Pharmaceutical Outsourcing Q3 2023 - 10

CONTRACT RESEARCH
A Review of How Real-World
Evidence Enhances
Regulatory Submissions
Jaclyn Bosco
VP & Global Head, Epidemiology, Real World Solutions,
IQVIA
Marni Hall
VP & Global Head, Regulatory Science & Strategy,
Real World Solutions
IQVIA
The importance of using real world evidence (RWE) for efficacy has
gained significant momentum over the past few years as regulators
are increasingly calling for the use of real-world data (RWD) and RWE.
Regulatory bodies across the world, including the U.S. Food and Drug
Administration (FDA) and the European Medicines Agency (EMA),
have released guidance to clarify their expectations for the use of
RWD and RWE for regulatory decision-making.
RWE can be used as supportive evidence to complement findings
from randomized clinical trials (RCTs), and there has been a growing
number of positive decisions where RWE, used during the drug
development phase, contributed to the substantial evidence. There
are many ways RWE and RWD can enhance and supplement clinical
development. A few examples of how RWE and RWD enhances and
optimizes clinical trials include RWD-driven protocol feasibility and
optimization, data-driven site selection and patient enrollment, more
patient-centric/decentralized approaches, leveraging non-identified
imaging or genomic data to identify the right patient population,
use disease registries for patient recruitment, or innovative extension
studies to gather long-term outcomes in new ways. RWE can also
be used to provide additional information and context for better
decision-making.
RWE Approaches to Support
Regulatory Decisions
RWE has been used to support drug approvals using an external
comparator to a single-arm trial (SAT), in which the use cases have
primarily related to therapeutic products for rare disease or oncology
settings. This is especially the case where there is a high unmet
need and it is unethical or not feasible to assign a parallel control
arm, and effect size is large (FDA, 2018). For instance, in 2017, the
FDA approved an immunotherapy drug which used RWE from a
retrospective analysis of patients with the disease to complement
data from a multicenter phase 2 trial. In 2018, the EMA approved a
new CAR-T cell therapy which utilized RWE as a comparator arm to
confirm data gathered in a SAT. In February 2023, the FDA released
a new draft guidance for externally controlled trials, which, in part,
addresses the potential role of RWE in these studies.
Pragmatic trials are another real-world approach that randomizes
patients to the treatment of interest or to standard of care and then
follows them under real world conditions to evaluate effectiveness
in broader populations than are typically included in RCTs. As an
example, in 2021, the FDA approved a label expansion for a biannual
schizophrenia treatment, which used a pragmatic trial
Pharmaceutical Outsourcing | 10 | July/August/September 2023
Pharmaceutical Outsourcing Q3 2023

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