First CSR Data Published in FDA's Clinical Data Summary Pilot Program

The FDA’s Center for Drug Evaluation and Research (CDER) has launched its pilot to publicly release data from pivotal clinical trials as part of a greater push for agency transparency in the approval of new drugs. The first product to have its pivotal trial information released is Janssen Biotech’s apalutamide (Erleada), an androgen receptor inhibitor indicated for the treatment of patients with non-metastatic castration-resistant prostate cancer, which was approved by the FDA in February 2018.

The pilot will include up to a total of 9 recently submitted new drug applications. If these applications are approved, CDER will release portions of the clinical study reports (CSRs) from the sponsors’ New Drug Applications. The CSR information—which comprises the study report’s body, the study’s protocol and amendments, and the statistical analysis plan—will be included in the information posted at the drug’s entry at the agency’s Drugs@FDA website.

In an FDA Voice blog post announcing publication of apalutamide’s data, Janet Woodcock, MD, director of CDER, wrote, “Many factors are involved in weighing the benefits a drug can provide against the risks associated with its use. To that end, after we approve a new drug, we also want to make sure the scientific community and the public can understand why we approved it. This can help inform future drug development and, in turn, may facilitate the approval of additional safe and effective medicines.”

With apalutamide in particular, Woodcock added, “It’s a novel drug and we believe the CSR information, together with the FDA review, label, and other supporting documents, will facilitate a deeper understanding of how we reached our approval decision.”

The transparency program was first announced in January 2018 by FDA Commissioner Scott Gottlieb, MD, who said that, once the pilot program is complete, the agency will seek public feedback through a Federal Register notice and a docket for public comments.

In addition to helping the public better understand the rationale for drug approval, the pilot program is intended to help with global regulatory alignment, as the European Medicines Agency (EMA) is working on a similar task to make approvals more accessible and easy to understand.

The EMA’s initiative entered into force in January 2015, and the agency publishes clinical data submitted by manufactures in support of their requests for marketing authorization. These data, reviewed by the Committee for Medicinal Products for Human Use, include a clinical overview, a clinical summary, study reports on individual clinical studies, and 3 appendices, including study protocol, a sample case report form used to collect data on individual patients, and documentation of statistical methods used to analyze study data.