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

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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. have exhausted all existing options. 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.

EURORDIS - The Voice of Rare Disease Patients in Europe