Executive Summary US FDA commissioner and others say they have put disagreements from controversial decision to approve Sarepta’s Duchenne muscular dystrophy drug behind them. FDA entrance sign 2016 FDA’s controversial approval of Sarepta Therapeutics Inc.’s Duchenne muscular dystrophy drug Exondys 51 elicited emotional disagreements among FDA staff, but officials maintain that they have largely been…
When I lost my only sister to breast cancer in 1986, patients like her had devastatingly few choices. Over the intervening decades, sustained commitment to biomedical research at the National Institutes of Health (NIH) and major technologic advances have led to transformative changes in cancer research and treatment. Advanced therapies and protocols have boosted survival…
Executive Summary US FDA, CMS and NIH are hoping to create a “single front door” for research. Article outlining collaboration on real-world evidence suggests one path forward. The heads of FDA and CMS are outlining a vision for collaboration on development of real-world evidence ahead of the transition to a new presidential administration. …
Executive Summary After a record-setting 2015 for new drug approvals, there were not enough applications with user fee goals in 2016 to reach that level again. FDA’s approval metrics could not remain at record levels forever, and it now appears the regression toward the mean has begun. Office of New Drugs Director John…
Executive Summary Center for Drug Evaluation and Research’s long-time director of oncology/hematology products office will continue as signatory on drug reviews even after new Cancer Moonshot-driven structure takes effect. FDA’s new Oncology Center of Excellence will not take Richard Pazdur away from his drug approval duties. Pazdur, who is the acting director of…
The FDA is considering ways to review more medical treatments by clinical area, following in the steps of the cancer center established this summer. “Historically, as almost everyone knows, FDA has been remarkably siloed,” Food and Drug Administration Commissioner Robert M. Califf said Nov. 4. “But what you hear loud and clear from patients…
Regarding the Nov. 2 PowerPost article “FDA continues to grapple with hundreds of vacancies”: For cancer patients, every day matters, and the Food and Drug Administration is a vital player in speeding lifesaving treatments to those who need them. Patients expect the FDA to hire and retain world-class talent. But for nearly a decade,…
The decision to approve a controversial rare-disease treatment has haunted the Food and Drug Administration in recent weeks. But instead of shying away from the controversy, the FDA has been remarkably transparent about how the decision was made. On Thursday night, the agency laid bare the disagreements between its highest officials by releasing 300 pages…
Executive Summary Bradley Thompson has been shepherding the Combination Products Coalition for more than a decade. In a recent interview, he said the recent policy spotlight on the combo-products space, including in a recent user-fee deal, is a welcome development after a period of neglect. He also spoke about the numerous reforms in the works…
Executive Summary Express Scripts’ Eichholz says clinicians may be less inclined to use a biosimilar in ‘life or death’ oncology indications compared to inflammatory diseases. Clinical decisions on whether to prescribe a novel biologic or a biosimilar may be driven, in part, by the condition and stage of disease, representatives from the pharmacy benefit,…
