Clinical trial success rate in lymphoma: fate of trials and agents from 2000 to 2019

Although previous studies have examined the drug development and clinical trial success rates in oncology, a comprehensive analysis of development success rate in lymphoma has not been performed. Therefore, we analyzed lymphoma trials initiated between 1 January 2000 and 31 December 2019, using the Trialtrove database and ClinicalTrials.gov. We identified phase 1, 2, and 3 trials that included patients with lymphoma and analyzed the transition rate of investigational agents across phases and their US Food and Drug Administration (FDA) approval rates to assess the efficiency and success of the clinical development process. A total of 1032 phase 1 trials and 510 phase 1/2 trials were conducted, with a total of 651 distinct agents being evaluated during the study period. There were 1027 phase 2, 20 phase 2/3, and 140 phase 3 trials conducted for lymphoma, with 200 agents proceeding from phase 1 to 2 and 46 agents proceeding from phase 2 to 3. The success rates of agents proceeding from phase 1 to 2 and phase 2 to 3 were 30.7% and 23.0%, respectively. A total of 40 agents reached FDA approval, with an overall approval rate of 6.1%. The average time from initiation of the first phase 1 trial to approval for lymphoma treatment was 7.9 years. Our study characterizes the clinical development landscape and timeline of novel therapies for lymphoma. Low approval rate from phase 1 trials in lymphoma suggests the need for improvement in trial design and in prediction of clinical response through preclinical studies to improve the likelihood of success.