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 voix des patients atteints de maladies rares en EuropeEURORDIS Donnant la voix, à l'international, aux personnes vivant avec une maladie rareRare Disease International Pour rapprocher patients, familles et experts sur un forum modéré multilingue, où partager leurs expériences. RareConnect Le programme Rare Barometer est une initiative d’EURORDIS qui vise à réaliser des sondages pour transformer les expériences des patients de maladie rare en faits et chiffres à communiquer aux décideurs politiques.Rare Barometer Campagne internationale de sensibilisation organisée chaque année le dernier jour de février, la Journée Internationale des Maladies Rares est une initiative d'EURORDISRare Disease Day Participez au plus grand événement réunissant tous les acteurs des maladies rares en Europe lors de Conférence européenne bisannuelle sur les maladies rares et les médicaments orphelins (European Conference on Rare Diseases and Orphan Products, ou ECRD). L'ECRD est une initiative d'EURORDISEuropean Conference on Rare Diseases