Health economics and social science research
Social sciences, heath economics and ethico-legal work underpin clinical research and are required for drug development and access in NMDs. Data on quality of life, burden of illness and patient preferences support decisions by companies, regulators and payers.
Our research has developed approaches for and provided first insights into the health economics of rare neuromuscular diseases such as Duchenne muscular dystrophy, spinal muscular atrophy and myotonic dystrophy, facilitated by collaborations with patient organisations, patient registries, academic networks and public-private partnerships. Shared decision-making and the ways individuals with neuromuscular diseases want to be engaged in research were explored at a recent ENMC workshop co-organised by Hanns and feature in the European IMI PREFER and Solve-RD projects.
Through our collaborative work in international projects we aim to ensure that the views and insights of individuals affected by rare diseases play a central role and that the impact of our research is maximised through secure mechanisms for data access and reuse.