Your Easy Guide to Clinical Studies

Orphan Drugs are:

• Medicinal Products for rare diseases (e.g. at most 5 out of 10,000 individuals in Switzerland are affected)
• Used to treat rare diseases that are:
  • Life-Threatening or serious
  • For which no treatment options or no satisfactory ones are available

 

Based on Swissmedic, a company may apply for so-called "orphan drug status" early during the development phase of the drug.

 

A product with orphan drug status:

• Profits from cost reductions during future authorisation procedures (no fees apply).
• Is only available for use after Swissmedic has authorised the drug for the rare disease in question. Thus, the recognition of an “orphan drug” status alone is not sufficient

 

As only a small fraction of the population is affected by rare diseases, the development of an orphan drug often depends on the support of the government and applicable agencies.

As a SP-INV / Site-INV get familiar with the definition of orphan drugs

External Links

Swissmedic – see in particular

• ZL100_00_002e_WL Guidance document Orphan Drug

Swiss Law

TPA – see in particular article

• Art. 4 Orphan drugs definition