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- What is an orphan drug?
- Improve the safety of your medicines
- Compassionate Use
- Role of EURORDIS
- Patient advocates involvement
- Access to orphan drugs
- Health technology assessment
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- List of the latest marketing authorisations and orphan medicinal products designations
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Compassionate Use
There is nothing worse for a patient, from a psychological and human standpoint, than being severely ill or even dying from a disease, when experimental treatments are out there, pending final evaluation.
Nowadays, patients are aware of new product developments; research is no longer a secret. In some countries, regulators have responded to this situation, and medicines can be obtained on a Compassionate Use basis prior to their marketing authorisation, to help treat patients who have no other options and who do not have the time to wait for the end of clinical trials and the authorisation process.
These pages inform patients and healthcare professionals about Compassionate Use possibilities. Compassionate Use can save lives.
EURORDIS position on compassionate use
EURORDIS position arguing for compassionate use programmes to be made more widely available to those patients in need of urgent help.
Good to know
This section explains the role of Compassionate Use Programmes: who are they for, and the conditions for accessing a medicine under compassionate use.
How to proceed
This section explains how your physician can request authorisation to prescribe to you a medicine on a compassionate basis
Resources
Useful services: where you can learn more about Compassionate Use Programmes.
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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