Simulated Side Effects: FDA Uses Novel Computer Model to Guide Kratom Policy

FDA Commissioner Scott Gottlieb issued a statement on Tuesday about the controversial plant Mitragyna speciosa, which is also known as kratom. According to Gottlieb, kratom poses deadly health risks. His conclusion is partly based on a computer model that was announced in his recent statement. The use of simulations to inform drug policy is a new development with implications that extend beyond the regulation of kratom. We currently live in the Digital Age, a period in which most information is in digital form. However, the Digital Age is rapidly evolving into an Age of Algorithms in which computer software increasingly assumes the roles of human decision makers. The FDA’s use of computer simulations to evaluate drugs is a bold first step into this new era. This essay discusses the potential risks of basing federal drug policies on computer models that have not been thoroughly explained or validated (using the kratom debate as a case study).

Kratom grows naturally in Southeast Asian countries such as Thailand and Malaysia where it has been used for centuries as a stimulant and pain reliever. In recent years, the plant has gained popularity in the United States as an alternative to illicit and prescription narcotics. Kratom advocates claim it is harmless and useful for treating pain and easing symptoms of opioid withdrawal. However, the FDA contends it has no medical use and causes serious or fatal complications. As a result, the US Drug Enforcement Agency (DEA) may categorize kratom in Schedule I, its most heavily restricted category.

Continue reading

How to “Lower Drug Prices” Without Lowering Drug Prices

Yesterday, Alex Azar was sworn in as the Secretary for the Department of Health and Human Services. A key question is whether Azar will take action against high drug prices, and if so, what he will do. At his confirmation hearing, Azar stated clearly that “drug prices are too high.” And during Azar’s swearing-in ceremony, the President stated that Azar was “going to get those prescription drug prices way down.” But I’m skeptical that Secretary Azar will do much to address the problem in the near term.

To be clear, I’m skeptical for a host of reasons, none of which are necessarily reflective of Secretary Azar. Much like health care, drug pricing is complicated. HHS should (and will) worry about potential unintended consequences of drug pricing proposals, proceeding cautiously and taking concerns seriously. HHS’ ability to act may be limited without Congressional involvement, and Congress has thus far been unable to act on this issue. Other proposals may take years to develop or implement, leaving patients without relief in the interim.

As a former President of Eli Lilly, Secretary Azar understands the drug pricing system deeply. He’s absolutely right that “there’s not one action that all of a sudden fixes this.” But if Azar is under pressure to deliver drug pricing changes in the short term, I’d expect to see focus in three main areas. Here’s the problem, though: at least two of these would not necessarily lower drug prices individually or drug spending overall. They might well increase overall spending. Importantly, that may not be a bad thing (as I’ll explain). But they won’t hurt the bottom line of the drug companies the President believes are “getting away with murder,” and they may well bolster it. The third area may lower prices – but it wouldn’t be Azar’s accomplishment.

Continue reading

“Right to Try” Does Not Help Patients

Co-Blogged by Christopher Robertson and Kelly McBride Folkers (research associate at the Division of Medical Ethics of the NYU School of Medicine)

In 2014, Arizonans overwhelmingly voted in favor of a ballot referendum that claimed to allow terminally ill patients the “right to try” experimental drugs that have not yet been approved by the Food and Drug Administration (FDA). Despite the policy’s broad support, it has yet to help a single patient in Arizona obtain an experimental drug that they couldn’t have gotten before. Thirty-seven other states have also passed right to try bills, but likewise have seen little real impact for patients.

“Right to try” has moved to the federal stage, as the U.S. Senate unanimously passed such a bill last August without even holding a hearing. The House Energy & Commerce Subcommittee on Health considered the bill in an October hearing, but it failed to garner much enthusiasm among committee members. Vice President Mike Pence has advocated for a federal right to try law, and he recently met with FDA Commissioner Scott Gottlieb and House leadership to encourage pass of the bill this year. Continue reading

Bioethicist Art Caplan: A New Mind-Body Problem

A new piece by Bill of Health contributor Arthur Caplan, with Lisa Kearns, in The Hastings Center Bioethics Forum:

Not since Rene Descartes gazed from his garret window in early 17th-century Paris and wondered whether those were men or hats and coats covering “automatic machines” he saw roaming the streets has the issue of personal identity and your cranium been of such import. Descartes feared a world that he alone occupied due to deception by the devil. Today we face a different mind-body challenge in the form of a devil we know: Italian neuroscientist Sergio Canavero. He recently announced that the first human head transplant is imminent.

