Latest Drugwonks' Blog
CMPI says requirements under federal health care reform would deprive Americans of medical innovation worth trillions over 10 years
New York, NY (June 12, 2012) --- Personalized medicine – treatments targeting individual risk of disease -- can increase life expectancy and generate $13 trillion in health value – but comparative effectiveness research would reduce investment in such innovations according to a report released today by the Center for Medicine in the Public Interest (CMPI), entitled: “Promoting Innovation and Health: Personalized Medicine or Comparative Effectiveness Research?”
"Our analysis shows that personalized medicine can increase life, well-being and the social value of health but also demonstrates that CER increases the cost and uncertainty of investing in such innovation," said John A. Vernon, Ph.D., a professor at the Purdue University, Krannert School of Management and co-author of the study. "CER will deny our nation better health and much needed biomedical investment."
The federal health reform law requires government-run comparative-effectiveness research, which will purport to evaluate the costs and benefits of different treatment options -- and use these studies to decide what medical innovations government and health plans should pay for and to limit what technologies doctors and patients can choose.
Proponents of CER claim it can increase investment in personalized medicine. But the CMPI study, conducted for its Personalized Medicine Acceleration Working Group, found that CER will reduce the level of investment in biomedical research by $75 billion over 10 years. There would be 60 fewer new personalized medicines available over the next decade if CER is required before marketing.
“We need to build upon the promise of personalized medicine,” said Robert Goldberg, Ph.D., Vice President of CMPI and co-author of the study. “Commercialization of innovations that reduce cost and extend life contribute to our nation’s prosperity and our global competitiveness. Policymakers should find ways accelerate the adoption of personalized medicine rather than adding regulatory requirements that increase the cost and risk of innovation.”
The study was conducted for The Personalized Medicine Acceleration Working Group, a CMPI project focusing on actionable steps that can be taken to speed up the commercialization and adoption of personalized medicine. The study and the working group were supported with a grant from the Ewing Marion Kauffman Foundation (www.Kauffman.org)
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The report -- "Uncertain Innovation: The Effect of Comparative Effectiveness Research (CER) On Personalized Medicine" -- can be downloaded here: http://cmpi.org/reports-newsletters/reports/uncertain-innovation
Reporters interested in scheduling an interview with the authors of the report should contact CMPI Vice President Bob Goldberg at rgoldberg@cmpi.org at 212-417-9169.
About CMPI
The Center for Medicine in the Public Interest, a non-profit public policy group dedicated to research-based free market reforms for the health care industry.
To support congressional work on user fee authorizing legislation and other FDA-related drug, device, and biological product provisions, Congressional Research Service analysts have prepared tables comparing provisions in the Senate passed bill that originated in the Senate Committee on Health, Education, Labor, and Pensions and the House suspension document that originated in the House Committee on Energy and Commerce.
• S. 3187, the Food and Drug Administration Safety and Innovation Act, passed by the Senate on May 24, 2012; and
• H.R. 5651, the Food and Drug Administration Reform Act of 2012, revised and dated May 24, 2012, as posted on the websites of the House Committee on Energy and Commerce and the House Committee on Rules.1
CRS has also provided brief summaries of relevant provisions in current law, mostly the Federal Food, Drug, and Cosmetic Act (FFDCA), but also the Public Health Service Act (PHSA), the Controlled Substances Act (CSA), and several others as noted.
The complete report can be found here.
CDER Staff:
It gives me great pleasure to announce the appointment of Gerald Dal Pan, M.D., M.H.S., as the permanent director of the newly reorganized Office of Surveillance and Epidemiology (OSE). CDER conducted a nationwide search for this position, and I am happy to say that the best candidate was right here in CDER.
OSE plays a critical role in monitoring and safeguarding drugs once they are on the market. As OSE director since 2005 and then, more recently, as acting director of the reorganized OSE (super office) since June 2011, Gerald has been instrumental in conceptualizing, standing up, and managing CDER’s postmarket drug safety and risk assessment programs. Throughout this time, he has provided strong leadership and direction to his staff and to the Center on a broad range of drug safety, epidemiological, risk management, and information technology activities.
Gerald has extensive knowledge of the scientific basis, regulatory laws, and best practice guidelines used to evaluate the essential components of postmarket drug safety: pharmacovigilance, pharmacoepidemiology, risk management, and medication error prevention. He has implemented numerous projects that have proven critical to advancing our postmarket drug safety programs. Under Gerald’s leadership, OSE has grown from a staff of approximately 116 to 250 over the last five years.
