Evidence Strategy Starts Early: Integrating HTA Needs Through PICO Simulation in Metastatic Castration-Resistant Prostate Cancer
Author(s)
Parth Joshi, M.Pharm.1, Michael Schmoeller, PhD2, Charlotte Mirland-Deschamps, MSc.3, Belen Neches Carretero, MSc.4, Dafne Mossoni, BSc.5, Damiano Bisagni, MSc.5, Liberty Fajutrao, MD, MSCE6, Enrico Grabbi, MSc.7, Valéry Risson, MBA, PhD8.
1Novartis Healthcare Private Limited, Hyderabad, India, 2Novartis Pharma GmbH, Munich, Germany, 3Novartis Pharma S.A.S., Rueil Malmaison, France, 4Novartis Farmacéutica, S.A., Barcelona, Spain, 5Novartis Farma S.p.A., Milan, Italy, 6Novartis Sverige AB, Stockholm, Sweden, 7Novartis Pharma AG, Geneva, Switzerland, 8Novartis Pharma AG, Basel, Switzerland.
1Novartis Healthcare Private Limited, Hyderabad, India, 2Novartis Pharma GmbH, Munich, Germany, 3Novartis Pharma S.A.S., Rueil Malmaison, France, 4Novartis Farmacéutica, S.A., Barcelona, Spain, 5Novartis Farma S.p.A., Milan, Italy, 6Novartis Sverige AB, Stockholm, Sweden, 7Novartis Pharma AG, Geneva, Switzerland, 8Novartis Pharma AG, Basel, Switzerland.
OBJECTIVES: To explore and evaluate potential Population, Intervention, Comparator, Outcomes (PICO) scenarios through a cross-country simulation exercise to support European Joint Clinical Assessment (EU JCA) readiness for a treatment under development in heavily pre-treated metastatic castration-resistant prostate cancer (mCRPC) patients.
METHODS: Base-case PICO was derived from the proposed clinical trial including advanced mCRPC patients who have received multiple lines of prior systemic therapies. Comparators included investigator’s choice of standard of care. A PICO prediction survey was conducted with colleagues from Germany, France, Italy, Spain, and the Nordics to understand their perspectives on relevance of the clinical trial design and the base-case PICO, as well as to elicit alternative relevant PICO scenarios. Multiple scenarios were developed primarily based on population variations and currently approved comparator treatment options.
RESULTS: Substantial heterogeneity was observed among the PICO inputs received from countries; eight alternative PICO scenarios with limited overlap across the countries were identified. Three of the eight PICO scenarios were based on alternative relevant comparators, whereas the remaining PICOs reflected patient population variations in terms of mutation status (n=2) or subgroups based on metastasis (n=1), functional status (n=1), and previously failed therapies (n=1). While there was a general alignment to the base-case outcomes across most markets, overall survival was the only endpoint considered most relevant by all the countries.
CONCLUSIONS: Identification of multiple and non-overlapping alternative PICO scenarios highlights the complexity and variability in country-level health technology assessment (HTA) requirements. Disease-specific challenges including population heterogeneity and evolving comparators suggest the need for regular stakeholder engagement and scenario testing. Early mapping of such variations provides a basis for further PICO refinement, gap analysis, and prioritization of evidence to support the overall EU JCA strategy.
METHODS: Base-case PICO was derived from the proposed clinical trial including advanced mCRPC patients who have received multiple lines of prior systemic therapies. Comparators included investigator’s choice of standard of care. A PICO prediction survey was conducted with colleagues from Germany, France, Italy, Spain, and the Nordics to understand their perspectives on relevance of the clinical trial design and the base-case PICO, as well as to elicit alternative relevant PICO scenarios. Multiple scenarios were developed primarily based on population variations and currently approved comparator treatment options.
RESULTS: Substantial heterogeneity was observed among the PICO inputs received from countries; eight alternative PICO scenarios with limited overlap across the countries were identified. Three of the eight PICO scenarios were based on alternative relevant comparators, whereas the remaining PICOs reflected patient population variations in terms of mutation status (n=2) or subgroups based on metastasis (n=1), functional status (n=1), and previously failed therapies (n=1). While there was a general alignment to the base-case outcomes across most markets, overall survival was the only endpoint considered most relevant by all the countries.
CONCLUSIONS: Identification of multiple and non-overlapping alternative PICO scenarios highlights the complexity and variability in country-level health technology assessment (HTA) requirements. Disease-specific challenges including population heterogeneity and evolving comparators suggest the need for regular stakeholder engagement and scenario testing. Early mapping of such variations provides a basis for further PICO refinement, gap analysis, and prioritization of evidence to support the overall EU JCA strategy.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA138
Topic
Health Technology Assessment
Topic Subcategory
Systems & Structure
Disease
Oncology