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Regulatory Focus™ > News Articles > PDUFA VI Reauthorization: Industry, FDA Discuss Broader Use of Real World Evidence

PDUFA VI Reauthorization: Industry, FDA Discuss Broader Use of Real World Evidence

Posted 23 November 2015 | By Zachary Brennan 

Representatives from industry groups BIO and PhRMA met with officials from the US Food and Drug Administration (FDA) twice last month to discuss new plans for using real world evidence to support benefit-risk assessments for medical products, according to the meeting minutes of a subgroup of the sixth reauthorization of the Prescription Drug User Fee Act (PDUFA).


Congress created the Prescription Drug User Fee Act (PDUFA) in 1992 to help FDA collect fees from companies that produce certain human drug and biologics to further expedite the drug approval process.

PDUFA must be reauthorized every five years, and was renewed in 1997 (PDUFA II), 2002 (PDUFA III), 2007 (PDUFA IV), and 2012 (PDUFA V). 

The current legislative authority for PDUFA expires in September 2017, so FDA, industry and Congress have until then to forge new agreements on what FDA should spend the funds on and what the agency should focus its efforts on. Separate legislation governing user fees for medical devices, biosimilars and generic drugs are also ongoing.

So far, the latest negotiations from September and October have involved: two stakeholder meetings (with various patient advocacy groups and other stakeholders), three steering committee meetings, three finance subgroup meetings, two post-market subgroup meetings, one regulatory decision tools subgroup meeting and an IT subgroup meeting.

Post-Market Subgroup

The first meeting on 7 October  -- between five CDER officials and three representatives from BIO (including Alexion and Alkermes employees) and two representatives from PhRMA (including an Eli Lilly employee) – introduced the proposals from both sides on real world evidence.

Industry called for using real world evidence in regulatory decisions for both safety and effectiveness.

“The plan calls for a regulatory science initiative to determine the benefits of and methodologies available for use of RWE [real world evidence] in this context, as well as the gaps that need to be overcome to achieve the goal of broader use of RWE in the post-market setting,” the minutes say.

FDA proposes to increase available pharmacovigilance data sources by expanding the capacities of the Sentinel System, which currently queries a group of automated healthcare data holders—like electronic health record systems, administrative and insurance claims databases, and registries—to evaluate possible medical product safety issues.

“The proposal seeks to enhance the Sentinel System by expanding the types of available data, and building new analytic methods. It also aims to fully integrate Sentinel into the existing FDA post-market safety regulatory review apparatus,” the minutes say.

At the second meeting a week later, FDA and industry voiced concern about the implications for using certain data sources such as social media for drug-safety reporting purposes.

Industry also discussed “a literature compendium completed to document published articles that describe uses of real world evidence (RWE), specifically in supporting benefit-risk assessment.”

They also mentioned specific case studies of the use of “RWE in benefit/risk assessment, and planned to present more details at a future meeting. Industry reiterated their view that, given significant advances in data analysis and available data, RWE likely has a role to play in augmenting or possibly replacing traditional data sources for benefit-risk decision making in the post-market setting.”

FDA Hiring, Other Notes

The meeting minutes from the steering committee hearing on 13 October also noted that FDA provided “a high-level overview of the current challenges FDA faces in hiring qualified individuals and presented a strategy to enhance targeted recruitment, onboarding, and retention of scientific personnel. Industry expressed support for FDA’s proposal.”

At the Regulatory Decision Tools working group on 7 October, both sides also discussed the potential for requiring new FDA guidance “to address a wide range of challenging methodological and process issues related to advancing the science of patient input.”

And at the Finance subgroup meeting on 14 October, FDA emphasized “the uncertainty with how much funding the workload adjuster will produce during a 5-year period and its goal of predictable funding in PDUFA VI. FDA explained obstacles to hiring new staff when funding for subsequent years is not guaranteed. Industry expressed concern about how the workload adjuster is applied and how funding generated from the workload adjuster does not appear to be allocated to the offices that manage the workload represented in the adjuster.”

PDUVA VI Negotiations


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