The FDA de-regulates the first genetically-engineered animal

By Joanna Sax

On November 19, 2015, the FDA de-regulated the AquAdvantage Salmon.  This salmon is genetically engineered to grow faster.  This is the first time the FDA has de-regulated a genetically engineered animal.

Let me just say from the outset that the scientific consensus is clear that genetically engineered food is as safe as conventional food.  Despite the onslaught of public outrage against GMO food, most of the main arguments against GMO food are just hype.

The genie came out of the bottle a long time ago and it’s not going back in.  This happens time and again with scientific advances.   Over the past few decades, our ability to understand, manipulate, edit, and otherwise employ the DNA of various organisms to facilitate human understanding has grown exponentially.  Efforts to resist, combat, or villain-ize the application of biotechnology to impact society might delay, but will not ultimately succeed in keeping the application of scientific discoveries at bay.

Continue reading

FDA Releases Report Detailing Problematic Laboratory-Developed Tests

Last week, the FDA issued a report presenting 20 case studies of laboratory-developed tests (LDTs) that have or may have harmed patients, in support of its ongoing efforts to impose greater regulatory oversight on LDTs.  The report came just before yet another House Energy & Commerce Committee hearing on the issue, and the timing of its release may have been motivated in part by questions FDA officials faced at the last Congressional hearing, about the existing evidence of harm to patients.  The report categorizes the 20 case studies into seven groups, organized by the primary problem posed by the LDT: those with a high degree of false positives (the test yields a positive result when the disease is not present), those with a high degree of false negatives (the test yields a negative result when the disease is present), those which yield both false positives and false negatives, those which test for a factor seemingly unrelated to the disease in question, those linked to treatments based on disproven scientific concepts, those which affirmatively undermined the drug approval process, and of course, “other.”

Continue reading

The Ongoing Push for E-Cigarette Regulations

By Katherine Kwong

Last week, the American Academy of Pediatrics publicly joined the group of advocates for federal regulations on e-cigarettes. The AAP urged the government to ban the sale of e-cigarettes to everyone under age 21 and prohibit advertising to minors, and advocated for high taxes on e-cigarette products similar to those on other tobacco products. In its announcement, the AAP cited developing brains’ vulnerability to nicotine and the potential harms to long-term health as reasons for its recommendations to keep e-cigarettes away from youths. It also recommended that smoke-free laws governing secondhand smoke explicitly include e-cigarettes, saying, “[t]he aerosol emitted from e-cigarettes is not harmless; it contains a variety of toxic chemicals, including some carcinogens and significant amounts of nicotine.”

A recently released poll found that a majority of Americans (57%) believe e-cigarettes should be regulated like tobacco products, while less than 25% of respondents felt they should not be. The Food and Drug Administration proposed e-cigarette regulations in 2014, and recently sent the regulations to the Office of Management and Budget for review. While the final form of the regulations is still unknown, the proposal banned the sale of e-cigarettes to minors and required e-cigarette labels include a list of ingredients and a disclosure that they contain nicotine.

There is growing concern about the potential health risks posed by e-cigarettes. Advocates for restrictions on e-cigarettes have long warned that unregulated e-cigarettes frequently expose users to the harmful effects of nicotine, as well as toxic chemicals such as formaldehyde, benzene, and other carcinogens. There have also been warnings about the risk that e-cigarette use may lead to greater social acceptance of smoking and higher rates of tobacco use. (Despite frequent claims that e-cigarettes may help with smoking cessation, longitudinal studies consistently find no evidence that e-cigarette use increases quitting rates.) Anecdotal evidence has linked e-cigarette use to pneumonia and other lung problems. Forty percent of e-cigarette users reported having health concerns about their use.

Continue reading

Lunch Talk & Book Launch Tomorrow, 10/28: FDA in the 21st Century

lync17118_frontBook Launch: FDA in the 21st Century
October 28, 2015 12:00 PM
Wasserstein Hall, Milstein East C
Harvard Law School, 1585 Massachusetts Ave., Cambridge, MA [Map]

In September 2015, Columbia University Press published FDA in the Twenty-First Century: The Challenges of Regulating Drugs and New Technologies, co-edited by Petrie-Flom Center Executive Director Holly Fernandez Lynch and Faculty Director I. Glenn Cohen. This edited volume stems from the Center’s 2013 annual conference, which brought together leading experts from academia, government, and private industry to evaluate the FDA and to begin charting a course for the agency’s future.

