Why medicine reimbursement is crucial for orphan drugs

Orphan drugs can play a crucial role in the lives of patients with rare diseases, but the journey from development to patient access is often challenging. Even after receiving market authorisation, these medicines may not be accessible without proper reimbursement. Medicine reimbursement is thus a crucial lifeline for patients with rare diseases. 

What are rare diseases? 

Rare diseases affect fewer than 1 in 2,000 individuals but account for around 5% of the global population. With over 6,100 rare diseases identified, many of which are genetic and life-threatening, effective treatments are essential. The complexity of these conditions, along with diagnostic and treatment challenges, can pose significant difficulties for healthcare systems. 

What are orphan drugs? 

Orphan drugs are vital for treating rare diseases, often providing the only option for symptom relief and improved quality of life. In the EU, medicine qualifies as an orphan drug if it targets a life-threatening or chronically debilitating disease affecting fewer than 5 in 10,000 people. Additionally, it must either provide a significant advantage over existing treatments or address a condition with no satisfactory treatment options. 

Orphan drug designation and market authorisation 

The Committee for Orphan Medicinal products (COMP), part of the European Medicines Agency (EMA), grants orphan drug status, offering incentives like a ten-year market exclusivity period to protect research investments.  

Once a medicine is granted orphan drug status, it must undergo evaluation by the EMA for market authorisation. The EMA assesses the drug’s safety and efficacy before granting approval. However, market authorisation does not guarantee patient access. 

Reimbursement decisions are made by national healthcare systems and are not automatic after market approval. Each drug undergoes a separate evaluation, considering clinical benefits, alternative treatments, and cost-effectiveness.  

Ensuring access to orphan drugs through medicine reimbursement 

While EMA approval is crucial, reimbursement often presents the biggest barrier to access. Even with market authorisation, a drug may not be reimbursed, limiting access through public healthcare systems. Securing reimbursement requires navigating complex regulatory and financial landscapes, with pharmaceutical companies needing expert guidance to ensure inclusion in national reimbursement lists. 

A collaborative approach for a brighter future 

The potential to improve the lives of rare disease patients is immense. Through collaboration across countries, medical disciplines, and sectors, healthcare communities can overcome access barriers. For pharmaceutical companies, working with market access, reimbursement, and rare disease care experts is key to ensuring orphan drugs reach those who need them most. Together, we can make life-changing medicines a reality for more patients across Europe.