Are The FDA’s New Definitions And Labeling Requirements Good For Us, Or Just Empty Calories?

By Diana R. H. Winters

[Crossposted from the Health Affairs Blog]

The Food and Drug Administration (FDA) has recently taken three steps toward providing consumers with more and better information about food products that the agency regulates. First, in response to several citizen petitions, the agency requested comments on the use of the term “natural” on food labeling. Second, the agency issued a statement in early May indicating that “in the near future” it planned to solicit comments reevaluating how nutrient content claims are regulated — including the term “healthy.” And third, the agency issued a final rule on an updated Nutrition Facts label, with which large companies must comply by July 2018.

With each of these actions the FDA is attempting to ensure that information provided to consumers by food manufacturers comports with the latest scientific understanding about food components. Indeed, the updated nutrition facts label will provide important information and potentially allow consumers to make more informed choices about what they eat. The agency, however, has set itself a far trickier task in defining words such as “natural” and “healthy.”

Act Naturally

In the past, the FDA has repeatedly declined to define the term “natural.” The Nutrition Labeling and Education Act (NLEA) of 1990 required the FDA to standardize definitions for nutrient content claims, like “fat free” or “high in fiber,” and to limit the use of health claims, like “heart healthy” (21 U.S.C. §§ 343(r)(1)(A), (B)). The word “natural,” however, does not fit into either of these categories.

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What do doctors know about FDA drug approval standards and the breakthrough therapy designation? Less than we’d hope.

By Dalia Deak

A study published this week in JAMA examined how much physicians know about FDA approval standards for new drugs and the breakthrough therapy designation. The investigators found major gaps in understanding with regard to both issues, despite intuitive beliefs to the contrary.

For the study, Kesselheim et al. conducted a national survey of board-certified internists and specialists. They selected a random sample of 300 clinically active internists and 900 specialists in endocrinology, hematology, and infectious diseases from the American Board of Internal Medicine’s diplomate list. Of the 1,148 physicians contacted, 692 physicians, or 60%, responded.

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Regulating genetically modified mosquitoes

By Dalia Deak

Fears of spreading zika virus have renewed interest in the use of genetically modified mosquitoes to suppress disease, with recent attention focused on the UK firm Oxitec. Last week, the developing public health crisis around zika prompted the federal government to tentatively clear a small-scale field test for the first time in the United States, pending a public comment process on a draft environmental assessment submitted by Oxitec. It should be noted that a final approval for the trial will not be made until the FDA completes the public comment process.

The genetically modified insects, which are male Aedes aegypti mosquitoes, are designed to breed with the female Aedes aegypti mosquito (primarily responsible for transmitting zika, dengue fever, and other diseases) and contain a gene lethal to their offspring. The female mosquitoes lay eggs but the larvae die well before adulthood. Oxitec claims that recent tests have shown up to a 90% decrease in the population of the Aedes aegypti mosquito, with a recent test in Piracicaba (~100 miles from Sao Paulo in Brazil) showing an 82% decline. Tests have also been conducted in the Cayman Islands and Malaysia.

In the United States, Oxitec is in the process of waiting for FDA approval to conduct trials in the Florida Keys. However, this is relatively unclear and uncharted territory for the federal government in terms of what group should be responsible for the review, and the decision for the CVM jurisdiction in this case remains hotly debated. Jurisdictional debate exists between the U.S. Department of Agriculture and the FDA’s Center for Drug Evaluation and Research (CDER) and Center for Veterinary Medicine (CVM).

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Jacobus and Catalyst Continue to Race for Approval of LEMS Drug

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

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:” 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

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.

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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.

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Tomorrow (1/29): A “Natural” Experiment: Consumer Confusion and Food Claims, a lecture by Efthimios Parasidis

A “Natural” Experiment: Consumer Confusion and Food Claims, a lecture by Efthimios Parasidis

Thursday, January 29, 2015, 12: 00 PM

Wasserstein Hall, Milstein West B                               Harvard Law School, 1585 Massachusetts Ave., Cambridge, MA [Map]

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

Efthimios Parasidis is Associate Professor at The Ohio State University Moritz College of Law and holds a joint appointment with the College of Public Health. He is an inaugural member of College of Medicine’s Center for Bioethics and Medical Humanities. His scholarship focuses on the regulation of medical products and human subjects research, the interplay between health law and intellectual property, and the application of health information technology to public health policy. He has published in leading law reviews and health policy journals, is co-authoring a casebook, and has a book under contract with Oxford University Press. The Greenwall Foundation awarded Professor Parasidis a Faculty Scholar in Bioethics fellowship for 2014-2017.