This event will provide a discussion of the book, including questions like: How is the agency faring in the 21st century? What are the greatest challenges to the FDA’s success, and what does success look like? What lessons has it learned and how can it best meet the challenges of today? Should we keep the agency we have, pull it apart, or rebuild from scratch?

Panelists: Continue reading

UDI Adoption: A Necessary Step Towards Better Care for Patients with Implanted Devices

By Dalia Deak

In the United States, though many millions of individuals live with implanted devices, it may shock you to know that it is easier to recall tainted dog food than it is to recall a faulty pacemaker. This is due in large part to the lag of the medical device world behind most other industries in the implementation of a standardized system that can uniquely identify and track medical devices as they move through the supply chain to a patient. Such an identification system has existed for most products since stores implemented the UPC and Congress mandated that drugs be labeled with the National Drug Code, both of which were introduced in the early 1970s.

To remedy this lag, Congress, in FDAAA of 2007, tasked the FDA with the creation of a unique device identification (UDI) system. In 2013, FDA published a Final Rule regarding manufacturer labeling of UDIs, to be rolled out by class in the coming years. While the establishment of such a system would certainly constitute an important step forward, another number on a label will do little to enhance patient safety on its own. Rather, the value of UDIs is in the uptake of the identifier at each point in a medical device’s life—from manufacturer to distributor to provider to patient to payer (see this report I co-authored on this very issue). Continue reading

Pre-Approval Access Symposium: Can Compassion, Business, and Medicine Coexist?

Dates: October 28 – 29, 2015
Location: The New York Academy of Sciences, 7 World Trade Center, NYC
Presented by: NYU School of Medicine and the New York Academy of Sciences
Event URL:

Explore challenges surrounding pre-approval access to investigational medicines through a series of debates featuring prominent representatives of governments, pharmaceutical companies, patient groups, NGOs, and foundations in this two-day colloquium.

Agenda topics include:

  • Perspectives from Patient Advocates on Compassionate Use and Expanded Access
  • The Case of Josh Hardy and Social Media’s Impact
  • Lessons Learned from the Ebola Virus Epidemic on Compassionate Use during a Crisis
  • Key Issues from Stakeholders’ Perspectives When Considering a Compassionate Use Request
  • Legislative or Regulatory Changes on Compassionate Use and Expanded Access

Continue reading

Does FDA Need a Dietary Supplement User Fee Act?

By Elizabeth Guo

Dietary supplements are dominating headlines these days – and not in a good way. Last Wednesday, Nevada officials found basketball star Lamar Odom unconscious at a brothel after taking cocaine along with ten pills of Reload, a sexual enhancement dietary supplement. That same week, the New England Journal of Medicine released an article finding that dietary supplements lead to roughly 23,000 emergency visits a year. Following these events, some officials have called on the Food and Drug Administration (FDA) to take a stronger role in regulating the dietary supplement industry.

Dietary supplements have had a long and storied past. As early as 1973, FDA tried to regulate dietary supplements regarding vitamin and mineral potency. The dietary supplement industry responded by challenging FDA in court, and Congress subsequently enacted the Proxmire Amendment, limiting FDA’s authority to regulate dietary supplements. However, by the 1990s, as consumers increasingly began to rely on dietary supplements, Congress passed the Dietary Supplement Health and Education Act of 1994, expanding FDA’s authority to regulate supplements by enacting special rules related to dietary supplement labeling and manufacturing.

Currently, FDA regulates dietary supplements as a special category of foods. Unlike manufacturers of over-the-counter drugs, dietary supplement manufacturers do not need to be registered with FDA and do not need list possible adverse events on supplement labeling. As Joanna Sax points out, this is a major problem because not all dietary supplements are the same. For example, certain weight loss or sexual enhancement supplements often contain chemicals associated with potentially serious side effects while other supplements containing chemicals such as Vitamin C pose less serious safety concerns.

