Spring Intro 2023

30/03/2023

Adaptive Pathways

Principles

• Iterative development • approval in stages, beginning with a restricted patient population then expanding to wider patient populations, or • confirming the benefit-risk balance of a product, following a conditional approval based on early data • Gathering evidence through real-life use to supplement clinical trial data • Early involvement of patient and HTA bodies Builds on regulatory processes already in place • Scientific advice • Compassionate use • Conditional approval mechanism • Patient registries and other PV tools for collection of real-life data Pilot project: March 2014 - August 2016

• EMA assessed 62 applications with mixed results • EMA to continue to explore the concept further

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Compassionate Use

Allows the use of an unauthorised medicine for patients with an unmet medical need • life-threatening, long-lasting or seriously debilitating illnesses • which cannot be treated satisfactorily with any currently authorised medicine

Individual national programmes

EMA can support with recommendations through CHMP • which national patient access programmes can consider

Medicine criteria • undergoing clinical trials or • have entered the marketing-authorisation application process • early studies will generally have been completed • but its safety profile and dosage not be fully established

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