The U.S. Government Accountability Office (GAO) issued a report on the Department of Health and Human Services’ (HHS) efforts to speed medical countermeasure innovation. As of June 2020, HHS had not selected a medical countermeasures innovation partner: an independent, nonprofit entity that the 21st Century Cures Act authorizes HHS to collaborate with in order to use venture capital (VC) practices and methods to invest in companies developing medical countermeasures. The GAO report reviewed relevant statutes and HHS documentation regarding its plans and actions taken to implement the authority, reviewed responses that HHS received to the two requests for information it used to collect information from VC and other stakeholders, interviewed HHS officials, and interviewed officials from the U.S. Department of Defense.
The FDA issued guidance on endpoints to support accelerated approval of neoadjuvant treatment for high-risk early stage breast cancer. The main focus of the guidance is to discuss the use of pathological complete response (pCR) in high-risk early stage breast cancer as a potential endpoint to support approval under the accelerated approval regulations. It summarizes what is known about the relationship between pCR and prognosis, and describes trial designs and patient populations in which pCR may be accepted as reasonably likely to predict clinical benefit.
The FDA issued a report on fiscal 2021 PDUFA user fees. The base revenue amount is about $1.06 billion, prior to adjustments for inflation, capacity planning, additional full-time employees, operating reserve and additional direct costs. Officials will adjust for inflation increases for the year by using two separate measures, one for personnel compensation and benefits (PC&B) and one for non-PC&B costs. The full report is here.
The NIH is seeking interest in collaborative research to develop autologous CD22 CAR T cells (m971BBZ lentivirus transduced) for the treatment of B-cell acute lymphocytic leukemia. The goal is the transfer of clinical development of the CD22-CAR program from National Cancer Institute to a drug development company. The collaborator will be responsible for the rapid scale-up and clinical manufacture of the agent to support the pivotal trial and subsequent BLA.