The lecture will be followed by an audience question and answer session moderated by Jacob Gersen, Professor of Law at Harvard Law School and Director of the Food Law Lab.

Cosponsored by the Food Law Lab and the Harvard Food Law and Policy Clinic at Harvard Law School.

European Responses to the Ebola Crisis: Initiatives at the European Medicines Agency (EMA)

By Timo Minssen

The current Ebola outbreak already attracted much attention on “Bill of Health” resulting in some excellent blogs on a horrible topic.

While it is evident that the current health crisis requires both immediate responses and more sustainable changes in health care policy, research and regulation, medicines regulators are collaborating internationally to find innovative solutions enhancing evaluation of and access to potential new medicines to fight Ebola outbreaks. In a statement announced by the International Coalition of Medicines Regulatory Authorities (ICMRA) in September 2014, regulators around the world led by the FDA and the EMA have vowed to collaborate in supporting accelerated evaluation of experimental new drugs to treat Ebola virus infections and say they will encourage submission of regulatory dossiers. This clearly backs up the World Health Organization’s (WHO) decision to test experimental Ebola treatments in infected patients in the current outbreak region in West Africa and to speed up the development of vaccines.

In the following I would like to summarize and discuss some of the recent European responses to the current crisis starting with an overview on recent initiatives at the EMA.

Like its US counterpart, the EMA leads a close and consistent dialogue with public and private developers of Ebola products and spends much effort in reviewing available information on the various experimental Ebola treatments currently under development. These experimental drugs range from experimental antivirals or vaccines based on the adenovirus or stomatitis vaccine to experimental therapies based on mono- and polyclonal antibody technologies. One of these unapproved antibody combination drugs – MAPP Biologicals’  ZMapp – has already been used in some care workers affected by Ebola. Other experimental drugs that are currently reviewed by the EMA include Biocryst’s BCX 4430, Fab’entech’s Hyperimmune horse sera, Sarepta’s AVI-7537, Toyama Chemicals and MediVector’s Favipiravir and Tekmira’s TKM-Ebola.

Other companies such as Bavarian Nordic  and the Russian Mikrogen are close to follow.

In addition to monitoring experimental drugs and enhancing global collaboration, the European Medicines Agency has like the FDA initiated several activities in order to support and speed up the development of these drugs towards market approval.  Continue reading

Upcoming Event: Emerging Issues and New Frontiers in FDA Regulation

lab_colored_beakers_slideEmerging Issues and New Frontiers for FDA Regulation

October 20, 2014 8:00 AM – 5:00 PM

Alston & Bird, The Atlantic Building, 950 F Street, NW, Washington, DC 20004-1404

Registration is now open online. A limited number of free seats are available to Harvard affiliates. For more information or to request a seat, please email us at petrie-flom@law.harvard.edu by October 7th.

Please join the Food and Drug Law Institute and the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School for an academic symposium on cutting-edge legal and regulatory issues facing FDA.  Leading academics will present papers on mobile health, stem cells, personalized medicine, and other novel medical product issues, as well as food regulation.  Papers will be available to registered attendees in advance, and will be published in an upcoming issue of the Food and Drug Law Journal.

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10/20/14: Emerging Issues and New Frontiers for FDA Regulation

lab_colored_beakers_slideEmerging Issues and New Frontiers for FDA Regulation

October 20, 2014 8:00 AM – 5:00 PM

Alston & Bird, The Atlantic Building, 950 F Street, NW, Washington, DC 20004-1404

Symposium co-sponsored by the Food and Drug Law Institute (FDLI) and the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School.

Please join the Food and Drug Law Institute and the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School for an academic symposium on cutting-edge legal and regulatory issues facing FDA.  Leading academics will present papers on mobile health, stem cells, personalized medicine, and other novel medical product issues, as well as food regulation.  Papers will be available to registered attendees in advance, and will be published in an upcoming issue of the Food and Drug Law Journal.

Registration is now open online. A limited number of free seats are available to Harvard affiliates. For more information or to request a seat, please email us at petrie-flom@law.harvard.edu.

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House Hearing on Regulation of Laboratory-Developed Tests Displays More Consensus Than Disagreement

The Health Subcommittee of the House Energy & Commerce Committee held a hearing last week on the FDA’s proposed draft guidance regarding laboratory-developed tests (LDTs), as part of its “21st Century Cures” initiative. The hearing, which can be viewed online (here and here), featured representatives from the FDA, industry, and research organizations. And although the various panelists offered differing views on the propriety of the FDA’s decision to begin exercising its regulatory authority over LDTs, there seemed to be more agreement than disagreement among the panelists.

