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,…
Molecular diagnostics are central to the delivery of personalized cancer care. Without these important tools, physicians would not be able to select the appropriate patients for targeted therapy, negating the advantages conferred by recent advances in genomics. In the past fifteen years, there has been an explosion of new technologies to interrogate the genes and…
In his first speech to Congress last February, President Trump revived a common misconception about drug regulation. The president’s comments reflect a perennial critique of the FDA, that the agency’s drug regulations impede timely access to new medicines. A few years ago, Friends of Cancer Research (Friends) explored whether these claims were true about cancer…
This is Data Driven, a new series that explore our nation’s health agencies’ publicly available data. This data, which is comprised of searchable online databases and files, presents a unique opportunity to analyze information on broad health care topics. We thought it would be interesting to explore data from agencies whose work most closely…
