Category: Pharmaceuticals

FDA Announces Draft Guidance That Would Limit Enforcement Discretion for FMT

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By Rachel Sachs

Yesterday, the FDA announced a new draft guidance regarding its exercise of its enforcement discretion around the investigational new drug (IND) requirements as they apply to fecal microbiota transplantation, or FMT.  For almost three years now, the FDA has exercised its enforcement discretion for FMT under a rather permissive set of guidelines, enabling patients to access FMT while companies shepherd a set of products through the clinical trial process.  I recently co-authored an article about the FDA’s regulation of FMT, and I’m concerned about this new guidance, in terms of safety, access, and regulatory clarity.  This is one of the wonkiest posts I’ve written in some time (and that’s saying something), so I’ll endeavor to be as clear as possible.

What is FMT and why is it important? To make a long story short, it’s a poop transplant.  Filtered stool from a healthy donor is transplanted into the gastrointestinal tract of a sick patient.  Although scientists are continuing to explore the use of FMT for a range of indications, we already know that FMT is a startlingly effective treatment for recurrent C. difficile infection.  C. difficile infections have become among the most common hospital-acquired infections in the United States, with more than 450,000 total incident infections annually.  Unfortunately, many of these infections are resistant to antibiotics: with those 450,000 infections came 80,000 recurrences and 29,000 deaths.  But FMT may provide a way forward.  A recent randomized trial of patients with recurrent C. difficile infections was stopped early, when 94% of patients in the FMT group were cured, as compared to roughly 30% of those getting only antibiotics.

How is the FDA involved? In May 2013, the FDA announced that fecal microbiota would be regulated as a drug.  All uses of FMT would therefore need to be part of an IND application, and patients who wanted to be treated with FMT for recurrent C. difficile would need to participate in a clinical trial to do so.  Physicians and scientists responded with concern, arguing that available evidence supporting FMT’s effectiveness as a therapy for recurrent C. difficile infection was too compelling for regulators to restrict its availability to clinical trials.  As such, in July 2013, the FDA announced that it would exercise enforcement discretion when FMT was used to treat patients “with C. difficile infection not responding to standard therapies,” so long as the treating physician obtained informed consent. Read More

Jacobus and Catalyst Continue to Race for Approval of LEMS Drug

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By Dalia Deak

The latest development in the race for approval between Jacobus Pharmaceutical Company and Catalyst Pharmaceuticals is a ‘refuse to file’ letter that the FDA issued to Catalyst indicating that Catalyst’s New Drug Application for Firdapse was incomplete. Both companies are competing for approval of slightly modified forms of a drug—3,4-diaminopyridine, or 3,4-DAP— to treat Lambert-Eaton myasthenic syndrome (LEMS). The winner will receive 7 years of exclusive marketing rights to the drug.

LEMS is an autoimmune disorder that affects an estimated 3,000 people in the United States. It is a rare, debilitating disorder that is marked by progressive weakening of the muscles that often begins in young adulthood. The drug in question was initially discovered in the 1970s in Scotland, with researchers in Sweden demonstrating its use in LEMS patients in the 1980s. Jacobus Pharmaceutical Company has been providing a free base form of the drug to patients with a LEMS diagnosis since the early 1990s at no cost (with the exception of postage), though the drug had never received FDA approval.

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Evolving Industry Structures in Biosimilar Development

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By Rachel Sachs

Yesterday, I had the privilege to moderate a fantastic event here at the Petrie-Flom Center on Assessing the Viability of FDA’s Biosimilars Pathway.  Bringing together expert panelists from legal practice (Donald R. Ware, Partner, Foley Hoag LLP), industry (Konstantinos Andrikopoulos, Lead IP Counsel, Manufacturing, Biogen, Inc.), and academia (W. Nicholson Price II, Assistant Professor of Law, University of New Hampshire School of Law), the event explored different aspects of the biosimilars issue, considering the guidances issued (and still to be issued) by the FDA, the role of the “patent dance” in biosimilar litigation, and whether Europe’s experience with biosimilars has helpful lessons for our own situation.  For those who weren’t able to make it, video of the event will be posted on the Petrie-Flom Center’s website soon.

But I wanted to write here about one of the very last questions we explored during the panel, because its implications are more far-reaching than we had the time to consider.  The situation is as follows:  In the decades after the Hatch-Waxman Act created a generic pathway for small-molecule drugs, companies typically specialized in developing either innovator or generic drugs, but not both.  And although generic drug companies had great capacity for innovating in manufacturing, they were not research companies in the way that we think about innovator companies.  The situation has changed somewhat over the years, as generic companies began to invest in innovative products, and as innovator companies put out authorized generics, but in general this broad division within industry has persisted.

