Pilot MCDA framework for valuing orphan medicines (Sussex et al.)
This framework helps assess the value of medicines for rare diseases when standard cost-per-QALY methods don't work well. It uses eight criteria—like how serious the disease is and how much the treatment improves life—that are not based on money.
At a glance
Use when
Evaluating orphan or ultra-orphan medicines with high uncertainty, small populations, or where QALYs are inadequate.
Avoid when
When robust clinical and economic data support standard cost-effectiveness analysis; in settings lacking capacity for MCDA implementation.
Inputs
Disease severity, progression rate, unmet need, rarity, comparative efficacy, safety profile, adherence requirements, caregiver burden.
Outputs
Structured value assessment of orphan medicines using weighted criteria scores, supporting reimbursement and pricing decisions.
How it works
A pilot Multi-Criteria Decision Analysis (MCDA) framework developed by Sussex et al. at the Office of Health Economics. It includes eight non-monetary criteria: four disease-related (severity, progression, unmet need, rarity) and four treatment-related (comparative efficacy, safety, adherence, caregiver impact). Designed for orphan medicines where traditional cost-per-QALY evaluation is inadequate due to high costs, small populations, and uncertain outcomes.
- HTA domains
- Clinical Effectiveness, Patient and Social Aspects, Aspects Beyond HTA
- Assumptions
- Decision-makers can reliably score non-monetary criteria; stakeholder preferences can be incorporated; QALY-based methods are insufficient for rare diseases.
- Strengths
- Addresses limitations of cost-per-QALY in rare diseases; incorporates patient and societal values; transparent and structured decision-making process.
- Limitations
- Requires subjective weighting and scoring; limited real-world validation in diverse settings; may be resource-intensive to implement.
- Also known as
- Sussex MCDA framework for orphan medicines, OHE MCDA framework (orphan drugs)
Questions this answers
- › How severe is the disease being treated?
- › Does the treatment offer meaningful improvement over existing options?
- › How does the treatment impact patient quality of life and daily living?
- › What is the level of unmet need for this condition?
- › How rare is the disease?
- › What are the safety and adherence implications of the treatment?
References & sources
Similar by meaning
Beta record. Based on the original catalogue summary; primary-source enrichment pending.