The Elusive PICO: Assessing Prediction in Joint Clinical Assessments (JCA): A Case Study in Soft Tissue Sarcoma (STS)
Author(s)
Vijay D'Souza, PhD1, Annete Njue, PhD1, Louise Hartley, PhD1, Komal Singh, PhD2, Michael Mueller, PhD3, Patrick Kieran Hopkinson, MBA, DPhil4, Caroline Ling, BSc, PhD1.
1RTI Health Solutions, Manchester, United Kingdom, 2Sun Pharmaceuticals, Princeton, NJ, USA, 3Sun Pharmaceutical Industries, Inc., Leverkusen, Germany, 4PHTA Consulting, London, United Kingdom.
1RTI Health Solutions, Manchester, United Kingdom, 2Sun Pharmaceuticals, Princeton, NJ, USA, 3Sun Pharmaceutical Industries, Inc., Leverkusen, Germany, 4PHTA Consulting, London, United Kingdom.
OBJECTIVES: To assess the process of defining PICO elements (Population, Intervention, Comparator(s), and Outcomes) ahead of JCA, focusing on STS, a condition characterized by disease subtype heterogeneity and multiple treatment options.
METHODS: We conducted a targeted literature review to inform PICO assessment across selected EU27 member states (MS), based on guidelines, health technology assessments (HTAs), randomized controlled trials (RCTs) and real-world evidence (RWE). A structured search using Nested Knowledge identified and summarized treatments and outcomes in published studies. Country-specific PICOs were summarized, and where feasible, we identified potential consolidated PICOs based on the HTA Coordination Group (HTACG) guidance.
RESULTS: PubMed searches, guideline databases, and desktop searching identified 222 RCTs, 1,050 RWE studies, 6 guidelines, and 24 HTAs in STS globally. Searches identified key themes, including (1) therapies used in first (1L) and later lines and (2) subpopulations with different treatment recommendations resulting in separate PICOs. Although there were clear standard of care (SoC) therapies in 1L STS, 13 different PICOs were identified. In 3L therapy, although there was no clear SoC, 11 possible consolidated PICOs were identified. The number of PICOs identified in the literature per country varied from 18 in France to 4 in Portugal.
CONCLUSIONS: JCA provides a harmonized, scientific rigorous assessment of clinical evidence that requires proactive efforts to anticipate and assess potential PICOs to inform indirect treatment comparisons (ITCs). Heterogeneous conditions such as STS are characterized by many possible treatments and a lack of clear, country-specific guidelines. This leads to varying treatment patterns across countries, multiple comparators and potential PICOs, with limited data to support selected comparisons and inform ITCs. The HTACG can facilitate the development of robust, fit-for-purpose submissions by providing additional guidance on PICO consolidation and working with companies and the MSs to ensure proposed comparators and outcomes align with treatment guidelines and data availability and minimize redundancy.
METHODS: We conducted a targeted literature review to inform PICO assessment across selected EU27 member states (MS), based on guidelines, health technology assessments (HTAs), randomized controlled trials (RCTs) and real-world evidence (RWE). A structured search using Nested Knowledge identified and summarized treatments and outcomes in published studies. Country-specific PICOs were summarized, and where feasible, we identified potential consolidated PICOs based on the HTA Coordination Group (HTACG) guidance.
RESULTS: PubMed searches, guideline databases, and desktop searching identified 222 RCTs, 1,050 RWE studies, 6 guidelines, and 24 HTAs in STS globally. Searches identified key themes, including (1) therapies used in first (1L) and later lines and (2) subpopulations with different treatment recommendations resulting in separate PICOs. Although there were clear standard of care (SoC) therapies in 1L STS, 13 different PICOs were identified. In 3L therapy, although there was no clear SoC, 11 possible consolidated PICOs were identified. The number of PICOs identified in the literature per country varied from 18 in France to 4 in Portugal.
CONCLUSIONS: JCA provides a harmonized, scientific rigorous assessment of clinical evidence that requires proactive efforts to anticipate and assess potential PICOs to inform indirect treatment comparisons (ITCs). Heterogeneous conditions such as STS are characterized by many possible treatments and a lack of clear, country-specific guidelines. This leads to varying treatment patterns across countries, multiple comparators and potential PICOs, with limited data to support selected comparisons and inform ITCs. The HTACG can facilitate the development of robust, fit-for-purpose submissions by providing additional guidance on PICO consolidation and working with companies and the MSs to ensure proposed comparators and outcomes align with treatment guidelines and data availability and minimize redundancy.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA315
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure, Value Frameworks & Dossier Format
Disease
Oncology