In recent months, the U.S. Food and Drug Administration has increased its compliance focus on the regenerative medicine industry, vowing to crack down on illicit stem cell clinics and others advertising the untested use of human cell and tissue-based products (HCT/P), many of which are used in orthopedics.
Through proper implementation of ICH Q12 tools and enablers, the industry could manage CMC changes effectively under a company’s pharma quality system with less need for oversight prior to implementation.
This article outlines six recent regulatory changes in China intended to protect patient safety as well as reduce regulatory burdens and minimize delays.
In today’s changing industry, it is critical we consider several factors about cell culture bioprocessing and the impact they will have on the future of patient treatment.
According to the inaugural annual report from the International Society for Cell and Gene Therapy (ISCT), the number of CGTs with marketing authorization increased by 45 percent between 2015 and September 2018.
A quick and nimble approach is often sought after in the cell and gene therapy product space due to the likely one-time or limited patient dosing and/or rare disease conditions limiting the number of available patients, which reduces the number of clinical studies.
The FDA published a new draft guidance on August 10, 2018 entitled, Expansion Cohorts: Use in First-In-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics. The comment period for the draft guidance closed on October 12, 2018 and approximately 20 comments were submitted to the docket. The guidance provides sponsors with recommendations for designing and conducting first-in-human (FIH) multiple expansion cohort trials within their oncology development programs.
CMOs are positioned to play a leading role in standardizing control strategies for gene therapy because so many gene therapy products are funneled through CMOs for commercial scale production.
The regenerative medicine sector is at a remarkable moment. Transformative products are now on the market and accessible to greater numbers of patients every day. Dozens of additional therapies are in late stage studies. The regulatory and policy environment has evolved rapidly alongside the science, enabling a surge of incoming innovation.
A number of challenges are hindering efforts to develop cell and gene therapies to treat rare diseases. While these challenges may seem common across other drug markets, in the case of rare diseases, they are exacerbated by limited patient populations