To collect relevant literature, structured searches were conducted across electronic databases, including PubMed, Scopus, Web of Science, and Google Scholar. Additional grey literature was sourced from official GCC government portals, MoH publications, national vision strategies (e.g., Saudi Vision 2030, UAE Health Sector Transformation Agenda). The reports from global health organizations such as the World Health Organization (WHO), OECD, and World Bank were also included.

To assess the landscape and preparedness for implementing VBH across GCC countries, particularly in the context of specialty pharmaceuticals, a qualitative focus group discussion was convened. This session, held in the UAE on April 11–12, 2025, engaged 24 key opinion leaders from the GCC countries. Figure 1 gives a countrywide distribution of the experts. The group included representatives from the pharmaceutical and healthcare sectors, clinical practitioners, policy advisors, and officials from government health agencies. Experts from different GCC nations were engaged. Their identification and selection were aimed at ensuring broad regional and inter-disciplinary representation. The panel was designed to reflect balanced representation across geography (6 GCC countries), professional backgrounds (clinical, academic, regulatory, and policy), and institutional affiliations (government, hospital, and academic). This minimized the risk of dominance by any single discipline or country and strengthened the representativeness of the consensus process.

Experts were selected based on the following inclusion criteria:

To minimize potential bias and ensure methodological rigor, the experts also declared potential conflicts of interest prior to participation; no material conflicts were identified.

The KSA has made notable progress under its Vision 2030 health transformation agenda, which includes key reforms such as digital health integration and a centralized procurement strategy (14, 18, 19). Oncology is a national priority under the cancer control strategy. However, policies for rare disease management remain underdeveloped and inconsistently applied (20, 21).

The vision outlines a comprehensive health reform agenda focused on preventive care, digital health, and emergency preparedness, public health campaigns promoting nutrition, physical activity, and smoking cessation, alongside school- and community-based programs. This aims to reduce the long-term burden of NCDs, offering a replicable model for other GCC countries (22, 23). The integration of digital tools such as electronic health records (EHRs), telemedicine, and artificial intelligence (AI) diagnostics has improved system efficiency and access to specialist care (24, 25). A review of Saudi MOH data showed rapid expansion of telemedicine during COVID-19, with 12 mobile apps (three launched during the pandemic) supporting millions of virtual consultations. “Sehhaty” was central, while “Mawid,” “Seha,” and the 937 hotline services saw dramatic increases in users and service volumes, reflecting a nationwide shift to digital healthcare (24).

Among GCC countries, KSA bore the largest share with 45% of NCDs related deaths, 49% of years of life lost, and 60% of economic losses (16). Therefore, KSA’s collaboration with the WHO to strengthen health emergency infrastructure provides a strategic framework for enhancing regional resilience (26).

The UAE’s approach to VBH emphasizes outcome-oriented and patient-centered care, underpinned by effective communication and technological integration. These reforms aim to improve clinical outcomes, enhance patient engagement, and reduce inefficiencies in healthcare delivery. While disparities in quality and care coordination persist, policymakers have acknowledged these challenges. They have demonstrated a strong commitment to embedding VBH principles into the national health strategy (19, 25).

The Department of Health has introduced an HTA roadmap to support universal health coverage. It focuses on making healthcare decisions more evidence-based and cost-effective by involving all stakeholders, including policymakers, providers, patients, and industry. The plan includes building local expertise, establishing governance, and promoting transparency through a phased five-year implementation. This initiative aims to create a more sustainable and inclusive healthcare system in the emirate (27).

Digital transformation is pivotal to advancing cancer care in the UAE, with e-health platforms, digital registries, and tele-oncology enabling earlier detection, better coordination, and more equitable access. Strengthening digital infrastructure and workforce training will be essential to deliver high-quality, affordable, and patient-centered cancer services. The UAE National Cancer Registry (UAE-NCR) was established to systematically collect, analyze, and report cancer incidence and mortality data across the country. The registry plays a crucial role in shaping national cancer control strategies, enabling early detection, screening programs, and resource allocation. It also highlights disparities in cancer incidence between UAE nationals and expatriates, and between genders (28).

Qatar demonstrates strong institutional readiness for VBH, owing to a highly centralized healthcare system and advanced digital infrastructure. The national cancer strategy supports robust outcome monitoring. However, care for rare diseases remains fragmented across the public and private sectors, limiting cohesive policy implementation (29). Qatar’s national health strategy focused on delivering person-centered care, improving healthcare outcomes, and ensuring the sustainability of the health system (22). The strategy promoted the integration of services, prioritization of prevention, and enhancement of care quality and efficiency, principles closely aligned with VBH. It also underscores the importance of data-driven decision-making and the use of health technology to improve patient outcomes and resource allocation. These initiatives reflect Qatar’s commitment to shifting from volume to value in healthcare delivery. Qatar’s national cancer framework builds on the earlier national cancer strategy to enhance cancer care by aligning services with patients’ needs. It outlines nine domains with defined activities and success metrics to guide implementation and evaluate outcomes (30).

These countries are in the early phases of VBH adoption, with policy discussions gaining momentum but limited operational structures currently in place. Their ongoing reforms primarily target improvements in quality of care and patient value, although standardized frameworks and outcome-based reimbursement models are still nascent (31).

Kuwait’s Vision 2035 “New Kuwait,” explicitly prioritizes improving healthcare quality, major hospital expansion, and digital transformation, giving VBH strong strategic alignment. The government has allocated a substantial healthcare budget and launched infrastructure expansion projects (new hospitals, partnerships with international centers) (32).

Oman Vision 2040 places health as a national priority, and the government is advancing initiatives to attract private investment and strengthen health sector capability helpful for mixed public/private VBH models (33).

Bahrain’s health sector is compact and well-regulated through the National Health Regulatory Authority (NHRA). It has piloted insurance reforms and has a mixed provider landscape, making it a strong testbed for value-based care. The National Health Plan and Economic Vision 2030 stress quality, sustainability, and data-driven outcomes, aligning with VBH principles. It has piloted insurance reforms and has a mixed provider landscape, making it a strong testbed for value-based care (34).

Oncology represents a critical and growing burden across GCC countries. It is driven by increasing incidence rates, rising treatment complexity, and the high costs associated with advanced therapeutics. Cancer is now among the leading causes of morbidity and mortality in the region. This burden is compounded by demographic shifts such as ageing populations and lifestyle-related risk factors, including obesity and tobacco use (40). The treatment landscape has evolved with the emergence of novel interventions, particularly immunotherapies, targeted therapies, and gene-modified cell therapies. This enables more precise and effective options for cancer care. However, these innovations come at substantial costs, posing financial challenges to health systems striving to ensure equitable and timely access. The introduction of VBH and MEA, such as risk sharing and outcome-based reimbursement models, offers a pathway for balancing clinical innovation with financial sustainability (41–44). All GCC countries bear a substantial economic burden due to the provision of free cancer care, underscoring the need for a robust framework to assess, register, and approve novel oncology treatments. Accelerating patient access, integrating patient-reported outcomes, and implementing value-based cancer care are critical to ensuring sustainability and improved health outcomes (17).

However, there are significant barriers to its implementation. This includes fragmented health infrastructure, variability in outcome measurement standards, limited local real-world data, and challenges in integrating VBH frameworks within existing procurement and reimbursement mechanisms. Moreover, programs such as compassionate use, though vital for patients with rare or advanced cancers, face ethical, regulatory, and logistical complexities that hinder broader adoption (45–48).

The rising prevalence of autoimmune conditions such as rheumatoid arthritis, Crohn’s disease, and psoriasis presents a growing challenge for health systems in the GCC, particularly given the chronic nature and high treatment costs of immunologic therapies (49, 50). Targeted treatments like Janus Kinase (JAK) and interleukin inhibitors offer improved efficacy with reduced systemic toxicity but come at a significant economic burden. Biosimilars provide a viable, cost-effective alternative to originator biologics, delivering comparable clinical outcomes and improving key health economic indicators (51). The adoption of biosimilars is increasing, driven by multiple factors such as regulatory reforms that streamline approval processes and the lowering of development costs, which encourage more market entry. The authors note that despite initial challenges, the case for broader biosimilar use is strengthening, and a significant market shift is imminent as prices continue to drop and market expansion occurs (52).

To facilitate VBH implementation in immunology, it is essential to align reimbursement policies with clinical guidelines, engage stakeholders early, communicate benefits transparently, and support switching policies with regulatory incentives (53). These measures are crucial to ensuring that innovation translates into broad and equitable patient access, supporting both fiscal sustainability and improved health outcomes across the region (23, 54).

Neurological disorders are a leading cause of disability and the second leading cause of death globally, with rising prevalence in the GCC (55). Diseases like Alzheimer’s disease (AD) and multiple sclerosis (MS) strain healthcare systems, as current therapies offer limited benefits or high-cost disease modification (56–59). Although specialty drugs, including monoclonal antibodies and neuroprotective agents, show promise, adoption is hindered by low investment, regulatory barriers, and limited reimbursement (60–63). Advancing VBH in neurology will require targeted policies, RWE generation, affordability programs. It will also depend on payer engagement to align innovation with outcomes (61–64).

The burden of rare diseases in the GCC countries is significant. It is largely driven by sociocultural factors such as high rates of consanguinity and large family sizes. This contributes to the elevated prevalence of genetic disorders (65). A retrospective case–control study on 112 pregnant women with sickle cell disease (SCD) was conducted in Bahrain. The findings reveal significantly increased maternal and fetal risks associated with the condition (66). Public awareness of SCD in Bahrain was widespread, but critical gaps remained in understanding its genetic inheritance and prevalence (67). In Qatar, whole genome studies have identified founder mutations specific to certain ancestries, underlining the value of population-specific genomic data (68). Similarly, the UAE reports over 1,365 unique gene variants, many likely pathogenic, underscoring the genetic complexity within its population (69). Oman’s carrier rate for SCD and thalassemia combined is around 6% (70). Collectively, these data reflect a high regional burden of rare diseases requiring long-term, resource-intensive care.

Despite increasing recognition of rare diseases, multiple challenges hinder the effective implementation of VBH in rare diseases. Diagnostic delays, limited access to genetic testing, and fragmented care pathways compromise early identification and management (71). The lack of centralized registries and the minimal availability of RWD impede outcome tracking and value-based reimbursement planning. Furthermore, affordability remains a concern due to the high cost of orphan drugs, which often lack reimbursement clarity and depend on ad hoc financing mechanisms (68, 72–74). Although the SFDA has included incentives like priority review and fee waivers, the lack of long-term outcome evaluation tools remains a barrier to full VBH integration (75).

Gene therapy offers a novel and potentially long-lasting solution for genetic disorders by directly addressing the underlying genetic defects. As a one-time treatment, it aims to repair or replace faulty genes, with the potential to restore normal function and alter the course of disease (76, 77). FDA-approved cell and gene therapies vary in cost-effectiveness, with some offering value despite high initial costs (78). However, progress in rare disease applications is limited by regulatory, financial, and market barriers. This underscores the need for sustainable models that reflect disease burden, existing treatments, and economic feasibility (79, 80).

While countries like the KSA and UAE have made notable progress, bureaucratic inertia and budgetary constraints continue to hinder public payer adoption (85). Despite universal healthcare coverage, resource allocation disparities, especially between citizens and expatriates, persist (2). Tools like MCDA offer a pathway to ethical and transparent decision-making (86, 87). Integrating ethics review boards and patient advocacy will further strengthen policy credibility and inclusiveness (87).

HTA infrastructure remains nascent in many GCC countries, with key limitations including insufficient local epidemiological and outcomes data, low HEOR publication rates, and limited professional capacity (40). These barriers delay VBH-aligned reimbursement decisions. Addressing these gaps will require investment in local research, cross-sector collaboration, and workforce development. Moreover, traditional pricing models often undervalue treatments in rare diseases and oncology due to unfavorable cost-effectiveness thresholds (88). Innovative approaches such as risk-sharing agreements (RSAs), value-based pricing (VBP), and patient assistance programs (PAPs) offer pragmatic alternatives (42, 89, 90). The SFDA has made strides by offering a structured orphan drug designation pathway, including incentives like regulatory support and fee waivers (26). Standardizing such frameworks regionally will be key to sustainable access.

RWD and RWE are critical enablers of VBH, especially in areas where clinical trial data are limited, such as rare diseases and oncology (25, 91–93). RWE supports regulatory compliance, post-marketing surveillance, and value-based pricing models. However, adoption in the GCC is challenged by infrastructural gaps, data privacy concerns, regulatory ambiguity, and limited technical expertise. Building effective RWD/RWE frameworks will require robust digital health systems, data governance policies, and stakeholder collaboration. Integrating local registries, especially for rare diseases, and capitalizing on the existing digital capabilities in the KSA and UAE will be essential to generating actionable evidence (25).

Digital infrastructure is pivotal to scaling VBH in the GCC. Advanced systems in the KSA, UAE, and Qatar support outcome tracking, bundled payment models, and integration of patient-reported outcomes (25, 94). In oncology and rare diseases, digital platforms facilitate registry development, real-time monitoring, and streamlined access to clinical trials (95). They also enable remote care delivery and administrative automation, enhancing both system efficiency and equity of access.