Most interestingly, as Representative Henry Waxman pointed out toward the end of the hearing, “[no]body on the panel [is] arguing that there shouldn’t be a very careful scrutiny of these tests. It seems like the question is who should do it: CLIA or the FDA.” Representative Waxman’s subsequent colloquy with Harvard Medical School Professor Christopher Newton-Cheh on this point particularly helped to differentiate the historical roles of CMS and the FDA in this space. But even those panelists who opposed the FDA’s involvement seemed supportive of expanding CMS’ authority under CLIA to conduct clinical validity analyses. (Anyone interested in the administrative law aspects of this issue should know that problems of shared regulatory jurisdiction have recently received increased scholarly attention, with Jody Freeman and Jim Rossi providing a particularly thorough treatment of the issue in their recent article, Agency Coordination in Shared Regulatory Space.)  Continue reading

Questioning Quorn

By Diana R. H. Winters
[Cross-posted on HealthLawProfBlog.]

There was an article a couple of weeks ago in the New York Times about “engineered foods,” and how “a handful of high-tech start-ups are out to revolutionize the food system by engineering ‘meat’ and ‘eggs’ from pulverized plant compounds or cultured snippets of animal tissue.”   The author discussed some of the business models, interviewed some of the entrepreneurs, and contemplated some of the implications of “Food 2.0.” The concerns she noted involved the nutritional impact of these foods, and the possibility of resource-intensive production.

But what sort of screening will these food products go through before they enter the food supply? How will FDA vet these new foodstuffs for toxicity or allergenic properties? It won’t. Manufacturers can self-affirm that food products are safe, based on their own studies, and market them on that basis with no prior FDA approval. This is the GRAS (generally recognized as safe) route to marketability. A manufacturer could also choose to submit to the formal food additive approval process, which is extremely time-consuming, but considering the breadth of the GRAS exception, they probably won’t. Many more GRAS notifications are filed per year than food additive petitions.  Continue reading

Public Health Trumps Corporate Speech

By David Orentlicher
[Ed Note: Cross-posted at HealthLawProfBlog.]

Reversing its previous deference to corporate speech interests, the U.S. Court of Appeals for the D.C. Circuit came down in favor of consumer protection in a July 29 decision. In American Meat Institute v. U.S. Dept. of Agriculture, the court upheld a federal government regulation requiring meat companies to disclose the countries of origin for their products. If your beef comes from Argentina or Canada, you will know that from its label.

More importantly, the court gave the Food and Drug Administration greater freedom to reduce tobacco use in the United States. In explaining its reasoning, the court repudiated the logic of an earlier decision by the court that rejected the FDA’s graphic warnings for cigarette packs. According to the meat labeling opinion, the cigarette warning decision did not allow sufficient leeway for the government to mandate warnings or other informational disclosures to consumers.

Perhaps the U.S. Supreme Court will restore the D.C. Circuit’s previous balance, but for now, the tide has turned in favor of the public’s health.

The Revival of Phage Therapy to Fight Antimicrobial Resistance – Part I: What are the legal implications?

Last week I blogged about recent publications concerning the global battle against anti-microbial resistance (AMR). I did not mention a recent paper published in the June 2014 issue of Nature, which describes how European and U.S. researchers and authorities are increasingly considering clinical research in unconventional areas to fight AMR. The news-report “Phage therapy gets revitalized” by Sara Reardon concentrates on the use of viruses (bacteriophages) to battle bacteria. The idea is not new, but apart from some applications in the former Soviet Union, it never was established as a major research area elsewhere. In particular the paper examines the European Phagoburn project, which is the first large, multi-centre clinical trial of phage therapy for human infections, funded by the European Commission. It involves a phase I-II trial of using viruses for the treatment of bacterial infection following burns. The European Union (EU) is contributing €3.8 million (US$5.2 million) to the Phagoburn study demonstrating that it is taking the approach seriously. Meanwhile, the US National Institute of Allergy and Infectious Diseases announced in March 2014  that it regards phage therapy as one of seven key areas in its strategy to fight antibiotic resistance.

So far Western practice has concentrated on treating complex or unidentified infections with broad-spectrum antibiotics. These antibiotics would typically eliminate multiple types of bacteria, including those who have beneficial effects to the human organism. Despite resulting in direct negative consequences for patients, e.g. gastrointestinal disorders, these “atomic bomb” approaches can result in biological niches where resistant “bad bugs” can prosper. This is the reason why scientists are turning towards more targeted approaches. This is where phage therapy comes into play. Like “guided missiles”, phage-therapy has the ability to kill just species of bacteria or strain. Quoting the US virologist Ryland Young and the head of the scientific council at the Eliava Institute in Tblisi (Georgia), Mzia Kutateladze, the Nature report explains how nature offers an almost unlimited source of different phages and that so far no identical phages have ever been found. For this reason it is fairly simple to identify a particular phage for a bacterial target. If the bacterium should become resistant against that particular phage, researchers would modify the viral cocktails that are used for treatment by adding or substituting phages. At the Eliava Institute such updates occur – according to the report – approximately every 8 months and the scientists would not be fully aware of the precise combination of phages in the cocktail.

