Beyond the Hospital Walls: A National Model for Lifelong Rare Disease Care
Redesigning Enzyme Replacement Therapy (ERT) Through Patient-Centered System Innovation
Program Overview
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Long before “Patient Engagement” became a healthcare buzzword, it was already being practiced, out of necessity, empathy, and strategic systems thinking.
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Between 2010 and 2013, a bold care-redesign initiative fundamentally changed how Enzyme Replacement Therapy (ERT) was delivered to patients with rare Lysosomal Storage Diseases (LSDs) across the Kingdom of Saudi Arabia (KSA).
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The goal was simple but transformative: remove access barriers that were undermining treatment compliance, family stability, and clinical outcomes, and replace them with a model designed around patients’ real lives.
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This initiative became what is now known as the ERT Relocation Program, a national success story in rare disease care, health system optimization, and true patient-centered innovision.
The Challenge: When Lifesaving Treatment Is Out of Reach
LSDs require lifelong intravenous ERT, administered weekly or biweekly under strict protocols. In KSA, where genetic metabolic disorders have a high prevalence, approximately 70% of patients receiving ERT lived outside Riyadh, yet treatment remained centralized at a single tertiary hospital.
For families, this meant:
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Weekly or biweekly long-distance travel
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Children missing school, parents missing work
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Significant and recurring travel and accommodation costs
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Emotional and physical exhaustion
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Declining compliance, despite the availability of effective therapy
In some cases, families stopped treatment altogether, not because of lack of understanding, but because the system left them with no viable alternative.
The Vision: A System Designed With Patients, Not Around Institutions
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Recognizing that treatment non-compliance reflected system-level barriers rather than patient behavior, the ERT Relocation Program emerged through a shared clinical vision to redesign care delivery for patients with Lysosomal Storage Diseases.
Dr. Nahya Awada, founder of DocNa, led this initiative in close collaboration with Dr. Hassnan and Dr. Sayyed to shape, operationalize, and advance this multi-dimensional initiative. Together, the team contributed to:
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The conceptual design and development of the ERT Relocation Program
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Coordinated national implementation efforts
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Training and capacity-building of healthcare professionals, including physicians, pharmacists, and nursing teams
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Development and adoption of standardized, evidence-based ERT preparation and administration guidelines
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Active engagement of patients and families to ensure continuity, safety, and trust throughout care transitions
The Solution: Three Integrated, Scalable Interventions
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1. Relocation of ERT to Local Hospitals (Outside Riyadh)
Between 2010 and 2012, 44 patients living outside Riyadh were successfully relocated to receive ERT safely in their local hospitals.
This required:
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Training physicians, nurses, and pharmacists in ERT preparation and administration
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Establishing protocols for central venous catheter management
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Coordinating drug logistics, cold-chain transport, and pharmacy readiness
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Actively preparing families to transition care with confidence
2. Home Enzyme Replacement Therapy (HERT) in Riyadh
For selected patients in Riyadh, especially those with severe mobility limitations, a home ERT model was designed and piloted. This innovation restored treatment continuity for patients who physically could not access hospital-based care.
3. National Standardization of ERT Guidelines
The creation of evidence-based ERT preparation and administration guidelines. These guidelines, which were used nationally and later regionally, standardized practice, reduced medication error risk, and enabled safe decentralization of care.
The Impact: Measurable Results, Sustainable Change
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* National Reach: Building Regional Capacity
Through the relocation program, ERT administration was initiated in 13 hospitals across 11 cities and provinces in Saudi Arabia. These centers were located at distances ranging from approximately 320 km to over 1000 km from Riyadh, significantly expanding access to specialized care across the Kingdom.
This rapid expansion—achieved within approximately 2.5 years—demonstrated that high-complexity rare disease care could be safely decentralized when supported by structured training, standardized protocols, and coordinated logistics.
* Compliance Before and After ERT Relocation
Treatment compliance improved markedly following relocation.
Among a subset of patients receiving ERT for Hunter syndrome and Maroteaux–Lamy syndrome, compliance was assessed over the 24 weeks before and after relocation, calculated as the proportion of scheduled infusions received.
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Median compliance increased from 54% before relocation to 92% after relocation
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This improvement was statistically significant (p = 0.002; n = 10)
Several patients demonstrated dramatic changes. Notably, two individuals whose compliance rates were only 8% and 17% before relocation achieved 100% compliance after relocation, underscoring the extent to which system barriers—rather than patient motivation—had driven prior non-adherence.
* Impact on Patients and Families
Relocating ERT administration closer to home reduced the required number of journeys to Riyadh from:
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52 trips per year for patients receiving weekly infusions, or 26 trips per year for patients receiving biweekly infusions
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To only two trips per year for follow-up care.
This translated into substantial cost savings:
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Annual travel costs decreased from approximately 78,000 or 39,000 Saudi Riyals (≈ USD 20,000 or 10,000)
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To approximately 3,000 Saudi Riyals per year (≈ USD 800), based on travel for three family members per visit
Beyond financial relief, the program significantly reduced disruption to family life, work, and schooling—contributing to improved quality of life and sustainability of lifelong therapy.
* Benefits for Hospitals and the Health System
The ERT relocation program delivered system-level benefits alongside patient-level gains.
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Healthcare providers at regional hospitals received specialized training and logistical support, strengthening local capacity for rare disease care
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At KFSH&RC, pressure on tertiary services was reduced, improving overall service efficiency.
As a direct result of relocation:
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1,820 ERT visits per year by patients from outside Riyadh were avoided
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This equated to approximately 10,920 hours of tertiary hospital bed utilization saved annually
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The freed capacity enabled KFSH&RC to focus resources on patients requiring advanced tertiary care, effectively creating national capacity through decentralization.
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Key results at a Glance:
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A Defining Example of Early Patient Engagement in Rare Disease Care
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This program was not just an operational success; it was a philosophical one.
It demonstrated that:
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Patient engagement is not a framework, it is a practice
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Equity in rare disease care requires structural redesign
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National impact is possible when clinical insight meets policy-ready execution
This program represents the true meaning of patient engagement, realized before the term was formalized, in one of the most complex areas of healthcare: rare disease.
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It is not just a program success. It is a reminder that when care is brought closer to home, hope follows.