In the Rush to Innovate for COVID-19 Drugs, Sound Science Is Still Essential

Following President Trump’s comments that these drugs could be a potential “game changer,” attention and prescriptions sky-rocketed, despite caution from experts. Some physicians stockpiled the drugs for personal use, and several hospitals have adopted hydroxychloroquine as COVID-19 standard of care. Although there have been efforts to help protect supply for those patients needing the drug for their proven indications, some of these patients have been told they may have to go without.

It is too soon to say whether chloroquine products work for COVID-19, since the few clinical studies are small and lack randomization or carefully matched control groups. Severe side effects have caused some hospitals to stop using them altogether.

There is a great need for rigorously conducted clinical trials on these products and their possible effectiveness in fighting COVID-19. But if physicians continue prescribing them off-label, without regard for appropriate testing, we’ll be left with anecdotes, not evidence.

Expanded Access to New Drugs
There are other drugs with some potential to combat COVID-19, but that have not yet been approved for any use and therefore may not be prescribed off-label. These drugs are currently under investigation in clinical trials around the U.S. and the globe. For seriously ill patients, the FDA has a pathway known as “expanded access” (sometimes called “compassionate use”) by which patients may be dosed with unapproved drugs for treatment use, if they are unable to enroll in a clinical trial. This eligibility restriction is critical because it ensures that patients cannot secure access by opting out of the trials designed to produce the evidence needed to confidently assess a product’s safety and efficacy.

Drug maker Gilead has emphasized this approach with its investigational antiviral drug remdesivir. Even as it opens its expanded access program through a wider pathway, the company has explained that participation in clinical trials will be the primary mode of patient access.

Focus on Science
Pragmatism is needed to collect data in real time, as patients are also in desperate need of treatment. That’s precisely the approach taken by the World Health Organization in its mega trial of four potential treatments for COVID-19, including remdesivir and chloroquine products, with over 70 countries participating. The trial is aptly named SOLIDARITY, and it is designed to minimize the burden on physicians and patients, while allowing random assignment and collection of systematic, anonymous data.

We simply have to stop guessing about what’s going to work for patients battling COVID-19. Patients today and tomorrow need a commitment from politicians, policymakers, companies and physicians to prioritize science and rigorous study. Off-label use and expanded access may be reasonable options for patients when there is no clinical trial available, but if there is, we have to prioritize enrollment.

The FDA has demonstrated its willingness to help speed trials and facilitate the collection of data. But its regulatory standards must not be short-circuited and its flexibility must be used judiciously. Federal policy in this area should be driven by scientific expertise, not false hope, hunches or short-sighted political demands.

Christopher Robertson is Professor of Law, University of Arizona. Alison Bateman-House is Research Assistant Professor, Division of Medical Ethics, New York University Langone Medical Center. Holly Fernandez Lynch is Assistant Professor of Medical Ethics, University of Pennsylvania. Keith Joiner is Professor of Medicine, Economics and Health Promotions Science, University of Arizona. This article is published courtesy of The Conversation.