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

By Ameet SarpatwariCharlie LeeFrazer Tessema, 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 pharmaceutical law and policy.

Below are the abstracts/summaries for papers identified from the month of April. The selections feature topics ranging from increases in Internet searches for hydroxychloroquine following promotional remarks by the President, to an evaluation of health gains from orphan drugs, to an assessment of clinical trials supporting new FDA drug approvals. A full posting of abstracts/summaries of these articles can be found on our website.

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Stacks of books against a burgundy wall

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

By Ameet Sarpatwari, Charlie Lee, Frazer Tessema, 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.

Below are the abstracts/summaries for papers identified from the month of March. The selections feature topics ranging from the utilization and cost of naloxone for patients at high risk of opioid overdose, to off-label and compassionate drug use in the COVID-19 pandemic, to public-sector financial support and sponsorship for gene therapy trials in the U.S. A full posting of abstracts/summaries of these articles may be found on our website.

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a covid 19 swab test being inserted into a vile

Maintaining Healthy Skepticism About Reported COVID-19 Findings

By Andy Podgurski and Sharona Hoffman

The COVID-19 pandemic presents special challenges to even well-informed and well-intentioned promulgators and consumers of medical research findings, including the legal community.

The stakes in the debate about handling the pandemic are extremely high in terms of lives, jobs, wealth, and political power. In addition, there are tremendous opportunities for researchers to receive attention and notoriety for influencing the debate.

All this means that perverse incentives exist to publicize initial scientific findings that are dubious, poorly vetted, and possibly dangerous to public welfare. The risk of promulgating false or misleading scientific claims is substantial, even when they are made by well-respected scientists affiliated with prestigious institutions. Government authorities must be extremely cautious about basing public policy decisions on inadequately vetted findings, no matter how much hype they get.

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Box of Hydroxychloroquine Tablets

Human Subjects Research in Emergencies: The Texas Nursing Home “Study” (Part II)

By Jennifer S. Bard

This post is the second in a series about conducting human subjects research in emergencies. These posts are being written in response to a rapidly evolving situation and will reflect the state of knowledge at the time of writing.

In April 2020, Dr. Robin Armstrong, medical director of the Resort, a nursing home in Texas City, Texas, reported “signs of improvement” after he gave hydroxychloroquine, a drug approved by the FDA to treat malaria, to 39 of his nursing home patients who were diagnosed with COVID-19.

At about the same time, information was emerging that now represents the current understanding that hydoxychloroquine isn’t only ineffective in treating COVID-19, but also may cause serious harm to patients. Tensions were raised even higher by the seemingly inexplicable enthusiasm for this treatment by the President and some media outlets.

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Researcher works at a lab bench

Human Subjects Research in Emergencies: An Ethical and Legal Guide (Part I)

By Jennifer S. Bard

This post is the first in a series about conducting human subjects research in emergencies. These posts are being written in response to a rapidly evolving situation and will reflect the state of knowledge at the time of writing.

The world is facing a medical emergency in the form of the rapid spread of a new virus, COVID-19, for which there is no known effective treatment and no preventive vaccine.

Without minimizing the need for haste or the significance of the threat, it is still important to remain aware of the risks inherent in rushing to treat patients with anything that might work and simultaneously conducting the research necessary to identify safety and effective interventions.

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Medicine law concept. Gavel and stethoscope on book close up

Free Online Ethics Resources Available from the Perelman School of Medicine

By Holly Fernandez Lynch

One of the silver linings of the COVID-19 pandemic has been seeing communities come together to offer support in ways big and small. Individuals are organizing drives to collect personal protective equipment for health care workers, media outlets are making pandemic content available for free, and children’s book authors are hosting online story times to offer a brief respite for parents suddenly thrust into homeschooling.

In that same spirit, the Department of Medical Ethics and Health Policy at the Perelman School of Medicine, University of Pennsylvania is hoping to ease the burden for bioethics faculty and bioethics and health professions students who may be in search of online content as their learning experiences have moved out of the brick-and-mortar classroom. We’re offering a variety of recorded video content in clinical and research ethics at no charge through at least June 30 – with lectures from two Petrie-Flom alums, Holly Fernandez Lynch and Emily A. Largent, as well as other faculty experts.

