Showing posts with label health care. Show all posts
Showing posts with label health care. Show all posts

Saturday, March 14, 2015

Disrupting Disruptive Physicians. By Bruce Gewertz


Viewpoint

Disrupting Disruptive Physicians

Bruce L Gewertz, MD
 
JAMA Surg. Published online March 11, 2015. doi:10.1001/jamasurg.2014.2911.


On Thursday mornings, our operating room management committee meets to handle items large and small. Most of our discussions focus on block-time allocation, purchasing decisions, and alike. However, too often we talk about behavioral issues, particularly the now well-characterized disruptive physician.

We have all seen it or been there before. A physician acts out in the operating room with shouting or biting sarcasm, intimidating colleagues and staff and impeding them from functioning at a high level. The most debilitating perpetrators of this behavior are repeat customers who engender such fear and uncertainty in all who contact them that the morale of the nursing staff and anesthesiologists is undermined, work becomes an unbearable chore, and performance suffers.

When one engages a difficult physician on his or her behavior, the physician responds in characteristic fashion. He or she defends his or her actions as patient advocacy, pointing out the shortcomings of the scrub nurse or instruments and showing limited, if any, remorse. He or she argues that such civil disobedience is the only way to enact change. In truth, disruptive physicians’ actions are often admired by a sizable minority of their colleagues as the only way to articulate real frustrations of working in today’s highly complex hospital. In extreme situations, these physicians become folk heroes to younger physicians who envy their fortitude in confronting the power of the bureaucracy.

A few days after a recent outburst by a particularly unpleasant and repeat offender, I was enjoying my daily interval on the stationary bicycle at my gym. My thoughts were wandering to a broad range of topics. I spent some time considering what really drives this nonproductive behavior and how otherwise valuable physicians could be channeled successfully into a more collegial state. As in the past, I was long on theory but short on conviction that it would make a difference.

After my workout as I prepared to shower, I received an urgent email. A patient I was consulting for upper extremity embolization had developed confusion and possible cerebral emboli despite full anticoagulation. I responded that I was on my way to see her and suggested a few diagnostic tests and consultations.

As I typed my message, a custodial employee of the gym reminded me that no cellular telephones were allowed in the locker room. I pointed out that I was not using my cellular telephone but rather an email function and I was not offending anyone by talking. He again pointed out that cellular telephones were not allowed under any circumstances. As I argued back, “I am a physician and this is an emergency.” My voice got louder and I became confrontational. I told him to call the manager. Another member next to me said quietly that the reason for the cellular telephone ban was the photographic potential of the devices and that I could have simply moved to the reception area and used the telephone any way I wished.

I felt like the fool I was. I trudged off to the showers feeling, as in the Texas homily, lower than a snake’s belly. After toweling off, I approached the employee and apologized for my behavior and for making his job more difficult. I told him he had handled the situation far better than me and I admired his restraint.

The lessons were stark and undeniable. Like my disruptive colleagues, I had justified my boorish behavior with patient care. I had assumed my need to break the rules far outweighed the reasonable and rational policy of the establishment; after all, I was important and people depended on me. Worse yet, I felt empowered to take out my frustration, enhanced by my worry about the patient, on someone unlikely to retaliate against me for fear of job loss.

I have come to realize that irrespective of disposition, when the setting is right, we are all potentially disruptive. The only questions are how frequent and how severe. Even more importantly, from a prognostic perspective, can we share the common drivers of these behaviors and develop insights that will lead to avoidance?

The most common approaches used today are only moderately effective. As in many other institutions, when physicians are deemed by their peers to have violated a carefully defined code of conduct, they are advised to apologize to any offended personnel. In many instances, these apologies are sincere and are, in fact, appreciated by all. Unfortunately, on occasion, the interaction is viewed as a forced function and the behavior is soon repeated albeit in a different nursing unit or operating room.

When such failures occur, persistently disruptive physicians are referred to our physician well-being committee. Through a highly confidential process, efforts are made to explore the potential causes for the behavior and acquaint the referred physician with the consequences of their actions on hospital function. Often, behavioral contracts are drawn up to precisely outline the individual’s issues and subsequent medical staff penalties if further violations occur.

That said, as well intentioned and psychologically sound as these programs are, there remains a hard core of repeat offenders. Despite the heightened stress and ill will engendered by disruptive physicians’ behavior, they simply cannot interact in other than confrontational fashion when frustrated by real or imagined shortcomings in the environment.

Based on nearly 20 years of physician management experience, it is my belief that in these few physicians, such behaviors are hard wired and fairly resistant to traditional counseling. An unfortunate end game is termination from a medical staff if the hostile working environment created by their outbursts is viewed as a liability threat by the institution. Such actions are always painful and bring no satisfaction to anyone involved. These high-stakes dramas, often involving critical institutional players on both sides, are played out behind closed doors. Few people are privy to the details of either the infraction or the attempts at remediation. Misunderstandings in the staff are common.

I suggest that an underused remedy is more intense peer pressure through continued education of those colleagues who might silently support these outbursts without fully realizing the consequences. This would begin by treating these incidents in the same way that we do other significant adverse events that occur in our hospitals. In confidential but interdisciplinary sessions, the genesis, nature, and consequences of the interaction could be explored openly. If indeed the inciting event was judged to be an important patient care issue, the problem could be identified and addressed yet clearly separated from the counterproductive interaction that followed. In addition to the deterrence provided by the more public airing of the incidents, the tenuous linkage between abusive behavior and patient protection could be severed. It is this linkage that provides any superficial legitimacy to the outbursts.

Through this process, peer pressure would be increased and provide a greater impetus for self-control and more productive interactions. Importantly, with such a direct and full examination of both the character and costs of poor conduct, whatever support exists for such behaviors within the medical staff would be diminished.
 
Bruce Gewertz, MD, Cedars-Sinai Health System Published Online: March 11, 2015. doi:10.1001/jamasurg.2014.2911.
Conflict of Interest Disclosures: None reported.

Saturday, November 15, 2014

Jonathan Gruber’s ‘Stupid’ Budget Tricks

Jonathan Gruber’s ‘Stupid’ Budget Tricks. WSJ Editorial
His ObamaCare candor shows how Congress routinely cons taxpayers.Wall Street Journal, Nov. 14, 2014 6:51 p.m. ET

As a rule, Americans don’t like to be called “stupid,” as Jonathan Gruber is discovering. Whatever his academic contempt for voters, the ObamaCare architect and Massachusetts Institute of Technology economist deserves the Presidential Medal of Freedom for his candor about the corruption of the federal budget process.

In his now-infamous talk at the University of Pennsylvania last year, Professor Gruber argued that the Affordable Care Act “would not have passed” had Democrats been honest about the income-redistribution policies embedded in its insurance regulations. But the more instructive moment is his admission that “this bill was written in a tortured way to make sure CBO did not score the mandate as taxes. If CBO scored the mandate as taxes, the bill dies.”

Mr. Gruber means the Congressional Budget Office, the institution responsible for putting “scores” or official price tags on legislation. He’s right that to pass ObamaCare Democrats perpetrated the rawest, most cynical abuse of the CBO since its creation in 1974.

In another clip from Mr. Gruber’s seemingly infinite video library, he discusses how he and Democrats wrote the law to game the CBO’s fiscal conventions and achieve goals that would otherwise be “politically impossible.” In still another, he explains that these ruses are “a sad statement about budget politics in the U.S., but there you have it.”

Yes you do. Such admissions aren’t revelations, since the truth has long been obvious to anyone curious enough to look. We and other critics wrote about ObamaCare’s budget gimmicks during the debate, and Rep. Paul Ryan exposed them at the 2010 “health summit.” President Obama changed the subject.

But rarely are liberal intellectuals as full frontal as Mr. Gruber about the accounting fraud ingrained in ObamaCare. Also notable are his do-what-you-gotta-do apologetics: “I’d rather have this law than not,” he says.

Recall five years ago. The White House wanted to pretend that the open-ended new entitlement would spend less than $1 trillion over 10 years and reduce the deficit too. Congress requires the budget gnomes to score bills as written, no matter how unrealistic the assumption or fake the promise. Democrats with the help of Mr. Gruber carefully designed the bill to exploit this built-in gullibility.

So they used a decade of taxes to fund merely six years of insurance subsidies. They made-believe that Medicare payments to hospitals will some day fall below Medicaid rates. A since-repealed program for long-term care front-loaded taxes but back-loaded spending, meant to gradually go broke by design. Remember the spectacle of Democrats waiting for the white smoke to come up from CBO and deliver the holy scripture verdict?

On the tape, Mr. Gruber also identifies a special liberal manipulation: CBO’s policy reversal to not count the individual mandate to buy insurance as an explicit component of the federal budget. In 1994, then CBO chief Robert Reischauer reasonably determined that if the government forces people to buy a product by law, then those transactions no longer belong to the private economy but to the U.S. balance sheet. The CBO’s face-melting cost estimate helped to kill HillaryCare.

The CBO director responsible for this switcheroo that moved much of ObamaCare’s real spending off the books was Peter Orszag, who went on to become Mr. Obama’s budget director. Mr. Orszag nonetheless assailed CBO during the debate for not giving him enough credit for the law’s phantom “savings.”

