GAO Report: Drug Companies Making Progress Expanding Drug Availability Beyond Trials

Federal regulators seek comments on draft guidance for clinical investigations involving children

Drug manufacturers are reporting positive strides in terms of increasing access to investigational drugs outside clinical trials, according to a Government Accounting Office (GAO) report issued Monday (September 9).

Of the 29 manufacturers interviewed, 23 posted their expanded access policies on their websites; four said they wouldn’t consider expanded access requests. The U.S. Food and Drug Administration (FDA) has been pushing for greater access as long as safety considerations are met, among other factors.

“Questions have been raised in recent years about whether clinical trial eligibility criteria are too narrow and exclude patients who are likely to be treated once a drug is approved, and FDA has historically provided guidance to manufacturers to help them consider the circumstances under which they could broaden these criteria without compromising study results or raising ethical issues,” the GAO report said.

Drug manufacturers also took the opportunity to promote clinical trials themselves. Officials from two drug manufacturers responded to the study and said broader eligibility criteria will allow more patients to access investigational drugs through clinical trial participation. It can also, officials said, help them obtain FDA approval for a drug that extends to a wider range of patients, if the drug is found to be safe and effective.

Further, officials from one of the two manufacturers noted that broader eligibility criteria, such as criteria that include patients with infections, could help streamline the process for conducting clinical trials—for example, by eliminating the need to conduct clinical testing to screen for the presence of infections.

Although most drug manufacturers in the review did not report efforts to broaden their eligibility criteria, many noted efforts to address other barriers to clinical trial participation. For example, to address geographic barriers, officials from six of the 10 manufacturers said they help cover costs for patients to travel to clinical trial sites, such as by reimbursing transportation and hotel costs for patients who travel long distances.

In addition, officials from one manufacturer said they completed a pilot clinical trial on diabetes in 2019 that used decentralized trial locations in three states, such as retail health clinics and patients’ homes, to help patients overcome challenges with obtaining transportation to trial sites.

Similarly, within the next two years, another manufacturer is planning to conduct a pilot clinical trial that is fully remote and expects the design to improve patient participation in rural communities.

To address the lack of information about upcoming and ongoing clinical trials that is available and tailored to patients, two manufacturers launched clinical trial registries in 2015 and 2016.

Author: Michael Causey