G.P.166: Comparative economic impact of therapeutic innovation on health care burden of Duchenne Muscular Dystrophy (DMD) using Becker Muscular Dystrophy (BMD) as a comparator for potential clinical outcome corridor

Abstract

DMD is a fatal incurable progressive disorder resulting in disability, reduced working capacity and high health care utilization often along with intense care. Promising innovative therapies (e.g. exon skipping) enabling transforming disease severity close to the milder BMD phenotype are currently under assessment. Clinical development of these promising therapies is costly and once approved these interventions may add high cost to health care budgets but also saving may be considerable. We aim to assess the cost of illness (COI) and quality of life (QoL) of DMD and BMD patients to pursue comparative modelling of innovative therapies from different perspectives. Patients and caring relatives were surveyed using an established German DMD/BMD patient registry. Direct and indirect cost were assessed in the perspectives of patients, caregivers, relatives and society. The patients’ current QoL was analyzed using a disease-specific questionnaire. 248 DMD and 117 BMD patients were included. Differences between the consumption of resources of direct medical services, indirect cost and QoL between DMD and BMD but also between the different clinical DMD stages were analyzed. Relevant socio-political implications have been identified and compiled. First results show that modifying the disease severity of DMD into a milder BMD clinical phenotype is likely to produce considerably lower cost while improving QoL. Early assessments of economic aspects and modelling of potential outcome corridors represent the first analytical steps towards a systematic health economic assessment of innovative DMD therapies. Our results can contribute to investment decisions on the developer side and may enable to speed up payer negotiations regarding pricing and reimbursement to contribute to the facilitation of a smooth translation of innovations from clinical research over marketing authorization to patient access to a reimbursed therapy.