Long regarded as a clinical trials backwater, the Middle East has experienced a tremendous surge in clinical research over the past five years. This boom is fuelled by a global demand for representational equity and an urgent need to correct a stark imbalance, as Middle Eastern populations currently account for just 1.24 percent of global genomic datasets.
“Global regulators and sponsors are increasingly prioritizing broader ethnic representation in clinical trials, moving to correct long-standing historical imbalances. Because this region has been so deeply underrepresented in the past, drug developers targeting a global marketplace face more pressure than ever to integrate a Middle Eastern cohort into their multi-country studies,” reckons Albarah Elkhani, COO of Integrated Health Solutions at M42.
This shift is also emblematic of how clinical research has suddenly become strategically integral to the national visions of wealthy Gulf petrostates like Saudi Arabia and the United Arab Emirates. “Historically, Middle Eastern nations were passive recipients of clinical research conducted abroad rather than active creators of medical innovation. However, the COVID-19 pandemic served as a major catalyst. It compelled countries with the financial resources to recognize the critical importance of embedding life sciences innovation domestically,” opines Alaa Assem, Managing Director for EMEA at CTI Clinical Trial & Consulting Services, an independently owned clinical research organization (CRO) with a long track record of operating in the region.
He flags up Saudi Arabia, with its Vision 2030 and National Biotechnology Strategy, and the UAE with its newly formed national regulatory body, the Emirates Drug Establishment, as exemplifying this transformation. “We are now seeing a steady rise in Phase I, first-in-human, and gene therapy studies across the region – milestones that were completely unimaginable barely a decade ago,” he insists.
The sheer scale of these long-term ambitions sets the GCC countries apart as they position themselves as active contributors to global biotechnology innovation. “The defining characteristic of the Gulf in recent years is the sheer scale of its ambition to become a core contributor to global biotech innovation. While many mature pharmaceutical markets today are rigid and structurally constrained, this region is entirely unburdened,” agrees Hisham Hagar, executive country manager for the GCC at Vertex Pharmaceuticals, a Boston-headquartered biotech renowned for its pioneering work in developing advanced therapies for cystic fibrosis.
“What we are witnessing across the GCC are sovereign states driven by clear national visions, backed by substantial government investment, and possessed by an appetite to lead rather than follow in advanced therapeutics. Saudi Arabia’s commitment to hosting diverse clinical trials, supported by major long-term capital, directly captures this ambition in action,” he adds.
While Saudi Arabia and the UAE might lead the way with scale and financial firepower, the entire regional hinterland has been gripped by a desire to get in on the action. Other countries in the region are fast following suit, despite a landscape that remains profoundly diverse economically, culturally, geographically, and linguistically.
“Following the path blazed by Saudi Arabia and Abu Dhabi, Qatar has advanced remarkably within a very short window, though it remains a more compact market with fewer active sites. Oman also shows strong research promise, even as its infrastructure continues to mature,” evaluates Assem. Beyond that regional core, Jordan and Lebanon both offer well-established regulations but limited market potential. Meanwhile, Egypt, with its vast population, enjoys significant potential but remains restricted by legislation that bans foreign-sponsored Phase I and early-phase trials.
Inbound Investment Surge
Little wonder, then, that this shifting landscape has seen a marked uptick in foreign direct investment, with multinational drug developers aggressively expanding their regional research footprints and pouring substantial capital into localizing clinical studies across the Middle East, particularly across the Arabian peninsula.
“We have been modernizing our clinical pipeline execution within the Gulf to build a mature, credible research presence, which directly aligns with our global evolution into a rare disease-focused organization,” confirms Diederik Kok, Biogen’s GCC head. “In one of our recent Saudi Arabian programs, local centres actually became top global enrollers. This was a clear signal that the regional ecosystem can deliver high performance under the right conditions. That success generated deep confidence internally and among investigators, serving as the foundation for our current expansion,” he reveals.
