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

ABSTRACT:

Background

Patient-reported outcome measures (PROMs) are used in health technology assessment (HTA) to measure patient experiences with disease and treatment, allowing a deeper understanding of treatment impact beyond clinical endpoints. Developing and administering PROMs for rare diseases poses unique challenges because of small patient populations, disease heterogeneity, lack of natural history knowledge, and short-term studies.

Objective

This research aims to identify key factors to consider when using different types of PROMs in HTA for rare disease treatments (RDTs).

Methods

A scoping review of scientific and grey literature was conducted, with no date or publication type restrictions. Information on the advantages of and the challenges and potential solutions wh

SUBMITTER: Whittal A 

PROVIDER: S-EPMC8357707 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature