FDA sets out vision to use real-world data in product
evaluations
In our centre, we have been interested in and following the
concept of Real World Evidence (RWE) derived from Real World Data (RWD) and
Real World Use (RWU) propounded by FDA USA, and their relation with Evidence
Based Medicine / Practice.
The times are moving for HICs and LMICs, clinicians,
researchers, pharmaceutical and technology industry, policy makers, health
systems and patients. As Jon asked where does Real World Evidence (RWE) sit in
the existing hierarchy of Evidence Based Medicine / Practice.
Courtesy of Jon Brassey on evidence-based-health@jiscmail.ac.uk,
we came across this interesting goings-on in FDA USA, that may affect how
regulatory bodies and countries approve drugs and devices in health care.
Insight – ‘’The FDA has long recognized that the groups of
patients enrolled in clinical trials bear only a passing resemblance to
populations treated in the real world. Seeking to eliminate confounding
factors, clinical trials enroll patients with fewer comorbidities and
concomitant medications than are found in the real world. These clinical trial
designs prioritize clear safety and efficacy signals over applicability to
real-world populations.
Regulators have tried to tackle the problem from multiple
angles. In 2013, the FDA told its staff to push sponsors to run clinical trials
in broad populations that mirror those found in the real world. More recently,
the agency has sought to leverage technological advances, notably electronic
health records (EHRs), to generate more evidence of safety and efficacy outside
of clinical trials.
‘The increasing accessibility of digital health data,
spurred in large part by the transition to electronic health records (EHRs),
together with rising costs and recognized limitations of traditional trials,
has renewed interest in the use of real-world data (RWD) to enhance the
efficiency of research and bridge the evidentiary gap between clinical research
and practice. RWD can be defined as data relating to patient health status or
the delivery of health care routinely collected from a variety of sources, such
as the EHR and administrative data
History - For hundreds of years, the development of new
medical treatments relied on “real-world” experience. Discoveries such as
citrus fruit curing scurvy described in the 1700s or insulin as a treatment for
diabetes in the 1920s long preceded the advent of the modern randomized
clinical trial. What these diseases had in common was a reliable method of
diagnosis, a predictable clinical course, and a large and obvious effect of the
treatment.
In the late 1940s, the medical community began to adopt the
use of randomized clinical designs for drug trials.1The recognition that anecdotal reports based on clinical
practice observations were often misleading led to the nearly complete
replacement of this “real-world evidence” (RWE) approach to evidence generated
using the modern clinical trial model. Although moving medical science toward
greater scientific rigor, this transformation to RCT simultaneously diminished
the use (and minimized the value) of evidence generated from practice-based
observations. Randomization and blinding became the gold standard for determining
the effect of treatment. With strict protocol-specified definition of eligible
patients, populations studied began to diverge from patients encountered in
clinical practice. Patients with wider ranges of disease severity and age,
taking a broader range of concomitant medications, and with more and varying
comorbidities were not as well represented in clinical trials.
By controlling for key sources of bias, assuring
appropriately matched study groups, modern clinical trials support drawing
strong causal inferences regarding the efficacy of treatments, and thereby
contribute to the substantial evidence of effectiveness necessary for
regulatory approval. On the other hand, such trials do have important
limitations, including high costs, extensive resource requirements, and often
long timelines. Restrictive enrollment criteria and the concentration of trial
sites in certain health systems make it challenging for some patients to
enroll, including those with comorbidities, especially if mobility or cognitive
abilities are affected. Thus, the trial population may not reflect the larger
population that will use the drug.’
Further reading on these changes are provided in:
.Joseph Ana
AFRICA
CENTRE FOR CLINICAL GOVERNANCE RESEARCH & PATIENT SAFETY
@Health Resources
International (HRI) WA.
National Implementing Organisation: 12-Pillar Clinical Governance
National Implementing Organisation: PACK Nigeria Programme for PHC
Publisher The BMJ West Africa & Journal of Health Practice
Nigeria: 8 Amaku
Street, State Housing & 20 Eta Agbor Road, Calabar.
Tel: +234 (0) 8063600642
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