Analysis report of more than 10,000 drug clinical projects over ten years: the success rate of clinical development is only 7.9%
2021-06-30 14:37:44
Recently, a new report on the success rate of drug clinical development shows that in the past ten years, the average success rate of drug development projects from Phase 1 clinical trials to FDA approval for marketing is 7.9%, and the average time required is 10.5 years. According to public information, the report was jointly released by BIO (Biotechnology Innovation Organization), one of the world’s largest biotechnology industry organizations, Informa Pharma Intelligence, and QLS. The research data covers 9,704 drugs in the past ten years (2011-2020). 12,728 projects in transition occurred in clinical development projects involving 1,779 companies.
Phase 2 clinical transformation has the lowest success rate, and the highest success rate from application submission to approval
According to the report, drug development transitions (phase transitions) are defined as withdrawals from the clinical trials in which the drug is located: for example, moving from Phase 1 to Phase 2, or on hold after completing Phase 1. By calculating the number of projects in the next phase / (the number of projects in the next phase of the number of projects on hold), the analysts evaluated the conversion success rate of the four stages of drug development. The analysis found that the success rate from Phase 1 to Phase 2 was 52.0% (n=4414), which was relatively high. One of the reasons was that the safety of the drug was mainly tested in this phase and was not dependent on the effectiveness of the candidate drug. Consistent with previous studies on translational success rates in drug development stages, the success rate from Phase 2 to Phase 3 was the lowest among the four phases, at 28.9% (n=4933), because Phase 2 was not only the first in humans Conducting proof-of-concept trials is also a critical node in deciding whether to continue clinical trials. The success rate from Phase 3 to submission of New Drug Application (NDA)/Biologics License Application (BLA) was 57.8% (n=1928). Among the four development stages, the highest translation success rate was from NDA/BLA submission to FDA approval, at 90.6% (n=1453).
The researchers also analyzed the translational success rates of drugs in different disease areas at various stages of development. In total, these clinical trials cover 21 major disease areas and involve 623 indications. The report analyzes 14 major disease areas including: Allergy, Autoimmunity, Cardiovascular, Endocrinology, Gastroenterology (non-IBD), Hematology, Infectious Diseases, Metabolism, Neurology, Oncology, Ophthalmology, Psychiatry, Respiratory and Urology division.
Phase 1 clinical translational success rates in different disease areas ranged from 40.9% to 71.6%, with an average success rate of 52.0% across all indications. Among them, ophthalmology and hematology were both well above the average, 71.6% (n=88) and 69.6% (n=92), respectively. Phase 2 had the lowest translational success rates in each disease area, ranging from 15.0% (urology, n=40) to 48.1% (hematology, n=106). Only hematology and metabolism (45.0%) achieved a conversion success rate close to 1/2. Similar to the phase 1 translation success rate, urology (15.0%), cardiovascular (21.0%) and oncology (24.6%) also had lower translation success rates in phase 2. It is worth mentioning in the field of gastroenterology, which has a lower than average translational success rate in Phase 1 clinical trials, but achieved an above-average translational success rate (34.2%) in Phase 2 clinical trials.
The average translational success rate of Phase 3 clinical trials across all disease areas was 57.8%, with the lowest rate in the oncology area (47.5%, n=495). The translational success rates of ophthalmic drugs were all above average in the early stages of development, but below average in Phase 3 clinical trials (51.2%, n=82). Conversely, urology had the lowest translation success rate in Phase 1 and Phase 2, but was second only to Hematology in Phase 3, at 69.2% (n=13). It is worth noting that the disease areas that are lower than the average success rate of phase 3 clinical translation have a relatively large patient base.
