Why Drug Repurposing for Small Molecule Drugs is Probably Best Done in Academia



There was a lovely paper in Nature Medicine recently by James McKerrow and colleagues from UCSF on the discovery that auranofin is a good anti-amoeba agent. As the name suggests, auranofin contains gold, and would not make many people's lists of drug like compounds, but hey it's a drug, a real drug!

Anyway, it got me thinking a bit about drug reuse, and the current drug development process is probably configured to make drug repurposing/indication expansion/rescue/whatever a sweet spot for academics, with relatively few incentives for pharma and biotech to actively pursue. I'm sure others have thought of this before, so sorry for being repetitive. I think biologicals will be a different kettle of fish.
  • Current patent life is too short to allow cautious post-approval studies in new indications in most cases, especially for diseases that are chronic and have a long read-out. What are the incentives for a company to perform these trials, when they are in reality probably getting a small bump on revenue at the tail end of the patent life, and building a market for generics. As an aside, I am coming firmly to the view that the patent system for drugs is just wrong. To base the system on reward for 21st Century R&D in healthcare on the length of medieval apprenticeships is just plain mad (see here for a little pointer to background).
  • Regulators are (quite rightly) cautious, and extra hurdles of cost-effectiveness and price negotiations again don't work towards the developing company for a drug actively pursuing new indications - there was a recent case for Aricept, where a recent study recommended use at an earlier stage of Alzheimer's, following many years of regulators turning down data from the developer's encouraging earlier use on the basis of their data. If it is the case that Aricept is helpful to patients with milder disease, for this to happen when the drug becomes generic is a bitter pill (no pun intended). I will check the facts on this and then update if it is way off.
  • Taken further - isn't one strategy for health providers with an eye on costs to resist new indications precisely until the drug is generic. Of course, this allows the building of a good safety record for the drug, across a far more diverse and ill patient population.
  • Academics (pre-clin and clinical) (and of course non-profits) have different drivers with respect to reward, and there is a pretty good alignment between the 'business model' of academic and drug repositioning studies maybe. However, other factors may kick in here, lack of funding, risk aversion, lack of experience and naivety of the process, and there are also sharp contrasts between the personality free operations of pharma (usually) and the personality rich environment of academia (usually).
  • Release of data from the initial compound developers is obviously a good thing - many pairs of eyes looking at the data, preventing wasted effort, etc. but what are the drivers for release of such data? It is expensive to do, and would be seen by some (investors and staff) as throwing money away.