The Office of Information and Regulatory Affairs (OIRA), the US government regulator of regulations, has announced its approval of a new report set to be published in two years by the US Food and Drug Administration (FDA) regarding its evaluation of its efforts to expedite the review of new molecular entities (NMEs), and original biologics license applications (original BLAs).
Under the terms of the fifth iteration of the Prescription Drug User Fee Act (PDUFA V), passed under the FDA Safety and Innovation Act (FDASIA), FDA agreed to establish a new review program aimed at improving transparency and communication between itself and members of industry.
Those goals, contained within Section II(B) of the PDUFA V commitment letter signed by FDA, describes assessment procedures meant to look back on the submission and review process associated with NMEs submitted via a new drug application (NDA) or original BLAs.
With the help of an independent contractor acting on behalf of FDA, the agency said it wants to collect and evaluate metrics regarding its performance in relation to PDUFA V's goals, including sending Day 74 letters, timing, handling of amendments, communications and its outlining of potential issues.
"A key aspect of the Program is an interim and final assessment that will evaluate how well the parameters of the Program have achieved the intended goals," FDA explained in a Federal Register posting due for publication on 19 February 2013.
The interim assessment is due by 31 March 2015, and is meant to allow for mid-cycle corrections of PDUFA V if the report determines that either industry or FDA isn't living up to its stated commitments. The second, final assessment is meant to drive discussions into the (likely) PDUFA VI reauthorization negotiations, which generally start about two years before the end date of the current PDUFA program. That report is due by 31 December 2016.
In February 2013, FDA announced that it had selected Eastern Research Group (ERG), a Virginia consulting firm, to generate the report.
And the report, FDA has explained, is set to function much as a series of exit interviews, with ERG conducting "independent interviews of applicants after FDA issues a first-cycle action for applications reviewed under the program."
Industry reaction to the report so far seems to be positive-not all that surprising given that it was industry that called for the process to begin with. But that doesn't mean its support has been without some concern.
In comments submitted to FDA's docket and relayed on to OIRA, the Pharmaceutical Research and Manufacturers of America (PhRMA) wrote that both ERG and FDA will need to "ensure that appropriate metrics are captured during the interview," but do so in such a way as to ensure the confidentiality of trade secrets and ensure the protection of commercially confidential information.
"FDA and ERG should ensure that any information obtained during the interviews is protected in accordance with the applicable confidentiality agreements," PhRMA wrote. "Applicant responses should be anonymized and aggregated prior to inclusion in the independent assessments and any presentation materials at public meeting to ensure the protection of applicants' confidential commercial information and trade secrets."
Study Details and Approval
In its own comments to OIRA, FDA said it agreed with PhRMA's comments with respect to commercially confidential information, stating that "ERG will not ask applicants for any confidential business information."
By the same token, however, "nor will ERG ask applicants to evaluate the performance of FDA staff."
"Instead, interviews will focus on applicant experiences with the new review process under the Program."
All data collected by ERG will be anonymized and aggregated to promote truthful responses, which it said may be presented at public meetings.
The plan calls for interviewing all NDA and BLA sponsors, of which there are an estimated 40 to 45 each year. Each interview will take approximately 1-1.5 hours to conduct and involve anywhere between one and three representatives from each sponsor, to be interviewed at the same time.
Data collection will involve a pre-test of five respondents, presumably to make sure the process is collecting the information it needs to, followed by an estimated 135 full interviews during the three-year process.
On 15 August 2013, OIRA announced that it has formally approved FDA's request to begin the study, meaning it will likely begin the pre-testing phase shortly because ramping up into its full exit interviews.
The exact interview questions can be found on OIRA's website.
Federal Register Announcement
Interview Questions (.docx format)