Latest Drugwonks' Blog
The WARFARIN (Warfarin Adverse Event Reduction for Adults Receiving Genetic Testing at Therapy Initiation) study, designed to demonstrate the value of genetic testing for guiding warfarin dosing, has received the thumbs-up from CMS and is a major step forward for future reimbursement decisions.
(The randomized, blinded, parallel-group study was designed and funded by the privately held Iverson Genetic Diagnostics, Inc., which sells a test that can be used to predict response to the generic injectable anticoagulant warfarin and help guide dosing.)
The trial design was authorized by Centers for Medicare and Medicaid Services under its coverage with evidence development program. The agency decided in 2009 that Medicare would not cover the genetic testing for routine use, but it would for randomized controlled trials. In issuing the decision, CMS made clear that it wanted to see prospective results examining use of the tests.
The 18-month WARFARIN study will include 7,000 patients over the age of 65 and assess the practical value of testing for these genetic mutations, based on rates of severe hemorrhage and thromboembolic adverse events. Per request by CMS, the study will be monitored by an independent data coordinating facility.
Via molecular diagnostics specifically called out in the amended FDA label, the health benefits and the resulting savings in health care costs generated by using personalized warfarin dosing decisions are estimated to prevent 85,000 serious bleeding events and 17,000 strokes annually – and that’s just in the United States. And estimates predict the reduced health care spending from integrating genetic testing into warfarin therapy to be $1.1 billion annually. And that’s the mid-range.
“Safety means doing the right things for patients.”
Janet Woodcock
Development of Novel Combination Therapies
February 16, 2011 (10.1056/NEJMp1101548)
www.nejm.org/doi/full/10.1056/NEJMp1101548It seems like there’s momentum to fund the FDA’s Sentinel Initiative via PDUFA. Although, according a report in the Pink Sheet, “Industry is not interested in blindly pumping user fee revenue into the project.”
Per industry suggestions, only specific Sentinel projects would be funded by user fees and an open process would be required to make the determinations,
FDA and industry also talked about possibilities of establishing a process for determining which Sentinel projects were eligible for user fees, according to minutes of a January 10 meeting.
(Ain't transparency grand?)
The proposal would limit user-fee funded Sentinel projects to those that would “emphasize safety issues that affect classes of drugs or multiple products,” according to the minutes.
Also required would be an assessment of Sentinel’s value to regulatory decision-making on safety issues and adjustments to resource needs accordingly, with the evaluations targeted for fiscal years 2015 and 2017.
The minutes indicated both sides have agreed to the proposal and will present it to the full committee for consideration.
FDA also agreed to better describe how regulatory science upgrades would be relevant to the review model and PDUFA.
Saying publicly what has to-date only been whispered privately, both sides seem to be reaching agreement that funding for the agency’s initiative on advancing regulatory science (the Advancing Regulatory Science Initiative or ARSI) is a PDUFA-worthy proposition.
Does this foreshadow new and robust agency/industry collaboration?Stand by.
"One must have a good memory to be able to keep the promises one makes."
-- Friedrich Nietzsche
Statistics, as the saying goes, are like a Speedo. What they show you is interesting, but what they conceal is essential.
Consider the recent comments by Ezra Klein in the Washington Post. Mr. Klein first asks us to all shed a tear for the health insurance industry, telling us that it’s profit margin is only 4.54%. That sounds like the same math used by Hollywood studios to explain why blockbuster movies that gross hundreds of millions actually lose money.
But Mr. Klein (in a blog post widely circulated by AHIP) is not only chief apologist for the private payer community, but is also its finger pointer-in chief. Why are healthcare costs so expensive? According to Mr. Klein the problem is caused by “new and incredibly expensive treatments.”
Per Mr. Klein the problem is … innovation ... of the pharmaceutical and medical technology varieties. In other words what we need are fewer new molecular entities (NMEs) and not too many cutting edge medical technologies. Really?
As Albert Einstein said, “Only two things are infinite, the universe and human stupidity, and I’m not sure about the former.”
This strategy of projection and finger pointing misdirection is not only wrong – it will boomerang against those making the argument. Every poll on the issue of healthcare reform makes it abundantly clear that patients and physicians are keenly aware and unhappy with those who support a short-term cost-based payer-friendly paradigm over a more holistic patient-centric health care solution.
As Harvard University health economist (and Obama healthcare advisor) David Cutler has noted, "Virtually every study of medical innovation suggests that changes in the nature of medical care over time are clearly worth the cost." The repercussions of choosing short-term stock prices over long-term results are pernicious to both the public purse and the public health.
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.
