books

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

By Ameet SarpatwariBeatrice Brown, Alexander EgilmanAviva Wang, 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.

Below are the citations for papers identified from the month of August. The selections feature topics ranging from an overview on the evolution of medical device regulation in the United States, to an analysis of the impact of the disclosure of expanded access policies mandated by the 21st Century Cures Act, to an evaluation of how litigation has impacted the success of the Biologics Price Competition and Innovation Act.

A full posting of abstracts/summaries of these articles may be found on our website.

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Society or population, social diversity. Flat cartoon vector illustration.

Engendering Equity in Biomedical Research by Meeting Communities Where They Are

By Rachele Hendricks-Sturrup

To address the root of both health disparities and community underrepresentation in biomedical research, it is mission-critical to teach early-stage career researchers how to empower underrepresented communities as partners in research while respecting and appreciating local history, context, and values.

As a researcher, I often encounter empirical studies in the literature that explore and experiment with institutionally– (versus community-) derived interventions that are meant to help boost underrepresented community engagement in biomedical research.

What if researchers took more time to intentionally harness their power and training to elevate, empower, and mobilize the voices of the communities they study to help design more impactful engagement interventions?

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Scientist analyzes DNA gel used in genetics, forensics, drug discovery, biology and medicine

Transplant Genomics: Ethical, Legal, and Social Implications

By Tamar Schiff

The appeal of precision medicine is of particular significance in transplantation. Treatment options are already integrally dependent on genetic factors – the donor-recipient match – and the demand for transplantable tissues far outstrips the available supply.

And the potential is only growing. Advances in genetic and genomic studies have identified an increasing number of novel biomarkers of potential use in transplant-related care. These include predictors of disease course, graft survival, response to immunosuppression, and likelihood of disease recurrence or other complications.

With wider availability of sequencing technologies and innovations in databanking, future clinical applications in transplant care may require ever-growing considerations of the significance of genetic variants, fair access to precision medicine therapeutics and participation in research, ethical approaches to data aggregation, and social determinants of health.

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Society or population, social diversity. Flat cartoon vector illustration.

Unequal Representation: Race, Sex, and Trust in Medicine — COVID-19 and Beyond

By Allison M. Whelan*

The COVID-19 pandemic has given renewed importance and urgency to the need for racial and gender diversity in clinical trials.

The underrepresentation of women in clinical research throughout history is a well-recognized problem, particularly for pregnant women. This stems, in part, from paternalism, a lack of respect for women’s autonomy, and concerns about women’s “vulnerability.” It harms women’s health as well as their dignity.

Over the years, FDA rules and guidance have helped narrow these gaps, and recent data suggest that women’s enrollment in clinical trials that were used to support new drug approvals was equal to or greater than men’s enrollment. Nevertheless, there is still progress to be made, especially for pregnant women. In the context of COVID-19 research, one review of 371 interventional trials found that 75.8% of drug trials declared pregnancy as an exclusion criteria, a concerning statistic given that recent data suggest that contracting COVID-19 during pregnancy may increase the risk of preterm birth.

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COVID-19’s Impact on Clinical Trials: Meeting Participants Where They Are

By Sarah V. Ferranti and Shine Chen Schattgen

“Decentralized” clinical trials (referred to as “DCTs”) are not novel, but nevertheless failed to gain real momentum given the regulatory and operational complexities involved. In light of COVID-19, however, it seems almost certain that the remote and virtual study activities that characterize DCTs, and a site and sponsor’s ability to flex to “meet the participant where they are” will be critical to the conduct of clinical trials going forward.

In the first half of 2020, as health care facilities prepared for capacity-exceeding patient volumes and equipment shortages, non-essential clinical care and non-COVID-related clinical trials came to a screeching halt. According to ClinicalTrials.gov, 1473 clinical trials were suspended, terminated, or withdrawn between December 1, 2019 and July 1, 2020, with a reported reason that explicitly mentioned COVID-19.

At the same time, initiation of clinical trials for COVID-19 vaccines and treatments exploded at speeds previously considered unachievable within the clinical trial industry. As of October 13, 2020, 811 COVID-19-related clinical trials had been initiated in the United States. To enable COVID-19 trials and, more recently, to restart previously paused non-COVID trials, clinical trial sponsors and sites have been forced to quickly adapt to protect participants and preserve the integrity of clinical trial data and results.

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Money.

Conflicts of Interest in the Hospital Sector: A Q&A with Rina K. Spence

By Chloe Reichel

Brigham and Women’s Hospital recently made headlines when the Boston Globe reported that the hospital’s president, Dr. Elizabeth Nabel, held a seat on the board of Moderna, a Cambridge biotech company that is working to develop an mRNA COVID-19 vaccine. The hospital has a major role in a national study of the vaccine.

The hospital maintained that safeguards were put in place to protect against conflicts of interest during the collaboration. Nevertheless, amid public outcry, Nabel stepped down from the board.

But this story is just one high-profile case of what is commonplace in the hospital sector. A 2014 research letter published in the Journal of the American Medical Association found that 40 percent of pharmaceutical company boards of directors had at least one member who also held, at the same time, a leadership role at an academic medical center.

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Racial Disparities Persist in Human Subjects Research

By Beatrice Brown

Human subjects research has long been plagued by racial inequality. While flagrant abuses have been curtailed, disparities have, unfortunately, persisted.

One area ripe for scrutiny is clinical trial enrollment. A 2018 study by William Feldman, Spencer Hey, and Aaron Kesselheim in Health Affairs documents racial disparities in trials that are exempt from typical requirements for informed consent from study participants.

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Researcher works at a lab bench

New Study Provides Insights into Potential Regulatory Treatment of COVID-19 Drugs

By Beatrice Brown

As the global pandemic continues, trials have been established to test whether existing drugs such as hydroxychloroquine could be repurposed to treat patients with COVID-19. There are also hopes that a novel drug will surface. But questions remain about when treatments and vaccines will become available.

There is currently great optimism that a treatment or vaccine will be developed quickly, but there is no assurance that such a vaccine or treatment will be highly effective or that normalcy will return in any particular timeframe. A recent study published in the Lancet ID by Jonathan J. Darrow, Mehdi Najafzadeh, Kristina Stefanini, and Aaron S. Kesselheim provides data that might help to temper enthusiasm with evidence.

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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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The Birth of GINA: An Interview with Jeremy Gruber

By Kaitlyn Dowling

Illustration of a gavel made out of a DNA helixIn a new, year-long series on Bill of Health, we’ll be exploring the legal scholarship on genetic non-discrimination. We’ll talk more about GINA and state laws protecting citizens from genetic discrimination. We hope these posts help shed light on this complex and ever-more-relevant area for legal scholars, policymakers, and the public at large.

The following is an interview with Jeremy Gruber, Senior Vice President at Open Primaries and the former President and Executive Director of the Council for Responsible Genetics. Jeremy Gruber led the passage of the Genetic Information and Nondiscrimination Act and other state-based genetic nondiscrimination legislation. As part of our series on GINA, he offered insights into GINA’s creation and what the future may hold for genetic nondiscrimination. The following interview has been lightly edited for length and clarity.

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