Bold New Policies for The Brave New Biologies: IPRs and Innovation in Synthetic Biology and Gene editing

Research Seminar at the University of Copenhagen debating intellectual property and innovation in synthetic biology, systems biology & gene editing.

New technologies in biology offer a brave new world of possibilities. Promising solutions to some of the most urgent challenges faced by humanity: climate change, environmental protection, growing population, renewable energy and improved health care. Scientific and technological progress has been remarkable. Simultaneously, emerging life science technologies raise outstanding ethical, legal and social questions.

In this research seminar, Prof. Esther Van Zimmeren from the University of Antwerp joins Prof. Timo Minssen, Postdoc Ana Nordberg and Ph.D. Student Jakob Wested from the Centre for Information and Innovation Law, debating bold new policies for intellectual property law and incentive to life science innovation.

Programme

15:00 – 15:10 Welcome
Prof. Timo Minssen, CIIR, Faculty of Law, University of Copenhagen.
15:10 – 15:30 Waiting for the Rumble in the Jungle: – An overview of current CRISPR/CAs9 patent disputes, central legal issues and some thoughts on conditioning the innovation system.
PhD Student Jakob Wested, CIIR, Faculty of Law, University of Copenhagen.
15:30 – 15:50 From FRAND to FAIR for Synthetic and Systems Biology? The Implications of Openness, IP Strategies, Standardization and the Huawei-case.
Prof. Esther van Zimmeren, Faculty of Law, University of Antwerp.
15:50 – 16:10 Keeping up with the technologies: IP Law and Regulation in the age of gene editing.
Postdoc Ana Nordberg, CIIR, Faculty of Law, University of Copenhagen.
16.10 – 17.00 Questions and panel debate

Time: 13 March 2017, 15:00 – 17:00

Venue: Meeting Room 7A-2-04 , Faculty of Law, Njalsgade 76, DK-2300 Copenhagen S

Registration:
The event is free to attend. Registration is mandatory. Please use this registration form no later than Monday, 13 March 2017, 11:00 at the latest.

Organizer: Copenhagen Biotech & Pharma Forum, at CIIR, Faculty of Law, University of Copenhagen

Manufacturers Of Biosimilar Drugs Sit Out The ‘Patent Dance’

This new post by Claire Laporte appears on the Health Affairs Blog in a series stemming from the Fifth Annual Health Law Year in P/Review event held at Harvard Law School on Monday, January 23, 2017.

Believe it or not, the Patient Protection and Affordable Care Act (Obamacare, or here, ACA) has intellectual property provisions. In addition to establishing mandates, subsidies and insurance exchanges, the ACA also created a new pathway for the approval of biosimilar drugs, which are akin to generic drugs. That pathway appears in a corner of the ACA that has its own title: the Biologics Price Competition and Innovation Act (BPCIA). The BPCIA is rich with intellectual property (IP) provisions that are now the subject of litigation in the Supreme Court.

Background: Generic Drugs And Biosimilars

Many of us take generic drugs for granted, but we have them only because the Hatch-Waxman Act (1984) provided an abbreviated pathway by which FDA could approve them. Under this pathway, a generic drug could be approved based on the safety and efficacy of the branded drug, plus a showing by the generic that it was essentially identical to the branded drug. This pathway also included provisions by which generic drug manufacturers could challenge the validity of patents protecting the branded drug. […]

Read the full post here.

Call for Papers – European Pharmaceutical Law Review (EPLR)

Dear Colleagues,

I am happy to announce that I have just joined the Board of Editors of the new journal “European Pharmaceutical Law Review” (EPLR). One of my first tasks is to spread the news about our “Call for Papers”. Further information is available here.

The European Pharmaceutical Law Review (EPLR) reports on key legislative developments in the EU and the Member States, and identifies and analyses important judgments that shape the interpretation and application of EU pharmaceutical law, in particular those by the European Courts, international courts and tribunals such as the WTO’s Dispute Settlement Body, and higher national courts.

In order to establish itself as a forum for dialogue between different stakeholders in pharmaceutical regulation and governance, it will invite contributions from academics, practitioners, regulators and civil society representatives. Topics covered by EPLR include:

  • Pharmaceutical law and policy in all jurisdictions (regional, national, international);
  • Commission decisions (EMA opinions) and regulatory guidelines;
  • National EU, and International Jurisprudence;
  • Medical devices;
  • Borderline cases: pharmaceuticals/food/cosmetics/chemicals
  • Patents /Trademarks;
  • Health Technology Assessment and pricing/reimbursement;
  • Digital health/Big data;

All contributions will be subject to double blind peer-review before acceptance for publication and are required to conform to the author guidelines.