For bioethicists, the moral critiques of this surgery practically write themselves: Are we merely our bodies? How can a person so ill as to wish to trade in his lifelong corporeal companion be considered competent to consent to such a drastic procedure? How can family members consent to donate a body that they could very well run into — and recognize — at the beach or gym? What if a left-handed person received a right-handed body? What if a lifelong Chicago Bears fan woke to find himself attached to the green-and-gold-tattooed torso of a former Packers fan? Would transplant recipients need to buy whole new wardrobes? Who will pay? […]

Read the full article here!

Register Now! The Policy, Politics & Law of Cancer Conference, Feb 8-9, 2018 at Yale Law School

The Opioid Crisis Requires Evidence-Based Solutions, Part II: How the President’s Commission on Combating Drug Addiction Ignored Promising Medical Treatments

Last year more than 64,000 Americans died of drug overdose, which is “now the leading cause of death” in people under 50. Opioids kill an estimated 91 Americans each day and are responsible for most drug-related deaths in the US. This public health crisis requires solutions that are supported by science and reason instead of emotion and political ideology. In Part I of this three-part series, I discuss how the President’s Commission on Combating Drug Addiction and the Opioid Crisis misinterpreted scientific studies and used data to support unfounded conclusions. In this second part of the series, I explore how the Opioid Commission ignored medical interventions that are used successfully in the U.S. and abroad. In Part III, I will discuss non-medical interventions such as drug checking and safe injection sites. The Commission’s failure to consider these options is likely driven by emotions such as fear and disgust rather than a careful review of scientific evidence.

Medical marijuana is currently accepted in 29 U.S. states and the District of Columbia. It is also permitted in at least 10 countries. However, the Opioid Commission outright rejected calls to consider the use of medical marijuana as an alternative to opioids for managing pain. Prior to the Commission’s first meeting, it solicited input from industry and members of the public on how to address the opioid crisis. In response, it received over 8,000 public comments. According to VICE News, which obtained the documents by submitting a Freedom of Information Act (FOIA) request, most comments were submitted by individuals urging the Commission to “consider medical marijuana as a solution to the opioid epidemic.” A spokesman for the Office of National Drug Control Policy, a body of the Executive Branch that provides administrative support to the Opioid Commission, reports receiving “more than 7,800 public comments relating to marijuana.” Despite these comments, in its final report, the Commission dismissed the notion that marijuana should play a role in treating chronic pain and opioid addiction. Its report cited a recent study from the American Journal of Psychiatry, which concluded that marijuana use was associated with an increased risk of opioid abuse. However, this study relied on data that was collected over twelve years ago. One of its authors, Columbia Medical School Professor Mark Olfson, told CNN that if the data were collected today, they could yield different results.

Continue reading

2017’s Word Of The Year In Health Law And Bioethics: Uncertainty

This is the first post by Carmel Shachar and I. Glenn Cohen that appears on the Health Affairs Blog in a series stemming from the Sixth Annual Health Law Year in P/Review event to be held at Harvard Law School on Tuesday, December 12, 2017.

2017 was a year of tremendous uncertainty for many areas of public policy. Health care policy was no exception, most prominently with an almost successful push by Congressional Republicans to radically revise the Affordable Care Act (ACA). Medical research and bioethics also faced uncertainty, with the struggle to ethically engage with new technologies and to better understand the boundaries around self-determination. As we look over the past year and anticipate the coming one, the overarching question remains: Is it possible to run a health law and health care system given this level of flux?

Healthcare Policy in Flux

2017 saw a new presidential administration and Congress. Seeking to capitalize on the Republican control of the White House and both Houses of Congress, Congressional Republicans sought to make good on their campaign promise to “replace and repeal” the ACA. The proposed legislation would have dramatically reshaped our health care landscape, including ending Medicaid’s financial status as an entitlement program, and undercutting the health insurance Marketplaces championed by the Obama administration. Despite the fact that the ACA is not yet a decade old, this would have been a seismic shift in the way many Americans receive their health care. […]

Read the full post here.

