‘Regulatory Risk’: A New Factor In Risk/Benefit Equation At US FDA?
Executive Summary
FDA’s evolving efforts to develop a ‘structured risk-benefit’ framework is playing an important role in accelerating access to drugs for rare diseases and specific subgroups in cancer; there may be less attention to an accompanying phrase that is growing in currency as a factor in the equation: ‘regulatory risk’ – the term for how FDA calculates the odds of making the wrong regulatory decision for a small-population treatment.
You may also be interested in...
AMAG’s Makena: Did Accelerated Approval Kill The Confirmatory Trial?
Sponsor’s assertion that recruitment of high-risk, US patients was negatively impacted by the preterm birth drug’s availability under accelerated approval led some advisory committee members to opine that Makena came to market too soon; the experience could make the FDA more cautious about granting accelerated approval unless confirmatory studies are at an advanced stage.
BIO 2019 Notebook: Lartruvo Lookback, M&A Soars, BIO Vs. ASCO
News and views from day four of the BIO International Convention: Former FDA official Amy McKee said "everything worked right" with accelerated approval and removal of Lilly's Lartruvo; emerging company M&A deals nearly double in 2018; and the ASCO effect on the 3-6 June BIO meeting.
BIO 2019 Notebook: M&A Soars, Lartruvo Lookback, BIO Vs. ASCO
News and views from day four of the BIO International Convention: emerging company M&A deals nearly double in 2018; former FDA official Amy McKee said "everything worked right" with accelerated approval and removal of Lilly's Lartruvo; and the ASCO effect on the 3-6 June BIO meeting.