He has been a dedicated leader on CDER’s Safety First Initiative--working effectively across lines within the Center to build collegial and team-based work groups. He has also forged new relationships with other federal health agencies and the international community—all of which are helping to advance our oversight of marketed drugs. Such efforts strengthen our credibility and transparency, and the public’s trust in our ability to protect them from harm.
Gerald first joined FDA in July 2000 as a medical officer in the Division of Anesthetic, Critical Care, and Addiction Drug Products. He became director of OSE (then known as the Office of Drug Safety) in November 2005. He holds a medical degree from Columbia University and a Master’s degree in clinical epidemiology from Johns Hopkins University. He is board certified in internal medicine and neurology.
Gerald has been a trusted and valued advisor to me due to his medical and scientific background, his ability to tackle complex regulatory issues, and his grasp of the expectations of the public and policymakers. He has done an excellent job in his role as acting director of OSE, especially as the depth and volume of work in OSE have continued to increase and the pace has quickened. I would like to extend my sincere appreciation for all that Gerald has accomplished, and for his dedication to our public health mission.
Please join me in congratulating Gerald on his permanent position and wishing him continued success in this role.
Janet Woodcock
China has overhauled its intellectual property laws to allow Beijing to issue compulsory licenses to eligible companies to produce generic versions of patented drugs during state emergencies, or unusual circumstances, or in the interests of the public.
For "reasons of public health", eligible drug makers can also ask to export these medicines to other countries, including members of the WTO.
According to Reuters, “All eyes are now trained on how China battles it out with big foreign drug exporters, especially from 2013 when the Geneva-based Global Fund to Fight AIDS, Tuberculosis and Malaria will no longer give grants to China to fight HIV.”
It seems that IP doesn't permeate the Great Wall. It's time for the Middle Kingdom to seek some middle ground.
If you’re attending the upcoming BIO convention, please join an important and timely conversation on the past, present and future of drug shortages.
Drug Shortages: How will the United States Ensure Patient Care is not Compromised will take place on June 18th from 2-3:30pm in Room 258B.
I will be joined by Jouhayna Saliba of the FDA, Nancy Davenport Ennis, CEO of the Patient Advocate Foundation, Louis DeGennaro of the Leukemia & Lymphoma Society, as well as representatives from industry to discuss and debate the regulatory, legislative, economic and provider aspects of this troubling issue.
Hope to see you there.
Eyebrows raised at ASCO over Abraxane vs paclitaxel study
Many observers have been puzzled by the presentation at the American Society of Clinical Oncology meeting in Chicago of a study which claimed that two new breast cancer drugs - Celgene Corp’s Abraxane and Bristol-Myers Squibb’s Ixempra - were no better than paclitaxel.
The Phase III study enrolled 799 patients with locally advanced or metastatic breast cancer who were randomised to receive one of the three therapies - paclitaxel (the standard of care), Abraxane (nanoparticle albumin bound -'nab' - paclitaxel) or Ixempra (ixabepilone) - on a weekly basis with each cycle consisting of three weeks of treatment followed by a one-week break. Some 98% of patients also received Roche's Avastin (bevacizumab), which had its approval for breast cancer revoked by the US Food and Drug Administration in November 2011.
The data from the study, presented at ASCO by lead investigator Hope Rugo at the University of California, San Francisco, stated that median progression-free survival was 10.6 months for those receiving paclitaxel, 9.2 months for nab-paclitaxel, and 7.6 months for ixabepilone.
Grade 3 or 4 non-haematologic toxicities were also lowest in the paclitaxel arm, including sensory neuropathy (16% versus 25% for both experimental arms), while haematologic toxicities were lowest in the ixabepilone arm, and highest for Abraxane (12% versus 51%), compared to paclitaxel (21%). Dr Rugo said that the study "demonstrates that we should not simply assume that newer drugs are always better than the standard therapies".
150mg dose used in study never used in practice
However the findings, particularly in the Abraxane arm, caused raised eyebrows among oncologists at the meeting. The dose of the Celgene drug used was 150mg, well above the 100mg for which Abraxane is approved in over 40 or so countries, and when asked by PharmaTimes World News as to why the higher dose was selected, Dr Rugo noted that it had been used in a Phase II trial by the company.
However, Brian Gill, head of global corporate communications at Celgene, noted that there is only one trial to date "that is a true head-to-head study between Abraxane and paclitaxel". Those results showed statistical significance in time to disease-progression, PFS and overall survival. He added that "it was very curious to see why the decision was made to use the 150mg rather than the successful dose on the label" and expressed his surprise over the use of Avastin.
