Several regions have developed orphan drug legislation to stimulate research and development of treatments for rare diseases, but do these go far enough in solving the issues of rare disease patients? Speakers from the USA and Europe discuss what the ideal system looks like and what elements need to be in place to ensure development of and access to appropriate treatments for rare disease patients.



Objectives:

Identify, compare and contrast the legislation and incentives available in different regions of the world to stimulate the research, development and placing on the market of treatments for rare diseases.

Identify the strengths and weaknesses of the different approaches and highlight areas where either joining up or local improvement could strengthen the availability of safe and effective treatments.

Identify the specificities of rare disease treatments, and discuss approaches that will address the challenges that this highly specialized sector poses to health-care systems around the world.