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abstract
Author Affiliations
- 1Pfizer Oncology, New York, NY, USA
- 2Pfizer Oncology, Cambridge, MA, USA
- 3Pfizer Oncology, Regional Medical Affairs, Berlin, Germany
- 4Pfizer, Worldwide Safety and Regulatory, Groton, CT, USA
An analysis of oncology regulatory
approvals since 2011 reveals that several new drugs demonstrate
exceptional clinical activity
in Phase 1 or Phase 2 trials. This has led to
increased use of regulatory mechanisms that allow initial approval based
on
these early data. However, full regulatory approval
is contingent upon subsequent verification of clinical benefit in
confirmatory
Phase 3 randomized controlled trials.
There has been a growing interest in the
systematic collection of real-world data to gain further insight into
the impact
of new drugs as they are introduced into clinical
practice. Furthermore, payers, physicians, and patients increasingly
require
more data on alternative comparators, specific
subgroups, patient-reported outcomes, and long-term toxicities - data
that
may be more appropriately collected in real-world
studies than in separate confirmatory trials.
We propose that for oncology drugs that
demonstrate “exceptional activity” in early trials and receive
accelerated/conditional
approval and/or Breakthrough Therapy Designation,
and for certain expanded indications, regulatory authorities should
consider
accepting data from prospectively agreed pragmatic
randomized clinical trials (pRCTs) to grant full regulatory approval.
The
“exceptional activity” would be defined as (1) an
objective response rate ≥50% to a single agent or (2) a hazard ratio of
<0.5 in an early randomized study. In pRCTs,
patients would be randomized to two or more interventions and then
treated and
followed up according to the investigators' usual
practice. Patients participating in pRCTs would be representative of the
real-world population. pRCTs would provide data
necessary for full regulatory approval and would address questions of
the
new drug's value for reimbursement purposes.
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