Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
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Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
Learn more
Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
Learn more
Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
Learn more
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VIEW ROLESAccelerated approval pathways in the United States have had a dramatic impact on oncology drug development, providing patients with rapid access to novel and effective drugs. The pathways are not limited to oncology, having originated in infectious disease and with significant use in immunologic and neurodegenerative disease. However, the use of accelerated approval requires a surrogate endpoint for the disease, a barrier that is infrequently overcome. The regulatory standards for surrogate endpoints for an accelerated approval or a full marketing approval are complex and difficult.
The FDA maintains a public list of accepted surrogate endpoints for regulatory purposes. The table distinguishes between traditional and accelerated surrogate endpoints. The FDA’s 2014 "Expedited Programs for Serious Conditions — Drugs and Biologics Guidance for Industry" guidance defines the two types of surrogates. A traditional surrogate endpoint is known to predict clinical patient response to a therapy with a clear mechanistic rationale while an accelerated or reasonable surrogate is likely to predict expected clinical patient response. A traditional surrogate endpoint is supported by clinical data providing strong evidence that an effect on the surrogate endpoint predicts a specific clinical benefit. In contrast, a reasonable surrogate endpoint is supported by strong mechanistic and/or epidemiologic rationale, but the amount of clinical data available is not sufficient to show it is a validated surrogate endpoint. A reasonable surrogate endpoint may support an application for accelerated approval. Surrogate endpoints, including candidate endpoints, with less supporting evidence, may also be used for decision-making without regulatory involvement.
Recent case studies of the use of accelerated surrogate endpoints highlight the complexity and requirements to meet a reasonable surrogate regulatory standard. Most recently, Tofersen obtained FDA accelerated approval for ALS with Neurofilament Light Chain (NfL) as a reasonable surrogate endpoint. NfL was supported by evidence for a mechanistic link between drug activity and endpoint status, a meta-analysis and regression analysis for a prognostic value of NfL in ALS and correlation analyses and a causal inference analysis demonstrating a relationship between change in NfL and reduction in ALSFRS‐R. Multiple publications preceded NfL’s use for Tofersen’s accelerated approval, supporting its linkage to neuronal damage and prognosis in neurodegenerative disease. The biomarker is now being explored as a surrogate endpoint in additional indications. The extent of data and analyses required for NfL and Tofersen’s approval described in the FDA ODAC meeting materials provides a detailed roadmap for future reasonable accelerated surrogate endpoints.
The accelerated approval pathway in the US has received recent critical review, with a few notable drugs failing in their confirmatory phase III trials following accelerated approval. However, the pathway will likely remain a key option for groundbreaking drugs in indications with high unmet need. Therefore, surrogate endpoints will remain a key area of research. It will be helpful for the field to acknowledge the requirements outlined in the Tofersen case study and to focus research on obtaining a reasonable level of validation.
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