Affiliations: National Centre for Rare Diseases, Istituto Superiore
di Sanità, Roma, Italy | Consorzio per Valutazioni Biologiche e Farmacologiche,
Pavia, Italy
Note: [] Corresponding author: Adriana Ceci, Consorzio per Valutazioni
Biologiche e Farmacologiche, Via Palestro, 26–27100 Pavia, Italy. Tel.: +39
0382 25075; Fax +39 0382 536544; E-mail: [email protected]
Abstract: The European Medicines Agency has expressed 50 positive opinions
recommending the granting of a marketing authorisation for an orphan medicinal
product since the Regulation on orphan medicinal products (OMPs) entered into
force in 2000. However, OMPs authorised at EU level are not always available at
Member States (MS) level. We developed and distributed a questionnaire to
collect information on the availability of 20 OMPs authorised before October
2006. The questionnaire included questions on the date of national market
availability; the possibility of pre-marketing access programme; the
distribution channel, the availability of a reimbursement policy. Twelve MS
provided information: Austria, Belgium, Czech Republic, Denmark, Estonia,
Finland, Hungary, Ireland, Italy, Latvia, Slovakia and UK. Results demonstrate
that the availability of OMPs varies greatly among the 12 MS considered and
market availability delays are highly variable. OMPs are often expensive drugs
and the different MS reimbursement policies are hindering access to OMPs. Data
show that since 2000 the number of OMPs has increased however issues including
costs and reimbursement policies at MS level represent major barriers to real
OMPs accessibility. This is a critical situation that deserves attention
because of the evident inequalities that do exist with regards to OMPs
accessibility among MS.
