Orphan Drug is a pharmaceutical product aimed at a rare disease. In the world, there are
more than 6000 rare diseases; 80% of them are of genetic origin, and are often chronic and …

This authoritative and comprehensive book makes the reader familiar with the processes of
bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare …

Highlights•Orphan drug development presents major challenges, eg study design and
execution.•New research methods aim to improve study design and analysis and patient …

Conclusion Orphan drug development is an area of pharmaceutical research that requires
significant investment, collaboration and innovation. It presents unique challenges but also …

Abstract© 2018 CSIC. Orphan drugs are intended for the treatment of diseases of low
prevalence, also known as rare diseases. Research and development into new therapies for …

Orphan drug is a drug intended for treatment of rare (orphan) diseases. No single definition
for an orphan disease exists worldwide, but it is generally a disease that affects a small …

An orphan disease refers to a disease with a low prevalence of less than 6.5–10 cases in
10,000 people. The National Organization for Rare Disorders (NORD) and Orphanet are the …

Orphan drugs have been defined as those drugs intended to treat either a rare disease or a
more common disease where the sponsor cannot make any profit. As per the definition US …

This chapter starts off with a concise overview of orphan drug development. It is generally
acknowledged that the introduction of specific orphan drug legislation in various jurisdictions …

In 2000, regulation on orphan medicinal products was adopted in the European Union with
the aim of benefiting patients who suffer from serious, rare conditions for which there is …