EVALUATING ENDPOINTS AND CHANGING TRENDS IN ADVANCED STAGES OF CANCER RELATED CLINICAL TRIALS
Author(s)
Kim H1, Lee J1, Song SY2, Kim E3
1Department of Pharmaceutical Industry Management, The Graduate School, Chung-Ang University, Seoul, Korea, Republic of (South), 2Chung-Ang University College of Pharmacy, Seoul, Korea, Republic of (South), 3Evidence-Based Research and Clinical Research Lab., Department of Health, Social and Clinical Pharmacy, College of Pharmacy, Chung-Ang University, Seoul, Korea, Republic of (South)
OBJECTIVES: Properly selected endpoints are essential for clinical trials aimed at developing new drugs. This study uses ClinicalTrail.gov registry as data source in evaluating the use of endpoints and changing trends in phase Ⅱ and phase Ⅲ trials in advanced stages of breast cancer. METHODS: We searched phase Ⅱ and phase Ⅲ clinical trials of advanced breast cancer registered in ClinilcalTrial.gov registry between October 2000 to September 2012, which was divided into two study periods (cohort A: October 2000 to September 2007 and cohort B: October 2007 to September 2012). The assessment of primary and secondary endpoints was conducted by two independent reviewers. RESULTS: In 398 phase II trials, there was a change in the most commonly used primary outcome measure from objective response rate in cohort A (60.6 %) to progression-free survival in cohort B (40.7 %). The trend was statistically significant with a decline in objective response rate selection (P < 0.001) and an increase in progression-free survival selection (P < 0.001). For 120 phase III trials, progression-free survival was the most frequently used primary outcome in both cohort groups (cohort A: 35.9 %; cohort B: 66.1 %; P < 0.001). CONCLUSIONS: This was the first study to assess endpoint selection in advanced breast cancer clinical trials over a decade. For both phase II and III trials, progression-free survival was the most frequently used primary outcome in general. However, in phase II trials studies, increasing trend in progression-free survival use in substitution of objective response rate was observed. As selection of proper endpoints is important for the success of clinical trials, changing trends should be considered when deciding upon primary and secondary outcome measures for the assessment of drug efficacy and safety.
Conference/Value in Health Info
2017-11, ISPOR Europe 2017, Glasgow, Scotland
Value in Health, Vol. 20, No. 9 (October 2017)
Code
PRM4
Topic
Clinical Outcomes
Topic Subcategory
Clinical Outcomes Assessment
Disease
Oncology, Reproductive and Sexual Health