Scoping the Future: Using NICE Scopes to Inform PICO Prediction for EU Joint Clinical Assessment
Author(s)
David Cork, PhD1, Callum C. Bannister, PhD1, Agnieszka Kocwin, MPharm2, Louise McEntee-Richardson, PhD1, Simon Pannett, BSc3, Patrycja Jaros, MSc, PharmD3, Cecile Remuzat, PharmD, MSc4.
1Putnam, Newcastle upon Tyne, United Kingdom, 2Putnam, Kraków, Poland, 3Putnam, London, United Kingdom, 4Putnam, Paris, France.
1Putnam, Newcastle upon Tyne, United Kingdom, 2Putnam, Kraków, Poland, 3Putnam, London, United Kingdom, 4Putnam, Paris, France.
OBJECTIVES: Advanced therapy medicinal products (ATMPs) are among the first wave of technologies eligible for European Joint Clinical Assessment (JCA). We used ATMPs illustratively to evaluate PICO (Population, Intervention, Comparator, Outcome) criteria within published scoping documents from the UK’s National Institute for Health and Care Excellence (NICE) to identify insights which can help health technology developers (HTDs) to predict JCA PICO.
METHODS: ATMPs approved by the EMA (2020-2025) were identified and corresponding NICE scoping documents were reviewed. The number of defined PICOs in each NICE scope were counted and changes between draft and final scopes were assessed. The EU PICO scoping pilot (JCA subgroup, 2024) was compared with the corresponding NICE scope.
RESULTS: Fifteen ATMPs were identified from the EMA, yielding 18 NICE appraisals. Both draft and final scopes were available for 17/18 appraisals. Our analysis identified 1-8 PICOs at draft scope and 1-6 at final, with variability primarily driven by multiple comparators. Changes between draft and final scopes occurred in 9/17 appraisals, most often reduction of PICOs (6/9), suggesting that stakeholder engagement often leads to PICO refinement. The JCA PICO scoping pilot for etranacogene dezaparvovec gave 7 distinct PICOs, while the corresponding NICE scope included 1 PICO. The greater number of JCA PICOs was driven by separation of comparators, while NICE grouped all comparators within “established clinical management”.
CONCLUSIONS: NICE’s consistently published PICO-based scoping documents can be used alongside other sources to support JCA PICO prediction, despite not being part of JCA. However, to accurately predict JCA PICO scopes and prepare for all necessary comparisons, HTDs must anticipate components of standard of care in each market, which may be grouped as a single comparator in NICE scoping documents. A transparent JCA scoping process with opportunity for HTD and other stakeholder inputs could help to reduce uncertainty in preparation for JCA.
METHODS: ATMPs approved by the EMA (2020-2025) were identified and corresponding NICE scoping documents were reviewed. The number of defined PICOs in each NICE scope were counted and changes between draft and final scopes were assessed. The EU PICO scoping pilot (JCA subgroup, 2024) was compared with the corresponding NICE scope.
RESULTS: Fifteen ATMPs were identified from the EMA, yielding 18 NICE appraisals. Both draft and final scopes were available for 17/18 appraisals. Our analysis identified 1-8 PICOs at draft scope and 1-6 at final, with variability primarily driven by multiple comparators. Changes between draft and final scopes occurred in 9/17 appraisals, most often reduction of PICOs (6/9), suggesting that stakeholder engagement often leads to PICO refinement. The JCA PICO scoping pilot for etranacogene dezaparvovec gave 7 distinct PICOs, while the corresponding NICE scope included 1 PICO. The greater number of JCA PICOs was driven by separation of comparators, while NICE grouped all comparators within “established clinical management”.
CONCLUSIONS: NICE’s consistently published PICO-based scoping documents can be used alongside other sources to support JCA PICO prediction, despite not being part of JCA. However, to accurately predict JCA PICO scopes and prepare for all necessary comparisons, HTDs must anticipate components of standard of care in each market, which may be grouped as a single comparator in NICE scoping documents. A transparent JCA scoping process with opportunity for HTD and other stakeholder inputs could help to reduce uncertainty in preparation for JCA.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA296
Topic
Health Technology Assessment
Topic Subcategory
Systems & Structure
Disease
No Additional Disease & Conditions/Specialized Treatment Areas