Session Type : | Free Webinar |
Topic / Module Title : | Introduction to Health Economics & Outcomes Research (HEOR) |
Speaker Name : | Bibhu Prasad Patra, PhD Scholar, NIPER Mohali |
PHARMIFY LIVE LEARNING SESSION
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Learning Objectives & Seminar Roadmap
Define HEOR scope and trace its historical evolution to value-based healthcare.
Evaluate Economic, Clinical, and Humanistic Patient Outcomes across care settings.
Contrast internal trial efficacy with real-world clinical effectiveness datasets.
Master QALY, DALY metrics and perform real-world ICER cost-utility calculations.
Understand Systematic Literature Reviews (SLR) and Network Meta-Analysis (NMA).
Explore high-demand global job roles across Pharma, CROs, Consultancies, and HTA.
Conceptual Mastery
ECHO Framework
RCTs vs RWD
Economic Metrics
Evidence Synthesis
Career Pathways
Formal Definition
Health Economics
Outcomes Research
What is HEOR? Definition & Scope
Health Economics and Outcomes Research (HEOR) is a multidisciplinary field that evaluates the clinical, economic, and humanistic value of healthcare interventions to inform pricing, reimbursement, and clinical decision-making.
Applies economic principles to optimize resource allocation, cost distribution, and financial sustainability across healthcare systems.
Examines the real-world impact of treatments on clinical endpoints, patient quality of life, survival rates, and mortality.
Healthcare decision-making has shifted dramatically over the last 50 years from subjective clinical intuition to structured value demonstration.
Key Takeaway:
1970s–1980s
Traditional Medicine
Physician intuition, expert opinion, fee-for-service reimbursement models.
1990s–2000s
Evidence-Based
Randomized Controlled Trials (RCTs), clinical guidelines, safety & efficacy focus.
2010s–2020s
Value-Based Care
Pay-for-performance, budget impact, Health Technology Assessment (HTA).
2025+ Era
RWE & AI Ecosystem
AI analytics, Real-World Evidence, patient preferences, holistic value frameworks.
Evolution of Healthcare Decision Making
Key Industry Drivers
Patients
Focus on quality of life, symptom relief, out-of-pocket costs, and functional independence.
Physicians
Focus on clinical efficacy, safety profiles, treatment guidelines, and comparative effectiveness.
Payers & Governments
Focus on cost-effectiveness, population budget impact, and resource allocation efficiency.
Rising Healthcare Costs: Global healthcare spending exceeds $10 Trillion, creating unsustainable public budgets.
Aging Demographics: Increasing global burden of multi-morbid chronic metabolic and cardiovascular diseases.
Precision Medicine: High-cost targeted gene therapies ($1M–$3M per dose) require novel reimbursement structures.
Stakeholder Perspectives in HEOR
RCTs
Primary clinical trial datasets establishing initial safety & efficacy endpoints.
Systematic Review
Exhaustive PICO search and PRISMA filtering to synthesize all global trial evidence.
Meta-Analysis
Quantitative pooling (Pairwise/NMA) to derive comparative effect sizes (HR, OR).
Economic Model
Inputs feed Markov cohorts & decision trees to simulate long-term cost-effectiveness.
HTA Decision
Reimbursement bodies (NICE, CADTH) approve pricing and market coverage.
Clinical Evidence Synthesis Workflow
Phase I / Preclinical
• Target Product Profile (TPP)
• Unmet need quantification
• Early economic modeling
Phase II / III Trials
• Embedding PRO surveys
• Health utility collection
• Resource utilization data
Regulatory & Launch
• Global Value Dossier (GVD)
• HTA dossier submissions
• Price & access negotiation
Post-Marketing
• Real-World Evidence (RWE)
• Label expansion studies
• Risk-sharing contracts
HEOR Integration Across Drug Lifecycle
The Role of SLR in HEOR & Market Access
Systematic Literature Reviews (SLR) form the foundational evidence baseline required by HTA bodies (e.g., NICE, CADTH) to evaluate safety, efficacy, and clinical burden.
SLR Workflow for Economic Modeling
Extracted trial endpoints (e.g., hazard ratios, treatment toxicities, response rates) serve as direct inputs for decision-tree and Markov cost-effectiveness models.
PICO Framework: Rigorous scoping of Population, Intervention, Comparator, and Outcomes.
