Orphan Drug Act

As part of the exploration that is the subject of this blog, I thought it imperative to explore the history of the industry; this has started with the reading of Shaping The Industrial Century - the remarkable story of the evolution of the modern chemical and pharmaceutical industries. Seems like a fairly unbiased account, right?

As I cracked it open and waded through the overview, there was a mention of the United States Orphan Drug Act (ODA) enacted in 1983, which is credited with a subsequent explosion of drug discovery. I had to take a further look...

An "orphan disease" is defined as one that affect less than 200,000 patients (Side note: I wonder if this number still has relevance given the population increase since 1983). The benefits of developing a drug that treats an orphan disease include tax incentives, enhanced patent protection, and subsidies for clinical trials, all in an effort to encourage pharmaceutical companies to apply resources to that development. Not only do we get new drugs for diseases that were previously considered untreatable (many in this category are for cancer treatment), but technological advances in this arena are good for R&D in general.

However, one interesting piece of information I noticed in an article on this topic from the Health Policy journal was that many of these orphan drugs do indeed become blockbusters! In fact "in 2006, a quarter of products which reached blockbuster status that year had one or more orphan designations" and one of them was Provigil (see previous post on Shift Work Disorder). Also of note, R&D for rare diseases has been stifled (as indicated in the number of new drug applications) in years when there has been political discussion around amending the ODA. Who wants to invest money if the incentives mentioned won't be available? It's great that legislation has the ability to spur such scientific breakthroughs, but it's no surprise that there are unintended results.

Hmm, just some food for thought :/