Requests for compassionate use of drugs not yet approved by the FDA have been making headlines, and experts predict this trend will continue. Last year, the FDA received 1,262 expanded access requests from drug companies, on behalf of patients, to use unapproved drugs outside of clinical trial programs.

Since 2010, the number of compassionate use requests has risen by nearly 25%. Many of these requests are for rare diseases with small patient populations, with highly engaged patients and caregivers connected to advocacy groups. With the increase in targeted therapies, the question is not whether research sponsors will have to address a request, but when and how prepared they are to respond.

The FDA has clear guidelines on requirements for meeting expanded access, but the onus is on companies to determine which cases to support and which to deny. Many companies are unprepared to respond quickly, having no formal process to follow. This approach has left the corporate reputations of several companies vulnerable.

For example, several years ago BioMarin was petitioned for access to its experimental phase III ovarian cancer drug. Like many pharma companies, BioMarin refused access. Andi Sloan, the petitioner, began a social media campaign in attempt to prolong her life with treatment. Word spread quickly through Facebook and Twitter, generating major media coverage critical of BioMarin, celebrity support from Wynonna Judd and Newt Gingrich and a Change.org petition signed by 150,000 people. A rival firm anonymously donated its own ovarian drug candidate to Sloan, but her condition worsened and she died.

As healthcare has become more consumer-driven, policy makers have stepped into the fray, proposing legislation aimed at cutting through government red tape that prevents access to investigational drugs in a timely manner. These “Right to Try” bills have made their way to legislatures in half the states in the U.S.

Some argue that these bills are empty legislation designed to raise the hope that patients have new rights. In fact, patients already have a path to request access to these drugs under current FDA rules, and over 98% of applications to the FDA to use experimental drugs are approved. These new laws contain no provision that compels companies developing experimental drugs to approve a patient’s request.

The public at large may not understand the impact of compassionate use on clinical research, but it has the potential to compromise the clinical trial process. The essence of right-to-try laws is to eliminate the role of the FDA from the process, which flies in the face of public safety.

Furthermore, the easier it is for patients to get access to experimental therapies, the less inclined they may be to enroll in a clinical trial. This is particularly troubling for rare diseases with small patient populations, where enrolling enough patients to generate significant comparative data is already a challenge.

Granting compassionate use of an investigational compound is a question of both good ethics and good business. Currently, the FDA offers no guarantee that an adverse outcome related to a compassionate use case does not threaten an ongoing clinical trial. At what cost is an individual’s access to therapy worth the ability to bring that therapy to hundreds or thousands of patients in a safe way?

Regardless of how the right-to-try movement plays out, this is an area that clinical research sponsors need to pay attention to. Experts forecast the global orphan drugs market to grow 10.05% annually between now and 2019. Companies like Shire, Baxalta and PTC Therapeutics, who have growth strategies focused on rare diseases, should anticipate an increase in compassionate use requests, and have appropriate decision systems in place. Instead of ad hoc evaluations of requests, they may want to test pilot programs that equitably and transparently distribute an experimental drug outside of its clinical trial program. 

Matthew Howes is executive vice president, Strategy & Growth, for PALIO, an inVentiv Health company. Email matthew.howes@inventivhealth.com.

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