I like to publish market research, data and facts to this site -- because they often interest me and it make it easy for me to find the information again in the future with the indexes.
I pulled this data from a GEN article by Gail Dutton that cites a Thomas Reuters study as the original source. The thrust of the article is that making orphan drugs makes good economic sense now. As a reminder, an orphan drug (in US) is one directed to a disease that affects fewer than 200,000 people, in Europe it is to a disease with an incidence lower than 5 in 10,000. The EMA recognizes 8,000 rare diseases and the FDA 6,000.
Since the Orphan Drug Act was passed in 1983, the FDA has approved 350 new drugs to about 200 diseases. Orphans grew at a CAGR of 26% between 2000-2010 versus a 20% CAGR for non-orphans. Orphans account for approximately 22% ($50 B) of total drug sales. It's further interesting, orphan drugs (developed and filed) have a 93% chance of success versus 88% success rate for non-orphans. It pays to be in a niche by yourself. The PV for the two drug types are virtually the same as well $637 M per year and $638 M per year respectively.
Companies (e.g. Shire, Genzyme, Biomarin) with Orphan Drug Business models are successful now and that would seem to be the case for the immediate future too. Here's the list for the Top Selling Orphan drugs:
- Rituximab Oncology
- Ranibizumab Opthamology
- Somatrophin EPR, metabolism
- Lenalidomide Oncology
- Imatinib mesylate Oncology
- Filgrastim Hematology
- Glatiramer acetate MSP
- Rec Factor VIII Hematology
- Bosentan monohydrate Cardiovascular
- Bortezomib Oncology Source: Thomas Reuters
Posted by Bruce Lehr Jan 30th 2013.