Introduction: Prior studies have evaluated the level of evidence behind treatment options listed in the National Comprehensive Cancer Network (NCCN) guidelines, but no study has categorized the life cycle of regimens listed in the NCCN guidelines. We longitudinally assessed the life cycle for each regimen for newly diagnosed multiple myeloma. We track the date of first clinical data, the date of regimen addition to NCCN guidelines, the date phase 3 data (if performed) were reported, and the results of phase 3 trials.
Methods: We systematically examined NCCN guidelines from January 2000 to April 2021. The primary objective of our study was to assess the life cycle of each drug/regimen listed on the NCCN guidelines. We systematically examined the following aspects for each regimen: (1) the inception of prospective clinical data, (2) its inclusion in the NCCN guidelines, (3) the completion of a randomized trial (if done), (4) the presence of an overall survival benefit in such trials, and (5) the removal of a regimen from NCCN guidelines (if done) and its corresponding timeline.
Results: Twenty-one regimens were added across 50 NCCN guideline document iterations during a 22-year period. The median time from when clinical data were first presented to when a regimen was first listed in the guidelines was 15 months. Phase 3 studies were conducted for 17 regimens (80%), with a surrogate endpoint (response rate or progression-free survival) as endpoint for all trials, other than one. The median time from a regimen being included in the NCCN guideline to its phase 3 data publication was 43 months. The primary endpoint was met for 13 trials (81%). No regimen was removed for a phase 3 endpoint not being met. Six regimens (38%) showed overall survival benefit. Five (23%) regimens were removed from NCCN guidelines, with none being due to failure in phase 3 testing.
Conclusion: Myeloma NCCN guidelines remain relevant and current, adding new regimens with promising early-phase data, and removing regimens that become obsolete over time. However, this process is inconsistent and may benefit from standardization.
Keywords: approvals; guidelines; myeloma; regimens.
© The Author(s) 2024. Published by Oxford University Press.