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The other evening I attended a dinner hosted by National Journal and AstraZeneca on the topic of “patient centricity.” Those around the table were payers, patient advocates, providers, pharmaceutical executives, IT geeks, and policy wonks.
Patient Centricity is a phrase we hear a lot and it’s lost a lot of it’s meaning – kind of like “personalized medicine.” But (in the immortal words of Jim Croce) “It doesn’t have to be that way.” "PC" is the new Healthcare.
In fact, when you think about it, both phrases really mean the same thing – driving positive therapeutic outcomes and, not surprisingly, the assembled diners agreed. After all, payers want (and are willing – mostly) to pay for treatments that work, physicians want treatments that provide the maximum therapeutic benefit, pharmaceutical companies want their products to work as well as they possibly can (so that physicians want to prescribe them to more patients and payers want to pay for them), and patients (last but not least) want to get well as swiftly and completely as possible.
(And, it’s no accident that many in Pharmaland have found religion in the wake of the intense focus on the prices of certain specialty medicines. It is now more urgent than ever to protect “sustainable innovation.”)
The point to remember is that a patient who is diagnosed earlier and treated with the most efficacious treatment as soon as possible is the least expensive over time. Alas, this means we must think long-term – not a trait the healthcare system is known for.
Patient Centricity = Driving Outcomes. Personalized Medicine, the right medicine in the right dose for the right patient at the right time, drives outcomes. Patient Centricity is Personalized Medicine.
QED.
Read More & Comment...One day before her resignation, HHS Secretary Kathleen Sebelius sent a letter to Congressman Bill Cassidy (R, LA). One of the subjects was academic detailing.
According to Secretary Sebelius:
AHRQ no longer conducts an academic detailing program as of September 2013.
But what happened to tax dollars already spent?
In October 2010, AHRQ awarded five grants for a program on academic detailing and the “communication of CER results to physicians”. This program to disseminate CER findings is supported by $29.5 million from the American Recovery and Reinvestment Act (aka, “the stimulus package”).
* One contract, for $11.7 million, went to Total Therapeutic Management (TTM) and is specifically intended for physician outreach and education
The goal of this contract was to integrate AHRQ’s comparative effectiveness research, products, and tools into clinical practice through 9,000 on-site, face-to-face visits with clinicians, nurses, health plan formularies, benefit managers, and other healthcare professionals.
According to TTM’s Barry Patel, “the AHRQ AD project to disseminate CER findings was a 3 year program that was scheduled to end in September 30th. The project has been over since Sept 30th 2013 as scheduled.”
Other recipient’s of the $29.5 million were:
* Ogilvy Public Relations Worldwide, Healthcare Division: $18 million to create a publicity center and another contract for $8.6 to create regional dissemination centers.
* Prime Education (an educational design and accreditation company focused on continuing medical education programs): $4 million as a “continuing education award.”
* IMPAQ International (a social science research and consulting firm that specializes in impact evaluations for a client base of predominantly U.S. government agencies): $2.4 million to evaluate the impact of the other four contracts.
Maybe it’s time for Congressman Paul Ryan to call a hearing to discuss the, um, outcomes of that $25.9 million.
Read More & Comment...Many of the claims about Zohydro are incorrect, and efforts to trump FDA's approval threaten the integrity of science-based regulation, FDA Commissioner Margaret Hamburg said in an interview with Steve Usdin of BioCentury This Week television. Edited excerpts follow.
BCTV: Why did FDA approve the drug?
Margaret Hamburg: I certainly understand the broader concerns that are motivating some of this in terms of the terrible public health burden of opiate addiction, abuse, misuse and overdose. But there is a need for adequate pain treatment of patients. And Zohydro, in fact, does represent an important pain medicine for people that respond better to hydrocodone - or if you need to rotate pain medicines because of chronic use - who don't want to be exposed to acetaminophen, which is a leading cause of liver toxicity, often even fatal liver toxicity.
And all of the other hydrocodone products currently in the marketplace are combined with acetaminophen.
BCTV: The governor of Massachusetts and several members of Congress have said Zohydro is superpotent and more addictive than other opioids.
MH: One of the common misperceptions is that this is the most potent opiate out there. And that simply isn't true. The second is that the other opiates out there have abuse deterrent formulations. And we wish that that were so. But in fact, almost without exception, the abuse deterrent claims of some of the products really don't hold water when tested.
And the one abuse deterrent formulation that has met the FDA criteria for labeling as abuse deterrent actually only provides deterrence against immediate injection or nasal intake - snorting. But it does not prevent oral abuse. And the majority of opiate overdose and abuse is through the oral route, not injection.
BCTV: Is there any deterrent now or even conceivable that's going to prevent somebody who's determined to abuse an opioid from doing that?
MH: Certainly not in terms of the formulation of the drug. There is nothing that can, at the present time, deter a determined abuser.
BCTV: Massachusetts and Vermont have tried to ban Zohydro. Other states are talking about it. What are the consequences of having states take that unprecedented action of overruling FDA approval?
