The political construction of orphan drugs market: between innovation & access to care

About 5000 identified diseases are classed as orphan because of the lack of diagnosis, prevention & treatment. Drug development for these diseases has been limited because of the prohibitive cost of investing in a novel drug with poor market potential. Orphan drugs (OD) is the term quoted to those drugs intended for the treatment of rare diseases & which qualify as drugs scientifically viable but not viable from an economic point. To encourage the development of such drugs, OD legislation came into effect in the European Union (EU) on 2000. These incentives have contributed to the development of many innovative biotechnology products. Indeed ODs give opportunities for pharma firms moving into more niche designations away from mass market at a time, the blockbuster business model have troubles constraining Big Pharma companies to find new markets & sources of profits. The increased availability of OD with their high cost, & long-term administration raises debate surrounding their affordability & cost-effectiveness, & so their public funding by national health services & innovation systems. Markets do not exist a priori but are a social & political construction designed by a political work of companies, patients, experts & State. As a political construction, it is a product of conflicts. While the EU OD act is encompassing its 10th anniversary, we might see the potential emergence of a new conception of control, based on niches and personalized medicine and the use of ODs to sustain and restructure Big Pharma business model.