Latest Drugwonks' Blog
FDA’s proposed process for qualifying drug development tools allows for the convening of advisory committee meetings or other public discussions about complicated submissions involving proposed biomarkers or patient-reported outcome instruments. A Center for Drug Evaluation and Research draft guidance on qualification of drug development tools (DDTs) states CDER “may choose to hold public discussions” for complex or controversial programs.
According to Marc Walton, associate director of CDER’s Office of Translational Sciences, “We certainly envision that for some things a public workshop might be appropriate,” Walton said. “For other things a formal advisory committee might be appropriate. It’s conceivable there might be some other venues that might be suitable in some cases. I think we would very much choose the mechanism to best suit the individual case.”
DDTs that are qualified for a specific context of use will be made publicly available to any sponsor for that purpose, and CDER reviewers will not need to reconfirm the tool’s utility each time. A tool’s qualified context of use may be expanded through additional data submissions. Companies that are developing a DDT for their own proprietary use should submit the necessary information with their investigational new drug application, NDA or BLA, rather than using the qualification process outlined in the guidance.
The agency says it will consider qualifying other clinical trial outcome measurement tools developed to support labeling claims, such as clinician and caregiver rating scales.
The agency is developing a Manual of Policies and Procedures that will lay out all the internal steps involved in the qualification process and contain more concrete goals for timeframes.
DDT qualification is not an activity currently supported under the Prescription Drug User Fee Act. However, if FDA gets its way in the current round of PDUFA reauthorization negotiations, future user fee funds may help support the process.
Sounds like something worth paying for – if the FDA can deliver the goods.
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.
Linda Gorman explains survival rates:
With funding from the Commonwealth Fund, Peter A. Muenning and Sherry A. Glied have produced a Health Affairs study of 15 year survival rates for men and women aged 45 and 65 in 13 countries for each year from 1975 to 2005.
A country’s survival rate is the percent of people of a given age who are still alive after 15 years. The authors ranked each country’s survival rate in each year. Because the U.S. relative ranking fell over the period while its per capita health spending rose, the authors conclude that the U.S. has a relatively inefficient health care system.
They blame the inefficiency on a rising number of people with “inadequate health insurance” and the reliance on “unregulated fee-for-service and specialty care” which they speculate may be “choking off public funding on more important life-saving programs.”
Little evidence is adduced to support any of these conclusions. In fact, the authors seem to jump through a myriad of speculative hoops to force the facts to conform to their preconceived views.
As is well known, age-adjusted survival rates are not by themselves a useful measure of the success or failure of a country’s health care system.
One reason that survival rates are not good measures of health system quality is that individual behavior has a large influence on health and, as a result, the health outcomes that produce survival rates. The authors do consider trans-national differences in smoking, obesity, and traffic accidents/homicide, three behavioral risk factors that receive a great deal of popular coverage. Unfortunately, their analysis of those factors is difficult to defend. At the same time, the authors ignore such factors as suicide, alcoholism, illegal drug use, poisoning, drowning, burns, and accidents other than homicide and traffic accidents.
Read Linda’s entire blog post here.
His written testimony to the Senate Finance Committee is an exercise in bowing and scraping to avoid prosecution. It is pathetic pablum and won't help. I am sure senators will have plenty of questions about MEDCAC, AHRQ, cuts to hospice care, comparative effectiveness research, his religious belief in the Dartmouth Institute, his claim that half of all medical spending is wasteful or harmful…
Here is Berwick's testimony.
Here is Berwick's testimony.
My article in the American Spectator today:
Are Medicare director Don Berwick and the Obama administration delaying or denying patients access to medical innovations? That's a question the Senate's Finance Committee should ask Berwick, who heads up the Center for Medicare and Medicaid Services (CMS), when he testifies November 17.
The timing couldn't be better. Medicare won't pay for Provenge, the first cancer vaccine, since it was approved in April. It's waiting for the Medicare Coverage Advisory Committee (MEDCAC) -- also meeting on the 17th -- to decide whether the Food and Drug Administration, the National Cancer Institute, and cancer experts are right in supporting Provenge use for prostate cancer patients.
MEDCAC was established in 1998 so CMS could ask leading scientists and doctors to recommend what information should be collected to determine the best use of new technologies. But recently MEDCAC has -- under CMS direction -- begun evaluating whether innovations are cost-effective.
In November 2008 Medicare bureaucrats asked MEDCAC: At current Medicare prices, how confident are you that CTC has a similar ratio of cost per LYS (Life Years Saved) as optical colonoscopy? In 2009 CMS asked MEDCAC: What are the desirable measures of the cost-effectiveness of screening genetic tests for the prevention or early detection of illness or disability? By 2010 CMS was simply asking MEDCAC whether CMS should cover new technologies unless there was evidence of its cost and clinical effectiveness from long and expensive studies. As one speaker at a MEDCAC hearing about genetic tests noted, "if clinical outcomes as defined… become[] a requirement for reimbursement, it will reduce investment in new genetic tests and the market introduction of these tests, and ultimately their use. "
That's the goal. But given Provenge's high profile, asking about its cost-effectiveness would be controversial. Since the one-time treatment runs about $90,000, CMS thought it could ask if Provenge was effective without mentioning cost. Hence, Dr. Louis Jacques, the director of the Coverage and Analysis Group at Medicare, told Forbes: "We've been getting questions from people," says Jacques. "'Well, what's up with Provenge? Is it a drug? Is it a biologic? Is it something else? Does it really work? It has been interesting to look at the evidence around it."
