Uncertainties in evaluating the health-related quality of life and disease burden of people with rare diseases and their caregivers in NICE HST submissions.

ABSTRACT:

Background and aims

The NICE Highly Specialised Technology (HST) programme evaluates interventions for very rare conditions within the UK. This review aimed to analyse previous NICE HST appraisals and determine commonly used methods to overcome uncertainties relating to health-related quality of life (HRQoL) and disease burden for people with rare diseases and their caregivers. The review also aimed to identify areas where further methodological development is required.

Approach and results

A targeted review of all previous NICE HST appraisals published by the 28th February 2022, in which at least one committee meeting had taken place, was conducted. A total of 24 appraisals were included (17 fully completed and seven ongoing). Data were extracted by one reviewer. The eviden

SUBMITTER: Looby A 

PROVIDER: S-EPMC11494764 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature