From a report I downloaded last month: Clinical Development Success Rates 2006-2015
"Executive Summary This is the largest study of clinical drug development success rates to date. Over the last decade, 2006-2015, a total of 9,985 clinical and regulatory phase transitions were recorded and analyzed from 7,455 development programs, across 1,103 companies in the Biomedtracker database. Phase transitions occur when a drug candidate advances into the next phase of development or is suspended by the sponsor. By calculating the number of programs progressing to the next phase vs. the total number progressing and suspended, we assessed the success rate at each of the four phases of development: Phase I, II, III, and regulatory filing. Having phase-by-phase data in hand, we then compared groups of diseases, drug modalities and other attributes to generate the most comprehensive analysis yet of biopharmaceutical R&D success.
This work was made possible due to the years of clinical program monitoring and data entry by Informa’s Biomedtracker service. BIO has long partnered with Biomedtracker to calculate success rates based on this data. More recently, BIO and Biomedtracker partnered with Amplion, the inventors of BiomarkerBase, to analyze the effects of biomarkers in clinical trial success.
Key takeaways:
• The overall likelihood of approval (LOA) from Phase I for all developmental candidates was 9.6%, and 11.9% for all indications outside of Oncology.
• Rare disease programs and programs that utilized selection biomarkers had higher success rates at each phase of development vs. the overall dataset.
• Chronic diseases with high populations had lower LOA from Phase I vs. the overall dataset. • Of the 14 major disease areas, Hematology had the highest LOA from Phase I (26.1%) and Oncology had the lowest (5.1%).
• Sub-indication analysis within Oncology revealed hematological cancers had 2x higher LOA from Phase I than solid tumors.
• Oncology drugs had a 2x higher rate of first cycle approval than Psychiatric drugs, which had the lowest percent of first-cycle review approvals. Oncology drugs were also approved the fastest of all 14 disease areas.
• Phase II clinical programs continue to experience the lowest success rate of the four development phases, with only 30.7% of developmental candidates advancing to Phase III."
Later:
"Phase Transition Success and Likelihood of Approval (LOA) - Overall Consistent with previous studies of drug development phase transition success rates, we found Phase II success rates to be far lower than any other phase.1 Phase I and III rates were substantially higher than Phase II, with Phase I slightly higher than Phase III. The highest success rate of the four development phases was the NDA/BLA filing phase.
The Phase I transition success rate was 63.2% (n=3,582). As this Phase is typically conducted for safety testing and is not dependent on efficacy results for candidates to advance, it is common for this phase to have the highest success rate among the clinical phases across most categories analyzed in this report. Phase I success rates may also benefit from delayed reporting bias, as some larger companies may not deem failed Phase I programs as material and thereby not report them in the public domain. The Phase II transition success rate (30.7%, n=3,862) was substantially lower than Phase I, and the lowest of the four phases studied. As this is generally the first stage where proof-of-concept is deliberately tested in human subjects, Phase II consistently had the lowest success rate of all phases. This is also the point in development where industry must decide whether to pursue the large, expensive Phase III studies and may decide to terminate development for multiple reasons including commercial viability. The second-lowest phase transition success rate was found in Phase III (58.1%, n=1,491). This is significant as most company-sponsored Phase III trials are the longest and most expensive trials to conduct.
The probability of FDA approval after submitting a New Drug Application (NDA) or Biologic License Application (BLA), taking into account re-submissions, was 85.3% (n=1,050). Multiplying these individual phase components to obtain the compound probability of progressing from Phase I to U.S. FDA approval (LOA) reveals that only 9.6% (n=9,985) of drug development programs successfully make it to market (Figure 1).
63.2% Phase I to Phase II
30.7% Phase II to Phase III
58.1% Phase III to NDA/BLA
85.3% NDA/BLA to Approval
9.6% Phase I to Approval
...(did some reformatting here as numbers were in a table)
Probablity of Success
All Diseases, All Modalities
Figure 1. Phase transition success rates and LOA from Phase I for all diseases, all modalities.
