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Abstract

Over the course of the 20th century, the U.S. Food and Drug Administration was given progressively greater authority to regulate medical products, ultimately requiring pre-approval upon a demonstration of safety and effectiveness. As standards tightened, increased certainty about the clinical benefit of new drugs was paired with inevitably slower access. In response, patients facing serious diseases with unmet treatment needs have pushed for more flexibility, both to access unapproved drugs and for drugs to be approved on weaker evidence. How should regulatory authorities balance the needs – and risk tolerance – of these patients with the need for confidence in drugs allowed on the market?

This lecture will provide a brief history of U.S. drug regulation, describe the pros and cons of pathways (in the U.S. and abroad) that allow early access to drugs whose benefit has not yet been proven, explore what role patient desperation should play in drug approval, discuss potential policy reforms, and identify important questions in need of further research. The U.S. approval of aducanumab for Alzheimer’s disease will be used as a case study, along with other examples.

 

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