Glossary

Glossary

Orphan Drug – Rare Disease Treatments & Regulatory Incentives


1. Orphan Drug Designation Criteria – EU vs. US.
 

European Union:

  • Affects <5 per 10,000 people
  • Regulating Authority: EMA (European Medicines Agency)
  • Incentives Provided: 10-year market exclusivity, fee waivers, protocol assistance

United States:

  • Affects <200,000 people per year
  • Regulating Authority: FDA (Food and Drug Administration)
  • Incentives Provided: 7-year market exclusivity, tax credits, fee reductions, grants
     

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.
  • US: 7 years of exclusivity after approval.

Tax Credits & Grants

  • EU & US: Research grants available for orphan drug development.
  • US: Up to 50% tax credit for clinical trial costs.

Fee Waivers & Reduced Costs

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

 Accelerated Development Pathways

  • Eligibility for priority review, fast-track designation, and expedited approvals.


3. Orphan Drug Approval Process

 Step 1: Apply for Orphan Drug Designation (ODD)

  • Must demonstrate the prevalence of the disease and medical need.
  • Applications submitted to EMA (EU) or FDA (US).

Step 2: Conduct Clinical Trials

  • Follows standard Good Clinical Practice (GCP) guidelines.
  • May qualify for expedited regulatory pathways.

 Step 3: Submit Marketing Authorisation Application (MAA/NDA/BLA)

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 $2.1 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.

 

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