In light of these advantages the recent interest of US and EU stakeholders in phage therapy comes as no surprise. However, the scientific and legal challenges confronting these projects are complex. After all we are talking about viruses here, which triggers alarm bells with regard to public perception, safety concerns, and the regulation of relevant research. It also appears questionable if – or under what circumstances – regulatory authorities would be willing to grant market approval for such a rapidly changing product like in the case of e.g. influenza vaccines. Another significant problem for the development of new phage therapies, also addressed in the paper, lies in the reluctance of pharmaceutical companies to invest into the field. The potential obstacles for more private involvement in phage therapy are many and range from considerable risks of failure, reputational damage, and unforeseeable side-effects to insufficient certainty with regard to intellectual property protection and guarantees of a profit.

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Capsule Endoscopy Instead of Colonoscopy? The FDA Approves the PillCam COLON

By Jonathan J. Darrow

In January, the Food and Drug Administration (FDA) approved the use of the PillCam COLON 2 as a minimally-invasive means of viewing the colon, a development that is sure to be welcomed by U.S. patients who currently undergo an estimated 14 million colonoscopies each year.  While the approval represents a major step forward, the PillCam is unlikely to supplant current procedures just yet.

The colon has traditionally been examined via optical colonoscopy, a procedure perceived by many to be uncomfortable and embarrassing that involves insertion through the rectum of a 5-6 foot long flexible tube as part of an examination that can take 30 to 60 minutes. Air must be pumped in through the rectum in a process called “insufflation.” Sedatives and pain medication are generally used to help relieve discomfort. In contrast, the PillCam COLON contains a power source, light source, and two tiny cameras encapsulated in an easy-to-swallow pill that produces no pain or even sensation as it moves through the colon. Reflecting the absence of discomfort, one report from a clinical researcher noted that a few patients have insisted on X-rays to confirm that the device had passed in their stool (FDA Consumer). The pill takes about 30,000 pictures before passing naturally from the body, which usually occurs before the end of its 10-hour battery life.

The safety record of capsule endoscopy, the category to which the PillCam COLON belongs, so far appears to compare favorably with the alternatives. Capsule endoscopy may be less likely to produce accidental colonic perforations or other serious complications, which occur in less than 1% of traditional colonoscopies despite the best efforts of the treating physician. Tears of the colon wall can in turn “rapidly progress to peritonitis and sepsis, carrying significant morbidity and mortality.” (Adam J. Hanson et al., Laparoscopic Repair of Colonoscopic Perforations: Indications and Guidelines, 11 J. Gastrointest. Surg. 655, 655 (2007)). Splenic injury or other serious complications also occur rarely with optical colonoscopies. Unlike “virtual colonoscopy,” which uses computed tomography (CT) to peer into the body, capsule endoscopy does not involve bombarding the body with radiation. A leading study published in the New England Journal of Medicine reported no serious adverse events among 320 subjects given the PillCam COLON, and concluded that use of the device was “a safe method of visualizing the colonic mucosa through colon fluids without the need for sedation or insufflation.” Continue reading

The FDA Strikes Again: Its ban on home testing kits is, as usual, likely to do more harm than good

Cross-post from PointOfLaw.

Richard A. Epstein is a professor of law at NYU Law School, a Senior Fellow at the Hoover Institution, a Senior Lecturer at the University of Chicago and a visiting scholar with the Manhattan Institute’s Center for Legal Policy. His forthcoming book is “The Classical Liberal Constitution,” from Harvard University Press.

On November 22, 2013, the Food and Drug Administration flexed its regulatory muscle by sending a warning letter to a genetic-testing company that goes under the stylish name of 23andme. The object of FDA scorn was a diagnostic kit that the tech company, backed by among others Google and Johnson & Johnson, sold to customers for $99. The kit contained an all-purpose saliva-based test that could give customers information about some 240 genetic traits, which relate to a wide range of genetic traits and disease conditions. The FDA warning letter chastised 23andme in no uncertain terms for being noncooperative and nonresponsive over a five-year period in supplying information that the FDA wanted to evaluate its product as a Type III device under the Medical Devices Act.