Likewise, Swiss biopharma giant Novartis has positioned the GCC as a senior-management priority region for early-stage global clinical trials. “In recent years, we have intentionally reinforced our local clinical trials footprint – specifically within rare diseases and advanced therapeutics – while heavily investing in real-world evidence and local data that truly reflects the regional patient population,” affirms Mohamed Ezz Eldin, head of the company’s GCC Cluster.
“Developing local registries and generating regional evidence is a core part of this strategy, ensuring the data gathered here meaningfully informs global clinical practice. Naturally, we work hand-in-hand with national stakeholders, aiming not just to run individual trials, but to build sustainable, long-term R&D capabilities,” he elaborates.
This momentum is not limited to Big Pharma. Emerging, early-clinical-stage biotechs are also reassessing the region, drawn by the Gulf’s unique capabilities in areas like population-wide genomic screening. “Establishing a footprint in the Middle East might seem unconventional for an early-stage biotech, but the rich data and scientific possibilities make it the ideal environment to test a new paradigm in drug discovery,” reasons David Bearss, co-founder and CEO of Halia Therapeutics, a biotech notable for studying genetic resilience rather than disease alone.
“Instead of looking exclusively at people who fall ill, we wanted to study individuals who remain perfectly healthy despite carrying heavy genetic risks. Historically, tracking these resilient individuals has been incredibly difficult because healthy people rarely interact with clinical trial networks. However, the rise of massive population genomics initiatives tied to integrated health data in the Middle East is changing the game, allowing us to study genetic resilience systematically for the very first time,” Bearss explains.
Having developed a drug designed to modulate an inflammatory pathway, Halia Therapeutics is now preparing to test it in individuals who carry the APOE4 gene and exhibit early biological signals associated with Alzheimer’s risk. “Abu Dhabi provides a peerless setting for this work. Combining population-scale genomic data with fully integrated medical records makes it possible to precisely identify these high-risk individuals and execute biomarker-driven clinical studies that would be exceptionally difficult to run anywhere else in the world,” insists Bearss.
Vertex Pharmaceuticals, having recently expanded into rare disease areas like sickle cell disease and transfusion-dependent beta-thalassaemia through the development of landmark gene-editing therapies, is similarly actively beefing up its Middle Eastern capabilities. “As we expand our clinical pipeline, we intend to fully leverage the region’s rapidly evolving infrastructure and scientific depth,” confirms Hagar. “Our commitment goes far beyond simply delivering new therapies; we want to actively co-develop next-generation medicines. By aligning with the bold national transformation agendas across the Gulf, we aim to help the region cement its place as a global leader in advanced therapeutics,” he affirms.
Meanwhile, clinical research partners like CTI are reporting a palpable increase in interest from biotechs evaluating the region as an optimal clinical trials destination for drug development in therapeutic niches such as rare diseases and gene therapies. The independently owned CRO, which boasts a broader Middle Eastern geographical footprint than many of the top global clinical research and development providers, notes that rare diseases now account for over 75 percent of its workload in the region, with more than half of those projects involving a cell and gene therapy component. “We are seeing a steady influx of inquiries from biotechs with highly specialized clinical needs. They are eager to leverage the region’s unique attributes, understand the logistical landscape, and explore strategic partnerships with us,” confides Assem.
Regulatory Renaissance
What are the drivers underpinning such momentum? On one hand, international pharma is reacting favourably to the tangible regulatory and infrastructural enhancements sweeping across the region. “Novartis has spent a quarter of a century supporting the evolution of clinical research in the Middle East, and the environment on the ground – especially in the GCC – has improved beyond measure. We are seeing highly enabling frameworks and much clearer regulatory pathways,” opines Eldin. He points out that, in Saudi Arabia, the Food and Drug Authority (SFDA) has introduced the Research and Investigational Drugs pathway to accelerate the development and review of investigational therapies, alongside orphan drug mechanisms that support research in rare diseases.
Abdulrazaq Al-Jazairi, deputy executive director for research and innovation at the King Faisal Specialist Hospital & Research Centre (KFSH&RC), very much concurs. “We have seen the strategic creation of powerful external agencies across the Kingdom, all designed to foster an environment where top-tier clinical research and drug discovery can thrive. These include the Saudi National Institutes of Health, modelled directly on the US NIH, and the Research, Development and Innovation Authority, which bridges the gap from early discovery to investigational new drug submissions and first-in-human trials.”
He additionally notes how his organization has worked closely with the National Insurance Council to develop national policy on including patients in clinical trials under the national insurance framework, and with the Saudi Authority for Data and Artificial Intelligence on data sharing and cybersecurity – particularly in the context of rare diseases and genomics, where data governance is especially consequential.
Setting up a local trial has become notably more efficient too, an important consideration when delays equate to higher costs. “Barely five years ago, securing clinical trial approvals in Saudi Arabia could easily take nine to twelve months. Today, standard start-up approvals are completed in just four to five months, and can move even faster for therapies addressing critical, unmet medical needs,” enthuses Assem.
Thanks to these improvements, KFSH&RC has reached a tempo where, at any given point in time, it has approximately 170 clinical trials open for recruitment, of which between 25 and 30 percent ordinarily involve advanced therapeutic medicinal products, including cell and gene therapies. “Our mandate for the end of the decade is even more ambitious: we aim to maintain a constant portfolio of at least 1,000 active clinical trials at any single moment,” Al-Jazairi proudly reveals.
Meanwhile, Ahmed Alaskar, executive director of the King Abdullah International Medical Research Center (KAIMRC) – the R&D arm of the Ministry of National Guard Health Affairs – explains how he has been positioning his entity as a catalyser of high-quality clinical research. This is achieved by spearheading multicentre studies with a dedicated team of clinical research coordinators, associates, auditors, monitors, and quality controllers trained to the highest international standards and tasked with upholding them.
“We have successfully executed multicentre trials led by local institutions that have expanded onto the international stage. Simultaneously, the Saudi Clinical Trial Enterprise continues to make great strides in seamlessly bridging the gap between global pharma, regulatory bodies, and our local hospital networks.”
For international biopharma like Biogen, such support structures make a massive difference and have not gone unnoticed. “The collaborative role of the Saudi National Institute of Health and the Ministry of Health has been incredibly constructive. When patient identification proved difficult during one of our studies, national stakeholders stepped in to coordinate enrolment across multiple regions rather than leaving individual sites to struggle in isolation,” recounts Diederik Kok. “This underscores a genuine commitment to industry collaboration and a clear understanding that attracting global trials requires active, systemic support to ensure operational success,” he believes.
Living Laboratories & Integrated Scale
The United Arab Emirates is likewise pulling out all the stops to position itself as an innovative pharma R&D powerhouse and a go-to clinical trials destination of choice. “Last year, we launched Abu Dhabi’s Life Science Cluster, HELM, in partnership with the Abu Dhabi Investment Office, and we are engaging deeply with leaders across the global life sciences space,” explains Her Excellency Dr. Noura Khamis Al Ghaithi, undersecretary of the Department of Health of Abu Dhabi.
“Our value proposition is highly straightforward: we offer an immense wealth of premium, rigorously governed data, a highly secure research environment, world-class medical cities, advanced wet and dry labs, strong academic ties, and an investment ecosystem tailored for both nimble start-ups and global multinational corporations. For our partners, this translates directly into accelerated clinical trial recruitment, seamless access to longitudinal population datasets, and the unique ability to turn clinical research into real-world patient outcomes within a single, unified health system,” she explains.
In its quest to dramatically increase clinical trial activity – doubling down on oncology, genetic diseases, neurodevelopmental conditions, and paediatric indications – the emirate is offering an end-to-end sandbox. This ecosystem allows drug developers to advance from hypothesis to real-world application rapidly by identifying niche patient cohorts at unprecedented speeds. “What makes this possible is our centralized data infrastructure. If a sponsor needs to pinpoint every multiple sclerosis patient aged 40 and above across Abu Dhabi – complete with their exact location, treatment history, current disease status, and long-term prognosis – that entire cohort can be identified securely through a single data query. This allows us to radically accelerate clinical enrolment, monitor patients seamlessly within our care network, and rapidly generate the real-world outcomes data needed to guide the next phase of development,” Al Ghaithi elaborates.
She challenges traditional research dogmas regarding trial size: “The traditional assumption that robust clinical trials require a massive geographic population is outdated; true scale today is determined by data integration and usability rather than raw population size. A fragmented medical system with ten times our population simply cannot match what we do here. Abu Dhabi operates as a living laboratory: we conduct the research, instantly translate it into active clinical care, measure the real-world impact, and rapidly iterate.”
Not wishing to be outpaced, Qatar is also ramping up its capabilities. The nation offers access to the largest genome sequencing facility in the Middle East and leverages Qatar Genome Project (QGP) data to construct complete Middle East genome reference maps from scratch. “By pairing a world-class clinical trial unit at Sidra Medicine with direct access to highly specific patient cohorts and rigorous safety protocols, we are seeing a significant influx of requests from global pharma companies looking to launch Phase I trials here,” claims Tawfeg Ben-Omran, division chief of genetics and genomic medicine at the state-of-the-art medical centre, which serves as the primary clinical, technological, and analytic engine for the QGP.
“Securing a Phase I trial requires a deep foundation of trust built over years, and the fact that global drug developers are bringing these early-stage studies to our ecosystem is a true testament to the unique scientific value we now bring to the table,” he adds.
For entities like Recordati Rare Diseases – a dedicated global business unit of the Italian Recordati Pharmaceutical Group focused on orphan and ultra-orphan conditions – the appeal of this infrastructure is clear. “The massive infrastructural investments and regulatory upgrades across the GCC have opened up unprecedented opportunities to translate national genomic data into precise, actionable clinical trial designs. This enables us to pinpoint the exact patient cohorts we need, optimize our study selection, and ultimately drive far better clinical outcomes. It’s a no-brainer that we are actively working to bring our global clinical trials to this region,” confirms Khaled Elrefae, the company’s general manager for the Middle East and North Africa.
Many industry insiders view this as evidence of a fast-maturing clinical research ecosystem. “It is clear that the region’s clinical research momentum is no longer being pushed solely by external commercial interest, which is the ultimate litmus test for a maturing ecosystem,” perceives Biogen’s Diederik Kok. “Local health authorities, hospital networks, and clinical investigators across Saudi Arabia, the UAE, and Qatar are proactively driving the research agenda. This reflects rapidly rising local capabilities, highly efficient operational timelines, and a clear national determination to weave clinical research directly into the fabric of healthcare transformation,” he observes.
“There’s an unmistakable sense of being on a powerful upward trajectory, and we are seeing an incredible appetite for research from centers of excellence across the region,” concurs Mohamed Abu Shawish, cluster general manager for the GCC at Japanese drug developer, Kyowa Kirin. “Our primary focus has been forming strategic alliances with these key centres, supporting localized evidence generation, driving clinical alignment through peer-to-peer exchanges, and ensuring tertiary centres are fully equipped to anchor complex global trials. The momentum here is entirely real,” he continues.
A Genetic Crucible
Alongside this unmistakable coming-of-age of the Middle East’s clinical research ecosystem, the region offers a singular, high-utility value proposition to biopharmaceutical companies specializing in rare diseases. This advantage is rooted in an abnormally high prevalence of genetic disorders and an unparalleled density of patient populations. “The distinct ethnic makeup and cultural factors of the MENA region, including historically high rates of consanguinity, have resulted in a remarkably high prevalence and diversity of rare genetic disorders compared to Europe or North America, making the region an essential global hub for genetic research,” explains Hussein Abhari, head of the Middle East and North Africa at QIAGEN.
“The high clinical volume of hemoglobinopathies like sickle cell anaemia and thalassaemia, alongside metabolic diseases, spinal muscular atrophy, and rare neurological conditions, gives global sponsors an unparalleled environment to test and validate targeted gene therapies and other advanced medicinal products,” he contends.
Fatma Al Jasmi, professor of biochemical genetics at the UAE University’s College of Medicine & Health Sciences, puts the sheer scale of this situation into sharp perspective. “In Western populations, genetic carrier rates discovered during pre-conception screening typically hover around two percent. However, our university’s pilot data shows that this figure jumps to 14 percent across the general UAE population, and approaches nearly 25 percent in areas with high consanguinity. Put simply, certain rare diseases are concentrated here in numbers that match or exceed case counts found across the rest of the globe combined. This genetic density should not be viewed merely as a clinical burden; it is an invaluable scientific resource for ultra-rare disease discovery.”
Beyond the raw frequency of these caseloads, the region has evolved into a global epicentre for unearthing entirely new scientific insights. “When we analyse our genomic databases to isolate unique regional variants, we frequently uncover severe disorders that are documented almost exclusively within Arab populations,” explains Tawfeg Ben-Omran. “Close-knit tribal structures and large family sizes naturally yield larger, highly concentrated cohorts of patients sharing identical genetic mutations. For example, we discovered that 98 percent to 99 percent of classical homocystinuria patients in Qatar share a single founder mutation. This precise genetic homogeneity has allowed us to rapidly design and execute highly targeted clinical trials for novel enzyme replacement therapies,” he continues.
Professor Al Jasmi further notes that researchers are regularly uncovering unprecedented genetic profiles that challenge traditional clinical frameworks. “The enduring tribal and consanguineous structures of this region, sustained over centuries, have yielded a genetic landscape that is fundamentally different from anything documented in global literature. Because these disease patterns remain concentrated within specific lineage lines, patients frequently present with two or three distinct genetic disorders simultaneously,” she elaborates.
“This co-occurrence creates highly complex ‘blended phenotypes’ – mixed clinical presentations that are incredibly difficult to diagnose using standard Western medical frameworks. This reality completely shifts how these patients must be managed, and highlights exactly why clinical research focused specifically on this population is so globally vital,” Al Jasmi argues.
From Recipient to Global Influencer
Armed with fresh insights from this wealth of localized research, Middle Eastern data is already actively influencing global drug development in a multitude of ways, from rewriting rare disease registries to reforming global treatment guidelines. Today’s established best practice for the treatment of thalassaemia serves as a primary case in point, with many of the globally accepted prevention and therapeutic advances originating from clinical investigations conducted within the region, initially pioneering out of Lebanon.
“Our early collaborative work with the American University of Beirut (AUB) revealed a profound gap in the global understanding of what drives morbidity and reduced life expectancy, particularly in patients labelled as having intermediate disease. Rather than blindly adopting international definitions that didn’t align with the massive patient cohorts we see in the Middle East, we built extensive longitudinal cohorts to map the true natural history of the disease, quantify real risk factors, and uncover modifiable clinical targets,” explains Khaled Musallam, chief research officer at Burjeel Holdings Group and deputy CEO for the Cancer Institute in Abu Dhabi’s Burjeel Medical City.
Middle Eastern-generated research was thus at the forefront of establishing new classifications of the disease, with local data proving instrumental in shifting the historically vague label of ‘thalassaemia intermedia’ into clinically precise categories. “Our findings fundamentally changed how the global scientific community categorizes the disease. Ambiguous and unhelpful labels were replaced by a far more practical clinical distinction: transfusion-dependent thalassaemia (TDT), where patients require lifelong transfusions every few weeks, and non-transfusion-dependent thalassaemia (NTDT), where patients produce enough haemoglobin to survive without routine transfusions. These categories accurately reflected real patient pathology and revolutionized both clinical care and trial architecture,” remembers Musallam.
