publication
Accelerating development, registration and access to medicines for rare diseases in the European Union through…
Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives…
publication
RAPS Orphan Medicinal Products Regulation in Europe
Regulation (EC) No. 141/2000 1 of the European Parliament and of the Council on Orphan Medicinal Products was drafted on…
publication
Progress in the development of Regulations for Orphan Drugs in Europe,Forum article for December 2002
Innovative therapies pose special problems for drug developers and regulators alike. By definition, such therapies tend to leap forward into…
publication
Orphan Drug Regulation in Europe – Fall 2002
The European Member States have decided that they cannot accept that certain individuals be denied the benefits of medical progress…
publication
Orphan Drug Regulation in Europe – Fall 2001
Rare diseases are referred to as “orphan diseases”, and the products intended to treat them as “orphan medicinal products”. In…
presentation
Smarter Orphan Drug Development
Rare diseases are characterized by a broad diversity of disorders and symptoms that vary not only from disease to disease…
whitepaper
Early Access Programmes / Compassionate Use Programmes: How to effectively deal with the challenges
Early access programmes (sometimes also called compassionate use or temporary use authorisation) were set up because of the rapid advances…