We are looking forward to receive and review the first submissions!

Best wishes/ Timo

PhRMA, Marathon Is Why You Can’t Have Nice Things

Yesterday, the FDA approved a steroid, deflazacort, for the treatment of Duchenne Muscular Dystrophy (DMD).  DMD is a rare, heartbreaking, and ultimately fatal genetic disease with few if any real treatments, and the steroid may be helpful to patients.  Deflazacort’s sponsor, Marathon, has offered the drug at a list price of $89,000 per year.  High, but actually much lower than the typical prices charged for new orphan drugs, which can easily run to $300,000 or more per year.

Here’s the big problem: deflazacort isn’t really a new drug.  As the Wall Street Journal and Endpoints have pointed out, the drug is approved in many other countries, and its list price is about $1,000-$1,600 in Canada and the UK.  Patients have been importing the drug and accessing it since the 1990s.  Now, patients will pay many times those prices for the same product they had already been purchasing.

But the drug had not previously been approved in the United States, and surely Marathon conducted new clinical trials to demonstrate the drug’s benefit?  Not clearly.  Marathon mostly relied on clinical trial data from the 1990s that had not been fully analyzed.  In return, Marathon gets 1) a seven-year market exclusivity period for the drug (as required by the Orphan Drug Act) and 2) a valuable priority review voucher (as required by law for rare pediatric diseases).

This is not acceptable.  Full stop.  It is the worst sort of gaming that other companies have engaged in over the years.  And at a time when the drug industry is under fire for its high prices, PhRMA cannot afford to have its members (of which Marathon is one) acting this way.  If PhRMA and patient groups funded by pharmaceutical companies are serious about drug pricing, here are three things they should do/encourage right now:

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Looking Forward: The Next Generation of Biosimilars

Looking Forward: The Next Generation of Bio
similars

February 7, 2017 12:00 PM

Description

Many of today’s important medications are biological products made from living organisms, manufactured through biotechnology, derived from natural sources, or produced synthetically. Biosimilars are a type of biological product approved by FDA on the basis of being highly similar to an already approved biological reference product, like a generic drug.

This panel of experts will discuss the current state of biosimilars in the healthcare ecosystem and what comes next from a technical and legal perspective. Topics include how the next generation of biosimilars can improve patient access to standard-of-care therapies, the concept of “biobetters,” economic and intellectual property considerations, and policy approaches to support existing and future biosimilars.

Panelists

This event is free and open to the public. Lunch will be provided.

Sponsored by the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School.

National Survey Suggests that Off-Label Status is Material to Informed Consent

As many readers of this blog know, the FDA requires that, prior to entering the market, companies prove safety and efficacy for each intended use of their products, but physicians are then free to prescribe the products for any other uses.  (Companies are not allowed to promote off-label uses however.)

A recent national survey by Consumer Reports includes two interesting findings:

  1. About two-thirds (63%) of Americans “would not take a doctor prescribed medication that has been approved by the FDA, but not for their specific condition.”
  2. Almost all Americans (94%) “say they have never been told by a physician that a medication they were taking was not approved by the FDA for their condition.”

Patients are right to be skeptical of off-label uses, though they may not appreciate just how common they are.  In fact, most off-label use is unsupported by scientific evidence as to safety and efficacy.  A new report by the FDA illustrates several off-label uses that were subjected to rigorous clinical trials and turned out to be ineffective or dangerous.   For example, Aliskiren is approved for treatment of hypertension and was used off-label for prevention of congestive heart failure (CHF) complications.  A large trial showed that, although it did not significantly improve CHF mortality, it did significantly increase rates of kidney failure for CHF patients.  We do not know how many other off-label uses would fail if similarly tested.   Continue reading

An Alternative Point of View: The Societal Benefits of the Amgen v. Regeneron Injunction

Special Guest Post by Beau H. Miller, Jefferies

This article was originally published as part of research issued by the Jefferies Biotechnology Equity Research group.

Since the Amgen v. Regeneron ruling that resulted in Judge Robinson of the District Court of Delaware granting a permanent injunction removing Regeneron’s Praluent from the market (pending appeal), there have been a number of media and twitter commentaries providing their insights on the case.