MH: I do think that it's quite troubling. And I understand the motivation behind some of these actions in terms of the pressing need to reduce the burden of opiate addiction and preventable disease and death. But I think people should step back and really think very carefully about what it means for states or the Congress to start dictating what drugs should be approved and which ones should be withdrawn from the marketplace.
You can easily look at scenarios that are increasingly worrisome where, because of ideological beliefs or dislike for certain patient categories or other criteria, drugs that could make a difference in people's lives get banned.
BCTV: Some members of Congress have written to the HHS secretary and asked her to overrule FDA's decision. What happens if we have a political system where political appointees can overrule FDA on decisions that members of Congress don't like?
MH: I think it's very worrisome. FDA has a very carefully defined legal regulatory framework for our decision-making. Most importantly, we are driven by the science, and there is a set of scientific database standards for product review and approval. When individuals who have very different backgrounds and are in the political environment, not the scientific environment, start making the decisions for the public, you can end up in some very worrisome places.
The full video interview is available at www.BioCenturytv.com.
Read More & Comment...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.
Read More & Comment...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.
Read More & Comment...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.
Read More & Comment...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.
Read More & Comment...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.
Read More & Comment...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.
Read More & Comment...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.
Read More & Comment...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.
Read More & Comment...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.
Read More & Comment...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.
Read More & Comment...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
Read More & Comment...As a wise man once said, “He who tooteth not his own horn, that horn shall go untooted.
This is a valuable lesson that the FDA has learned to be true. And it couldn’t have happened at a better time. Specifically, I refer to opioids.
The FDA has taken the bit out of its mouth and taken hold of the reins of pain.
While scene-stealing members of Congress, some governors, and a gaggle of state attorneys general are trying to run roughshod over science-based regulation to great attention from the media, the FDA has been quietly doing the right thing without anybody noticing.
But when you do the right thing and don’t tell anyone about it, the assumption is that you’re not doing anything. That began to change with FDA Commissioner Hamburg’s aggressive testimony in front of the Senate HELP Committee defending the agency’s October approval of Zohydro ER.
And it continued yesterday with the agency’s approval of EVZIO™ (naloxone hydrochloride injection) for the emergency treatment of known or suspected opioid overdose. Smartly, the FDA used the approval to speak, more broadly, to the topic. There was a lot to say – and it’s about time the FDA said it.
Peggy’s complete remarks can be found here.
During the stakeholder teleconference the Commissioner laid it all on the table. It turns out that the FDA is doing a lot to mitigate opioid risk after all! Most importantly, they are doing so while understanding the need to ensure appropriate access for the tens of millions of Americans suffering from chronic pain.
She got specific:
Combatting the serious public health problem of misuse, abuse, addiction and overdose from opioid analgesics is a high priority. Since 2001 the FDA has taken a number of actions designed to help address prescription opioid abuse and to encourage the development of new drug treatments for pain. These actions include:
Revising the labeling for opioid medications to foster their safe and appropriate use, including recent changes to the indications and safety warnings of extended-release and long-acting opioids.
Requiring that manufacturers conduct studies of the safety of long-term use of prescription opioids.
Improving appropriate prescribing by physicians and use by patients through educational materials required as a part of a risk mitigation strategy for extended-release and long-acting opioids.
Using the agency’s expedited review programs to advance development of new non-opioid medications to treat pain with the goal of bringing new non- or less-abusable products to market.
Working with other federal agencies and scientists to advance our understanding of the mechanisms for pain and how to treat it, including the search for new non-opioid medications for pain.
Recommending that hydrocodone-containing combination products have additional restrictions on their use by rescheduling them from Schedule III to Schedule II.
Strengthening surveillance efforts to actively monitor the changing nature of prescription opioid abuse and to identify emerging issues.
And, importantly, encouraging the development of medications to treat opioid abuse, such as buprenorphine for use in medication-assisted treatment, and to reverse opioid overdoses, such as naloxone.
I can’t say I agree with all of these things. (Upscheduling impacts appropriate access), but it’s a pretty powerful list.
And, it seems, recalling drugs that are currently on the market isn’t on the agenda.
In the immortal words of Don Draper, “If you don't like what is being said, then change the conversation.
Peggy returned again and again to the role the FDA must play in facilitating physician education, not only through labeling language but physician education. She specifically mentioned CME and working to develop (with a broad constituency) validated tools for physicians to use in determining which patients may be more prone to slide into abuse so they can choose their therapeutic recommendations more precisely.
“It all comes back to provider education,” she said. Amen.
Provider education – the Hamburg Manifesto.
That’s not regulatory mission creep; it’s the appropriate application of the agency’s Safe Use of Drugs initiative. The way you make a drug “safer” is to ensure that it is used by the right patient in the proper manner.
Importantly, the Commissioner regularly referred not to “abuse” but to “misuse and abuse.” That’s more than a rhetorical flourish since it recognizes that misuse is a gateway to abuse.