Then again, Mr. Gruber told a Holy Cross audience in 2010 that although ObamaCare “is 90% health insurance coverage and 10% about cost control, all you ever hear people talk about is cost control. How it’s going to lower the cost of health care, that’s all they talk about. Why? Because that’s what people want to hear about because a majority of Americans care about health-care costs.”

***

Both political parties for some reason treat the CBO with the same reverence the ancient Greeks reserved for the Delphic oracle, but Mr. Gruber’s honesty is another warning that the budget rules are rigged to expand government and hide the true cost of entitlements. CBO scores aren’t unambiguous facts but are guesses about the future, biased by the Keynesian assumptions and models its political masters in Congress instruct it to use.

Republicans who now run Congress can help taxpayers by appointing a new CBO director, as is their right as the majority. Current head Doug Elmendorf is a respected economist, and he often has a dry wit as he reminds Congressfolk that if they feed him garbage, he must give them garbage back. But if the GOP won’t abolish the institution, then they can find a replacement who is as candid as Mr. Gruber about the flaws and limitations of the CBO status quo. The Tax Foundation’s Steve Entin would be an inspired pick.

Democrats are now pretending they’ve never heard of Mr. Gruber, though they used to appeal to his authority when he still had some. His commentaries are no less valuable because he is now a political liability for Democrats.

Saturday, May 10, 2014

China moves to free-market pricing for pharmaceuticals, after price controls led to quality problems & shortages

China Scraps Price Caps on Low-Cost Drugs. By Laurie Burkitt
Move Comes After Some Manufacturers Cut Corners on Production
Wall Street Journal, May 8, 2014 1:15 a.m.
http://online.wsj.com/news/articles/SB10001424052702304655304579548933340544044

Beijing

China will scrap caps on retail prices for low-cost medicine and is moving toward free-market pricing for pharmaceuticals, after price controls led to drug quality problems and shortages in the country.

The move could be a welcome one for global pharmaceutical companies, which have been under scrutiny in China since last year for their sales and marketing practices.

The world's most populous country is the third-largest pharmaceutical market, behind the U.S. and Japan, according to data from consulting firm McKinsey & Co., but Beijing has used price caps and other measures to keep medical care affordable.

Price caps will be lifted for 280 medicines made by Western drug companies and 250 Chinese patent drugs, the National Development and Reform Commission, China's economic planning body, said Thursday. The move will affect prices on drugs such as antibiotics, painkillers and vitamins, it said.

The statement said local governments will have until July 1 to unveil details of the plan. In China, local authorities have broad oversight over how drugs are distributed to local hospitals.

Aiming to keep prices low, some manufacturers cut corners on production, exposing consumers to safety risks, said Helen Chen, a Shanghai-based partner and director of L.E.K. Consulting. Many also closed production, creating shortages of low-cost drugs such as thyroid medication.

"It means the [commission] recognizes that forcing prices down and focusing purely on price does sacrifice drug safety, quality and availability," said Ms. Chen.

Several drug makers, including GlaxoSmithKline PLC, didn't immediately respond to requests for comment. Spokeswomen for Sanofi and Pfizer Inc. said that because implementation of the new policy is unclear, it is too early to understand how it will affect their business in China.

The industry was dealt a blow last summer when Chinese authorities accused Glaxo of bribing doctors, hospitals and local officials to increase sales of their drugs. The U.K. company has said some of its employees may have violated Chinese law.

The central government, which began overhauling the country's health-care system in 2009, has until now largely favored pricing caps and has encouraged provincial governments to cut health-care costs and prices. Regulators phased out five years ago premium pricing for a list of "essential drugs" to be available in hospitals.

Chinese leaders want health care to be more accessible and affordable, but there have been unintended consequences in attempting to ensure the lowest prices on drugs. For instance, many pharmaceutical companies registered to sell the thyroid medication Tapazole have halted production in recent years after pricing restrictions squeezed out profits, experts say, creating a shortage. Chinese patients with hyperthyroidism struggled to find the drug and many suffered with increased anxiety, muscle weakness and sleep disorder, according to local media reports.

In 2012, some drug-capsule manufacturers were found to be using industrial gelatin to cut production costs. The industrial gelatin contained the chemical chromium, which can be carcinogenic with frequent exposure, according to the U.S. Centers for Disease Control and Prevention.

"Manufacturers have attempted to save costs, and doing that has meant using lower-quality ingredients," said Ms. Chen.

The pricing reversal won't necessarily alleviate pricing pressure for these drugs, experts say. To get drugs into hospitals, companies must compete in a tendering process at the provincial level, said Justin Wang, also a partner at L.E.K. "It's still unclear how the provinces will react to this new national list," Mr. Wang said.

If provinces don't change their current system, price will remain a key competitive factor for drug makers, said Franck Le Deu, a partner at McKinsey's China division.

"The bottom line is that there may be more safety and more pricing transparency, but the focus intensifies on creating more innovative drugs," Mr. Le Deu said.

  —Liyan Qi contributed to this article.

Saturday, January 25, 2014

Number of new antibacterial-drug approvals in the US


Source: Drug Makers Tiptoe Back Into Antibiotic R&D. By Hester Plumridge
As Superbugs Spread, Regulators Begin to Remove Roadblocks for New Treatments
WSJ, Jan 23, 2014
http://online.wsj.com/news/articles/SB10001424052702303465004579322601579895822

Saturday, December 28, 2013

MRSA Infections, swine effluent lagoons, and farm consolidations

Answering to some comments in a book review, 'In Meat We Trust,' by Maureen Ogle (http://online.wsj.com/news/articles/SB10001424052702303482504579177742158078278), WSJ, Dec. 17, 2013 6:36 p.m. ET:

A recent paper* in a FAO publication summarizes advances in hog manure management. Obviously, the cases mentioned are small in comparison with the great consolidated farms, but even so, there are multiple ways to manage better the effluents and some useful ways to profit from the lagoons/catchments are shown here.

@Mr Evangelista: I got access to the paper** you mentioned. If interested you may ask for it. I'd like, though, to calm down things. As it says other paper*** published at the same time, which it is likely it is the one Mr Blumenthal mentioned:
"In 2011,we estimated the overall number of invasive MRSA infections was 80 461; 31% lower than when estimates were first available in 2005"

The reasons are not well understood (several explanations are offered), but that is not relevant now. The important idea is that despite increasing consolidation of farm operations and an increasing population (from approx 295 million in 2005 to approx 311 million in 2011), there are 31% less MRSA infections.


References

* Intensive and Integrated Farm Systems using Fermentation of Swine Effluent in Brazil. By I. Bergier, E. Soriano, G. Wiedman and A. Kososki. In Biotechnologies at Work for Smallholders: Case Studies from Developing Countries in Crops, Livestock and Fish. Edited by J. Ruane, J.D. Dargie, C. Mba, P. Boettcher, H.P.S. Makkar, D.M. Bartley and A. Sonnino. Food and Agriculture Organization of the United Nations, 2013. http://www.fao.org/docrep/018/i3403e/i3403e00.htm

** High-Density Livestock Operations, Crop Field Application of Manure, and Risk of Community-Associated Methicillin-Resistant Staphylococcus aureus Infection in Pennsylvania. By Joan A. Casey, MA; Frank C. Curriero, PhD, MA; Sara E. Cosgrove,MD, MS; Keeve E. Nachman, PhD, MHS; Brian S. Schwartz, MD,MS. JAMA Intern Med. Vol 173, No. 21, doi:10.1001/jamainternmed.2013.10408

*** National Burden of InvasiveMethicillin-Resistant Staphylococcus aureus Infections, United States, 2011. By Raymund Dantes, MD, MPH; Yi Mu, PhD; Ruth Belflower, RN, MPH; Deborah Aragon, MSPH; Ghinwa Dumyati, MD; Lee H. Harrison, MD; Fernanda C. Lessa, MD; Ruth Lynfield, MD; Joelle Nadle, MPH; Susan Petit, MPH; Susan M. Ray, MD; William Schaffner, MD; John Townes, MD; Scott Fridkin, MD; for the Emerging Infections Program–Active Bacterial Core Surveillance MRSA Surveillance Investigators. JAMA Intern Med. Vol 173, No. 21, doi:10.1001/jamainternmed.2013.10423

Friday, December 6, 2013

New meat regulations could spark a trade war with Canada and Mexico and will raise costs

This Label Will Raise the Cost of Your Steak. By Scott George and Randy Spronk
New meat regulations could spark a trade war with Canada and Mexico.
Wall Street Journal, Dec. 5, 2013 6:42 p.m. ET
http://online.wsj.com/news/articles/SB10001424052702303670804579234642364948248

Right before Thanksgiving, while Congress was on break, federal meat labeling regulations took effect that could result in Americans paying higher prices on everything from beef and pork to apples and maple syrup. While legislators, as part of the continuing farm bill negotiations, are considering a fix to the Country of Origin Labeling (Cool) statute, the regulations implementing it went into effect Nov. 23.