Does it really work?!
Maybe Dr. Jacques didn't get the memo about the FDA approving Provenge. The FDA said Provenge "substantially improved survival to patients with a fatal disease. The risks… are minor relative to the benefit of improved survival." Or perhaps he didn't see the May 6 National Cancer Institute statement asserting: "The field of cancer immunotherapy received an important boost last week with the FDA's approval" of Provenge. On May 29 the National Comprehensive Cancer Network (NCCN) Drugs & Biologics Compendium added Provenge to its list of standard therapies. On June 15 Aetna said it would cover Provenge.
Federal law requires that CMS cover any cancer drug approved by the FDA or NCCN compendium therapy. Instead, on June 30, an anonymous individual requested that CMS hold a MEDCAD hearing on Provenge before it covered the drug. CMS immediately accepted the query from the secretive party. Perhaps the Finance Committee can find out who that was.
MEDCAC will render its decision based on a just released evaluation of the FDA data conducted by the Agency for Health Research and Quality (AHRQ). AHRQ tried to pretend it wasn't second-guessing the FDA and the NIH. In October, the report name was changed on AHRQ's website from "The Efficacy and Safety of Sipuleucel T" (Provenge) to "The Outcomes of Sipuleucel T." The reviewers determined the FDA data used to approve Provenge was "adequate" but not entirely convincing. You might wonder: What expertise in prostate cancer did the authors use to draw to that conclusion? The answer is none unless you count nursing, a master's degree in statistics, or a PhD in sociology. Apparently CMS believes AHRQ's collective wisdom towers over the oncology expertise of the FDA, NCI, and NCCN.
The AHRQ report understates the impact of Provenge on survival. First, it begrudges the fact that many patients receiving chemotherapy after taking Provenge live longer. It focuses on the median survival benefit of 4.5 months (which tells you the midpoint of patient survival but not how many patients lived longer and for how long). Then it raises doubts about safety. And finally the study glosses over the finding that terminal prostate cancer patients who received Provenge were 40 percent more likely to be alive in three years than those who did not receive it. The AHRQ report is ideology masquerading as medical facts. The routine and expanding using of AHRQ to guide life or death decisions undermines the legitimacy of real science.
MEDCAC meets this week but CMS can take months to decide. This callous and possibly illegal process reflects Berwick's stated belief that only a centralized entity should decide what's best for us. People with prostate cancer have died and will die waiting. If that's not a death panel, I don't know what is.
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.
The media’s favorite economist Paul Krugman is under fire for these comments he made this past weekend on ABC regarding federal spending on health care.
“Some years down the pike, we’re going to get the real solution, which is going to be a combination of death panels and sales taxes.”
Krugman is now trying to correct the record, or so he says:
I said something deliberately provocative on This Week, so I think I’d better clarify what I meant (which I did on the show, but it can’t hurt to say it again.)
So, what I said is that the eventual resolution of the deficit problem both will and should rely on “death panels and sales taxes”. What I meant is that
(a) health care costs will have to be controlled, which will surely require having Medicare and Medicaid decide what they’re willing to pay for — not really death panels, of course, but consideration of medical effectiveness and, at some point, how much we’re willing to spend for extreme care
This is merely a euphemistic way of saying the government must decide who lives and who dies.
Do we really want Medicare bureaucrats determining the “medical effectiveness” of any given treatment or medication?
Sounds like a very slippery slope.
Via this week’s edition of Ad Age:
Drug Makers Brace for Possible Cuts to FDA
After Midterm Elections, Pharma Wary of Congressional Appetite for Reduced Spending on Federal Agency
The Nov. 2 elections that reshaped the structure of the U.S. House of Representatives are causing a headache in the pharmaceutical industry, which could lose millions as a hamstrung FDA leads to a clogged up drug pipeline.
With many congressional candidates riding into office on a platform of reduced spending, drug makers are bracing for what they believe could be cuts to government agencies, particularly to the Food and Drug Administration.
Astra Zeneca CEO David Brennan and FDA Commissioner Dr. Margaret Hamburg last week both told the Reuters Health Summit in New York that cuts to the agency would be detrimental. "I don't think that would be good for our industry. It doesn't look to me like that would speed things up," Mr. Brennan told Reuters, speaking of the time it takes the FDA to review new products, where delays of several months can cost pharma companies millions.
Ms. Hamburg cautioned the new Congress to think twice about FDA cuts.
"Not every function of government can be cut to the same degrees using the same tools. I think we should proceed with real care," she told Reuters. "It should be recognized if we can't do our job and do it well, there isn't any other entity that will backstop behind us. What we do really matters to health."
"I think the threat to cut FDA funding is real and it's a signal that FDA has to deliver on the promises that it made during the last PDUFA negotiations," said former FDA associate commissioner Peter Pitts, referring to the Prescription Drug User Fee Act, or the fees that pharmaceutical companies pay to the FDA. "It's a shot across the bow."
For the rest of the story, see here.
Joel Ario of HHS’ Office of Insurance Exchanges comparing Mitt Romney’s health system overhaul in Massachusetts to the federal health care law:
“You could say Obamacare was Romneycare before it was Obamacare.”
CMPI recently hit the streets of New York City to ask people if they expected the health care law to help them.
Watch the video below to hear their answers:
CMPI at Columbus Circle from CMPI on Vimeo.
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.
