Latest Drugwonks' Blog
Anyone who has been diagnosed with cancer knows the importance of having an arsenal of therapeutic weaponry. So why are federal officials asking patients with advanced breast cancer to unilaterally disarm?
The weapon in jeopardy is Avastin, a biologic drug that cuts off the blood supply to tumors. Clinical studies prove that for women with stage 4 breast cancer, Avastin buys them weeks or months in which the cancer doesn't spread.
But the Food and Drug Administration isn't satisfied this time is “enough,” and will decide by Dec. 17 whether to revoke its approval of Avastin as a treatment for breast cancer.
Patients and their doctors have proclaimed they should decide what constitutes “enough.” Cancer treatment can't be one-size-fits-all. Some research has found, on average, Avastin doesn't prolong life. But scientists universally agree that it does prolong the quality of life. One study demonstrated that Avastin plus chemotherapy delayed tumor growth a median of about 11 months — five months longer than chemo alone.
Focusing only on averages ignores patients that are “super-responders.” Erin Howarth, was 31 when she learned her stage 4 breast cancer had spread to her spine, skull, pelvis and right leg.
“I got the impression that it was just like, ‘Well, you're going to come here for chemo every week . kind of until you die,'” Howarth told a newspaper.
But she sought out other doctors who put her on Avastin. Within seven months, her cancer was in remission. Labeling herself the poster child for Avastin, she concluded “it worked really well for me.”
Susan G. Komen for the Cure and Ovarian Cancer National Alliance wrote to the FDA urging them to preserve Avastin. They contended that removing it from the FDA's list of approved drugs for breast cancer could discourage future drug development.
Since the FDA fast-tracked approval of Avastin two years ago, it has been prescribed to about 17,500 patients a year with metastatic breast cancer.
This summer, a 13-member FDA advisory panel — with only two breast cancer oncologists — recommended that the FDA withdraw its approval of Avastin for breast cancer patients.
Doctors could still prescribe Avastin without FDA approval by going “off label.” But if the FDA revokes its breast cancer seal of approval for Avastin — insurance companies, Medicaid and Medicare are also likely to deny coverage.
The latest vote of confidence in Avastin comes from the National Comprehensive Cancer Network — a not-for-profit alliance of nearly two dozen of the world's top cancer centers. Last month, they announced it had reviewed and affirmed its guidelines for using Avastin to treat metastatic breast cancer.
The FDA should listen to these experts, and to patients. Avastin may well be the best weapon they've got to fight cancer — and the government shouldn't take it out of their hands.
Actions have consequences – often unintended ones. And the same is true for inaction.
Remember the medical device “gap” of the 1990s? That’s when Europe was outpacing the United States in bringing new medical technologies to market. Well – the gap is back, it’s growing – and it has consequences.
The #1 consequence is that Americans don’t have access to new technologies that make a difference. It also means that American companies (and their investors) wonder whether continued investment in research and development is worth it.
And what’s on the other side of the equation? One would think, well, safety. After all, nobody wants the FDA to approve unsafe medical technologies. Sure enough. But there’s no evidence that these newer options available in Europe are anything except safe and effective. Hence, there is a gap in care but none is device safety. What’s wrong with this picture?
All this while we debate reform to (among other things) the 510(k) process. Reform? Good. Better? Sure. But we must also address the issues of better with faster. We must learn from Europe. We must harmonize with Europe. And we mustn’t ignore the reality that the medical technology gap is widening and that this fact has consequences for the public health in the United States today as well as for the competitiveness of the American medical technology industry in the future.
A new survey of medical technology companies and investors (by Josh Makower, MD Consulting Professor of Medicine, Stanford University; Aabed Meer MD-MBA, and Lyn Denend Research Associate, Stanford University – with support from the Medical Device Manufacturers Association, the National Venture Capital Association, and multiple state medical industry organizations) is important reading -- and not just for industry and investors, but for thought leaders and policy makers – and especially those at CDRH.
The full report can be found here.
Don Berwick's coming out party was a little over an hour long. He was shepherded and sheltered by Finance Committee Dems who ate up the clock with softball questions about how Obamacare repeal would affect seniors. Berwick said disastrous without explaining how stoping cuts in hospice care and Medicare advantage and using comparative effectiveness to delay access to breakthroughs like Provenge are harmful. Republicans didn't even have enough time to clear their throats before the hearing was adjourned.
Berwick recycled comments he has made since the story about his love affair with rationing, centralized decisioning making and the NHS was broke by yours truly: He told the Committee seniors should get "all the care they want and need, when and where they want and need it."
He didn't mention that what people want and need will be limited by what government defines as quality care.
And he already broke his promise by delaying access to Provenge and rationing diabetes strips.
Read Linda’s entire blog post here.
Here is Berwick's testimony.
Innovation? Unmet needs? Where's the action?
An interesting new paper by Robert Kneller (University of Tokyo, Research Center for Advanced Science and Technology) The importance of new companies for drug discovery: origins of a decade of new drugs.
Here’s the abstract:
Understanding the factors that promote drug innovation is important both for improvements in health care and for the future of organizations engaged in drug discovery research and development. By identifying the inventors of 252 new drugs approved by the US Food and Drug Administration from 1998 to 2007 and their places of work, and also classifying these drugs according to innovativeness, this study investigates the contribution of different types of organizations and regions to drug innovation during this period. The data indicate that drugs initially discovered in biotechnology companies or universities accounted for approximately half of the scientifically innovative drugs approved, as well as half of those that responded to unmet medical needs, although their contribution to the total number of new drugs was proportionately lower. The biotechnology companies were located mainly in the United States. This article presents a comprehensive analysis of these data and discusses potential contributing factors to the trends observed, with the aim of aiding efforts to promote drug innovation.
Some relevant facts:
* Of the 252 new drugs approved by the FDA from 1998 to 2007
o 58% from pharmaceutical companies
o 18% from biotech companies
o 16% from universities, transferred to biotech
o 8% from universities, transferred to pharma
* 123/252 (49%) new drugs approved by FDA from 1998 to 2007 were for an unmet medical need
* 118/252 (46%) new drugs approved by the FDA from 1998 to 2007 were scientifically novel
* 24/252 (21%) new drugs approved by the FDA from 1998 to 2007 had an orphan designation
The full paper can be found here.
This is merely a euphemistic way of saying the government must decide who lives and who dies.
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.
