Patient group submission form for re-appraisal of a rare disease treatment after an OBMEA
This is a form that patient groups can use to share real-life experiences about a rare disease treatment that was accessed through an OBMEA (a type of early access program). It helps describe how patients’ lives changed before and after treatment, and highlights any practical challenges during the access period. HTA bodies can adapt this form or use it as a guide for interviews or focus groups when re-evaluating the treatment.
At a glance
Use when
When an HTA body is re-appraising a rare disease treatment previously accessed via an OBMEA and seeks structured patient input to inform the review.
Avoid when
When patient experiences are unavailable or too heterogeneous to summarize meaningfully, or when a rapid assessment without patient engagement is required.
Inputs
Patient, caregiver, and family experiences collected through surveys, interviews, or focus groups during or after participation in an OBMEA program.
Outputs
Structured qualitative and narrative feedback summarizing patient-reported outcomes, treatment impact, practical issues, and recommendations for HTA re-appraisal.
How it works
A structured template designed for patient organizations to systematically report patient, caregiver, and family experiences related to a rare disease intervention following an OBMEA (Observe & Measure Early Access). Captures qualitative and practical data on treatment impact, quality of life, symptom changes, and access barriers. Intended for adaptation by HTA agencies during re-appraisal processes to inform decision-making with patient-centered evidence.
- Project
- IMPACT HTA
- Funding
- Horizon 2020
- Project status
- Completed 2021
- HTA domains
- Aspects Beyond HTA
- Categories
- Pricing/Payer
- Technology
- Medicines
- Assumptions
- Patient experiences during OBMEA reflect real-world treatment effects and are valuable inputs for HTA re-evaluation; patient groups can accurately represent collective experiences.
- Strengths
- Captures rich, real-world patient insights that may not be evident in clinical trials; supports equitable inclusion of patient voices in HTA; flexible for adaptation or use in interviews/focus groups.
- Limitations
- May be subject to selection or recall bias; lacks quantitative standardization; dependent on patient group capacity to collect and report data.
- Also known as
- Patient submission form for OBMEA re-appraisal, OBMEA patient experience template, Rare disease patient feedback form post-OBMEA
Questions this answers
- › What was life like for patients before they received this treatment through early access?
- › How has the treatment changed patients’ daily lives and symptoms?
- › What challenges did patients face in accessing or using the treatment during the OBMEA?
- › How do patients and families feel about the benefits and side effects of the treatment?
- › What information should HTA bodies consider from patient perspectives when re-evaluating this treatment?
- › Can patient experiences help explain real-world effectiveness and safety?
Similar by meaning
Beta record. Based on the original catalogue summary; primary-source enrichment pending.