While Avastin is "a wonderful drug," lots of peer-reviewed publications note that the Roche drug, used in combination with other therapies, exacerbates toxicities, he told PharmaTimes World News. Between using the higher dose with Avastin, it appears that about 50% of patients actually discontinued the therapy in the latest study.
William Sikov of the Brown University School of Medicine, said he had no problems about the conduct of the study but questioned its design in terms of the 150mg dose, which would have led to missed and delayed doses in that arm. He was surprised by the broad statement that patients can do equally well on paclitaxel than Abraxane, a stance he felt was "contradictory" given that the latter is highly effective when used at the right dose.
When asked by PharmaTimes World News whether the 150mg dose was ever used in clinical practice, Dr Sikov gave a clear 'no', although occasionally 125mg is used. He noted that the 150mg used in the Phase II study had appeared to be the most active, although toxic as well, "but it is very different going from a 75-patient Phase II study to a 200-plus patient trial".
Other breast cancer specialists that spoke to this publication were also surprised that ASCO had chosen to highlight this study, with one suggesting that while making a case for the use of cheap generics is a valid one, in the case of Abraxane versus pacitaxel, the argument falls down.
Celgene has recently stated that peak sales of Abraxane, which is also being studied in lung and pancreatic cancer, as well as melanoma, could be in the region of $1.5 billion, with as much as a quarter of that coming from pancreatic cancer.
The report and Whitehead's comments come ahead of discussions about implementing the U.K. government's proposed value-based pricing system, which is slated to come into effect at the start of 2014.
He’s right.
When it comes to innovation in health-care technologies, there are some tough but important basic principles:
Innovation is slow. As any medical scientist will tell you, there are few "Eureka!" moments in health research. Progress comes step by step, one incremental innovation at a time. Biopharmaceutical companies more often profit by improving existing molecules and making processes more efficient than by revolutionizing the whole field with new "miracle" products.
Innovation is difficult. Today, it takes about 10,000 new molecules to produce one FDA-approved medicine. And if that's not frightening enough, only three in 10 new medicines earn back their research and development costs. And here's the kicker: Unlike other R&D-intensive industries, biopharmaceutical investments generally must be sustained for more than two decades before the few that make it can generate a profit.
Innovation is expensive. In 2003, researchers at the Tufts Center for the Study of Drug Development estimated the costs to bring a new medicine to market at $802 million. Others suggest that the total cost is closer to $1.7 billion. And that number is on the rise.
Innovation is under attack. From accusations of the "me-too" variety, to crackpot schemes to replace pharmaceutical patents with a "prize" system, life for innovator pharmaceutical companies is rough and tough. The late Israel Makov, founder of the generics giant Teva, once said that he wasn't really in the pharmaceutical business, but rather "the litigation business."
But innovation is important -- and not just for biopharmaceutical industry profits. Increases in life expectancy resulting from better treatment of cardiovascular disease from 1970 to 1990 have been conservatively estimated at bringing benefits worth more than $500 billion annually. In 1974, cardiovascular disease was the cause of 39 percent of all deaths. Today, it's responsible for about 25 percent.
Cerebrovascular diseases were responsible for 11 percent of deaths back then. In 2004, they caused 6.3 percent of deaths. Kidney diseases were linked to 10.4 percent of deaths then, and now they're associated with 1.8 percent. And that's just in the United States.
And, with all due respect to the pharmaceutical industry, innovation mustn't be only about medicines. We have to embrace innovative technologies for medical records and prescribing medications. We need innovative clinical trial designs and molecular diagnostics so that we can develop better, more personalized medicines faster and for far less than the current $1 billion-plus delivery charge.
So we'd better start taking innovation -- of both the incremental and discontinuous varieties -- seriously. And that means spending more on more complex developmental R&D (with concomitant higher investment risks).
Shame on the National Coalition on Health Care. The NCHC is foolishly calling on the FDA not to insist that biosimilars carry different names than their innovator precursors. Joining the NCHC are the AARP, AFL-CIO, the American Federation of Federal, State, County and Municipal Employees, Blue Cross and Blue Shield Association, California Public Employees Retirement System, and Service Employees International Union.
Why do these well-intentioned groups (including the Blues and the AARP – two of our nation’s biggest payer organizations) feel the urgency to remove an important level of safety and … knowledge for physicians (who write the prescription), pharmacists (who fill them), and patients (who ingest them)? Could it be a question of transitory cost savings over patient care? Just asking.
(In Europe, many healthcare systems are developing HTA standards and practices to ensure that product quality and therapeutic value are not inappropriately influenced by short-term cost considerations. We should learn from their experience.)
And, after all, regardless of your position on narrow therapeutic index, biosimilarity and interchangability (not to mention pharmacovigilance) – isn’t knowledge power?
