Model-Based Drug Development: A Rational Approach to Efficiently Accelerate Drug Development

TLDR

The pharmaceutical industry faces high costs, low success rates, and rising investment, with late‑stage failures mainly due to insufficient efficacy. The article aims to show how model‑based drug development (MBDD) can accelerate and optimize compound development strategies. It illustrates MBDD approaches through a series of examples. Published in Clinical Pharmacology & Therapeutics, 2013, 93(6):502–514 (doi:10.1038/clpt.2013.54).

Abstract

The pharmaceutical industry continues to face significant challenges. Very few compounds that enter development reach the marketplace, and the investment required for each success can surpass $1.8 billion. Despite attempts to improve efficiency and increase productivity, total investment continues to rise whereas the output of new medicines declines. With costs increasing exponentially through each development phase, it is failure in phase II and phase III that is most wasteful. In today's development paradigm, late-stage failure is principally a result of insufficient efficacy. This is manifested as either a failure to differentiate sufficiently from placebo (shown for both novel and precedented mechanisms) or a failure to demonstrate sufficient differentiation from existing compounds. Set in this context, this article will discuss the role model-based drug development (MBDD) approaches can and do play in accelerating and optimizing compound development strategies through a series of illustrative examples. Clinical Pharmacology & Therapeutics (2013); 93 6, 502–514. doi:10.1038/clpt.2013.54