HTA appraisal framework for rare disease treatments
A structured approach to assess the value and impact of treatments for rare diseases, considering clinical benefits, costs, safety, and patient experiences.
At a glance
Use when
Assessing new or existing treatments for rare diseases within HTA processes, especially when evidence is sparse or heterogeneous
Avoid when
Evaluating common conditions with abundant evidence; when a rapid assessment without detailed patient input is needed
Inputs
Clinical trial data, real-world evidence, cost data, patient-reported outcomes, epidemiological data
Outputs
Appraisal report summarizing clinical effectiveness, economic impact, safety profile, and patient and social implications
How it works
The framework provides a comprehensive methodology for evaluating rare disease treatments within health technology assessment processes, integrating multiple domains such as clinical effectiveness, economic evaluation, safety profiles, and patient-centered outcomes. It supports decision-making in contexts where evidence may be limited due to small patient populations.
- Project
- IMPACT HTA
- Funding
- Horizon 2020
- Project status
- Completed 2021
- HTA domains
- Aspects Beyond HTA
- Categories
- Appraisal
- Technology
- Medicines
- Assumptions
- Evidence may be limited due to small sample sizes; long-term outcomes might be extrapolated; patient perspectives are critical in decision-making
- Strengths
- Comprehensive multi-criteria assessment; accommodates uncertainty and small population sizes; emphasizes patient and social aspects
- Limitations
- May require subjective judgments due to limited data; resource-intensive to implement; not all criteria may be equally applicable across different rare diseases
Questions this answers
- › What are the clinical benefits of a rare disease treatment?
- › How cost-effective is the treatment compared to existing options?
- › What are the safety concerns associated with the treatment?
- › How does the treatment affect patients' quality of life?
- › What organizational challenges exist in delivering the treatment?
- › How should uncertainty in evidence be handled in appraisal?
Related methods
Similar by meaning
- IMPACT-HTA multicriteria value framework
- Patient group submission form for re-appraisal of a rare disease treatment after an OBMEA
- Guidance on the use of PROMs in HTA of rare diseases
- Framework on HTA methods for addressing drug repurposing challenges
- Analytical framework for determinants of HTA recommendations
Beta record. Generated from the primary source via AI extraction and independent audit, pending final human review.