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Everything You Wanted to Know About Expanded Access but Were Afraid to Ask, Part 3

By Alison Bateman-House, Hayley M. Belli, and Sage Gustafson

This series is adapted from a webinar hosted by PRIM&R on August 5, 2021: IRB Review of Expanded Access Protocols that Collect Real World Data: Considerations and Guidance. Read Part 1 and Part 2.

Part 3: What’s an IRB to do?

EA is considered treatment, not research. EA was not established as a means to collect research data, even though certain safety data must be collected and shared with the FDA and the sponsor. But, once sponsors decide to capture/share EA-derived data above and beyond that needed to report SAEs, what should IRBs do when reviewing such plans: view this as research, and thus hold it to (higher) research standards, or continue to view this as treatment?  This distinction is important for patients’ rights and welfare.

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The International Weaponization of Health Data

By Matthew Chun

International collaboration through the sharing of health data is crucial for advancing human health. But it also comes with risks — risks that countries around the world seem increasingly unwilling to take.

On the one hand, the international sharing of health-related data sets has paved the way for important advances such as mapping the human genome, tracking global health outcomes, and fighting the rise of multidrug-resistant superbugs. On the other hand, it can pose serious risks for a nation’s citizens, including re-identification, exploitation of genetic vulnerabilities by foreign parties, and unauthorized data usage. As countries aim to strike a difficult balance between furthering research and protecting national interests, recent trends indicate a shift toward tighter controls that could chill international collaborations.

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Everything You Wanted to Know About Expanded Access but Were Afraid to Ask, Part 2

By Alison Bateman-House, Hayley M. Belli, and Sage Gustafson

This series is adapted from a webinar hosted by PRIM&R on August 5, 2021: IRB Review of Expanded Access Protocols that Collect Real World Data: Considerations and Guidance. Read Part 1 here.

Part 2: Possible Value of “Real World Data” Collected from Expanded Access

Real world data (RWD) are data relating to patient health status and/or the delivery of health care such as medical bills/claims, electronic health records, and product/disease registries. RWD are derived from sources outside of randomized controlled trials (RCTs). Real world evidence (RWE) may be derived from the analysis of quality RWD.

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Everything You Wanted to Know About Expanded Access but Were Afraid to Ask, Part 1

By Alison Bateman-House, Hayley M. Belli, and Sage Gustafson

This series is adapted from a webinar hosted by PRIM&R on August 5, 2021: IRB Review of Expanded Access Protocols that Collect Real World Data: Considerations and Guidance.

Part 1: What is Expanded Access and How Does it Work?

Expanded Access (EA) is a regulatory mechanism that allows patients, through their physicians, to request the use of an unapproved medical product in a treatment setting.

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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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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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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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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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