> Why HEOR? > Costs, Consequences and Perspectives > Key Stakeholders in HEOR > What is Health Economics and Pharmaco-economic Research? > Economic Evaluations > Incremental Cost Effectiveness Ratio (ICER) > Concept of HRQoL > Comparative Effectiveness Research (CER) > Pragmatic Clinical Trials > Observational Studies > Systematic Reviews and Meta-Analysis > Application of CER > Health Technology Assessment (HTA) > Real World Evidence (RWE) > Patient Reported Outcomes (PROs) > Patient Focused Drug Development (PFDD) > Application of Health Economic Evaluations  > Challenges and Barriers

1. Fundamentals of HEOR:
An Industrial Perspective
Dr. Amit Dang
Founder and CEO | MarksMan Healthcare Communications
President | ISPOR - Mumbai

2. Why HEOR?
 Rapidly growing healthcare industry in India
 Inadequate healthcare budget
 80% OOP expenses
 Scarcity and uncertainty of resources
 Decision making - therapeutic effectiveness v/s cost-effectiveness
 Allocation of healthcare expenditure
 Inadequate health budget/low health coverage
 Absence of central health reimbursement system
 Catastrophic payments/financial toxicity
 Absence of consensus
 Absence of uniformity in delivery of health services
 Absence of willingness-to-pay thresholds

3. Costs, Consequences and Perspectives
Costs
Direct cost
•Medical Cost
•Non-Medical Cost
Indirect cost
•Lost productivity/time off
work
•Value of lost productivity
•Caregiver time
Intangible cost
•Quality of life
•Pain and suffering
Consequences
Monetary
benefits
Effectiveness of
therapy
Utility
•QALY
•HALY
Perspectives
Medical Scientist
•Clinical
effectiveness
Patient
•Costs
Hospital
administrator
•Costs and
consequences
Societal and
National
Multidimensional
health outcomes are
reduced to a single
index using health
utilities.

4. Key Stakeholders in HEOR
Patients
Hospital
administrators
Healthcare
providers
Pharmaceutical
industry
Insurance
companies
Government

5. What is Health Economics?
 Branch of economics that examines as well as evaluates issues related to effectiveness and value of resources in health care.
 Theoretical framework to help healthcare professionals, decision-makers or governments to make choices on…
…HOW to maximize the health of population, given constrained health
producing resources
WANTS
AFFORD
BUDGET

6. What is Pharmaco-economic Research?
 Comparison between costs (resources) and consequences or outcomes (clinical, economic, humanistic)
 Types of Economic Evaluations:
 Cost minimization Analysis (CMA)
 Cost-effectiveness Analysis (CEA)
 Cost-utility Analysis (CUA)
 Cost-benefit Analysis (CBA)
 Cost-illness Evaluation (CIE)
 Cost-consequence Analysis (CCA)
 Impact (positive or negative) of substituting existing drug therapies or treatments with newer ones
Description and analysis of the cost and consequences of alternative drug therapy and pharmaceutical
services and their impact on individuals, healthcare systems and society

7. Economic Evaluations
• Serve as an effective tool to help with healthcare decision making

8. Incremental Cost Effectiveness Ratio
(ICER)
Identify Measure Value Compare
• Statistic used to summarize the cost-effectiveness of a health
care intervention.
• Defined by the difference in cost between two possible
interventions, divided by the difference in their effect.
• The ICER can be estimated as:
Where C1 and E1 are the cost and effect in the intervention group and C2 and E2 are the cost
and effect in the control care group

9. ICER Plane

10. Concept of HRQoL
• Quality of life
Broad concept, includes all aspects of life; where and how one lives
and plays; family circumstances; finances; housing and job
satisfaction.
• Health-related quality of life (HRQoL)
Narrower concept, that only includes aspects of life dominated or
significantly influenced by mental or physical well-being.
• Often studied in the field of outcomes and patient-centric research
• Encompasses a wide range of human experience, including functioning and
subjective responses to illness
• Serves as a common measure of gains from any technology
• Patient groups/regulators can use these measures to compare the values of new
technologies
Simplified HRQoL Framework

11. Comparative Effectiveness Research (CER)
• CER involves comparison of two or more drugs, or treatment regimens,
or surgical techniques, or devices, or healthcare policies which are
considered true therapeutic alternatives in actual clinical practice.
• Purpose:
• to evaluate the relative effectiveness of different health care
treatment options
• to encourage the development and use of clinical registries, clinical
data networks, and other forms of electronic data to generate
outcomes data
• CER involves not only the conduct of the trials, but also, synthesis of
results by systematic reviews and meta-analysis and dissemination of the
results.

