D10.2 Guidance on Use of Patient Reported Outcome Measures in HTA Appraisals of Rare Disease Treatments
| Main Authors: | Elena Nicod, Michela Meregaglia, Amanda Whittal, Sheela Upadhyaya, Karen Facey, Michael Drummond |
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| Format: | info Lainnya Journal |
| Terbitan: |
, 2021
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| Online Access: |
https://zenodo.org/record/5018959 |
Daftar Isi:
- The objective of this workstream conducted within IMPACT-HTA WP10 was to create a guidance document on the use of patient reported outcome measures (PROMs) in health technology assessment (HTA) for rare disease treatments (RDTs). PROMs of all types can be used in HTA, but in some jurisdictions they take the form of health state utility values (HSUVs), since these can be used to calculate the quality-adjusted life-years (QALYs) gained from treatments. We defined the nuances around use of PROMs/HSUVs in rare diseases, illustrated these with examples on use of PROMs/HSUVs in HTA in different countries, and provided guidance on when methodological approaches that differ from the standard may be suitable to measure impact on quality of life (QoL) in rare diseases. A mix of approaches was used including literature scoping/systematic reviews, documentation around use of PROMs in appraisal of RDTs and exploration of better use for evidence of clinical effectiveness and cost-effectiveness.