FDA Releases First Clinical Study Report in Data Transparency Pilot Program
The FDA published its first batch of clinical data summary documents under a transparency pilot program launched earlier this year.
By making portions of CSRs publically available, the FDA hopes to increase stakeholder understanding of the basis for the agency’s approval decisions, and enhance the accuracy of information used in scientific publications, said CDER Director Janet Woodcock. The FDA plans to post up to nine total CSRs from recently approved NDAs volunteered by participating sponsors across a range of disease areas.
“As an added benefit, our pilot program can help with global alignment, as our counterparts at the European Medicines Agency are similarly working to make information about their approvals more accessible and easier to understand,” Woodcock said.
The EMA launched its clinical data transparency program, which publishes clinical study reports and protocols from all new drug submissions, regardless of approval status, in October 2016 (CWWeekly, Oct. 24, 2016). At the one-year anniversary of the EMA’s initiative, the agency had published more than 3,000 clinical documents covering 50 medicines, totaling 1.3 million pages. The publicly available database also registered more than 3,600 users.
The data from the FDA’s first release covers the pivotal trial for Janssen Biotech’s Erleada (apalutamide) — approved in February as the first drug to treat non-metastatic, castration-resistant prostate cancer, and the first to use the endpoint of metastasis-free survival.
The released portions of Janssen’s clinical study report include complete summaries of the trial’s results, as well as its protocol, amendments and statistical plan, totaling about 1,000 pages.
The pilot program redacts or excludes confidential commercial information, trade secrets and personal privacy information, as well as patient-level data, Woodcock said. Previously, the FDA’s drug approval packages and clinical trial snapshots typically included the agency’s review letters and product labeling, as well as breakdowns of participant demographics.
“I am impressed with the release of such a data set so soon after Scott Gottlieb’s announcement of FDA’s proposal,” said David Forster, chief compliance officer of WIRB-Copernicus Group.
During his announcement of the project in January, FDA Commissioner Gottlieb said the disclosures can help sponsors address challenges in development, identify areas that may require additional postmarket research and generate the data necessary for approval.
In addition, the agency hopes to use the pilot to streamline CDER’s clinical review processes, by consolidating disparate staff review documents into a single memo (CWWeekly, Jan. 22).
In the Phase III trial, Erleada, an antiandrogen, showed a robust effect in its novel primary endpoint of prolonging the time until metastasis or death, according to Richard Pazdur, director of the FDA’s Oncology Center of Excellence. “This demonstrates the agency’s commitment to using novel endpoints to expedite important therapies to the American public,” he said. Erleada was granted priority review.
The placebo-controlled, multi-center trial — launched in October 2013, with last patient, last visit being completed in May 2017 — randomized a total 1,207 patients 2:1, demonstrating a median metastasis-free survival of 40.5 months in the Erleada arm, compared to 16.2 months in the placebo arm.
Erleada’s full approval package is available here: www.accessdata.fda.gov/drugsatfda_docs/nda/2018/Erleada_210951_toc.cfm.