We will be discussing these issues and more at the Sixth Annual Health Law Year in P/Review conference, held on December 12, 2017, at Harvard Law School in Cambridge, MA. If you find these issues interesting, we invite you to join us as the event is free and open to the public (registered required). For those unable to join us in Cambridge, some of our conference presenters will participate in a blog series to follow  at the Health Affairs Blog. Stay tuned!

What does the Orphan Drug Tax Credit tell us about the Costs of Clinical Trials?

By James Love

Summary:

  • The number of patients enrolled in the trials used to support the registration of novel orphan product are significantly smaller that non-orphan products.  One measure of this is the difference in the enrollment of trials cited in the FDA drug trials snapshots.
  • Since 2015, the average number of trials cited in the FDA trials snapshots for novel drugs were 439 for orphan products, and 2,736 for non-orphans.
  • Data from the Orphan Drug Tax Credit provides insights into the costs of drug development, or more specially, the costs of the clinical trials used to support an FDA approval.
  • From 2010 to 2016, the average qualifying trial costs claimed for the orphan drug credit was $86 million to $102 million, per FDA approved orphan indication (assuming 2 to 3 year average years of lag between the credit claimed and the approval date).  Companies were able to take a credit of $43 to $51 million, on average, for each FDA approval.
  • The $86 to $102 million in pre-credit outlays is far lower than the average of $965 million on trial costs for a new drug approval, estimated by DiMasi and others in 2016.  Some of the differences are explained by the smaller trials for orphan drugs and other differences in methodologies, although both figures include the costs of failed trials and exclude pre-clinical or cost of capital costs.
  • In 2013, the last year for which we have actual rather than projected data on the credit (from the IRS Statistics of Income), the total amount of the credit from all 132 corporate tax returns that claimed the credit was just over $1 billion, nearly the same amount as the DiMasi estimate of $965 million for a single drug. But in 2013, the FDA granted 265 orphan designations and approved 33 orphan indications, including 8 novel products which were approved for an orphan drug lead indication.
  • The data from the orphan drug tax credit illustrates the large gap between the known facts about the costs for R&D for orphan drug development, and the astronomically larger R&D costs claimed by DiMasi (and frequently quoted by other researchers, policy makers and journalists) as averages that should guide policy making.
  • These data underline the need for greater transparency of R&D costs, and more sophistication and realism by policy makers regarding the costs of research and development for drugs qualifying as orphan products.
  • The data from the orphan drug tax credit also provides additional perspective on the estimates of drug development costs provided by Vinay Prasad and Sham Mailankody in their 2017 JAMA paper.

Continue reading

TOMORROW, 11/9! Book Launch: Specimen Science – Ethics and Policy Implications

Book Launch: Specimen Science: Ethics and Policy Implications
November 9, 2017 12:00 PM
Countway Library, Lahey Room
Harvard Medical School, Boston, MA

In September 2017, MIT Press will publish Specimen Science: Ethics and Policy Implications, co-edited by Holly Fernandez Lynch (outgoing Petrie-Flom Executive Director), Barbara Bierer, I. Glenn Cohen (Faculty Director), and Suzanne M. Rivera. This edited volume stems from a conference in 2015 that brought together leading experts to address key ethical and policy issues raised by genetics and other research involving human biological materials, covering the entire trajectory from specimen source to new discovery.  The conference was a collaboration between The Center for Child Health and Policy at Case Western Reserve University and University Hospitals Rainbow Babies & Children’s Hospital; the Petrie-Flom Center  for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School; the Multi-Regional Clinical Trials Center of Harvard and Brigham and Women’s Hospital; and Harvard Catalyst | The Harvard Clinical and Translational Science Center. It was supported by funding from the National Human Genome Research Institute and the Oswald DeN. Cammann Fund at Harvard University.

Continue reading

Orphan Drugs Designations and Approvals have Something to Say about Risks

This brief essay examines data from the U.S. Orphan Drug Act, including specifically the FDA designations of an indication for a drug to treat an orphan disease, and the likelihood that once the designation is made, the FDA will approve the drug for that indication. This is one empirical measure of the risks associated with the development of new drugs to treat U.S. defined orphan diseases.  Note that 75 percent of all novel cancer drugs approved in the United States from 2010 to 2016 qualified as orphan products.    The essay also reports the average time between the FDA designation and the FDA approval for orphan indications.

