Beyond business

new approaches to an old problem

Rare Partners represents a new approach aimed at giving a solution to the well-known problem of finding resources for development of drugs with limited economic return on investment (the so-called orphan drugs).

In this respect, we can not speak of a new “business model” (in most cases there is no business), but rather of a new way to approach the problem and a new opportunity of partnership between the non profit world, the scientific community and the pharmaceutical/biotech companies.

There are very few precedents of “non profit pharmaceutical companies”. We took inspiration from the experience of the Institute for One World Health (iOWH, now a drug development program of PATH), which was founded by Victoria Hale in year 2000 and is very active in the field of neglected diseases, strongly supported by Bill & Melinda Gates Foundation. In 2006 iOWH has obtained the marketing approval in India for the use of paromomycin, an off-patent antibiotic agent, in visceral leishmaniasis, a disease that affects each year about 500,000 people and is almost always fatal if left untreated.

We believe that the same successful approach of iOWH can be applied to the rare diseases field. There are already examples of non profit organizations engaged into development of therapies for orphan genetic diseases. In France Genethon is conducting clinical trials for treating gamma-sarcoglycanopathy and Wiskott-Aldrich syndrome. In Italy Telethon has developed, in collaboration with the Istituto Scientifico San Raffaele, a gene therapy protocol for ADA-SCID, which has been already successfully applied to 14 children and has been recently the subject of a strategic alliance between Telethon and GlaxoSmithKline (GSK).