Swissmedic Annual Report 2018

30 Market access | Marketing authorisation
Special categories of human medicinal products
Orphan drugs
Swissmedic may, upon application, recognise the status as
a medicinal product for a rare disease (orphan drug). Applicants
must prove that the medicinal product is used for the
diagnosis, prevention or treatment of a rare, life-threatening
or chronically debilitating disease that affects at most
five out of 10,000 people in Switzerland, or that it has been
granted this status by a country with comparable medicinal
product control. Most applications are based on the recognition
of the status in another country with comparable
medicinal product control, and in particular by the European
Medicines Agency (EMA) or the US Food and Drug Administration
(FDA). A medicinal product can be authorised
as an orphan drug either in parallel to or, more usually, as
the result of recognition of the orphan drug status. The
authorisation procedure for orphan drugs is exempt from
processing fees.
Activities
• 27 applications for recognition of orphan drug status
were received, and 30 applications were completed.
• Orphan drug status was granted 28 times, distributed
across 25 preparations.
• 14 preparations were authorised as new orphan drugs,
while additional orphan indications were approved for
13 existing orphan drugs.
• The status was discontinued for five products.
Paediatric medicinal products
Since the entry into force of EU Regulation EC 1902/2006
and the Food and Drug Administration Amendment Act
(FDAAA), it has been mandatory for pharmaceutical firms
to submit their paediatric investigation plans (PIPs) to the
authorities and to develop their medicinal products for use
by children in accordance with these plans. The Swissmedic
Paediatrics Working Group is responsible for the consistent
handling of this specific group of medicinal products. The
Ordinance on Fees permits a reduction of 90% in fees for
the authorisation of medicinal products with exclusively
paediatric indications and for corresponding major variations.
This measure is intended to encourage developments
in the area of paediatric medicines. Inspections relating to
paediatric clinical trials
are considered important: Annual planning will continue to
take account of inspections to assess compliance with GCP.
Activities
• In 2018, the submission of PIPs once again proved
helpful in the notification of paediatric clinical trials. A
total of 29 paediatric trials was authorised.