In the not-so-distant past, clinical trials were considered an option only for the young and fit. Enrolling older people “used to be thought unethical,” said Janet Woodcock, MD, Director of the U.S. Food and Drug Administration (FDA)’s Center for Drug Evaluation and Research (CDER), as she opened the 2017 FDA-ASCO Geriatric Oncology Workshop. “We’ve…
When 29-year-old Carly Bastiansen was diagnosed in January 2016 with advanced pancreatic cancer, doctors told her a clinical trial was her best shot at slowing the notoriously quick-killing and hard-to-treat disease. She found one that appeared promising and went through the screening process. But the trial would not accept her. “Participating in a clinical…
Executive Summary Celgene’s ASH briefing notes ‘unprecedented’ MRD-negative data in Phase I study in multiple myeloma development program for CAR-T therapy bb2121; firm sees potential opportunity for the marker to pave way for approval in earlier-line settings of the disease. Celgene Corp. believes minimal residual disease (MRD) may someday be the ticket to approval of…
FDA officials are floating a proposal for a review process informally dubbed “reverse accelerated approval.” This mechanism would come into play in situations where the data that have already been provided to the agency may justify approval of supplemental indications. This way, sponsors would not be required to conduct new confirmatory trials when…
Executive Summary Discussion on cutting edge medical technology demonstrates the strong reputation FDA has built as a regulator. Witnesses in the gene editing field said FDA is doing a good job – and Congress is likely to leave it at that. The US FDA’s early approach to regulation of gene editing is “robust” and…
Executive Summary Importance of randomized, controlled trials and need for a thoughtful, considered approach to development of rational combinations are highlighted by the increased mortality risk seen with pembrolizumab treatment in two halted multiple myeloma studies, FDA officials say. The troubling mortality results from two multiple myeloma trials of Merck & Co. Inc.’s PD-1 inhibitor…
Executive Summary Commissioner Gottlieb says new centers would follow template of Oncology Center of Excellence. US FDA Commissioner Scott Gottlieb has two additional centers of excellence already in mind – immunology and neuroscience – should the model implemented for oncology work as planned. Both, like the Oncology Center of Excellence that launched at the…
FDA is developing a policy by which the agency can approve subsequent indications of cancer drugs with less rigorous follow-up trials, FDA Commissioner Scott Gottlieb announced Thursday (Nov. 30). The agency will also provide more guidance on how FDA believes it can approve treatments based on intermediate clinical endpoints, which one expert explained may be…
Executive Summary Commissioner Gottlieb tells Congress that concept could be used for some clinical endpoints when a large benefit is seen in a small trial, which could benefit rare disease drug development. The US FDA is exploring a potentially dramatic expansion of the accelerated approval mechanism. Under the concept, products showing a substantial, early effect…
Executive Summary In citizen petition response involving Takeda’s Velcade, agency says ANDA and 505(b)(2) applicants may add more words to the indication statement in order to carve-out protected uses from the reference product’s broadly written indication. The US FDA appears to have established a new “carve in” approach to “carving out” a reference product’s protected…