The new Cool rules require more detailed labels on meat derived from animals born outside the United States. Labels must now list the country in which livestock were born, raised and slaughtered. For example, a package of rib-eye steak might be labeled: "Born in Canada, Raised and Slaughtered in the United States."

The previous Cool rules required less detailed labeling, such as "Product of Canada and the United States." Ironically, the U.S. Department of Agriculture issued the new rules in May in an effort to improve the previous Cool rules, which the World Trade Organization last year ruled discriminated against Canada, Mexico and other U.S. trading partners.

Not surprisingly, Canada and Mexico are also fighting the new, more stringent rules at the WTO. Should the trade organization rule in their favor, our North American neighbors will likely retaliate against U.S. products through tariffs that will limit U.S. exports and kill American jobs. Canada, the second-largest export market for U.S. agricultural products, valued in 2012 at $20.6 billion, already has a preliminary retaliation list that includes fresh pork and beef, bakery goods, rice, apples, wine, maple syrup and furniture.

U.S. cattle ranchers and hog farmers who purchase livestock from Canada or Mexico will be affected by those retaliatory tariffs in a number of ways. Most crucially to those of us in the industry, the duties will prompt U.S. beef and pork exports to fall while American farmers and ranchers who import animals will see significant cost increases.

Alpha 3 Cattle Company in Amarillo, Texas, for example, imports roughly 38,000 feeder cattle a year from Mexico. When the original Cool law took effect in 2009, meat packers, fearing consumers would be less inclined to buy meat labeled "Product of Mexico and the United States" and incurring added costs to label mixed-origin meat, discounted Alpha 3's Mexican-origin animals by $35 a head. That alone cost Alpha 3 more than $1 million.

Under the new Cool regulations, the company expects the discount to be even higher, or for packing plants to stop processing Mexican-born cattle altogether. Why? Because under the new regulations those animals—and the meat from them—now need to be tracked, verified and segregated from U.S.-born cattle. (The 2009 law allowed co-mingling of animals.)

A Michigan hog farmer who gets most of his feeder pigs from Canada, and who took a financial hit when the labeling law took effect in 2009, has been told by the packing plant to which he sends his animals that he'll have a 10-hour window each week to get his Canadian-born hogs to market. That will be nearly impossible to accomplish—it's 32 truckloads—and it will be extremely costly.

That's because the new regulations will force the packing plant to shut down the lines processing U.S.-born hogs and switch to processing Canadian-born ones—which spend five of their six months in the U.S.—so that pork cuts can be tracked, labeled and kept separate. That's a logistical headache and a huge expense for the plant, which will likely pay the hog farmer less for his Canadian-born hogs and charge consumers more for the meat from those animals.

So why is the U.S. risking trade retaliation and prohibitive cost increases on American producers and consumers of meat? Groups that support Cool, such as the U.S. Cattlemen's Association and the Consumer Federation of America, think U.S. consumers will buy American if they see a "Product of the United States" label. But since the 2009 law went into effect, the USDA says there's been little effect on demand for U.S. meat, and that consumers buy primarily based on taste and price. Most Americans know, even if their legislators don't, that all meat products, regardless of their country of origin, must pass the same USDA safety regulations.

When the Cool proposal was first debated in Congress, the U.S. meat industry said it would be a costly program with little if any benefit to consumers. The USDA estimated it would cost $2.5 billion to implement and nearly $212 million annually over 10 years to maintain.

With our North American neighbors set to impose tariffs on dozens of U.S. products, livestock producers and meat packers facing greater costs and American consumers ultimately bearing higher prices, it appears that assessment was an understatement.

Mr. George is a cattleman from Cody, Wyo., and president of the National Cattlemen's Beef Association. Mr. Spronk is a hog farmer from Edgerton, Minn., and president of the National Pork Producers Council.

Wednesday, November 27, 2013

Alzheimer's Disease - The Puzzles, The Partners, The Path Forward

Alzheimer's Disease - The Puzzles, The Partners, The Path Forward
PhRMA, November 26, 2013
http://www.innovation.org/index.cfm/NewsCenter/Newsletters?NID=218

Alzheimer's is a debilitating neurodegenerative disease that currently afflicts more than 5 million people in the U.S. If no new medicines are found to prevent, delay or stop the progression of Alzheimer's disease, the number of affected people in America will jump to 15 million by 2050 and related healthcare costs could increase five-fold to $1.2 trillion, according to the Alzheimer's Association. In contrast, a medicine that delays onset of Alzheimer's disease by five years would lower the number of Americans suffering from the disease by nearly half and save $447 billion in related costs, in 2050.

America's biopharmaceutical companies are currently developing 73 potential new treatments and diagnostics for Alzheimer's, according to a recent report released by PhRMA. At a recent all day-forum, "Alzheimer's: The Puzzle, The Partners, The Path Forward," the Alzheimer's Association, Alzheimer's Drug Discovery Foundation, and PhRMA convened key stakeholders from the Alzheimer's community to discuss these therapies presently in development to treat the disease, as well as the current state of innovation and R&D for Alzheimer's disease treatments and diagnostics.

Among the key areas of discussion were pre-competitive partnerships, including potential areas for collaboration and public-private partnerships, as well as pre-symptomatic clinical trials, which may help researchers understand the clinical heterogeneity of the disease and subsequent challenges in the use and adoption of clinical and functional endpoints in new clinical trial design.

Panelists included top industry and academic scientists, policymakers, patients, payers, and many others. Executives from the Alzheimer's Association and Alzheimer's Drug Discovery Foundation also discussed the path forward for Alzheimer's disease in relation to science and policy.

Continue the conversation online using the event hashtag #ALZpov

Saturday, August 3, 2013

Nearly 450 Innovative Medicines in Development for Neurological Disorders

Neurological Disorders
innovation.org
July 30, 2013
www.innovation.org/index.cfm/FutureofInnovation/NewMedicinesinDevelopment/Neurological_Disorders


Nearly 450 Innovative Medicines in Development for Neurological Disorders

Neurological disorders—such as epilepsy, multiple sclerosis, Alzheimer’s disease, and Parkinson’s disease—inflict great pain and suffering on patients and their families, and every year cost the U.S. economy billions of dollars. However, a growing understanding of how neurological disorders work at a genetic and molecular level has spurred improvements in treatment for many of these diseases.

America’s biopharmaceutical research companies are developing 444 medicines to prevent and treat neurological disorders, according to a new report released by the Pharmaceutical Research and Manufacturers of America (PhRMA). 

The report demonstrates the wide range of medicines in development for the more than 600 neurological disorders that affect millions of Americans each year. These medicines are all currently in clinical trials or awaiting Food & Drug Administration (FDA) review. They include 82 for Alzheimer’s disease, 82 for pain, 62 for brain tumors, 38 for multiple sclerosis, 28 for epilepsy and seizures, 27 for Parkinson’s disease, and 25 for headache.

Many of the potential medicines use cutting-edge technologies and new scientific approaches. For example:

  • A medicine that prompts the immune system to protect neurons affected by amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease
  • A gene therapy for the treatment of Alzheimer’s disease
  • A gene therapy to reverse the effects of Parkinson’s disease
These new medicines promise to continue the already remarkable progress against neurological disorders and to raise the quality of life for patients suffering from these diseases and their families. Read more about selected medicines in development for neurological disorders.

Alzheimer's Disease

Every 68 seconds someone in America develops Alzheimer’s disease, according to the Alzheimer’s Association, and by 2050 it could be every 33 seconds, or nearly a million new cases per year. Disease-modifying treatments currently in development could delay the onset of the disease by five years, and result in 50 percent fewer patients by 2050.

There are also potential cost savings offered by innovative disease-modifying treatments. As the 6th leading cause of death in the United States and one of the most common neurological disorders, Alzheimer’s disease currently costs society approximately $203 billion. This number could increase to $1.2 trillion by 2050; however, delaying the onset of the disease by five years could reduce the cost of care of Alzheimer’s patients in 2050 by nearly $450 billion.

Additional Resources

Friday, May 31, 2013

241 Medicines in Development for Leukemia, Lymphoma and Other Blood Cancers

241 Medicines in Development for Leukemia, Lymphoma and Other Blood Cancers
PhRMA, May 2013
www.innovation.org/index.cfm/FutureofInnovation/NewMedicinesinDevelopment/Leukemia_and_Lymphoma

Biopharmaceutical research companies are developing 241 medicines for blood cancers—leukemia, lymphoma and myeloma. This report lists medicines in human clinical trials or under review by the U.S. Food and Drug Administration (FDA).

The medicines in development include:

• 98 for lymphoma, including Hodgkin and non-Hodgkin lymphoma, which affect nearly 80,000 Americans each year.
• 97 for leukemia, including the four major types, which affect nearly 50,000 people in the United States each year.
• 52 for myeloma, a cancer of the plasma cells, which impacts more than 22,000 people each year in the United States.
• 24 medicines are targeting hematological malignancies, which affect bone marrow, blood and lymph nodes.
• 15 each for myeloproliferative neoplasms, such as myelofibrosis, polycythemia vera and essential thrombocythemia; and for myelodysplastic syndromes, which are diseases affecting the blood and bone marrow.

