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Recent study results showed larotrectinib was effective in patients with tropomyosin receptor kinase fusion-positive cancer, regardless of the age of the patient or the tumor type. In addition to implications for the treatment of genetic alterations across tumor types, these study results underscore the importance of molecular profiling of tumors, through which patients were identified for the studies, on ensuring precision medicine is used in practice while simultaneously providing a cost-effective tool.

CAR T-cell therapies tisagenlecleucel (Kymriah, Novartis) and axicabtagene ciloleucel (Yescarta, Kite Pharma/Gilead) may come with hefty price tags, but the cost-effectiveness of both therapies fell below or within commonly cited thresholds of $50,000 to $150,000 per quality-adjusted life years, according to a report by the Institute for Clinical and Economic Review.

When a drug becomes first-line, I would like to think that as a clinician I would have access to that, but most importantly that my patients would not have to have a bake sale or take out a second mortgage on their homes to get therapies that are designed specifically for them, said A. Mark Fendrick, MD, director of the Center for Value-Based Insurance Design at the University of Michigan.

Biomarkers of renal dysfunction, endothelial dysfunction, and inflammation were associated with incident heart failure with reduced ejection fraction. By contrast, only natriuretic peptides and urinary albumin to creatinine ratio were associated with heart failure with preserved ejection fraction (HFpEF), a finding that highlights the need for future studies focused on identifying novel biomarkers of the risk of HFpEF, according to a study in JAMA Cardiology.

The FDA has launched a new pilot program that will evaluate whether disclosing certain information within clinical study reports following approval of a new drug application enhances public access to drug approval information, according to a statement by FDA Commissioner Scott Gottlieb, MD. The agency is also working to add a trial's ClinicalTrials.gov identifier number to the agency’s materials for future drug approvals.

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