Patients, payers and developers of Orphan Medicinal Products: lessons learned from 10 years’ multi-stakeholder dialogue on improving access in Europe via MoCA

Orphanet Journal of Rare Diseases

Research

Hans-Georg Eichler¹  Yann Le Cam²  Maria Cavaller-Bellaubi²  Simone Boselli²  Evert Jan Van Lente³  Johan Pontén³  Emanuele Degortes⁴  Wills Hughes-Wilson⁵  Anna Bucsics⁶  Marc Van de Casteele⁷  Šárka Kubinová⁸ 

[1] Association of Austrian Social Insurances, Vienna, Austria;EURORDIS – Rare Diseases Europe, Paris, France;MoCA Steering Committee Members, Brussels, Belgium;Medicine Evaluation Committee (MEDEV), Brussels, Belgium;MoCA Steering Committee Members, Brussels, Belgium;Menarini Group, Florence, Italy;Mereo BioPharma Plc, London, United Kingdom;MoCA Steering Committee Members, Brussels, Belgium;MoCA Steering Committee Members, Brussels, Belgium;Rijksinstituut Voor Ziekte- en Invaliditeitsverzekering (RIZIV-INAMI), Brussels, Belgium;Medicine Evaluation Committee (MEDEV), Brussels, Belgium;MoCA Steering Committee Members, Brussels, Belgium;State Institute for Drug Control, Prague, Czech Republic;MoCA Steering Committee Members, Brussels, Belgium;

关键词: Access to medicines;    Orphan medicinal products;    Multi-stakeholder;    Early dialogues;    Value evaluation;   

DOI  :  10.1186/s13023-023-02774-7

 received in 2023-03-16, accepted in 2023-06-04,  发布年份 2023

来源: Springer

【 摘 要 】

BackgroundThe Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA) was established in 2013 with the intention of developing a coordinated mechanism between volunteering EU stakeholders and developers of Orphan Medicinal Products (OMPs) to support the exchange of information aimed at enabling informed decisions on pricing and reimbursement at Member State level and to evaluate the value of an OMP based on a Transparent Value Framework. The objective of the collaborative approach was to support more equitable access to authorised therapies for people living with rare diseases, rational prices for payers and more predictable market conditions for OMP developers. Over the past 10 years, the MoCA has conducted a series of pilot projects, examining a variety of different products and technologies at different stages of development; and with contributions from a variety of patient representatives, participation from EU payers from a range of Member States and, recently, with EUnetHTA members and the European Medicines Agency participating in the meetings as observers.Results10 years on from the establishment of the MoCA, the European landscape has significantly evolved, not only in the field of drug development with increasingly transformative therapies based on novel technologies, but also in terms of larger numbers of approved treatments, increased budget impact and the resulting associated uncertainties; as well as in terms of stakeholder collaboration and interactions. The value of early dialogue with OMP developers, including the EU payer community via their national decision-making authorities, is a key element within this early interaction and contributes to identifying, managing and reducing uncertainties allowing a prospectively planned approach earlier in development and, consequently, to support more timely, sustainable and equitable access to new OMPs, particularly where there is a high unmet medical need.ConclusionsThe voluntary, informal nature of the MoCA interactions creates a flexible framework for non-binding dialogue. A forum for such interactions is needed to achieve the aims of the MoCA and both to support healthcare systems in planning as well as to underpin timely, equitable and sustainable access to new therapies for patients with rare diseases within the EU.

【 授权许可】

CC BY   
© The Author(s) 2023

【 图 表 】

Fig. 4