Legal Regulation of 23andme

There is no doubt that the FDA is on solid legal ground. This case is not like the processes involved in Regenerative Sciences, LLC v. United States, where the FDA asserted that physicians’ use of certain stem-cell procedures for joint disease involved the use of a drug that required FDA approval before it could be approved for use. In an earlier essay for the Manhattan Institute, I argued that this classification was in fact both legally incorrect and socially mischievous. In this case, the legal arguments are not available to 23andme because the current definition of “medical devices” covers not only those devices intended for use on the human body, but also those used for the diagnosis of disease. The Type III classification means that this device has to receive premarket approval from the FDA, which in turn requires that it be shown to be safe and effective for its intended use. Getting approval under this standard is arduous business, because any such approval must be for each of the tests separately. 240 tests thus require that number of approvals. The costs are prohibitive, and the delay enormous.

The FDA Warning Letter is significant both for what it says and for what it does not say. Continue reading

A “Torrent of Studies” on Direct-to-Consumer Advertising: Is FDA Shoring Up Its Defenses?

By Kate Greenwood

Cross-Posted at Health Reform Watch

At Regulatory Focus earlier this week, Alexander Gaffney wrote about what he characterized as “a torrent of studies” that FDA is conducting or has proposed conducting on prescription drug promotion, and, in particular, on direct-to-consumer advertisements.  The studies include, among others, a survey study aimed at sussing out “the influence of DTC advertising in the examination room and on the relationships between healthcare professionals and patients”, a study exploring similarities and differences in the responses of adolescents and their parents to web-based prescription drug advertising, and a study that will use eye tracking technology to collect data on the effect of distracting audio and visuals on participants’ attention to risk information. 

Gaffney speculates that “the proposed studies could indicate coming changes in FDA’s regulatory approach toward advertising[.]”  Another possibility is that the studies are part of an effort by FDA to build up the evidence base supporting its current regulatory approach.  In a Tweet commenting on Gaffney’s article, Patricia Zettler–a  Fellow at Stanford Law School’s Center for Law and the Biosciences who was formerly an Associate Chief Counsel for Drugs at FDA’s Office of Chief Counsel–asks whether the data generated by the studies could help insulate FDA from First Amendment challenges. Continue reading

Our Bodies, Our Cells: FDA Regulation of Autologous Adult Stem Cell Therapies

By Mary Ann Chirba, J.D., D.Sc., M.P.H. and Alice A. Noble, J.D., M.P.H.

Stem cells have been an endless source of fascination and controversy since Dolly the sheep was cloned in 1996. This month’s announcement of a cloned human embryo from a single skin cell [1] came on the heels of Sir John B. Gurdon and Dr. Shinya Yamanaka’s receipt of the 2012 Nobel for Physiology and Medicine for their work with induced pluripotent stem cells. Pluripotent stem cells can be embryonic or induced. Embryonic stem cells (ESCs) can generally be obtained from human embryos or by cloning embryos through somatic cell nuclear transfer (SCNT), as was done for Dolly.  Gurdon and Yamanaka demonstrated that pluripotent cells may also be formed by reprogramming adult cells to an embryonic state, resulting in induced pluripotent stem (iPS) cells without having to use eggs or cloning, or destroy embryos. However derived, pluripotent cells are capable of differentiating into virtually any cell type in the human body. This imbues them with great promise for scientific breakthroughs and medical advances, but also raises serious ethical, legal and safety concerns about their use.

Less controversial are “multipotent” adult stem cells (ASCs) which do not involve embryos or raise as many safety concerns as pluripotent cells.  ASCs are found throughout the body.  Their ability to differentiate is more limited than pluripotent cells but is vast nonetheless. The NIH’s clinicaltrials.gov site lists some 4500 ASC trials as compared with 27 for embryonic stem cells and 21 for induced pluripotent stem cells. Recent announcements of new stem cell treatments usually involve ASCs, such as last month’s news that a toddler born without a trachea received a new one made from her own adult stem cells.  It is therefore no surprise that ASCs have captured the attention of researchers, investors, physicians, patients and – increasingly – regulators, both here and abroad.

A growing number of physicians routinely offer treatments involving ASCs to their patients which can be performed in their offices.  Autologous adult stem cells, used to treat a variety of conditions, are harvested from the patient, processed, and returned to the same patient. It is no surprise that moving ASCs from laboratories to physician offices raises complex questions of law. We consider one of the more pressing ones: to what extent can the FDA regulate a physician’s ability to treat a patient with that patient’s own stem cells?  In the coming months, the D.C. Circuit Court of Appeals will hear oral arguments on this very issue in United States v. Regenerative Sciences.[2]

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