These medicines in development offer hope for greater survival for the thousands of Americans who are affected by these cancers of the blood.

Definitions for the cancers listed in this report and other terms can be found on page 27. Links to sponsor company web sites provide more information on the potential products. See full report: http://t.co/JSbXhBVG7t

Sunday, April 28, 2013

"What a civilised society, I thought to myself"

From the speech by Lee Kuan Yew at the Imperial College Commemoration Eve Dinner, Oct 22, 2002 (http://www3.imperial.ac.uk/newsandeventspggrp/imperialcollege/alumni/pastukevents/newssummary/news_26-2-2007-14-0-12):

Looking back at those early years, I am amazed at my youthful innocence. I watched Britain at the beginning of its experiment with the welfare state; the Atlee government started to build a society that attempted to look after its citizens from cradle to grave. I was so impressed after the introduction of the National Health Service when I went to collect my pair of new glasses from my opticians in Cambridge to be told that no payment was due. All I had to do was to sign a form. What a civilised society, I thought to myself. The same thing happened at the dentist and the doctor.

I did not understand what a cosseted life would do to the spirit of enterprise of a pe ople, diminishing their desire to achieve and succeed. I believed that wealth came naturally from wheat growing in the fields, orchards bearing fruit every summer, and factories turning out all that was needed to maintain a comfortable life.

Only two decades later when I had to make an outdated entrepot economy feed a people did I realise we needed to create the wealth before we can share it. And to create wealth, high motivation and incentives are crucial to drive a people to achieve, to take risks for profit or there will be nothing to share.

It is remarkable that powerful minds like Sir William Beveridge's, who thought out this egalitarian welfare system, did not foresee its unintended consequences. It took more than three decades of gradual decline in performance before Margaret Thatcher set out to reverse it, to restore individual incentives and the motivation to succeed, to encourage risk-taking, necessary for a successful entrepreneurial economy.

h/t: Haseltine, William A. Affordable Excellence: The Singapore Health System. Brookings Institution Press with the National University of Singapore Press, Apr 2013


The most interesting this, to me is that this once was the norm:
Perhaps the most impressive sight I came upon was when I emerged from the tube station at Piccadilly Circus. I found a little table with a pile of newspapers and a box of coins and notes with nobody in attendance. You take your newspaper, toss in your coin or put in your 10-shilling note and take your change. I took a deep breath - this was a truly civilised people.

But, as he added:
Five decades ago, London was a grimy, sooty, bomb-scarred city, with less food, fewer cars, and deprived of the conveniences of the consumer society. But the people, then homogeneous, white, and Christians, were admirable, self-confident and courteous.

From that well-mannered Britain to the yobs and football hooligans of the 1990s took only 40 years. I learned that civilised living does not come about naturally.

Friday, March 1, 2013

Robust Biopharmaceutical Pipeline Offers New Hope for Patients

Robust Biopharmaceutical Pipeline Offers New Hope for Patients
innovation.org 
January 31, 2013 
http://www.innovation.org/index.cfm/NewsCenter/Newsletters?NID=209

According to a new report by the Analysis Group, the biopharmaceutical pipeline is innovative and robust, with a high proportion of potential first-in-class medicines and therapies targeting diseases with limited treatment options. The report, “Innovation in the Biopharmaceutical Pipeline: A Multidimensional View,” uses several different measures to look at innovation in the pipeline.

The report reveals that more than 5,000 new medicines are in the pipeline globally. Of these medicines in various phases of clinical development, 70 percent are potential first-in-class medicines, which means that they have a different mechanism of action than any other existing medicine. Subsequent medicines in a class offer different profiles and benefits for patients but first-in-class medicines also provide exciting new approaches to treating disease for patients. Potential first-in-class medicines make up as much as 80% of the pipeline for disease areas such as cancer and neurology.

Many of the new medicines in the pipeline are also for diseases for which no new therapies have been approved in the last decade and significant treatment gaps exist.  For example, there are 158 potential medicines for ovarian cancer, 19 for sickle cell disease 61 for amyotrophic lateral sclerosis, and 41 for small cell lung cancer.

The authors also found that personalized medicines account for an increasing proportion of the pipeline, and the number of potential new medicines for rare diseases designated by the FDA each year averaged 140 per year in the last 10 years compared to 64 in the previous decade.

The record 39 new drugs approved by the FDA in 2012 – a 16 year high – and the robust pipeline of drugs in development reflect the continuing commitment of the biomedical research community, including industry, academia, government researchers, patient groups, and others to develop novel treatments that will advance our understanding of disease and improve patient outcomes.

New medicines have brought tremendous value to the U.S. health care system and the economy more broadly. But more progress is needed to address the most costly and challenging diseases facing patients in America and across the globe. As our population ages, the need will only grow. Researchers are working to deliver on the promise of unprecedented scientific advances. 

Saturday, December 22, 2012

Novel Drug Approvals Strong in 2012

Novel Drug Approvals Strong in 2012
Dec 21, 2012
http://www.innovation.org/index.cfm/NewsCenter/Newsletters?NID=208

Over the past year, biopharmaceutical researchers' work has continued to yield innovative treatments to improve the lives of patients. In fiscal year (FY) 2012 (October 1, 2011 – September 30, 2012), the U.S. Food and Drug Administration (FDA) approved 35 new medicines, keeping pace with the previous fiscal year’s approvals and representing one of the highest levels of FDA approvals in recent years.[i] For the calendar year FDA is on track to approve more new medicines than any year since 2004.[ii]

A recent report from the FDA highlights the groundbreaking medicines to treat diseases ranging from the very common to the most rare. Some are the first treatment option available for a condition, others improve care for treatable diseases.

Notable approvals in FY 2012 include:
  • A breakthrough personalized medicine for a rare form of cystic fibrosis;
  • The first approved human cord blood product;
  • A total of ten drugs to treat cancer, including the first treatments for advanced basal cell carcinoma and myelofibrosis and a targeted therapy for HER2-positive metastatic breast cancer;
  • Nine treatments for rare diseases; and
  • Important new therapies for HIV, macular degeneration, and meningitis.
The number of new drugs approved this year reflects the continuing commitment of the biomedical research community – from biopharmaceutical companies to academia to government researchers to patient groups – to advance basic science and translate that knowledge into novel treatments that will advance our understanding of disease and improve patient outcomes.

Building on these noteworthy approvals, we look to the new year where continued innovation is needed to leverage our growing understanding of the underpinnings of human disease and to harness the power of scientific research tools to discover and develop new medicines.

To learn more about the more than 3,200 new medicines in development visit http://www.innovation.org/index.cfm/FutureOfInnovation/NewMedicinesinDevelopment.

Thursday, September 13, 2012

The Rough Road to Progress Against Alzheimer's Disease

The Rough Road to Progress Against Alzheimer's Disease
 PhRMA
Sep 13, 2012
http://www.innovation.org/index.cfm/NewsCenter/Newsletters?NID=205


Two high-profile Alzheimer’s drug development failures were announced in recent weeks shining a spotlight on the challenges and frustrations inherent in Alzheimer’s research. Alzheimer’s disease is among the most devastating and costly illnesses we face and the need for new treatments will only become more acute as our population ages.

Understanding a disease and developing medicines to treat it is always a herculean task but Alzheimer’s brings particular challenges and long odds. A new report from the Pharmaceutical Research and Manufacturers of America (PhRMA), "Researching Alzheimer’s Medicines: Setbacks and Stepping Stones", examines the complexities of researching and treating Alzheimer’s and drug development success rates in recent years.

Since 1998, there have been 101 unsuccessful attempts to develop drugs to treat Alzheimer’s—or as some call them “failures,” according to the new analysis. In that time three new medicines have been approved to treat the symptoms of Alzheimer’s disease; however, for every research project that succeeded, 34 failed to yield a new medicine.

These “failures” may appear to be dead ends – a waste of time and resources – but to researchers they are both an inevitable and necessary part of making progress. These setbacks often contribute to eventual success by helping guide and redirect research on potential new drugs. In fact, the recent unsuccessful trials have provided a wealth of new information which researchers are now sifting through to inform their ongoing research.

Alzheimer’s disease is the sixth leading cause of death in the United States today, with 5.4 million people currently affected.[i]  By 2050, the number of Americans with the disease is projected to reach 13.5 million at a cost of over $1.1 trillion unless new treatments to prevent, arrest or cure the disease are found.[ii]  According to the Alzheimer’s Association a new medicine that delays the onset of the disease could change that trajectory and save $447 billion a year by 2050.

According to another new report, researchers are currently working on nearly 100 medicines in development for Alzheimer’s and other dementias. Although research is not a straight, predictable path, with continued dedication, we will make a difference for every person at risk of suffering from this terrible, debilitating disease.



