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Common pitfalls in oncology drug applications aiming for conditional marketing authorization.
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  • Sinan Sarac,
  • Peter Kiely,
  • Simona Stankeviciute,
  • Jorge Camarero,
  • Amy McKee
Sinan Sarac
PAREXEL International Denmark

Corresponding Author:sinan.sarac@parexel.com

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Peter Kiely
Parexel International
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Simona Stankeviciute
Parexel International
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Jorge Camarero
Merck & Co Inc
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Amy McKee
Parexel International Corporation Bethesda
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Abstract

Early approval mechanisms, such as conditional approval in the EU, have been used extensively to provide timely access to therapeutic innovations to cancer patients with unmet medical needs. While based on promising early evidence, such approvals are challenging from many perspectives due to the lack of comprehensive data. The limitation typically relates to data that demonstrates clinical benefit via particular endpoints and is only acceptable when the early evidence is particularly convincing to assume that the benefits of early access are greater than the potential harms. This paper describes the requirements for conditional approval and reviews common pitfalls in oncology, such as misunderstandings about the strength of evidence from exploratory trials and secondary analyses, lack of planning, and opportunities to improve communication. Thereafter, we present a framework (“EDGE”) on how to improve the submission and evaluation of drug applications for conditional approval in the EU.
25 Jul 2024Submitted to British Journal of Clinical Pharmacology
25 Jul 2024Submission Checks Completed
25 Jul 2024Assigned to Editor
25 Jul 2024Review(s) Completed, Editorial Evaluation Pending
26 Jul 2024Reviewer(s) Assigned