ERGOMED

Rare Diseases

Publications

Ergomed is committed to the Orphan/Rare Disease sector and is pleased to have co-authored a major work in this area. 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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