Only company-sponsored, FDA registration-enabling development
programs were included in this analysis. Investigator-sponsored studies and combinations with other
investigational drugs were excluded.
* NDA - New Drug Application / BLA - Biologic License Application.
Citation formats
U.S. development Phase success rates for rare and common disease drugs 2011-2020
This statistic shows the probability of success for new rare disease and high prevalence disease drugs in the U.S. through the various stages of development, from , to **. It was found that while the probability that a new drug for a chronic high prevalence disease makes it from phase I to approval was only *** percent, while it was ** percent for rare disease drugs.
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Biotechnology Innovation Organization, & Pharma Intelligence, & QLS Advisors. (February 26, 2021). Probability of success for new rare disease and high prevalence disease drugs in the U.S. by development phase from 2011 to 2020 [Graph]. In ÌÇÐÄÆƽâ°æ. Retrieved August 03, 2026, from /statistics/597822/drug-development-phases-probability-of-success-rare-high-prevalence-disease-drugs/
Biotechnology Innovation Organization, und Pharma Intelligence, und QLS Advisors. "Probability of success for new rare disease and high prevalence disease drugs in the U.S. by development phase from 2011 to 2020." Chart. February 26, 2021. ÌÇÐÄÆƽâ°æ. Accessed August 03, 2026. /statistics/597822/drug-development-phases-probability-of-success-rare-high-prevalence-disease-drugs/
Biotechnology Innovation Organization, Pharma Intelligence, QLS Advisors. (2021). Probability of success for new rare disease and high prevalence disease drugs in the U.S. by development phase from 2011 to 2020. ÌÇÐÄÆƽâ°æ. ÌÇÐÄÆƽâ°æ Inc.. Accessed: August 03, 2026. /statistics/597822/drug-development-phases-probability-of-success-rare-high-prevalence-disease-drugs/
Biotechnology Innovation Organization, and Pharma Intelligence, and QLS Advisors. "Probability of Success for New Rare Disease and High Prevalence Disease Drugs in The U.S. by Development Phase from 2011 to 2020." ÌÇÐÄÆƽâ°æ, ÌÇÐÄÆƽâ°æ Inc., 26 Feb 2021, /statistics/597822/drug-development-phases-probability-of-success-rare-high-prevalence-disease-drugs/
Biotechnology Innovation Organization & Pharma Intelligence & QLS Advisors, Probability of success for new rare disease and high prevalence disease drugs in the U.S. by development phase from 2011 to 2020 ÌÇÐÄÆƽâ°æ, /statistics/597822/drug-development-phases-probability-of-success-rare-high-prevalence-disease-drugs/ (last visited August 03, 2026)
Probability of success for new rare disease and high prevalence disease drugs in the U.S. by development phase from 2011 to 2020 [Graph], Biotechnology Innovation Organization, & Pharma Intelligence, & QLS Advisors, February 26, 2021. [Online]. Available: /statistics/597822/drug-development-phases-probability-of-success-rare-high-prevalence-disease-drugs/
