Get JCA-Ready: Mastering PROs for Market Access Success
Author(s)
Xenia Sitavu-Radu1, Edel Falla, MSc2, Livia Lai, MSc3, Montserrat Casamayor, MSc, PhD, MD4, McVin Cheen, MSc5, Mohamed Gad, MA, MD2, Sofia Oliveira Martins, Msc6, Tom Halmos, BSc, MRes2.
1Health Economist, IQVIA, London, United Kingdom, 2IQVIA, London, United Kingdom, 3IQVIA, Reading, United Kingdom, 4IQVIA, Barcelona, Spain, 5IQVIA, IJmuiden, Netherlands, 6IQVIA, Lisbon, Portugal.
1Health Economist, IQVIA, London, United Kingdom, 2IQVIA, London, United Kingdom, 3IQVIA, Reading, United Kingdom, 4IQVIA, Barcelona, Spain, 5IQVIA, IJmuiden, Netherlands, 6IQVIA, Lisbon, Portugal.
OBJECTIVES: With the implementation of the joint clinical assessment (JCA) in 2025, increasing evidence expectations demand early, strategic planning. As patient reported outcomes (PROs) fall within JCA scope and require thorough justification, we examined how these evolving evidence requirements will impact health technology developers (HTDs).
METHODS: We reviewed EU HTA Coordination Group guidance on outcomes (2024), JCA dossier (2024) and the Food and Drug Administration (FDA, 2022) guidance. Activities and mitigation strategies required to meet JCA PRO requirements were also assessed.
RESULTS: FDA requirements mandate detailed evidence on PROs when seeking a regulatory PRO labelling claim, and HTDs typically develop a PRO statistical analysis plan (SAP), an aspirational PRO narrative, and PRO dossier. Conversely, due to evidence requirements there is a need to plan for JCA when developing PRO strategy even if no PRO labelling claim is sought. Based on EU HTAR guidance, HTDs need to provide details on the validity, reliability and interpretability of the PRO instruments used. In lieu of detailed PRO dossier and SAP typically prepared for FDA, HTDs can deploy a different strategy to address JCA requirements, including a gap analysis focusing on payer-relevant PROs to determine evidence generation needs, targeted literature review to address gaps, pragmatic PRO dossier for key outcomes, PRO statistical analysis plan, and an effective PRO narrative. Adequate timing for analyses and interpretation is needed to ensure JCA requirements are met.
CONCLUSIONS: To ensure alignment with JCA requirements, HTDs must implement a structured, strategic timeline that incorporates JCA-specific activities and integrates the PRO strategy from the earliest stages of development. HTDs need to consider the rigorous PRO evidence requirements for JCA and align to these requirements alongside FDA evidence needs. Pragmatic, evidence-based methodologies that align with JCA requirements and enhance the credibility and acceptability of PRO data are needed to ensure a successful submission.
METHODS: We reviewed EU HTA Coordination Group guidance on outcomes (2024), JCA dossier (2024) and the Food and Drug Administration (FDA, 2022) guidance. Activities and mitigation strategies required to meet JCA PRO requirements were also assessed.
RESULTS: FDA requirements mandate detailed evidence on PROs when seeking a regulatory PRO labelling claim, and HTDs typically develop a PRO statistical analysis plan (SAP), an aspirational PRO narrative, and PRO dossier. Conversely, due to evidence requirements there is a need to plan for JCA when developing PRO strategy even if no PRO labelling claim is sought. Based on EU HTAR guidance, HTDs need to provide details on the validity, reliability and interpretability of the PRO instruments used. In lieu of detailed PRO dossier and SAP typically prepared for FDA, HTDs can deploy a different strategy to address JCA requirements, including a gap analysis focusing on payer-relevant PROs to determine evidence generation needs, targeted literature review to address gaps, pragmatic PRO dossier for key outcomes, PRO statistical analysis plan, and an effective PRO narrative. Adequate timing for analyses and interpretation is needed to ensure JCA requirements are met.
CONCLUSIONS: To ensure alignment with JCA requirements, HTDs must implement a structured, strategic timeline that incorporates JCA-specific activities and integrates the PRO strategy from the earliest stages of development. HTDs need to consider the rigorous PRO evidence requirements for JCA and align to these requirements alongside FDA evidence needs. Pragmatic, evidence-based methodologies that align with JCA requirements and enhance the credibility and acceptability of PRO data are needed to ensure a successful submission.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA164
Topic
Health Technology Assessment
Topic Subcategory
Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas