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The specific case of a named-patient Compassionate Use Programme
Compassionate use on a named-patient basis is another possibility for early access to a medicinal product
A named-patient prescription is delivered for only one named patient not taking part in biomedical research, under the responsibility of the prescribing physician, and when the medicinal product is likely to present a benefit for this patient.
These are medicinal products whose efficacy/safety ratio is supposed to be favourable considering the available data or, exceptionally, medicinal products prescribed because a fatal outcome for the patient in the near futture is unavoidable, given the current state of available therapies for the patient's condition.
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The voice of rare disease patients in Europe
The international voice of people living with rare diseases
Bringing together patients, families and experts to share experiences in a moderated multi-language forum.
The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.
An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiative
Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiative
The international voice of people living with rare diseases
Bringing together patients, families and experts to share experiences in a moderated multi-language forum.
The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.
An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiative
Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiative