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Regulatory Focus™ > News Articles > 2021 > 4 > Rare diseases, STAR expansion considered in PDUFA VII premarket negotiations

Rare diseases, STAR expansion considered in PDUFA VII premarket negotiations

Posted 16 April 2021 | By Kari Oakes 

Rare diseases, STAR expansion considered in PDUFA VII premarket negotiations

Newly released minutes from meetings between industry and the US Food and Drug Administration (FDA) give observers more insight into premarket review issues in play during the negotiations for the reauthorization of the Prescription Drug User Fee Act (PDUFA VII).
FDA and industry looked at commitment language related to new molecular entity (NME) milestones and postmarketing requirements (PMR) in a 27 January meeting of the premarket negotiations subgroup. For both standard and priority NME new drug applications and original biological license applications, FDA is agreeing to formalize pre-approval communication timelines as commitments under PDUFA VII, rather than regarding these timelines as best practices.
The subgroup also discussed how to review existing (PMR) during the post-approval phase, considering timelines for release from PMR and what information FDA should include in its decision letters.
Rare diseases; RWE
As part of an ongoing focus on rare diseases, FDA and industry discussed a proposed pilot program to identify novel endpoints for rare diseases, envisioning “potentially a limited number of common disease programs with innovative endpoints that have applicability to rare diseases,” according to the 21 January minutes. FDA had requested resources for this pilot project, and sought to include information about the process and the criteria used to select novel endpoints in the commitment language. By 27 January, FDA had scaled back its resource request for this area, “which would correspondingly reduce the program’s outputs,” noted the minutes of that date.
As industry and FDA continue to ponder how to incorporate real-world data and real-world evidence into the clinical trial and presubmission process, they envision a pilot program “to develop new methods and knowledge” to flesh out how real-world data can play into regulatory decisions. Even in the scaled-back version of the pilot, FDA is still contemplating that its biologics and drug evaluation branches would host an unspecified number of public workshop to address the issue.
Combo products
Another rapidly evolving area, combination products, also received attention during the 21 January meeting. Here, FDA is looking for resources to bolster its review of use-related risk analysis and human factor protocol reviews to enable the agency to stay on the 60-day review timeline for human factor protocols that was established in PDUFA VI. “Industry noted preliminary agreement” with FDA’s resource request, according to the 21 January minutes.
On 27 January when the premarket subgroup reconvened, FDA clarified that human factor protocols already have available guidance for industry and are currently evaluated without an accompanying use-related risk analysis.
Both the biologics and drug evaluation arms of FDA need to boost capacity and expertise in bioinformatics so that the agency can “provide detailed and consistently timed feedback to Industry earlier in the development cycle,” agreed the parties during the 21 January meeting. Negotiators are envisioning a suite of guidance documents addressing bioinformatics, with a particular emphasis on international harmonization of standards and methodologies used by regulators and sponsors.
STAR program extension
Finally, the January negotiations picked up a thread started in December 2020 related to real-time application review. Both FDA and industry are interested in expanding the Split Real-Time Application Review (STAR), currently available for oncology therapeutics, into all therapeutic areas. The program speeds review of certain efficacy supplements.
27 January premarket subcommittee minutes
21 January premarket subcommittee minutes


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