These reactions have overall been of the opinion that it is a “shocking” precedent for a judge to remove an FDA-approved drug from the market for patent infringement – a remedy which patent owners, even pharmaceutical manufacturers, are justly able to obtain under the circumstances outlined in eBay v. MercExchange.

Given this, there are a few facts that have been overlooked in these reviews that lead us to disagree with some of the conclusions they reach, and also which led us to anticipate an injunction in this case. Continue reading

Looking Forward: The Next Generation of Biosimilars

Looking Forward: The Next Generation of Bio
similars

February 7, 2017 12:00 PM

Description

Many of today’s important medications are biological products made from living organisms, manufactured through biotechnology, derived from natural sources, or produced synthetically. Biosimilars are a type of biological product approved by FDA on the basis of being highly similar to an already approved biological reference product, like a generic drug.

This panel of experts will discuss the current state of biosimilars in the healthcare ecosystem and what comes next from a technical and legal perspective. Topics include how the next generation of biosimilars can improve patient access to standard-of-care therapies, the concept of “biobetters,” economic and intellectual property considerations, and policy approaches to support existing and future biosimilars.

Panelists

This event is free and open to the public. Lunch will be provided.

Sponsored by the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School.

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

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. Continue reading

Preventing a post-antibiotic world

Nick Bagley and I have an op-ed in today’s New York Times calling for serious economic incentives for antibiotics, delinking revenues from sales volumes with a $4 billion prize system.

From the piece:

On Friday, the Centers for Disease Control and Prevention released a disturbing report about the death of an elderly woman in Washoe County, Nev. What killed her wasn’t heart disease, cancer or pneumonia. What killed her were bacteria that were resistant to every antibiotic doctors could throw at them.

This anonymous woman is only the latest casualty in a war against antibiotic-resistant bacteria — a war that we are losing. Although most bacteria die when they encounter an antibiotic, a few hardy bugs survive. Through repeated exposure, those tough bacteria proliferate, spreading resistance genes through the bacterial population. That’s the curse of antibiotics: The more they’re used, the worse they get, especially when they’re used carelessly. […]

Read more here.

Over Before It Started: CMS Abandons New Payment Pilot

By Zack Buck

With little fanfare, last month, the Centers for Medicare and Medicaid Services (CMS) abandoned its proposal to begin a payment pilot in which Medicare Part B would change the way it pays for pharmaceutical drugs.  As I blogged about last March, under the proposed pilot, providers’ payments would be changed from the Average Sales Price (ASP) plus 6 percent of drugs’ costs (ASP+6), to ASP plus 2.5 percent of the drugs’ costs plus a flat fee per drug per day (of $16.80). This new proposed pilot would have been time-limited, and would have allowed officials to observe the effects of such a reimbursement change on prescribing patterns in an effort to cut Medicare’s substantial drug costs.

Following an outburst of negative reaction from Medicare’s providers, the pharmaceutical industry, and Congress (including the new nominee to be Secretary of Health and Human Services Tom Price), CMS announced in December that more than 1300 public comments were submitted in reaction to the proposal.  And following November’s election and the public comments shared with CMS, the agency announced that it had decided that “the complexity of the issues and the limited time available led to the decision not to finalize the rule at this time.”

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PFC Spotlight Series: Faculty Affiliate Ameet Sarpatwari

Learn more about the Petrie-Flom Center’s work through our Spotlight Series, which features interviews with Student and Academic Fellow alumni, as well as current Faculty Affiliates.

sarpatwari_peopleThis week’s post features Ameet Sarpatwari, J.D., Ph.D., who is an Instructor in Medicine at Harvard Medical School, an Associate Epidemiologist at Brigham and Women’s Hospital, and Assistant Director of the Program On Regulation, Therapeutics, And Law (PORTAL). His research draws upon his interdisciplinary training as an epidemiologist and lawyer and focuses on the effects of laws and regulations on therapeutic development, approval, use, and related public health outcomes.

Read the article to learn more about his contributions to the Center and its mission!

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

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. 

Agenda

8:30 – 9:00am, Registration

A continental breakfast will be available.

9:00 – 9:05am, Welcome Remarks

  • I. Glenn Cohen, Professor of Law and Faculty Director, Petrie-Flom Center, Harvard Law School
  • Holly Fernandez Lynch, Executive Director, Petrie-Flom Center and Faculty, Center for Bioethics, Harvard Medical School

9:05 – 10:30am: The End of ObamaCare? Health Care Reform Under A New Administration

  • Joseph R. Antos, Wilson H. Taylor Scholar in Health Care and Retirement Policy, American Enterprise Institute
  • David Blumenthal, President, The Commonwealth Fund
  • Michael K. Gusmano, Research Scholar, The Hastings Center
  • John McDonough, Professor of the Practice of Public Health, Director of the Center for Executive and Continuing Professional Education, Harvard T. H. Chan School of Public Health
  • Abigail R. Moncrieff, Associate Professor of Law and Peter Paul Career Development Professor, Boston University School of Law
  • Moderator: Einer Elhauge, Caroll and Milton Petrie Professor of Law and Founding Faculty Director, Petrie-Flom Center, Harvard Law School

10:30 – 10:45am, Break

10:45 – 11:10am, Precision Medicine Initiative/Cancer Moonshot

11:10 – 11:35am, Common Rule Update

  • Holly Fernandez Lynch, Executive Director, Petrie-Flom Center and Faculty, Center for Bioethics, Harvard Medical School

11:35am – 12:00pm, Clinical Trial Data Sharing

  • TBD, MRCT Center at Harvard

12:00 – 12:25pm, All-Payer Claims Databases

  • Gregory D. Curfman, Editor-in-Chief, Harvard Health Publications, Harvard Medical School

12:25 – 1:00pm, Lunch

Lunch will be provided.

1:00 – 1:25pm, Defining Death, Aid in Dying, and Family Rights

  • Paul Ford, Lecturer, Harvard Medical School, Winter 2017; Director, NeuroEthics Program, Cleveland Clinic; Director of Education, Department of Bioethics, Cleveland Clinic; Associate Professor, CCF Lerner College of Medicine of CWRU

1:25 – 1:50pm, Patient Advocacy, FDA, and Right to Try

  • Jerry Avorn, Professor of Medicine, Harvard Medical School and Chief of the Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital

1:50 – 2:15pm, Drug Pricing and Cost

  • Ameet Sarpatwari, Instructor in Medicine, Harvard Medical School and Brigham and Women’s Hospital

2:15 – 2:40pm, Health IP

2:40 – 2:55pm, Break

2:55 – 3:20pm, Women’s Health

  • Aziza Ahmed, Professor of Law, Northeastern University School of Law

3:20 – 3:45pm, Reproductive Technology and Regulatory Oversight

  • I. Glenn Cohen, Professor of Law and Faculty Director, Petrie-Flom Center, Harvard Law School

3:45 – 4:10pm, Legal Responses to Zika

  • George Annas, William Fairfield Warren Distinguished Professor and Chair of the Department of Health Law, Bioethics & Human Rights, Boston University School of Public Health; Professor in the Boston University School of Medicine, and School of Law

4:10 – 4:35pm, Flint, Water Safety, and Public Health Infrastructure

  • Wendy Parmet, Matthews Distinguished University Professor of Law, Director of the Center for Health Policy and Law, and Associate Dean for Interdisciplinary Education and Research Support; Professor of Public Policy and Urban Affairs, Northeastern University School of Public Policy and Urban Affairs

4:35 – 5:00pm, Concussion Litigation and Legislation in Sports

  • Christopher Deubert, Senior Law and Ethics Associate, Petrie-Flom Center Law and Ethics Initiative, Football Players Health Study at Harvard University

5:00pm, Adjourn

Learn More

How did our prognosticators do in predicting health law and policy developments they expected in 2016? Check out videos of all of the presentations at the 4th Annual Health Law Year in P/Review event, held in January 2016, and find out!

Register Now!

This event is free and open to the public, but seating is limited and registration is required. Register now!

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. 

The Best-Laid Plans For Health Care

This new post by Petrie-Flom’s Faculty Director I. Glenn Cohen appears on the Health Affairs Blog as the first entry in a series that will stem from our Fifth Annual Health Law Year in P/Review event to be held at Harvard Law School on Monday, January 23, 2017.

“The best-laid plans of mice and men often go awry.” This phrase, adapted from the 1785 Robert Burns Poem “To a Mouse” and made as the source of the title of a Steinbeck novella, may become the mantra for health policy in 2017.

The Affordable Care Act (ACA) was the largest and most ambitious alteration to American health policy in a generation. By the middle of 2016, it appeared to be largely “settling into place,” and the quartet of Supreme Court encounters with the law have by now been largely resolved. The Constitutional commerce and taxation clause challenges of NFIB v. Sebelius have been decided, with the Court weakening Medicaid expansion and causing other problems, albeit not ones that threatened the vitality of the overarching statutory scheme due to preservation of the individual mandate.

