SpinalMuscularAtrophy
A European Policy & Access Tracker
About the Project
Spinal muscular atrophy (SMA) is a rare, genetic, neuromuscular disease affecting approximately 1 in 10,000 live births globally, with an estimated incidence in Europe ranging from 1 in 3,900–16,000 live births.
Charles River Associates (CRA) conducted a comparative assessment of the policy and access landscape for spinal muscular atrophy (SMA) patients across 23 European countries.
European Comparisons
Interactive Map
5 key policy and access ‘areas’ are covered to ensure that the ideal policy and access environment is analysed with sufficient breadth and depth.
Across the 5 areas, 11 specific metrics were identified for which a detailed analysis has been conducted in each country. Each of the 11 metrics selected for inclusion within the policy and access tracker covers one of the issues that most greatly impacts access to the necessary care for SMA patients in line with the objectives of the study.
SMA
What is Spinal Muscular Atrophy?
Spinal muscular atrophy (SMA) is a rare, genetic, neuromuscular disease affecting approximately 1 in 10,000 live births globally, with an estimated incidence in Europe ranging from 1 in 3,900–16,000 live births.
The SMA Tracker sheds a light on existing gaps affecting SMA patients and allow decision-makers to address them. While people living with SMA can now benefit from different care options, a number of challenges to access these options or connected care continue to exist in various European countries.
David Nestor ,Head of Neuromuscular Diseases at ECP (Europe, Canada and Partner Markets) Biogen
Action
Policy Asks
- Rare diseases should be high on the political agenda, and effective strategies to fight RD should be implemented so that no patient is left behind
- Healthcare systems should ensure that information about the needs of the SMA patient population is always up to date and that the infrastructure system for the provision of SMA treatment and care is adequately aligned with these needs
- Nationwide SMA newborn screening and rapid access to diagnostic procedures should be the routine approach to ensure early disease detection and timely patient access to treatment and care
- Access provisions should be available to support fast patient access to SMA treatment, and reimbursement pathways should consider the specifics of rare disease products to fully capture the value they deliver to rare disease patients and their carers
- Treatment and care approaches should ensure equal access for all SMA patients and their carers