The drugs which are developed for treatment of rare diseases and conditions are referred to as orphan drugs. The rare diseases such as Tourette syndrome, Huntington’s disease, muscular dystrophy, myoclonus, and ALS (Lou Gehrig’s disease) are some of those diseases for treatment of which adequate drugs have not been developed, and hence these diseases are treated by orphan drugs. Orphan drugs are developed by the pharmaceutical companies for public health need, and the sales generated by this drugs are very minimal when compared to normal drugs. The orphan diseases manifest in patient populations representing 6-8% of the global population. Symptoms of some orphan diseases may appear at birth or in childhood including cystic fibrosis, infantile spinal muscular atrophy, familial adenomatous polyposis (FAP), and some others.
Numerous collaborative research programs across North America and Europe is owing to growth of orphan drugs market
Regional segmentation of orphan drugs market by Coherent Market Insights comprises of North America, Latin America, Europe, Asia Pacific, Middle East, and Africa. North America dominates the market globally due to factors such as established regulatory and reimbursement framework and incentives given by U.S. Food and Drug Administration (FDA) for research and development in orphan drugs sector. The various collaborative research programs across United States and Europe provides research data on rare diseases to manufacturers, patients and health experts which gives timely medical care, thus boost the orphan drug market. Whereas the prevalence of genetic diseases and growth of population are some factors which might allow the orphan drugs manufacturers to invest in the Asia Pacific region.
Key players operating the orphan drugs market are Acorda Therapeutics, Actelion Pharmaceuticals Ltd., Amgen, Alexion Pharmaceuticals, Baxter international, Bayer healthcare pharmaceuticals, Biomarin Pharmaceuticals, Novartis, F.Hoffmann-La Roche AG, Johnson and Johnson and many others.