Syringe being filled from a vial. Vaccine concept illustration.

What Does the Good News on the Vaccine Front Mean?

By Dorit Rubinstein Reiss

In the past weeks, three companies in advanced stages of COVID-19 vaccine trials reported good news. Moderna and Pfizer reported, respectively, 94.5% and 95% effectiveness of their mRNA vaccines in preventing symptomatic disease and similarly high effectiveness in preventing severe disease.

This was shortly followed by news that the AstraZeneca vaccine had over 70% effectiveness, and 90% with a different dosage regime.

The companies have also reported a favorable safety profile, with no serious harms attributed to the vaccine, though the vaccines do cause a high rate of temporary and unpleasant side effects, including local reactions and temporary flu-like symptoms.

Pfizer has already applied for an Emergency Use Authorization (EUA) from the FDA, and I would expect Moderna and AstraZeneca to follow suit.

What does this mean? First, a note of caution. These are reports from the companies; the FDA has not yet finished examining the data. Examination may raise questions. The data submitted has to pass dual review.

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books

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

By Ameet SarpatwariBeatrice Brown, Neeraj Patel, 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 October. The selections feature topics ranging from a commentary calling for reconsideration of the FDA’s risk evaluation and mitigation strategy (REMS) program for mifepristone, to an analysis of clinical development times for biosimilars seeking FDA approval, to an editorial describing the challenges of using the Defense Production Act to address drug shortages. A full posting of abstracts/summaries of these articles may be found on our website.

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Pill pack.

Fortifying the US Pharmaceutical Supply Chain

By Laura Karas

The COVID-19 pandemic triggered supply chain disruption across the globe. The United States, in particular, is susceptible to interruptions in the supply chain for pharmaceutical drugs because many of the raw materials, active pharmaceutical ingredients, and manufacturing processes needed to produce domestically marketed prescription drugs have been outsourced beyond U.S. borders.

Is it time to bring some of these processes back to our shores? This post will demystify the pharmaceutical supply chain and explore some key considerations as we head toward 2021.

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Map of United States made up of pills.

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

By Ameet SarpatwariBeatrice Brown, Neeraj PatelandAaron 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 September. The selections feature topics ranging from commentaries on political pressures and questions of integrity facing the FDA, to a critique of the financial incentive structure for antibiotic development, to an estimation of how much NHS England would spend if it paid U.S. Medicare Part D prices. A full posting of abstracts/summaries of these articles may be found on our website.

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Washington, USA- January13, 2020: FDA Sign outside their headquarters in Washington. The Food and Drug Administration (FDA or USFDA) is a federal agency of the USA.

Experts Question FDA Approval of Remdesivir for COVID-19

By Sravya Chary

Experts are calling into question the recent decision of the U.S. Food and Drug Administration (FDA) to approve remdesivir (sold under the brand name Veklury) for the treatment of COVID-19 and casting scrutiny as to whether the decision is truly in the public’s interest.

Evaluating and approving an effective treatment for SARS-CoV-2 virus has been a top priority for regulatory authorities, especially in the absence of a viable vaccine. On October 22, 2020, the FDA approved Veklury for the treatment of COVID-19 in adult and pediatric patients requiring hospitalization.

The FDA cited three randomized, controlled clinical trials as the evidence supporting its decision to approve Veklury. The findings from the three studies were as follows: first, that the median time to recovery from COVID-19 was 5 days sooner in the Veklury group compared to the placebo group. Second, that the odds of a research subject’s COVID-19 symptoms improving were statistically significantly higher in the five-day treatment group than the standard of care group (the 10-day treatment group did not show a statistically significant difference from the standard of care group). Third, that there were no statistically significant differences in recovery or mortality rates between subjects in the five-day Veklury group versus the ten-day Veklury group.

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

The Cost of Exclusion in Psychedelic Research

By Xinyuan Chen, Mackenzie Bullard, Christy Duan, Jamilah R. George, Terence Ching, Stephanie Kilpatrick, Jordan Sloshower, and Monnica Williams

In the last two decades, researchers have started to reexamine psychedelics for their therapeutic potential. Though initial results seem promising, the research has a significant shortcoming: the lack of racial and ethnic diversity among research teams and study participants.

In the 1960s, psychedelic substances such as LSD, psilocybin, and mescaline were a major part of American counterculture. Less well-known is that, concurrently, researchers were studying potential therapeutic uses of these mind-altering substances. Unfortunately, psychedelics were classified as Schedule I drugs in 1970, halting research into their therapeutic benefits.

The recent renaissance of psychedelic research shows these substances have significant capabilities for treating anxiety, depression, posttraumatic stress disorder (PTSD), and substance use disorders. But these promising results are limited in their applicability: an analysis from 2018 showed that 82.3% of all study participants in psychedelic trials internationally were non-Hispanic Whites, and only 2.5% were African-American.

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Young male doctor in telehealth concept

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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computer and stethoscope

Is Real-World Health Algorithm Review Worth the Hassle?

By Jenna Becker

The U.S. Food and Drug Administration (FDA) should not delay their plans to regulate clinical algorithms, despite challenges associated with reviewing the real-world performance of these products. 

The FDA Software Pre-Certification (Pre-Cert) Pilot Program was designed to provide “streamlined and efficient” regulatory oversight of Software as a Medical Device (SaMD) — software products that are regulable by the FDA as a medical device. The Pre-Cert program, in its pilot phase, is intended to inform the development of a future SaMD regulatory model.

Last month, the FDA released an update on Pre-Cert, highlighting lessons learned from pilot testing and next steps for developing the program. One key lesson learned was the difficulty in identifying and obtaining the real-world performance data needed to analyze the clinical effectiveness of SaMDs in practice. Although this challenge will be difficult to overcome in the near future, the FDA’s plans to regulate should not be slowed by insufficient postmarket data.

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Washington, USA- January13, 2020: FDA Sign outside their headquarters in Washington. The Food and Drug Administration (FDA or USFDA) is a federal agency of the USA.

A New Step for the FDA in Regulating Digital Health Products

By Vrushab Gowda

On September 22, the U.S. Food and Drug Administration (FDA) announced the establishment of a new initiative to regulate digital health products – the Digital Health Center of Excellence (DHCoE).

In some ways, the announcement does not come as a surprise; FDA Commissioner Stephen Hahn’s predecessor, Scott Gottlieb, outlined the DHCoE in a press release two years ago. What does remain to be seen is whether DHCoE represents a true paradigm shift in FDA’s approach to regulating digital health products.

According to Hahn, the DHCoE aims to (1) build partnerships, (2) share knowledge across FDA and with stakeholders, and (3) innovate regulatory approaches. It will be led by the current Director of CDRH’s Division of Digital Health, Bakul Patel.

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Person typing on computer.

Telehealth Policy Brought to the Fore in the COVID-19 Pandemic

By Vrushab Gowda

The COVID-19 pandemic has highlighted the value of telehealth as both a tool of necessity (e.g., minimizing infection risk, conserving thinly stretched healthcare resources, reducing cost) as well as of innovation.

Telehealth services have surged in recent months; in April alone, they constituted over 40 percent of primary care visits nationwide and over 73 percent of those in Boston. “Increasing Access to Care: Telehealth during COVID-19,” a recent publication in the Journal of Law and the Biosciences, dissects the issues that have accompanied the growth of telehealth and identifies further areas of potential reform.

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