Rare Diseases: Drug Shortages Threaten Patients

Imagine living with a chronic, rare disease, and that your health depends on a specific medication. Then, one day, it turns out to be unavailable. You do not know when it might be on-stock again, there are only limited alternatives, and the future looks uncertain. Many patients face this scenario driven by drug shortages resulting from disruptions in supply chains, manufacturing delays, raw material scarcity, and geopolitical tensions.

Despite medical progress, the global supply of essential medicines remains vulnerable. In the field of rare diseases, where only one or two treatment options may exist, a single disruption can have devastating consequences. Drug shortages may threaten not just individual health but also trust in healthcare systems.

Patients at Risk, Especially in Rare Diseases

Chronic conditions particularly require continuity of care to ensure patients' long-term wellbeing.

More Than One Option Is Not a Luxury - It’s a Necessity

When a therapy becomes unavailable and alternative treatments exist, they should be accessible to all patients - which means they must be included in reimbursement schemes. Countries should ensure more than one therapeutic option is approved and supplied to meet patients’ needs.

Supplier Diversity Strengthens Healthcare

In rare diseases, the number of suppliers is often limited. Encouraging diversity in suppliers not only builds resilience in the supply chain but also drives medical innovation. A healthcare system reliant on a single source is a vulnerable one.

Our Commitment to Patients

“AOP Health is a trusted partner in the field of rare and complex diseases”, says Jenny Maouas, Global Therapeutic Area Director Hemato-Oncology at AOP Health.

As a company focused on unmet medical needs, AOP Health continues to advocate for secure access to essential medicines, no matter how rare the condition or how complex the path.

For deeper insights into the topic, see the publication The Importance of Diversity of Supply in Rare Diseases Markets by The Office of Health Economics (OHE) dated January 23, 2025. OHE is an independent health economics research organization. (https://www.ohe.org)

Photo credit: Patient A: AdobeStock_714609392, Jenny Maouas: Ludwig Schedl

About AOP Health

AOP Health is a global enterprise group with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions to address unmet medical needs, particularly in the fields of rare diseases and intensive care medicine. The group has established itself internationally as a pioneer in integrated therapy solutions and operates worldwide through subsidiaries, representations, and a strong network of partners. With the claim "Needs. Science. Trust." the AOP Health Group emphasizes its commitment to research and development, as well as the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company’s actions.

Press Contact.

Mag. Nina Roth, MAS

Head of Corporate Communications
nina.roth[at]aop-health.com