Patient Care Access News

FDA Must Clarify Outcomes Data Use in Expanded Access Drug Program

The GAO reported call on FDA to make outcomes data clarifications in its expanded access drug program.

outcomes data expanded access drug program

Source: Thinkstock

By Sara Heath

- FDA must communicate clearer intent for outcomes data use in its expanded access program. This will ensure that drug manufacturers participate in the program and offer broader treatment access to extremely sick patients, according to a new Government Accountability Office report.

The FDA expanded access program helps patients with life-threatening and serious illness with no comparative treatment options obtain access to investigational drugs. The FDA and drug manufacturing companies carry out an extensive application process to deliver these treatments to patients.

FDA collects outcomes data from expanded access participants primarily related to adverse events. Currently, FDA does not provide sufficient clarification for its outcomes data use, the report found.

“Several stakeholders we spoke with, including the selected manufacturers we interviewed, raised concerns that FDA is not clear about how it uses expanded access adverse events data in its review of drugs being considered for sale and marketing in the United States,” GAO reported.

FDA maintains that it rarely uses that data during clinical trials because the expanded access program does not have the same controls as a typical clinical trial.

That fact notwithstanding, a review of communication materials between FDA and drug manufacturers showed that FDA’s guidance and transparency on drug data use is not sufficient. This can damage the intent of the program, GAO and stakeholders explained.

“These manufacturers noted that the lack of clear information can influence their decision whether to give patients access to their drugs, because of their concerns that an adverse event will result in FDA placing a clinical hold on their drug, which could delay its development,” GAO stated.

“This could impact FDA’s goal of facilitating expanded access to drugs for treatment use by patients with serious or life-threatening diseases or conditions, when appropriate.”

GAO recommended FDA simplify its data use standards and spell out its protocol for using expanded access data.

This current report built upon a previous GAO report published in July 2017. The previous report noted the efficacy FDA had in implementing the expanded access program and ensuring critically ill patients had access to investigational medications that could be life-saving.

GAO found that the agency obliged 99 percent of the nearly 5,800 applications it received. Almost 96 percent of those granted requests came from individual patients, and the rest came from groups of patients with like ailments requesting the same drug.

Per federal regulations, FDA has a 30-day window in which it can respond to expanded access requests. FDA usually took a few hours to respond to applications, but in cases where it did take longer, the wait period always fell within the 30-day period.

When the FDA did not grant access to the investigational drug, it was usually because of:

  • Incomplete applications
  • Unsafe dosing
  • Lack of efficacy for drug’s proposed use
  • There were adequate available treatments
  • Inadequate information provided on completed applications

GAO concluded the July 2017 report by commending FDA for its efforts in implementing the expanded access program. Additionally, GAO praised FDA and its partners for improving the program through administrative simplification.

States and the FDA have taken steps to improve the expanded access programs. Some states have enacted “Right to Try” laws, which in most cases lets terminal patients try investigational drugs as a last-ditch effort.

In response to both reports, FDA agreed with GAO’s recommendations and stated it would work to fulfill the suggestions.


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