Explore the latest in drug development from The JAMA Network, including evolving methodologic, economic, and regulatory challenges to new drug innovation.
This Viewpoint describes the Priority Antimicrobial Value and Entry (PAVE) award, which has been proposed to address the problem of antimicrobial-resistant infections using limited public funds to shift to value-based payment for new, high-priority antimicrobial drugs.
This Viewpoint discusses the drug development portfolio—a family of trials assessing a drug for different indications or in different combinations with other drugs—and summarizes some of the challenges drug portfolios pose for policy, care, and human protections.
This analysis of US Securities and Exchange Commission filings provides a contemporary estimate of research and development spending to develop 10 new cancer drugs.
This study uses public US Food and Drug Administration (FDA) data to characterize drug trials used to obtain FDA accelerated approval and to describe the existence, timing, and characteristics of postapproval trials mandated by the FDA as part of the accelerated approval decision.
This study describes publication rates and characteristics of postmarket drug studies by application type, regulatory authority, study type, and drug class.
This meta-analysis examines the association of minimal residual disease status with acute lymphoblastic leukemia in children and adults.
This randomized clinical trial evaluates the safety and efficacy of adjuvant girentuximab on disease-free survival and overall survival in patients with localized completely resected high-risk clear cell renal cell carcinoma.
This Viewpoint uses the example of the US FDA’s 2017 approval of desmopressin for treatment of nocturia to discuss the risks of approving a drug for symptoms rather than disease and the ways that the drug approval process is influenced by manufacturers and by overreliance on statistical vs clinical significance.
This study describes withdrawals, boxed warnings, and safety communications affecting pharmaceuticals and biologics approved by the US Food and Drug Administration between 2001 and 2010, and associations between characteristics known at the time of approval and those events.
This Viewpoint argues that publication by the European Medicines Agency of clinical study reports as part of its approval process for commercial drugs sets a standard of transparency that could challenge the reputation of the US Food and Drug Administration as the international standard-bearer in drug and device approval.
This Viewpoint from former US FDA Commissioner Robert Califf discusses existing agency decision support tools for assessing the benefits and risks of medical products under review and the advantages of harmonizing the currently separate frameworks used for evaluating devices and for drugs and biologics.
This Viewpoint discusses potential threats to public health that arise from provisions of the 2016 21st Century Cures Act that allow drug manufacturers to use surrogate measures of safety and that encourage the US Food and Drug Administration to emphasize speed over science in the drug approval process.
This Viewpoint proposes a framework for promoting pharmaceutical innovation while ensuring access and affordability of new drugs on a global scale.
This study examines potential benefits of cancer drugs approved by the FDA on the basis of surrogate endpoints.
This Viewpoint discusses the Pediatric Research Equity Act, a 2003 law authorizing the US Food and Drug Administration to require the study of new therapies for pediatric populations, and proposes improvements to the law as it comes up for reauthorization in 2017.