are diseases affect close to 440 million people globally, yet around 95% lack approved therapies. Families face diagnostic odysseys lasting years, often enduring worsening symptoms while waiting for diagnosis or treatment. In India, over 70 million people may be affected, but most remain unrecognized in formal registries and policy priorities.

iostatisticians are misperceived as only appearing at the end of a clinical trial to analyze the collected data and present the results. Yet nothing could be further from the truth. In practice, these statistical analysts are active participants from the earliest stages of research planning until long after approval.

ou could hear the “Aha!” moment in the room. The conference delegates and members of the panel had come to a realization. One of the reasons why the implementation of Risk-Based Quality Management (RBQM) is so difficult: everyone does it differently. The international guidance in ICH E6 (R3) is very high level, and organizations have taken many varied approaches.

or years, clinical research has wrestled with the fundamental challenge of ensuring that the subjects enrolled in studies reflect the patients who will ultimately use the therapies. Diversity, equity, and inclusion (DEI) initiatives reflected in FDA guidance, NIH grantmaking, and/or sponsor-led action plans were meant to push the industry towards that ideal. However, the evolving legal and political landscape in the United States has pushed DEI to effectively be on life support.

n the rapidly evolving global pharmaceutical research and development landscape, China is transitioning from harmonization with international standards to becoming a vital innovation engine.

hina and Japan, two large economies in Asia, have shared a long and intricate history of cultural exchange and mutual influence dating back to ancient times.

s part of the revision to the UK clinical trials legislation coming into force on 28 April 2026, the Medicines and Healthcare products Regulatory Agency (MHRA) undertook a review of requirements and timelines associated with safety management and reporting. As a regulator, we support a pragmatic and risk-proportionate approach, and the revised legislation encourages sponsors to embed risk proportionality within their operations. In today’s context of evolving clinical trials, technology, and innovative products, this revision places emphasis on the sponsor to review safety signals identified during product development and transparently describe to the regulator how these are being managed in the context of their ongoing clinical trials.

he UK has recently undertaken the most significant overhaul of its clinical trials regulations in two decades. Developed by the Medicines and Healthcare Products Regulatory Agency (MHRA) in partnership with the Health Research Authority (HRA), the updated regulations are designed to protect trial participants, reduce unnecessary burdens on researchers, and facilitate the conduct of high-quality, trusted research in the UK. The Medicines for Human Use (Clinical Trials) (Amendment) Regulations 2025 will come into force on 28 April 2026 across the whole UK.

ngoing challenges in US prescription drug pricing have led many pharmaceutical companies to create financial support or assistance programs to help patients offset the price of their prescription drugs. One recent article reports that there are currently 2,000 active patient assistance programs (PAPs) offered by approximately 500 companies. But this author has observed that such programs, designed to expand patient access to safe and efficacious drugs, can unintentionally complicate downstream programs designed to protect patient safety.