12. Methods of CER
Gap
identification
Evidence
generation
Pragmatic
trials
Observational
studies
Evidence
synthesis
Systematic
reviews
Meta-analysis

13. Pragmatic Clinical Trials
• The question of ‘pragmatic trials’ is whether the intervention actually
works in real life, which is in contrast to the question of ‘explanatory trials’
• ‘Pragmatic trials’ aim to generate Real World Evidence (RWE) from the
routine daily settings
• Characteristics of pragmatic trials:
• Questions are raised from daily practice
• Heterogeneous settings
• Diverse patient populations
• Comparators arms are those interventions used in daily practice
(instead of placebo)
• Multiple outcomes relevant for daily practice
• Broader inclusion criteria, and patient-centered outcomes
• Patients may be passively followed through routine care with the
help of hospital registries

14. Observational Studies
• Used in routine clinical practice setting
• Involvement of diverse population, large sample size, real world
setting, high external validity, simple design and low cost
• The study environment is not tightly controlled
• Participants are followed in the normal routine set-up
• Can evaluate adverse effects associated with short-term and long-term
use of treatments
• Can compare multiple regimes simultaneously
• Becoming more popular because of easy access to clinical registries,
electronic health records, and administrative databases

15. Systematic Reviews and Meta-Analysis
• A systematic review is a literature review using a highly
structured process to identify, appraise and synthesize all
evidence relevant to a specific health care question.
• Narrative reviews on the other hand…
• Does not involve a systematic search of the literature
• Generally focuses on a subset of studies
• High risk of selection bias
• Reinforces the preconceived thoughts/ideas of the authors
• No quantitative information
Meta-
Analysis
Systematic
Reviews
RCTs
Cohort Studies
Case Control Studies
Case Series/ Case Reports
Background Information / Expert Opinion
Animal Research / Laboratory Studies

16. Application of CER
CER
Generates new
scientific
evidence
Reduces clinical
uncertainty
Disseminates
evidence
Guides
healthcare
choices
Identifying the
treatments
which are not
effective
Summarizing the
likely magnitude
of benefits of
effective
treatments
Identifying
unanticipated
risks of
apparently
effective
treatments
Identifying gaps
of knowledge
Auditing the
quality of
existing RCTs

17. Health Technology Assessment (HTA)
• Evaluation of health technologies for improving
the healthcare investments and to achieve
maximum benefit for the population.
• Useful for:
• the success of universal health coverage
• health policy making
• drug pricing
• development of clinical protocols
• implementation of evidence based medicine
Challenges faced by healthcare systems across the world

18. Health Technology Assessment (HTA)
 The common methods for HTA include
 systematic reviews
 meta-analysis
 cost-effectiveness analysis
 comparative analyses
 modeling
 clinical trials
 epidemiological/observational analyses
 expert opinions
 post-marketing surveillance
 group judgments
 The common end-points include:
 cost/life-year saved
 cost/event avoided
 cost/quality-adjusted life year
 Cost-effectiveness/cost-utility analyses are the most common types of
economic analysis

19. Real World Evidence (RWE)
• Real world data (RWD) - data collected from sources
outside of traditional clinical trials (US FDA).
• These can be:
 Large simple trials or pragmatic clinical trials
 Prospective observational or registry studies
 Retrospective database studies
 Case reports
 Administrative or healthcare claims
 Electronic health records
 Data obtained as part of public health investigation
 Routine public health surveillance
 Registries

20. Sources of RWE

21. Patient Reported Outcomes (PROs)
• A PRO is any report of the status of a patient’s health
condition that comes directly from the patient, without
interpretation of the patient’s response by a clinician or
anyone else.
• The outcome can be measured in absolute terms or as a
change from a previous measure.
• The concepts measured by PRO instruments are used in
support of labeling claims - patient’s symptoms, signs, or an
aspect of functioning directly related to disease status.

22. Significance of PROs
 Provide clinical information for medical decision-making
 Identify patient’s areas of concern that may be unrecognized by the
provider
 Contribute information for pre-planning of visits by the patient care
team,
 Assist clinicians in monitoring patient status longitudinally
 Provide an important source of information about treatment response
and long term outcomes
 To take reimbursement decisions and influence health policy
 USFDA issued guidance documents for industry in 2006 (updated 2009)
to be followed while developing PRO instruments for supporting label
claims

23. Patient Focused Drug Development (PFDD)
• Allows patients to voice their opinions regarding their disease
• How it impacts their life
• Their opinions on the current treatments available
• What they expect from new drugs and therapies in the future
• ‘Voice of the Patient’ reports.
• Impact:
• Gives the patients’ viewpoint to the type of drugs needed in the
market
• Enable the researchers to get a better insight to the problems and
newer avenues for drug discovery
• Helps in the development of Patient Reported Outcomes (PRO)
• Gives payers an insight into how the drug will be accepted post its
launch, by giving feedback from the end users of the drug well
before the launch
• Ensures that the industry is made aware of the needs of the people
or what they expect from the drug.
• Reduces the time taken for patients to gain access to new drugs
Patient
Empowerment
Patient Participation
Patient
Involvement

24. Application of Health Economic Evaluations
• Framework for evaluation of costs and outcomes
• Basis for guiding decisions on healthcare policy and resource allocation
• Valuable tool for priority setting and healthcare decision-making
• Pricing and reimbursement of new drugs
• Investment in new technology
• Implementation of general screening programs
• Making choices between therapies
• Establishment of treatment guidelines
• Prioritization for research funding
• Assessment of new technologies

25. Challenges and Barriers
 When valuing costs and benefits, it is important to determine the most appropriate perspective to
take i.e. patient, third-party payer or society
 Ethics - is alright to base decisions only on monetary values in a particular case?
 Education and training
 Barriers in India
 lack of awareness
 limited knowledge and expertise
 absence of policies/guidelines/consensus
 no central health reimbursement process
 no willingness-to-pay thresholds
 non-uniform delivery of healthcare services
 dearth of data

26. Thank you!
amit.d@marksmanhealthcare.com