Perhaps most significantly, this regional work challenged the long-held assumption that NTDT was an inherently mild condition. Serious complications, including thrombosis and pulmonary hypertension, were documented for the first time, while chronic anaemia combined with progressive iron overload were shown to be clinically meaningful even in patients who were not receiving regular transfusions. “We unlocked a far deeper understanding of the critical role baseline haemoglobin plays, demonstrating that thalassaemia is a highly complex, multidisciplinary disease that critically impacts cardiology, hepatology, and endocrinology alongside traditional hematology. These insights radically transformed how comprehensive care is structured worldwide,” confirms Ali Taher, professor of medicine at the AUB. Musallam agrees, adding, “We were not just describing what we saw. We were building the evidence base that would eventually change how the disease was defined globally.”
Regional investigations uncovering these severe, hidden complications provided the critical impetus for the development of Luspatercept – a subcutaneous injection given every three weeks that promotes red blood cell maturation, serving as the first novel disease-modifying therapy to reach regulatory approval. Furthermore, strong research participation from the UAE, Saudi Arabia, and Lebanon has been critical to the clinical approval of Mitapivat, the first oral disease-modifying therapy for thalassaemia-related anaemia.
“This trajectory clearly demonstrates how our region can actively inform and guide the next generation of global medical science for rare and genetic diseases. Ultimately, this model is highly transferable because it is powered by dense patient concentration rather than just physical infrastructure,” argues Musallam.
Steep Learning Curve
For all its much-vaunted untapped potential, there is widespread recognition that the Middle East’s clinical research ecosystem – even within the Gulf states boasting the most advanced infrastructure – is still navigating a steep learning curve. True global competitiveness will naturally require time and sustained operational maturity.
“While it is highly impressive that the Middle East is developing deep expertise in cutting-edge areas like cell and gene therapy trials, the absolute volume of these studies remains modest, often concentrated across a handful of active sites,” observes Alaa Assem.
This is because clinical expertise is forged solely through delivery: successfully enrolling patients, securing regulatory approvals, activating sites, and managing complex treatment protocols. “These are incredibly sophisticated clinical projects that demand flawless synchronization between sponsors, regulatory agencies, hospital sites, and CROs. The sheer depth of logistics and regulatory detail involved is immense, meaning the region must navigate an unavoidable operational learning curve before achieving true mastery,” he insists.
Diederik Kok agrees that the region is still building its foundation relative to mature markets. “There is still work to be done to compete day-in and day-out with deeply entrenched trial hubs in Europe or Japan. We need to focus on enhancing centre readiness, securing long-term investigator commitments, and building a critical mass of operational experience to run complex trials at scale. Patience is essential, but the structural advantages and foundational elements are undeniably already in place.”
Perceptual impediments also persist that cannot realistically be remedied overnight, requiring a concerted effort to showcase the region’s actual capabilities. “An analytical gap still remains between international perception and our current clinical reality. Many global sponsors are genuinely stunned when they visit because their baseline expectations were set too low. Getting international drug developers to step onto the ground and tour our facilities is the single most effective way to shatter those outdated perceptions,” counsels Assem.
“When global sponsors engage face-to-face with regulators and principal investigators at flagship institutions like KFSH&RC, KAIMRC, SSMC, or Cleveland Clinic Abu Dhabi, it builds immediate institutional trust. Every major clinical hub followed this exact path. Eastern Europe, for instance, started with basic infrastructure, but nations like Poland and the Czech Republic are now global trial leaders. Global pharma invested heavily and drove that evolution; the Middle East is tracking along that exact same trajectory,” he posits.
The ultimate breakthrough for the Middle East will depend on moving beyond merely hosting trials to anchoring them within a self-sustaining, localized biotechnology engine. Abdulrazaq Al-Jazairi underscores this paradigm shift, warning that true leadership requires unifying a historically fragmented landscape. “To unlock the next level of maturity, we must seamlessly integrate our entire value chain – unifying basic science discovery, translational research, clinical trials, and real-world evidence generation so that insights flow continuously back into the discovery loop. Historically, these sectors have operated in silos,” he cautions. “Our absolute focus moving forward must be to connect these pieces deliberately, systematically, and permanently.”