Continue reading

Regulating Tobacco Standards: An Easy Fix in a Scientifically Uncertain Field

Last week, the New England Journal of Medicine published a study finding that smokers using reduced nicotine cigarettes smoked 30% fewer cigarettes and had reduced cravings at the end of the study compared to smokers using standard cigarettes. The lower-nicotine cigarettes had 0.4 mg of nicotine/gram compared to 15.8 mg of nicotine/gram for the standard cigarettes. Commentators were quick to point out that such studies could provide the evidence FDA needs to establish new nicotine standards for combustible cigarettes.

FDA has explicit authority from Congress to set low nicotine standards for cigarettes. The Family Smoking Prevent and Tobacco Control Act (FSPTCA) allows FDA to set “tobacco product standards,” including provisions for “nicotine yields of the product” where such standards are “appropriate for the protection of public health.” With a mountain of evidence showing that combustible cigarette addiction can lead to cancer, heart disease, and death, FDA should have no problem proving that nicotine standards for combustible cigarettes are appropriate.

FDA may have a harder task trying to show that nicotine reduction standards for other tobacco products are “appropriate for the protection of public health.” As one commentator noted, if the nicotine in combustible cigarettes declines, addicted smokers might switch to other nicotine-containing products, including smokeless-tobacco products, e-cigarettes, and e-pipes. Currently, no study has conclusively shown that smokeless tobacco products are safe or unsafe. Some studies suggest that smokeless tobacco products may lead to smoking later in life, while other studies show that smokeless tobacco may be helpful for smokers hoping to quit.

Continue reading

A Cost Conundrum for Treating Small Patient Populations

By Dalia Deak

The issue of drug pricing has been thrust center stage (again) after Turing Pharmaceuticals raised the price of daraprim from $13.50 to $750 per dose. The public issued a loud outcry, the pharmaceutical industry condemned the move, and presidential candidates are now discussing drug prices (as discussed previously on this blog). The reactions were so swift and loud that Turing eventually backed down, indicating that they will lower the drug’s price, though it is unclear by how much.

The drug in question in this debate, daraprim, is a 62-year old drug used to treat toxoplasmosis, a parasite that is particularly dangerous in infants, AIDS patients, and cancer patients. The curiosity of this case in particular is that the usual host of development incentives implicated in driving up the cost of a drug (e.g., patents, market exclusivity) was not in play. The reason Turing was able to raise the cost of daraprim is because no other generic competitor for the drug is on the market to drive down the cost. This is largely a result of the small market for daraprim, which had 13,000 prescriptions filled for it last year. This begs the questions: specifically for disease areas where populations are small, will drug prices, even for generics, remain stubbornly high?

Continue reading

Government Regulation of Commercial Speech: is Amarin Pharma’s Breakout Moment?

Government regulation of off-label promotion by pharmaceutical companies is now an important First Amendment issue. The Food and Drug Administration (FDA) has historically restricted truthful and non-misleading speech by pharmaceutical companies under the Food, Drug and Cosmetic Act (FDCA). The FDCA prohibits introducing “misbranded” products into interstate commerce. The FDA has interpreted this to prohibit pharmaceutical companies recommending uses not already approved by the agency (these uses appear in a drug’s labeling). Drugs promoted for unapproved uses may also be considered “new” drugs which require FDA approval. Pharmaceutical companies can also face liability under the False Claims Act for off-label promotion.

United States v. Caronia was the first time the FDCA’s misbranding provisions were successfully challenged under the First Amendment. In 2012, the Second Circuit held that the FDA’s regulations failed the test for commercial speech announced in Central Hudson. Namely, the Court held that restricting truthful speech did not directly advance a government interest (rather, the regulations paternalistically prevented dissemination of truthful information), and the Court held that the FDA’s regulations were more extensive than necessary. The Court did not even analyze the regulations under the test announced in Sorrell v. IMS Health, decided by the Supreme Court in 2011, which held that heightened scrutiny was warranted where restrictions are content- and speaker-based. The FDA did not seek en banc review or writ of certiorari.