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Vials of medications with syringe and needle.

Is “Implied Consent” Ethically Permissible in WHO’s Malaria Vaccine Pilot Introduction?

By Beatrice Brown

A recent BMJ article has exposed ethical concerns with the informed consent process in the World Health Organization’s (WHO) large, randomized cluster trial of the world’s first licensed malaria vaccine, RTS,S, known as Mosquirix. The study is being conducted in Malawi, Ghana, and Kenya, and 720,000 children will receive the vaccine. The vaccine is currently limited to pilot implementation because of residual safety concerns from previous clinical trials, including: a tenfold rate of meningitis in those who received the vaccine versus those who did not, “increased cerebral malaria cases, and a doubling in the risk of death in girls.” Rather than engaging in the traditional informed consent process, the WHO is utilizing an implied consent process, leading several bioethicists, including Charles Weijer, Christine Stabell Benn, and Jonathan Kimmelman, to voice concern.

The WHO has defended their use of implied consent to BMJ on the grounds that “the study is a ‘pilot introduction’ and not a ‘research activity.'” A WHO spokesperson explained that in an implied consent process, “parents are informed of imminent vaccination through social mobilisation and communication, sometimes including letters directly addressed to parents. Subsequently, the physical presence of the child or adolescent, with or without an accompanying parent at the vaccination session, is considered to imply consent.” However, as Weijer rightly points out, this is not truly consent, as “We have no assurance that parents, in fact, received information about the study let alone that they understood it.” After the publication of the original article criticizing the WHO for going against international ethical standards for research involving human participants, the WHO released a response in BMJ and on their own website, contending that this implied consent process is “used for all vaccines provided through the Expanded Programme on Immunization” and that the study is in accordance with international ethical standards. Here, I further explore whether this implied consent process is ethically permissible in this specific trial by exploring the guidelines set out by two organizations.

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A white hospital hallway

The ‘Nazi Problem’ in Human Subject Research Regulation

As Holocaust Martyrs’ and Heroes’ Remembrance Day draws near, it is heartening to note that our collective horror against Nazis and Nazism is still sufficiently strong as to make them the archetype evil villains in movies, TV, and dystopian literature. The evil doctor conducting fiendish experiments lives on in fiction because we recall the all-too-real horrific acts of torture conducted by Nazi physicians against children held captive and killed in the millions.

(Here are the Personal Statements of people who survived to tell their stories.)

Yet, as important as it is to never forget these crimes, we should resist the temptation of letting these memories lead us to a false story about the origin of human subject research regulation in the United States. Read More

Zoom in of a dashboard focusing on the "App Store" widget

Nobody Reads the Terms and Conditions: A Digital Advanced Directive Might Be Our Solution

Could Facebook know your menstruation cycle?

In a recent Op-ed Piece, “You Just Clicked Yes. But, Do you Know Terms and Conditions of that Health App?,” I proposed that a mix of factors have given rise to the need to regulate web-based health services and apps. Since most of these applications do not fall under the Health Insurance Portability and Accountability Act (HIPAA), few people actually read through the Terms and Conditions, and also, the explosive growth of web-based health applications, the need for solutions is dire. Read More

Image of Normal blood cells next to a sickle blood cell, colored scanning electron microscope image.

Recharting the Course of Sickle Cell Disease – Who will Benefit?

By Vence L. Bonham and Anitra Persaud

Scientific advancements in gene therapy and the implications of leveraging this technology to develop new curative therapies are at the forefront of medical research. Sickle cell disease (SCD), the most common genetic blood disorder, stands center stage. Last month, 60 Minutes aired a segment showcasing the story of a patient at the NIH Clinical Center who is on her journey to a cure of sickle cell disease (SCD) with the help of an experimental gene therapy.

Preliminary clinical trial findings suggest that gene therapy has an acceptable level of safety and can help individuals with the disease produce normal red blood cells instead of the sickle-shaped ones that underlie the physiological basis of the disease and its complications. Given these promising results, there is hope that gene therapy may catalyze a turning point for the SCD population, a community that has long suffered the debilitating effects of not only their disease, but of longstanding neglect within the medical system and research enterprise. Read More