The main findings are that since 2010, the average time from orphan designation to approval is 5.3 years, and the likelihood of FDA approval for an orphan indication, which varies over time and across business cycles, was .22 from 1990 to 2017, and since 2010, was .25.

The essay concludes with a comparison to other studies of the risks of drug development.


On January 5, 1983, the U.S. Orphan Drug Act became law as Public Law 97-414. Over the past 34 years the Act has been amended numerous times, often extending or expanding the benefits, which currently include a 50 percent tax credit for qualifying clinical trials, exemptions or discounts on prescription drug user fees, an easier and faster path to FDA approval, and seven years of marketing exclusivity for an approved orphan indication. Continue reading

Federal “Right to Try” Legislation – Perpetuating a Misguided Skepticism Towards the FDA

Cross-posted from the CRITical Thinking blog.

By Jeanie Kim

The “right to try” (RTT) movement presents a narrative that pits patients against the FDA. Supporters of RTT, powered by the libertarian Goldwater Institute, have pushed for laws that let terminally ill patients bypass regulators to access unapproved treatments.

As of September 2017, 37 states have enacted RTT laws. Earlier this year, the Senate and the House introduced federal RTT bills, and on August 3, 2017, the Senate unanimously passed an amended RTT bill without an opportunity for debate. There is pressure on the House to follow suit, but it is unclear whether the House will consider the originally introduced RTT bill (“RTT 1.0”) or the Senate’s amended version (“RTT 2.0”), or even take up the legislation at all.

Despite the recent legislative backing, RTT is not a new concept. It is a variation on an age-old skepticism towards the FDA that has been around as long as the agency’s inception. At the core of RTT is the previously rejected, yet persistent argument that the FDA’s approval standards for safety and efficacy should not matter for terminally ill patients who have nothing to lose [1]. Continue reading

Be Very, Very Concerned About What Allergan Just Did

Yesterday, it was announced that Allergan had transferred the ownership of the patents on its billion-dollar drug Restasis, used for the treatment of chronic dry eye, to the Saint Regis Mohawk Tribe. The Tribe then exclusively licensed the drug back to Allergan, in exchange for tens of millions of dollars in both licensing and royalty fees. Although it may not sound like it, this transfer is potentially huge news in the drug pricing world. It is also extremely complex, and its full implications have yet to be determined.

Enormous caveat before we begin: I am by no means an expert on tribal sovereign immunity. I may well be wrong here. (In fact, I would very much like to be wrong here.) There is little (any?) case law on sovereign immunity’s impact in the Hatch-Waxman area, and much of what follows is extrapolated from case law on tribal sovereign immunity both in IP and in other contexts, state sovereign immunity in the IP area, and discussions with other law professors. Please let me know if this is your area of expertise and you believe I’ve gotten the analysis wrong!

In short, if repeated and taken to its logical conclusion, this transfer has the potential to prevent most invalidity challenges to drug patents. Would-be generic competitors could not seek to initiate inter partes review (IPR) actions before the Patent and Trademark Office (PTO). They could not bring declaratory judgment actions in federal court. And – both most importantly and most unclear – they could not bring Paragraph IV invalidity counterclaims under Hatch-Waxman, preventing generic companies from independently challenging patents’ invalidity and potentially requiring us all to wait until the very end of patent expiration to experience generic competition.

Continue reading

Two Views About the Gene Editing ‘Breakthrough’ that Are Not Getting Enough Attention (IMHO)

As has now been well-covered in the news, Nature just published a paper from Mitalipov’s lab at the Oregon Health and Science University that used CRISPR/Cas 9 gene editing to correct the MYBPC3 mutation associated with hypertrophic cardiomyopathy — a heart muscle disease that affects 1 in 500 people. The more impressive element of the story is that by doing the alteration simultaneous with the sperm fertilizing the egg (not after fertilization) they were able to avoid the mosaicism that problematized early attempts in China — in mosaicism not all cells are repaired due to failure in the editing. The media coverage thus far, sadly but predictably, has focused on the soundbite of “designer babies” and “hope and hype” (indeed as my friend Hank Greely has suggested perhaps “overhype”.) These are worthy narratives to tell, to be sure, but here are two other narratives that I think are not getting the air time they deserve:

