Endpoint surrogacy is an important concept in oncology trials. Using a surrogate endpoint like progression-free survival as the primary endpoint-instead of overall survival-would lead to a potential faster drug approval and therefore more cancer patients with an earlier opportunity to receive the newly approved drugs.
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http://dx.doi.org/10.1038/s41416-020-0896-5 | DOI Listing |
J Cancer Policy
December 2024
Department of Epidemiology and Biostatistics, University of California San Francisco, San Francisco, CA, USA. Electronic address:
Background: In April 2024, the Oncology Drug Advisory Committee (ODAC) voted to approve minimal residual disease (MRD) as a new regulatory endpoint for multiple myeloma (MM) despite its poor trial-level surrogacy. This is expected to result in faster MM drug approvals, a potential boon for the pharmaceutical companies that make them. This study investigates the prevalence of financial conflicts of interest (FCOIs) with these companies among United States (US)-based physician speakers at the meeting.
View Article and Find Full Text PDFJ Endovasc Ther
December 2024
Medical Research and Biometrics Center, National Clinical Research Center for Cardiovascular Diseases, National Center for Cardiovascular Diseases, Fuwai Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China.
Purpose: Most clinical trials of drug-coated balloon (DCB) for the treatment of femoropopliteal artery stenosis chose 12-month primary patency rate (PPR) or 6-month late lumen loss (LLL) as the primary endpoint. It is still debatable whether 6-month LLL can be served as an appropriate surrogate endpoint for 12-month PPR. This study aimed to identify whether LLL can serve as an appropriate surrogate endpoint in peripheral DCB clinical trials, and shed light on the selection of primary outcome for subsequent confirmatory clinical trials of DCB in the treatment of femoropopliteal artery stenosis.
View Article and Find Full Text PDFMed Decis Making
January 2025
Biostatistics Research Group, Department of Population Health Sciences, University of Leicester, Leicester, UK.
Background: Multi-indication cancer drugs receive licensing extensions to include additional indications, as trial evidence on treatment effectiveness accumulates. We investigate how sharing information across indications can strengthen the inferences supporting health technology assessment (HTA).
Methods: We applied meta-analytic methods to randomized trial data on bevacizumab, to share information across oncology indications on the treatment effect on overall survival (OS) or progression-free survival (PFS) and on the surrogate relationship between effects on PFS and OS.
Background: Establishing surrogate endpoints for overall survival (OS) may expedite assessment of new therapies in esophageal cancer (EC) and gastroesophageal junction cancer (GEJC). This study aimed to evaluate disease-free survival (DFS) as a surrogate endpoint for OS.
Methods: Patients from the Surveillance, Epidemiology, and End Results (SEER)-Medicare database aged ≥66 years with resection after primary diagnosis of stage 2 or 3 EC/GEJC between 2009 and 2017 were analyzed (N = 925; median follow-up 26.
ESMO Open
November 2024
Department of Oncology, Queen's University, Kingston, Canada; Department of Public Health Sciences, Queen's University, Kingston, Canada; Division of Cancer Care and Epidemiology, Queen's University, Kingston, Canada; Queen's Global Oncology Program, Kingston, Canada. Electronic address:
Background: The importance of surrogate endpoints, magnitude of clinical benefit of cancer drugs, and their prices have often been debated in the oncology world. No study, however, has systemically explored oncologists' perception regarding these issues.
Methods: We conducted a mixed-methods study including in-depth qualitative interviews of medical oncologists prescribing cancer drug therapy in India.
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