BIO Industry Analysis | 7"
"Executive Summary This is the largest study of clinical drug development success rates to date. Over the last decade, 2006-2015, a total of 9,985 clinical and regulatory phase transitions were recorded and analyzed from 7,455 development programs, across 1,103 companies in the Biomedtracker database. Phase transitions occur when a drug candidate advances into the next phase of development or is suspended by the sponsor. By calculating the number of programs progressing to the next phase vs. the total number progressing and suspended, we assessed the success rate at each of the four phases of development: Phase I, II, III, and regulatory filing. Having phase-by-phase data in hand, we then compared groups of diseases, drug modalities and other attributes to generate the most comprehensive analysis yet of biopharmaceutical R&D success.
This work was made possible due to the years of clinical program monitoring and data entry by Informa’s Biomedtracker service. BIO has long partnered with Biomedtracker to calculate success rates based on this data. More recently, BIO and Biomedtracker partnered with Amplion, the inventors of BiomarkerBase, to analyze the effects of biomarkers in clinical trial success.
Key takeaways:
• The overall likelihood of approval (LOA) from Phase I for all developmental candidates was 9.6%, and 11.9% for all indications outside of Oncology.
• Rare disease programs and programs that utilized selection biomarkers had higher success rates at each phase of development vs. the overall dataset.
• Chronic diseases with high populations had lower LOA from Phase I vs. the overall dataset. • Of the 14 major disease areas, Hematology had the highest LOA from Phase I (26.1%) and Oncology had the lowest (5.1%).
• Sub-indication analysis within Oncology revealed hematological cancers had 2x higher LOA from Phase I than solid tumors.
• Oncology drugs had a 2x higher rate of first cycle approval than Psychiatric drugs, which had the lowest percent of first-cycle review approvals. Oncology drugs were also approved the fastest of all 14 disease areas.
• Phase II clinical programs continue to experience the lowest success rate of the four development phases, with only 30.7% of developmental candidates advancing to Phase III."
Later:
"Phase Transition Success and Likelihood of Approval (LOA) - Overall Consistent with previous studies of drug development phase transition success rates, we found Phase II success rates to be far lower than any other phase.1 Phase I and III rates were substantially higher than Phase II, with Phase I slightly higher than Phase III. The highest success rate of the four development phases was the NDA/BLA filing phase.
The Phase I transition success rate was 63.2% (n=3,582). As this Phase is typically conducted for safety testing and is not dependent on efficacy results for candidates to advance, it is common for this phase to have the highest success rate among the clinical phases across most categories analyzed in this report. Phase I success rates may also benefit from delayed reporting bias, as some larger companies may not deem failed Phase I programs as material and thereby not report them in the public domain. The Phase II transition success rate (30.7%, n=3,862) was substantially lower than Phase I, and the lowest of the four phases studied. As this is generally the first stage where proof-of-concept is deliberately tested in human subjects, Phase II consistently had the lowest success rate of all phases. This is also the point in development where industry must decide whether to pursue the large, expensive Phase III studies and may decide to terminate development for multiple reasons including commercial viability. The second-lowest phase transition success rate was found in Phase III (58.1%, n=1,491). This is significant as most company-sponsored Phase III trials are the longest and most expensive trials to conduct.
The probability of FDA approval after submitting a New Drug Application (NDA) or Biologic License Application (BLA), taking into account re-submissions, was 85.3% (n=1,050). Multiplying these individual phase components to obtain the compound probability of progressing from Phase I to U.S. FDA approval (LOA) reveals that only 9.6% (n=9,985) of drug development programs successfully make it to market (Figure 1).
63.2% Phase I to Phase II
30.7% Phase II to Phase III
58.1% Phase III to NDA/BLA
85.3% NDA/BLA to Approval
9.6% Phase I to Approval
...(did some reformatting here as numbers were in a table)
Probablity of Success
All Diseases, All Modalities
Figure 1. Phase transition success rates and LOA from Phase I for all diseases, all modalities.
BIO Industry Analysis | 7"
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