Swissmedic has published an updated guidance for" Orphan Drug" aimed at streamlining the regulatory process for medicinal products treating rare diseases. Effective from January 15, 2025, this guidance provides comprehensive instructions for applicants seeking Orphan Drug Designation (ODS) and authorization in Switzerland.
An orphan drug is defined as a medicinal product aimed at diagnosing, preventing, or treating life-threatening or chronic debilitating rare diseases.
Rare diseases are those affecting no more than 5 in 10,000 people in Switzerland. Swissmedic also recognizes products designated as orphan drugs by countries with equivalent regulatory standards.
Swissmedic allows applications for ODS at any stage—before, simultaneous with, or after the marketing authorization application. The applicant must submit the following:
Scientific evidence demonstrating the rarity and significance of the disease.
Documentation showing recognition of ODS in other jurisdictions, if applicable.
Proof of product identity matching the one recognized abroad.
Timing: Applications for ODS are typically reviewed faster than marketing authorization applications.
Fees: Fees for ODS applications and related marketing authorizations are waived, reducing financial burdens for applicants.
Indication Extensions: Products with ODS can extend their indications to additional rare diseases. If new indications do not meet ODS criteria, the applicant may either modify the existing product or file a new application.
Document Protection: Orphan drugs receive 15 years of document protection, safeguarding intellectual property.
Swissmedic mandates that applicants notify the agency of changes to ODS granted abroad. This includes impending withdrawals, voluntary discontinuation, or ongoing review procedures. ODS can be withdrawn if the drug no longer meets the criteria for rarity. Swissmedic maintains an online list of orphan drugs in Switzerland. This includes:
Active substance(s).
Applicant or authorization holder.
Treated disease.
Authorization details, including dates and status.
This list is updated monthly, ensuring transparency and accessibility for stakeholders.
By offering simplified authorization processes, fee waivers, and extended document protection, the guidance encourages the development and availability of treatments for rare diseases.
For detailed instructions and specific requirements, refer to the full guidance document available on the Swissmedic website.
コメント