The Federation of American Scientists (FAS) is putting forth a “bold” menu of breakthrough science projects for consideration by the incoming administration under the banner Day One 2025. Among those projects are An Innovation Agenda for Addiction, a piece written by Nicholas Reville, executive director of the Center for Addiction Science, Policy and Research, and medical entrepreneur, Willy Chertman, Biotechnology Fellow at the Institute for Progress.

The authors come out swinging with a blunt call to “expand the FDA’s priority review voucher program (PRV) and provide market exclusivity advantages to encourage the development of medications for addiction.”

The PRV is a series of incentives for drug makers to develop drugs for rare conditions where there is not enough of a market to warrant the costs of drug development. When companies do this, they earn vouchers that can be used to speed reviews of other, more lucrative drugs in the pipeline. These review vouchers can be traded and sold to other drug companies.

Addiction to opioids is not a “rare condition,” but it is a problem area in drug development for a variety of reasons. One is the health of the patients in trials, which can lead to deaths during trials that are unrelated to the trial. There is also a reliability problem with patients being treated for substance use disorders (SUDs). The dropout rate in trials is high, biasing any results. The authors recommend:

The PRV program should be expanded to cover opioid use disorder, alcohol use disorder, stimulant use disorder, and smoking.

The authors make their case quite forcefully, with charts that show the shocking lack of development for addiction recovery medications. In the U.S., in the near quarter-century from 2000 to 2023, the annual death rate from SUDs averaged 767,000 and the annual death rate from cancer averaged 609,000. Yet over that same period, 222 medications for cancer treatment were approved, and six for SUDs!

The authors note the decades-long gap since the last medications approved for opioid use disorder (buprenorphine) and alcohol use disorder (acamprosate). “None of the ten largest pharmaceutical companies have active addiction medicine programs or drug candidates,” the authors lament.

What about GLP-1 agonists such as Ozempic and Mounjaro, which have shown anecdotal evidence of reducing withdrawal symptoms for opioid use disorders (OUDs) and alcohol use disorders (AUDs)? Reville and Chertman dig deep on the subject to explain why we have not seen drug companies conducting SUD trials with GLP-1 agonists:

Many addiction scientists now consider these compounds to be the biggest breakthrough in decades. However, Novo Nordisk and Eli Lilly, who own the drugs currently in the market, do not plan to run phase 3 addiction trials on them, due to fear of adverse events in substance use disorder populations.

The drug makers are enjoying stunning profits from GLP-1s for weight loss and they see no reason to potentially jeopardize that market with an adverse result from a SUD trial. Academic researchers have shown amazing results with GLP-1 agonists, but the drug development won’t scale without support from drug makers.

To stimulate drug makers to develop anti-addiction medications, the authors recommend first expanding the PRV program to SUDs, and secondly extending exclusivity on the drugs developed. Like the review vouchers, exclusivity vouchers would be transferable to other medications. These vouchers have proven to be very effective in encouraging the development of “orphan drugs.”

The policy recommendations conclude with a series of suggestions for the Food and Drug Administration (FDA) regarding the approval process for anti-addiction medications, including:

• The FDA should provide increased consideration for addiction medicines for breakthrough, fast-track, and priority review designations

• The FDA should modernize standards of efficacy for substance use disorder trials

• The FDA should “shift away from abstinence-based endpoints and towards more meaningful consumption-based endpoints”

• The FDA should work to adopt endpoints and standards of efficacy that mirror standards for other disease areas

The authors’ emphasis on energizing market systems to stimulate drug development should be music to the ears of both drug companies and federal regulators. Given the severity of the problem, which the authors outline in grim detail, the underfunding of research is a disgrace and needs to be addressed to reduce preventable deaths.

Written by Steve O’Keefe. First published January 8, 2025.

Sources:

“An Innovation Agenda For Addiction: Breakthrough Medicines That Scale,” Federation of American Scientists, January 6, 2025.

“Charting the fourth wave: Geographic, temporal, race/ethnicity and demographic trends in polysubstance fentanyl overdose deaths in the United States, 2010–2021,” Addiction, September 13, 2023.

“Regulatory Explainer: Everything You Need to Know About FDA’s Priority Review Vouchers,” Regulatory Focus, February 24, 2020.

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