(1) The Importance of Genetic Ties: This use of CRISPR/Cas 9, as with most reproductive technologies, are attempts to allow those with disease-causing genes or other obstacles to reproduce genetically to do so. Investment and development of these technologies reifies the importance of genetic ties, as opposed to the kinds of ties associated with adoption, step-parenting, etc. It confuses a right to be a genetic parent, with a right to be a parent. We might have one right or both, but we should be clear they are different rights claims. Françoise Baylis has written eloquently about this issue in the context of In Vitro Gemetogenesis, and others (myself included) have mused on what claims the infertile have on society to have the state pay for these kinds of technologies instead of adopting. The National Academies report on gene editing suggested that clinical use of gene editing to eliminate disease be restricted to cases where there is an “absence of reasonable alternatives,” but does not take a position on when adoption is a reasonable alternative. Of course, in the U.S. at least, adoption is not easy and not available for everyone and there are a ton of interesting normative questions I have gestured at (including whether it matters for “reasonability” whether the child is of a certain age, race, or lacks developmental delay).

(2) The Importance of Embryo Sparing: A different alternative to gene editing in some cases is to fertilize large numbers of embryos and engage in preimplantation genetic diagnosis to eliminate those embryos that carry the disease-causing genes. There is a lot of obstacles to doing this: the fact that women may not retrieve enough eggs to do this, the cost (physical and financial) of repeated egg retrievals and PGD, the fact that this may not work for all genetic problems, etc. But one problem that vexes some is that this results in the destruction of large numbers of embryos (“discard” is sometimes used as the euphemism). Gene editing may be a solve for this problem. The Mitalipov group in their Nature paper have a line to this effect, “When only one parent carries a heterozygous mutation, 50% of the embryos should be mutation-free and available for transfer, while the remaining carrier embryos are discarded. Gene correction would rescue mutant embryos, increase the number of embryos available for transfer and ultimately improve pregnancy rate” (emphasis mine). This raises to me a very interesting question: some religious conservatives have tended to oppose both attempts to transform the human genome & embryo destruction (especially in the stem cell debate context). Could gene editing offer an olive branch to them as an alternative to the “greater evil” of routine PGD plus discard? Does it matter that to get to a place where we could achieve this we would have to actually destroy numerous embryos to perfect the research? (The Mitalipov embryos were not implanted, it seems under current U.S.  law that they could not be/) Is the right way to think about this consequentialist — destroy some embryos today to develop embryo sparing technologies to save many more tomorrow — or is this a case of complicity where the wrongfulness of the basic research taints what comes later?

Monthly Round-Up of What to Read on Pharma Law and Policy

By Ameet SarpatwariMichael S. Sinha, and Aaron S. Kesselheim

Each month, members of the Program On Regulation, Therapeutics, And Law (PORTAL) review the peer-reviewed medical literature to identify interesting empirical studies, policy analyses, and editorials on health law and policy issues relevant to current or potential future work in the Division.

Below are the abstracts/summaries for papers identified from the month of June. The selections feature topics ranging from physicians’ views of the Sunshine Act; to a biomarker-based drug development case study of CETP inhibitors, to the potential return on public investment in detecting adverse drug effects. A full posting of abstracts/summaries of these articles may be found on our website.

  1. Chimonas S, DeVito NJ, Rothman DJ. Bringing Transparency to Medicine: Exploring Physicians’ Views and Experiences of the Sunshine Act. Am J Bioeth. 2017;17(6):4-18.
  2. Dong K, Boehm G, Zheng Q. Economic Impacts of the Generic Drug User Fee Act Fee Structure. Value Health. 2017;20(6):792-8.
  3. Hey SP, Franklin JM, Avorn J, Kesselheim AS. Success, Failure, and Transparency in Biomarker-Based Drug Development: A Case Study of Cholesteryl Ester Transfer Protein Inhibitors. Circ Cardiovasc Qual Outcomes. 2017;10(6).
  4. Huybrechts KF, Kesai RJ, Park M, Gagne JJ, Najafzadeh M, Avorn J. The Potential Return on Public Investment in Detecting Adverse Drug Effects. Med Care. 2017;55(6):545-51.
  5. Sommers BD, Maylone B, Blendon RJ, Orav EJ, Epstein AM. Three-Year Impacts Of The Affordable Care Act: Improved Medical Care And Health Among Low-Income Adults. Health Aff (Millwood). 2017;36(6):1119-28.
  6. ‘t Hoen EFM, Boulet P, Baker BK. Data exclusivity exceptions and compulsory licensing to promote generic medicines in the European Union: A proposal for greater coherence in European pharmaceutical legislation. J Pharm Policy Pract. 2017;10:19.

