eCliniqua, a publication aimed at those who conduct clinical trials, recently published an interesting look at myriad factors driving increased interest by the bio-pharmaceutical industry in researching and developing orphan drugs http://www.ecliniqua.com/2010/08/16/orphan-drug.html.
Among the factors cited are financial incentives, unmet medical needs, advances in science, and unusual regulatory flexbility. The piece points out that, while activity in this field is picking up, much remains to be done. For instance, GSK's Marc Dunoyer, who heads up our rare diseases unit, told eCliniqua that there are 8,000 rare diseases and as few as 10% have approved treatments. And although the FDA told eCliniqua that nearly 40% of all new drugs now approved by the agency are orphan drugs, reportedly only 359 orphan drugs for 150 rare diseases have been approved in total.
address this, the National Organization for Rare Disorders, academia, industry
and regulators are working to find ways to overcome obstacles that are slowing
efforts to help those who suffer from rare diseases. We're hopeful these efforts will accelerate
the availability of more treatments for rare diseases.