Facilitating Both Evidence and Access: Improving FDA's Accelerated Approval and Expanded Access Pathways

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21 U.S. Code §355 (2018).

For diseases and conditions that lack even unvalidated surrogates, FDA should further clarify which intermediate clinical endpoints will suffice for Accelerated Approval.

Because the course of COVID-19 is relatively quick — patients either recover or die within a matter of weeks — reliance on surrogate endpoints and Accelerated Approval is not needed for drugs to treat this disease. Instead, Accelerated Approval is better suited for diseases and conditions with a longer trajectory that would require much longer trials to evaluate ultimate outcomes related to clinical benefit or irreversible morbidity and mortality.

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Larger companies are typically able to cover these costs, although even they may be overwhelmed by logistics in the event of high request volume, as we have seen during the COVID-19 pandemic. O'Day, supra note 36.

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H.R. 7071, 2020, available at <https://iamals.org/wp-content/uploads/2020/06/Accelerating-Access-to-Critical-Therapies-for-ALSAct.pdf?utm_source=Social&utm_medium=Twitter&utm_campaign=ACT%20for%20ALS%20Bill> (last visited June 3, 2020); “Quigley, Fortenberry Create New Pathways for Treatment in Fight Against ALS, Neurodegenerative Diseases,” June 1, 2020, available at <https://quigley.house.gov/media-center/press-releases/quigley-fortenberry-create-new-pathways-treatment-fight-against-als> (last visited June 2, 2020). This bill would also create a Center of Excellence for Neurodegenerative Diseases at FDA, modeled after the Center of Excellence in Oncology.

Joffe and Fernandez Lynch, supra note 7; Fernandez Lynch, Zettler, and Sarpatwari, supra note 7.

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