Post-Approval Studies are an Essential Phase of Drug Development with Important Implications for Life Science Companies. observed in clinical trials creating treatment and safety gaps, which can undermine return on investment for all healthcare stakeholders.

1. 9400 Henri-Bourassa Boulevard West
Montreal, Quebec, Canada, H4S 1N8
Tel: 514-9346116 Fax: 514-9349913
www.jssresearch.com
A science-based
contract research
organization where
scientific expertise
meets operational
excellence
Post Approval Clinical and Epidemiological
Studies (PACES)
Post Market Observational Studies (PMOS)
Health Economics and Outcomes Research
(HEOR)
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2. JSS Medical Research
Headquartered in Montreal
>25 years in business
Privately held and financially stable
Experienced management team
An international full-service CRO with study
involvement in over 20 countries
Largest Canadian-owned CRO
Approximately 200 ongoing projects in fiscal 2016
Growing client list includes global big pharma (14 of
top 25) biotechnology and device companies
80% repeat business
290 full time + 49 regionally based contract staff
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2015
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We offer solutions and insights for all developmental
phases and post-approval needs for a wide range of
therapeutic areas worldwide.

3. A full service CRO with PACES experience and expertise
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JSS Medical Research was founded by Dr. John S. Sampalis, a Clinical Epidemiologist
trained at McGill University and the Division of Clinical Epidemiology of the Montreal
General Hospital. Dr. Sampalis is currently a professor of Surgery and Epidemiology and
Biostatistics at McGill University, University of Montreal and University of Laval.
The epidemiologists, biostatisticians and project managers at JSS Medical Research
possess the exact training and experience that is required to conceive, design, and
execute the study, analyze the data and disseminate results for regional Canadian
PACES. Clinical operations, project management and quality assurance at JSS Medical
Research are guided by the clinical research and epidemiology nucleus.
Consequently, JSS Medical Research is the ideal full service Clinical Research
Organization (CRO) partner for conducting post-approval studies in a wide range of
therapeutic areas. The publication record of over 200 publications in peer reviewed
journals and over 300 presentations at scientific conferences is testimony to our
scientific excellence.
Aug
2015

4. Post Approval Clinical & Epidemiological Studies (PACES)
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2015
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that of the Phase II and III Registrational
studies.
While Phase II – III controlled clinical
trials provide evidence of efficacy under
ideal conditions, PACES are the only
source of information that allows for the
assessment of real-life effectiveness. In
addition, ongoing surveillance for safety
signals under routine clinical practice that
allows for the detection of rare but
potentially serious adverse events can
only be accomplished by the
accumulation of data from several PACES
that capture data from large populations
and over sufficient periods of treatment
exposure.
Post-Approval Clinical and
Epidemiological Studies
(PACES) that include Phase
IV and Post-Marketing
Observational Studies
(PMOS) have evolved into an
integral and essential phase of
the drug development life
cycle with implications that
have comparable weight to

5. must take into consideration regional
needs and treatment gaps that are less
relevant in global studies. It follows that,
at the minimum, country specific PACES
will be required to conduct regional
evaluations of marketed treatments.
However, the aggregation of evidence
from several regions or countries could be
employed to provide global assessments.
Given the importance of PACES and the
potential implications of the results to
health care stakeholders, it is essential
that these studies be conducted with the
same scientific rigor as Phase II and
Phase III studies.
Post Approval Clinical & Epidemiological Studies (PACES) continued
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2015
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One of the most important
considerations of PACES is
regional specificity. This is due
to regional variations in the
patient profile, cultural
influences, practice patterns
and local reimbursement
policies affecting access to
care. Consequently, the
design and conduct of PACES

6. Post Approval Clinical & Epidemiological Studies (PACES) continued
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2015
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incorporate advanced methods to
compensate for the lack of randomization,
non-standardized follow up and the
observational nature of the design. As
such, PACES must be conducted under
the aegis of clinical or health outcomes
departments of pharmaceutical sponsors
in collaboration with third parties that
have experience in the design, conduct
and analyses of epidemiological studies
with appropriate training in the field.
Clinical epidemiology provides
the only methodological
platform upon which PACES
can be developed and
executed. Essential elements
of properly conducted PACES
include well defined
hypotheses addressing
knowledge gaps that define
clear objectives and research
questions. Statistical
considerations must include
precise sample size
requirements that ensure
sufficient power to address the
research objectives, as well as
statistical analyses that

7. Post Approval Clinical & Epidemiological Studies (PACES) continued
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2015
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which should be considered as the real-
life extension of the Phase III
Registrational studies. Under the Phase
IV studies, treatment effectiveness for
specific patient subgroups or enriched
populations can be evaluated.
Randomization for the assessment of
differences with respect to treatment
effectiveness is also possible in Phase IV
studies.
While Phase IV studies
emulate a real-life setting, the
fact that the sponsor provides
medication to the patient at no
cost affects the generalizability
with respect to access of care
and compliance that is
artificially enhanced in the
Phase IV setting.
Furthermore, Phase IV studies
are conducted under pre-
determined protocols dictating
patient follow up schedules
and assessments.
Nevertheless, there is
significant value in Phase IV
studies,

