Challenges in PICO Definition for HTDs: Lessons From Durvalumab Exercise
Author(s)
Hend Jedidi, MD1, Aleksandra Caban, Head of PRMA department2.
1Market Access, Clever Access, Tunis, Tunisia, 2Market Access, Clever-Access, Cracow, Poland.
1Market Access, Clever Access, Tunis, Tunisia, 2Market Access, Clever-Access, Cracow, Poland.
OBJECTIVES: Since January 2025, Joint Clinical Assessment (JCA) became mandatory for oncology and advanced medicinal products. Its scope, based on the population, intervention, comparator, and outcome (PICO) framework, should align with clinical guidelines and current practice. Six PICO exercises were published to test the scoping process. This research critically analyses from health technology developers (HTDs) perspective the exercise about durvalumab for the first-line treatment of adults with advanced or unresectable hepatocellumar carcinoma.
METHODS: The JCA-published PICO exercise on durvalumab was reviewed with reference to regulatory data, European guidelines and health technology assessment reports.
RESULTS: PICOs 1-4 incorporated the full label population, while PICOs 5-13 targeted subpopulations defined by prognosis (disease severity, operability, performance status). For PICOs 10-11, eligibility for locoregional therapy was specified, but treatment line was missing even though it should align with the same line according to scoping Guidance. This ambiguity arisess from the broadly defined population. Comparators varied across PICOs: immunotherapy combinations, multi-kinase inhibitors (MKIs), procedures, and best supportive care. Guidelines support immunotherapy as the standard of care, with MKIs as alternatives for ineligible patients; some recommended procedures were not reflected in the exercise. Outcome definitions also varied: PICOs 1-4 were consistent, PICO 7 included a broader set of efficacy outcomes, while PICOs 10-11 had limited efficacy and QoL data and omitted safety. Differences between PICOs 5 and 12 reflect the inclusion of more specific QoL measures.
CONCLUSIONS: Although these exercises were designed as methodological tests rather than final PICO scopes, the durvalumab exercise lacked full alignment with guidelines and created a burden for HTDs in terms of feasibility, forecasting, raising concerns for future access to innovative therapies. Early anticipation of PICOs is essential to allow timely data generation and treatment comparisons, especially given the strict 100-day evidence submission deadline after scoping is finalized.
METHODS: The JCA-published PICO exercise on durvalumab was reviewed with reference to regulatory data, European guidelines and health technology assessment reports.
RESULTS: PICOs 1-4 incorporated the full label population, while PICOs 5-13 targeted subpopulations defined by prognosis (disease severity, operability, performance status). For PICOs 10-11, eligibility for locoregional therapy was specified, but treatment line was missing even though it should align with the same line according to scoping Guidance. This ambiguity arisess from the broadly defined population. Comparators varied across PICOs: immunotherapy combinations, multi-kinase inhibitors (MKIs), procedures, and best supportive care. Guidelines support immunotherapy as the standard of care, with MKIs as alternatives for ineligible patients; some recommended procedures were not reflected in the exercise. Outcome definitions also varied: PICOs 1-4 were consistent, PICO 7 included a broader set of efficacy outcomes, while PICOs 10-11 had limited efficacy and QoL data and omitted safety. Differences between PICOs 5 and 12 reflect the inclusion of more specific QoL measures.
CONCLUSIONS: Although these exercises were designed as methodological tests rather than final PICO scopes, the durvalumab exercise lacked full alignment with guidelines and created a burden for HTDs in terms of feasibility, forecasting, raising concerns for future access to innovative therapies. Early anticipation of PICOs is essential to allow timely data generation and treatment comparisons, especially given the strict 100-day evidence submission deadline after scoping is finalized.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA71
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology