Once regarded as a strategic afterthought by international biopharma, the Middle East has undergone a profound transformation in recent years, emerging as an early adopter of next-generation therapies for rare diseases. Regional regulatory authorities are accelerating approval timelines, reflecting an aggressive institutional ambition to secure speedy access to cutting-edge medical science.
“In some cases, regulatory authorities are managing approvals timelines closely aligned to the US FDA, reflecting both the pace of reform and the ambition of governments to ensure their populations gain access to the very best and brightest medical innovations without delay,” observes Mayada Al-Ahmad, general manager for the GCC at BMS. “We’re already treating Saudi Arabia and the UAE among our phase one early launch markets,” she confides.
“From an industry standpoint, innovation is reaching the region much earlier – in some cases close to global benchmarks – which signals a broader recognition of the Gulf Countries as a strategic market rather than a secondary one,” confirms Sherif Basha, head of rare diseases for the MENA at Versalya Pharma, an affiliate of Italfarmaco Group. “Looking back over the past two decades, the change has been substantial. Rare diseases were once addressed almost exclusively through distributor-led models focused on basic supply, often several years after approvals in the US or Europe. That approach has now shifted decisively, with regional business now contributing strongly to global performance,” he observes.
Khaled Elrefae, general manager for the MENA at Recordati Rare Diseases, reinforces this sentiment, noting that commercial momentum has accelerated rapidly across high-value Gulf markets. “We have been aggressively expanding our portfolio of innovative hematology-oncology, endocrinology, and metabolic therapies,” he attests. “Compared to where we were merely a year ago, we have already achieved significant traction across key markets such as Saudi Arabia, Kuwait, and Qatar,” he reveals.
Evolving Perceptions
The forces driving such a turnaround are manifold. “From a rare disease perspective, the Middle East represents a significant market opportunity. The region boasts a population of more than half a billion, and there is a uniquely high incidence of rare disease due to ethnic, demographic and socio-culture factors,” explains Elrefae. “This concentration of genetic traits presents a vast pool of unmet medical need that global life science firms can no longer overlook.”
Underpinning this change of perceptions is the enhanced capital and infrastructural maturity of many Middle Eastern health systems, alongside granular demographic insights unlocked by nationwide genome projects. For these structural advancements have turned a historically opaque market into a transparent, data-driven environment capable of absorbing complex therapeutic modalities. “Biotechnology companies have woken up to the relevance of the region because local data increasingly makes the unmet need visible,” reflects Adeeb Al Attar, managing director at Genpharm, a Dubai-based specialty life science partner for global biotechs. “Moreover, the more affluent countries, notably the Gulf petrostates, have attained levels of health system maturity where this marketplace can finally be tapped.”
Al Attar recalls that as recently as 2016, regional therapeutic options were limited mostly to conventional enzyme replacements. “At that stage, when gene therapies were only just beginning to emerge, there was little practical reason to pursue deep genetic characterisation,” he reflects. “However, as medical science advanced, healthcare systems across the region began to respond in tandem, setting the stage for global biopharma to re-evaluate the merits of direct market entry.”
This has been matched with massive state-backed capital deployment. Over the past decade, national genomics initiatives have scaled rapidly. Programs like the Saudi Human Genome Program, the Emirati Genome Programme, and Qatar’s sequencing efforts at Sidra Medicine have built comprehensive, population-level datasets. These platforms providing the robust infrastructure required for precise diagnostics and targeted clinical interventions.
Tailored Market Entry
Commercial success, however, remains contingent on recognizing that the region is not a homogenous landscape. Significant disparities exist in health system maturity, screening coverage, and institutional capacity, necessitating highly granular engagements.
Recordati, for example, has rejected generalised market entry strategies, opting instead to concentrate resources on high-priority jurisdictions to secure operational speed and precision. “We ensured that we had the right focus and resources in our priority markets, particularly Saudi Arabia and the Gulf,” reasons Elrefae. “This has allowed us to move faster, execute more effectively across our portfolio, and provided the basis for our strong operational performance.”
Ultimately, navigating the fragmented regulatory and reimbursement pathways of the MENA region requires an intricate understanding of localized legislative tools, from orphan drug status to international reliance mechanisms. “There are rich pickings to be had if you approach the region intelligently, but the practical implication is that there is no single route to market,” insists Al Attar. “Strategies need to be adapted country by country, using tools such as orphan designation, expedited pathways, or reliance mechanisms. Our role is to help biotechs navigate that complexity and build a compliant, realistic approach from the outset.”
At the same time, the manner of expected engagement from industry has evolved too: shifting the paradigm from transactional vendor to strategic public health ally. “The commercial mindset has matured,” perceives Sherif Basha. “The assumption that access was simply a matter of price in wealthy markets has been replaced by a partnership-driven approach centred on structured dialogue with governments and shared objectives. National priorities around genomics place growing emphasis on local data generation, clinical research, and real-world evidence. As a result, market entrants are increasingly expected to contribute through research collaborations, registry development, and the use of advanced analytics and AI to improve diagnosis.”
Saudi Arabia and the UAE exemplify this collaborative shift. Regulatory bodies have transitioned from bureaucratic obstacles into proactive partners. “The Saudi FDA now positions itself less as a gatekeeper and more as a collaborative partner, open to early dialogue when patient burden is clear,” affirms Al Attar.
To navigate this newly collaborative ecosystem, drug developers have been systematically restructuring their regional corporate footprints to establish direct, localized entities that allow them to deepen relationships with sovereign healthcare decision-makers.
“From a regional perspective, PTC Therapeutics was already present in the MENA through established partnerships,” confides Mohamed Abu Shawish’s counterpart, Zeina Sfeir Lahoud. “The opening of the regional office in Dubai Science Park in 2025, however, marked an important step towards a more direct and structured presence. Today, we operate a hybrid model, continuing to work with partners for some products while managing newer therapies more directly. This approach allows us to deepen engagement with local stakeholders while retaining flexibility.”
This trend toward establishing localized corporate hubs is further evidenced by recent expansions into the Saudi capital. Companies like Recordati have recognized that physical proximity is mandatory to support the complex, data-driven partnership models required for launching next-generation therapeutics. “Maintaining a strong physical in-country presence on the ground is essential to facilitate the more complex partnerships that will underpin our future product launches,” insists Khaled Elrefae.
Vertex has similarly reinforced its strategic commitment by beefing up its in-Kingdom competencies. This physical immersion allows the enterprise to engage intimately across the entire value chain fully capturing the momentum of a region moving at an unprecedented pace. “We made a conscious decision to establish our regional HQ in Riyadh precisely because physical proximity to stakeholders is essential to the kind of collaboration we are pursuing,” attests Hisham Hagar. “Regulatory approvals, reimbursement discussions, clinical development partnerships – all of these benefit from being conducted in close, sustained engagement rather than from a distance.”
Confronting the Cost of Advanced Interventions
Nonetheless the economic realities of cell and gene therapies have been creating profound affordability barriers, even for the most cash-flush Gulf economies, forcing a wholesale reassessment of traditional reimbursement paradigms across the region.
“The most pressing challenge, and one that transcends any single country, is the cost of advanced therapies,” notes Fatma Al Jasmi, dean of the College of Medicine & Health Science at UAE University (UAEU). “As a clinician, I will always advocate for my patients to access the treatments they need, but the economic realities of gene therapies and other advanced interventions create genuine barriers. Finding ways to develop treatments that are effective, accessible, and affordable is a problem the global rare disease community must address together,” she warns.
Dimitris Moulavasilis, group CEO at the healthcare tech group, M42 very much concurs. “Healthcare systems need to move credibly from episodic treatment to prevention-first models,” he argues. “In most markets, the Gulf not excepted, health expenditure continues to outpace economic growth, while medical science is becoming more complex and costly. Disease categories that were once treated uniformly are now molecularly stratified into multiple subtypes, each requiring targeted therapeutic strategies. Systems must provide access to these advances, yet the existing cost trajectory is not sustainable,” he explains.
For Ali Taher, professor of medicine at the American University of Beirut, the only feasible way forward is to revert to precision medicine. “Rather than applying a high-cost therapy universally when only a proportion of patients will respond optimally, we should be identifying the clinical and molecular variables that predict response and directing resources accordingly,” he opines. “In other words, we must match the right intervention to the right patient, ensuring that those who do not require the most expensive option receive excellent, appropriately targeted care instead. A therapy that benefits 60 percent of recipients while the remaining 40 percent receive it unnecessarily, at substantial cost, is simply not a model that serves public health,” he insists.
Pivoting Towards Prevention
Driven by this economic imperative, sovereign Gulf states have been aggressively embedding preventative healthcare, precision medicine, and advanced molecular diagnostics into their overarching national strategies. Blueprints such as Saudi Vision 2030 and Oman’s national health initiatives explicitly position early identification as a financial defence mechanism, recognizing that shortening the time to diagnosis significantly reduces downstream catastrophic care costs. “Substantial budgets and strategic frameworks now entrench prevention and early diagnosis as fundamental priorities,” notes Hussein Abhari, head of the Middle East and North Africa at QIAGEN. “This creates a transformative opportunity to shift from late diagnosis to early identification.”
Qatar offers a compelling template for this operational shift. At Sidra Medicine, the integration of cutting-edge genomics into frontline paediatrics has dismantled the protracted “diagnostic odyssey” that historically plagued rare disease patients, substituting years of clinical uncertainty with immediate, targeted interventions. “We now utilise whole genome sequencing as a first-tier diagnostic test, which directly contributes to diagnosing more patients at the critical right time,” explains Tawfeg Ben-Omran, Sidra’s Division Chief of Genetics and Genomic Medicine. “Administering early treatment decreases hospitalisations, reduces intensive care stays, and fundamentally relieves families. Now, particularly in acute or intensive care settings, we can obtain whole genome results within one week, considerably alleviating the burden on the healthcare system and society.”
This democratization of advanced diagnostics is mirrored in Saudi Arabia. Once confined to specialized elite bastions like the King Abdullah International Medical Research Center (KAIMRC) and King Faisal Specialist Hospital & Research Centre (KFSH&RC), screening capabilities have been systematically scaled nationwide under Vision 2030 guidelines. For global specialty biopharma, this expanding infrastructure is a critical commercial enabler that ensures patients are identified and funnelled into optimal care pathways before irreversible disease progression occurs. “Our treatments are most effective when patients are diagnosed early and referred to the right specialists,” reveals Mohamed Abu Shawish, cluster general manager for the GCC at Kyowa Kirin. “We see genomics and newborn screening as important enablers of timely diagnosis, not just from a technology ownership standpoint, but from a patient-journey standpoint and health system sustainability perspective too.”
Early Access Frameworks
Regulatory frameworks across the Middle East have progressed in concert with these commercial and diagnostic shifts. Where ambiguity once surrounded the registration of orphan drugs for small patient populations, national regulators, aided by newly formed HTA apparatuses, are increasingly offering structured pathways, including dedicated orphan designations and expedited review timelines.
“Where uncertainty once surrounded pathways for therapies addressing small patient populations, most Middle Eastern markets now offer clearer frameworks, including orphan designations and accelerated reviews,” notes Sherif Basha. “In practice, these pathways are significantly faster than the conventional route, and that speed reflects a deeper change in how rare diseases are now viewed by policymakers. Twenty years ago, rare conditions were often treated as marginal or theoretical. Today, when a rare disease is raised with local regulators, the discussion is structured and grounded in evidence. Authorities typically start by examining patient numbers, the quality of the clinical data, and the sources supporting prevalence estimates, before assessing whether there is a clear unmet need and a genuine gap in available treatment options.”
While terminology varies by jurisdiction, the underlying principle of regulatory acceleration remains consistent. In Saudi Arabia, the SFDA has institutionalized formal accelerated pathways – including Priority Review, Orphan Drug Designation, and the Breakthrough Medicines Programme – explicitly designed to truncate approval timelines for high-unmet-need therapies. Similarly, the UAE’s Emirates Drug Establishment operates a fast-track registration pathway for innovative and orphan medicines, governed by strictly defined review milestones. Furthermore, regulators are aggressively leveraging abridged or verification review mechanisms. By utilizing reliance models that honour prior approvals from trusted reference bodies like the US FDA or the EMA, regulators can fast-track critical therapeutics into the local market with unprecedented efficiency.
Recent high-profile approvals illustrate the real-world impact of these expedited frameworks. “Saudi Arabia demonstrated genuine leadership by being the first country globally to approve the first CRISPR-based therapy through the SFDA’s accelerated access pathway, facilitating and expediting patient access,” points out Vertex’s Hagar. “That regulatory speed reflects a seriousness of intent that we find enormously encouraging as a partner,” he exclaims.
Concurrently, the UAE’s rapid authorization and clinical administration of Novartis’s gene therapy for spinal muscular atrophy marked another landmark milestone, establishing the country as the second globally to authorize the treatment. “The SMA milestone is a clear example of how the UAE’s healthcare ecosystem functions when regulatory frameworks, operational capability, and partnership are aligned,” enthuses Mohamed Ezz Eldin, head of the GCC Cluster at Novartis. “In this case, accelerated review pathways enabled approval shortly after the US FDA, positioning the UAE as the second country globally to grant authorisation.”
Financial Risk Sharing
Balancing rapid patient access with long-term fiscal sustainability has meanwhile forced a comprehensive reassessment of traditional reimbursement paradigms. Local healthcare systems have been decisively transitioning away from classic fee-for-service models toward sophisticated value-based healthcare frameworks that reward quantifiable patient outcomes rather than the volume of clinical interventions.
“The conversation has clearly moved beyond price alone,” observes Diederik Kok, head of the GCC at Biogen. “Middle Eastern policymakers are increasingly looking at health technology assessment, pharmacoeconomic evidence, disease burden and long-term budget impact, which has elevated market access from a technical function to a central strategic pillar.”
