Multi-criteria decision analysis (MCDA): testing a proposed MCDA framework for orphan drugs

Area of Science:

• Pharmacoeconomics
• Health Technology Assessment
• Rare Disease Drug Evaluation

Background:

• Traditional reimbursement models may not suit orphan drugs.
• The National Institute for Health and Clinical Excellence suggests broader criteria for orphan drug reimbursement.
• Orphan drugs present unique challenges in cost-effectiveness evaluations.

Purpose of the Study:

• To evaluate the Multi-Criteria Decision Analysis (MCDA) framework for orphan drugs.
• To compare MCDA aggregate scores with average annual drug costs.
• To establish MCDA's utility in assessing orphan drug value relative to price.

Main Methods:

• Developed an MCDA framework using nine criteria for orphan drug evaluation.
• Applied a numerical scoring system (1-3) for each criterion.
• Analyzed correlations between average annual drug costs and MCDA scores, testing different attribute weightings and including drug cost.

Main Results:

• A strong correlation (R²=0.79) was found between MCDA scores and average annual cost per patient.
• The study visually represented the relationship between drug scores and costs.
• Analysis explored the impact of including drug cost within the aggregate index.

Conclusions:

• The MCDA framework provides valuable insights into orphan drug valuation.
• A significant relationship exists between MCDA scores and average annual drug costs.
• Despite limitations, MCDA is a useful tool for assessing orphan drug value and pricing.