s we approach the end of 2025 and begin to look forward to 2026, I want to share a quick update about Global Forum’s publication schedule. Beginning this year, we’ll be taking a short end-of-year break — our way of giving our volunteer editors and contributors some time to rest, recharge, and reflect on the year’s accomplishments — and we will not publish a December issue.

ecent advances in drug discovery, development, manufacturing, and safety monitoring technologies, including the adoption of automation, robotics, simulation, and other digital capabilities, have allowed sponsors and manufacturers to reduce sources of error, optimize resources, and reduce patient risk.

here is a paradox keeping the rare disease research community awake at night. The challenge is the required statistically significant evidence of efficacy.

This mathematical impossibility can block therapeutic development for thousands of rare diseases. Conventional power calculations, designed for diseases with large populations, fail when patient numbers drop below critical thresholds.

The result? People with ultrarare conditions face a cruel challenge: too few of them exist to prove that a treatment works, even when it does.

Fortunately, a quiet revolution is underway. Artificial intelligence and advanced statistical methods are rewriting the rules of what’s possible with small sample sizes. Regulators are paying attention.

ancer is not a single disease confined to one tissue. It’s a dynamic, systemwide disorder that exploits the body’s own biological networks. This article explores how cancer testing has evolved from early animal models to today’s advanced in vitro and computational methods, and why understanding the whole organism remains essential for developing effective therapies.

ell and gene therapies (CGTs) continue to demonstrate extraordinary potential to transform treatments, particularly for rare diseases and cancer. Yet despite major scientific advances, the field faces persistent barriers that slow progress from discovery to patient access. Manufacturing remains complex and costly, regulatory expectations vary widely across regions, and uncertainty around reimbursement and long-term sustainability continues to deter investment and delay adoption.

se of Direct-to-Patient (DTP) shipments of investigational products and study treatments has received increasing attention since the COVID-19 pandemic began in 2020. DTP shipments are generally defined as any method of investigational treatment delivery directly to a trial participant’s home. Two strategies are common: Site-to-Patient (STP) shipments and Warehouse-to-Patient (WTP) shipments. The STP method involves shipping investigational treatment from the investigative site to the patient’s home—excluding one-time shipments that are not part of the study design (i.e., one-off or ad hoc shipments made to accommodate a patient’s specific limitations, but not included in the study design). The WTP approach is the shipment of investigational treatment from a warehouse, depot, or central pharmacy directly to the patient’s home.

n conjunction with this Tufts CSDD survey, DIA asked the Patient Partner Program Fellows from the last several DIA Global Annual Meetings if they had any experience with DTP components in a clinical trial.

nput from patients, as users of medicines, can inform medicines development, enhance regulatory decision making, and result in more patient-relevant outcomes. Clinical trial data for example typically constitute snapshots in time. Patient experience data collection helps to paint a clearer picture of how patients and families feel and function as a result of a clinical trial or lived experience with a disease. In a recent draft reflection paper, the European Medicines Agency (EMA) acknowledges the usefulness of such Patient Experience Data (PED) gained directly from patients. The paper is now open for public comment until January 31, 2026. It is now time to read and react.

ow do we move from “checking boxes” to truly meaningful patient involvement in clinical trials? From emerging global trends through redesigning the consent process to creative solutions to real-world barriers, meaningful collaboration across the ecosystem is key—and the reason why we’re only at the beginning of what’s possible. Several recent initiatives are aiming to bring these possibilities into practice in the EU.

n August 2025, during an official mission of the Brazilian government to Mexico, representatives of the Brazilian regulatory authority ANVISA signed a new Memorandum of Understanding (MOU) with the Mexican Agency COFEPRIS. A MOU is a formal, nonbinding agreement that details the mutual goals, responsibilities, and intentions of two or more parties, as a framework for collaboration. In this interview, Ana Carolina Marino, head of the International Relations Office of ANVISA, provides more details on this bilateral agreement.

verybody should benefit from the advancements in care and treatments made through research with no one, or no group, unfairly excluded. This can only happen if we understand how interventions work for different groups of people. To gather this evidence, research must be representative of the people who stand to benefit from it.

hanges to product labeling guidances are coming soon to improve the information available to patients and healthcare providers, aiming to make drug labeling a continually updated safety tool—a tool that remains dynamic, adaptable to new evidence, responsive to public health crises, and able to balance patient access with safety.