Background The number of orphan drug approvals is currently increasing globally. This
creates a significant burden on payers and healthcare systems. This study aimed to
create a multi-criteria decision analysis (MCDA) tool for evaluating orphan drugs
within the United Arab Emirates (UAE). The intended result of the tool is to provide
evidence-based guidance to decision-makers in reimbursement and procurement decisions.
Methods We conducted a literature search and local expert interviews to identify relevant
preliminary criteria for the MCDA tool. Then we conducted a structured consensus-building
session for healthcare experts and decision-makers in the UAE to develop the Emirati
MCDA tool for orphan drugs. The experts voted for the criteria to be included in the
tool and their ranking according to importance, as well as the weight of each criterion
and its scoring function. To improve understanding and facilitate the voting process,
experts were provided with a brief illustration of similar tools conducted in other
countries before the voting sessions. Finally, the tool was developed in a Microsoft
Excel sheet (Microsoft Corporation, Redmond, Washington, United States), and it was
validated and tested based on real case studies, then it was fine-tuned accordingly
based on the experts’ discussions. The final tool was provided to the attendees to
guide their decisions in the reimbursement and procurement of orphan drugs. Results
The created tool provides a score for each analyzed orphan drug based on its value.
Ten criteria were included in the final MCDA tool. These were cost-effectiveness (25.1%
of the weight), magnitude of health gain (20.1%), availability of therapeutic alternative
(14.3%), disease severity (11%), budget impact (7.9%), disease rarity (5.6%), strength
of clinical evidence (5.6%), burden on households (4.5%), indication uniqueness (3.2%),
and patients’ age (2.6%). Conclusions Implementation of evidence-based healthcare
necessitates assessing the fair value of each health technology. Addressing the high
unmet medical needs and improving healthcare for patients with rare diseases are priorities
within the UAE. The created Emirates MCDA tool for orphan drugs has the potential
to help decision-makers implement value-based and evidence-based reimbursement decisions
for orphan drugs.
