There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was limited interest from the pharmaceutical industry to develop treatment for very small patient groups, owing to the fact that similar levels of investment are needed from a pharmaceutical company to bring a drug to market for both small and large patient groups.

This authoritative and comprehensive book describes the processes involved of bringing orphan drugs to the global market. The journey from application of an orphan drug designation to a reimbursed market approved drug is long and many obstacles occur during the journey. Chapters offer insights into who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns, such as patients and patient organizations, researchers and treating physicians, industry, regulatory and reimbursement bodies and explain the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients. Patients and researchers, industry representatives and authorities contribute short case studies on the challenges they faced in developing or getting access to orphan drugs.

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