8. Post Market Observational Studies (PMOS)
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2015
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by the patients themselves. PMOS
provide evidence on real-life effectiveness
and safety in combination with
assessment of health care utilization,
direct and indirect health care costs that
are essential for a comprehensive health
economic evaluation, and assessment of
treatment impact on burden of illness.
Post-Marketing Observational
Studies (PMOS) are true
epidemiological studies in
which treatment is provided to
patients without any
intervention by the sponsor or
the investigator. In the PMOS,
payment for treatment is
provided as per real life,
including insurance plans or

9. Importance & benefits of Phase IV & PMOS studies
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2015
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• Assess real-life efficacy (effectiveness)
and safety
• Assess compliance and adherence
• Assess patient & physician satisfaction
• Assess patient outcomes such as
quality of life, productivity, satisfaction,
health care utilization
• Determine treatment and safety gaps
• Assess subpopulation
• Generate hypothesis
• Increase familiarity with treatment
Phase IV studies &
Observational studies are
important and beneficial to:

10. Aug
2015
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Marie Maxime Hubert, B.Sc., M.Sc.
Manager, Health Economics and Outcomes Research
Ms. Hubert holds a bachelor in biomedical sciences, a graduate diploma in drug development and
a master’s degree in pharmaceutical sciences with a double specialization in
pharmacoepidemiology and pharmacoeconomics. She gained a practical understanding of
economics, data analysis and drug policies as economic consultant atIMS Health. She has
experience in market access from roles in multinational pharmaceutical companies; preparing the
launch of a new chemical entity (dronedarone, Sanofi-Aventis) as well as generating
pharmacoeconomic evidence for established indications (adalimumab, AbbVie – formerly Abbott).
Ms. Hubert also led multidisciplinary teams in the design and execution of treatment adherence
studies. Ms. Hubert has built reimbursement dossiers for individual provinces, private drug plans,
Quebec’s Institut national d’excellence en santé et en services sociaux, the common drug review
(CDR) and the pan Canadian Oncology Drug Review. She also reviewed reimbursement
submissions as part of her duties as health economist for the CDR at the Canadian agency for
drugs and technologies in health. After completing a contract with JSS Medical Research as
independent consultant, Ms. Hubert joined the team as Manager, Health Economics and
Outcomes Research (HEOR). She is currently expanding the HEOR franchise at JSS Medical
Research.

11. Health Economics and Outcomes Research (HEOR)
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2015
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Increasingly, payers and healthcare
service providers around the world are
becoming more knowledgeable about this
and require HEOR to be conducted with
rigorous scientific methods.
HEOR measures the indirect treatment
comparison that can be described as a
way of pooling clinical evidence to
establish the relative efficacy of
treatments never compared against each
other but compared against a common
comparator, without breaking
randomization.
Health Economics and
Outcomes Research (HEOR)
demonstrates the cost-
effectiveness of drugs by
comparing to the gold
standard treatment in current
practice. This is expressed as
a ratio of the incremental cost
per incremental health benefit.

12. Health Economics and Outcomes Research (HEOR)
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2015
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• Scientific rationale
• Clinical summary
• Burden of illness
• Cost of illness
• Pharmacoeconomic assessment:
• Cost-utility analysis
• Cost-effectiveness analysis
• Cost-minimization analysis
• Net impact (adopting a societal
perspective)
• Budget impact analyses
•
Leveraging expertise in health
economics, experience working in
the multinational pharmaceutical
companies, consultation firms and
the Canadian reimbursement
agency, JSS Medical Research
HEOR management can lead the
development of the reimbursement
dossier or its adaptation for
practice, including:

13. Health Economics and Outcomes Research (HEOR)
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2015
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It should also take into consideration
healthcare policies, patient associations
and the reimbursement landscape.
As a science-based CRO operated by
professionals with years of experience in
the pharmaceutical industry, our value
proposition is based on high quality
analyses that can convincingly
demonstrate the value of your product,
performed under the sponsor’s timelines,
with the support of key opinion leaders,
without compromising the scientific
rationale.
Given its core capabilities in
biostatistics, data analysis, meta-
analysis and modeling, JSS Medical
Research can design and perform
the appropriate analyses to allow
you to gain or optimize
reimbursement. In a holistic
approach, the submission dossier
should contain a value proposition
supported by clinical, economic, and
humanistic data.

14. At JSS Medical Research we offer solutions & insights for all
developmental phases & post-approval needs
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2015
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D r u g d e v e l o p m e n t
Clinical Phase
I-III
Late and post-
approval phase
Market Access &
HEOR
Across the
lifecycle
• Phase I
• Phase II
• Phase III-IIIb
• Medical Devices
• Phase IV
• PMOS
• Chart Review
• Registries
• Databases Analysis
• Epidemiological Surveys
• Health Technology
Assessment
• Value Proposition
• Budget Impact Analysis
• Strategic Assessments
• Study Design
• Risk Management
• Epidemiological Study
• Patient Reported Outcomes
• Literature Review
• Advanced Biostatistics
• Investigators Initiated Studies
• Rescue Analysis
• Survey
• Monitoring
• Other: Staffing, Courses,
QA & GCP
JSS Medical Research Capabilities

15. 9400 Henri-Bourassa Boulevard West
Montreal, Quebec, Canada, H4S 1N8
Tel: 514-9346116 Fax: 514-9349913
www.jssresearch.com
A science-based
contract research
organization where
scientific expertise
meets operational
excellence
For More Information Contact
Ann Vawter
Director, Business Development & Marketing
Ph: +1.919.424.3332
Email: ann.vawter@neeman-medical.com
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Thank You