BACKGROUND: The 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. RESULTS: 10 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. CONCLUSIONS: The 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.

Association of Austrian Social Insurances Vienna Austria

EURORDIS Rare Diseases Europe Paris France

Medicine Evaluation Committee Brussels Belgium

Menarini Group Florence Italy

Mereo BioPharma Plc London United Kingdom

MoCA Steering Committee Members Brussels Belgium

Rijksinstituut Voor Ziekte en Invaliditeitsverzekering Brussels Belgium

State Institute for Drug Control Prague Czech Republic

Zobrazit více v PubMed

EU Regulation on Orphan Medicinal Products (Regulation (EC) No 141/2000). https://eur-lex.europa.eu/eli/reg/2000/141/oj. Accessed 19 May 2023.

Breaking the Access Deadlock to Leave No One Behind. EURORDIS. 2018. http://download2.eurordis.org.s3.amazonaws.com/positionpapers/eurordis_access_position_paper_final_4122017.pdf. Accessed 19 May 2023.

Corporate Responsibility in the field of Pharmaceuticals. European Commission. 2010. https://ec.europa.eu/commission/presscorner/detail/en/MEMO_10_442. Accessed 19 May 2023.

Process on Corporate Social Responsibility in the Field of Pharmaceuticals Platform on Access to Medicines in Europe Working Group on Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA-OMP). European Commission. 2014. http://download2.eurordis.org.s3.amazonaws.com/moca/history/WG%20MoCA-OMP%20Transparent%20Value%20Framework.pdf. Accessed 19 May 2023.

Medicine Evaluation Committee (MEDEV). https://www.medev-com.eu/. Accessed 19 May 2023.

EURORDIS-Rare Diseases Europe. https://www.eurordis.org/. Accessed 19 May 2023.

MoCA Pilot Project Stage 2: Revised Terms of Reference. MEDEV. 2016. http://download2.eurordis.org.s3.amazonaws.com/moca/today/MOCA%20Revised%20Terms%20of%20Reference%20endorsed%20Jan-2016.pdf. Accessed 19 May 2023.

Mechanism of Coordinated Access to orphan medicinal products (MoCA) webpage. EURORDIS. https://www.eurordis.org/moca/. Accessed 19 May 2023.

Survey monkey. https://www.surveymonkey.com/. Accessed 19 May 2023.

European Medicines Agency - Payer Community meeting 2017. https://www.ema.europa.eu/en/events/european-medicines-agency-payer-community-meeting. Accessed 19 May 2023.

Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency. https://health.ec.europa.eu/system/files/2016-11/reg_2004_726_en_0.pdf. Accessed 19 May 2023.

European Medicines Agency - Payer Community meeting 2019. https://www.ema.europa.eu/en/events/european-medicines-agency-european-union-payer-community-meeting Accessed 19 May 2023.

European Medicines Agency - Payer Community meeting 2021. https://www.ema.europa.eu/en/events/european-medicines-agency-payer-community-meeting-0. Accessed 19 May 2023.

European Medicines Agency – Scientific Advice. https://www.ema.europa.eu/en/human-regulatory/research-development/scientific-advice-protocol-assistance. Accessed 19 May 2023.

Joint Scientific Consultations. EUnetHTA. https://www.eunethta.eu/jsc/. Accessed 19 May 2023.

BeNeLux-I-A. https://beneluxa.org/collaboration. Accessed 19 May 2023.

FINOSE.https://www.fimea.fi/web/en/development/therapeutic_and_economic_value_of_medicines/nordic-collaboration-finose-. Accessed 19 May 2023.

Regulation (Eu) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU. https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32021R2282&from=EN. Accessed 19 May 2023.

EMA tools. Patient Registries. https://www.ema.europa.eu/en/human-regulatory/post-authorisation/patient-registries. Accessed 19 May 2023.

REQqueST tool. https://www.eunethta.eu/request-tool-and-its-vision-paper/. Accessed 19 May 2023.