Category: Clinical Research

Hundred dollar bills rolled up in a pill bottle

Ketamine Is the New Viagra

Vincent Joralemon Leave a comment

By Vincent Joralemon

Spravato, the first FDA-approved psychedelic therapy, just outsold Viagra. Johnson & Johnson’s ketamine-based formulation generated $183 million in Q3, surpassing Pfizer’s erectile dysfunction (ED) drug, which earned $110 million over the same period. Remarkably, a therapeutic made from ketamine, once dismissed as a “club drug” or “horse tranquilizer,” now sells more than one of the most notable 21st-century pharmaceuticals. 

Yet, these two drugs have more in common than meets the eye, and the path Spravato has taken looks strangely similar to that of Viagra. By looking at the journey traversed by Viagra over the past twenty years, we can predict where Spravato (and other psychedelic therapies) are headed.

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Silver Spring, MD, USA - June 25, 2022: The U.S. Department of Health and Human Services (HHS), U.S. Public Health Service (USPHS) and FDA logos are seen at the FDA headquarters, the White Oak Campus.

FDA Solicits Feedback on the Use of AI and Machine Learning in Drug Development

Matthew Chun Leave a comment

By Matthew Chun

The U.S. Food and Drug Administration (FDA), in fulfilling its task of ensuring that drugs are safe and effective, has recently turned its attention to the growing use of artificial intelligence (AI) and machine learning (ML) in drug development. On May 10, FDA published a discussion paper on this topic and requested feedback “to enhance mutual learning and to establish a dialogue with FDA stakeholders” and to “help inform the regulatory landscape in this area.” In this blog post, I will summarize the main themes of the discussion paper, highlighting areas where FDA seems particularly concerned, and detailing how interested parties can engage with the agency on these issues.

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On Experiencing IBD as a Woman

Amalia Sweet Leave a comment

By Amalia Sweet

At the end of last summer I stopped eating. It wasn’t that I wasn’t hungry — I was, constantly — but rather that pretty much everything I tried to put in my stomach triggered excruciating abdominal pain. 

While still in Chicago where I was working toward my master’s degree, I went to University Health Services. When tests revealed I was anemic but free of ulcers and Celiac disease, they suggested I work to reduce my stress and follow up with a gastroenterologist when I returned home to Boston later that month. 

I called every medical practice I could think of in the greater Boston area and no one had availability sooner than four months out. Without a primary care physician and desperate for a diagnosis, I went to the ER. In spite of my anemia and the fact that I had lost a scary amount of weight in a short period of time, the ER refused to provide a prioritized referral and told me my symptoms were a product of me being sedentary when in fact I was sedentary because of my symptoms. 

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

The Petrie-Flom Center Staff Leave a comment

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

Matthew Chun Leave a comment

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

The Petrie-Flom Center Staff Leave a comment

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

The Petrie-Flom Center Staff Leave a comment

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

Ameet Sarpatwari Leave a comment

By Ameet Sarpatwari, Beatrice Brown, Alexander Egilman, Aviva 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

The Petrie-Flom Center Staff Leave a comment

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

The Petrie-Flom Center Staff Leave a comment

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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