Latest Drugwonks' Blog
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.
I was recently interviewed by Context Matters on Expedited Reviews and the Current State of Drug Development. Here’s Part One of the conversation
Regardless of what you want to call it, whether it’s expedited review or special protocols, limited use medicines, the theory is, “How can the FDA help bring important therapies to the market faster?” And, there are a lot of pieces to that puzzle.
At the end of the day, the agency still has to meet with the sponsor, who still has to have data. They have to meet and decide how to move forward.
There are two issues that also have to come into this conversation:
- The patient voice
- Benefit / risk profile
Those are two things that really are not usually discussed. Generally speaking, in this type of conversation what comes about in the discussion is time. How quickly can something happen? Obviously faster is better than slower, but you don’t want to ever sacrifice proper review.
Blood From a Stone: Expedited or Standard, It’s All the Same FDA Resources
A key issue not being widely talked about is that the FDA isn’t hiring a whole new team to deal with expedited reviews. It’s the same people. So does an expedited review take precedence over a non-expedited review? How does the agency determine who gets the resources at what point in time?
Obviously, if you’re going to file a regular old-style NDA, you get a PDUFA (Prescription Drug User Fee Act) date and the agency is responsible for that. How do you weigh that against “critical medical need”?
The answer is it’s on a case-by-case basis. You can’t say, “Hey, we’re not going to make the PDUFA date because we’re reviewing someone else’s drug,” that’s a non-starter. You have to view expedited review situations in the broader context of everything else the agency has to do.
And then the agency is then going to define (on a case-by-case basis) what it feels is most important to the public health. Here’s an example: MS (multiple sclerosis) or IPF (Idiopathic pulmonary fibrosis) – Are either of those more or less important from an expedited review / resources perspective than a new anti-infective? It depends who you talk to. If MRSA issues are on the top of your agenda, then it’s one thing. If it’s orphan diseases, it’s another or if it’s pain-mitigation it’s something else again. Where you stand on these issues depends on where you sit.
I don’t really think having people specifically tasked to working on expedited reviews is the way to go, unless you have more people. And then the question is, where do they come from and do you take the best and brightest away from standard review, to put them there? How do you weigh the general importance of staff time and agency resources? Do you want it to be for a disease that is not an orphan disease that’s an important therapy, or do you want it to be on an orphan disease that can have a profound difference on people’s lives? Those are really philosophical questions, but it comes down to resources. I don’t think you can have dedicated teams when you don’t have people that can dedicate their time exclusively, otherwise it is just rhetoric. It’s just another committee on which the same people sit.
Comparing the Agencies
What might be interesting (and is generally not part of this conversation) is a move towards greater FDA / EMA harmonization on certain disease categories. There’s been a lot of chatter on this issue, but at the end of the day nothing really happens. I think the EMA would be interested in that, probably more than the FDA, because one of the main differences is the type of data accepted by the EMA. The EMA casts a wider net in terms of the types of data they accept for any given application.
One of the reasons the EMA has gone beyond the FDA is that their national economies are so much more financially strapped than we are here. It is important to remember that EMA decisions are only the first part of gaining market access. After the EMA, drugs go on to be evaluated by different country Health Technology Assessment (HTA) agencies to determine reimbursement and pricing in various markets. And from a reimbursement perspective, the HTA agencies are moving away from the QALY (quality-adjusted life year) towards a more value-based insurance design.
From a review proposition, from the FDA’s perspective, obviously they don’t get involved in cost issues, only clinical issues. I think what innovator companies are going to find out (especially with the expedited review pathways) is that the risk of getting a drug yanked quicker (meeting a quick death) rises. So they are going to have to understand, from a business perspective, the risk of asking the FDA for these special medical use reviews – it could backfire unless you understand what you are asking the FDA to do. The FDA is trying to signal that there are multiple ways to get an important new therapy to market quicker, but that has to be based on a public health need and not on a marketing strategy.
Governor Deval Patrick wants to ban Zohydro in Massachusetts.
That's what he wants, but what he needs is an intro level course in federal jurisdiction -- and he's about to get one.
US District Court Judge Rya W. Zobel would not grant an immediate restraining order as requested by drugmaker Zogenix, but scheduled a followup hearing for Monday, saying it appeared that Zogenix would have a likelihood of winning the case.
“I think, frankly, the governor is out of line on this,” Zobel said..”
She urged both sides to discuss the issue before Monday.
“I do not expect this to be a very long hearing,” he said.
