Sidra Medicine is building a vertically integrated academic medical centre where clinical care, research, and education operate as a single system to accelerate precision medicine, particularly in rare and complex paediatric diseases. In this interview, Professor Khalid Fakhro explains how the institution leverages national-scale patient cohorts, genomics infrastructure, and global partnerships to advance diagnostics, personalised therapies, and translational research while positioning Qatar as an emerging hub for biomedical innovation.
What is your mandate as Chief Research Officer at Sidra Medicine, and how does it connect to your broader roles within Qatar’s research ecosystem?
I serve as Chief Research Officer at Sidra Medicine, a women’s and children’s academic medical centre in Qatar, where I also chair the Precision Medicine Program. At the national level, I hold faculty appointments at Weill Cornell Medicine-Qatar (and at the graduate school at WCM-New York) and Hamad Bin Khalifa University, part of Qatar Foundation, where we have helped establish graduate programmes in biomedical sciences, genomics, and precision medicine. I also serve on the boards of the Qatar Precision Medicine Institute and Diabetes Qatar, alongside multiple roles on advisory committees in the health sector as well as several local universities and high schools focused on STEM. While these roles contribute to shaping the broader research landscape in the country, my primary operational focus is Sidra Medicine.
Sidra Medicine functions as an academic medical centre dedicated to women’s and children’s health, although our paediatric services represent the core of our national mandate. In practice, we serve as Qatar’s principal referral centre for complex paediatric cases, meaning that children requiring specialised or multidisciplinary care are referred to us from across the country. Any child referred for tertiary care is covered under the national plan, which allows Sidra Medicine to operate as a private hospital serving a public benefit. This structure eliminates any barriers due to cost, and ensures that children with rare and complex conditions are concentrated within a single institution, supported by more than 30 paediatric subspecialties spanning areas such as neonatology, paediatric intensive care, neurology, pulmonology, genetics, and complex surgical care.
For patients and families, this integrated structure simplifies what is often a complicated clinical journey. Children with rare diseases frequently require input from multiple specialists, and at Sidra Medicine these referrals take place within our hospital walls, rather than across multiple institutions. From a research perspective, the benefits are even more significant. Because the entire patient journey, from diagnostics and imaging to laboratory testing and long-term follow-up, is captured within a single electronic health record (EHR) system, we generate exceptionally rich longitudinal datasets. In effect, every child with a tertiary condition (e.g. T1D, epilepsy, congenital malformation) in Qatar enters the same clinical environment and remains within that system throughout their care.
Sidra Medicine’s model also benefits from Qatar’s geography, where patients can reach the hospital from almost anywhere within roughly 20 minutes, which facilitates access to specialised care and strengthens continuity of clinical data. To offer the most for patients, Sidra Medicine operates as a fully integrated academic medical centre. In many healthcare systems, research, clinical care, and education are distributed across separate institutions that collaborate through formal agreements. While that model has proven effective in some regions, it often requires significant time to develop the culture and coordination needed for genuine integration.
At Sidra Medicine, we adopted a different approach by embedding research directly within the hospital as a core operational branch. Our founding vision is to be a beacon of learning, discovery, and exceptional care, and we see these elements as inseparable. To reinforce this, our hospital was named after the Sidra tree, which appears in the Qatar Foundation logo, which is not only a symbol of resilience and life in our harsh climate, but also a metaphor for the institution being held up by three interconnected branches: clinical care, medical education, and research. By structurally integrating research into the hospital itself, we have been able to accelerate the translation of discovery into clinical practice and build a model that increasingly enables what we describe as ‘research-guided care’ as part of a patient’s journey.
How does Sidra Medicine ensure that its research activities translate into meaningful improvements in clinical care?
At Sidra Medicine, we maintain our own Institutional Review Board (IRB), which allows investigators to submit patient-facing studies for internal ethical review while receiving operational and financial support from our research infrastructure. Many of our physicians have trained at leading academic medical centres and naturally identify clinically interesting cases that can be channelled into research programmes. And many of our full time researchers have likewise trained at world-class institutions bringing their incredible skills and expertise to crack those same diseases. This creates a practical bridge between clinical observation and scientific investigation, ensuring that discoveries can emerge directly from the patient journey and be evaluated within the same clinical environment.
Our approach is grounded in a broader philosophy about the role of research in healthcare. Many of the technologies that clinicians rely on today began as research projects and took years of validation before becoming part of standard medical practice. In many ways, research environments, and their myriad advanced technologies, operate several years ahead of routine clinical care. When patients with rare or undiagnosed conditions exhaust conventional diagnostic pathways, they are often referred to academic medical centres precisely because those institutions have access to advanced research technologies. Sidra Medicine was designed with this principle in mind. As early as 2014, we established a high-throughput genomics centre equipped with next-generation sequencing platforms, enabling us to integrate genomic analysis into the study of rare and complex diseases and support national initiatives such as the Qatar Genome Programme in pioneering precision health at population scale.xx
Our research strategy is structured around four pillars. The first focuses on precision diagnostics, bringing advanced platforms such as clinical genomics, microbiome analysis, flow cytometry, advanced imaging, and functional disease modelling closer to the point of care. These capabilities help shorten the diagnostic journey for children with complex conditions by allowing clinicians to access research-grade technologies within the clinical setting.
The second pillar centres on personalised therapies, recognising that many rare or complex diseases require treatment strategies tailored to the biology of each patient rather than a single standard approach. To support this, Sidra Medicine established Qatar’s first GMP facility within the hospital. This facility enables the production of clinical-grade biologics, cell therapies, and gene-based treatments on site and has already allowed us to launch the country’s first paediatric hematopoietic stem cell transplant programme. For children with conditions such as leukaemia, this means that highly specialised procedures such as bone marrow transplantation can now be performed locally rather than requiring travel abroad. The facility also prepares us to participate in emerging therapeutic modalities, including gene-editing approaches (most recently, Casgevy) and other advanced treatments that are currently progressing through clinical trials.
The third pillar of our strategy focuses on implementation science. While much attention in biomedical research centres on disease mechanisms and drug development, there is also a need to study how healthcare systems function in practice. At Sidra Medicine, our aim is to function as an academic learning health system, where areas such as health economics, patient experience, and the cost-effectiveness of new technologies constantly inform healthcare policy and system design. This perspective reflects our ambition to position Sidra Medicine not only as a centre for clinical care and discovery, but also as an open platform for rigorous academic projects studying how healthcare can be delivered more effectively.
Finally, the fourth pillar centres on building a world-class research ecosystem. We aim to attract leading clinicians and scientists, strengthen collaborations with academic institutions, and work closely with biotechnology and pharmaceutical partners through our newly formed innovation lab. We believe the opportunities in Qatar for Life Sciences are only going to grow, and see Sidra Medicine as a central player along the entire value chain, from capacity building and early career scientist training, to building a sustainable and resilient infrastructure advancing patient care.
To operationalise this strategy, we have organised our activities into six translational research programmes covering the following major disease areas: neuroscience, metabolic and Mendelian disorders, paediatric cancer, congenital malformations, immune dysregulation, and reproductive and perinatal health. Each programme focuses on what we call “gold cohorts”, disease populations for which Sidra Medicine sees the majority of cases in the country and where clinical expertise, patient access, pharma interest, and research platforms converge. These programmes allow us to build detailed patient registries, advance genomic research, and progressively translate research findings into clinical practice, reinforcing Sidra Medicine’s role as an emerging centre for genomic medicine and paediatric translational research.
How is Sidra Medicine balancing investigator-led research with industry-sponsored studies, and how do you see this evolving in the coming years?
At present, most of the research conducted at Sidra Medicine remains investigator-led, reflecting both the academic foundation of the institution and the relatively recent expansion of our clinical trials infrastructure. The pandemic delayed several key developments, including the activation of our GMP facility and CRO activity in the region, which in turn slowed the pace at which we could scale industry-sponsored studies. Today we have a modest number of patients enrolled in trials and several additional studies in preparation, while discussions are ongoing with >20 industry sponsors. That said, the intention is clearly to expand this activity to have a substantial fraction of ongoing research projects (currently >270 in number) be clinical trials; however, one must approach this goal in a thoughtful way, prioritising collaborations that align with the patient populations, building strong partnerships with industry and CROs, and ensuring the clinical expertise exists within each target subspecialty for running trials at Sidra Medicine.
Both the regulatory environment and types of patients we treat naturally shape this approach. Many of the children referred to Sidra Medicine present with rare or highly complex conditions for which standard therapeutic options are limited. In such cases, early-phase clinical research may represent one of the few realistic avenues available to families. Because Sidra Medicine functions as a national referral centre for these complex paediatric cases, rare diseases targeted by pharma become ‘common’ in our health system, and our strong research focus ensures we are well positioned to contribute to early-stage and translational studies. We have already seen how this can work in practice. In one industry-sponsored paediatric study for a rare condition, for instance, we worked closely with the Ministry of Public Health and our internal IRB to review the protocol, assess the available safety data, and secure approval for a Phase 1 trial in children. Experiences like this illustrate that, when there is a clear medical need, the regulatory and clinical environment in Qatar can support carefully designed experimental research.
In parallel, we have taken steps to strengthen the broader life sciences innovation ecosystem around Sidra through the creation of the Sidra Medicine Innovation Lab. This initiative provides a structured entry point for biotechnology companies, pharmaceutical partners, and technology developers seeking to collaborate with our clinicians and researchers. Many of the organisations approaching us are early-stage companies developing artificial intelligence tools, digital health platforms, or exciting new therapeutic approaches to key disease areas (e.g. gene editing for local founder mutations).. Through the Innovation Lab they can get access to high-quality datasets and real-world validation environments, leveraging resources such as EHRs, imaging repositories, and multi-omics platforms, translating those insights into clinical studies conducted within the hospital.xx
The strong interest we have seen since the Innovation Lab’s launch reflects a broader reality within the regional ecosystem. For many years, large multinational pharmaceutical companies maintained only a commercial presence in the Middle East, but significant research and development infrastructure remains concentrated elsewhere. Rather than waiting for that landscape to evolve, we have chosen to engage directly with emerging biotechnology companies whose technologies intersect with the disease areas we treat. The Innovation Lab has already attracted considerable attention from startups seeking a trusted clinical partner in the region.
I see these private enterprises as crucial for closing the “implementation gap.” In most countries, research moves from bench to bedsides through commercialisation vehicles and companies that are funded by the private sector, and which consequently have a greater ability to take risks as well as access to private capital to hire, spend and execute. Without them, we risk falling into the “valley of death” in terms of implementation from bench to bedside.
Over time, our ambition is to build a translational ecosystem in which innovation, clinical research, and patient care reinforce one another, positioning Sidra Medicine as a credible partner for advanced paediatric research and therapeutic development.
Where do you see the greatest opportunities for Sidra Medicine and Qatar to establish international leadership?
One area where Sidra Medicine is particularly well positioned to contribute globally is in rare disease diagnostics and therapy. Because complex paediatric cases from across the country (and, increasingly, the region) are concentrated within a single institution, we are able to work with highly defined patient populations that are often difficult to assemble elsewhere. This creates an opportunity to collaborate with partners developing new diagnostic platforms or novel therapeutic approaches for rare conditions. For biotechnology and pharmaceutical companies working in this space, Sidra Medicine can serve as a trusted clinical partner where emerging technologies and therapies can be evaluated in a high-quality research environment, with the potential to accelerate both discovery and clinical translation.
At the same time, Qatar’s size and tightly integrated national ecosystem for science allows for multidisciplinary initiatives that extend beyond traditional biomedical research. Our work on autism is a good example of this model. There is a strong national commitment to improving the diagnosis, treatment, and long-term support of individuals and families with autism spectrum disorder, reflected in initiatives such as the BARAKA-Qatar Study, which investigates the genetic and environmental drivers of autism in the Qatari population. Because autism represents a broad spectrum of conditions, meaningful progress requires collaboration across multiple disciplines, which is why today this study has grown to encompass several collaborating sites, including clinical centers, schools, and local faculty working in the areas of stem cell biology, artificial intelligence, social sciences, and education, all working to explore new ways of understanding and supporting affected individuals. By combining these perspectives within a coordinated national framework, Sidra Medicine and its partners have an opportunity to influence public policy and develop more comprehensive approaches to complex conditions that eventually also inform international research and care models.
Looking ahead, what milestones would you hope to highlight for Sidra Medicine over the next few years?
Over the next several years, we expect to see a significant expansion in the scale and scope of clinical trials and translational research conducted at Sidra Medicine. At the same time, we are placing increasing emphasis on implementation science, with the goal of ensuring that research findings translate into meaningful changes in healthcare delivery and policy, both within Qatar and more broadly. An equally important priority is capacity building. We have invested heavily in developing local expertise by working closely with universities across Qatar to mentor master’s and PhD students within Sidra Medicine. Over the past decade, we have helped establish national training programmes in areas such as genomics, disease modeling, precision medicine, bioinformatics, artificial intelligence and genetic counselling. These programs are designed to build a sustainable national workforce that is able to scale genomic medicine across the country.
Another area of growth is the transition from sick care to health care. The last decade of diagnostic research has revealed a slew of biomarkers and founder mutations specific to our population, which we continue to compile. As our knowledge of these grows, and the costs to perform advanced testing goes down, we are piloting innovative screening programs for families. A good example of this is the use of whole genome sequencing, which has long served as the gold standard for rare disease diagnosis. With dropping costs and growing reference databases, we have now started a newborn genomic screening program, alongside similar initiatives leveraging microbiome, immune biomarkers and imaging testing in different cohorts. We envision transforming the Qatari health system from reactive to proactive and believe this could serve as a model for other systems around the world. This goes back to the implementation pillar I referenced earlier, as we need good implementation science to measure the utility, cost-effectiveness, and degree of success in integrating evidence-based practice into routine healthcare.
Finally, Qatar has long-been a champion of international dialogue and learning, and science is no exception. At Sidra Medicine, we focused relentlessly over the past decade on hosting specialised workshops and international conferences that convene researchers, clinicians, and industry leaders working in fields such as genomics, advanced imaging, computational biologydisease modelling and precision health. Our flagship event is the Precision Medicine and the Future of Genomics (PMFG) summit, which we launched in 2014 and which is now approaching its twelfth edition in December. What began as a largely academic meeting has gradually evolved into a broader platform that attracts participants from across the biotechnology and investment communities as well. Over time, these events have played an important role in connecting Sidra Medicine with leading institutions worldwide, helping us establish collaborations and write multi-million dollar grants with more than fifty partners across local, regional, and international networks over the years.
Taken together, these initiatives reflect a broader effort to build a durable global ecosystem around Sidra Medicine. Through sustained investment in training, research partnerships, and scientific convening, we have been able to strengthen our research profile and develop credibility among leading investigators who see Sidra Medicine as a place where ambitious ideas can be pursued within an integrated clinical and research environment. Our goal over the coming years is to continue building on that foundation, positioning Sidra Medicine not only as a centre for clinical care and discovery, but also as a living lab for international collaboration in precision medicine and research-driven care.
