Latest Drugwonks' Blog
By now you’ve surely seen the news (the good news) about Sarepta Therapeutics and it’s investigational drug for Duchenne muscular dystrophthe. The FDA (in a reversal of it’s original position) has indicated a willingness to consider the treatment for accelerated approval.
Much made of the high-pressure tactics employed by patient groups. But, if this decision is viewed as “the uncaring FDA caving in to desperate patients,” we’re being unfair to the agency, misrepresenting the intellectual potency of the patient voice, and missing a very important nuance.
In it’s Page One coverage of this story the Washington Post, after much FDA bashing (and towards the very end of the article), writes,
Not everyone faults the FDA for taking so long to decide how to proceed. Eric Hoffman, director of the Center for Genetic Medicine Research at Children’s National Medical Center and a longtime Duchenne researcher, said valid questions remain about eteplirsen’s dystrophin-producing abilities and how effective Sarepta’s drug really is.
“I’ve personally been very impressed with the FDA,” he said. “There’s every indication they are taking this extremely seriously.”
Hoffman said he understands the families’ sense of urgency, and he shares optimism about exon skipping. But he said the public campaign to pressure the FDA has divided the community more than united it.
“It becomes this form of bullying that puts everybody between a rock and hard place,” he said. “Unless you’re supporting kicking out the head of the FDA and granting accelerated approval as quickly as possible, then you’re supporting killing Duchenne kids.”
He said that the reality is more nuanced and that FDA reviewers do want to help patients but are wary about setting a precedent by approving high-priced drugs they aren’t yet convinced will work.
Precisely.
The Duchenne muscular dystrophthe case also serves as an important example as to why the FDA’s nascent Special Medical Use program shouldn’t be limited to anti-biotics/anti-infectives. There’s much work to be done – and the more options that are open to bring important new therapies to patients the better.
Accelerated Approval is good – but, as a recent blog from Context Matters points out, the FDA’s previously established programs to expedite therapies (including Fast Track, Accelerated Approval, and Priority Review) have not produced lasting improvements in approval time. “Based on our preliminary analysis, in 2008 the average cycle time for ‘Priority’ was 10.1 months, versus the ‘Standard’ which was 21.2 months. In 2011, the average cycle time for ‘Priority’ was 19.5 months, versus ‘Standard’ which was 17.5 months,” the blog authors wrote. “What’s interesting here is that over time the fast lane has apparently become just another standard lane; in 2011 it was actually even slower.”
What’s standing in the way of the FDA Special Medical Use Super Highway?
The FDA needs to have the proper infrastructure and support to successfully reduce approval times. Clearly, when you’re looking at products that have less data behind them, you need more senior people who can devote greater resources to studying them, and that also takes time away from other programs that the agent needs to review. It’s one thing to give the FDA more authority, it’s another thing to give the FDA greater responsibility, but if you don’t give them the resources to get it done, to a large degree it is just rhetoric.
And there’s more than enough of that to go around.
Dr. Michael Weber, hotshot cardiologist, Chairman of the Center for Medicine in the Public Interest, and all-around good guy weighs in on medication adherence and the value of apps.
The AP reports:
Medicine only helps if you take it properly. And adhering to an exact schedule of what to take, and when, can be challenging for patients who are forgetful or need to take several medications.
Doctors warn about the consequences and urge patients to use various techniques, such as using divided pill boxes or putting their pill bottles beside their toothbrush as a reminder to take their morning and bedtime medicines.
Still, only about half of patients take medication as prescribed, resulting in unnecessary hospital admissions and ER visits that cost the U.S. health care system an estimated $290 billion a year.
To help combat the problem, many doctors are trying a more high-tech approach: They're recommending smartphone apps that send reminders to patients to take their medications and record when they take each one.
"I think it's going to become pretty standard" for doctors to recommend them, said Dr. Michael A. Weber, a cardiologist at SUNY Downstate Medical Center. Weber began recommending apps to patients a few months ago and already has seen better lab results from a few using them.
"Some people say, 'That's a great idea,'" Weber said. "Even ones who claim they're conscientious, like the reminders."
He said the apps are particularly helpful for patients with symptomless conditions, such as high blood pressure or high cholesterol. Those patients are less likely to regularly take their medications than someone with pain or an infection.
"I don't think they're going to change the world," Weber said, though he recognizes benefit of apps. Even so, he said smartphone apps won't do much to help people who simply don't like taking medicine, fear side effects or can't afford their prescriptions.
The full AP story can be found here.
In a March 25, 2014 tweet and blog post, I inaccurately referred to Gabriel Levitt as an “admitted felon” and to his business, PharmacyChecker.com, as having “felonious business interests.” Mr. Levitt’s attorney has assured me that Mr. Levitt has never been charged with or convicted of a felony, nor has PharmacyChecker.com. Per Mr. Levitt’s attorney, PharmacyChecker.com provides pharmacy verification and drug price comparisons.
In today’s New York Times, Andrew Pollack reports:
Saying they can no longer ignore the rising prices of health care, some of the most influential medical groups in the nation are recommending that doctors weigh the costs, not just the effectiveness of treatments, as they make decisions about patient care.
And further:
In practical terms, new guidelines being developed by the medical groups could result in doctors choosing one drug over another for cost reasons or even deciding that a particular treatment — at the end of life, for example — is too expensive. In the extreme, some critics have said that making treatment decisions based on cost is a form of rationing.
This is an urgent discussion that has been going on for a while minus any real imput from providers. It’s good to have them join the conversation. But here’s the bad news:
The cardiology societies, for instance … plan to rate the value of treatments based on the cost per quality-adjusted life-year, or QALY — a method used in Britain and by many health economists.
It’s important to point out that NICE in England (not Britain – that’s an important distinction) has publicly stated that it is moving away from using QALY-based reimbursement decisions, moving to a value-based insurance design (VBID) strategy.
Here’s the important difference – QALY is based on cost, VBID is based on outcomes.
Everyone should welcome physicians to the crucial discussion over reimbursement. But the focus should be on paying for what works, not for what’s cheapest.
As for an open and honest debate over the costs/benefit of end-of-life care – it’s important, but politically problematic.
According to a new study published in JAMA Dermatology, dermatologists who receive free drug samples are more likely to give their patients prescriptions for expensive medicines.
Not surprisingly, that results in headlines such as “Free samples of prescription drugs are costly to patients, study says” (Los Angeles Times) and “Doctor’s free samples have a hidden cost” (NBC News). Sexy, but is that the real story?
The study reports that, for a single visit, the average retail cost of prescriptions for patients whose doctors received free samples from drug makers was about $465, compared with about $200 for patients whose doctors did not receive free samples.
What the study does not report (because they did not collect the data) was whether the free samples were the more therapeutically appropriate choice.
The reporting on the article (various news media) makes it sound as though generic and brand name drugs that treat the same condition are identical (and equally appropriate and efficacious) to each other. Not so. But why let important facts get in the way of a good story.
Most nauseating is the following study factoid:
In contrast, such drugs (sampled brand name drugs) only accounted for 17 percent of prescriptions written by doctors at an academic medical center that doesn't allow its doctors to accept free drug samples.
Well, duh. No samples in, no samples out. (Not to mention the fact that academic medical centers often dictate narrow formulary choices. Not surprisingly, that fact wasn’t mentioned in the study) It would also be interesting to match up patient outcomes of those derms who dispense samples vs. their ivory tower colleagues. Will any of these additional data sets be included in a follow-up study? Inquiring minds want to know.
The full study can be found here.
As the saying goes, everything you read about in the news is true –except for those things you know personally. Case in point: coverage of the FDA’s approval of the pain medicine, Zohydro.
Yesterday, U.S. District Court Judge Rya Zobel granted a request by a drug manufacturer Zogenix for an injunction temporarily halting Gov. Deval Patrick’s attempt to ban the prescribing of the painkiller Zohydro.
