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The Use of Patient‐Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment

Amanda Whittal, Michela Meregaglia, Elena Nicod
January 2021

Patient-reported outcome measures (PROMs), used in health technology assessment (HTA) to measure patient experiences and quality of life with disease and treatment, can enable a deeper understanding of treatment impact beyond clinical endpoints. These are particularly relevant for rare disease treatments (RDTs) considering they most often aim to improve the very poor quality of life affecting patients living with a rare disease. However, unique challenges to measure PROMs in RDTs exist because of the small populations, disease heterogeneity, or lack of natural history knowledge inherent to rare diseases. This scoping review aimed to identify key factors to consider when using different types of PROMs in HTA for RDTs. In total 32 peer reviewed papers and 12 reports from the grey literature were included. Findings revealed key challenges and advantages of using the different types of PROMs, as well as potential solutions, including pooling data, using computer-assisted technology, or using generic and diseases-specific PROMs in a complementary way.

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