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Federal Register / Vol. 86, No. 161 / Tuesday, August 24, 2021 / Notices
3. Advancing Real-World Evidence for Use in Regulatory Decision Making In accordance with Section 3022 of the 21st Century Cures Act, and by providing earlier and increased Agency advice, FDA proposes a new pilot program around real-world evidence RWE to improve the quality and acceptability of RWE-based approaches in support of new intended labeling claims, including approval of new indications of approved medical products or to satisfy post-approval study requirements.
E. Enhancing Regulatory Decision Tools To Support Drug Development and Review Building on the success of PDUFA VI, the enhancements under this section focus on enhancing regulatory decision tools to support drug development and review in the areas of patient focused drug development, benefit-risk assessment in regulatory decision making, drug development tools for qualification pathway for biomarkers, model-informed drug development, and complex innovative clinical trial designs. The details of these enhancements can be found in section I.L of the proposed PDUFA VII
commitment letter.

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F. Enhancement and Modernization of the FDA Drug Safety System FDA will continue to utilize user fees to enhance the drug safety system, including adopting new scientific approaches, improving the utility of existing tools for the detection, evaluation, prevention, and mitigation of adverse events, modernizing REMS
assessments, and coordinating regulatory activity in the premarket and postmarket settings. Enhancements to the drug safety system will improve public health by increasing patient protection while continuing to enable access to needed medical products.
Specifically, PDUFA VII user fees will provide support for modernization and improvement of REMS assessments and optimization of the Sentinel Initiative https www.fda.gov/safety/fdassentinel-initiative through: 1
Maintenance of Sentinel Initiative capabilities and continued integration into FDA drug safety activities and 2
enhancement of the analytic capabilities of the Sentinel Initiative to address questions of product safety and advance the understanding of how RWE can be used for studying effectiveness. These enhancements are described in section I.M of the proposed PDUFA VII
commitment letter.

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G. Enhancements Related to Product Quality Reviews, Chemistry, Manufacturing, and Controls Approaches, and Advancing the Utilization of Innovative Manufacturing Technologies To ensure new and innovative products are developed and available to patients in a timely manner, FDA
proposes several enhancements related to communication between FDA and sponsors during product quality reviews, CMC approaches, and advancing use of innovative manufacturing technologies.
For product quality reviews, these enhancements would include promoting the use of structured information requests, a third-party assessment on current practices related to information requests, and a goal to notify sponsors of certain pre-approval inspections.
Given the accelerated development of certain human drug products, FDA also proposes a new pilot program to facilitate the expedited CMC
development of products under an IND
based upon the anticipated clinical benefit of earlier patient access to products. Additionally, FDA proposes holding a public workshop to help advance utilization and implementation of innovative manufacturing by facilitating and discussing best practices, barriers, and overall strategies. These enhancements are described in section I.N of the proposed PDUFA VII commitment letter.
H. Enhancing CBERs Capacity To Support Development, Review, and Approval of Cell and Gene Therapy Products To ensure that new and innovative cell and gene therapy products are developed and available to patients in a timely manner, FDA proposes to build on the success of the Cell and Gene Therapy Program CGTP in CBER to further support and advance a balanced approach to product development and regulation. To this end, FDA will strengthen staff capacity and capability to meet the increasing challenges and demands in this growing field.
Increasing staff capacity will overcome existing resource limitations, allowing staff to spend additional time on meetings and submission reviews including those with breakthrough or regenerative medicine advanced therapy designations, expand stakeholder outreach, invest in new policy and guidance, and facilitate development and use of regulatory tools and scientific technologies. These enhancements are described in section I.O of the proposed PDUFA VII commitment letter.

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I. Supporting Review of New Allergenic Extract Products FDA proposes to incorporate and include new allergenic extract products into the PDUFA program. Allergenic extract products licensed after October 1, 2022, would generally be included in user fees. Allergenic extract products licensed before October 1, 2022, and standardized allergenic extract products submitted pursuant to a notification to the applicant from the Secretary of Health and Human Services regarding the existence of a potency test that measures the allergenic activity of an allergenic extract product licensed by the applicant before October 1, 2022, would remain excluded from PDUFA.
All performance goals, procedures, and commitments in this letter apply to the allergenic products included in the PDUFA program under PDUFA VII.
These enhancements are described in section I.P of the proposed PDUFA VII
commitment letter.
J. Continued Enhancement of User Fee Resource Management FDA is committed to ensuring the sustainability of PDUFA program resources and to enhancing the operational agility of the PDUFA
program. FDA will build on the financial enhancements included in PDUFA VI and continue activities in PDUFA VII to ensure optimal use of user fee resources and the alignment of staff to workload through the continued maturation and assessment of the Agencys resource capacity planning capability. This would also include an independent assessment of the resource capacity planning capability. FDA will also continue activities to promote transparency of the use of financial resources in support of the PDUFA
program through annual public meetings, publishing a 5-year financial plan along with annual updates, and additional reporting in the annual PDUFA Financial Report. These enhancements are described in section II of the proposed PDUFA VII
commitment letter.
K. Enhancing Transparency and Leveraging Modern Technology FDA is committed to enhancing the transparency of its information technology IT activities and modernization plans and will continue maintaining catalogs, standards, and plan updates that are published regularly to FDAs website in addition to the publication of a Data and Technology Modernization Strategy document and sharing regular updates on CBER IT modernization progress.

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