MEAs are structured arrangements between pharmaceutical companies and payers or regulators that facilitate access to high-cost, innovative therapies while managing clinical and financial uncertainties. These agreements are increasingly relevant in GCC as countries adopt VBH models. MEAs can be classified as performance-based, linking reimbursements to specific clinical outcomes. They can also be financial-based, incorporating mechanisms such as price discounts or caps to mitigate budgetary impact (96, 97). Despite their promise, implementation faces several challenges in the GCC, including limited local outcome data systems, and a shortage of pharmacoeconomic expertise. Additional barriers include the absence of standardized frameworks, and cultural resistance to alternative reimbursement models. Figure 5 shows the four phases of MEA implementation.

MCDA is gaining traction in the GCC as a structured tool to support transparent, value-based healthcare decisions. It incorporates factors beyond cost-effectiveness, including clinical impact, equity, and patient preferences (98). In the KSA, researchers demonstrated the feasibility of implementing MCDA to establish a national evaluation framework, and stakeholders endorsed its role in enhancing transparency (99). Traditional HTA, while essential, may inadequately capture the nuances of rare diseases and oncology. In these areas, small populations and uncertain outcomes complicate standard evaluation models (89). In response, UAE researchers developed a tailored MCDA tool for orphan drugs, incorporating 10 weighted criteria to guide reimbursement decisions. These criteria include patient age, indication uniqueness, household burden, clinical evidence strength, disease rarity, budget impact, disease severity, therapeutic alternatives, health gain magnitude, and cost-effectiveness (100). Similarly, to address rising pharmaceutical costs and enhance transparency in purchasing decisions, Kuwait developed an MCDA tool. It was co-created by a diverse group of stakeholders, including pharmacists, regulators, and academics, through a structured seven-step process. The initiative aimed to support more consistent, value-based decisions, ultimately improving the sustainability of Kuwait’s healthcare system (101).

Key considerations for adapting MCDA frameworks in the region include addressing limited long-term clinical data, small patient populations, ethical dilemmas, and the need for stakeholder-inclusive decision-making (102) (Table 1).

Early diagnosis plays a pivotal role in improving outcomes for rare diseases and cancers. GCC countries are investing in genomic programs such as the Saudi Human Genome Program, UAE Genomics Council initiatives, and the Qatar Genome Program (103–105). Additionally, expanded newborn screening and national cancer screening initiatives across GCC nations support timely detection (103, 105). However, limitations in HTA frameworks and data infrastructure hinder the integration of these tools into reimbursement systems (106).

RSAs provide a pragmatic solution to the uncertainties in real-world effectiveness and cost of high-priced therapies, particularly in oncology and rare diseases. They include performance-based models connected to clinical outcomes and financial-based models involving discounts or refunds (97). The KSA is actively adopting RSAs as part of its Vision 2030 health reform, offering a mechanism to balance patient access with fiscal sustainability (107). By adapting global models, the KSA can establish robust RSA frameworks tailored to local needs and regulatory conditions.

In the initial phase, efforts need to be directed toward expanding MEAs in the KSA and across the GCC to improve access to high-cost innovative therapies. Clearly delineated roles across regulatory bodies and procurement entities are essential; the consumer health informatics (CHI) can drive evidence generation, the SFDA may oversee regulatory compliance. The pilot implementation of MCDA frameworks, particularly for high-cost oncology and rare disease medications, will enable transparent and value-oriented pricing mechanisms. Concurrently, integrating PROMs and establishing institutional RWE registries can facilitate patient-centered care and digital health transformation.

Over the medium term, the focus shifts to harmonizing HTA processes across the GCC to enable consistent, evidence-based decision-making. Establishing a regional RWE consortium aims to address current gaps in data quality, governance, and regulatory harmonization, while fostering inter-country collaboration. Additionally, value-based insurance models can be piloted for chronic, high-burden therapeutic areas such as autoimmune and neurodegenerative conditions.

Long-term strategies include developing digital ecosystems capable of supporting outcome-based payment models and real-time tracking of bundled healthcare services. A GCC-wide orphan disease access fund can be instituted as part of long-term initiatives to mitigate inequities in access to ultra-rare therapies. The creation of a unified regional platform to negotiate value-based pricing contracts with pharmaceutical manufacturers is recommended, supporting scale and economic sustainability.

In parallel, several cross-cutting strategies may be implemented to enhance the uptake and equitable access of specialty therapies throughout the GCC region (60, 63, 64, 128, 129):