Drugmakers Support FDA’s Expanded Trial Eligibility Plan, GAO Says

September 16, 2019

Drug manufacturers are making progress toward the FDA’s goal of increasing access to investigational drugs outside clinical trials, the GAO says in a report released last week.

Of 29 manufacturers GAO surveyed, 23 companies reported having an expanded access policy and only four stated they would not consider expanded access requests.

One of the four sponsors said it wanted to ensure its investigational drugs were administered to patients only through clinical trials where safety could be closely monitored. Another cited limited resources, stating that the company chose to
focus resources solely on conducting clinical trials.

Among the 19 manufacturers willing to consider requests for investigational drugs outside of clinical trials, 18 stated that patients must have a serious or life-threatening disease or condition with no comparable therapies available and have been ineligible for a clinical trial of the requested drug.

Seventeen stated that the treating physician must determine for the patient seeking access that the risk of taking the investigational drug is not greater than the anticipated benefit. Ten sponsors stated that supply of investigational drugs was a consideration.

Five sponsors referenced specific drugs they would consider providing, including three investigational drugs for bladder cancer, influenza and HIV from one manufacturer.

Reaction was mixed among drugmakers to the agency’s plan to broaden trial eligibility. Most reported taking steps to open trials to wider populations, as recommended in four draft guidances the FDA issued in March (CenterWatch Weekly, March 18, 2019).

But some respondents expressed concern that broadening eligibility could adversely affect a study’s ability to identify the effects of a drug. According to four drugmakers responding to GAO, “Broader criteria must be carefully balanced with the need to collect evidence from a well-defined population,” the report says.

Another objection raised, GAO reports, is that “removing standard exclusion criteria, such as excluding patients who use other medications, could interfere with the success of their clinical trial if those medications make it difficult to identify the effects of the studied drug.”

But two sponsors said they agree that broader eligibility criteria will allow more patients access to investigational drugs through clinical trial participation and said they would follow the agency’s lead because “it will facilitate the drug approval process.”

Most manufacturers did not report efforts to broaden eligibility criteria specifically, but many said they were taking other steps to increase inclusion in trials, such as covering patients’ travel costs, and establishing decentralized trial locations convenient to patients.

One sponsor said it completed a pilot clinical trial on diabetes this year that decentralized trial locations across three states. Yet another manufacturer is planning to conduct a clinical trial that is fully remote in the next two years.

Manufacturers also are planning to better involve historically underrepresented groups in clinical trials, with one manufacturer conducting workshops to train minority investigators. Sponsors also are considering locating trial sites in areas with minority populations of more than 25 percent.

Read the full report here:

By Colin Stoecker