How the FDA Produces Knowledge (and Is Not So Weird)

Credit: SalFalko

The Federal government has wrested billions of dollars from the drug and device industry in settlements of claims that the companies broke the law by promoting their products “off-label” for uses not approved by the FDA.  In response, companies have asserted that promotions are a form of speech, protected by the First Amendment. Speech regulations are especially worrisome when motivated by paternalism.  This argument has received some traction in the courts, and is now getting a favorable look by the Trump administration.

I have argued (here, here, and here) that this law is not actually a speech regulation.  Nor is it paternalistic.  Instead, it is simply a vanilla regulation of a behavior (shipment of product in interstate commerce), which depends on various sources of evidence (including speech) as revealing whether the actor has an illicit intent (an unapproved use of the product).  The pre-market approval system, which requires that companies prove safety and efficacy for all intended uses, solves a collective action problem to produce information as a public good.  This is our key social mechanism for producing knowledge about safety and efficacy.  If this law is unconstitutional in the off-label context, the entire pre-market approval system would seem to be as well.

In a new piece out on SSRN, my physician co-author Victor Laurion develops the example of the drug Seroquel XR, to show how a federal prosecution for off-label promotion caused the company to perform scientific research on two new indications (general anxiety disorder and major depression).  A detailed discussion of the regulatory record shows how physician prescribing was improved by this public information, regardless of whether the FDA approved the new indication.  In this way, the FDA protects the liberty of physicians and patients to try drugs for new uses, even while holding companies to the proof of any uses that they actually intend.  The fact that the company’s intention is shown by speech evidence is immaterial. Continue reading

FDA v. Opana ER: Opioids, Public Health, and the Regulation of Second-Order Effects

Earlier this month, the FDA announced that it is asking Endo Pharmaceuticals to remove the opioid Opana ER from the market.  Opana ER is an extended-release pain reliever often abused by those who take it.  While opioid abuse is nothing new, and many opioids leave those who take them addicted to narcotics or heroin, Opana ER is particularly dangerous because of how people misuse it.  The pill was designed to prevent would-be abusers from crushing and snorting it —  a popular means of ingesting prescription opioids.  Without the ability to crush and snort the drug, however, abusers turned to dissolving the pills and injecting them intravenously, leading to outbreaks of Hepatitis C, HIV, and other blood-borne diseases.  In Indiana’s Scott County, for instance, the prevalence of HIV has skyrocketed since the introduction of Opana ER to the local population, with 190 new cases since 2015.

While this foray into public health is somewhat surprising — given the anti-regulatory stance of the current administration and its billionaire backers — it is precisely the type of initiative the FDA should be taking.  Public health is a central part of the FDA’s mission statement, which notes that the agency “is responsible for protecting the public health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, and medical devices.”  Traditionally, though, the FDA’s efforts to ensure safety and efficacy have been limited to the narrow context of individual patients taking medications as directed under physician supervision.  As the FDA noted in its Opana ER press release, this is the first time it has requested that an opioid be taken off the market as a result of its susceptibility to abuse and the associated public health consequences.

Continue reading

The First Human Body Transplant – Ethical and Legal Considerations

By Ana S. Iltis, PhD

brain_glowingprofileTo what lengths should we go to preserve human life? This is a question many are asking after hearing that three men plan to make medical history by conducting the first human head transplant. Or, rather, whole body transplant. Italian neurosurgeon Dr. Sergio Canavero and Chinese surgeon Dr. Xiaoping Ren plan to provide a Russian volunteer, Valery Spiridonov, a new body. During the procedure, Spiridonov’s body and head would be detached and, with the help of a crane, surgeons would move the head and attach it to the donor body.  But is this ethical? What role might law and regulation play in monitoring them or in assessing their conduct after the fact?