Returning to the subject of expanded access to developmental medicines (When Compassion Isn’t Enough), I want to be clear that it wasn’t me who coined the term “expanded access.” As one of my former FDA colleagues commented, “In March 1990 the IOM Roundtable for the Development of Drugs and Vaccines Against AIDS held a workshop “Expanding Access to Investigational Therapies for HIV Infection and AIDS.” FDA staff, including me, participated in the Keystone national policy dialogue (Expanded access to promising therapeutic drugs for HIV infection and AIDS with implications for other life-threatening diseases) in the early 1990s. Also in the early 90s, FDA used the term expanded access at advisory committee meetings and at meetings of the National Task Force on AIDS Drug Development.”
Naming issues aside, this remains a highly contentious issue – and for all the wrong reasons. A new paper from the Goldwater Institute, “Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of their Treatment,” points the finger at the FDA as a roadblock to access, “Sadly, over half a million cancer patients and thousands of patients with other terminal illnesses die each year as the bureaucratic wheels at the FDA slowly turn.”
Nothing could be further from the truth.
What the paper presents a libertarian platform, “The burdens imposed on a terminal patient who fights to save his or her own life are a violation of personal liberty.” Maybe so, but the Supreme Court has ruled otherwise. In January 2008, the U.S. Supreme Court, without comment, opted not to accept an appeal of Abigail Alliance v. von Eschenbach. In other words, the federal appeals court ruling that patients do not have a constitutional right to experimental drugs stands
The paper continues, “Such people should have the option of accessing investigational drugs which have passed basic safety tests, provided there is a doctor’s recommendation, informed consent, and the willingness of the manufacturer of the medication to make such drugs available.”
I don’t think that anyone of those constituencies has any argument on that point. But should the FDA be cut out of the process? According to the paper, “… bureaucratic impediments violate an individual’s fundamental right to try to save his own life.”
But that’s consistent with the author’s libertarian philosophy. She believes that the “vast new granting of power (of the Kefauver-Harris Amendments) “was unwarranted.” So, consider the source of the argument.
Alacrity is important, certainly. But process is important too, as is collecting data on expanded access use.
The paper does raise important questions, such as at what point in the drug development process should an investigational product be available to patients? The author argues for Phase I. That’s an aggressive position, but one worth debating.
One item that paper ignores is that for the FDA to address single patient INDs with both more careful attention and speed is funding. That’s more than the 800-pound gorilla sitting in the room – it’s the 800-pound gorilla sitting on the chests of desperately ill patients who want access to investigational medicines.
The author quotes Patty Delaney. Patty (who passed away in 2008) was the FDA’s main liaison to the cancer community and a tireless soldier for “doing the right thing.” She was a pit bull on behalf of patients.
According to the paper, “As Patty Delaney, the former director of the FDA’s cancer liaison program explained in 2007, “the patient has a right to be heard, but in the end, it’s the data that matters. FDA opinions about safety and efficacy are always based on data.”
I’ll side with Patty.
Much chatter about Janet Woodcock’s Energy & Commerce Subcommittee on Health testimony last week. Much of it ill-informed.
Dr. Woodcock stood firm that separate labeling for generic drugs will advance the public heath by advancing 21st century drug safety. Subcommittee Chairman Joe Pitts (no relation) didn’t agree. His comment, “The only outcome I see is confusion,” demonstrates his own confusion.
“I’d like to dispel the notion that labels are the same now with respect to safety information,” said Janet. Representative Pitts was not mollified, moving on to suggest the agency’s motives were other than for advancing the public health – specifically that “trial lawyers” were to blame for the agency’s actions.
Trial lawyers? Clearly he’s met with representatives of the GPhA.
Janet made it clear that the decision to move forward on the labeling change rule was CDER and no one other than CDER. “The personnel in the Center for Drugs did not meet with the trial layers.”
Mr. Pitts’ suspicions were not assuaged. Whatever.
(PS/Representative Waxman commented that he found the GPhA’s report that distinct labeling would increase consumer spending on generic drugs by $4 billion annually “highly invalid.”)
Dr. Woodcock also clarified that the discussion about generics labeling did not extend to biosimilars.
Per Janet, “This rule does not apply to that because those would be under the Pubic Health Service Act – and they’re not considered generics, so that’s a separate issue.”
Much consternation over this last remark. Some see it as a nod and a wink that the agency is going to allow biosimilars to have the same name and labeling language as innovators. I disagree for two reasons:
(1) Janet was precisely correct in stating that generic drugs and biosimilars are two distinct things. Biosimilars are not generic drugs.
(2) The fact that the FDA has made distinct generic drug labeling such an important policy initiative certainly does not send a signal that they will view biosimilars in a more laissez faire manner. Au contraire.
So, for people trying to read too much into Janet’s statement that generic drug labeling is different from biosimilar naming and labeling, relax. If anything, the news is good.
Prediction is very difficult, especially about the future. – Niels Bohr
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.
