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To Burr, with Love
BioCentury reports that Senator Richard Burr (R-N.C.) threatened Thursday to delay reauthorization of medical device and prescription drug user fee legislation unless FDA implements steps to improve and speed product reviews. Speaking at a Senate committee on Health, Education, Labor and Pensions hearing, Burr said user fees, especially for medical devices, have not improved FDA performance and expressed skepticism that increasing user fees would improve the situation. He said reauthorization will become "a very slow and laborious process" unless the new legislation has measurement tools to track whether a fee system produces better outcomes
HELP committee Chairman Sen. Tom Harkin (D-Iowa) pushed back, saying that safety and efficacy are more important than speed to market. He also said FDA is understaffed and underfunded, so more money could improve its review performance.
They’re both right.
PhRMA wants dedicated biosimilars funding
The merits of creating a dedicated appropriation for biosimilars reviews has emerged as a point of contention in closed door FDA-hosted biosimilars user fee stakeholder discussions. In a July 24 letter from the Pharmaceutical Research and Manufacturers of America (PhRMA) to FDA, the trade association came down solidly on the side of creating a funding stream for biosimilar reviews that is separate from PDUFA-funded drug reviews.
PhRMA also called for a separate biosimilars user fee "trigger," or minimum amount Congress must allocate for biosimilars reviews to enable FDA to spend user fees. A trigger was built into PDUFA with the goal of making user fees supplement, not replace, federal funding. The law creating a biosimilars pathway called for FDA to fund biosimilars reviews from PDUFA funds until October 2012, when biosimilars user fees are expected to kick in. Applying PDUFA to biosimilars past October 2012 would drain resources from reviews of innovative medicines, according to the PhRMA letter.
Lack of money is the root of all evil.
-- George Bernard Shaw
This week the mainstream media discovered patent expirations and the headlines rang, “drug prices plummet!”
But they missed the real story.
From JAMA:
Implementation of Medicare Part D and Nondrug Medical Spending for Elderly Adults With Limited Prior Drug Coverage
1. J. Michael McWilliams, MD, PhD;
2. Alan M. Zaslavsky, PhD;
3. Haiden A. Huskamp, PhD
Author Affiliations
1. Author Affiliations: Department of Health Care Policy, Harvard Medical School (Drs McWilliams, Zaslavsky, and Huskamp); and Division of General Internal Medicine and Primary Care, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School (Dr McWilliams), Boston, Massachusetts.
Abstract
Context Implementation of Medicare Part D was followed by increased use of prescription medications, reduced out-of-pocket costs, and improved medication adherence. Its effects on nondrug medical spending remain unclear.
Objective To assess differential changes in nondrug medical spending following the implementation of Part D for traditional Medicare beneficiaries with limited prior drug coverage.
Design, Setting, and Participants Nationally representative longitudinal survey data and linked Medicare claims from 2004-2007 were used to compare nondrug medical spending before and after the implementation of Part D by self-reported generosity of prescription drug coverage before 2006. Participants included 6001 elderly Medicare beneficiaries from the Health and Retirement Study, including 2538 with generous and 3463 with limited drug coverage before 2006. Comparisons were adjusted for sociodemographic and health characteristics and checked for residual confounding by conducting similar comparisons for a control cohort from 2002-2005.
Main Outcome Measure Nondrug medical spending assessed from claims, in total and by type of service (inpatient and skilled nursing facility vs physician services).
Results Total nondrug medical spending was differentially reduced after January 1, 2006, for beneficiaries with limited prior drug coverage (−$306/quarter [95% confidence interval {CI}, −$586 to −$51]; P = .02), relative to beneficiaries with generous prior drug coverage. This differential reduction was explained mostly by differential changes in spending on inpatient and skilled nursing facility care (−$204/quarter [95% CI, −$447 to $2]; P = .05). Differential reductions in spending on physician services (−$67/quarter [95% CI, −$134 to −$5]; P = .03) were not associated with differential changes in outpatient visits (−0.06 visits/quarter [95% CI, −0.21 to 0.08]; P = .37), suggesting reduced spending on inpatient physician services for beneficiaries with limited prior drug coverage. In contrast, nondrug medical spending in the control cohort did not differentially change after January 1, 2004, for beneficiaries with limited prior drug coverage in 2002 ($14/quarter [95% CI, −$338 to $324]; P = .93), relative to beneficiaries with generous prior coverage.
Conclusion Implementation of Part D was associated with significant differential reductions in nondrug medical spending for Medicare beneficiaries with limited prior drug coverage.
http://jama.ama-assn.org/content/306/4/402.full.pdf+html
Recently BIO released a white paper on post-PDUFA FDA reform. (Full details can be found here.)
Many good ideas – but one that gets the blood flowing (whether you’re for or against): “progressive approvals.”
