Glossary

1. Orphan Drug Designation requirements
​

There are two main requirements for Orphan Drug Designation in the EU:

• The medicinal product must be intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than 5 in 10,000 persons in the EU.

Alternatively, it can also be intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition for that – without incentives – it is unlikely that its marketing in the EU would generate sufficient return to justify the necessary investment;

• There exists no satisfactory method of diagnosis, prevention or treatment of the condition that has been authorised in the EU. If, however, such a method exists, the medicinal product will be of significant benefit to those affected by the condition.

The key requirements for Orphan Drug Designation in the USA are similar, but not identical:

• The condition for which the medicinal product is intended must affect fewer than 200,000 people in the United States (i.e. approximately 5-6 per 10,000 population).
• If the medicinal product is a vaccine, diagnostic drug, or preventive drug, it will be administered to fewer than 200,000 persons per year in the United States.
• In all other cases, there must be no reasonable expectation that costs of research and development of the medicinal product for the specific indication can be recovered by sales of the medicinal product in the United States.

2. Key Regulatory Incentives for Orphan Drugs

Governments provide special benefits to pharmaceutical companies developing orphan drugs, including:

Market Exclusivity

• EU: 10 years of exclusivity after approval + 2 additional years if an agreed PIP is available at the time of assessment of the ODD application.
• USA: 7 years of exclusivity after approval.

Fee Waivers & Reduced Costs

• EU: EMA waives or reduces scientific advice and regulatory fees.
• USA: FDA waives application and approval fees (PDUFA fees).

Others:

• EU: Protocol assistance and access to the centralised authorisation procedure.
• USA: Tax credit for clinical trial costs.

3. Orphan Drug Designation Process

 
Step 1: Apply for Orphan Drug Designation (ODD)

• Demonstrate the prevalence of the disease and (for the EU) the related medical need in the application.
• Prepare all relevant forms and other documents; for doing this, use the respective templates as required by EMA (EU) or FDA (USA).

Steps 2: Submission

• Submit the application
  • EU: Via IRIS
  • USA: Via the CDER NextGen portal, email or postal mail

Step 3: Evaluation by EMA/FDA

• EU: The Committee for Orphan Medicinal Products (COMP) will assess the ODD application within 90 days. Subsequently, the decision of the European Commission is adopted within 30 days.
• USA: The FDA division “Office of Orphan Products Development” will evaluate ODD applications within 90 days. 

4. Orphan Drug Success Stories

Several life-saving medicines have benefited from orphan drug designation:

Spinraza (nusinersen) – First approved treatment for spinal muscular atrophy (SMA).
Kalydeco (ivacaftor) – Breakthrough drug for cystic fibrosis.
Zolgensma (onasemnogene abeparvovec-xioi) – Gene therapy for SMA, priced at ca. 2 million € per dose.

Why Orphan Drug Designation is Important

• Encourages Development of Treatments for Rare Diseases.
• Reduces Financial Barriers for Drug Manufacturers.
• Provides Faster Access to Life-Saving Therapies.