Gottlieb Unveils New Transparency Initiatives
FDA has launched a new pilot to offer more transparency with drug approval decisions. “One place where we are evaluating how we can release information that may better inform scientists, providers, and patients is clinical study reports (CSRs),” FDA commissioner Scott Gottlieb said 1/16. A CSR is the portion of the drug file that “contains detailed summaries of the bottom line information on the methods and results of a clinical trial,” he said in a statement.
The pilot will assess whether disclosing certain information included within CSRs after NDA approval improves public access to drug approval information. “In this pilot, we will select up to nine recently-approved NDA 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,” Gottlieb said. “As part of this effort, we intend to post the parts of the CSRs that were most important to the FDA’s assessment of the safety and efficacy of the drug. Specifically, we’ll include 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 web page on the FDA’s Web site 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.”
Gottlieb announced the pilot during a Johns Hopkins forum, which examined a Journal of Law, Medicine, and Ethics special issue on FDA transparency. He told moderator and former principal deputy director Josh Sharfstein that releasing the CSRs will ultimately make the drug review process more efficient. “Over time,” Gottlieb said, “manufacturers will see inherent value in allowing the CSR reports to be public because a lot of the review memos are cutting and pasting from review documents. The bottom line clinical information that informs the review isn’t very accessible in those documents, and so when we are able to link to the bottom line information in the CSRs it is going to make the development of the review document much more efficient and accessible, and could provide a lot of efficiencies to the review process itself.
The CSR pilot is also part of a broader reform underway in CDER’s Office of New Drugs, where it is trying to come up with a more consolidated review memo. “Right now if you look at review process, a lot of the documents made public at the time of approval are different documents related to the different consultative aspects of the review process,” Gottlieb told the forum. “So there are multiple memos, and we want to work in the direction of a consolidated memo to have a more team-based approach where everyone is working together to develop a consolidated document that explains the agency's philosophy, consensus and the disagreements in one central place, and released upon product approval.”
Gottlieb said the agency will soon be contacting sponsors about participating in the pilot. “We will also continue to protect patient privacy, trade secret, and confidential commercial information in the CSRs we release as part of the pilot,” he said. Once the CSR pilot is complete, FDA will seek public feedback through a Federal Register notice.
Another transparency effort aims to improve the tracking of trials registered on the National Institutes of Health’s database (ClinicalTrials.gov). “Many of the global trials listed on ClinicalTrials.gov relate to research involving new drugs, and may eventually form the basis of an application seeking FDA approval,” Gottlieb said. “Yet right now, tracking a specific clinical trial listed on ClinicalTrials.gov and correlating that trial to its relevance in informing FDA related activities – from advisory committee meetings to FDA approval decisions and to the inclusion of the results of a clinical trial into a drug product’s label – can be challenging.”
FDA is adding the ClinicalTrials.gov identifier number (NCT #) to its materials that support a future FDA approval action. “This number will make it easier to associate the clinical trial listings on ClinicalTrials.gov to FDA communications about specific drugs, including product labeling and even our advisory committee meeting materials,” Gottlieb said. “Members of the patient, academic and scientific communities can easily use this number to identify and track clinical research from a drug’s development throughout the regulatory process. Including this number on FDA materials could greatly benefit all those interested in following the progress of specific clinical research.”
Sharfstein asked Gottlieb to address whether FDA will begin releasing information contained in “complete response letters” issued when the agency needs more information before approving a drug product. He said the agency is evaluating whether it can release certain information about safety concerns that “may reach across a class of drugs or information that could inform different classes of drugs... I'm not sure if this is doable and we have someone going through them to see if there are some identifiable issues. Letters with safety issues could be made public to start.”
Gottlieb told the forum that there could be a way to get to “the subset of letters important to public health, for example: where we are asking for additional clinical trial data related to a safety issue where there are other marketed drugs that may have some relevancy. I don’t want to be eating a lot of resources to redact these documents just for purposes of market efficiency and this has to have some bottom line value for patients.”