Sounds good (at least in theory) to many. But there are some tough questions. As an FDA insider opined, What exactly would the standard for progressive approval be? And would every one of the progressive approvals come with some sort of access control?”
Attempting to address some of these unknowns (via an op-ed in the Wall Street Journal) are Michael Boldrin (chair of the economics department at Washington University in St. Louis) and S. Joshua Swamidass (medical professor at Washington University).
They write:
“We can reduce the cost of the drug companies' bet by returning the FDA to its earlier mission of ensuring safety and leaving proof of efficacy for post-approval studies and surveillance.”
I’m not sure where they get the “earlier mission” statement, but let’s allow them to continue.
“In exchange for this simplification, companies would sell medications at a regulated price equal to total economic cost until proven effective, after which the FDA would allow the medications to be sold at market prices.”
Leaving the difficulty of determining what such a “regulated price” might be (and don’t for a minute believe that the devil isn’t in the details), an even tougher question (and a real rabbit hole of one) is the issue of “until proven effective.”
What does “effective” mean? Does it mean “cure” or “remission?” Does it mean "cost effective?" Or extension of life? And, if so, for how long? 5 years? 5 months? 5 minutes? And who makes the call?
According to Boldrin and Swamidass, “Doing so will improve all of our lives, decrease the cost of health care, and unleash the next wave of medical innovation.
It’s a weak argument in support of an important discussion.
Do we really want to open the door for tacit price controls and healthcare technology assessment in return for a very questionable upside?
Safety without efficacy? Really?
Better to pursue a path last publicly discussed in April 2009 when Merck agreed to peg what the insurer Cigna pays for the diabetes drugs Januvia and Janumet to how well Type 2 diabetes patients are able to control their blood sugar.
Now that’s progressive.
Stories reporting on the study that demonstrated that even when human cram themselves with BPA-heavy diets scientists "find the substance (in urine and blood) below our ability to detect them, and orders of magnitude lower than those causing effects in rodents exposed to BPA"? 17
As Trevor Buttorworth points out in his blog on Forbes: " the media have ignored the stunning finding – double checked before publication – that overturns pretty much everything the press has told the public about this common chemical." tinyurl.com/3wcgbl6
The Enviromental Protection Agency's response to the study?
EPA considers new call for toxicity testing of BPA
"The Environmental Protection Agency solicited public comment, July 26, about whether to require new toxicity testing and environmental sampling of bisphenol A, an ingredient in many plastics and food-contact resins."
All of which will be duly reported by the media in he-said, she-said fashion.
www.sfgate.com/cgi-bin/article.cgi
Lives wasted as FDA stalls on diabetes care
Wednesday, July 27, 2011
For more than 20 years, my daughter Piper has lived with the constant, frightening, deceptive and malicious disease called type 1 diabetes. Piper has always been prone to the kind of hypoglycemic - low blood sugar - life-threatening attacks that come on hard, fast and without warning. She almost drowned as a youngster after becoming unconscious from low blood sugar. In college, she went into hypoglycemia while she slept and didn't wake up in the morning. Fortunately, she was discovered and emergency care saved her life.
Unfortunately, the Food and Drug Administration has been dragging its feet on technologies that could revolutionize diabetes care and make these kinds of episodes a thing of the past. Key trials are on hold and it looks to be years more before these proven, life-saving technologies are available for patients in the United States. Meanwhile, kids are dying.
Every hour of every day, individuals with type 1 diabetes have to balance insulin, food and activity to try to prevent low and high blood sugars, and the devastating and costly complications: seizures, comas, kidney failure, heart disease, blindness and amputations. The human cost is incalculable; the economic cost isn't: Diabetes costs our nation more than $174 billion a year and $1 in $3 of Medicare spending goes to care for people with diabetes.
Perhaps the most gut-wrenching story of diabetes is the specter of "dead in bed" - kids found dead in the morning after a completely normal evening. Dead in bed occurs because blood sugar levels can suddenly change. When this happens while sleeping you are unable to adjust insulin to right the body's blood sugar, which can be life threatening.
We know how to prevent these attacks, but we don't - at least not here in the United States. Breakthrough technologies that protect against dangerous diabetes episodes are already available elsewhere, but not at home. Low-glucose suspend systems have been approved for nearly three years and used safely in more than 40 countries worldwide, but they are not available in the United States because of the FDA's unnecessarily slow process.
These pumps stop delivering insulin automatically when a monitor indicates that the body's glucose levels are low. The low-glucose suspend technology is the first phase of an artificial pancreas, a combination of a continuous glucose monitor and an insulin pump with software that would communicate between the two to automatically monitor glucose levels and administer insulin doses. The artificial pancreas would address both high and low blood sugar levels. In 2006, the FDA recognized the importance of this technology and placed the artificial pancreas on its Critical Path Initiative. But now key trials are on hold until the FDA provides a roadmap for outpatient studies. A draft is promised in December.
It should not have taken this long, and must not take any longer. When I testified before Congress, my message was simple: this technology could revolutionize diabetes care and it is imperative that the FDA provide reasonable guidance immediately. Waiting is not an option. My daughter's life, and those of millions of people with diabetes, depends on it.
Pam Sagan of Los Altos is a former board member of the Juvenile Diabetes Research Foundation International.
If you have to protect 3 million people from a brand-new law, it probably wasn’t very well written in the first place.
Mission creep is a worrisome thing – especially at the FDA.
Awhile back there were some folks at CDRH who believed that the mobiles that you hang over a baby’s crib should be classified as a medical device because they can impact vision development.
No – really.
Fortunately, cooler minds prevailed and sanity won the day.
Today, the issue is whether or not some mobile apps can be considered medical devices. It’s important for many reasons, not the least of which is that over-regulation or the threat of FDA action will slow both the development and adoption of mobile technologies for a variety or urgent public health purposes. Adherence and compliance come to mind as well as safety issues relative to appropriate use/safe use.
To that end, an interesting audio interview in the Burrill Report. It's with Joe Smith, chief medical and science officer for the Gary & Mary West Wireless Health Institute about new draft guidance from the FDA on medical apps, how the agency is approaching these products, and whether this provides the clarity needed to promote investment and innovation in this new world of digital health.
The interview can be found here.
This issue, BTW, is yet another reason why the name of CDRH (the Center for Devices and Radiological Health) needs to change to the Center for Medical Technology.
But FDA advisors are too scared about the possible risks of the drugs to allow diabetics and doctors to see how they work in the real world.
"Several committee members said they could have voted either way.
“I changed my mind about four times in the last 10 seconds,” said Erica H. Brittain, a statistician at the National Institutes of Health who voted no. "
The fate of a new drug should be decided by FDA's Hamlets?
The biggest safety concern was that in clinical trials, patients who got the drug were more likely to develop breast and bladder cancers than those in the control groups.
About 0.4 percent of women taking the drug got breast cancer, compared with 0.1 percent of the women in the control groups. About 0.3 percent of men getting the drug got bladder cancer, compared with about 0.05 percent of men in the control groups.
The numbers were very small, however, making it hard to draw definitive conclusions. Bristol-Myers and AstraZeneca argued that many of the cancers occurred too soon to have been caused by the drugs."
And get this:
"The committee members agreed that more study of the possible cancer risks and other safety questions would be needed. Those who voted no mainly believed that the studies needed to be done before approval, even though that might delay approval by years. "
How many people will die from diabetes related complications because this medicine is not approved in order to organize trials that will likely never resolve the issue?
This is pathetic. The advisory committee members are being haunted by the ghost of Steve Nissen.
Stephen Salzburg, who runs genomic research at the University of Maryland scores a direct hit on the Sun and the quacks whose crap it published in Fighting Pseudoscience
I also wrote a letter to Michael Cross-Barnet who is in charge of op-Eds at the Sun. Here it is in the likely case they don't print my piece:
The Baltimore Sun published two articles that ignore the scientific evidence about the importance and safety of evidence-based vaccination and then make discredited claims about how to make vaccines and immunization safer. It should be ashamed of itself for doing so.
Medical science is not a he-said, she-said process. It is an incremental process of proving and disproving hypothesis based on biological evidence established through experimentation. When facts don’t fit a theory or an assertion, it’s the latter that is wrong.
By giving two pseudo-scientists, Margaret Dunkel and Mark Geier, access to it’s press, the Sun has legitimized misleading and dangerous claims about vaccine safety and about the role vaccines play in causing all sorts of childhood disorders, particularly autism. It perpetuates assertions that contribute to the rise of vaccine preventable diseases such as measles, whooping cough and cervical cancer. And it has legitimized the idea that wild claims about a product causing autism are “science” even if such claims have never been proven scientifically or have been disproven. To the Sun, just raising the possibility of danger is enough to merit publication.
I will not restate the scientific evidence about the significant benefits and incredibly small risks associated with vaccines. One can read Stephen Salzberg’s editorial in Forbes for a concise discussion.
The problem is not with the Geiers and Dunkels of the world who peddle their conspiracy theories and lethal prescriptions for assuring vaccine safety. The problem is with newspapers, new shows and politicians who promote fearmongering.
Would the Sun allow those ‘scholars’ who deny the Holocaust or claim astronauts never landed on the moon access to its editorial page? Both types of conspiracy driven twaddle exist in spite of the facts, not because of them. Yet the Sun, in giving Geier and Dunkel a platform, has given the scientific equivalent of Holocaust denialism legitimacy and renewed strength.
In so doing, it has shamed itself and empowered quacks to endanger the lives of children.
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.
