Earlier this year, in one of his first public addresses, FDA Commissioner Scott Gottlieb discussed the agency's on-going “Blueprint for Transparency” initiative.
This week the FDA officially launched its pilot project to publicly release portions of clinical trial-related summaries from pivotal trials, with initial data coming from Janssen's recently approved Erleada (apalutamide), which is for patients with prostate cancer that has not spread.
About 1,000 pages of Janssen's partially redacted clinical study reports for Erleada were published, including information on the trials' protocols and statistical analysis plans. A Janssen spokesperson told Focus that the company's interest in such a transparency initiative and in furthering other research efforts coincided with its submission for Erleada. The spokesperson also said Janssen did not redact the study report or the protocol and also did not review the FDA assessment report prior to its posting.
Up to eight more recently approved new drug applications (NDAs), whose sponsors volunteer to participate, are expected to be a part of the pilot program.
Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, wrote in a blog post that by releasing the clinical study reports, 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.
The FDA has long said it is sharply limited in what information it can release because it often is dealing with drug companies' proprietary material.
That may be changing. In January, the agency started a pilot program to release clinical study reports for recently approved drugs. These summaries, which are generated by drug-company sponsors of the treatments, spell out the methods and results of clinical trials. The data don't include patient-identifiable information.
The release of the study reports, which can run hundreds of pages, will allow researchers and others (per Gottlieb) “to do more analysis around our decision-making,” especially on the safety and efficacy of new drugs. Some of the information is already released by the agency but in a format that is difficult for lay audiences to use. So, in some respects “transparency” will also mean “more user-friendly. One key question to ponder then is, “more user-friendly” for whom?
The FDA is also going to make it easier to track clinical-research information by adding a study's identifier number from ClinicalTrials.gov to all FDA materials for a specific product. ClinicalTrials.gov is the database of studies maintained by the National Institutes of Health. This has been long discussed and is long over-due.
On another transparency issue, Gottlieb said the agency is exploring whether there is a “subset” of “complete response letters” (CRLs) that can be released. Such letters to drug companies detail why their drugs were not approved. He said the FDA is looking at possibly releasing information involving safety issues. Critics of the pharmaceutical industry have long complained that the companies don't always give the public accurate and comprehensive explanations of why their products were rejected. It's also been a thorn in the side of agency reviewers who are regularly blamed by companies when they receive CRLs. The release of (properly redacted) CRLs will ensure that the reasoning behind them (and the appropriate assignation of blame) will be more balanced.
Gottlieb understands that regulatory transparency cannot be a “for thee but not for me” proposition. Per the Commissioner, “We should be making sure that we try to provide as much information back into the market of ideas as possible. There are places across this agency where we bottle up too much information.”
He singles out complete response letters as a “place where we should ask hard questions because there's some very important information in those communications.” Releasing this information could advance public health, for example by publicizing a safety threat, or by helping other companies avoid known pitfalls and thereby get drugs onto the market more quickly.
This week the FDA officially launched its pilot project to publicly release portions of clinical trial-related summaries from pivotal trials, with initial data coming from Janssen's recently approved Erleada (apalutamide), which is for patients with prostate cancer that has not spread.
About 1,000 pages of Janssen's partially redacted clinical study reports for Erleada were published, including information on the trials' protocols and statistical analysis plans. A Janssen spokesperson told Focus that the company's interest in such a transparency initiative and in furthering other research efforts coincided with its submission for Erleada. The spokesperson also said Janssen did not redact the study report or the protocol and also did not review the FDA assessment report prior to its posting.
Up to eight more recently approved new drug applications (NDAs), whose sponsors volunteer to participate, are expected to be a part of the pilot program.
Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, wrote in a blog post that by releasing the clinical study reports, 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.
The FDA has long said it is sharply limited in what information it can release because it often is dealing with drug companies' proprietary material.
That may be changing. In January, the agency started a pilot program to release clinical study reports for recently approved drugs. These summaries, which are generated by drug-company sponsors of the treatments, spell out the methods and results of clinical trials. The data don't include patient-identifiable information.
The release of the study reports, which can run hundreds of pages, will allow researchers and others (per Gottlieb) “to do more analysis around our decision-making,” especially on the safety and efficacy of new drugs. Some of the information is already released by the agency but in a format that is difficult for lay audiences to use. So, in some respects “transparency” will also mean “more user-friendly. One key question to ponder then is, “more user-friendly” for whom?
The FDA is also going to make it easier to track clinical-research information by adding a study's identifier number from ClinicalTrials.gov to all FDA materials for a specific product. ClinicalTrials.gov is the database of studies maintained by the National Institutes of Health. This has been long discussed and is long over-due.
On another transparency issue, Gottlieb said the agency is exploring whether there is a “subset” of “complete response letters” (CRLs) that can be released. Such letters to drug companies detail why their drugs were not approved. He said the FDA is looking at possibly releasing information involving safety issues. Critics of the pharmaceutical industry have long complained that the companies don't always give the public accurate and comprehensive explanations of why their products were rejected. It's also been a thorn in the side of agency reviewers who are regularly blamed by companies when they receive CRLs. The release of (properly redacted) CRLs will ensure that the reasoning behind them (and the appropriate assignation of blame) will be more balanced.
Gottlieb understands that regulatory transparency cannot be a “for thee but not for me” proposition. Per the Commissioner, “We should be making sure that we try to provide as much information back into the market of ideas as possible. There are places across this agency where we bottle up too much information.”
He singles out complete response letters as a “place where we should ask hard questions because there's some very important information in those communications.” Releasing this information could advance public health, for example by publicizing a safety threat, or by helping other companies avoid known pitfalls and thereby get drugs onto the market more quickly.
