Oncology clinical trials are increasingly conducted globally, with recent trends indicating a growing share of trial activity outside of the U.S. In 2025, 80% of patients in pivotal trials supporting novel cancer drug approvals by the FDA were enrolled outside of the U.S.1 Such global trials are critical for generating data across distinct regional and…
The Prescription Drug User Fee Act (PDUFA) allows the U.S. Food and Drug Administration (FDA) to collect fees from drug sponsors that streamline reviews of new drug applications, promoting timely patient access without compromising safety. When the Act is reauthorized every five years, industry and FDA discuss goals to support and improve the drug review process. With PDUFA negotiations for the 2027 reauthorization now…
As FDA and stakeholders begin preparations for the reauthorization of the Prescription Drug User Fee Act (PDUFA), Friends of Cancer Research’s new Data Driven-Insight provides a high-level overview of the program’s history, impact, and upcoming priorities. This primer provides essential context as public discussions begin around PDUFA VIII. It also highlights how user fee programs…
Introduction and Background The Accelerated Approval (AA) program is a drug development pathway that was established to expedite the delivery of promising treatments to patients, primarily for serious and life-threatening conditions. AA is granted based on surrogate endpoints, such as tumor size reduction in cancer patients, that are reasonably likely to predict clinical benefit. These…
In 2011, Friends published a study in Health Affairs that challenged the prevailing narrative of the time, which suggested that the U.S. Food and Drug Administration (FDA) was slower and less efficient than other regulatory authorities, such as the European Medicines Agency (EMA). This study provided evidence to the contrary, demonstrating that the FDA approved…
While significant progress has been made in cancer treatment and care, this progress has not always easily translated to new treatments for pediatric oncology patients. In 2003, the Pediatric Research Equity Act (PREA) passed requiring the study of cancer therapeutics in certain pediatric cases.
This is the first of two blog posts highlighting Friends’ upcoming September 20th event discussing the Breakthrough Therapy designation (BTD). In this post, we will summarize the recent history of this innovative drug approval pathway and also address potential areas of improvement. BTD is a process that expedites the development and review of drugs intended…
Since 1992, Accelerated Approval has facilitated earlier patient access to promising therapies that treat serious and life-threatening illnesses. While a traditional approval is often based on a therapy’s ability to demonstrate meaningful impact upon a clinical endpoint (e.g. overall survival), Accelerated Approval is granted based upon demonstration of an effect on a surrogate or intermediate endpoint…
Physicians rely on a variety of sources of information to provide high-quality care to patients. In oncology, some of the most prominent resources that inform provider decision-making are the American Society of Clinical Oncology (ASCO) and the National Comprehensive Cancer Network (NCCN) clinical practice guidelines; drug compendia; electronic point-of-care decision support tools, such as UpToDate;…
The U.S. Food and Drug Administration (FDA) fulfills its mission to protect and advance the public health by overseeing the development and approval of new medical products and ensuring that they are safe and effective for American consumers. When taking into consideration the full range of products the FDA oversees, including not just medical products,…
