Conventional wisdom about orphan drugs turned on its head

Conventional wisdom has it that pharmaceutical companies won't invest in drugs for rare, or 'orphan' diseases because it's just not profitable.  Many diseases are rare and the cause unknown, but there are some rare diseases for which enough is known that, in principle at least, one can envision developing a targeted medicinal approach.  Many of these are also known to be, or seem to be genetic, and that at least leads to the idea of a targetable problem for Pharma.  The costs of the research can be so high that pharmaceutical companies just don't want to wade in, or this has been the usual view. But, in an unusual twist on the story, it turns out there's some reason for optimism for those with one of the 8000 or so such diseases, with 250 more discovered every year.

Rare diseases are defined by the National Institutes of Health Office of Rare Disease Research as those that affect fewer than 200,000 people in the US.  Some, far fewer than 200,000.  A new study, reported in Medical Marketing and Media, and published in the July Drug Discovery Today, suggests that in fact there is money to be made in rare disease pharmaceuticals.  And that's potentially good news for a lot of people.  

The government offers an "Orphan Drug Designation" program, which allows tax credits for what can be prohibitively expensive RandD for drugs for rare diseases.  In addition it offers grants to cover costs, waived FDA fees, which can be high, and the promise of seven years of exclusivity.  Granted, there's a lot that's unsavory about the workings of the pharmaceutical industry, but patents, of course, are the prime protection that pharmaceuticals have for recouping RandD costs and eventually profiting from a drug.   And, given that most orphan drugs are novel, "biosimilars" are not as much of a threat when the patent expires as they are for more popular compounds such as Viagra or Lipitor, say.

And, the MMM piece points out that marketing to a small target population is much cheaper than the population at large, clinical trials can be less expensive, and in fact the orphan drug market grew at a higher rate than the drug market in general.  

There's a lot to be said against the pharmaceutical industry, but that's not our point today.  Of course bottom line is the primary consideration when it comes to developing new drugs, but if it really does become financially attractive to develop new drugs for rare diseases, this is good news for a lot of people.  The scientific challenges are still real, but at least there's motivation to take them on.  

Now to hope that it becomes profitable to invest in research into drugs for another and much larger group of forgotten people, those with "neglected tropical diseases."