Quality of Life and Rare Disease: Lessons from Spinal Muscular Atrophy
The measurement of quality of life in the context of spinal muscular atrophy (SMA) is challenging. This is because the disease is experienced by children and is rare, which makes data collection difficult. Our latest Briefing reports on a symposium…
The measurement of quality of life in the context of spinal muscular atrophy (SMA) is challenging. This is because the disease is experienced by children and is rare, which makes data collection difficult. Our latest Briefing reports on a symposium that outlined some lessons that can be learnt from the SMA context that might be more widely applicable.
The Briefing outlines each speaker’s perspective on quality of life measurement in SMA and rare diseases more broadly. Based on the discussion, we highlight four possible strategies for improving the quantity and quality of data available to inform decisionmakers in the context of rare diseases:
Bespoke data collection which is relevant to health technology assessment decisionmakers;
Simple economic modelling methods, which reflect the evidence available at the time of the assessment;
Collaboration among the different parties involved; and
Identifying what is ‘good enough’ to inform decision-making on use at the time of launch or of the health technology assessment process.
New approaches to research could facilitate health technology assessment processes and improve patients’ access to cost-effective treatments for rare diseases.
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