In the biologic context, by contrast, the biosimilar applications being filed with the FDA are more typically being filed by innovator companies themselves or by subsidiaries thereof.  For instance, the only biologic approved in the United States thus far is marketed by Sandoz, which is part of the innovator company Novartis.  Instead of a situation in which innovators battle generic companies for access to the market, now innovator companies are battling themselves.  There are a host of reasons for this development, most notably including the complex manufacturing processes involved in the biologics space and the need for the development of expertise there.

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Why Senator Markey’s Message Hurts Children

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Last week, Senator Edward Markey (D-MA) placed a hold on the Senate’s nomination of Robert Califf’s as head of the Food and Drug Administration (FDA). The move was less against Califf and more as political leverage against FDA’s approval of OxyContin. In August 2015, FDA approved OxyContin, a prescription painkiller, for pediatric patients ages 11 to 17. OxyContin is the painkiller most associated with United State’s prescription drug abuse epidemic, accounting for an increase in drug overdose and death over the last decade. FDA’s approval of OxyContin for children drew concern from Markey and others that the approval would lead to an increase in drug misuse for children and their family members. Markey, who has prioritized the fight against opioid addiction in his legislative agenda, hopes he can use the hold to convince FDA to reverse its August decision.

Senator Markey’s message is well intentioned, but may ultimately do more harm than good for children.

Before FDA approved OxyContin in August, children who suffered from severe, chronic pain due to cancer, extensive trauma, or serious surgeries had few drugs approved to treat their pain. Many physicians treating severe pain in children prescribed OxyContin off-label, without proof that OxyContin could safely and effectively treat children. These physicians often relied on their experience or intuition to not under or overprescribe the drug. FDA’s approval in August meant the pharmaceutical manufacturer finally provided physicians with instructions, backed by controlled studies, explaining how physicians could safely use OxyContin to treat children with severe pain. Read More

TOMORROW, 1/29! Fourth Annual Health Law Year in P/Review Symposium

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MORE SEATS AVAILABLE! Fourth Annual Health Law Year in P/Review
January 29, 2016 8:00 AM – 5:00 PM
Wasserstein Hall, Milstein West AB 
Harvard Law School, 1585 Massachusetts Ave., Cambridge, MA

The Fourth Annual Health Law Year in P/Review symposium will feature leading experts discussing major developments during 2015 and what to watch out for in 2016. The discussion at this day long event will cover hot topics in such areas as health insurance, health care systems, public health, innovation, and other issues facing clinicians and patients.

In addition to presenting at the conference, many of our speakers will write about their topics for a collaborative blog series that will begin in February 2016 on the Health Affairs Blog.

This year’s Health Law Year in P/Review is sponsored by the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School, the New England Journal of Medicine, Health Affairs, the Hastings Center, Harvard Health Publications at Harvard Medical School, and the Center for Bioethics at Harvard Medical School, with support from the Oswald DeN. Cammann Fund at Harvard University.

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Four Key Issues In Health Law That Are As Relevant As Ever In 2016

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Petrie-Flom’s Executive Director Holly Fernandez Lynch has a new post over at the Health Affairs Blog as the first entry in a series that will stem from our Fourth Annual Health Law Year in P/Review conference, to be held at Harvard Law School on Friday, January 29, 2016.

In it, Holly takes stock of which major issues in health law policy remain outstanding from years past, and which are coming down the pike, including:

  • The ACA (and the contraceptives coverage mandate, in particular)
  • The state of health care reform generally
  • Efforts to control health care costs
  • Emerging regulatory pathways to speed patient access to new products
  • And more…

Read the full post here.

March-In Rights Alone Won’t Solve Our Drug Pricing Problems

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By Rachel Sachs

On Monday, a group of over 50 members of Congress sent a letter urging the Department of Health and Human Services (HHS) and the National Institutes of Health (NIH) to use a little-known statutory provision to take action against high drug prices.  Their goal is laudable, and HHS and the NIH should certainly offer guidance in this area – but the existing law offers only a partial solution to the problem as the legislators describe it.

The members of Congress wrote to remind HHS and the NIH of a provision in the 1980 Bayh-Dole Act giving the government “march-in rights” to patents resulting from government-funded research.  More specifically, the statute spells out a range of conditions under which the government may require a patentholder to grant licenses on reasonable terms to others to practice the patent.  The government may require such a license where “action is necessary to alleviate health or safety needs which are not reasonably satisfied,” 35 U.S.C. § 203(a)(2), or where the benefits of the invention are not being made “available to the public on reasonable terms,” 35 U.S.C. § 201(f).  The legislators argue that many drugs today violate these conditions, as even many insured Americans cannot access prescription drugs without incurring significant financial harm.

