Waiel Al Naeem

Hemophilia A management in the United Arab Emirates (UAE) is still largely driven by treatment acquisition costs. This may limit access to innovative therapies such as emicizumab despite their broader clinical value. This paper aimed to identify barriers to value-based reimbursement and develop policy recommendations to strengthen health technology assessment (HTA) for hemophilia A in the UAE. An evidence-informed multi-stakeholder roundtable was held during the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) UAE Chapter 2026 Conference. The discussion was informed by published evidence, regional clinical guidance, and expert perspectives. The roundtable discussion was transcribed and summarized using descriptive qualitative analysis, and the manuscript synthesizes the main policy themes identified by the participants. Four priority areas emerged: adopting value-based assessment beyond acquisition cost, strengthening the use of evidence in reimbursement decisions, establishing national hemophilia registries with standardized data collection, and improving timely access to innovative therapies for high-risk patients. Overall, participants agreed that stronger HTA processes and better evidence generation are needed to support reimbursement decisions. Implementing these recommendations could improve access to innovative therapies and support more consistent, patient-centered, and sustainable hemophilia care in the UAE.

Background In the evolving healthcare landscape of the United Arab Emirates (UAE), establishing cost-effectiveness thresholds (CETs) is pivotal to informing decision-makers about the value of health technologies. Objective This study aimed to establish CETs for the UAE that harmonise with international standards while reflecting the nation’s unique healthcare needs and economic context. Setting United Arab Emirates. Methods A multitiered methodology was employed, involving a literature review, a panel of national experts and workshops with key stakeholders, including healthcare providers, government health departments and healthcare payers. The panel and workshops were integral in assessing global CET practices and their applicability to the UAE providing a preliminary framework for CET in the UAE. Structured voting sessions were then conducted allowing voting on crucial aspects of CET to determine the baseline threshold, multipliers for severity, rarity and health gain, and methodologies for quantifying disease severity. Results CETs were linked to the economic status of the UAE, with a baseline threshold of 0.75 times the gross domestic product per capita for one quality-adjusted life year gained. A multiplier system was introduced to reflect societal views on disease severity, disease rarity and the relative health benefit of health technologies. Based on the voting results, disease rarity was deemed the most crucial factor, receiving a maximum multiplier of 3X, while severity and health gain were assigned a maximum of 2X. The multiplier values for both disease severity and relative health gain would be determined on a continuous scale. The proportional or relative shortfall method would be used to assess disease severity. Conclusions The proposed CET framework for the UAE will be dependent on local generation of cost-effectiveness evidence. Periodic review of CETs based on initial experiences ensures the responsiveness of policymakers to the changing healthcare and economic environment.