[i]Alzheimer's Association, “Factsheet,” (March 2012), http://www.alz.org/documents_custom/2012_facts_figures_fact_sheet.pdf 
[ii]Alzheimer's Association, 2012 Alzheimer's Disease Facts and Figures, Alzheimer's and Dementia, Volume 8, Issue 2

Tuesday, July 10, 2012

Quality of Government and Living Standards: Adjusting for the Efficiency of Public Spending

Quality of Government and Living Standards: Adjusting for the Efficiency of Public Spending. By Grigoli, Francesco; Ley, Eduardo
IMF Working Paper No. 12/182
Jul 2012
http://www.imf.org/external/pubs/cat/longres.aspx?sk=26052.0

Summary: It is generally acknowledged that the government’s output is difficult to define and its value is hard to measure. The practical solution, adopted by national accounts systems, is to equate output to input costs. However, several studies estimate significant inefficiencies in government activities (i.e., same output could be achieved with less inputs), implying that inputs are not a good approximation for outputs. If taken seriously, the next logical step is to purge from GDP the fraction of government inputs that is wasted. As differences in the quality of the public sector have a direct impact on citizens’ effective consumption of public and private goods and services, we must take them into account when computing a measure of living standards. We illustrate such a correction computing corrected per capita GDPs on the basis of two studies that estimate efficiency scores for several dimensions of government activities. We show that the correction could be significant, and rankings of living standards could be re-ordered as a result.

Excerpts:

Despite its acknowledged shortcomings, GDP per capita is still the most commonly used summary indicator of living standards. Much of the policy advice provided by international organizations is based on macroeconomic magnitudes as shares of GDP, and framed on cross-country comparisons of per capita GDP. However, what GDP does actually measure may differ significantly across countries for several reasons. We focus here on a particular source for this heterogeneity: the quality of public spending. Broadly speaking, the ‘quality of public spending’ refers to the government’s effectiveness in transforming resources into socially valuable outputs. The opening quote highlights the disconnect between spending and value when the discipline of market transactions is missing.

Everywhere around the world, non-market government accounts for a big share of GDP and yet it is poorly measured—namely the value to users is assumed to equal the producer’s cost.  Such a framework is deficient because it does not allow for changes in the amount of output produced per unit of input, that is, changes in productivity (for a recent review of this issue, see Atkinson and others, 2005). It also assumes that these inputs are fully used. To put it another way, standard national accounting assumes that government activities are on the best practice frontier. When this is not the case, there is an overstatement of national production.  This, in turn, could result in misleading conclusions, particularly in cross-country comparisons, given that the size, scope, and performance of public sectors vary so widely.

Moreover, in the national accounts, this attributed non-market (government and non-profit sectors) “value added” is further allocated to the household sector as “actual consumption.” As Deaton and Heston (2008) put it: “[...] there are many countries around the world where government-provided health and education is inefficient, sometimes involving mass absenteeism by teachers and health workers [...] so that such ‘actual’ consumption is anything but actual. To count the salaries of AWOL government employees as ‘actual’ benefits to consumers adds statistical insult to original injury.” This “statistical insult” logically follows from the United Nations System of National Accounts (SNA) framework once ‘waste’ is classified as income—since national income must be either consumed or saved. Absent teachers and health care workers are all too common in many low-income countries (Chaudhury and Hammer, 2004; Kremer and others, 2005; Chaudhury and others, 2006; and World Bank, 2004). Beyond straight absenteeism, which is an extreme case, generally there are significant cross-country differences in the quality of public sector services. World Bank (2011) reports that in India, even though most children of primaryschool age are enrolled in school, 35 percent of them cannot read a simple paragraph and 41 percent cannot do a simple subtraction.

It must be acknowledged, nonetheless, that for many of government’s non-market services, the output is difficult to define, and without market prices the value of output is hard to measure. It is because of this that the practical solution adopted in the SNA is to equate output to input costs. This choice may be more adequate when using GDP to measure economic activity or factor employment than when using GDP to measure living standards.

Moving beyond this state of affairs, there are two alternative approaches. One is to try to find indicators for both output quantities and prices for direct measurement of some public outputs, as recommended in SNA 93 (but yet to be broadly implemented). The other is to correct the input costs to account for productive inefficiency, namely to purge from GDP the fraction of these inputs that is wasted. We focus here on the nature of this correction. As the differences in the quality of the public sector have a direct impact on citizens’ effective consumption of public and private goods and services, it seems natural to take them into account when computing a measure of living standards.

To illustrate, in a recent study, Afonso and others (2010) compute public sector efficiency scores for a group of countries and conclude that “[...] the highest-ranking country uses onethird of the inputs as the bottom ranking one to attain a certain public sector performance score. The average input scores suggest that countries could use around 45 per cent less resources to attain the same outcomes if they were fully efficient.” In this paper, we take such a statement to its logical conclusion. Once we acknowledge that the same output could be achieved with less inputs, output value cannot be equated to input costs. In other words, waste should not belong in the living-standards indicator—it still remains a cost of government but it must be purged from the value of government services. As noted, this adjustment is especially relevant for cross-country comparisons.

...

In this context, as noted, the standard practice is to equate the value of government outputs to its cost, notwithstanding the SNA 93 proposal to estimate government outputs directly. The value added that, say, public education contributes to GDP is based on the wage bill and other costs of providing education, such as outlays for utilities and school supplies. Similarly for public health, the wage bill of doctors, nurses and other medical staff and medical supplies measures largely comprises its value added. Thus, in the (pre-93) SNA used almost everywhere, non-market output, by definition, equals total costs. Yet the same costs support widely different levels of public output, depending on the quality of the public sector.

Note that value added is defined as payments to factors (labor and capital) and profits. Profits are assumed to be zero in the non-commercial public sector. As for the return to capital, in the current SNA used by most countries, public capital is attributed a net return of zero—i.e., the return from public capital is equated to its depreciation rate. This lack of a net return measure in the SNA is not due to a belief that the net return is actually zero, but to the difficulties of estimating the return.

Atkinson and others (2005, page 12) state some of the reasons behind current SNA practice: “Wide use of the convention that (output = input) reflects the difficulties in making alternative estimates. Simply stated, there are two major problems: (a) in the case of collective services such as defense or public administration, it is hard to identify the exact nature of the output, and (b) in the case of services supplied to individuals, such as health or education, it is hard to place a value on these services, as there is no market transaction.”

Murray (2010) also observes that studies of the government’s production activities, and their implications for the measurement of living standards, have long been ignored. He writes: “Looking back it is depressing that progress in understanding the production of public services has been so slow. In the market sector there is a long tradition of studying production functions, demand for inputs, average and marginal cost functions, elasticities of supply, productivity, and technical progress. The non-market sector has gone largely
unnoticed. In part this can be explained by general difficulties in measuring the output of services, whether public or private. But in part it must be explained by a completely different perspective on public and private services. Resource use for the production of public services has not been regarded as inputs into a production process, but as an end in itself, in the form of public consumption. Consequently, the production activity in the government sector has not been recognized.” (Our italics.)

The simple point that we make in this paper is that once it is recognized that the effectiveness of the government’s ‘production function’ varies significantly across countries, the simple convention of equating output value to input cost must be revisited. Thus, if we learn that the same output could be achieved with less inputs, it is more appropriate to credit GDP or GNI with the required inputs rather than with the actual inputs that include waste. While perceptions of government effectiveness vary widely among countries as, e.g., the World Bank’s Governance indicators attests (Kaufmann and others 2009), getting reliable measures of government actual effectiveness is a challenging task as we shall discuss below.

In physics, efficiency is defined as the ratio of useful work done to total energy expended, and the same general idea is associated with the term when discussing production. Economists simply replace ‘useful work’ by ‘outputs’ and ‘energy’ by ‘inputs.’ Technical efficiency means the adequate use of the available resources in order to obtain the maximum product. Why focus on technical efficiency and not other concepts of efficiency, such as price or allocative efficiency? Do we have enough evidence on public sector inefficiency to make the appropriate corrections?

The reason why we focus on technical efficiency in this preliminary inquiry is twofold. First, it corresponds to the concept of waste. Productive inefficiency implies that some inputs are wasted as more could have been produced with available inputs. In the case of allocative inefficiency, there could be a different allocation of resources that would make everyone better off but we cannot say that necessarily some resources are unused—although they are certainly not aligned with social preferences. Second, measuring technical inefficiency is easier and less controversial than measuring allocative inefficiency. To measure technical inefficiency, there are parametric and non-parametric methods allowing for construction of a best practice frontier. Inefficiency is then measured by the distance between this frontier and the actual input-output combination being assessed.

Indicators (or rather ranges of indicators) of inefficiency exist for the overall public sector and for specific activities such as education, healthcare, transportation, and other sectors. However, they are far from being uncontroversial. Sources of controversy include: omission of inputs and/or outputs, temporal lags needed to observe variations in the output indicators, choice of measures of outputs, and mixing outputs with outcomes. For example, many social and macroeconomic indicators impact health status beyond government spending (Spinks and Hollingsworth, 2009, and Joumard and others, 2010) and they should be taken into account. Most of the output indicators available show autocorrelation and changes in inputs typically take time to materialize into outputs’ variations. Also, there is a trend towards using outcome rather than output indicators for measuring the performance of the public sector. In health and education, efficiency studies have moved away from outputs (e.g., number of prenatal interventions) to outcomes (e.g., infant mortality rates). When cross-country analyses are involved, however, it must be acknowledged that differences in outcomes are explained not only by differences in public sector outputs but also differences in other environmental factors outside the public sector (e.g., culture, nutrition habits).

