Review and Evaluation of Health Equity Considerations in Health Technology Assessments in Cystic Fibrosis

OBJECTIVES: A gap exists between considerations of health equity and the implementation into health technology assessment (HTA) frameworks. This review aims to describe the extent of health equity incorporation within global HTA evaluation of cystic fibrosis medications.

METHODS: National HTA bodies were identified using the International Network of Agencies for Health Technology Assessment (INAHTA) List and independent assessment bodies (European Medicine Agency and Institute for Clinical and Economic Review). Included HTA bodies had public reports in English on cystic fibrosis medications, identified using following search terms: cystic fibrosis, lumacaftor, elexacaftor, tezacaftor, and ivacaftor. Included submissions were reviewed utilizing the Framework for Equity by Benkhalti et al. and the health equity key questions within each HTA evaluations were descriptively reported. Some considerations included: stakeholders involvement, outcome measures and methodological approach.

RESULTS: Of the 33 HTA bodies identified, 12 had at least one report on cystic fibrosis medication. Three HTA bodies were included in the analysis, representing United States, European Union, and Canada. Canada and US included patient engagement and advocacy considerations. There was variable to no consideration of medication access and its impact on outcomes. No health equity methodological considerations were identified. Among the identified HTA reports, there were significant gaps in the level of incorporation and implementation of health equity considerations. Limitations include the exclusion of non-English reports, significantly reducing the sample size.

CONCLUSIONS: The study underscores the need to address gaps in the application and standardization of health equity principles and frameworks. This challenge is further exacerbated by variabilities amongst different countries in terms of their health equity guidance, if any exists. By shedding light on the challenges and opportunities specific to cystic fibrosis, this study provides valuable insights for enhancing the integration of health equity in HTA assessments, especially in disease areas with high unmet needs.