ESTIMATING THE RETURN ON PATIENT ENGAGEMENT IN CLINICAL TRIALS: EVIDENCE FROM A RISK-ADJUSTED NET PRESENT VALUE MODEL FOR A SOLID TUMOR ONCOLOGY PRODUCT
Author(s)
Diego Civitelli, BSC, MSc1, Caitlin Main, BA, MSc1, Kavyashree Satish, MSc1, Jean Mossman, BSc1, Allan Hackshaw, BSC, MSc2, Silvy Mardiguian, PhD3, Tricia Mullins, BSc4, Panos Kanavos, BSc, MSc, PhD1.
1The London School of Economics, London, United Kingdom, 2University College London, London, United Kingdom, 3BeiGene, Amersham, United Kingdom, 4BeiGene, Encinitas, CA, USA.
1The London School of Economics, London, United Kingdom, 2University College London, London, United Kingdom, 3BeiGene, Amersham, United Kingdom, 4BeiGene, Encinitas, CA, USA.
OBJECTIVES: To quantify the financial impact of patient engagement (PE) in clinical trials for a non-orphan solid tumor oncology product and identify relevant value drivers.
METHODS: A targeted literature review across six databases (2015-2025) identified 17 studies on PE practices and impacts. Semi-structured interviews with representatives from five cancer-focused patient organizations informed a framework mapping PE across drug development and reimbursement. A risk-adjusted Net Present Value (rNPV) model for a non-orphan solid tumor drug projected cash flows over 40 years from preclinical research to post-exclusivity. Phase-specific costs, transition probabilities, and durations were drawn from published estimates; regulatory and reimbursement inputs were revenue-weighted across seven markets representing ~72% of global pharmaceutical sales. Three engagement scenarios were compared with a base case of no PE.
RESULTS: In the base case, rNPV was $78.5 million, with break-even at Year 14.5. Scenario 1 (avoidance of one phase 2 protocol amendment plus 90-day phase 3 reduction) yielded $86.1 million (+9.6%, break-even 13.9). Scenario 2 (additional 10% reduction in regulatory approval time) reached $87.2 million (+11.0%, break-even 13.8). Scenario 3 (further 10% reduction in time to reimbursement) reached $88.3 million (+12.4%, break-even 13.7). Gains were driven primarily by extended effective patent life rather than direct cost savings.
CONCLUSIONS: Patient engagement at the protocol design stage could generate returns substantially exceeding its cost, primarily by compressing development timelines and extending market exclusivity. As regulatory and HTA frameworks increasingly mandate patient perspectives, the financial and policy cases for PE appear to be aligned.
METHODS: A targeted literature review across six databases (2015-2025) identified 17 studies on PE practices and impacts. Semi-structured interviews with representatives from five cancer-focused patient organizations informed a framework mapping PE across drug development and reimbursement. A risk-adjusted Net Present Value (rNPV) model for a non-orphan solid tumor drug projected cash flows over 40 years from preclinical research to post-exclusivity. Phase-specific costs, transition probabilities, and durations were drawn from published estimates; regulatory and reimbursement inputs were revenue-weighted across seven markets representing ~72% of global pharmaceutical sales. Three engagement scenarios were compared with a base case of no PE.
RESULTS: In the base case, rNPV was $78.5 million, with break-even at Year 14.5. Scenario 1 (avoidance of one phase 2 protocol amendment plus 90-day phase 3 reduction) yielded $86.1 million (+9.6%, break-even 13.9). Scenario 2 (additional 10% reduction in regulatory approval time) reached $87.2 million (+11.0%, break-even 13.8). Scenario 3 (further 10% reduction in time to reimbursement) reached $88.3 million (+12.4%, break-even 13.7). Gains were driven primarily by extended effective patent life rather than direct cost savings.
CONCLUSIONS: Patient engagement at the protocol design stage could generate returns substantially exceeding its cost, primarily by compressing development timelines and extending market exclusivity. As regulatory and HTA frameworks increasingly mandate patient perspectives, the financial and policy cases for PE appear to be aligned.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR240
Topic
Patient-Centered Research
Topic Subcategory
Patient Engagement
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology