Category: Pharmaceuticals

Unified Patents and Brexit: Britain’s Back on Board

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By Seán Finan

After several failed attempts, years of protracted negotiations and a glacial ratification process, it seemed as if Brexit would finally put an end to the nascent European Unitary Patent. Last week, however, the UK confirmed its intention to ratify the Unified Patent Court Agreement and to move forward with the plans for the creation of a single European Patent. If the UK goes ahead, the agreement would only be a German ratification away from coming into force. This means that we could see a single European patent by 2017.

So, what is the European Unitary Patent? How would it interact with the current patent regime in the EU? What would its introduction mean for the future of pharma and biotechnology in Europe?

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What is the Right Number of Unsafe, Ineffective Drugs for the FDA to Approve?

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By Rachel Sachs

Later today, the Senate will begin voting on the 21st Century Cures Act, which passed the House overwhelmingly last week. I’ve blogged repeatedly about the Act (most recently here), and many academics and commentators are rightfully worried about the Act’s efforts to lower FDA approval standards in different ways. I write here to put some of these concerns more plainly (and more bluntly), by asking a simple question: what is the right number of unsafe or ineffective drugs for the FDA to approve? I would like to hear the Act’s supporters answer this question. Below, I offer some thoughts of my own on how we should think about and evaluate this question.

More generally, when we think about FDA approval of new pharmaceuticals, we have to consider how the FDA should balance Type I and Type II errors. You may think the FDA ought to focus on minimizing the number of unsafe or ineffective drugs that it approves (minimizing Type I errors). After all, we don’t want the FDA putting its stamp of approval on drugs that harm patients or that don’t work. Over time, this would lead to an erosion of public trust in the FDA as a tool for consumer protection. More generally, this is the entire reason we’ve given the FDA its powers to begin with. Scandals involving unsafe or ineffective drugs prompted Congress to give the FDA more, greater powers over the years, in large part to prevent such products coming to market in the first instance.

Instead, you may think that the FDA should focus on minimizing the number of safe, effective drugs it fails to approve (minimizing Type II errors). In other words, it is worse for the FDA to deny patients access to a drug that is safe and effective than it is for the FDA to approve a drug that later turns out to be unsafe or ineffective. On this view, the FDA should still perform some screening against drugs with significant safety signals or against drugs with no plausible mechanism of action, and perhaps should require post-market surveillance studies, but the FDA ought to be enabling sick patients to access drugs more quickly. This view of the FDA’s role places greater responsibility on insurers, physicians, and patients to gather, process, and act on information about a drug’s safety and efficacy.

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REGISTER NOW (1/23/17)! PFC’s 5th Annual Health Law Year in P/Review

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p-review-logoHealth Law Year in P/Review
Featured Panel: The End of ObamaCare? Health Care Reform Under A New Administration
January 23, 2017 
Wasserstein Hall, Milstein West AB
Harvard Law School, 1585 Massachusetts Ave., Cambridge, MA
Register for this event

The Fifth Annual Health Law Year in P/Review symposium will feature leading experts discussing major developments during 2016 and what to watch out for in 2017. The discussion at this day-long event will cover hot topics in such areas as health policy under the new administration, regulatory issues in clinical research, law at the end-of-life, patient rights and advocacy, pharmaceutical policy, reproductive health, and public health law.

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, Harvard Health Publications at Harvard Medical School, Health Affairs, the Hastings Center, the Program On Regulation, Therapeutics, And Law (PORTAL) in the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital, and the Center for Bioethics at Harvard Medical School, with support from the Oswald DeN. Cammann Fund. 

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Your Weekly Reminder That FDA Approval and Insurance Coverage Are Often Linked

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By Rachel Sachs

In recent days, it seems like the din of voices arguing that the FDA should approve pharmaceuticals more speedily and on less evidence has grown louder.  It is a central theme of the 21st Century Cures Act, which the House may vote on today and which I seemingly will never finish blogging about (most recent post here, with links to previous ones).  It is the premise that underlies other legislation recently introduced into Congress.  And it was the topic of a Wall Street Journal opinion piece just last week.  In the view of these critics, sure, the FDA has some role to play in ensuring safety and some basic level of effectiveness.  But the current standard for demonstrating effectiveness is, in their view, much too strict.  Instead, we ought to approve drugs more quickly and allow insurance companies and physicians to decide which products have enough supporting evidence to merit reimbursement.

