Early access to medicines in Europe: Compassionate use to become a reality

EURORDIS has published a position arguing for compassionate use programmes for medicines to be made more widely available to those patients in need of urgent help.

At the moment the length of time needed to fully develop a medicine is too long for some patients. A compassionate use programme which takes places before the authorisation process has been carried out can offer a hope of treatment to those patients who are willing to take a higher risk with a non-tested product. Allowing early access to these promising new treatments, before their efficacy and safety have been fully established, can save lives.

At the moment not all countries benefit from a compassionate use programme, and different patients can access them at different times depending on where they live. EURORDIS proposes different policy ideas to help counteract this and offers recommendations to patients’ organisations, industry, member states and European authorities.

The Paper also takes ethical principles into consideration: namely the need for equal access for all patients to the treatment; transparency of the programme for the patients and general public; and a clear definition of who will benefit from the programme.

Please find the annexes for the Position Paper here.

 

 
 
La voce dei malati rari in EuropaEURORDIS Rare Diseases International, la voce internazionale dei malati rariRare Disease International RareConnect è un'iniziativa di EURORDIS che mette in contatto malati, famiglie ed esperti per condividere esperienze in un forum multilingua e moderato RareConnect Il Programma Rare Barometer è un’iniziativa di EURORDIS volta a condurre indagini con l’obiettivo di trasformare le esperienze delle persone affette da malattie rare in cifre e fatti che possono essere condivisi con i decisori politici.Rare Barometer La Giornata delle Malattie Rare, una campagna di sensibilizzazione internazionale che si svolge l'ultimo giorno di febbraio di ogni anno.Rare Disease Day La Conferenza Europea biennale sulle Malattie Rare è il più grande incontro di soggetti interessati al tema delle Malattie Rare in Europa. L'ECRD è un'iniziativa di EURORDISEuropean Conference on Rare Diseases