Clinical Value of Molecular Targets and FDA-Approved Genome-Targeted Cancer Therapies.

Importance: The number of new genome-targeted cancer drugs has increased, offering the possibility of personalized therapy, often at a very high cost.

Objective: To assess the validity of molecular targets and therapeutic benefits of US Food and Drug Administration-approved genome-targeted cancer drugs based on the outcomes of their corresponding pivotal clinical trials.

Design And Settings: In this cohort study, all genome-targeted cancer drugs that were FDA-approved between January 1, 2015, and December 31, 2022, were analyzed.

Benefit of adjuvant bisphosphonates in early breast cancer treated with contemporary systemic therapy: A meta-analysis of randomized control trials.

Background: The absolute and relative benefits of adjuvant bisphosphonates on disease-free survival and overall survival in patients receiving contemporary systemic therapy for early breast cancer is uncertain.

Methods: Data from randomized trials of adjuvant bisphosphonates that recruited patients exclusively after 2000 and reported disease free survival and overall survival was utilized. Five-year disease-free survival and overall survival in bisphosphonates and control group along with associated hazard ratios were extracted.

Prognostic value of human leukocyte antigen G expression in solid tumors: a systematic review and meta-analysis.

Introduction: Identification of modulators of the immune response with inhibitory properties that could be susceptible for therapeutic intervention is a key goal in cancer research. An example is the human leukocyte antigen G (HLA-G), a nonclassical major histocompatibility complex (MHC) class I molecule, involved in cancer progression.

Methods: In this article we performed a systematic review and meta-analysis on the association between HLA-G expression and outcome in solid tumors.

Changes in circulating tumor DNA and outcomes in solid tumors treated with immune checkpoint inhibitors: a systematic review.

Background: Quantification of circulating tumor DNA (ctDNA) levels is a reliable prognostic tool in several malignancies. Dynamic changes in ctDNA levels in response to treatment may also provide prognostic information. Here, we explore the value of changes in ctDNA levels in response to immune checkpoint inhibitors (ICIs).

Association between control group therapy and magnitude of clinical benefit of cancer drugs.

Little is known about the impact of control group therapy on clinical benefit scales such as American Society of Clinical Oncology Value Framework (ASCO-VF), European Society for Medical Oncology Magnitude Clinical Benefit Scale (ESMO-MCBS), National Comprehensive Cancer Network (NCCN) Evidence Blocks and ASCO Cancer Research Committee (ASCO-CRC). We searched Drugs@FDA to identify cancer drugs approved between January 2012 and December 2021 based on randomized trials (RCTs). Definition of substantial clinical benefit was based on recommendations for each scale.

Three-year disease-free survival in randomized trials of neoadjuvant chemotherapy and HER2-targeted therapy in breast cancer: A meta-analysis.

Background: Outcomes for breast cancer patients with residual disease (RD) after neoadjuvant chemotherapy (NACT) and HER2-targeted therapy may be better than anticipated leading to a smaller absolute benefit of adjuvant trastuzumab emtansine (T-DM1). Therefore, accurate estimates of 3-year disease-free survival (DFS) can aid in treatment planning.

Methods: We reviewed randomized trials of NACT and HER2-targeted therapy in breast cancer (excluding T-DM1) and calculated mean 3-year DFS weighted by study sample size.

Associations With Definitive Outcomes and Clinical Benefit of Cancer Drugs at the Time of Marketing Approval and in the Postmarketing Period.

Background: Most anticancer drugs are approved by regulatory agencies based on surrogate measures. This article explores the variables associated with overall survival (OS), quality of life (QoL), and substantial clinical benefit among anticancer drugs at the time of approval and in the postmarketing period.

Methods: Anticancer drugs approved by the FDA between January 2006 and December 2015 and with postmarketing follow-up until April 2019 were identified.

Clinical benefit and cost of breakthrough cancer drugs approved by the US Food and Drug Administration.

Background: The clinical benefit and pricing of breakthrough-designated cancer drugs are uncertain. This study compares the magnitude of the clinical benefit and monthly price of new and supplemental breakthrough-designated and non-breakthrough-designated cancer drug approvals.

Methods: A cross-sectional cohort comprised approvals of cancer drugs for solid tumors from July 2012 to December 2017.

Assessment of Frequency and Reporting of Changes in Cancer Trial Design After Initiation of Patient Accrual.

This study matches entries in ClinicalTrials.gov for cancer drug trials at initiation of the trial with later entries and with journal reports of the trials to assess modifications in trial design, external factors associated with such modifications, and transparency in reporting them.

Magnitude of Clinical Benefit of Cancer Drugs Approved by the US Food and Drug Administration Based on Single-Arm Trials.

This study uses the European Society of Medical Oncology Magnitude of Clinical Benefit Scale to evaluate the clinical benefit of anticancer drugs that gained approval by the US Food and Drug Administration based on single-arm rather than randomized clinical trials.

Postmarketing Modifications of Drug Labels for Cancer Drugs Approved by the US Food and Drug Administration Between 2006 and 2016 With and Without Supporting Randomized Controlled Trials.

Purpose Modifications in cancer drug indications, dosing, and related toxicities after Food and Drug Administration approval are common. It is unclear whether drug approval without a supporting randomized controlled trial (RCT) influences the probability of such modifications. Methods We searched the Drugs@FDA Web site for new drug indications for solid tumors approved between January 2006 and December 2016.

Magnitude of Clinical Benefit of Cancer Drugs Approved by the US Food and Drug Administration.

Background: It is uncertain whether drugs approved by the US Food and Drug Administration (FDA) have clinically meaningful benefit as determined by validated scales such as the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS).

Methods: We searched the Drugs@FDA website for applications of anticancer drugs from January 2006 to December 2016. Study characteristics, outcomes, and regulatory pathways were collected from drug labels and reports of registration trials.