The average success rate of drug development projects is 7.9%, and the success rate of CAR-T therapy and RNAi therapy is higher
In the report, the researchers used the probability of obtaining FDA approval (LOA) from phase 1 clinical trials as one of the key indicators to evaluate the success rate of drug clinical development. The LOA success rate is the product of the success rates for all four stages of clinical development. For example, if each stage has a 50% success rate, the success rate from Phase 1 clinical stage to approval would be 0.5 x 0.5 x 0.5 x 0.5 = 6.25%.
Through computational analysis, the researchers found that of these drug development programs (n=12,728), only 7.9% successfully progressed from Phase 1 clinical trials to the final FDA-approved marketing stage. However, this success rate also varies widely by disease area. Among them, the field of hematology has the highest development success rate of 23.9% (n=352), which is almost 7 times that of the field of urology (3.6%, n=88). In addition, the success rate of drug development in the five disease areas of infectious diseases (13.2%), ophthalmology (11.9%), autoimmunity (10.7%), allergy (10.3%), and gastroenterology (8.3%) is also higher than 7.9% average of. The success rates of drug development in the fields of oncology, neurology, cardiovascular and other fields are all below average.
Of the 12,728 projects counted by the researchers, oncology drug development projects accounted for 33%. The success rate of drug development in the field of oncology is only 5.3% (n=4179), which is much lower than the success rate of drug development in non-oncology indications (9.3%, n=8,549). Low success rates in the field of oncology are also an important factor contributing to the lower average success rates in overall drug development. It is worth mentioning that the development success rate of tumor immunotherapy is 12.4%, which is higher than the average development success rate of all tumor drugs (5.3%). An analysis of the drug development success rates for rare diseases and high-incidence chronic diseases (with tumor indications excluded from the statistics) found that the drug development success rate for rare diseases was 17.0%, almost three times that of chronic diseases with high prevalence (5.9%). .
The translation success rate of different types of drug clinical trials also varies greatly. The analysis found that the development success rate of generic drugs (14.7%, n=2,161) was twice that of innovative drugs (6.8%, n=10,527). In the field of new drug development, the probability of new molecular entities (NMEs, mainly small molecules) from phase 1 clinical to approval was 5.7% (n=6803), which was lower than that of biological products (9.1%, n=3412) or vaccines (9.7%) %, n=312), of which the development success rates of CAR-T therapy and RNAi therapy were higher, 17.3% (n=67) and 13.5% (n=70), respectively. Among generic drugs, biosimilars had a higher development success rate of 32.3% (n=277).
In addition, the researchers analyzed the impact of biomarkers on the success rate of drug development. The analysis found that drug development programs using the patient’s preselected biomarkers had twice the success rate (15.9%) as programs that did not use the biomarkers (7.6%).
The average time from phase 1 clinical to market approval is 10.5 years
The researchers analyzed 6,151 successful translational drug development projects from 2011-2020 and found that it took an average of 10.5 years for a drug to go from phase 1 clinical to final regulatory approval. Among them, it takes an average of 2.3 years from Phase 1 to Phase 2, 3.6 years from Phase 2 to Phase 3, 3.3 years from Phase 3 to submission of a marketing application, and an average of 1.3 years from submission of a marketing application to approval.
Of the 14 major disease areas analyzed by the researchers, allergy had the shortest drug development time at 9.2 years, and other disease areas with less than 10 years of development time included metabolism, infectious diseases, and ophthalmology. The drug development time in the three disease areas of urology, cardiovascular and neurological diseases is relatively long, all of which exceed 11 years, of which the urology field has reached 12.2 years.
The average time for drug development in oncology is 10.3 years. From the perspective of different development stages, the longest clinical transition time for Phase 1 is oncology and urology (both 2.7 years), the longest clinical transition time for Phase 2 is in the field of urology (5.0 years), and the duration of Phase 3 clinical projects The longest was in the cardiovascular field (4.2 years). It is worth mentioning that the time from the submission of NDA/BLA to the approval of drugs in the field of oncology is the shortest, which is 0.8 months, which is also the only field in which the regulatory approval time is less than 1 year among the 14 disease fields.