Although the “march-in rights” provision has existed in the statute since 1980, it has never been exercised by the federal government, even when it has been specifically asked.  And some of these cases have been paradigmatic examples where “health or safety needs” are not being satisfied.  A December 2015 study notes that in Bayh-Dole’s history, there have only been five petitions requesting that the NIH exercise its march-in rights.  Three of those requests were based on high prices for drugs for HIV/AIDS and glaucoma, and one was based on a persistent drug shortage which may have caused the deaths of people with Fabry disease, a rare condition.  (The fifth petition involved a medical device under patent litigation at the time.)  The NIH denied each request.  Some scholars argued that if the NIH denied the Fabry disease petition, where Genzyme’s drug shortages lasted for multiple years and caused great suffering, possibly including death, there may be no circumstances under which the NIH would grant such a petition.

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Peeling the Onion: How to Promote Pharmaceutical Innovation and Access to Medicine

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By Timo Minssen

As mentioned in my earlier blog post, I decided to conclude this year by publishing a introductory speech that I gave on April 14th, 2015 at the 2015 Broad Institute Innovation & Intellectual Property Symposium. The speech was part of the session “Bringing Therapies to the Patients” and introduced a panel-discussion with Entrepreneur and Professors of Law and Business about the failures of the patent system to support new therapeutics. The text is below:

Peeling the Onion:
How to Promote Pharmaceutical Innovation and Access to Medicine

Speaking about frustrations over the IP system in pharmaceutical innovation, sometimes feels like – to lend the words of the late German Nobel Prize winner Günter Grass – “peeling an onion:” Read More

Happy New Year: From “Weltschmerz” to Pharmaceutical Innovation

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By Timo Minssen

Dear readers and colleagues,

I would like to take this opportunity to wish you all a very happy, healthy and peaceful year 2016.

Reaching the end of 2015, I cannot stop thinking about the year that has passed. Being a native German, living in Sweden and commuting every week over the bridge to Copenhagen in Denmark – most recently with thousands of terrified refugees and border controls on the way back to Sweden – this year has left me with much astonishment and concern about the state of the European Union and our global situation. It appears to me as if the EU and other global leaders have focused far too much on tiny technicalities, while leaving the bigger issues untouched and disregarding crucial lessons of history. There are so many things that we must learn from 2015’s terrible events and alarming decisions, but also from the hope-giving agreements, incidents and initiatives. For me one of the most important take-aways is that everything is connected and that sustainable, realistic solutions not only require immediate actions. In my view, we need to think about long-term strategies both in more detail and from a bigger perspective. Due to the complexity of our most pressing problems this is a colossal task. It demands more knowledge, better communications, more collaboration and a more effective coordination of  the considerable skills and different competences that are already out there.

Returning to the actual topic of this blog, it becomes evident that this is also very much true for the health sector and the bio-pharmaceutical area. Not only the Ebola outbreak, global health crises, IPR debates, dreadful business models and controversial FTA negotiations, but also scientific break troughs, new therapies, legislative action and novel US and EU approaches demonstrate very clearly how this area is left with many challenges and opportunities. The recently approved US 21st Century Cures Act and the new EU Clinical Trials Regulation, for example, show how legislative activities pursuing laudable goals might lead to unwanted adverse effects if they are not carefully enough considered. Read More

Bioethicist Art Caplan: Shkreli Isn’t to Blame For High Drug Prices in U.S.

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A new piece by Bill of Health contributor Art Caplan on NBC News:

Should we care about Martin Shkreli, the man I call the “Wolf of Pharma Street”? His hoodie-wearing perp walk sparks outrage, but he is diverting attention from far bigger and more important systemic problems regarding the cost of drugs for all Americans.

Shkreli, the former Turing Pharmaceuticals CEO, has been indicted by the feds for allegedly running a Ponzi scheme to keep his various drug company investments afloat.

Before the feds came calling to charge him with securities fraud, Shkreli had secured the manufacturing license for Daraprim which is used to treat nasty, often fatal protozoal infections in, among others, those with AIDS. Shkreli, grinned, flipped the rest of humanity the bird, and raised the 62 year-old drug’s price by 5,500 percent; from $13.50 to $750 per tablet overnight — thus retiring the “Biggest Jerk in Health Care Award” forever. […]

Read the full article here.