Using Proms in Health Technology Assessment for RARE Disease Treatments
Author(s)
Whittal A*1;Nicod E1;Meregaglia M2, Drummond M3
1Bocconi University, Milan, MI, Italy, 2Bocconi University, Milan, Italy, 3University of York, York, YOR, UK
OBJECTIVES: Using patient-reported outcome measures (PROMs) in Health Technology Assessment (HTA) can enable understanding of treatment value beyond clinical endpoints by measuring patient experiences. In rare diseases, the use of PROMs to evaluate new treatments can be extremely beneficial, but also entails challenges due to small patient populations, disease heterogeneity and poor natural history knowledge. This objective of this study is to identify key aspects that should be taken into account when using different types of PROMs in HTA for rare disease treatments (RDTs).
METHODS: Study approach: Literature Review & Synthesis. We conducted a scoping review of scientific and grey literature with no time or publication type restrictions. Information was extracted related to the opportunities, challenges, and potential solutions of using different types of PROMs for RDTs, including psychometric property implications.
RESULTS: Twenty-seven of 66 identified publications were included from PubMed, as well as five records from the grey literature. Data collection and psychometric challenges resulting from small populations and disease heterogeneity arise regardless of PROM type. Generic PROMs are often comparable across diseases, but not sensitive to a disease; this is a particular issue for RDTs because the small, heterogeneous samples and variation in treatment impact increase the possibility of generic PROMs being poorly applicable. Disease-specific PROMs, one the other hand, are sensitive but often not validated or do not exist for rare diseases. Creating new PROMs requires substantial time and resources. Multi-site or international data collection, use of computer assisted technology and application of different types of PROMs in a complementary way are examples of some potential solutions to the existing challenges.
CONCLUSIONS: The usefulness of PROMs in HTA for RDTs may be undermined by practical challenges. A better understanding of the advantages, challenges and solutions of using PROMs for RDTs can help improve their use in HTA.
METHODS: Study approach: Literature Review & Synthesis. We conducted a scoping review of scientific and grey literature with no time or publication type restrictions. Information was extracted related to the opportunities, challenges, and potential solutions of using different types of PROMs for RDTs, including psychometric property implications.
RESULTS: Twenty-seven of 66 identified publications were included from PubMed, as well as five records from the grey literature. Data collection and psychometric challenges resulting from small populations and disease heterogeneity arise regardless of PROM type. Generic PROMs are often comparable across diseases, but not sensitive to a disease; this is a particular issue for RDTs because the small, heterogeneous samples and variation in treatment impact increase the possibility of generic PROMs being poorly applicable. Disease-specific PROMs, one the other hand, are sensitive but often not validated or do not exist for rare diseases. Creating new PROMs requires substantial time and resources. Multi-site or international data collection, use of computer assisted technology and application of different types of PROMs in a complementary way are examples of some potential solutions to the existing challenges.
CONCLUSIONS: The usefulness of PROMs in HTA for RDTs may be undermined by practical challenges. A better understanding of the advantages, challenges and solutions of using PROMs for RDTs can help improve their use in HTA.
Conference/Value in Health Info
2020-11, ISPOR Europe 2020, Milan, Italy
Value in Health, Volume 23, Issue S2 (December 2020)
Topic
Health Technology Assessment, Patient-Centered Research, Study Approaches
Topic Subcategory
Decision & Deliberative Processes, Literature Review & Synthesis, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Rare and Orphan Diseases