Increased Focus Being Placed On Using “Real World Evidence” In
Regulatory Decision Making
Jobs For
Epidemiologists Have Increased Several Fold
The
traditional pathway to getting new drugs approved has been through the
conduct of randomized control trials (RCTs). More recently, an
intensifying focus on using non-clinical trial, real world data (RWD)
and real world evidence (RWE) as part of a pathway to drug licensure
is creating new possibilities and raising new challenges for all
parties involved--- regulators, scientists, and drug and insurance
companies. At the same time, this new pragmatism about observational
data is creating a huge boost for epidemiologists already inside
biopharma and is creating new job opportunities for others.
New Realities
These new realities were the focus of a presentation
made by Amgen’s Cathy Critchlow at the recent American College
of Epidemiology meeting in New Orleans last month. The session was
organized around the theme of epidemiology in the current political
climate, and she provided her thoughts based on her work in the
pharmaceutical Industry.
According to Critchlow, RWD includes electronic health
records, administrative/claims data, patient generated/reported data,
prospective cohort studies, and other non-clinical trial
health-related data such as that collected from wearable devices.
Questions Addressed
Among the types of questions being addressed or uses
for RWE are a better understanding of a product’s benefit/risk profile
and drug utilization in real world (clinical practice) settings, a
better picture of the characteristics of patients most likely to
benefit from a drug, better grasp of the patient response to therapy
targets from genomics data linked to electronic health records, the
extent of unmet medical need and size of populations that might
benefit from specific drugs.
Drivers
Some of the drivers for this invigorated interest in
using non-clinical trial data comes from the fact that there are
simply more and better data being collected from a variety of sources
and computing power, methodology, and data science competency are
“advancing significantly”, according to Critchlow. In addition,
requirements embedded in the Prescription Drug User Fee Act VI and the
21st Century Cures legislation call for the Food and Drug
Administration (FDA) to utilize real-world observational data and to
evaluate the use of RWE in informing regulatory decisions.
Case Examples
Given the opportunities but at the
same time the limitations of RWE, all parties involved are trying to
decipher just how RWE can be effectively used. There are as yet no
official standards by which to evaluate the quality of RWE, and this
is a hot topic in the field, says Critchlow. Companies are beginning
to accumulate a body of case examples to stimulate discussions of
potential ways in which RWE could be used in reaching decisions about
a variety of product labeling, drug safety, reimbursement, and other
issues. The hope is that accumulating a body of case examples will
together inform and help establish acceptable non-clinical trial data
pathways to approval for drug utilization.
Cautions
Needless to say, it is critical to ensure that any RWE used in
regulatory submissions is of sufficient quality to enable valid
assessment of a drug’s benefits and risks, yet increasing costs of
drug development, the greater need to address questions that cannot be
addressed by clinical trials,
and the greater availability
of
RWD, all argue for
taking a more pragmatic approach.
Issues
According to Critchlow, some of the issues being faced to allow for
more impactful use of RWE are:
1)
Data accessibility, quality, and standards
2)
Interoperability of electronic medical records and other systems
3)
Analytic methods that ensure study validity
4)
Preserving data privacy
The Future
In
looking toward the future, Critchlow told the ACE audience that she
sees growing capabilities to leverage real world data to inform
decision making by regulatory and reimbursement agencies. However,
with greater use will come greater scrutiny, and valid study designs
and methods will remain critical. ■
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