Polish MCDA tool for orphan-drug pricing and reimbursement
This tool uses multiple-criteria decision analysis to evaluate orphan drugs for pricing and reimbursement decisions in Poland. It considers ten criteria beyond traditional health technology assessment, helping decision makers weigh clinical, economic, and social factors to determine whether an orphan drug should be funded.
At a glance
Use when
Evaluating orphan drugs for pricing and reimbursement in settings where traditional HTA may overlook broader value elements; when decision makers seek a more comprehensive, transparent appraisal framework.
Avoid when
When rapid decisions are needed without capacity for multi-criteria scoring; when there is insufficient evidence to populate all MCDA criteria; in systems with rigid, cost-effectiveness-dominated reimbursement rules that do not allow for value flexibility.
Inputs
Orphan drug-indication pairs, evidence on clinical effectiveness, safety, economic data, and patient/social impact; scoring inputs across ten MCDA criteria derived from literature and policy context.
Outputs
MCDA score for each drug-indication pair, classification as positive or negative reimbursement recommendation based on threshold (e.g., >50% of max score), comparison with HTA outcomes.
How it works
The Polish MCDA tool was developed through a four-step process: (1) systematic literature review to identify relevant MCDA criteria, (2) creation of a database of orphan drugs, (3) categorization of HTA recommendations and performance of MCDA appraisals, and (4) comparison of MCDA and HTA outcomes. The tool includes ten criteria and assigns scores to drug-indication pairs, with a positive outcome defined as exceeding 50% of the maximum score (base case), tested further at 25% and 75% thresholds in sensitivity analysis. The study validated the tool using 27 drug-indication pairs, revealing discrepancies between MCDA and standard HTA outcomes, indicating added value in capturing broader aspects of decision-making.
- HTA domains
- Clinical Effectiveness, Costs & Economic Evaluation, Patient and Social Aspects
- Assumptions
- The ten selected MCDA criteria adequately represent relevant decision-making factors in the Polish context; scoring is consistent and transparent; a threshold of >50% of maximum points indicates sufficient value for reimbursement.
- Strengths
- Incorporates broader value elements beyond traditional HTA, such as patient and social aspects,Transparent and structured decision framework,Validated using real-world HTA recommendations and orphan drug data,Includes sensitivity analysis on scoring thresholds,Highlights discrepancies between MCDA and conventional HTA, supporting policy reflection
- Limitations
- Criteria weighting and scoring may reflect expert judgment rather than stakeholder consensus,Limited economic data availability for some drug-indication pairs,Findings may not be generalizable to non-orphan drugs or other healthcare systems,Does not replace budget impact or affordability considerations
- Also known as
- Polish MCDA framework for orphan drugs, MCDA tool for orphan drug reimbursement in Poland
Questions this answers
- › How might MCDA change orphan drug reimbursement decisions in Poland compared to standard HTA?
- › What additional criteria beyond clinical and economic factors influence the value of orphan drugs?
- › Can MCDA uncover value in orphan drugs that traditional HTA processes may overlook?
- › How sensitive are MCDA outcomes to scoring thresholds?
- › Do drugs rejected by HTA receive more favorable appraisals under MCDA?
- › What is the impact of including broader decision criteria on reimbursement outcomes?
References & sources
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