https://orcid.org/0000-0002-9907-8363
Key Points
The approval rate of new investigational agent from all phase I trials in lymphoma is only 6.1%.
The median duration required from phase I trials to approval has been shortening suggesting recent improvement in drug development process.
While previous studies have examined the drug development and clinical trial success rates in oncology, a comprehensive analysis of the development success rate in lymphoma has not been performed. We thus analyzed lymphoma trials initiated between January 1, 2000, and December 31, 2019 utilizing Trialtrove database and ClinicalTrials.gov. We identified phase I, II, and III trials that included patients with lymphoma and analyzed the transition rate of investigational agents across phases and their FDA approval rates to assess the efficiency and success of the clinical development process. A total of 1,032 phase I trials and 510 phase I/II trials were conducted with a total of 651 distinct agents being evaluated during the study period. There were 1,027 phase II, 20 phase II/III and 140 phase III trials conducted for lymphoma, with 200 agents proceeding from phase I to phase II and 46 agents proceeding from phase II to phase III. The success rate of agent proceeding from phase I to II and phase II to III 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 I 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 I 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.
