US policy changes impacting commercialization of cell and gene therapies

Aimee O'Driscoll, Writer | March 16, 2023

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In this Q&A, Kylie Stengel, Policy Consultant, and Mark Von Eisenburg, Market Access Consultant, explore FDA challenges at each stage of the product life cycle—from preclinical to post-approval—and share their insights on the changing US regulatory and policy reforms impacting commercialization of cell and gene therapies.

The cell and gene market is evolving at lightning speed. Since the approval of the first CAR-T therapy in 2017, more than 20 cell and gene therapies have been approved by the FDA. This includes six CAR-T and four gene therapies. Today, the pipeline is filled with hundreds of these novel candidates, often designed to treat therapeutic areas with high unmet need and limited alternative treatments.

This has led to a dynamic landscape in which the FDA has struggled to upskill in the complex science and the rising demand for regulatory expertise. As a result, it is now turning to legislative and/or regulatory updates to help expedite approvals.

In turn, biopharmaceutical companies are compelled to think critically about evidence generation to meet the approval and post-approval demands of various stakeholders, including regulators and payers, as evidence of novel cell and gene therapy effectiveness may be more pivotal to approval and coverage decisions, while also being harder to obtain in a traditional clinical trial setting.

Manufacturers will need to employ a robust strategy and implement meticulous planning procedures early in the drug development process to account for the evolving regulatory and policy landscape. This includes an increased focus on areas such as health equity, patient-focused drug development, and clinical trial diversity.

Avalere, which joined Fishawack Health in June 2022, is a leading healthcare consulting firm based in Washington DC. Avalere’s team of more than 200 advisors offers vast experience and expertise in federal policy and policy developments across all 50 states.

Offering services in market access, due diligence, and transformation, Avalere partners with life science companies, providers, and others to bring unique solutions to intricate healthcare challenges. Combining Avalere’s experience with Fishawack Health’s global capabilities results in unparalleled global insights and expertise within the cell and gene product life cycle.

We heard from Avalere representatives Kylie Stengel, Policy Consultant, and Mark Von Eisenburg, Market Access Consultant, who shed light on the key FDA regulatory challenges and the impact of US policy changes at each stage of the cell and gene product life cycle. They focus on the impact on product developers, highlighting considerations that will equip companies to plan effectively.


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