A recent review of medical research spending across the public and private sectors, in the US and worldwide, has some interesting findings (JAMA).
Analysis of spending from 1994-2004, and 2004-2012 revealed some notable changes in those decades. While pharmaceutical company spending grew in the first period, it was flat in the last decade. In contrast, spending by medical device and biotechnology companies continued to grow over the entire 1994-2012 spell.
With the number of new drug approvals relatively flat, and concentrated in a few therapeutic areas, the authors note that:
Many new basic discoveries that have probable clinical value are stymied by financial constraints at the critical proof-of-concept stage, where utility in humans is demonstrated.
In industry-funded research, phase 3 studies account for the largest, and fastest-growing component of spending (36% of industry-funded research in 2011, vs 26% in 2004). While phase 1 and 2 spending is also growing, preclinical is declining (from 26 to 22% in the same timeframe). This may reflect the growing complexity of clinical trials, and the size of pivotal trials required to show drug effectiveness in disease sectors where there are existing effective therapies.
One way to address these issues would be to do fewer phase 3 trials and obtain more data at the clinical proof-of-concept phase. While relatively few phase 3 studies fail (~30% according to Paul et al.) these failures are hugely expensive. Much discussion about improving the efficiency of clinical trials centres on adaptive trial design. At Java Clinical Research, we believe that modern laboratory techniques are under-utilised in clinical trials, with much more informative data about drug effects going uncaptured in early-phase clinical studies. Modern approaches using targeted assays, proteomics and genomics allow drug targets to be sampled and analysed with unprecedented precision and breadth, yet these techniques are largely confined to preclinical research.
Learning more about the drug being developed in phase 1 and 2 studies has several advantages. It can be used to demonstrate early clinical proof of concept, which can help with financing, licensing or sale of a compound. It allows translational medicine, confirming basic research results into the clinic and allowing clinical data to inform basic research. Most importantly, it enables more informed decision-making prior to phase 3 trials, taking some of the gamble out of drug development.