Recently, research on a promising drug for a disease that currently affects 1,500 people in the United States annually was halted due the financial reasons, according to Peter Saltonstall of the National Organization for Rare Diseases. The root of these financial reasons stems from the cost of research versus the expected amount of profits that the pharmaceutical company can expect over the long term.
Saltonstall declines to name the particular illness in question, but the identity is hardly important. Such financial issues have long been a difficulty when it comes to spurring interest in research for virtually all rare diseases. Sadly, drug development is a profit-driven business – a fact that has long proven detrimental to “small potatoes” illnesses that affect less than 200,000 people annually. However, it should be noted that combined, more than 7,000 rare diseases affect 20 to 30 million people in the United States each year.
In an effort to improve interest in such research, the United States passed the Orphan Drug Act (ODA) in 1983. The ODA serves to increase incentives for pharmaceutical companies to pursue research within the rare disease sector. Such incentives include federally funded grants, tax credits on costs associated with clinical trials and a 7-year exclusive marketability of any drugs that eventually come to market.
In a lot of ways, the ODA has been very successful. Since 1983, the FDA has approved 357 rare-disease drugs. Additionally, 2,100 products entered the testing pipeline. In comparison, only 10 drugs for rare disease achieved FDA-approval prior to the Act.
However, many experts on the subject claim additional measures need to be taken. The Food and Drug Administration’s Office of Orphan Products Designation is responsible for reviewing studies that may or may not be eligible for orphan status. According to Tom Cote, who heads the department, “[Pharmaceutical companies] frequently come out with press releases saying how important orphan products are to them…[but] they infrequently pass anything substantive over my desk.”
In an effort to further bolster interest in rare diseases, a 2010 appropriations bill is calling for a review process for orphan drugs. The review may result in additional measures that can decrease the cost of cancer research for rare illnesses. For example, initiating statistical models that require fewer patient participants may drop research costs significantly. Of course, the concern is that shallower pools of data may reduce the efficacy of results. Still, Cote is willing to be flexible when it comes to spurring initial research.
As a complement to the proposed revisions, the FDA is already attempting to spur interest by holding on-site workshops that help provide guidance for maximizing current ODA incentives. Hopefully, these and other measures will result in a renewed interest in research for rare diseases.