The decision in King v. Burwell left funding for the insurance Exchanges intact. Controversy over the contraceptive coverage requirements stemming from the Act remains, with the Court punting on the extent to which its analysis from Burwell v. Hobby Lobby ought to apply to challenges raised by other types of objectors in Zubik v. Burwell, leaving the litigants with a strange “Can’t you guys just work this out on remand?” sort of resolution. […]

Read the full post here!

Let’s All Worry About The Effects of Patent Injunctions Against Drug Manufacturers

Yesterday, a federal district judge made an important ruling in the ongoing patent dispute between Amgen’s cholesterol-lowering drug Repatha and Sanofi and Regeneron’s drug, Praluent.  Early in 2016, Amgen’s patents covering the products had been found both valid and infringed, and now Judge Sue Robinson has granted Amgen’s request for an injunction against Sanofi and Regeneron, blocking the two companies from selling Praluent.  (The injunction takes effect in 30 days, giving the companies time to appeal.)

This is very strange.  Let’s be clear: Judge Robinson looked at a situation involving two competing, chemically distinct (though similar) drugs for the same condition and opted to kick one of them off the market, putting Amgen in a monopoly position and taking some number of patients off of the drug they’ve been taking.  As far as Pharma Policy Twitter (h/t Forbes’ always-excellent Matthew Herper) can tell, an injunction of this type happens about once a decade – in 2008 with Amgen and Hoffman-LaRoche regarding an EPO product, and in 1996 with Novo Nordisk and Genentech over hGH products.  (Please send along other examples, if you have them!)

A number of commentators have already weighed in on Judge Robinson’s order, with Professor Jake Sherkow providing a particularly thoughtful tweetstorm on the subject. I largely agree with Professor Sherkow’s analysis, but I want to emphasize two aspects of the case that have not yet received sufficient attention: the first is the decision to ask for the injunction, and the second is the practical effect the injunction will have on patients, on the market, and on the gathering of information about PCSK9 products going forward.

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

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. Continue reading

LIVE ONLINE TODAY @ NOON: President-Elect Trump’s Health Policy Agenda: Priorities, Strategies, and Predictions

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Webinar: President-Elect Trump’s Health Policy Agenda: Priorities, Strategies, and Predictions

Monday, December 19, 2016, 12:00 – 1:00pm

WATCH LIVE ONLINE!: http://petrieflom.law.harvard.edu/events/details/president-elect-trumps-health-policy-agenda

Submit your questions to the panelists via Twitter @PetrieFlom.

Please join the Petrie-Flom Center for a live webinar to address what health care reform may look like under the new administration. Expert panelists will address the future of the Affordable Care Act under a “repeal and replace” strategy, alternative approaches to insurance coverage and access to care, the problem of high drug prices, innovation policy, support for scientific research, and other topics. The panel will discuss opportunities and obstacles relevant to President-elect Trump’s proposals, as well as hopes and concerns for health policy over the next four years. Webinar participants will have the opportunity to submit questions to the panelists for discussion.

Panelists

  • Joseph R. Antos, Wilson H. Taylor Scholar in Health Care and Retirement Policy, American Enterprise Institute
  • Lanhee J. Chen, David and Diane Steffy Research Fellow, Hoover Institution; Director of Domestic Policy Studies and Lecturer, Public Policy Program; affiliate, Freeman Spogli Institute for International Studies, Stanford University
  • Douglas Holtz-Eakin, President, American Action Forum
  • Moderator:Gregory Curfman, Editor-in-Chief, Harvard Health Publications

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Patients in Research: What the Professionals Don’t Get

Guest Post by Rebecca Dresser

When scientists and doctors get together to talk about accelerating medical advances, someone inevitably brings up the need for more research subjects.  Not enough patients are participating in clinical trials, experts complain.  If more patients were part of medical studies, we could make more progress in treating disease and improving human lives.

Why do relatively few patients join studies?  The National Academy of Sciences and similar groups blame the problem on regulatory impediments and an inadequate supply of physician-researchers willing to ask patients to enroll in trials.  They want more funding to support physicians’ research efforts, and they want review committees like IRBs to streamline their work.

But these measures won’t be enough to fix the problem.  To really understand why patients don’t enroll in trials, and why many who do enroll drop out early, you have to ask them. I know, because I was one of the patients who turned down a trial.

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Unified Patents and Brexit: Britain’s Back on Board

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?

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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