Why drugs fall short in late-stage trials

Kick-ass article from McKinsey reviewing why drugs fail in P3, based on >650 small molecule trials since 1990.

Notes:

-over all diseases, 42% of leads failed P3, slightly worse than most models anticipate. (The Milken Model anticipates a 33% failure rate at this stage for small molecules.)

-of the failures that could be studied, 50% failed due to efficacy. (Kinda late to find that out, eh?)

-biology still matters in P3 - novel mechanisms of action generated a disproportionate amount (2X) of drop-outs, even in P3.

-quantitative endpoints (biomarkers) improve the likelihood of P3 success.