Today, a House appropriations subcommittee will mark up a bill that would give FDA $44 million less to spend in Fiscal 2013 than a measure produced by the Senate Appropriations Committee. Look to a conference committee to be the final arbiter of agency funding -- again.
From the Wall Street Journal
Inside ObamaCare's Grant-Making
George Washington University is getting $1.9 million for a project to reduce health costs by $1.7 million.
By STEVEN E. GREER
Early this year, I was briefly involved with one of the Affordable Care Act's bureaucracies called the Center for Medicare and Medicaid Innovation, or CMMI. Despite its lofty ideals, it is one more pork program and venue for political cronyism, as I learned firsthand.
The innovation center is supposed to test better ways to deliver and pay for health care than the current fee-for-service system, and the outfit will spend $10 billion over the next decade on awards, grants and contracts. In a recent letter to Congress defending its work, the center touted its "structured clearing process" and "rigorous evaluation of models." But I found there are few safeguards and little transparency in practice.
This January, I was invited to review grant applications for something called the Health Care Innovation Challenge. Local health systems, state Medicaid programs and the like could apply for awards ranging from $1 million to $30 million, with priority for "projects that rapidly hire, train and deploy new types of health care workers." I was selected to become one of the chairmen overseeing the volunteer panels of outside experts. Our grant application reviews were supposed to help the CMMI in making the final award decisions. There were more than 3,000 grant applications in total.
Having written numerous other federal grant applications as a medical researcher, I was surprised by the very short time allotted to review 12 applications, each of which ran more than 100 pages. We had only two weeks to assemble a team and grade the applications on such criteria as the promise of the project design and its workforce goals.
Applications to the government's National Institutes of Health or the Patient-Centered Outcomes Research Institute, by contrast, undergo months of thoughtful review by scientists who are well-regarded in their fields. I began to wonder how much CMMI was interested in high-quality input from the grant reviewers.
To make matters more challenging, the computer system that handled our grant-scoring input was malfunctioning and deleting the painstaking comments and scores that our reviewers were inputting. When I reported the problems to the outside IT contractor, a support technician accused me and my team of never writing the reviews in the first place. All but one of my reviewers quit due to the digital hassle and the time-consuming workload.
That is when I sent a memo to CMMI Director Richard Gilfillan and Health and Human Services Secretary Kathleen Sebelius. I said that the rushed process was simply collecting "junk in, junk out" reviews. When I received no response, I left the CMMI program as well. Based on my experience, it seems unlikely that the 3,000 applications were closely vetted.
In May, the innovation center announced the first batch of grant recipients, 26 in all. George Washington University earned $1,939,127 because it expected to reduce health costs by a mere $1.7 million. Similarly, the Center for Health Care Services in San Antonio received $4,557,969 to save $5 million. At least CMMI didn't pick one of the thinly veiled handout requests that I personally oversaw, such as a for-profit company that claimed to offer holistic healing through human touch.
Of particular note was a $5,862,027 grant to the University of Chicago medical center to "train and create new jobs for an estimated 90 individuals from this high-poverty, diverse community." The program is part of the school's Urban Health Initiative. Perhaps creating this is a noble use of federal funds, but job creation seems far afield from the CMMI mission to innovate.
Dr. Donald Berwick was Administrator of the Centers for Medicare and Medicaid Services from July 2010 to December 2011. CMMI, which was established during his tenure, started another program called the Partnership for Patients. That $500 million initiative is supposed to reduce hospital-acquired conditions and hospital readmissions.
In December 2011, the Partnership for Patients awarded a contract to the Health Research and Education Trust, which in turn awarded a subcontract to the Boston-based Institute for Healthcare Improvement—which Dr. Berwick ran for 19 years before he moved to Medicare. A source involved with Partnership for Patients told me about the relationship.
I emailed Dr. Berwick in May to confirm the subcontracts between the institute and the trust. "I don't think there are contracts between them, but they're good friends," he replied. He was careful to note that he is no longer the institute's CEO, though he now works out of the institute's Boston offices as an adviser. The Health Research and Education Trust and the Institute for Healthcare Improvement have not responded to requests for information about the subcontract.
Even if the innovation center's grants were chosen via a squeaky-clean peer-review system and awarded solely on merit, they would still be a waste of taxpayer money because Medicare should not be straying from its core competencies into areas like job creation. Congress ought to dismantle and defund the program if CMMI survives the Supreme Court ruling on the Affordable Care Act.
Dr. Greer, a surgeon, is CEO of the Healthcare Channel (thehcc.tv) and a financial analyst.
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
