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

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 uni...

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Veröffentlicht in:The patient : patient-centered outcomes research Jg. 14; H. 5; S. 485 - 503
Hauptverfasser: Whittal, Amanda, Meregaglia, Michela, Nicod, Elena
Format: Journal Article
Sprache:Englisch
Veröffentlicht: Cham Springer International Publishing 01.09.2021
Springer Nature B.V
Schlagworte:
Data collection
Disease
Health Administration
Health Economics
Health technology assessment
Humans
Medicine
Medicine & Public Health
Patient Reported Outcome Measures
Patients
Pharmacoeconomics and Health Outcomes
Preferences
Psychometrics
Public Health
Quality of Life
Quality of Life Research
Quantitative psychology
Questionnaires
Rare diseases
Rare Diseases - therapy
Review
Review Article
Technology Assessment, Biomedical
ISSN:1178-1653, 1178-1661, 1178-1661
Online-Zugang:Volltext
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Zusammenfassung: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 when using different types of PROMs for RDTs, including psychometric properties, was extracted and synthesized. Results Of 79 records from PubMed, 32 were included, plus 12 records from the grey literature. PROMs for rare diseases face potential data collection and psychometric challenges resulting from small patient populations and disease heterogeneity. Generic PROMs are comparable across diseases but not sensitive to disease specificities. Disease-specific instruments are sensitive but do not exist for many rare diseases and rarely provide the utility values required by some HTA bodies. Creating new PROMs is time and resource intensive. Potential solutions include pooling data (multi-site/international data collection), using computer-assisted technology, or using generic and disease-specific PROMs in a complementary way. Conclusions PROMs are relevant in HTA for RDTs but pose a number of difficulties. A deeper understanding of the potential advantages of and the challenges and potential solutions for each can help manage these difficulties.
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ISSN:1178-1653
1178-1661
1178-1661
DOI:10.1007/s40271-020-00493-w