Accelerated Drug Approval Processes in Oncology

Accelerated approval processes have become central to bringing novel cancer therapies to patients more rapidly by allowing regulatory decisions on the basis of earlier or less comprehensive data than traditional pathways. These mechanisms, spearheaded by agencies such as the US Food and Drug Administration and the European Medicines Agency, include designations like accelerated approval, priority review and conditional marketing authorisation. They often rely on surrogate endpoints—biological or radiological measures reasonably likely to predict clinical benefit—to shorten time to market. Postauthorisation obligations, including confirmatory trials and ongoing safety monitoring, seek to resolve residual uncertainties in efficacy and adverse-event profiles. As oncology increasingly incorporates targeted agents and immunotherapies, expedited pathways balance urgent clinical need against the imperative for rigorous evidence, highlighting global collaboration on regulatory standards and the integration of real-world data to support regulatory and clinical decision-making.

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Accelerated Drug Approval Processes in Oncology publication trend

The graph below shows the total number of articles in accelerated drug approval processes in oncology across all publications each year (not limited to Nature Index journals).

Technical terms

Accelerated approval: A regulatory pathway granting early authorisation on the basis of surrogate or intermediate endpoints, contingent on postmarketing confirmatory studies.

Surrogate endpoint: A marker, such as tumour shrinkage or progression-free survival, used in trials as a proxy for direct clinical outcomes like overall survival.

Priority Review: A designation reducing the standard review period for regulatory bodies, aimed at therapies for serious conditions with significant unmet medical need.

Conditional Marketing Authorisation: An approval in the European Union granted on the basis of less complete data, subject to obligations to provide additional evidence post-authorisation.

Confirmatory trial: A post-approval study designed to verify and describe the clinical benefit of a therapy initially authorised under an accelerated or conditional pathway.

References

1. Trends in utilization of FDA expedited drug development and approval programs, 1987-2014: cohort study. The BMJ (2015).
2. Assessment of Clinical Trials Supporting US Food and Drug Administration Approval of Novel Therapeutic Agents, 1995-2017. JAMA Network Open (2020).
3. Approval of Cancer Drugs With Uncertain Therapeutic Value: A Comparison of Regulatory Decisions in Europe and the United States. Milbank Quarterly (2020).