Evidence-Based Decision Making | Strategic Planning | Real-World Evidence
HEOR/Market access professional with 10+ years designing and executing value strategies to support clinical development, pricing, reimbursement, and commercialization. Experienced in engaging cross-functional teams and external stakeholders. Demonstrated success in generating impactful RWE, economic models and payer engagements across multiple therapeutic areas including rare disease, infectious and cardiovascular diseases.
Explore comprehensive case studies demonstrating expertise in Integrated Evidence Planning, market access strategy, and outcomes research across diverse therapeutic areas.
Comprehensive IEP development for a novel immuno-oncology agent, addressing global market access challenges and HTA requirements across multiple jurisdictions.
Strategic evidence generation plans including ultra-rare metabolic disease and comprehensive Polycythemia Vera HEOR strategy with comparative effectiveness framework across multiple comparators.
Real-world evidence studies across comparative effectiveness research and hepatitis D clinical outcomes and economic burden.
Complete health technology assessment submission for NICE, including economic model development, cost-effectiveness analysis, and budget impact assessment.
Comprehensive PRO strategy framework across influenza, oncology, and liver disease. Includes instrument selection (Flu-iiQ, EORTC QLQ-C30/CR29, FACT-C, EQ-5D, SF-36), stakeholder messaging, and economic modeling for HTA submissions.
State-transition Markov model for chronic disease management, including deterministic and probabilistic sensitivity analyses with comprehensive validation.
Claims databases, electronic health records (EHRs), registries and observational cohort data
Strategic evidence planning and health economics & outcomes research
Comprehensive market access strategies for global markets
Evidence generation strategies across product lifecycle
Cost-effectiveness models, budget impact analyses, and value frameworks
Patient-reported outcomes and clinical endpoint strategy
Design and execution of prospective research initiatives
Cross-functional collaboration and stakeholder engagement
End-to-end project leadership and execution
Specialized expertise in orphan drug development and rare disease evidence
HEOR and evidence strategies for medical devices and diagnostics
A comprehensive literature review tool for systematic evidence synthesis and analysis is currently under development. This tool will enable efficient literature searches, evidence extraction, and synthesis across multiple databases and sources.
Name
Prajakta Bhounsule
Developed a comprehensive IEP for a first-in-class checkpoint inhibitor targeting advanced melanoma, designed to support global regulatory approval and market access across 20+ countries.
Conducted systematic review of HTA requirements across EU5, US, Canada, and Australia. Analyzed competitor evidence strategies and payer perspectives.
Mapped clinical development program outputs against HTA evidentiary requirements. Identified 12 critical evidence gaps requiring additional data generation.
Designed 5-year evidence generation roadmap including RCTs, RWE studies, and PRO collection. Developed economic model conceptual framework.
Facilitated cross-functional workshops with clinical, regulatory, commercial, and medical affairs. Obtained senior leadership approval for $15M evidence budget.
Systematic HTA Research: Analyzed submission guidelines from NICE, IQWiG, HAS, CADTH, PBAC, and regional payers.
Evidence Mapping: Created comprehensive matrix linking clinical trial endpoints to HTA decision criteria across jurisdictions.
Stakeholder Engagement: Conducted 25+ interviews with KOLs, payers, and patient advocacy groups to inform evidence priorities.
Economic Modeling: Developed cost-effectiveness model framework aligned with reference case requirements across major HTA bodies.
Develop a comprehensive IEP for X designed to support global regulatory approval and market access across in the US and EU5 countries.
Orphan drug for ultra-rare lysosomal storage disorder (prevalence: 1:100,000) with limited natural history data and no existing approved therapies. Challenge: demonstrating value to payers despite small patient population and high treatment costs.
Established international patient registry (n=150) to characterize disease progression, healthcare utilization, and quality of life impacts over 3-year follow-up period.
Optimized Phase III trial design with innovative endpoints combining clinical, functional, and patient-reported outcomes. Included health economics data collection.
Designed expanded access program with prospective data collection to supplement pivotal trial evidence and address long-term effectiveness questions.
Created multi-stakeholder value framework emphasizing prevention of irreversible complications, caregiver burden reduction, and societal benefits.
Small Population Challenges: Implemented Bayesian approaches and adaptive trial designs to maximize limited patient pool.
Societal Perspective: Developed broader value framework including caregiver productivity, educational impacts, and prevention of long-term complications.
Early Engagement: Facilitated Scientific Advice meetings with EMA and FDA to align on evidence requirements and accelerated pathways.
Comprehensive HEOR strategy for Polycythemia Vera asset including integrated evidence planning, real-world evidence generation, and comparative effectiveness framework across multiple treatment comparators.
Targeted analysis across key patient populations:
Real-World Evidence:
Quality of Life:
Cost-Effectiveness:
Authors: Schneider DB, Krajcer Z, Bonafede M, Thoma E, Hasegawa J, Bhounsule P, Thiel E
Journal: Journal of Comparative Effectiveness Research. 2019 Dec;8(16):1381-1392
This retrospective real-world evidence study compared clinical and economic outcomes between patients undergoing arterial repair using the Perclose ProGlide vascular closure device versus surgical cutdown following large-bore arterial access procedures.
Study Population: Matched patients who underwent transcatheter aortic valve replacement/repair (TAVR), endovascular abdominal aortic aneurysm repair (EVAR), thoracic endovascular aortic repair (TEVAR), or balloon aortic valvuloplasty between January 2013 and April 2017.
The use of Perclose ProGlide for arterial repair was associated with significantly lower transfusion rates, shorter index hospitalization, and lower hospitalization costs compared with surgical cutdown, supporting the clinical and economic value of percutaneous vascular closure devices in real-world practice.
Note: Study conducted at IBM Watson Health with support from Abbott Vascular. Analysis utilized real-world electronic health records and claims data with rigorous matching methodology.
AASLD 2024 Conference Presentations at The Liver Meeting® | San Diego, CA | November 2024
Lead Author: Prajakta Bhounsule, Director, Health Economy and Marketing, Vir Biotechnology, Inc.
Co-Authors: Elizabeth Kinter, Nancy Reau, Benjamin Liebov
Presentation: Poster #1174 at The Liver Meeting® 2024 (AASLD Annual Conference)
Session: Hepatitis – Other Infections | November 15, 2024 | San Diego, CA
Link: ResearchGate Publication
Hepatitis D virus (HDV) infection is associated with higher risk of adverse clinical outcomes including cirrhosis, hepatocellular carcinoma (HCC), and mortality compared to chronic hepatitis B alone. HDV co-infection accelerates progression towards liver cancer and liver-related death by almost a decade compared to HBV mono-infected persons. To support the development of new therapies for this severe form of viral hepatitis, it is critical to understand the evidence on surrogate endpoints and non-invasive assessment methods.
This evidence review addresses a critical gap in hepatitis D drug development by evaluating the validity and utility of non-invasive biomarkers. Given that HDV is considered by the World Health Organization to be the most severe form of chronic viral hepatitis, with limited treatment options and no cure, establishing validated surrogate endpoints is essential for accelerating therapeutic development.
Clinical Impact: FibroScan (VCTE) has been validated in large cohorts worldwide across multiple liver diseases including hepatitis B and C. The technology provides non-invasive assessment of liver fibrosis and has demonstrated ability to predict liver-related events and mortality, making it a valuable tool for monitoring disease progression in HDV patients.
Presenter: Prajakta Bhounsule, Director, Health Economy and Marketing, Vir Biotechnology, Inc.
Presentation: Poster #3294 at The Liver Meeting® 2024 (AASLD Annual Conference)
Session: Health Services and Public Health Research | November 17, 2024 | San Diego, CA
Announcement: Vir Biotechnology Press Release
This health economics research evaluated the economic burden of hepatitis D virus (HDV) infection across different patient segments, providing critical insights into the societal and healthcare system costs associated with this severe liver disease.
Disease Severity: Chronic hepatitis delta (CHD) affects nearly 5% of people with chronic HBV infection. HDV co-infection represents the most severe form of chronic viral hepatitis with accelerated progression to cirrhosis and hepatocellular carcinoma.
Unmet Need: With no cure and limited treatment options, understanding the economic burden of HDV is essential for:
Value to Stakeholders: This research provides critical evidence for payers, policymakers, and healthcare systems to understand the true cost of HDV infection and the potential value proposition of emerging therapies targeting this underserved patient population.