Empirical efficiency measurement methods first construct a reference technology based on observed input-output combinations, using econometric or linear programming methods. Next, they assess the distance of actual input-output combinations from the best-practice frontier. These distances, properly scaled, are called efficiency measures or scores. An inputbased efficiency measure informs us on the extent it is possible to reduce the amount of the inputs without reducing the level of output. Thus, an efficiency score, say, of 0.8 means that using best practices observed elsewhere, 80 percent of the inputs would suffice to produce the same output.

We base our corrections to GDP on the efficiency scores estimated in two papers: Afonso and others (2010) for several indicators referred to a set of 24 countries, and Evans and others (2000) focusing on health, for 191 countries based on WHO data. These studies employ techniques similar to those used in other studies, such as Gupta and Verhoeven (2001), Clements (2002), Carcillo and others (2007), and Joumard and others (2010).

? Afonso and others (2010) compute public sector performance and efficiency indicators (as performance weighted by the relevant expenditure needed to achieve it) for 24 EU and emerging economies. Using DEA, they conclude that on average countries could use 45 percent less resources to attain the same outcomes, and deliver an additional third of the fully efficient output if they were on the efficiency frontier. The study included an analysis of the efficiency of education and health spending that we use here.

? Evans and others (2000) estimate health efficiency scores for the 1993–1997 period for 191 countries, based on WHO data, using stochastic frontier methods. Two health outcomes measures are identified: the disability adjusted life expectancy (DALE) and a composite index of DALE, dispersion of child survival rate, responsiveness of the health care system, inequities in responsiveness, and fairness of financial contribution. The input measures are health expenditure and years of schooling with the addition of country fixed effects. Because of its large country coverage, this study is useful for illustrating the impact of the type of correction that we are discussing
here.

We must note that ideally, we would like to base our corrections on input-based technical efficiency studies that deal exclusively with inputs and outputs, and do not bring outcomes into the analysis. The reason is that public sector outputs interact with other factors to produce outcomes, and here cross-country hetereogenity can play an important role driving cross-country differences in outcomes. Unfortunately, we have found no technical-efficiency studies covering a broad sample of countries that restrict themselves to input-output analysis.  In particular, these two studies deal with a mix of outputs and outcomes. The results reported here should thus be seen as illustrative. Furthermore, it should be underscored that the level of “waste” that is identified for each particular country varies significantly across studies, which implies that any associated measures of GDP adjusting for this waste will also differ.

...

We have argued here that the current practice of estimating the value of the government’s non-market output by its input costs is not only unsatisfactory but also misleading in crosscountry comparisons of living standards. Since differences in the quality of the public sector have an impact on the population’s effective consumption and welfare, they must be taken into account in comparisons of living standards. We have performed illustrative corrections of the input costs to account for productive inefficiency, thus purging from GDP the fraction of these inputs that is wasted.

Our results suggest that the magnitude of the correction could be significant. When correcting for inefficiencies in the health and education sectors, the average loss for a set of 24 EU member states and emerging economies amounts to 4.1 percentage points of GDP.  Sector-specific averages for education and health are 1.5 and 2.6 percentage points of GDP, implying that 32.6 and 65.0 percent of the inputs are wasted in the respective sectors. These corrections are reflected in the GDP-per-capita ranking, which gets reshuffled in 9 cases out of 24. In a hypothetical scenario where the inefficiency of the health sector is assumed to be representative of the public sector as a whole, the rank reordering would affect about 50 percent of the 93 countries in the sample, with 70 percent of it happening in the lower half of the original ranking. These results, however, should be interpreted with caution, as the purpose of this paper is to call attention to the issue, rather than to provide fine-tuned waste estimates.

A natural way forward involves finding indicators for both output quantities and prices for direct measurement of some public outputs. This is recommended in SNA 93 but has yet to be implemented in most countries. Moreover, in recent times there has been an increased interest in outcomes-based performance monitoring and evaluation of government activities (see Stiglitz and others, 2010). As argued also in Atkinson (2005), it will be important to measure not only public sector outputs but also outcomes, as the latter are what ultimately affect welfare. A step in this direction is suggested by Abraham and Mackie (2006) for the US, with the creation of “satellite” accounts in specific areas as education and health. These extend the accounting of the nation’s productive inputs and outputs, thereby taking into account specific aspects of non-market activities.

Wednesday, November 30, 2011

Over 900 Biotechnology Medicines in Development, Targeting More than 100 Diseases

Over 900 Biotechnology Medicines in Development, Targeting More than 100 Diseases
September 14, 2011
http://www.innovation.org/index.cfm/FutureOfInnovation/NewMedicinesinDevelopment/Biotechnology_Medicines

Biotechnology has opened the door to the discovery and development of new types of human therapeutics. Advancements in both cellular and molecular biology have allowed scientists to identify and develop a host of new products. These cutting-edge medicines provide significant clinical benefits, and in many cases, address therapeutic categories where no effective treatment previously existed.

Innovative, targeted therapies offer enormous potential to address unmet medical needs of patients with cancer, HIV/AIDS, and many other serious diseases. These medicines also hold the potential to help us meet the challenge of rising healthcare costs by avoiding treatment complications and making sure each patient gets the most effective care possible.

Approved biotechnology medicines already treat or help prevent heart attacks, stroke, multiple sclerosis, leukemia, hepatitis, congestive heart failure, lymphoma, kidney cancer, cystic fibrosis, and other diseases. These medicines use many different approaches to treat disease as do medicines currently in the pipeline.

America's biopharmaceutical research companies have 901 biotechnology medicines and vaccines in development to target more than 100 debilitating and life- threatening diseases, such as cancer, arthritis and diabetes, according to a new report [http://www.phrma.org/sites/default/files/1776/biotech2011.pdf] by the Pharmaceutical Research and Manufacturers of America (PhRMA). The medicines in development—all in either clinical trials or under Food and Drug Administration review—include 353 for cancer and related conditions, 187 for infectious diseases, 69 for autoimmune diseases and 59 for cardiovascular diseases.

The biotechnology medicines now in development make use of these and other state-of- the-art approaches. For example:

•A genetically-modified virus-based vaccine to treat melanoma.
•A monoclonal antibody for the treatment of cancer and asthma.
•An antisense medicine for the treatment of cancer.
•A recombinant fusion protein to treat age-related macular degeneration.


SELECTED BIOTECHNOLOGY MEDICINES IN DEVELOPMENT

Autoimmune Diseases: Autoimmunity is the underlying cause of more than 100 serious, chronic illnesses, targeting women 75 percent of the time. Autoimmune diseases have been cited in the top 10 leading causes of all deaths among U.S. women age 65 and younger, representing the fourth largest cause of disability among women in the United States.


Blood Disorders: Hemophilia affects 1 in 5,000 male births. About 400 babies are born with hemophilia each year. Currently, the number of people with hemophilia in the United States is estimated to be about 20,000, based on expected births and deaths since 1994.


Sickle cell disease is an inherited disease that affects more than 80,000 people in the United States, 98 percent of whom are of African descent.


Von Willebrand disease, the most common inherited bleeding condition, affects males and females about equally and is present in up to 1 percent of the U.S. population.


Cancer: Cancer is the second leading cause of death by disease in the United States—1 of every 4 deaths—exceeded only by heart disease. This year nearly 1.6 million new cancer cases will be diagnosed, 78 percent of which will be for individuals ages 55 and older.


Cardiovascular Diseases (CVD): CVD claims more lives each year than cancer, chronic lower respiratory diseases, and accidents combined. More than 82 million American adults—greater than one in three—had one or more types of CVD. Of that total, 40.4 million were estimated to be age 60 and older.


Diabetes: In the United States, 25.8 million people, or 8.3 percent of the population, have diabetes. An estimated 18.8 million have been diagnosed, but 7 million people are not aware that they have the disease. Another 79 million have pre-diabetes. Diabetes is the seventh leading cause of death in the United States.


Genetic Disorders: There are more than 6,000 known genetic disorders. Approximately 4 million babies are born each year, and about 3 percent-4 percent will be born with a genetic disease or major birth defect. More than 20 percent of infant deaths are caused by birth defects or genetic conditions (e.g., congenital heart defects, abnormalities of the nervous system, or chromosomal abnormalities).


Alzheimer’s Disease: In 2010 there were an estimated 454,000 new cases of Alzheimer’s disease. In 2008, Alzheimer’s was reported as the underlying cause of death for 82,476 people. Almost two-thirds of all Americans living with Alzheimer’s are women.


Parkinson's Disease: This disease has been reported to affect approximately 1 percent of Americans over age 50, but unrecognized early symptoms of the disease may be present in as many as 10 percent of those over age 60. Parkinson's disease is more prevalent in men than in women by a ratio of three to two.

Asthma: An estimated 39.9 million Americans have been diagnosed with asthma by a health professional within their lifetime. Females traditionally have consistently higher rates of asthma than males. African Americans are also more likely to be diagnosed with asthma over their lifetime.


Skin Diseases: More than 100 million Americans—one-third of the U.S. population—are afflicted with skin diseases.