PRISMA Compliance: Standardized reporting ensuring transparency and reproducibility.
Evidence Synthesis: Identifies clinical trial data gaps and benchmarks standard-of-care.
Clinical Evidence: Systematic Literature Reviews
Direct Pairwise Meta-Analysis
Combines quantitative findings across multiple homogenous trials using Fixed or Random Effects models to determine pooled effect sizes (OR, RR, HR).
Network Meta-Analysis (NMA)
Enables indirect treatment comparisons when head-to-head clinical trial data between novel therapies are missing.
Indirect Comparison: Evaluates Drug A vs Drug B via a common comparator Drug C.
HTA Impact: Essential for generating comparative efficacy metrics demanded by reimbursement agencies globally.
Quantitative Synthesis: Meta-Analysis & NMA
Clinical Relevance:
Economic
Direct medical costs, indirect productivity losses, and emergency room utilization savings.
Clinical
Medical events, biomarker measurements, survival rates, and adverse event frequencies.
Humanistic
Patient-Reported Outcomes (PROs), health-related quality of life (HRQoL), and treatment satisfaction.
The ECHO Model & Patient Outcomes
Decision Rule:
QALY vs DALY
Quality-Adjusted Life Year (QALY): Combines quality and quantity of life lived. (1 QALY = 1 Year in Perfect Health).
Disability-Adjusted Life Year (DALY): Measures overall disease burden (Years of Life Lost + Years Lived with Disability).
Incremental Cost-Effectiveness Ratio
Calculates economic value gained per additional unit of health benefit.
Key Quantitative Metrics: QALY, DALY & ICER
Real Trial Case Scenario
New Drug A Total Cost: $120,000
Standard Care B Cost: $60,000
Incremental Cost (Δ Cost): +$60,000
Incremental Benefit (Δ QALY): +1.50 QALYs
ICER Result & Threshold
Payer Threshold Assessment:
$50,000 / QALY
COST-EFFECTIVE & REIMBURSED!
Incremental Cost-Effectiveness Ratio (ICER) Calculation
Method | Cost Measurement | Outcome Measurement | Primary Application |
Cost-Minimization (CMA) | Monetary ($) | Proven Equivalent Efficacy | Generic vs Brand Drug Comparisons |
Cost-Effectiveness (CEA) | Monetary ($) | Natural Clinical Units (mmHg, life years) | Comparing treatments in same therapeutic area |
Cost-Utility (CUA) | Monetary ($) | QALYs or DALYs (Utility) | Cross-disease resource allocation (Gold Standard) |
Cost-Benefit (CBA) | Monetary ($) | Monetary ($) Net Benefit | Broad public health & government interventions |
Budget Impact Analysis (BIA):
Cost of Illness (COI):
The 4 Primary Economic Evaluation Models
Randomized Controlled Trials (RCTs)
Efficacy in highly controlled, ideal environments with strict inclusion criteria.
Real-World Data (RWD) & Evidence
Effectiveness in routine, heterogeneous patient populations derived from clinical practice.
High internal validity; low risk of bias
Limited external generalizability
Short follow-up duration; small sample size
Insurance claims & Electronic Health Records (EHR)
Patient registries and wearable biosensors
High external validity across diverse populations
Clinical Evidence: RCTs vs Real World Data
Feature / Criteria | Randomized Controlled Trials (RCTs) | Real-World Data (RWD) & Evidence |
Primary Objective | Evaluate clinical efficacy under ideal conditions | Evaluate real-world effectiveness & long-term safety |
Study Environment | Highly controlled, artificial protocol-driven settings | Routine, heterogeneous clinical practice & healthcare systems |
Patient Population | Homogeneous, strictly defined inclusion/exclusion criteria | Diverse patient demographics, multi-morbidities, real age spread |
Internal vs External Validity | High internal validity (low risk of confounding bias) | High external validity (broad generalizability to practice) |
Primary Data Sources | Clinical trial Case Report Forms (CRFs), blinded studies | Insurance claims, Electronic Health Records (EHR), registries |
Clinical Evidence: RCTs vs Real-World Data (RWD)
Case Example (Oncology):
Phase I / Preclinical
Target Product Profile
Early economic modeling, unmet need analysis, establishing required price points.
Phase II / III Trials
Trial Integration
Embedding PRO questionnaires, health utility weights, and resource utilization metrics.
