Fundamentals of Health Economics and Outcomes Research (HEOR)

> 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 Multidimensional health outcomes are reduced to a single index using health utilities.

4. Key Stakeholders in HEOR

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

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

16. Application of CER

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

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

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