Wednesday, October 12, 2011

Personalized Therapies Mark Significant Leap Forward in Fight Against Cancer


Personalized Therapies Mark Significant Leap Forward in Fight Against Cancer
http://www.innovation.org/index.cfm/NewsCenter/Newsletters/Newsletters?NID=191
 
October 12, 2011 

This year marks the 40th anniversary of the signing of the National Cancer Act of 1971. Indeed, the 12 million cancer survivors living in the U.S. today attest to the significant progress in cancer prevention and treatment we have made over the past decades. Despite the remarkable advances we have made there are still more than 550,000 men and woman who lose their battle to cancer each year.
 
Recently released scientific data demonstrate that the collective commitment to cancer research is unwavering and our knowledge of the biology of cancer and ability to treat it continues to expand. One promising trend in cancer research: drug developers are harnessing an improved understanding of the molecular basis of many types of cancer to develop therapies uniquely targeted to these pathways.
 
For example, a newly approved drug for lung cancer called crizotinib is targeted to a mutation in a gene called anaplastic lymphoma kinase, or ALK. Mutations in the ALK gene are found in approximately 5% of patients with non-small-cell lung cancer. In data presented at this year’s meeting of the American Society of Clinical Oncology (ASCO), 54% of patients who received crizotinib were still alive after two years compared to just 12% in a control group. Crizotinib received fast-track review by the U.S. Food and Drug Administration (FDA) and was approved in August ahead of the six-month priority review schedule.
 
Dramatic advances are being made in the treatment of the skin cancer melanoma as well. More than 60 drugs are currently in development for the disease and this year two new medicines have been approved – the first approvals for the disease in 13 years. The first, ipilimumab, was approved in March and was the first treatment ever approved by FDA to show a survival benefit for patients with metastatic melanoma. In August the second, a new personalized medicine called vemurafenib, was approved to treat this deadliest form of skin cancer. This drug, which is taken orally, selectively inhibits a mutated form of the BRAF kinase gene. The mutated gene is associated with increased tumor aggressiveness, decreased survival, and is found in approximately half of all malignant melanomas. Recently reported clinical trials results demonstrate that the medicine reduces the risk of death by 63% percent.
 
Personalized medicine holds great potential beyond these two select examples in lung cancer and melanoma. MD Anderson Cancer Center recently reported on the results of a large-scale clinical trial examining the effect of matching targeted therapies with specific gene mutations across many cancer types. According to the results of the study, patients who received a targeted therapy demonstrated a 27% response rate compared to 5% for those whose therapy was not matched. This clinical trial marks the largest examination of a personalized approach to cancer care to date, and as principal investigator Apostolia-Maria Tsimberidou, M.D., Ph.D. concludes, "This study suggests that a personalized approach is needed to improve clinical outcomes for patients with cancer."
 
As these and many other studies illustrate, a dramatic transformation in cancer diagnosis and treatment is underway. Therapies targeted to the genetic and molecular underpinnings of disease are being developed, and patient outcomes are improving as a result. The studies highlighted above only begin to scratch the surface of the remarkable potential of personalized, targeted therapies, but are an indication of the great reward of years of research and investment, as well as great promise for continued innovation in the years to come.

Wednesday, August 17, 2011

USAID Expands Life-Saving Malaria Prevention Program in Africa

USAID Expands Life-Saving Malaria Prevention Program in Africa


FOR IMMEDIATE RELEASE
August 17, 2011
Public Information: 202-712-4810

http://usaid.gov/press/releases/2011/pr110817.html


WASHINGTON, D.C. - The U.S. Government, through the U.S. Agency for International Development (USAID), announced the expansion of its Indoor Residual Spraying (IRS) program. IRS is the application of safe insecticides to the indoor walls and ceilings of a home or structure in order to interrupt the spread of malaria by killing mosquitoes that carry the malaria parasite. Malaria is the number one killer in Africa.

Through the new IRS contract, the President's Malaria Initiative, led by USAID and implemented together with the Centers for Disease Control and Prevention (CDC), will provide technical and financial support to the Ministries of Health and National Malaria Control Programs in African countries to build country-level capacity for malaria prevention activities. The $189 million, there-year contract awarded by USAID to Abt. Associates will cover the implementation of IRS activities in Angola, Benin, Burkina Faso, Ethiopia, Ghana, Liberia, Madagascar, Mali, Mozambique, Nigeria, Rwanda, Senegal, Zambia, and Zimbabwe, with the possibility of expansion based on malaria control needs and availability of resources.


Activities include assessing the environment to ensure safe and effective use of insecticides, evaluating mosquito abundance and susceptibility to the insecticides, educating residents about IRS and how they should prepare their house for spraying, training spray teams, procuring insecticide and equipment, and monitoring and evaluating spraying activities.

"Here in Washington, a mosquito bite is a fleeting nuisance. But in all too many places, that sudden sting and scratch can be a death sentence. In a world being bound ever closer together, those places do not seem so far away," said Rear Adm. (RET) Tim Ziemer, U.S. Malaria Coordinator. "Preventing and treating malaria saves lives, contributes to a reduction in all-cause under-five mortality, improves the health of children in malaria-burdened regions, and contributes to socioeconomic development in areas most affected by malaria."

The United States is focusing on building capacity within host countries by training people to manage, deliver, and support the delivery of health services, which will be critical for sustained successes against infectious diseases like malaria. PMI continues to introduce and expand four proven and highly effective interventions in each of the target countries. Scale-up of the four interventions is complemented by a strong focus on extending expanding access in rural and underserved communities and further expanding community engagement for malaria prevention and control.

According to the World Health Organization, the estimated number of global malaria deaths has fallen from about 985,000 in 2000 to about 781,000 in 2009. In spite of this progress, malaria remains one of the major public health problems in sub-Saharan Africa, where malaria is the leading cause of death for children under five. Because malaria is a global emergency that affects mostly poor women and children, malaria perpetuates a vicious cycle of poverty in the developing world. Malaria-related illnesses and mortality cost Africa's economy alone $12 billion per year.

Friday, December 24, 2010

Europeans are approving important new drugs more rapidly than we are

The FDA Is Evading the Law. By SCOTT GOTTLIEB
Europeans are approving important new drugs more rapidly than we are.
WSJ, Dec 24, 2010
http://online.wsj.com/article/SB10001424052748704034804576025981869663212.html

This year, the Food and Drug Administration rejected the only medicine capable of treating the rare and fatal lung disease known as idiopathic pulmonary fibrosis. Pirfenidone, which has been available in Japan since 2008 and was just approved in Europe, was spurned by the FDA because the drug only showed efficacy in a single big trial—not the two large studies the FDA now requires. The decision to ban the drug is one of a rash of recent decisions that shows the FDA is making it more and more difficult for promising drugs to reach severely ill patients.

Last week, the FDA revoked an approval for the cancer drug Avastin because it said that evidence supporting its use in breast cancer wasn't strong enough. (The drug was judged ineffective because it merely stalls the spread of tumors.) European regulators, looking at the same data, made the opposite decision.

It wasn't supposed to be this way. In 1997, Congress passed the FDA Modernization Act, which gave the FDA broad discretion to reduce the quantity and rigor of clinical data needed to approve drugs targeting grave illnesses. The purpose of the law was to save lives by reducing the cost and time needed to launch such medicines.

But the FDA has steadily disregarded many of the law's provisions. Longer, larger trials that require drug makers to evaluate "hard" endpoints (like how long a cancer patient lives) rather than "surrogate" endpoints (like a drug's ability to shrink tumors) give FDA reviewers more statistical confidence. Reviewers prefer these drawn-out trials because they insulate the FDA from critics who say that it isn't focused enough on safety. But bigger trials increase the time needed to develop a drug, keeping it out of the hands of patients.

The Modernization Act also allowed the FDA to conduct drug trials in which patients are treated with an experimental medicine in a single group or "arm," and the trial can be completed in less than a year. But the FDA doesn't often opt for such trials. The agency commonly requests more complex, "multi-arm" and "placebo controlled" studies, which can take three years to finish and are much more expensive. Each patient enrolled in a trial adds over $30,000 to drug-development costs.

Of 76 cancer drugs approved since 2005, the FDA gave only 13 "accelerated approval"—another process created under the Modernization Act to expedite drug development. From 2001 to 2003, 78% of the novel cancer drugs approved were granted accelerated approval. Since then only 32% got the designation.

What's more, the clinical trial requirements that the FDA is imposing on cancer drugs with accelerated approval are now as burdensome as the requirements imposed on regular drugs. So, practically speaking, having "accelerated approval" doesn't mean anything.

Europeans are now approving novel drugs an average of three months more rapidly than we do. Of 82 novel drugs that were submitted for approval in both the U.S. and Europe between 2006 to 2009, 11 were approved only in Europe. One is for relapsed ovarian cancer, another for bone cancer.

To reverse these discouraging trends, Congress should reaffirm the provisions of the Modernization Act. It should spell out in legislation that the FDA "shall"—rather than "may"—approve drugs for severe conditions on the basis of a single study, or a more lenient statistical orthodoxy than "two, randomized, placebo controlled trials."

