Minimal residual disease as an early endpoint for accelerated drug approval in myeloma: A roadmap Review
| Authors: | Landgren, O.; Devlin, S. M. |
| Review Title: | Minimal residual disease as an early endpoint for accelerated drug approval in myeloma: A roadmap |
| Abstract: | SIGNIFICANCE: The acceptance of MRD-negative complete response as an endpoint that is reasonably likely to predict clinical benefit will allow for the design of streamlined clinical trials for accelerated approval, enabling significantly faster patient access to novel therapies. Cooperative efforts were required to obtain and analyze clinical trial data from multiple sponsors and to determine the best approach to analysis with a relatively limited number of available datasets. The process to evaluate MRD as an intermediate endpoint, undertaken jointly by myeloma researchers and industry, with feedback from the FDA, serves as a roadmap for other areas of oncology to develop intermediate endpoints. ©2024 The Authors; Published by the American Association for Cancer Research. |
| Keywords: | antineoplastic agents; clinical trials as topic; drug approval; antineoplastic agent; multiple myeloma; minimal residual disease; neoplasm, residual; bioassay; drug therapy; endpoint determination; clinical trial (topic); humans; human |
| Journal Title: | Blood Cancer Discovery |
| Volume: | 6 |
| Issue: | 1 |
| ISSN: | 2643-3230 |
| Publisher: | American Association for Cancer Research |
| Date Published: | 2025-01-01 |
| Start Page: | 13 |
| End Page: | 22 |
| Language: | English |
| DOI: | 10.1158/2643-3230.Bcd-24-0292 |
| PUBMED: | 39630969 |
| PROVIDER: | scopus |
| PMCID: | PMC11707509 |
| DOI/URL: | |
| Notes: | Review -- Source: Scopus |
