Guidance on the use of PROMs in HTA of rare diseases
This document provides recommendations on how to incorporate Patient-Reported Outcome Measures (PROMs) when assessing the effectiveness and impact of health technologies for rare diseases, ensuring patient perspectives are included in decision-making.
At a glance
Use when
Conducting HTA for orphan drugs or treatments for rare diseases; developing health technology submission dossiers requiring patient-reported evidence; designing clinical trials with patient-centered endpoints
Avoid when
PROMs are not relevant to the health outcome of interest; no capacity to collect or validate patient-reported data; when disease severity prevents reliable patient reporting
Inputs
Patient-reported data, choice of PROM instruments, disease-specific characteristics, regulatory and HTA requirements
Outputs
Recommendations for PROM selection and use, guidance on data collection methods, framework for integrating patient-reported evidence into HTA dossiers
How it works
The guidance outlines methodological considerations for selecting, applying, and interpreting PROMs in Health Technology Assessment (HTA) processes specific to rare diseases, addressing challenges such as small sample sizes, heterogeneity of conditions, and lack of validated instruments, while promoting standardization and patient involvement.
- Project
- IMPACT HTA
- Funding
- Horizon 2020
- Project status
- Completed 2021
- HTA domains
- Clinical Effectiveness, Costs & Economic Evaluation
- Technology
- Medicines
- Assumptions
- PROMs are feasible to collect despite small populations; existing PROMs can be adapted or validated for rare diseases; patient input improves decision relevance
- Strengths
- Supports patient-centered assessment; enhances validity of benefit-risk evaluation in low-prevalence settings; promotes consistency across HTA bodies
- Limitations
- Limited availability of validated PROMs for specific rare diseases; challenges in statistical power due to small samples; potential bias in self-reported data
- Also known as
- PROMs in rare diseases HTA, Patient-Reported Outcomes in HTA for rare diseases
Questions this answers
- › What are PROMs and why are they important in rare diseases?
- › How can patient-reported outcomes be reliably measured when patient numbers are very small?
- › Which PROMs are suitable for use in rare disease assessments?
- › How should PROM data be collected and interpreted in HTA submissions?
- › What challenges exist in using PROMs for rare diseases and how can they be addressed?
- › How can patient perspectives be integrated into HTA using PROMs?
Related methods
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Beta record. Generated from the primary source via AI extraction and independent audit, pending final human review.