This document presents research on developing a multi-criteria decision analysis (MCDA) framework to enhance patient access to orphan drugs. The research aims to test an MCDA model based on criteria suggested by Hughes-Wilson et al. (2012) and assess the proposed criteria versus those in literature. The study developed an MCDA framework, selected study drugs, collected data on criteria like disease severity and effectiveness uncertainty, and scored the drugs. Results showed relationships between individual criteria and overall scores, and average annual costs versus total scores. The value of the MCDA is that it provides a rational, transparent assessment using multiple attributes to facilitate comparisons and support reimbursement decisions for orphan drugs with high unmet need.

1. The health economics of orphan drugs: Testing a
MCDA framework to enhance patient access
9th
European Patients’ Rights Day, Brussels, May 2015
Carina Schey, Unit of Pharmacoepidemiology and Pharmacoeconomics
PhD Researcher
Supervisor: Prof. Dr. Maarten Postma
Health Economist at Global Market Access Solutions

2. • Increased demand on healthcare results in cost containment
initiatives
• Current health technology appraisals might disregard several key
disease attributes
• Orphan drugs are unlikely to achieve “accepted” cost/ QALY
• Results in poor access to orphan drugs in many countries
• New methods may facilitate reimbursement decision making
which will enhance the access by patients to life-sustaining drugs
Background
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3. • To develop a multi-criteria decision analysis model based on
the criteria and framework suggested by Hughes-Wilson et
al, 2012 (1)
• To assess the:
• Proposed criteria versus criteria highlighted in a
literature review
• Outcomes of the MCDA
Aims
1. Hughes-Wilson W, et al. Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for
payers in Europe to take account of new rare disease treatments? Orphanet Journal of Rare Diseases. 2012, 7: 74.
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4. • Conducted a literature review
• Developed the MCDA framework
• Selection of study drugs
• Cost calculations
• Developed “scoring” system for different attributes
• Collected data for each criterion for each drug
Methods
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5. The proposed criteria
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• Disease severity
• Level of impact on disease
• Level of research undertaken
• Unmet need
• Manufacturing complexity
• Follow up measures
• Level of effectiveness uncertainty
• Disease rarity
• Uniqueness of the indication

6. Results: Individual criteria in relation to the overall
drug score
PAH – Pulmonary arterial hypertension MPS II & VI – Mucopolysaccharidosis II & VI
PNH – Paroxysmal nocturnal haemoglobinuria LGS – Lennox-Gastaut Syndrome
MDS – Myelodysplastic syndromes
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7. Results: Average annual cost in relation to the total
drug score
PAH – Pulmonary arterial hypertension MPS II & VI – Mucopolysaccharidosis II & VI
PNH – Paroxysmal nocturnal haemoglobinuria LGS – Lennox-Gastaut Syndrome
MDS – Myelodysplastic syndromes
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8. › Rational and transparent
› Includes a wide range of attributes
› Robust assessment
› Facilitates within-therapy and across-therapy comparisons
› Supports decision making
› Demonstrates true drug benefit and enhances patient access
where there is huge unmet need
Value of MCDAValue of MCDA
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9. › University of Groningen
Acknowledgements
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10. › University of Groningen
Acknowledgements
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