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‘Regulatory Risk’: A New Factor In Risk/Benefit Equation At US FDA?

 
• By Cole Werble

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.

FDA is trying to formalize the inherently subjective process of weighing risks against benefits when making a drug approval decision by developing and implementing a structured risk/benefit assessment as a routine part of the review process.

That calculation necessarily relies on relatively formal, quantifiable criteria: an analysis of the condition, current treatment options, summaries of the...

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