Office of Health Economics Briefing, 2 April 2019
- Based on discussion of current approaches to the measurement of quality of life in Spinal Muscular Atrophy, the report highlights four possible strategies for improving the quantity and quality of data available to inform decision makers in the context of rare diseases:
- Bespoke data collection which is relevant to health technology assessment decision makers;
- 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.
