Compassionate Use: FDA Is on Board, but Are Drug Manufacturers?

Expanded access by providing patients access to a drug outside of a clinical trial, prior to FDA approval, has been on the policy agenda for a while now, and the 21st Century Cures bill includes a provision to force manufacturers to publicly share their compassionate use policy. Results from an Avalere Health study underscore the need for such a legislation. A survey by the company found that only 19% of drug makers publicly post policies about their compassionate use programs.

For its survey, Avalere reviewed the websites of 100 publicly traded pharmaceutical and biotechnology companies (classified as large, medium, and small) to document:

• Websites that had posted their policy
• Ease of finding the compassionate use policy
• Specific attributes of the posted policy

While the analysis found that only 19 of 100 companies surveyed had a policy posted to their website, larger companies were more likely to publicize their compassionate use policies:

• 13 of 25 large companies did so
• Only 4 of 28 medium companies did so
• Only 2 out of 47 small companies did so

With respect to ease of finding relevant information on compassionate use or expanded access, the authors found that 75% of the posted policies were easy to access on the companies’ websites. Unfortunately, multiple labels were used to describe or name the policy, including “compassionate use,” “expanded access,” “access to investigational drugs/medicines,” “pre-approval access,” and “early (patient) access.”

The variability and the lack of easy access to this information can prove a significant barrier for terminally ill patients and their families seeking information on alternate options during desperate times. According to STAT news, the federal government allows terminally ill patients ineligible for a clinical trial to apply to the FDA for its expanded access program, which approved most of the applications (nearly 1900) it received in 2015. Another alternative is the Right to Try law. The law has been passed by 33 states in the United States and bypasses the FDA to provide access to phase 1 drugs for those who are terminally ill.

The Avalere findings “ought to make it clear that the FDA role is utterly secondary and corporate decision-making is the driver and primary actor in giving access to investigational products.,” Arthur Caplan, PhD, a bioethicist at New York University Langone Medical Center told STAT. “The FDA doesn’t get involved until a company has made a decision about granting access. And if a company doesn’t have a policy on its website, it’s a huge obstacle to patients and doctors more than anything else.”

The Cures bill would be a much needed push to streamline this important process because it will force drug manufacturers to publish:

• Contact information for the manufacturer or distributor to facilitate communication about requests
• Procedures for making compassionate use requests
• General criteria for approving requests
• Length of time anticipated to acknowledge receipt of requests