Swissmedic updates guidance on orphan drugs and paediatric investigation plans

The Swiss Agency for Therapeutic Products (Swissmedic) has updated their guidance on orphan drugs and paediatric investigation plans (PIPs). Topics addressed include orphan drug fees and the documentation companies need to supply to support European and American PIPs.

Revisions to the orphan drug guidance focus on three sections, for example a new section on rare disease indication extensions is one of the more substantive changes. If a medicine has orphan drug status in one rare disease, then Swissmedic will allow use in another rare disease under the same name and authorisation number. However, if the new disease does not meet the orphan drug criteria, then either the medicine will lose it status or be sold under a different name and number for the new indication.

The opposite applies when a company would like to expand use of an approved drug without orphan drug status into a rare disease. Swissmedic have outlined that they require manufacturers to sell the drug for the rare disease under a different name and authorisation number.

In the updated guidance for orphan drugs, Swissmedic also describes the process for transferring orphan drug status to another company and have added a section which states Swissmedic will waive the flat-fee rates for applications of medicines with orphan drug status.

Revisions were also made to multiple documents related to PIPs, including the documents that companies must submit in relation to investigation plans newly produced for Switzerland, and those already prepared for authorities in the United States or in Europe.

Provided that the same indications and dosage forms are covered in each territory, Swissmedic will accept PIPs approved by the European Medicines Agency (EMA) and equivalent plans cleared by the United States Food and Drug Administration (FDA). Conditions of fulfilment differ depending on whether a PIP is specific to Switzerland or not. Regarding Swiss PIPs, Swissmedic expects companies to document results within 60 days of the creation of the final study report.

To read Swissmedic’s updated guidance on orphan drugs, please click here.

To read Swissmedic’s updated guidance on PIPs, please click here.