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Comment & Response
August 26, 2021

Randomized Clinical Trials in the Era of Precision Oncology—The Role of End Points, Industry Funding, and Medical Writing Integrity

Author Affiliations
  • 1Medical Oncology, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italia
JAMA Oncol. 2021;7(10):1577. doi:10.1001/jamaoncol.2021.3335

To the Editor In their study, Del Paggio and colleagues1 highlight that most of contemporary oncology randomized clinical trials (RCTs) use putative surrogate end points and are funded by the pharmaceutical industry. The authors are to be commended for their attempt to provide a deeper insight into these interesting but controversial topics.

We believe that some aspects deserve discussion. First, recent studies have suggested that nearly half of anticancer drug approvals are based on end points that have never been evaluated and validated as surrogates for overall survival (OS). For example, only 13.9% of drugs approved by the US Food and Drug Administration between 2008 and 2012 on the basis of progression-based end points reported an improvement in OS in subsequent RCTs.2 Del Paggio et al1 assert that the “marked rise in use of PFS [progression-free survival] is of further concern, particularly given that PFS trials are more likely to be positive.” Nonetheless, despite that the number of clinical settings with validated surrogate end points for OS is altogether limited, there is no doubt that PFS has shown adequate accuracy in several tumors.3 In addition, the multiplication of lines of treatment in a large number of metastatic malignant neoplasms could suggest that controlling disease progression may be as important as achieving an OS benefit. From a purely clinical perspective, although PFS and time to progression have not been shown to be good surrogates for OS in some cancer types, these progression-based end points may provide fundamental information.

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