Explore the latest in drug development, including evolving methodologic, economic, and regulatory challenges to new drug innovation.
This narrative review examines whether angiotensin II receptor blockade coupled with potentiation of key biological signaling pathways can favorably alter the course of heart failure with reduced ejection fraction.
This cohort study evaluates the characteristics, completion rate, and transparency of study design and results for mandatory pediatric postmarketing studies required under the Pediatric Research Equity Act.
This study characterizes the duration of clinical trials submitted to the US Food and Drug Administration for pediatric drug approvals, with a focus on drugs used for long-term therapy.
This Viewpoint discusses whether the changes in the health care marketplace, the evolution of precision medicine, and the uniqueness of drug development and labeling for cancer diagnoses make the Orphan Drug Act less necessary and less relevant today than at its inception.
This study assesses the benefits of the US Food and Drug Administration’s pediatric exclusivity program in terms of new safety and efficacy data in pediatric populations and the costs to all consumers of extending market exclusivity by 6 months.
This Viewpoint reviews provisions of the US Right to Try Act, which allows patients with life-threatening conditions access to investigational drugs without FDA approval, and calls for clarifications around patient and drug eligibility, reporting requirements, and differences from the FDA’s Expanded Access program to minimize potential harms from the law.
This phenome-wide association study assesses clinical associations between interleukin 6 receptor (IL6R) single-nucleotide polyporphisms and known IL6R drug effects and whether large biobanks and genetics can be used to assess potential beneficial and adverse effects of therapeutic agents with known pathways and related genes.
This review discusses the problems associated with funding for development of drug therapy to treat cancer in children and proposes a financial model to address the challenges.
This study used the Drugs@FDA database to review FDA-approved “breakthrough” therapies from 2012 to 2017 (characterizing the pivotal clinical trials that serve as the basis of FDA approval), and premarket development and review times to assess the strength of evidence supporting breakthrough approvals.
This study describes expanded access and compassionate use programs created through 2017 for experimental drugs and biologics, and the timing and duration of existence of those programs relative to the date of US Food and Drug Administration (FDA) final approval.
This review describes all malignant hematology and oncology accelerated approvals from inception of the US Food and Drug Administration accelerated approval program.
This data analysis compares the analytical validity of US Food and Drug Administration–approved companion diagnostics and laboratory-developed tests for 3 genetic analytes using uniform reference materials from the College of American Pathologists Molecular Oncology Committee.
This Viewpoint discusses concerns about the approval of nusinersen sodium by the US Food and Drug Administration for the treatment of spinal muscular atrophy.
This Viewpoint summarizes the present state of 3,4-methylenedioxymethamphetamine–assisted psychotherapy and key questions associated with US Food and Drug Administration approval.
This Viewpoint examines the data and circumstances behind the US Food and Drug Administration approval of sunitinib as adjuvant treatment for patients with resected renal cell carcinoma who are at a high risk of relapse.
This Viewpoint discusses Right to Try laws and examines the potential effects these laws could have on drug approval and clinical trials.
Customize your JAMA Network experience by selecting one or more topics from the list below.
Create a personal account or sign in to: