Orphan Drugs in Germany: Funding and Insurance Coverage for Rare Diseases

What are “Orphan Drugs”?
“Orphan Drugs” are medicines developed specifically to treat rare diseases (Seltene Erkrankungen). In the EU, a disease is considered rare if it affects:
fewer than 5 people per 10,000.
Examples include cystic fibrosis, inherited muscular dystrophies, certain rare cancers, and inherited metabolic disorders.

How are these medicines funded in Germany?
Although orphan drugs are often very expensive, the German healthcare system ensures funding mainly through:

Statutory Health Insurance (Gesetzliche Krankenversicherung – GKV)

• Most orphan drugs are generally covered by statutory insurance

• Patients typically only need a prescription from a specialist

• The medicine is covered almost fully, sometimes with a small co-payment (Zuzahlung)

Compensation for manufacturers
Due to high R&D costs and a small number of patients:

• Germany often allows relatively higher prices for orphan drugs to offset investment

• some companies also receive EU-level research incentives (EU Orphan Incentives)

How are orphan drugs approved in Germany?
They fall under EU pharmaceutical law, and the orphan designation is granted by:

• the European Medicines Agency (EMA)
  which can speed up approval and facilitate funding.
  In Germany, orphan drugs are often exempt from the traditional health-economic assessment under the AMNOG procedure, unless annual sales exceed €50 million.

Why are prices so high?

• very small patient groups → costs cannot be spread widely

• clinical trials are rare and expensive

• development is often aimed at urgent unmet medical needs (unmet medical needs)
  Nevertheless, they are funded because they can be the only hope for patients with no therapeutic alternatives.

Can a patient request a medicine that is not listed?
Yes, if:

• the specialist provides a detailed medical report

• the lack of therapeutic alternatives is demonstrated

• the insurer approves after reviewing the request
  In rare cases, requests are rejected and can be appealed via:

• the MDK (medical service of health insurance)

• or through the social court (Sozialgericht)

Statistics

• more than 130 orphan drugs are currently approved in Germany

• they cover treatments for more than 300 rare diseases

• funding costs the system billions annually, but is considered medically and ethically necessary

Summary

Note: The editorial team strives to provide accurate information through thorough research. However, errors may occur or some information may be unconfirmed. Please treat this content as an initial reference and consult the competent authorities for verified information.