While Congress may not want to get into the business of establishing the FDA's analytical methods, it can call on the agency to convene an advisory panel to cultivate principles that are more permissive when it comes to very bad diseases. And it could go a step further, empowering patient groups with a mechanism to seek review of FDA decisions.

Congress also needs to modernize the way the FDA's review process is organized in order to increase efficiency and enable more cooperation. The science embedded in the most novel drugs is increasingly complex, requiring collaboration across many disciplines, including clinical medicine, pharmacology and statistical modeling.

But in recent years, the FDA has carved out each scientific discipline into its own distinct office. In addition, new work rules allow drug reviewers to spend two days each week working from home. The result is that FDA scientists don't collaborate well. Reviewers rarely meet as full teams, so they struggle to resolve internal debates and provide timely feedback to drug makers. The FDA's scientists should be organized around areas of therapeutic expertise—not broken into discrete offices based on what degree they have.

Finally, the FDA should be required to disclose its reasons for rejecting a drug.

The next Congress will reauthorize a user fee program that funds the FDA's review process. It should use this legislation to revive the FDA's fundamental mission: giving very sick Americans the best medical options available.

Dr. Gottlieb, a former deputy commissioner of the Food and Drug Administration, is a fellow at the American Enterprise Institute and a practicing internist. He invests in and consults with drug companies.

Friday, May 28, 2010

This week Medicare sent a flyer to seniors: The act passed by Congress "will provide you and your family greater savings and increased quality health care"

Medicare and Double Standards. WSJ Editorial
An ObamaCare mailer tells some howlers.WSJ, May 28, 2010

In the full-circle department, recall the moment last September when Senator Max Baucus and Medicare went after the insurer Humana for having the nerve to criticize one part of ObamaCare. It turns out those same regulators have different standards for their own political advocacy.

This week Medicare sent a flyer to seniors, ostensibly to inform them of what ObamaCare "means for you." Many elderly Americans are worried—and rightly so—about where they'll rank in national health care, given that the new entitlement is funded by nearly a half-trillion dollars in Medicare cuts. They must have been relieved to hear that "The Affordable Care Act passed by Congress and signed by President Obama this year will provide you and your family greater savings and increased quality health care."

That's the first sentence of the four-page mailer, and it gives a flavor of the Administration's respect for the public's intelligence. It goes on to mention "improvements to Medicare Advantage," the program that Democrats hate because it gives nearly one out of four seniors private health insurance options. "If you are in a Medicare Advantage plan, you will still receive guaranteed Medicare benefits."

But that's not what Medicare's own actuary thinks. In an April memo, Richard Foster estimated that the $206 billion hole in Advantage will reduce benefits, cause insurers to withdraw from the program and reduce overall enrollment by half. Doug Elmendorf and his team at the Congressional Budget Office came to the same conclusion, as did every other honest expert.

That's also what Humana told its customers, warning that seniors "could lose many of the important benefits and services that make Medicare Advantage so valuable." Medicare threatened the Kentucky-based company with fines and regulatory punishments for "misleading and confusing" beneficiaries, then issued a blanket gag order on Advantage insurers. The agency later backed down, once its Cosa Nostra message had been signed, sealed and delivered.

Medicare's flyer includes answers to other pressing questions in Boca Raton and Scottsdale, such as allowing children up to age 26 to remain on their parents' health plans, and further misleading commentary about keeping the program "strong and solvent." Dave Camp, the ranking Republican on the Ways and Means Committee, believes the mailer may violate the prohibition on using taxpayer dollars for political propaganda.

The larger issue is the White House's view of political opposition. It seems to think its assertions will be true if they are repeated often enough, as long as no one is allowed to disagree.

Thursday, May 27, 2010

Obamacare's Cooked Books and the “Doc Fix”

Obamacare's Cooked Books and the “Doc Fix”, by James Capretta
May 26, 2010

The Obama administration continues to insist (see this post from White House budget director Peter Orszag) that the recently enacted health-care law will reduce the federal budget deficit by $100 billion over ten years and by ten times that amount in the second decade of implementation. They cite the Congressional Budget Office’s cost estimate for the final legislation to back their claims.

And it is undeniably true that CBO says the legislation, as written, would reduce the federal budget deficit by $124 billion over ten years from the health-related provisions of the new law.
But that’s not whole story about Obamacare’s budgetary implications — not by a long shot.

For starters, CBO is not the only game in town. In the executive branch, the chief actuary of the Medicare program is supposed to provide the official health-care cost projections for the administration — at least he always has in the past. His cost estimate for the new health law differs in important ways from the one provided by CBO and calls into question every major contention the administration has advanced about the bill. The president says the legislation will slow the pace of rising costs; the actuary says it won’t. The president says people will get to keep their job-based plans if they want to; the actuary says 14 million people will lose their employer coverage, many of whom would certainly rather keep it than switch into an untested program. The president says the new law will improve the budget outlook; in so many words, the chief actuary says, don’t bet on it.

All of this helps explain why the president of the United States would be so sensitive about the release of the actuary’s official report that he would dispatch political subordinates to undermine it with the media.

It’s not the chief actuary’s assignment to provide estimates of non-Medicare-related tax provisions, so his cost projections for Obamacare do not capture all of the needed budget data to estimate the full impact on the budget deficit. But it’s possible to back into such a figure by using the Joint Tax Committee’s estimates for the tax provisions missing from the chief actuary’s report. When that is done, $50 billion of deficit reduction found in the CBO report is wiped out.

And that’s before the other gimmicks, double counting, and hidden costs are exposed and removed from the accounting, too.

For instance, this week House and Senate Democratic leaders are rushing to approve a massive, budget-busting, tax-and-spending bill. Among its many provisions is a three-year Medicare “doc fix,” which will effectively undo the scheduled 21 percent cut in Medicare physician fees set to go into effect in June. CBO says this version of the “doc fix” would add $65 billion to the budget deficit over 10 years. The entire bill would pile another $134 billion onto the national debt over the next decade.

If the Obama administration gets its way, this three-year physician-fee fix will eventually get extended again, and also without offsets. Over a full 10-year period, an unfinanced “doc fix” would add $250 to $400 billion to the budget deficit, depending on design and who is doing the cost projection (CBO or the actuary).

Administration officials and their outside enthusiasts (see here) say the Democratic Congress shouldn’t have to find offsets for the “doc fix” because everybody knows a fix needs to be enacted and therefore should go into the baseline. (By the way, the history of the sustainable growth rate [SGR] that Ezra Klein provides at the link above is a misleading one. The SGR was a replacement for a predecessor program that too had run off the rails — the so-called “Volume Performance Standard” enacted by a Democratic Congress in 1989.)

But supporting a “doc fix” is not the same as supporting an unfinanced one on a long-term or permanent basis. Not everybody in Congress is for running up more debt to pay for a permanent repeal of the scheduled fee cuts, which is why such a repeal has never been passed before. In the main, the previous administration and Congresses worked to find ways to prevent Medicare fee cuts while finding offsets to pay for it.

But that’s not the policy of the Obama administration. The truth is the president and his allies in Congress worked overtime to pull together every Medicare cut they could find — nearly $500 billion in all over ten years — and put them into the health law to pay for the massive entitlement expansion they so coveted. They could have used those cuts to pay for the “doc fix” if they had wanted to, as well as for a slightly less expansive health program. But that’s not what they did. That wasn’t their priority. They chose instead to break their agenda into multiple bills, and “pay for” the massive health entitlement (on paper) while claiming they shouldn’t have to find offsets for the “doc fix.” But it doesn’t matter to taxpayers if they enact their agenda in one, two, or ten pieces of legislation. The total cost is still the same. All of the supposed deficit reduction now claimed from the health-care law is more than wiped out by the Democrats’ insistent march to borrow and spend for Medicare physician fees.

And the games don’t end there. CBO’s cost estimate assumes $70 billion in deficit reduction from the so-called “CLASS Act.” This is the new voluntary long-term-care insurance program that hitched a ride on Obamacare because it too created the illusion of deficit reduction. People who sign up for the insurance must pay premiums for at least five years before they are eligible to draw benefits. By definition, then, at start-up and for several years thereafter, there will be a surplus in the program as new entrants pay premiums and very few people draw benefits. That’s the source of the $70 billion “savings.” But the premiums collected in the program’s early years will be needed very soon to pay actual claims. Not only that, but the new insurance program is so poorly designed it too will need a federal bailout. So this is far worse than a benign sleight of hand. The Democrats have created a budgetary monster even as they used misleading estimates to tout their budgetary virtue.

There is much more, of course. CBO’s cost projections don’t reflect the administrative costs required to micromanage the health system from the Department of Health and Human Services. The number of employers looking to dump their workers into subsidized insurance is almost certainly going to be much higher than either CBO or the chief actuary now projects. And the price inflation from the added demand of the newly entitled isn’t factored into any of the official cost projections.

We’ve seen this movie before. When the government creates a new entitlement, politicians lowball the costs to get the law passed, and then blame someone else when program costs soar. Witness Massachusetts. Most Americans are sensible enough to know already that’s what can be expected next with Obamacare.