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

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.

Co-ordination and management of chronic conditions in Europe: the role of primary care--position paper of the European Forum for Primary Care.

Healthcare systems in Europe struggle with inadequate co-ordination of care for people with chronic conditions. Moreover, there is a considerable evidence gap in the treatment of chronic conditions, lack of self-management, variation in quality of care, lack of preventive care, increasing costs for chronic care, and inefficient use of resources. In order to overcome these problems, several approaches to improve the management and co-ordination of chronic conditions have been developed in European healthcare systems. These approaches endeavour to improve self-management support for patients, develop clinical information systems and change the organisation of health care. Changes in the delivery system design and the development of decision support systems are less common. Almost as a rule, the link between healthcare services and community resources and policies is missing. Most importantly, the integration between the six components of the chronic care model remains an important challenge for the future. We find that the position of primary care in healthcare systems is an important factor for the development and implementation of new approaches to manage and coordinate chronic conditions. Our analysis supports the notion that countries with a strong primary care system tend to develop more comprehensive models to manage and co-ordinate chronic conditions.