Switzerland – Swissmedic Updates Guidance on Orphan Drugs, Pediatric Investigation Plans

The Swiss Agency for Therapeutic Products (Swissmedic) has updated its guidance on orphan drugs and pediatric investigation plans (PIPs). The revisions address topics, including orphan drug fees and the documentation to supply in support of European and American PIPs.

Swissmedic’s changes to the orphan drug guidance center on three sections. A new section on rare disease indication extensions is among the more substantive changes. If a medicine has orphan drug status in one rare disease, Swissmedic will allow its use in another rare disease under the same name and authorization number. If the new disease does not meet the orphan drug criteria, the medicine will either lose its status or be sold under a different name and number in the new indication.

The reverse is true when a company wants to expand use of an approved drug without orphan drug status into a rare disease. Swissmedic will make manufacturers sell the drug for the rare disease under a different name and authorization number.

Swissmedic also outlined the process for transferring orphan drug status to another company and added a section stating that it will waive the flat-rate fees for applications of medicines with orphan drug status…