Joining Dr. Hamburg on the call were Michael Botticelli, Acting Director, Office of National Drug Control Policy, Douglas Throckmorton, M.D., Deputy Director for Regulatory Programs, Center for Drug Evaluation and Research, FDA, Melinda Campopiano, M.D., Senior Medical Officer, Center for Substance Abuse Treatment, Substance Abuse and Mental Health Services Administration, and Wilson Compton, M.D., Deputy Director, National Institute on Drug Abuse, National Institutes of Health.
Oh, and HHS Secretary Kathleen Sebelius, who never mentioned the word “recall” either. It looks like the specter of Secretarial interference is off the table. Kudos to Secretary Sebelius for making it clear this is a cross-departmental priority.
The take away message was loud and clear –misuse and abuse of opioids is a serious issue that must be addressed in an appropriate manner.
And, per today’s teleconference, “appropriate” means science-based and patient-centric.
Read More & Comment...Media Inquiries: Sandy Walsh, 301-796-4669, sandy.walsh@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA
FDA Commissioner Margaret A. Hamburg Statement on Prescription Opioid Abuse
For more than a decade, the U.S. Food and Drug Administration has been working to address the important public health problems associated with the misuse, abuse, addiction and overdose of opioid analgesics, while at the same time working to ensure continued access to effective and appropriate medications for millions of Americans currently suffering from pain. I firmly believe that these goals are compatible, and that actions to address one should not be at the expense of the other.
Tragically, the most recent data shows that more than 16,000 lives are lost each year due to opioid-related overdoses. In fact, drug overdose deaths, driven largely by prescription drug overdose deaths, are now the leading cause of injury death in the United States – surpassing motor vehicle crashes. We know that the illegal diversion, misuse, and abuse of prescription opioids are often fueled by inappropriate prescribing, improper disposal of unused medications, and the illegal activity of a small number of health care providers. This highlights the important role that education of prescribers and patients can play in addressing this epidemic. The FDA has taken steps to address this but more work remains to be done.
Combatting the serious public health problem of misuse, abuse, addiction and overdose from opioid analgesics is a high priority. Since 2001 the FDA has taken a number of actions designed to help address prescription opioid abuse and to encourage the development of new drug treatments for pain. These actions include:
Revising the labeling for opioid medications to foster their safe and appropriate use, including recent changes to the indications and safety warnings of extended-release and long-acting opioids.
Requiring that manufacturers conduct studies of the safety of long-term use of prescription opioids.
Improving appropriate prescribing by physicians and use by patients through educational materials required as a part of a risk mitigation strategy for extended-release and long-acting opioids.
Using the agency’s expedited review programs to advance development of new non-opioid medications to treat pain with the goal of bringing new non- or less-abusable products to market.
Working with other federal agencies and scientists to advance our understanding of the mechanisms for pain and how to treat it, including the search for new non-opioid medications for pain.
Recommending that hydrocodone-containing combination products have additional restrictions on their use by rescheduling them from Schedule III to Schedule II.
Strengthening surveillance efforts to actively monitor the changing nature of prescription opioid abuse and to identify emerging issues.
And, importantly, encouraging the development of medications to treat opioid abuse, such as buprenorphine for use in medication-assisted treatment, and to reverse opioid overdoses, such as naloxone.
Today’s FDA approval of Evzio (naloxone autoinjector) provides an important new tool in our arsenal to more effectively combat the devastating effects of opioid overdose, which is one part of our comprehensive work to support opioid safety. Reflecting the FDA’s commitment to encouraging important new therapies, the FDA’s review of Evzio was granted priority status, and the application was reviewed by the FDA in just 15 weeks.
This product is the first auto-injector designed to rapidly reverse the overdose of either prescription or illicit opioids. While the larger goal is to reduce the need for products like these by preventing opioid addiction and abuse, they are extremely important innovations that will help to save lives.
The FDA will continue to work to reduce the risks of abuse and misuse of prescription opioids, but we cannot solve this complex problem alone. A comprehensive and coordinated approach is needed; one that includes the White House Office of National Drug Control Policy, the Drug Enforcement Administration and many of our sister agencies within the Department of Health and Human Services, as well as state and local governments, public health experts, health care professionals, addiction experts, researchers, industry, and patient organizations.
I am confident that this can be accomplished, but we will all need to work together to invest in strategies and responsible approaches that deter or mitigate the effects of abuse while preserving access to pain medicines for the patients that need them the most.
Read More & Comment...
On Tuesday I appeared on Al Jazeera America program, “The Stream.” The episode was titled “Inside the medicine cabinet,” and here’s how it was described:
Nearly 70% of Americans are on at least one medication. But for millions, combining too many of them could lead to dangerous consequences – even with safeguards in place to protect patients. We’ll explore what you need to know about your medicine cabinet.
Hosted by Lisa Fletcher, my fellow panelists were
Michael Carome
Director, Health Research Group at Public Citizen
Dr. Natalie Boulware
Naturopathic Physician, Tulsi Holistic Living
Dr. Caleb Alexander
Co-Director, Johns Hopkins Center for Drug Safety and Effectiveness
David S.H. Lee
Assistant Professor, Oregon State University College of Pharmacy
Good topic. Good panelists.
Click here for the full video.
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