Common pitfalls in oncology drug applications aiming for conditional
marketing authorization.
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.