OBJECTIVES: Despite the recent success in orphan drug development, limited patient access still represents a major challenge for patients with rare diseases. In most instances, orphan drugs fail to prove cost-effectiveness when assessed against conventional cost-effectiveness thresholds (CETs) and are therefore denied reimbursement. The CET framework of Egypt allowed the introduction of a differential threshold to assess orphan drugs, where its value is based on a multiplier of the CET. This study aims to develop a multi-criteria decision analysis (MCDA) tool to evaluate the reimbursement of orphan drugs based on several criteria in Egypt to define which orphan drugs deserve preferential reimbursement.

METHODS: A scoping review was conducted to identify the criteria used to grant a drug an orphan status, as well as the criteria used to evaluate orphan drugs using different MCDA tools. This was followed by a workshop attended by stakeholders representing several governmental bodies to rank, weigh, and assign scoring functions for the identified criteria. The created MCDA tool provides a score for each drug that can be used as a proxy for the CET framework multiplier.

RESULTS: A broad list of criteria were identified through the literature and refined to create the MCDA tool. According to participants’ votes, six criteria were included in the final tool: “disease severity” criterion was on top of the criteria list with a weight of 38.6%, 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%).

CONCLUSIONS: An MCDA tool was created to assess the eligibility of orphan drugs for a higher cost-effectiveness threshold compared to that of common health technologies in Egypt. The developed MCDA tool offers a fair chance for orphan drugs to be reimbursed according to acceptable criteria, therefore increasing patient access to treatment.