Patient group submission form for re-appraisal of a rare disease treatment after an OBMEA
This template helps patient groups share real-life experiences of patients and carers who received a rare disease treatment through an Outcomes-Based Managed Entry Agreement (OBMEA). It guides them to report on how the condition and treatment affected daily life, access challenges, and outcomes not captured in clinical data, to inform HTA re-appraisal decisions.
At a glance
Use when
During re-appraisal of a rare disease treatment following an Outcomes-Based Managed Entry Agreement; when HTA bodies seek structured patient input on real-world treatment experiences and unmet needs.
Avoid when
When no OBMEA has been conducted; when patient groups lack capacity to gather representative input; when only quantitative data is required for re-appraisal.
Inputs
Qualitative and experiential data from patients, carers, and families who participated in an OBMEA; input from patient groups on data collection methods and international insights.
Outputs
Structured patient group submission for HTA committee consideration during re-appraisal, including key messages on treatment impact, unmet needs, and recommendations for future use.
How it works
A structured template developed for patient group submissions during the re-appraisal phase of rare disease treatments following an OBMEA. It includes seven sections covering group background, data collection methods, pre-treatment disease burden, OBMEA access and implementation issues, treatment experiences, additional international insights, and key messages for HTA committees. Designed to capture patient-relevant outcomes and contextual evidence beyond formal OBMEA data collection, supporting more holistic HTA decision-making.
- Project
- IMPACT HTA
- Funding
- Horizon 2020
- Project status
- Completed 2021
- HTA domains
- Aspects Beyond HTA
- Categories
- Pricing/Payer
- Technology
- Medicines
- Assumptions
- Patient groups can collect and report representative experiences from those treated under OBMEA; HTA bodies value balanced patient input; real-world experiences complement clinical and economic evidence in re-appraisal.
- Strengths
- Structured yet flexible format allows comprehensive patient input; focuses on outcomes important to patients and carers; supports transparency and consistency in patient involvement; adaptable across health systems; promotes balanced reporting of benefits and challenges.
- Limitations
- Relies on patient group capacity and resources; may be influenced by selection bias if only certain patients participate; not all patient voices may be captured equally; requires adaptation to local HTA processes.
- Also known as
- Patient Group Submission Form for OBMEA Re-appraisal, IMPACT HTA Patient Submission Template, Template for Patient Input in Rare Disease Re-appraisal
Questions this answers
- › What impact did the treatment have on patients' daily lives that wasn't measured in the OBMEA?
- › How did the condition affect carers and families before and after treatment?
- › Were there problems accessing or staying in the OBMEA?
- › Did patients experience unexpected benefits or side effects?
- › What should the HTA committee know about real-world patient experiences?
- › How can patient input improve future use of this treatment in routine care?
References & sources
- websiteimpact-hta.eu ↗
- paperDOI: 10.1007/s40273-021-01050-5 ↗
Related methods
Similar by meaning
Beta record. Generated from the primary source via AI extraction and independent audit, pending final human review.