Orphan drug authorisation in Switzerland: the simplified procedure and Art. 13 TPA

What the simplified authorisation procedure actually simplifies

The simplified procedure changes the evidentiary expectations and the administrative load, not the legal standard. Swissmedic still has to be satisfied that quality, safety and efficacy are demonstrated. What changes is that the rarity of the disease is taken into account when preclinical and clinical data are assessed, and that the application is processed with priority.

Articles 24 to 26 TPLO set out the specific provisions for authorising a medicine that holds the status. They are read together with Art. 4 to 7 TPLO, which govern the status itself, so an applicant works with two connected sets of rules in the same ordinance.

How Swissmedic evaluates the evidence

The assessment starts from the same triad as any other application: quality, safety and efficacy must be demonstrated. Swissmedic then factors in that patient numbers are small and that adequately powered trials are harder to run. In justified cases it accepts published results in place of complete trial documentation, which is a concession on data format, not on scientific substance.

• Quality, safety and efficacy remain the legal test and are not waived by the status.
• The rarity of the disease is taken into account in the appraisal of preclinical and clinical data.
• Published results may replace complete trial data in justified cases.
• A scientifically insufficient dossier is not made acceptable by the status.

The practical consequence is that the burden shifts towards argumentation. An applicant has to explain why a given design was the only feasible one, why the endpoints are meaningful in the disease and why the published evidence it relies on is adequate for the claimed indication.

The Art. 13 TPA route for products already authorised abroad

Article 13 TPA is the second lever for orphan drugs. Where a medicinal product is already authorised in a country with comparable medicinal product control, the results of the tests carried out for that authorisation are taken into account appropriately by Swissmedic. The same concept of comparable control underpins the recognition route for the status itself.

Article 13 TPA does not create automatic recognition. Swissmedic conducts its own assessment and decides what weight the foreign results carry. Applicants therefore submit the foreign dossier in a form that lets Swissmedic follow the original evaluation, rather than a summary of the foreign decision.

The same concept also supports the status itself, where recognition as an important medicinal product for rare diseases in a country with equivalent medicinal product control replaces a Swiss prevalence argument. The two Art. 13 TPA routes are separate decisions and are documented separately.

What the authorisation dossier has to contain

The authorisation dossier is a complete quality, safety and efficacy dossier, prepared in the knowledge that the rarity of the disease will be considered. Where the product relies on a foreign authorisation, the material submitted to that authority and its assessment belong in the file so that Art. 13 TPA can be applied.

1. Establish or confirm orphan drug status, ideally before or in parallel with the authorisation application under Art. 4 para. 3 TPLO.
2. Assemble the quality module and the preclinical and clinical documentation, flagging where published results stand in for complete trial data and why that is justified.
3. Where the product is authorised abroad, include the documentation submitted to that authority so Swissmedic can take the foreign test results into account under Art. 13 TPA.
4. Prepare the product information for the authorisation decision in German, French and Italian.
5. File the application with Swissmedic under the simplified procedure of Art. 14 para. 1 let. f TPA and Art. 24 TPLO and request priority processing.

Priority processing applies to the authorisation application, so the dossier should be complete when filed. A priority queue does not compensate for missing modules, and queries that stop the clock remove much of the benefit the status was meant to deliver.

Indication extensions and the fate of the status

Adding an indication to an authorised orphan drug forces a decision about the status. If the new indication itself meets the orphan drug criteria, the applicant files a new orphan drug status application together with the indication extension as a major variation of type II. Name and authorisation number can stay and the status is retained.

If the new indication does not meet the criteria, there are two outcomes. Either the product loses the orphan drug status, or a separate new medicinal product is authorised for the non-rare indication while the orphan product keeps its status for the rare one.

A new dosage form of an already authorised orphan drug needs no separate orphan drug status application. The cover letter refers to the existing status. This keeps line extensions within the ordinary variation framework instead of reopening the rarity assessment.

Fees and what the status does not cover

The fee relief is narrower than often assumed. Article 9 let. a of the Fees Ordinance for Swissmedic (SR 812.214.5) waives the flat-rate fee for the new authorisation application. Everything after that follows the ordinary tariff, and the surcharge for a fast-track procedure remains payable even for a product with orphan drug status.

• Waived: the flat-rate fee for the new authorisation application under Art. 9 let. a FeeO-Swissmedic.
• Payable: variations, which have been subject to fees since 1 January 2019.
• Payable: the fast-track surcharge, despite the status.
• Available on request: 15 years of document protection under Art. 11b para. 4 TPA, instead of any form of market exclusivity.

Document protection has to be requested; it is not granted automatically with the status. Since Switzerland grants no market exclusivity at all, the request under Art. 11b para. 4 TPA is the only protection instrument tied to the orphan drug route and should be diarised early.