2nd Annual Orphan Drug Congress 2012

Venue: Barcelona

Location: Barcelona, Spain, Spain

Event Date/Time: Jun 07, 2012 End Date/Time: Jun 08, 2012
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Successful development plan for Orphan Drug

Regulatory challenges surrounding Orphan drug development

Developing patient registries

Commercial aspects of orphan drug development

Patient perspective

Ms. Lise Aagaard (University of Copenhagen) will cover the important Paediatric regulatory environment in EU for Orphan Drugs
President of OrphaNet, Ms. Segolene Ayme will present International Rare Disease Research Initiative

Treatments of rare diseases has long represented market niche in the pharmaceutical industry. Recently the importance of developing medicines for rare and obscure diseases have forced traditional pharma companies to re-evaluate their position on orphan and ultra orphan drugs. Despite the incentives relating to marketing exclusivity, taxes and fees, protocol design and research grants for the development of orphan drugs, there remain significant challenges.



Additional Information

Topic Introduction There are thousands of rare diseases. To date, six to seven thousand rare diseases have been found and approximately five new diseases are described every week in the medical literature. This number also depends upon the accuracy of the definition. Whether a single pattern is considered unique depends on the state of our knowledge, on the accuracy of clinical and investigative analysis and on the way we choose to classify diseases in general. The specificities of rare diseases are limited number of patients and scarcity of relevant knowledge and expertise. Some are rare forms or rare complications of common diseases. While signs may be detected at birth or in childhood, more than 50% of rare diseases appear during adulthood, and are often life-threatening or chronically debilitating. Usually there is no effective treatment, but screening for early diagnosis, followed by suitable care, can improve quality of life and life expectancy. Long considered niche markets and the domain of their smaller biotech brethren, traditional pharmaceutical companies are now re-evaluating their position on orphan drugs. A confluence of market dynamics and scientific advances seems to be driving this change.