From PICOs to comparative evidence: Preparing orphan medicines for EU Joint Clinical Assessment
The landscape of health technology assessment (HTA) in Europe is undergoing a significant shift, and nowhere will the impact be felt more acutely than in the development of orphan medicines. With the EU Health Technology Assessment Regulation (EU HTAR) now in force and the Joint Clinical Assessment (JCA) process expanding to include rare disease therapies from 2028, developers are entering a new era of rigorous, centralized evidence evaluation.
At the World Evidence, Pricing & Access Congress 2026, Patrick Hopkinson and Caroline Ling from RTI Health Solutions discussed the emerging methodological and operational challenges shaping this transition. Drawing on early implementation experience, they highlighted how the JCA framework intensifies long-standing rare disease constraints, including small trial populations, limited epidemiology, heterogeneous comparators, and sparse real-world evidence. These challenges are further compounded by the potential for multiple Member State–driven PICOs, creating evidence requirements that may exceed what rare disease datasets can realistically support.
At the same time, early signals are beginning to emerge. The first JCA reports, expected in mid-2026, will provide important insight into how assessors interpret uncertainty, comparative evidence, and methodological approaches in practice. Early assessments already suggest increasing complexity, with some dossiers including as many as 7–13 PICOs.
This Deep Dive brings together these early insights and practical considerations, offering a clear view of how companies can begin preparing now. From evidence planning to submission strategy, it outlines what developers need to consider as orphan medicines move into the JCA framework.
About the speakers
Caroline Ling
Vice President for Value and Access, RTI Health Solutions
Caroline Ling has over 20 years of experience with market access and health economic consultancies, and is able to deliver a broad spectrum of strategic and tactical market access projects for both global and local clients, including JCA and HTA submissions, systematic and pragmatic literature reviews, global value dossiers, , interactive payer tools, value communication materials, and payer research. Dr Ling has experience in a wide range of therapeutic areas, including rare diseases, hematology, oncology, autoimmune disorders, renal disease, diabetes, mental health, and cardiovascular disorders.
Patrick Hopkinson
Independent Consultant, Founder and Managing Director, PHTA Consulting
Patrick Hopkinson is a Global Market Access/HEOR expert with over 30 years experience. He is an independent consultant, founder and managing director of PHTA Consulting. He is a senior leader in the life sciences industry with extensive expertise gained from working across multiple functions in global pharmaceutical organizations at an executive leadership level.
A former Vice President of Global HEOR at Bristol Myers Squibb, Patrick has a specialist focus on Market Access, HEOR, HTA Policy and RWE combined with extensive commercial experience including P&L responsibility. An HTA expert, he has delivered multiple launches, over 400 submissions across ~35 countries overcoming HTA and Access challenges to optimize commercial performance.
As an EU HTAR expert, Patrick led the BMS enterprise readiness, was a member of the EFPIA HTA working group and is a regular speaker at key conferences. Patrick provides support for organizations preparing for JCA/JSC, i.e., embedding requirements into the product development process, cross-functional working, JSC, PICO predication, JCA strategy, stakeholder planning and dossier submission.
He has been a member of key access policy working groups at EFPIA, PhRMA and ABPI, plus he served as a NICE Appeal Panel member for 10 years. Previously he worked at GSK in Global and Country roles and as a Clinical Oncology Pharmacist in the UK NHS. Patrick is a thought leader and regular conference speaker who is passionate about transforming the life science industry and healthcare stakeholder relationship by focusing on value and the sustainability of healthcare.
Sponsorship for this Deep Dive was provided by RTI Health Solutions