Massachusetts Governor Deal Patrick and others who want to ban Zohydro argue that the FDA Advisory Committee voted against approval.
True, but not necessarily accurate.
At an FDA advisory committee, the agency is asked to defend its scientific thinking in public, before a panel of experts who can dissect results, challenge conclusions, and ensure no clinical stone goes unturned. Seldom reported, however, is that advisory committee votes are recommendations. They aren’t binding on the FDA.
An analysis of advisory committee recommendations compared to agency actions shows FDA followed committee advice 74% of the time. Interestingly, the agency overruled “no” votes only three times: (Tarceva for maintenance therapy in lung cancer, Avastin for breast cancer, and Micardis to lower blood pressure.) Since their approval, these medicines have saved, extended, and improved hundreds of thousands of lives.
So, what about the Zohydro decision? The soundbite you’ve likely heard is that the vote was against approval of the drug. That’s true. What you probably don’t know is that, by a vote of 11-2, the experts affirmed that there was no evidence to suggest Zohydro had greater abuse or addiction potential than any other opioid.
When the committee voted, Dr. Bob Rappaport, Director of the FDA’s Division of Anesthesia, Analgesia, and Addiction, asked members to explain their votes. All but two said that while Zohydro had met their requirements for approval, their votes were meant to call greater attention to the agency’s regulation of opioids in general – not Zohydro specifically.
The FDA decided to approve Zohydro based on the agency’s judgment (and the advisory committee’s concordance) that the medicine is safe and effective. But the FDA also heeded the expert panel’s advice for better post-approval regulation of opioids. Shortly before Zohydro’s approval, the agency strengthened opioid labeling and post marketing requirements to address the concerns raised by the advisory committee.
A report by the Institute of Medicine found that 100 million Americans now live with chronic pain. That’s a third of the U.S. population. Ten million of those have pain so severe they are disabled by the pain.
The vast majority of people who use opioids do so legally and safely. A subset, about four percent, use them illegally. In the debate over safe and effective pain medicines, sound bites make headlines, but context matters more.
As many as 36 million Americans narrowly dodged a recent proposal from the Centers for Medicare and Medicaid Services (CMS) that would have advanced unprecedented government intrusion within the Medicare Part D prescription drug program.
CMS never offered a satisfactory reason for its proposed tinkering with something that's working. Part D is a real rarity -- a genuinely popular and cost-effective federal health program. The proposed reforms were not only unnecessary; they would have compromised affordability, limited treatment options for patients, and stifled innovation in the insurance industry.
Though the misguided proposal has been shelved, Part D is still at risk. The Obama administration has pledged to push through a similar plan in the near future.
By all accounts, Medicare Part D is overwhelmingly successful. The program offers drug coverage to seniors for a monthly average of $31 and enjoys a 90 percent approval rating among beneficiaries.
Unlike other healthcare benefits, Part D is also cost-effective for taxpayers. The nonpartisan Congressional Budget Office (CBO) reports expenditures are now 45 percent below original cost projections.
And because Part D provides access to vital medicines, the program helps seniors avoid expensive stays in hospitals and nursing homes. Over half of beneficiaries report they would be "more likely to cut back or stop taking medicine altogether" without Part D coverage. In this way, Part D saves our healthcare system $12 billion annually, according to a study in the Journal of the American Medical Association.
In light of the program's remarkable track record, it's inexplicable why CMS would attempt to tamper with important aspects of Part D.
As part of the proposed changes to Part D, CMS wanted to eliminate rules that provide "protected class" status to mental health drugs and drugs used for autoimmune diseases and organ transplant patients.
If the reform had taken effect, private plan providers may no longer have covered many of the medicines included in these designated categories. Countless Part D beneficiaries would have lost access to the drugs they were currently receiving and need to stay healthy.
CMS claimed that the proposal would lower prices by giving private plan providers better negotiating leverage with pharmaceutical companies. But that rationale ignores the fact that costs for these drugs have already been falling for years. From 2006 to 2010, in fact, prices for protected-class drugs dropped by two percent, despite an explosion in overall healthcare costs.
The idea that federal regulators can intervene to help negotiate better prices is dubious at best. And CMS didn't offer any quantitative evidence that restricting vital medications would benefit either seniors or the federal government.
Indeed, Part D works specifically because it encourages open competition between private insurers who are eager to serve Medicare's huge prescription drug market.
Nevertheless, federal regulators sought to reinterpret certain parts of Part D's "non-interference clause" -- the part of the law that forbids regulators from distorting the market by intervening in drug price negotiations. The proposed changes would have allowed the government access to all agreements struck by drug manufacturers, pharmacies, and insurers.
The new rules would have also limited the number of bids an insurance plan may offer in a region to two. By reducing the number of plans available, CMS would have weakened competition between insurers while also preventing plan providers from experimenting with new policies. Uncle Sam wants to reduce competition? What would the Federal Trade Commission have to say about that?
On top of all that, the agency suggested changes that would encourage nearly every employer who offers health insurance for retirees to drop prescription drug coverage.
There was one common thread running through these proposed changes to Part D: they all restricted choice and discouraged competition. The Obama administration, it seems, isn't content with a healthcare entitlement that relies on market forces -- no matter how cost-effective or popular the program may be.
When the Obama team shelved its plan, CMS Administrator Marilyn Tavenner only promised to pull it only "at this time." In fact, she committed to "advancing some or all of the changes in these areas in future years." So Part D isn't yet safe. In the future, if regulators want to sabotage the most successful federal health program in the country, the least they could do is explain why.
Last week’s 14th annual meeting of Pharmaceutical Research and Manufacturers of America (PhRMA) was a memorable pharmitzvah. Smart folks, old friends, challenging panels, and a bevy of good speeches on challenging topics.
And while there was the appropriate serving of rah-rah, there was significant substance alongside the Beltway style.
This was best summed up by Ian Read (Chairman and CEO of Pfizer) in his inaugural remarks as the incoming Chairman of PhRMA. It wasn’t a Morning in America oration. Read shared his concern about the industry’s failure in getting the message out about “the value we generate.” His key message, “We need to fix the misperception gap.”
Specifically he talked about the industry’s need to broaden the conversation from the economic performance of biopharmaceutical companies to the value that accrues to society and called for a “dialogue with society.” Bravo.
He asked, “Where are the headlines?” They’re not about societal value – and they need to be. There’s a strong story to tell. It’s not happening. And it needs to, because minus that narrative, nothing the industry wants to make happen (with government being a focus since the meeting was in Washington, DC) will be possible.
Read called for “industry speaking for itself.” After all, if you can’t be your own best advocate, you’re suspect in the minds of many – and rightfully so. He spoke to “better ideas and clarity” versus “more tactics.” That’s a foundational shift and a timely one. Innovators win when the discussion is about the future.
Read called for transparency in data sharing and physician relationships. Doing it is one thing – and most members of the pharmaceutical brethren are doing the right thing. But it’s equally important to tell the industy’s various constituencies what programs are in place, what they are designed to do, and how they work. Transparency mustn’t be allowed to be a cudgel used to beat innovators but a tool in it’s own communications arsenal.
Value to society. It was Read’s mantra. It needs to be the industry’s manifesto.
Our elected officials should act to protect medical progress in chronic pain relief. One hundred million people in the United States deal with debilitating chronic pain and need access to approved medications every day to stay productive and attain some measure of quality of life.
We must protect medical access for those afflicted with the most severe pain
The FDA did its job correctly by approving a unique treatment option based on its safety and efficacy data.
Do not let politicians overturn the FDA’s legitimate and expert approval of a pain medicine option that can bring relief. Do not infringe on patient access to approved pain medication options in any State. Help us send a message to Washington and state capitals across the nation to do the right thing.
Stand up and show your support for people with chronic pain. Please sign (and share) our petition today.
The on-line petition can be found here.
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.
