Large molecules enjoy greater success rates than small

Published: 13-Sep-2016

From early phase to approval, small molecules have a 5% chance of success compared with 13% for large

A recent assessment of R&D success rates by KMR Group based on data released by the Pharmaceutical Benchmarking Forum (PBF) reveals that large molecules continue to outperform small molecules, particularly in the early phases of development.

Formed in 1997, the PBF consists of many of the world’s leading pharmaceutical companies, including AbbVie, Allergan, Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, Eli Lilly, Johnson & Johnson, Merck Research Labs, Novartis, Pfizer, Roche, Sanofi and Takeda, and has emerged as the industry’s premier source on R&D performance metrics.

Overall, from start of GLP Tox to approval, small molecules have a 5% chance of success, compared with 13% for large. In preclinical small molecules have a 63% rate of success versus 79% for large molecules; the difference in Phase I is 41% for small molecules vs 52% for large. The only phases where there is a statistically significant variance (p < 0.05) in molecule size is in preclinical and Phase I, but large molecules tend to have higher rates in all phases.

In recent years, Phase I has been relatively stable for both small and large molecules, while Phase II has shown significant improvement for small molecules while large molecules have seen their rates decline. In late development (Phase III and registration) both small and large molecules have improved success rates.

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