Regulatory & Launch
Global Value Dossier
Submitting HTA dossiers (NICE, CADTH), pricing & reimbursement negotiations.
Post-Marketing
RWE & Expansion
Comparative effectiveness studies, label expansion, post-approval safety commitments.
HEOR in Pharma R&D & Drug Lifecycle
Global Health Technology Assessment (HTA) agencies act as "gatekeepers" evaluating whether new drugs should be covered by public healthcare systems.
Major Global HTA Agencies
Global Value Dossier (GVD)
Comprehensive submission document detailing epidemiological burden, systematic reviews, economic models, and budget impact.
NICE (UK): National Institute for Health and Care Excellence
ICER (USA): Institute for Clinical and Economic Review
G-BA / IQWiG (Germany): Federal Joint Committee
CADTH (Canada) & PBAC (Australia)
Market Access & HTA Bodies
Major Employer Sectors
Key High-Demand Job Roles
Pharma & Biotech: Pfizer, Novartis, Roche, Sanofi, AstraZeneca, GSK, Eli Lilly
CROs & Consultancies: IQVIA, ICON, Parexel, Syneos Health, Evidera
HTA & Government: NICE, ICER, WHO, Public Health Agencies
HEOR Scientist / Health Economist
Real-World Evidence (RWE) Analytics Lead
Global Market Access & Pricing Director
Decision Modeler / Biostatistician
Global Career Opportunities in HEOR
Technical & Quantitative Skills
Strategic & Soft Skills
Statistical Software: R, Python, SAS, SPSS, SQL, Excel VBA
Economic Modeling: Decision Trees, Markov Cohort Models, Partitioned Survival Analysis
Evidence Synthesis: Systematic Literature Reviews (SLRs), Network Meta-Analysis (NMA)
Database Analytics: Claims databases (Truven, CPRD), EHR extraction
Scientific Writing: Publishing peer-reviewed manuscripts in Value in Health or Pharmacoeconomics
Strategic Communication: Translating complex economic models for commercial leadership and payers
Project Management: Managing cross-functional vendor and academic research teams
Regulatory Insight: Navigating evolving FDA RWE guidance and EU Joint Clinical Assessment
Essential HEOR Competency Framework
Top Professional Organization:
1. Academic Foundation
Leverage PharmD / MBBS / MPharm clinical training. Take elective courses in biostatistics, epidemiology, and health economics.
2. Certifications & Tools
Complete ISPOR Distance Learning modules, Coursera Johns Hopkins Public Health courses, or University HEOR certificates.
3. Portfolio & Network
Publish a systematic review, join student ISPOR chapters, optimize LinkedIn profile, and apply for HEOR internships at CROs.
Student Roadmap & Entry Strategy
Generative AI
AI algorithms automating Systematic Literature Reviews, clinical data abstraction, and rapid value dossier generation.
Digital Health
Developing novel health economic evaluation models specifically tailored for software-as-a-medical-device (DTx).
Gene Therapies
Navigating multi-million dollar curative therapies via novel outcome-based risk-sharing agreements.
EU JCA Mandate
EU Joint Clinical Assessment harmonizes clinical effectiveness evidence across all member states.
Future Trends in HEOR (2025–2030)
Key Seminar Takeaways
Case Scenario: Rare Disease Drug
Scenario: A novel gene therapy for Spinal Muscular Atrophy costs $2.1 Million per single injection.
HEOR bridges clinical trial data and real-world health insurance coverage.
Cost-Utility Analysis (CUA) using QALYs is the global benchmark metric.
RWE complements RCTs by providing long-term real-world effectiveness.
High global demand for trained clinical & quantitative HEOR specialists.
Discussion :
Summary & Workshop Practical Case
Contact- bibhuprasad1281998@gmail.com
linkedin.com/in/bibhu-prasad-patra
Questions & Discussion
Thank you for your active participation in this introductory session.
Image Sources
https://library.medicine.yale.edu/wp-content/uploads/2025/01/evidencesynthesis-22-1-scaled.jpg
Source: library.medicine.yale.edu
https://www.healthknowledge.org.uk/sites/default/files/documents/interactivel/fae/making_sense_results/RBC_plot.JPG
Source: www.healthknowledge.org.uk
https://static.biospace.com/fb/d5/3d72d33a4fbcb9c9fae45dc5418b/image001.jpg
Source: www.biospace.com
THANK YOU