Here’s the problem: that is not the way we’ve set up the system.  FDA approval is often linked to insurance coverage.  Medicaid must cover essentially all FDA-approved drugs, and Medicare similarly has limited ability to decline to cover FDA-approved drugs.  Even private insurers are generally required to cover at least some prescription drugs, although in some cases this may be on a more limited basis.  Take Exondys, a drug that recently won accelerated approval from the FDA for the treatment of a small number of patients with Duchenne Muscular Dystrophy (I’ve blogged about Exondys here).  Because Exondys was approved based on a surrogate endpoint and not actual evidence of clinical improvement (Exondys’ label actually says that “[a] clinical benefit of Exondys 51 has not been established”), it would seem to be a poster child for these arguments about the FDA.  Allow insurers to cover it or not as they choose, since we don’t yet know if it works.  Yet many insurers are legally required to pay the $300,000 a year on average the company is charging for the drug.

I’ll put it another way.  If we lower the FDA’s approval standards and do nothing to coverage requirements, we will all almost certainly end up paying more money for drugs that don’t work.  The pronoun “we” here is important: because an enormous amount of these expenditures will come through Medicare and Medicaid, which are funded by all of us as taxpayers, it costs all of us financially when ineffective or unsafe drugs are approved by the FDA.  Many people who argue for a decrease in FDA standards also believe that we spend too much through Medicare and Medicaid, yet they don’t seem to put these two pieces of the argument together.

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The Precision Medicine Initiative and Access

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By Leslie Francis

Persistent differences in participation in clinical trials by race and ethnicity are well known; for example, the 2015 Report of the Working Group on Precision Medicine (PMI) relies on statistics that only 5% of clinical trial participants are African-American and only 1% are Hispanic. A recently-launched website of the FDA, “Drug Trials Snapshots,” confirms this dismal picture.

Designed to “make demographic data more available and transparent,” and to “highlight whether there were any differences in the benefits and side effects among sex, race and age groups,” the website reveals instead an impressive lack of information. Reported on the website are 70 new drug approvals for 78 different indications. These data report only evidence about differences by the census categories for race (White, Black or African-American, Asian, American Indian or Alaska Native, Native Hawaiian or Other Pacific Islander, and Unknown). In nine of the reported trials data were considered sufficient to report detected differences in efficacy or side-effects in all racial categories, in two data were considered sufficient to report these differences for African-Americans and Asians, in seven data were considered sufficient to report these differences for Asians, and in two data were considered sufficient to report these differences only for African-Americans. No data are reported about ethnicity, socioeconomic status, disability, or other categories that might be important to the PMI and the benefits data about the planned cohort might bring. Read More

Losing the Arms Race: Health Policy and Anti-Microbial Resistance

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By Seán Finan

And scattered about it, some in their overturned war-machines, some in the now rigid handling-machines, and a dozen of them stark and silent and laid in a row, were the Martians–dead!–slain by the putrefactive and disease bacteria against which their systems were unprepared; slain as the red weed was being slain; slain, after all man’s devices had failed…

H.G. Wells, The War of the Worlds  

The WHO World Antibiotic Awareness Week ran from 15-22 November. It coincided with similar European and American initiatives. So, in the interests of raising awareness, I thought I would highlight a few figures.

Photo by Rocky Mountain Laboratories, NIAID, NIHAntimicrobial resistance currently causes an estimated 70,000 deaths annually. If current practices continue, the death toll is expected to hit to ten million per year by 2050. That works out at about one death every three seconds.

The threat isn’t limited to increased mortality. Anti-microbial resistance could cast medical practice back to turn-of-the-century standards. Turn of the 20th century, that is. Without antibiotics, the chance of infection turns chemotherapy and invasive surgeries into mortal gambles. During these procedures, the body’s immune system is subject to massive exposure and needs antibiotic support. Even ordinary nicks and scratches can lead to fatal infections without effective antibiotics.

