Download Post-Approval Studies are an Essential Phase of Drug Development

Post-Approval Studies are an Essential Phase of Drug
Development with Important Implications for Life Science
Companies
PMOS provide evidence on real–life
by Dr. John S. Sampalis
effectiveness and safety...
The intrinsic differences between clinical trials and real-life utilization of drugs
It is important that biopharmaceutical companies consider post-approval studies as part
create treatment and safety gaps that can undermine return on investment for
of their clinical programs. This is due to regional variations in the patient profile, cultural
drug manufacturers. Safety issues and reduced efficacy observed in real-life can
influences, practice patterns and local reimbursement policies affecting access to care.
limit the success of marketed drugs. In addition, these gaps also increase costs to
Consequently, PACES must be designed and conducted taking into consideration regional
society while maintaining a high burden of illness. It is essential for all stakeholders
needs and treatment gaps while global-wide studies are less relevant. It follows, that
to continue to generate more evidence to achieve optimal effectiveness and safety.
at a minimum, country specific PACES will be required to conduct regional evaluations
of marketed treatments. The benefits from these studies will be improved treatment
While Phase II – III controlled clinical trials provide evidence of efficacy under ideal
effectiveness with improvement in health and wellness of population at large and a
conditions, Post-approval clinical and Epidemiological Studies (PACES) are the
reduction in the burden of illness to society.
only source of information that allows the assessment of real – life effectiveness.
Furthermore, ongoing surveillance for safety signals under routine clinical
PACES have evolved into an integral and essential phase of the drug development life cycle
practice allowing the detection of rare but potentially serious adverse events can
with implications that have comparable weight to that of Phase II and III registrational
only be accomplished by the accumulation of data from several PACES capturing
studies. Given the importance of the PACES and the potential implications of the results
data from large populations and over sufficient periods of treatment exposure.
to health care stakeholders it is essential that these studies are conducted with the
In addition to long term assessment of effectiveness and safety, these studies
same scientific rigor as Phase II and Phase III studies. Essential elements of properly
are ideal for generating evidence relating to patient adherence/compliance and
conducted PACES include well defined hypotheses addressing knowledge gaps that
preference for therapy.
define clear objectives and research questions. Statistical considerations must include
precise sample size requirements that ensure sufficient power to address the research
There are numerous possible designs of PACES. Phase IV studies emulate the real–
objectives and statistical analyses that incorporate advanced methods to compensate
life setting and allow assessment of treatment effectiveness for specific patient
for the lack of randomization, non–standardized follow up and the observational nature
subgroups or enriched populations. These studies typically provide treatment at
of the design. Scientific rigor will increase the studies’ credibility in the scientific
no cost. There is significant value in Phase IV studies, which should be considered
community and with government. A high scientific standard is also essential to support
as the real–life extension of the Phase III registrational studies. Post Marketing
promotional claims.
Observational Studies (PMOS) are true epidemiological studies in which treatment
is provided to patients without any intervention by the sponsor or the investigator.
The life science industry should view PACES as an important ongoing evaluation to help
Unlike Phase IV studies, in the PMOS payment for treatment is provided as per
achieve optimal effectiveness & safety outcomes of treatments within populations.
real life, including insurance plans or by the patients themselves. PMOS provide
evidence on real–life effectiveness and safety in combination with assessment
John S Sampalis, Ph.D.
of health care utilization, direct and indirect health care costs, and the impact
Professor of Surgery and Medicine McGill University, University of Montreal and
of access to treatment. These are essential for a comprehensive health economic
University of Laval, CEO and Chief Scientific Officer, JSS Medical Research Inc.
evaluation. Healthcare administrative databases, chart reviews and registries are
a means to expedite retrospective studies with large number of patients. These
studies are ideal for validation or generation of hypotheses for future studies. The
right design and approach depends on the objective, the desired outcomes and
other limitations such as time and data.
Montreal, Quebec
Medellin, Colombia
+1 866 934 6116