What is it? Why is it important?

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.

What do I need to do?

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

Where can I get help?

Your local Research Support Centre can assist you with experienced staff regarding this topic

  • Basel, Departement Klinische Forschung (DKF), dkf.unibas.ch

  • Lugano, Clinical Trials Unit (CTU-EOC), ctueoc.ch

  • Bern, Department of Clinical Research (DCR), dcr.unibe.ch

  • Geneva, Clinical Research Center (CRC), crc.hug.ch

  • Lausanne, Clinical Research Center (CRC), chuv.ch

  • St. Gallen, Clinical Trials Unit (CTU), h-och.ch

  • Zürich, Clinical Trials Center (CTC), usz.ch

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
Abbreviations
  • CTU – Clinical Trials Unit
  • Site-INV – Site-Investigator
  • SP-INV – Sponsor-Investigator
Basic ↦ Drug or Device ↦ Therapeutic Products ↦ Orphan Drugs
Study
Basic

Provides some background knowledge and basic definitions

Basic Monitoring
Concept

Starts with a study idea

Ends after having assessed and evaluated study feasibility

Concept Statistic Methodology
Concept Drug or Device
Development

Starts with confidence that the study is feasible

Ends after having received ethics and regulatory approval

Development Drug or Device
Set-Up

Starts with ethics and regulatory approval

Ends after successful study initiation

Set-Up Ethics and Laws
Set-Up Statistic Methodology
Set-Up Quality and Risk
Set-Up Drug or Device
Conduct

Starts with participant recruitment

Ends after the last participant has completed the last study visit

Conduct Statistic Methodology
Conduct Drug or Device
Completion

Starts with last study visit completed

Ends after study publication and archiving

Completion Drug or Device
Current Path (click to copy): Basic ↦ Drug or Device ↦ Therapeutic Products ↦ Orphan Drugs

Please note: the Easy-GCS tool is currently under construction.