Pharmaceutical Outsourcing Q3 2023 - 11

CONTRACT RESEARCH
SECTION TITLE
design. Regardless of the RWE approach, the FDA has outlined
an RWE framework with three criteria (a) are the RWD fit for study
purpose, (b) is the trial or study design likely to provide adequate
and well-controlled scientific evidence to answer or help answer
the regulatory questions, and (c) does the study conduct meet FDA
regulatory requirements?
Determining When RWE is Fit-For-Purpose
Identifying
quality
data
sources
that will meet regulatory
requirements can be challenging. In the FDA's draft guidance on
data standards, there is a strong emphasis on data quality from the
point of collect and curation to research analyses. Due to the wide
variety of RWD sources, challenges arise due to inconsistent data
formats and standards and governance requirements depending
on the country or region of origin. There may also be missing data
or differing use of vocabulary or coding systems that can make
standardization difficult.
When using RWD there are important considerations for selecting
the optimal data source and determining whether the data are fitfor-purpose
by applying a rigorous evaluation of the appropriateness
to use a given database. The reliability and relevance to the research
questions must be established. When selecting the optimal database,
researchers using RWD need to ask questions about the data
provenance - where did it come from, who recorded it, why was it
recorded, who is represented, how many years does it cover, is there a
lag between data recording and data availability?
It is also important to understand whether the data are accessible
and if so, what the process is to access the data and ethics approval,
and whether there are any restrictions for third party access. Finally,
the curation process of the data can be learned by obtaining a data
dictionary that includes coding systems and understanding what
verification and validation checks are performed are essential. In
terms of determining the fitness of the data, there are frameworks
such as the Structured Process to Identify Fit-for Purpose Data (SPIFD)
that can be applied as it is a systematic process to identify fit-forpurpose
data (Gatto et al., 2022).
What This Means Moving Forward
The opportunities to advance disease research and drug development
are growing with the global increase in expanded use of RWE by
regulatory bodies. The rich repository of RWD available coupled
with rapidly evolving technologies, and novel study designs, enables
more innovative ways to consider evidence generation for regulatory
decision-making. The fitness of RWD for the specific study question
must be evaluated and there is a need to distinguish when RWE is
intended to be used for substantial evidence or supportive evidence.
The requirements for substantial evidence are no different than they
have been before, and the fit-for-purpose data feasibility assessments
need to be transparent regarding data provenance, access, curation,
and linkage as well as communicating and discussing with regulators
early and often are essential. The use of RWD and RWE can enhance
drug development and enable advancements in treatment options
for patients.
pharmoutsourcing.com | 11 | July/August/September 2023
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Pharmaceutical Outsourcing Q3 2023

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