Almost immediately, the case was heralded as a landmark decision that would have a profound impact on drug regulation. However, that has yet to occur. Since the case was decided the FDA has already generated large settlements with companies like Amgen for violating agency regulations on off-label promotion. That may be because of uncertainty regarding Caronia’s reach, and because for large companies a relatively cheap settlement makes more sense than risking felony indictments and exclusion from government programs. For the agency’s part, it has tried to avoid fully vetting constitutional issues surrounding its regulations. The FDA stated after Caronia that the ruling would not alter its enforcement policy. Although, the agency has stated it is developing new guidances concerning off-label promotion.

The newest development in this story came last month in Amarin vs. United States. Continue reading

NPRM Summary from HHS

As Michelle noted, the Notice of Proposed Rule Making (NPRM) on human subjects research is out after a long delay. For my (and many Bill of Health bloggers’) view about its predecessor ANPRM, you can check out our 2014 book, Human Subjects Research Regulation: Perspectives on the Future.

Here is HHS’s own summary of what has changed and what it thinks is most important:

The U.S. Department of Health and Human Services and fifteen other Federal Departments and Agencies have announced proposed revisions to modernize, strengthen, and make more effective the Federal Policy for the Protection of Human Subjects that was promulgated as a Common Rule in 1991.  A Notice of Proposed Rulemaking (NPRM) was put on public display on September 2, 2015 by the Office of the Federal Register.  The NPRM seeks comment on proposals to better protect human subjects involved in research, while facilitating valuable research and reducing burden, delay, and ambiguity for investigators. It is expected that the NPRM will be published in the Federal Register on September 8, 2015.  There are plans to release several webinars that will explain the changes proposed in the NPRM, and a town hall meeting is planned to be held in Washington, D.C. in October. Continue reading

NOW AVAILABLE: FDA in the 21st Century: Get 30% Off When You Order through the Press!

lync17118_frontJust out from Columbia University Press, FDA in the Twenty-First Century: The Challenges of Regulating Drugs and New Technologies! This volume, co-edited by Petrie-Flom Center Executive Director Holly Fernandez Lynch and Faculty Director I. Glenn Cohen, stems from the Center’s 2013 annual conference, which brought together leading experts from academia, government, and private industry to evaluate the FDA and to begin charting a course for the agency’s future.

Use promo code FDA21 and save 30% if you order now at the Columbia University Press website!

And join us at Harvard Law School on October 28 for a book launch and panel discussion featuring editors Holly Fernandez Lynch and Glenn Cohen!

New DTCA Guidance — Enough to Empower Consumers?

Bill of Health contributor Christopher T. Robertson has a new Op-Ed out in the New England Journal of Medicine:

As one of only two countries that permit direct-to-consumer advertising (DTCA) of pharmaceuticals, the United States tasks the Food and Drug Administration (FDA) with regulating that advertising to ensure that it doesn’t mislead consumers. When a drug maker publishes or broadcasts a claim that its drug has benefits in a particular disease, the FDA requires it to include information on the product’s risks as well. Since it’s not feasible for companies to include all the important information about their products in a television ad, the FDA requires them to refer viewers to more complete information, such as that in a printed magazine ad. Companies have tended to comply with this requirement by supplementing colorful, persuasive ads with one or two pages of dry text providing the required disclosures, often simply using language that the FDA has approved for other purposes, such as package inserts for prescribers. But research shows that most patients who attempt to read these disclosures find them difficult to understand, and many don’t even try to make sense of them.1 Now, the FDA is in the process of adjusting its DTCA rules, aiming to provide greater assurance that patients receive due warning of the most significant risks — but its tweaks probably don’t go far enough to really empower consumers to make smart decisions about the drugs they put into their bodies. […]

Read the full article here.

Bioethicist Art Caplan: All Hat, No Cattle—The False Hope of Right-to-Try Laws

Bill of Health contributor Arthur Caplan and Alison Bateman-House have a new piece up in Harvard Health Policy Review:

Are right-to-try laws a good idea? In 2014, they began appearing throughout the United States, first in Colorado and now in 23 states, with several more considering passing versions of this popular legislation.

At first glance these bills may seem worth enacting. Right-to-try (RTT) laws promise terminally ill patients that they can obtain experimental drugs or medical devices in their quests to save or extend their lives. These investigational medical products are still in development and have not yet met the requirements set by the Food and Drug Administration (FDA) in order to be approved for sale or use in the United States. In some cases, the products are still being tested and patient access would primarily be by enrolling in a clinical trial – something terminally ill people may be too unwell to do. Less commonly, the products have completed clinical testing and are waiting for an approval verdict from the FDA. In either case, RTT laws are about providing terminally ill patients access to unapproved drugs or devices. […]

Read the full article here.

