Drug regulation articles from across Nature Portfolio

Patients with chronic kidney disease often require complex polypharmacy, require dose adjustments or discontinuation with changes in kidney function, and can be susceptible to the adverse effects of medications. This Review discusses the principles of drug stewardship — that is, the effective, safe and sustainable use of medications — for people with chronic kidney disease.

Administrative patent review procedures are an effective way of correcting erroneously granted biologic patents and may help promote timely drug competition for the benefit of patients and the US healthcare system.

In 2023, the US FDA approved several new cancer drugs and biologic agents, including seven small-molecule inhibitors, four bispecific T cell engagers, two anti-PD-1 antibodies and one cell therapy product. Regulatory focus areas included analyses of biomarker-positive subgroups that drive efficacy, equipoise in randomized controlled trials and a new authority to require confirmatory trials be underway before accelerated approval.

The current drug development pipeline is time-consuming, costly and inefficient. To better model interactions between pharmaceuticals and human physiology and, thus, increase the likelihood of drug success in clinical trials, the effect of pharmacokinetic drug profiles on cellular behaviour should be tested early in drug development.

The FDA approval of perioperative pembrolizumab, an approach that combines neoadjuvant and adjuvant therapy with this agent, for patients with early stage non-small-cell lung cancer (NSCLC) contradicts its own stated standard for combination therapies. Given the large population of patients with early stage NSCLC and the high costs of pembrolizumab, whether the adjuvant component provides incremental benefit is an important question.

Pharmaceutical companies continue to advocate for the use of in vitro models towards the reduction of animal use in drug discovery and development while acknowledging that further advancements are needed to heighten the models’ current state of readiness.