Repurposing & collaborative drug development for rare diseases.

• Author: Muthyala, Ramaiah

The label "rare/orphan disease" refers to diseases that affect a small number of people compared to common diseases. In this category, more than 7,000 diseases have been identified so far. 80% of them are of genetic origin and often affect children. A small fraction of these diseases have treatments, but often are very expensive. Due to rapid population migration in the last 3-5 decades, the genetic diseases are no longer confined to any one geographic location, just as infectious diseases such as malaria, TB, etc., are restricted to developing nations and are generally prevalent in poor communities. Rare diseases do not distinguish national boundaries; making them a concern to all nations--rich or poor, all races, religions and social affiliations. Therefore, orphan drug development need not and can't be in any one country. It is a shared responsibility.

Pharmaceutical companies were reluctant to develop drugs for rare diseases under the assumption that they can't make reasonable profit. In recent years this mindset has changed, thanks to the U.S. Orphan Drug Act (ODA), and recent developments in gene technologies. Under the incentives of the ODA, pharmaceutical companies are willing to develop drugs for rare diseases, and this signifies an increasingly important component of the pharmaceutical market; but, they are slow in taking advantage of academic knowledge, new technologies and the power from the advocacy of...