Journal of Law and the Biosciences: Call for Harvard Student Submissions

JLB coverThe Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School collaborates with Stanford and Duke Universities to publish the Journal of Law and Biosciences  (Oxford University Press), an online, open-access, peer-reviewed journal. JLB includes a Notes & Developments section, comprised  of  brief summaries and commentary on recent legislation, regulation, and case law written by graduate students at the collaborating schools. The Petrie­Flom Center is responsible for providing the Notes & Developments for one issue per annual volume.

We are currently seeking Harvard graduate students to contribute papers to be published in JLB’s Notes & Developments section in early 2016. In previous years, Notes & Developments have been generated from scratch specifically for JLB, based on selection from submitted proposals. This year, we are taking a different approach by publishing already complete (or to-be-completed by the deadline) original student papers (such as student notes, course papers, etc.) written by graduate students from any Harvard school.  Notes & Developments are limited to 5000 words, including footnotes and references,  and  should  be  on  a  topic  of  relevance  to  law  and  the  biosciences,  in particular a topic of relatively recent concern, controversy, or change. They should focus on  describing  the  issue  at  hand,  explaining  why  it  is  relevant to scholars and practitioners, and providing analysis and questions for further consideration.

Interested students should submit their papers and CVs for consideration no later than September 7, 2015 (earlier  is  welcome). Up to four papers will be selected for publication in the New Developments section of JLB. Applicants will be notified by the end of September. Selected students will receive comments on their papers by the end of October, and will also be responsible for providing comments to the other selected students. Revisions will be due by the end of November, and final submissions to JLB will be due by the end of December 2015.

Please send all application materials, and direct all questions, to Holly Fernandez Lynch,

Bureaucracy Can Save Lives – The Legacy of Dr. Frances Kelsey

By Robert Field

What adjective would most people associate with the word “bureaucrat”? For many, it would be “inefficient,” “inept,” or “incompetent.” But another that is just as descriptive is “lifesaving.”

Dr. Frances Kelsey, who died this month at the age of 101, was celebrated as an American hero for her work as a medical officer at the Food and Drug Administration (FDA). She saved thousands of lives and prevented untold suffering by using techniques that earn bureaucrats a bad name, delay and obstruction, to keep the drug thalidomide from reaching the market in the United States in 1961.

Thalidomide is a sedative that had been approved for sale in Europe four years earlier and was prescribed for morning sickness during pregnancy. The American manufacturer, Richardson-Merrell, saw a large potential market in the United States. However, Dr. Kelsey, who was assigned to review its application for marketing approval, was troubled by questionable safety data. The law in effect in 1961 required that she issue a decision within 60 days, but she was able to buy more time by asking for additional information.

Continue reading

North, West, and the Direction of FDA Enforcement in the Social Media Age

Co-blogged with University of Arizona Fellow, Jonathan Loe

Breathlessly, many news outlets reported yesterday that Kim Kardashian West was in trouble with the FDA for misleading social media advertising of the drug Diclegis. For example, the reliably hyperbolic Daily Mail led with “Kim Kardashian slammed by FDA.” 

As followers of this blog may not know, Mrs. Kardashian West is pregnant with her second child.  Following on the disappointing news that the soon-to-be sibling of baby “North West” will not be named South, the celebrity-for-celebrity’s-sake shared a post on Instagram (and Facebook, and linked to from Twitter, naturally).  The post announced for the world that “OMG” her “#morningsickness” had benefited from a prescription of Diclegis—with “no increased risk to the baby.” The FDA issued a warning letter, because the social media post failed to communicate any risk information.

But is the FDA really concerned with people, however famous, commenting on their personal experiences with drugs?

The answer is…

Continue reading

Health Law Year in P/Review: Until Next Year

This new post by Holly F. Lynch, I. Glenn Cohen, and Gregory Curfman appears on the Health Affairs Blog as the final entry in a series stemming from the Third Annual Health Law Year in P/Review event held at Harvard Law School on Friday, January 30, 2015.