Conference Context: Both presentations were part of Vir Biotechnology's comprehensive data package at AASLD The Liver Meeting® 2024, which included clinical trial results from the Phase 2 SOLSTICE trial evaluating investigational therapies (tobevibart and elebsiran) for chronic hepatitis delta, alongside real-world evidence and health economics research.
Complete HTA submission for novel GLP-1 receptor agonist for Type 2 diabetes mellitus, following NICE Single Technology Appraisal (STA) process and meeting all methodological requirements of the NICE reference case.
Systematic literature review and network meta-analysis of 47 RCTs comparing efficacy and safety versus 8 relevant comparators. GRADE quality assessment and bias evaluation.
Patient-level simulation model (validated UKPDS OM2) projecting 40-year outcomes including microvascular and macrovascular complications, QALYs, and costs from NHS perspective.
5-year budget impact model estimating financial impact on NHS considering eligible population, uptake rates, and displaced therapies.
Comprehensive DSA (50+ parameters), PSA (10,000 iterations), and scenario analyses addressing committee concerns about long-term adherence and cardiovascular benefits.
Outcome: Positive recommendation for reimbursement as second-line therapy in patients with inadequate glycemic control on metformin.
Restrictions: Limited to patients with BMI ≥30 kg/m² or specific circumstances where weight loss would benefit other comorbidities.
Implementation: Recommendation published and incorporated into NHS formulary within 3 months of final guidance.
Methodological Rigor: Adherence to NICE reference case and use of validated diabetes model (UKPDS OM2).
Stakeholder Input: Early engagement with patient groups and clinical experts strengthened value proposition.
Responsive Analysis: Rapid turnaround of requested sensitivity analyses and scenario modeling during appraisal process.
PRO Strategy is a structured plan for how patient-reported outcomes will be selected, collected, analyzed, and used across a clinical development program to support:
It helps to capture the patient perspective on symptoms, functioning, and quality of life beyond health markers and supports payer needs.
Comprehensive overview of patient-reported outcome instruments with measurement details, value messages, and stakeholder relevance.
PRO:
What does it measure:
Value Message:
Patients with intervention report higher health-related quality of life
Stakeholders:
PRO:
What does it measure:
Value Message:
Patients with intervention report higher QoL compared to placebo or comparator. Used for health utility / cost-effectiveness modelling.
Stakeholders:
PRO:
What does it measure:
Value Message:
Patients with intervention report higher health-related quality of life. Used for health utility / cost-effectiveness modelling.
Stakeholders:
State-transition Markov cohort model evaluating cost-effectiveness of novel triple therapy inhaler versus dual therapy and long-acting bronchodilator monotherapy in moderate-to-severe COPD patients.
Five health states defined by GOLD severity classification (moderate, severe, very severe), acute exacerbations (moderate, severe requiring hospitalization), and absorbing state (death).
3-month cycles over lifetime horizon (30 years) with half-cycle correction. Annual discount rate of 3.5% for costs and QALYs (UK perspective).
Derived from meta-analysis of pivotal trials (n=3 RCTs, 8,000+ patients) for treatment effects. Background progression and mortality from published cohort studies and UK life tables.
NHS reference costs for hospitalizations and primary care. EQ-5D utilities from trial data with published decrements for exacerbations and GOLD progression.
Internal Validation: Extreme value testing, null comparator checks, Excel formula auditing by independent analyst.
External Validation: Model predictions compared against published COPD natural history studies and registry data. Good agreement achieved for 5-year exacerbation rates and mortality.
Cross-Validation: Results replicated independently in TreeAge and R to ensure programming accuracy.
Deterministic SA: Tornado diagram identified exacerbation rates, utility values, and hospitalization costs as key drivers. Results remained <£20K/QALY across plausible ranges.
Probabilistic SA: 10,000 Monte Carlo iterations with distributional assumptions (beta for probabilities/utilities, gamma for costs, lognormal for RRs). Cost-effectiveness acceptability curve demonstrated 96% probability of cost-effectiveness at £30K/QALY threshold.
Scenario Analyses: Alternative time horizons (5, 10, 20 years), discount rates (0%, 6%), treatment effect waning (none, 5 years), and societal perspective (including productivity losses).