FDA Moving to Enhance Drug Approval Transparency, Gottlieb Says

New pilot program to evaluate disclosure of clinical study reports

As part of its efforts to enhance transparency around drug approval decisions, the FDA is exploring ways it can continue to build on its obligation to share information, says FDA Commissioner Scott Gottlieb, MD.

The agency is especially focused on information that can improve patient care and better inform providers about the products they prescribe. One place where it is evaluating how it can release information that may better inform scientists, providers, and patients is clinical study reports (CSRs), Dr. Gottlieb wrote in a statement.

Currently, when the FDA approves a drug, the agency releases certain information that it used when reviewing the new drug application (NDA). This includes summaries written by its medical reviewers that capture their assessment of the data, the proposed labeling or other requirements, and other important, relevant data supporting safe and effective use. This information is included in the agency’s drug approvals database, Drugs@FDA.

These summaries provide important context on the basis for FDA approval decisions. But they are packaged in a format that can sometimes make it difficult for external audiences to extract all of the detailed clinical evidence that supported the FDA’s approval decisions, according to the statement.

The FDA has launched a pilot program to evaluate whether disclosing certain information included within CSRs following approval of an NDA improves public access to drug approval information. In this pilot, the agency will select up to nine recently approved NDAs across a range of disease areas whose sponsors volunteer to participate and post portions of clinical trial-related summaries from the pivotal trials that were submitted to the FDA by the drug’s sponsor on Drugs@FDA.

The FDA intends to post the parts of the CSRs that were most important to the agency’s assessment of the safety and efficacy of the drug, specifically, the study report body, the protocol and amendments, and the statistical analysis plan for each of the participating product’s pivotal studies. The CSRs will be posted on a new page on the FDA’s website that describes the pilot program, in addition to appearing on Drugs@FDA along with the drug’s approval information, soon after a drug is approved.

The FDA intends for initial participation in the pilot to reflect products that are novel (including drugs that are new molecular entities) and of scientific interest (e.g., certain NDA efficacy supplements). The agency will begin contacting sponsors “soon” to see if they are interested in participating in the pilot. The FDA will continue to protect patient privacy, trade secrets, and confidential commercial information in the CSRs that are released as part of the pilot.

Once the new program is complete, the FDA will seek public feedback through a Federal Register notice and docket for public comments.

Dr. Gottlieb’s full statement is available at: www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm592566.htm.

Source: FDA; January 16, 2018.