It’s been our great pleasure to collaborate with the Health Affairs Blog on this series stemming from theThird Annual Health Law Year in P/Review symposium at Harvard Law School. This annual event takes a look back over the prior year and previews the year to come with regard to hot topics in health law.

After the symposium, we asked our speakers to keep the conversation going online by expanding on their topics from different angles or by honing in on particularly intriguing features. These pieces were published on the Health Affairs Blog through the spring and into summer.

We heard more from Kevin Outterson on how to promote innovation in the development of new antibiotics, from Rachel Sachs on whether the Food and Drug Administration’s proposal to regulate laboratory-developed tests will really stifle innovation, and from Claire Laporte on the impact of recent Supreme Court decisions on bio-IP.

George Annas weighed in on the Ebola outbreak, which has already almost faded from public consciousness but offers important public health lessons, while Wendy Parmet and Andrew Sussman tackled important developments in tobacco control. […]

Read the full post here.

The FDA’s Determination On Artificial Trans Fat: A Long Time Coming

By Diana R. H. Winters

[cross-posted at Health Affairs Blog]
On June 16, 2015, the Food and Drug Administration (FDA) released its final determination withdrawing the generally recognized as safe (GRAS) designation for partially hydrogenated oils (PHOs), which are the main source of artificial trans fat in processed foods. The agency gave the food industry three years, until June 18, 2018, to phase out the use of PHOs. The FDA’s order was expected based on the agency’s tentative determination that PHOs were no longer GRAS, published in November 2013.

This action is a milestone in — although perhaps not the culmination of — the FDA’s decades-long attempt to grapple with increasing scientific recognition that trans fat poses a serious health risk to consumers. The action is also unusual, in that it is quite rare for the FDA to withdraw GRAS status from a food product, a move that most likely will mean the ingredient is no longer used in foods.

What is not unusual, unfortunately, is the lengthy timeframe of this regulatory trajectory in the context of FDA action. To be sure, this action will lead to a dramatic reduction in the use of PHOs in processed foods, which in turn, will lead Americans to eat less trans fat – a good thing. What this regulatory action does not do, however, is speak to problems with the GRAS process as a whole.

Continue reading

FDA’s Non-Response Response to My New York Times Op-Ed on Gay Blood Ban

On May 21, along with my frequent co-author Eli Adashi, I published an op-ed in the New York Times raising some questions about FDA’s proposed guidance recommending a ban on taking the blood on any man who has had sex with another man in the past year, or in other words imposing a one year celibacy requirement on gay men if they want to donate blood. This built on our critique last July in JAMA, wherein we argued that FDA’s then-lifetime ban on gay men and MSM donating blood was out of step with science and the practice of our peer countries, as well as potentially unconstitutional.

Thanks to our work, and a concerted effort by public health, medical, and gay rights groups, FDA has finally moved off of that prior policy and recognized that it was unjustified, and discriminatory.

Just to put this in context It took more than 30 years to convince FDA that it was problematic to ban blood donation for a lifetime any man who ever had sex with another man, even if both have repeatedly tested negative for HIV, while it imposed only a one year ban on people who had sex with individuals known to be HIV positive or a sex worker. FDA is appropriately a conservative agency, but on this issue of the lifetime ban its willingness to listen and reconsider has gone beyond conservatism to the point of lunacy. [By the way to be clear, I *love* FDA. I represented them while at the DOJ and have a new book coming out about FDA in the fall. You can think highly of an agency but think they have a bad track record on an issue. This is critique not hater-aide].

Well with that background, one should be not so quick to assume that a move to a one year ban — a de facto lifetime ban for any gay man who is sexually active, even one who is monogamously married with children — is the best policy. To put it bluntly, refusing to change a lifetime ban for such a long period makes me skeptical we should accept a “just trust us” line on their new restrictive policy.

The question we raised in our op-ed was whether FDA had adequately justified retaining a one year ban in light of the evidence from places like South Africa (with a much shorter time period ban), Italy (which does individualized risk assessment instead of stigmatizing all gay men as high risk for disease), etc.

Well FDA responded…sort of … through a NY TImes Letter To the Editor.

Here is what FDA said with my analysis in bold:

Continue reading