So what is antimicrobial resistance? How does it come about? What can we do to combat it and prevent the “antibiotic apocalypse”?

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Violations of federal antifraud provisions alleged against two hepatitis B treatment producers

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By Wendy S. Salkin

Two investor class-action suits have been filed within days of one another against two different California-based pharmaceutical companies both of which produce hepatitis B treatments, Dynavax Technologies and Arrowhead Pharmaceuticals. The named plaintiffs in both shareholder class-action suits, David Soontjens and Yaki J. Meller, are represented by counsel at Pomerantz, LLP.[1]

Meller v. Arrowhead Pharmaceuticals, Inc., et al., complaint filed (C. D. Cal. Nov. 15, 2016)

On November 15th, named plaintiff Yaki J. Meller filed a Class Action Complaint in the United States District Court for the Central District of California against Pasadena-based Arrowhead Pharmaceuticals, Inc., its President and CEO (Christopher Anzalone), and its CFO (Kenneth Myszkowski). Arrowhead is a biopharmaceutical company that, according to its website, “develops medicines that treat intractable diseases by silencing the genes that cause them.”

Among its clinical stage drugs are ARC-520 and ARC-521, which “are designed to treat chronic hepatitis B virus infection by reducing the expression and release of new viral particles and key viral proteins with the goal of achieving a functional cure.” ARC-520 is the drug at issue. According to the Complaint, Arrowhead knew but failed to disclose that ARC-520 “could be fatal at its higher doses and that the FDA was unlikely to approve the treatment as a result.”[2] In particular, the Complaint alleges that Arrowhead:

made false and/or misleading statements and/or failed to disclose that: (i) the Company’s ARC-520 was unsafe at certain doses and caused deaths in an ongoing primate toxicology study; and (ii) as a result, Arrowhead’s public statements were materially false and misleading at all relevant times.

According to Meller’s Complaint, in so doing, Arrowhead violated Sections 10(b) (“Position Limits and Position Accountability for Security-Based Swaps and Large Trader Reporting.”) and 20(a) (“Liability to contemporaneous traders for insider trading.”) of the Securities Exchange Act of 1934, “Position Limits and Position Accountability for Security-Based Swaps and Large Trader Reporting” and “Liability to contemporaneous traders for insider trading,” respectively, and Securities and Exchange Commission Rule 10b-5, “Employment of manipulative and deceptive devices.”

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The Newest 21st Century Cures Draft Moderates, But Doesn’t Eliminate, Controversy

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By Rachel Sachs

Earlier this evening, the House of Representatives released the most recent draft of the 21st Century Cures Act. This is the fifth time I’ve blogged about the Act (prior posts here, here, here, and here), which has ballooned from a 200-page discussion draft in April 2015 to a 996-page draft version today. (The House has a 44-page summary here for those with more limited time.) To be fair, the Act now contains a whole set of provisions around mental health, substance abuse, and child and family services which were not originally part of the Act. The 21st Century Cures Act is the biggest Christmas tree bill I’ve ever had occasion to read.

There will be an enormous amount of commentary on different parts of the bill, so here are some quick thoughts on the new draft, focusing not only on the provisions which are likely to attract the most attention, but also on a few quieter provisions that are nonetheless worthy of scrutiny.

Some controversial provisions have been eliminated entirely or softened greatly. One of the most controversial provisions in the last draft of the bill would’ve “farm[ed] out the certification of safety of modified devices to third parties, circumventing the FDA altogether.” That provision seems to be absent from the new draft. The last draft, in creating a program for breakthrough review of medical devices, controversially called for the use of “shorter or smaller clinical trials” for those devices. The new draft asks the Secretary only to ensure that the design of such clinical trials is “as efficient and flexible as practicable, when scientifically appropriate” (section 3051).

