Situation
Rare diseases, defined as conditions affecting fewer than 1 in 2,000 individuals, often require extensive experience as well as specialized care and ongoing treatment. In Saudi Arabia, patients receiving therapies such as intravenous infusions may need to make frequent visits to specialized healthcare facilities, creating logistical and geographic barriers to consistent treatment.
While efforts are underway by the Saudi Ministry of Health to strengthen support for people living with chronic and rare diseases, there remains a need for solutions that address the practical realities of receiving specialized care over time.
Solution
Recognizing these difficulties, a multinational pharmaceutical company identified the need for a Patient Support Program (PSP) tailored to address logistical, geographical, and compliance barriers for patients with rare diseases.
The PSP, launched in 2022 in collaboration with Axios International, was envisioned to provide holistic and personalized support to such patients that included services such as home administration for IV infusions, medication delivery between hospitals and directly to patients, and the provision of essential medical devices. It also was designed to align with the company’s commitment to improving access to healthcare and monitoring medication usage in the market to ensure efficacy and patient safety. Initially, the program focused on rare diseases requiring Enzyme Replacement Therapy (ERT), such as Gaucher disease, Fabry disease, Pompe disease, MPS1, and ASMD. However, the broader objective of the program extends beyond disease-specific care to include a robust support system that addresses logistical and operational challenges in delivering healthcare across the Kingdom. The program also facilitates the delivery of medications to the treating hospital or the patient’s home as per convenience. It also ensures the provision of medical devices and, thereby helps patients receive the necessary infusion sets required for their therapies, enabling smooth and uninterrupted treatment at home.
The program expanded in July 2024 to enhance treatment options. Under the program, both the patients and caregivers are provided with detailed education and awareness on proper medication storage, administration, and adherence practices.
Results
The program worked with referring physicians across 13 hospitals in Saudi Arabia and has enrolled more than 60 patients since its inception.
By bringing medication delivery, home administration and medical device support together, the program addresses practical barriers that can make ongoing treatment difficult for patients with rare diseases.