Some countries have addressed structural budget pressure through dedicated rare disease funds, particularly for paediatric populations, recognising that rare conditions require a different financing logic. Others are employing managed entry agreement frameworks whereby outcomes are tracked once patients begin treatment, allowing reimbursement to be informed by real-world performance rather than international trial data alone. “This approach supports more accurate forecasting, better budget management, and more confident access decisions,” believes Kok.
“There is a growing recognition that innovation only fulfils its purpose when patients are able to access it,” believes Mohamed Ezz Eldin. While the cost of advanced therapies, including gene therapies, remains an important consideration, discussions in the region increasingly centre upon the longer-term value these treatments can deliver. “In some cases, these therapies have the potential to fundamentally change disease trajectories, and local payers are proving willing to look beyond upfront cost to consider the broader clinical and economic impact over time. In parallel, there is a clear eagerness, on the part of regional policymakers to explore more innovative access and financing models,” he concedes, noting that “Novartis is collaborating directly with authorities on approaches such as risk-sharing.”
Abu Dhabi’s Department of Health is perhaps emblematic of this newly enlightened approach, blending rigorous clinical oversight with an entrepreneurial posture to evaluate and finalize insurance reimbursement pathways within tight operational windows. “Our vision is to be intelligent and efficient – not simply to spend, but to spend where we will achieve demonstrable impact on population health,” details Dr Noura Khamis Al Ghaithi, Undersecretary at the Department of Health in Abu Dhabi.
“Submissions are reviewed within a maximum of ten to 14 days, with coding, reimbursement, and insurance coverage determined within that same window. Deploying, evidence-based methodologies, we are no classic regulator, but instead maintain a corporate mindset – agile, evidence-based, and increasingly AI-assisted – oriented around approving new technologies as rapidly as possible without compromising rigour,” she claims.
The emirate has also established concrete benchmarks to systematically transition to outcome-based health spending. “We’ve set a clear target to shift 50 percent of our payments to a value-based model,” Al Ghaithi declares, pointing that their fertility reimbursement programme provides an early example. “Centres are paid a base amount, with additional reimbursement tied to success rates, patient satisfaction, and the adoption of AI-enabled infrastructure. That model – rewarding outcomes rather than activity – is the direction of travel across the system,” she discloses.
Industry has taken note. “One key milestone for us was the 2024 memorandum of understanding with the Department of Health Abu Dhabi, focusing on advancing data utilisation and real-world evidence to support more informed clinical and policy decisions,” notes Mohamed ElShaarawy, general manager of Roche Pharmaceuticals in the UAE. “This type of partnership reflects a broader shift: moving from episodic access discussions to long-term value conversations grounded in evidence.”
Such financial evolution is mirrored in Saudi Arabia, where major public health institutions are intertwining clinical documentation with risk-sharing structures. By leveraging robust electronic health record (EHR) infrastructures to track real-world clinical endpoints, the Kingdom’s apex medical centres can seamlessly tie industry compensation directly to therapeutic efficacy. “Rather than paying the full list price for high-cost therapies upfront, we have structured reimbursement agreements with industry partners that are contingent on patients achieving defined clinical outcomes,” explains Abdulrazaq Al-Jazairi, Deputy Executive Director for Research & Innovation at King Faisal Specialist Hospital & Research Centre (KFSH&RC).
“We pay for results, not for prescriptions. This model is not only fiscally prudent – it is the only sustainable approach to deploying these therapies within a budget-constrained government healthcare system. Our electronic health record infrastructure makes it operationally feasible: capturing the clinical endpoints required to trigger or withhold reimbursement is entirely manageable within our existing systems,” he elaborates.
From Product Access to Ecosystem Readiness
While the region’s success in broadening access to advanced biotherapies for rare diseases is undeniable, industry insiders stress that the next frontier requires maturing ecosystem readiness.
“Execution requires a completely different mindset,” asserts Mayada Al-Ahmad of BMS. “You are not simply introducing a treatment; you are building the diagnostic and care infrastructure around it. This means mobilizing the broader ecosystem to close system gaps and strengthen pathways for earlier diagnosis and integrated disease management.”
To illustrate, Al-Ahmad points to BMS’ successful introduction of breakthrough therapies for obstructive hypertrophic cardiomyopathy in Saudi Arabia. The launch required extensive ecosystem engineering, including partnering with the National Heart Centre to redesign diagnostic pathways, develop registries, and coordinate care to ensure early patient identification.
Ultimately, the focus must shift to uniting these elements into a seamless, end-to-end system that links awareness, early diagnosis, referral, treatment, and outcome tracking. “Fostering this level of coordination, anchored by reliable data and real-world evidence, is absolutely critical,” Lahoud concludes. “It will enable the region’s transition toward locally driven clinical and research models, ultimately delivering top-tier care to one of the world’s most prominent rare disease communities.”
