Is the New EU HTA Process Fit for Engagement of Patients With Rare Diseases?

Author(s)

Bahar N1, Chamoux C2, Outteridge G3
1Rare Disease Patient Expert, Basel, Switzerland, 2AESARA Europe, RUEIL-MALMAISON, France, 3AESARA Europe, London, LONDON, UK

Patient engagement in Health Technology Assessment (HTA) processes across Europe is still not widely established. The EUnetHTA mandate was to prepare for a future centralized EU HTA. During the Joint Action 3 period (2016-2021), patient engagement strengthened with the involvement of structured patient associations. Building on this experience, the EU HTA regulation (2021/2282) states the importance of engaging stakeholders, including patients, throughout the Joint Clinical Assessment (JCA) process.

OBJECTIVES: Assess how the JCA Implementing Act (IA) was adapted to account for the concerns raised by rare disease patient representatives during public consultation.

METHODS: Feedback from patients representing rare diseases was compiled, drawn from the public consultation on the draft IA (open for comments March-April 2024). The key themes of interest were identified; their inclusion in the IA published 24 May 2024 was checked.

RESULTS: The draft IA received feedback from 47 patient representatives. Twelve organizations were represented and/or included rare disease patients. There were repeated concerns for patient engagement to begin at the very preparatory stages. Timelines were frequently mentioned with concerns around tight, poorly communicated schedules which could impede inclusive patient perspectives. There were many requests to ensure availability of plain language translations, in multiple languages, for wider and more diverse patient participation.

CONCLUSIONS: Rare disease patient representation in EU HTA is progressively increasing. The IA outlines that patient selection will start “as early as possible” and the JCA Sub-group “may” consult European reference networks in recognition and inclusion of patients with rare and complex diseases. Practical measures around representativeness, timelines, and translations are not explicitly cited and remain a concern to rare disease patients.

Conference/Value in Health Info

2024-11, ISPOR Europe 2024, Barcelona, Spain

Value in Health, Volume 27, Issue 12, S2 (December 2024)

Code

HTA304

Topic

Health Technology Assessment

Topic Subcategory

Decision & Deliberative Processes

Disease

No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases

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