Introduction High pricing of orphan drugs often restricts patient access. As orphan medicines are usually not cost-effective according to traditional thresholds, countries may choose not to consider the cost-effectiveness criterion in pricing and reimbursement decisions or may adopt higher thresholds for orphan drugs. The objective of this research was to develop a multi-criteria decision analysis (MCDA) framework to serve as a multiplier for determining a differential cost-effectiveness threshold (CET) for orphan drugs in Egypt. Methods A scoping review was conducted to identify criteria used for the evaluation of orphan drugs. This was followed by a policy workshop with the representation of the relevant governmental bodies to develop the MCDA tool by ranking and weighing the final list of criteria. The MCDA score is then converted to a CET multiplier for each orphan drug. Results A broad list of criteria was identified through the literature and was used as a starting point to create the tool. Based on participants’ voting, six criteria were included in the final MCDA tool: “disease severity” criterion was on top of the list with 38.6% weight, followed by “rarity” (24.2%), “budget impact” (16.1%), “robustness of the data” (10.2%), age of patients” (6.4%) and “societal impact” (4.6%). Conclusion Using multiple cost-effectiveness thresholds for common technologies and orphan drugs is increasingly applied in many countries based on different criteria. An MCDA tool was created to calculate the multiplier for orphan drugs for a higher threshold. The tool will improve the evidence, transparency and reproducibility of pricing and reimbursement decisions for orphan drugs.
Fasseeh et al. (Sun,) studied this question.