Other controversial provisions remain, sometimes under new names. One of the most troubling provisions in the previous draft of the bill would’ve created a program for the use of “clinical experience” evidence in drug approvals. Rather than relying on the gold standard of randomized clinical trials, this provision “would[‘ve] require the Secretary to establish a draft framework for implementing” such evidence. The new draft keeps this provision but changes the term “clinical experience” to “real world evidence” (section 3022). To be sure, this provision gives enormous discretion to the Secretary to limit (and maybe even reject) the use of such evidence. But in light of recent high-profile clinical trial failures, most notably just two days ago, we ought to be concerned about claims that the FDA is too slow and imposes too stringent requirements on drug approvals.

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Jordan Paradise on ‘The Week in Health Law’ Podcast

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By Nicolas Terry and Frank Pasquale

Subscribe to TWIHL here!

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Loyola Chicago law professor Jordan Paradise joins us to discuss some of her recent work in life sciences law. Jordan’s recent interests span nanotechnology, synthetic biology, precision medicine, gene editing, and electronic cigarettes.  Her publications have appeared in both peer-reviewed and legal publications.

We start with a review of some of the regulatory issues involving e-cigarettes, and discuss the 2016 FDA regulations. We then move into a discussion of FDA regulation of biologics and biosimilars and Jordan explains naming and substitution issues.

Jordan’s recent publications include research on how the FDA could regulate e-cigarettes, strategic misuse of risk evaluation and mitigation strategies (REMS), and cutting edge developments in the law of biosimilars.

The Week in Health Law Podcast from Frank Pasquale and Nicolas Terry is a commuting-length discussion about some of the more thorny issues in Health Law & Policy. Subscribe at iTunes, listen at Stitcher Radio, Tunein and Podbean, or search for The Week in Health Law in your favorite podcast app. Show notes and more are at TWIHL.com. If you have comments, an idea for a show or a topic to discuss you can find us on twitter @nicolasterry @FrankPasquale @WeekInHealthLaw

CALL FOR ABSTRACTS, DUE 12/2! 2017 Annual Conference, “Transparency in Health and Health Care: Legal and Ethical Possibilities and Limits”

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Medical care prices against a white background

2017 Annual Conference Call for Abstracts: Transparency in Health and Health Care – Legal and Ethical Possibilities and Limits
April 28, 2017 
Wasserstein Hall, Milstein East ABC (2036)
Harvard Law School, 1585 Massachusetts Ave., Cambridge, MA

The Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School is pleased to announce plans for our 2017 annual conference, entitled: “Transparency in Health and Health Care: Legal and Ethical Possibilities and Limits.”

Transparency is a relatively new concept to the world of health and health care, considering that just a few short decades ago we were still in the throes of a “doctor-knows-best” model. Today, however, transparency is found on almost every short list of solutions to a variety of health policy problems, ranging from conflicts of interest to rising drug costs to promoting efficient use of health care resources, and more. Doctors are now expected to be transparent about patient diagnoses and treatment options, hospitals are expected to be transparent about error rates, insurers about policy limitations, companies about prices, researchers about data, and policymakers about priorities and rationales for health policy intervention. But a number of important legal and ethical questions remain. For example, what exactly does transparency mean in the context of health, who has a responsibility to be transparent and to whom, what legal mechanisms are there to promote transparency, and what legal protections are needed for things like privacy, intellectual property, and the like?  More specifically, when can transparency improve health and health care, and when is it likely to be nothing more than platitude?

This conference, and anticipated edited volume, will aim to: (1) identify the various thematic roles transparency has been called on to play in American health policy, and why it has emerged in these spaces; (2) understand when, where, how, and why transparency may be a useful policy tool in relation to health and health care, what it can realistically be expected to achieve, and when it is unlikely to be successful, including limits on how patients and consumers utilize information even when we have transparency; (3) assess the legal and ethical issues raised by transparency in health and health care, including obstacles and opportunities; (4) learn from comparative examples of transparency, both in other sectors and outside the United States.  In sum, we hope to reach better understandings of this health policy buzzword so that transparency can be utilized as a solution to pressing health policy issues where appropriate, while recognizing its true limitations.

Call for Abstracts

We welcome submissions on both the broad conceptual questions described above and more specific policy issues, including: Read More