Top

Published in:

Open Access 01-11-2012 | Meeting abstract

Compassionate use programmes for rare diseases: proposals for actions

Authors: François Houÿez, Chantal Bélorgey, Arielle North, Etelka Czondi, Michele Lipucci di Paola

Published in: Orphanet Journal of Rare Diseases | Special Issue 2/2012

Excerpt

Patients’ advocates, regulators and doctors have the constant objective to make the best treatments quickly available to patients. Compassionate use programmes (CUP) as defined by the EU Regulation (EC) № 726/2004 article 83.2 are designed to provide early access before the marketing authorisation (MA) of a medicine. It is necessary to evaluate the achievements of the present legislations on CUP in Europe in order to propose actions that could integrate CUP into an improved orphan drug development model. …

Title: Compassionate use programmes for rare diseases: proposals for actions
Authors: François Houÿez
Chantal Bélorgey
Arielle North
Etelka Czondi
Michele Lipucci di Paola
Publication date: 01-11-2012
Publisher: BioMed Central
Published in: Orphanet Journal of Rare Diseases / Issue Special Issue 2/2012
Electronic ISSN: 1750-1172
DOI: https://doi.org/10.1186/1750-1172-7-S2-A28

At a glance: The STEP trials

Springer Medicine

Compassionate use programmes for rare diseases: proposals for actions

Excerpt

At a glance: The STEP trials

Springer Medicine

Excerpt

Please log in to get access to this content

Other articles of this Special Issue 2/2012

Developing a cure for Black Bone Disease

Exon skipping for DMD

Transition from childhood to adulthood in Duchenne muscular dystrophy (DMD)

A coordinated EU approach to informed access decisions: CAVOD process proposals – the possibility to turn concept into reality?

Professional clinical guidelines for rare diseases: methodology

Leveraging existing opportunities for improved Orphan Drug approval in the EU