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.

Participants at ENMC workshop on shared decision-making

Relevant publications

Landfeldt, E, Alemán, A, Abner, S, Zhang, R, Werner, C, Tomazos, I et al.. Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis. J Neuromuscul Dis. 2024.11 (3)579-612 PMID:38669554

Marcì, M, Crescimanno, G, Vaccaro, P. [Sudden cardiac death in Duchenne muscular dystrophy]. G Ital Cardiol (Rome). 2024.25 (4)294 PMID:38526366

Landfeldt, E, Phung, K, Zaman, F, Åström, E, Abner, S, Lochmüller, H et al.. Bisphosphonates in Glucocorticoid-Treated Patients With Duchenne Muscular Dystrophy: A Systematic Review and Grading of the Evidence. Neurology. 2024.102 (2)e207948 PMID:38165327

Landfeldt, E, Aleman, A, Abner, S, Zhang, R, Werner, C, Tomazos, I et al.. Factors Associated with Respiratory Health and Function in Duchenne Muscular Dystrophy: A Systematic Review and Evidence Grading. J Neuromuscul Dis. 2024.11 (1)25-57 PMID:37980679

Landfeldt, E, Abner, S, Pechmann, A, Sejersen, T, McMillan, HJ, Lochmüller, H et al.. Caregiver Burden of Spinal Muscular Atrophy: A Systematic Review. Pharmacoeconomics. 2023.41 (3)275-293 PMID:36515815

Landfeldt, E, Pechmann, A, McMillan, HJ, Lochmüller, H, Sejersen, T. Costs of Illness of Spinal Muscular Atrophy: A Systematic Review. Appl Health Econ Health Policy. 2021.19 (4)501-520 PMID:33576939

Landfeldt, E, Nikolenko, N, Jimenez-Moreno, C, Cumming, S, Monckton, DG, Faber, CG et al.. Change over time in ability to perform activities of daily living in myotonic dystrophy type 1. J Neurol. 2020.267 (11)3235-3242 PMID:32542526

Landfeldt, E, Pogoryelova, O, Sejersen, T, Zethraeus, N, Breiner, A, Lochmüller, H et al.. Economic Costs of Myasthenia Gravis: A Systematic Review. Pharmacoeconomics. 2020.38 (7)715-728 PMID:32363541

Mascalzoni, D, Dove, ES, Rubinstein, Y, Dawkins, HJ, Kole, A, McCormack, P et al.. International Charter of principles for sharing bio-specimens and data. Eur J Hum Genet. 2015.23 (6)721-8 PMID:25248399

Landfeldt, E, Lindgren, P, Bell, CF, Schmitt, C, Guglieri, M, Straub, V et al.. The burden of Duchenne muscular dystrophy: an international, cross-sectional study. Neurology. 2014.83 (6)529-36 PMID:24991029