From the Valley of Death to the Crossroads of Opportunity: A Discussion of Evolving Benefit/Risk Evaluation Standards

Therapeutic Innovation & Regulatory Science
Peter J Pitts, Patrick Brady

Abstract

A series of recent US Food and Drug Administration (FDA) approvals (such as Sarepta's Exondys 51, Merck's Keytruda, and Portola's Bevyxxa) has generated significant interest within the drug development ecosystem. Facilitated regulatory pathways aimed toward expediting medicines to patients suffering from serious and life-threatening conditions are a good thing, even if it raises curiosity and introduces some degree of uncertainty. Over the last 20 years, two key words in drug development have been speed and innovation. Going forward, the patient voice, data quality, and evidence generation must be added to that list. There is a raging debate over the level of evidence expected to first introduce a treatment to patients. Some argue for less data followed by postapproval follow-up, others for more adaptive clinical trial designs and end-point modification driven by patient-focused drug development and use of real-world evidence. The transition in the regulatory framework is happening in front of our eyes. How are these shifts in regulatory science interpreted within the context of 21st-century drug development-and how can these learnings help advance patient care while placing into context the expected uncertainty we find in bene...Continue Reading

References

Nov 10, 2005·Clinical Cancer Research : an Official Journal of the American Association for Cancer Research·Boris Freidlin, Richard Simon
Jun 14, 2016·Journal of Thoracic Disease·Sai-Bo PanYing Chai

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Citations

Aug 29, 2020·Therapeutic Innovation & Regulatory Science·Alfredo Aram PuliniGregory Katz
Sep 2, 2020·Therapeutic Innovation & Regulatory Science·Mark Robberson, Christopher D Breder
Jul 29, 2020·Trends in Biotechnology·Jonathan D Linton, Wei Xu

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