Critics call the plan crazy, unethical, and sure to fail. The likelihood of success is very low and the risk of Spiridinov dying is high. Spiridonov says that as soon as animal studies confirm the possibility of survival, the risks will be worth taking. He has Werdnig-Hoffmann Disease, a genetic disorder that destroys muscle and nerve cells. He is confined to a wheelchair and has lived longer than expected. Body transplantation offers him the best chance at a life worth living. Continue reading

Call for Abstracts: Wiet Life Sciences Law Scholars Conference

Loyola University Chicago’s nationally acclaimed Beazley Institute for Health Law and Policy is pleased to invite original research submissions for its inaugural Wiet Life Science Law Scholars Conference on Friday, October 13, 2017.

The conference is designed to provide a new intellectual venue for life science professors, scholars, and practitioners to convene and discuss current research and scholarship. The phrase “life science law” aims to capture research and disciplines spanning food and drug law, health law, intellectual property (IP), biotechnology, environmental, administrative, antitrust, and other realms that involve the life sciences in some meaningful respect. Our goal is to foster recognition of life science law as a cohesive, dynamic, area of legal study and strengthen connections among national life science law scholars.

Loyola is currently soliciting 750-1,000 word abstracts reflecting early or mid-stage ideas for the purpose of workshopping with other conference scholars. Modeled after successful events for law professors and scholars in other areas, participants will be organized in topical panels of three to five authors with approximately 15-20 minutes allotted to each abstract presentation, followed by discussions with scholar attendees. Abstracts from the authors will be distributed one week prior to the conference; authors may also submit draft articles for distribution to conference attendees.

SUBMISSION AND REVIEW TIMELINE: The deadline for 750-1,000 word abstracts, including author contact information is June 15. Submit via email to health-law@luc.edu with subject line Wiet Life Science Law.

Authors will be notified of speaker selections by email on or before July 15.

Continue reading

Jelly Beans, Booze, and B-Vitamins

By Seán Finan

The FDA’s policy guidelines on nutritional fortification include the so-called “jelly-bean rule:” the FDA considers it inappropriate to fortify candy or soda with nutrients because to do so would allow “misleading health claims” to be made about a putatively unhealthy product. Candy companies that tried to add vitamins their products to market them as “healthier” have already been targeted by the FDA. But take a quick glance at the shelves of any convenience store: the “healthy”, vitamin enriched snacks and drinks are so full of sugars, flavors and sweeteners that it would take a doctorate in metaphysics, rather than medicine, to distinguish them from the candy and soda. So, maybe the FDA’s stance on adding a spoonful of sugar to help the medicine go down has relaxed. With that in mind, here’s a little thought experiment. I’d like to bring a proposal back from the eighties: that inexpensive alcoholic beverages be fortified with allithiamine, a fat-soluble analogue of Vitamin B1.[1] Why? The fortification could dramatically reduce the incidence of Wernicke’s encephalopathy and Korsakoff’s Syndrome among the homeless and alcoholic population.

Continue reading

DTC Genetic Risk Reports Back on Market

By Kayte Spector-Bagdady, JD, MBE & Michele Gornick, PhD, MA

On Thursday, April 6th, the FDA announced that it will allow the direct-to-consumer (DTC) genetic testing company 23andMe to market “Genetic Health Risk” (GHR) tests for 10 diseases or conditions including early-onset Alzheimer’s and Parkinson’s Diseases. This is in addition to 23andMe’s current offering of ancestry, wellness (e.g., lactose intolerance), trait (e.g., hair color), and autosomal recessive carrier screening (e.g., sickle cell anemia) test reports.

The decade since 23andMe entered the market has been a regulatory labyrinth of twists and turns. But what direction are we headed now?

The way we were

23andMe was a pioneer of the field, entering the DTC genetics market in 2007 with a product offering 13 health-related reports for $999. By December 2013, it was offering more than 250 reports; including carrier status, drug response, and over 100 GHRs. In response to a set of FDA Untitled Letters that went out in 2010, 23andMe filed for de novo 510(K) premarket clearance for some tests… but also concurrently marketed them in a national television and web campaign. Continue reading