Spring Intro 2023

30/03/2023

US Accelerated Approval

Program

Criteria

When to Submit

Features/Considerations

Serious condition AND

Discuss during development

Approval based on an effect on a surrogate or intermediate clinical endpoint that is reasonably likely to predict drug’s clinical benefit Post-approval confirmatory trials Submission of promotional material

•

•

Meaningful advantage over available therapies, AND

•

Surrogate endpoint reasonably likely to predict clinical benefit or a clinical endpoint that can be measured earlier than an effect on irreversible morbidity or mortality

•

Accelerated Approval

•

(designation)

•

Potential of withdrawal of accelerated approval

•

The Organisation for Professionals in Regulatory Affairs

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Other expedited pathways

Approval under exceptional circumstances • Comprehensive data on the efficacy and safety cannot be obtained even after authorization because – comprehensive information cannot be gathered (very rare disease, scientific knowledge not available), ethical. • Early and continuous dialogue (Pre and Post approval) Adaptive Pathways • Iterative development with product approval in stages, beginning with a restricted patient population then expanding to wider patient populations • Gathering evidence through real-life use to supplement clinical trial data • Early involvement of patient and HTA bodies • Use scientific advice and consider (and agree) all avenues for evidence generation 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 • Governed by national regulation • Product has entered marketing authorisation or has a global approval

The Organisation for Professionals in Regulatory Affairs

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