United States: US FDA Launches New Pilot Program aiming at Enhanced Transparency About New Drug Approvals

The United States Food and Drug Administration (USFDA) on March 19, 2018, launched its pilot project to publicly release portions of clinical trial-related summaries from pivotal trials46, with initial data coming from the recently approved Erleada (Apalutamide), meant for patients with prostate cancer that has not spread. Erleada (Apalutamide) is from Janssen Biotech and was approved by the agency in February 2018.

Clinical Data Summary Pilot Program

The pilot program will include up to nine recently submitted new drug applications (NDAs). For these applications (if approved), Center for Drug Evaluation and Research (CDER) will release portions of the clinical study reports (CSRs) from the sponsor's NDA along with the action package following approval. The CSRs are summaries of clinical information used in making market approval decisions and will be included in the information posted at the drug's entry at Drugs@FDA47.

During the pilot phase, the agency will post key portions of the Clinical Study Reports (CSRs) – documents that sponsors create for FDA on each of their clinical studies. These portions will contain complete summaries of the study results, the protocol and protocol amendments, and the statistical plan. The agency also said that it plans to release these portions of the CSRs for the pivotal studies that supported the approval. The reports will be redacted by the agency to exclude confidential commercial information, trade secrets, and any privacy information. Further, the agency will not release patient-level data. The agency's goal is to share more directly complete summaries of the clinical trial information we have evaluated to determine whether a drug is safe and effective.

Currently the agency posts its review documents online – through material called drug approval action packages. While the action packages include a significant amount of information pulled in from the sponsor's application, the information is frequently separated into different sections and does not provide a complete summary of the results of any given study. This makes it difficult for academic researchers, regulators in other agencies, and other stakeholders to gain an in-depth understanding of the studies supporting approval. By providing the CSRs the agency hopes to:

  • Enhance the accuracy of information used in scientific publications;
  • Increase stakeholders' understanding of the basis for FDA's approval decisions; and,
  • Inform physicians and other healthcare providers about the detailed results that regulatory decisions were based on.

During this program, the FDA pilot will post the CSRs from up to 9 approved new drug applications of participating sponsors. The agency hopes that reviewing the CSRs will help the scientific community better understand the information FDA used to evaluate an application and make an approval decision. At the end of the pilot phase, the agency will seek comment from the public through a Federal Register notice to hear first-hand how the information was accessed and used.

 The first pilot participant is Janssen Biotech for the approval of Erleada (apalutamide), the first FDA-approved treatment for non-metastatic, castration-resistant prostate cancer, as well the first to use the clinical trial result, or endpoint, of metastasis-free survival48.

The agency has posted the CSR of the pivotal study with the regular action package. Over 1000 pages of Janssen's partially redacted clinical study reports for Erleada have been published, including information on the trials' protocols and statistical analysis plans. Erleada (apalutamide) is a novel drug and the agency believes that the CSR information, together with the FDA review, label, and other supporting documents, will facilitate a deeper understanding of how the agency reached the approval decision.

The agency also believes that this pilot program can help with global alignment, as the European Medicines Agency is also working similarly, to make information about their approvals more accessible and easier to understand through their clinical data publication program.

Conclusion:

Through this pilot program the agency's goal is to directly share complete summaries of the clinical trial information that it evaluates to determine whether a new drug is safe and effective. The Clinical Summary Pilot is the agency's effort towards enhancement of transparency about new drug approvals.

Footnotes

46 https://www.fda.gov/Drugs/DevelopmentApprovalProcess/ucm589210.htm

47 https://www.fda.gov/Drugs/DevelopmentApprovalProcess/ucm589210.htm

48 https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm596768.htm

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