The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted 17-0, with one abstention, that the benefits of Pfizer Inc.-Biontech SE pediatric formulation of its two-dose COVID-19 vaccine outweigh its risks for children ages 5 through 11. Many of those votes came with caveats, as the VRBPAC members struggled with the unknowns of the vaccine and the fear that schools would mandate its use, even though it would still be considered experimental if the FDA grants it an emergency use authorization.
The Biden administration may have jumped ahead of the FDA review when it announced last month that it planned to roll out COVID-19 boosters by Sept. 20, but that’s not likely to happen when it comes to the timing of vaccines for young children.
The Research to Accelerate Cures and Equity for Children Act is leading to deep changes in pediatric cancer research. Passed in 2017 and fully implemented in 2020, the RACE Act requires companies to investigate targeted drugs for adult cancers in pediatric cancers as well “when the molecular target of the drugs are substantially related to a pediatric cancer.”
The potential for using real-world data (RWD) to support the development of cancer drugs for children has yet to be realized as researchers, sponsors and regulators continue to struggle with the collection of meaningful data.
Despite the demonstrated benefits of using patient-reported outcomes (PROs) in clinical trials assessing new cancer drugs in adults, PROs are rarely used in pediatric cancer trials. Hoping to change that, the FDA is convening the pediatric subcommittee of its Oncologic Drugs Advisory Committee (ODAC) May 11 to discuss how to effectively use a pediatric form of the PRO version of the common terminology criteria for adverse events.
PERTH, Australia – Regenerative medicine company Mesoblast Ltd. saw its stock drop 37% on the news that it received an FDA complete response letter to its BLA for Ryoncil (remestemcel-L) for the treatment of pediatric steroid-refractory acute graft-vs.-host disease.
PERTH, Australia – Regenerative medicine company Mesoblast Ltd. saw its stock drop 37% on the news that it received an FDA complete response letter to its BLA for Ryoncil (remestemcel-L) for the treatment of pediatric steroid-refractory acute graft-vs.-host disease.
Menlo Park, Calif.-based startup Tusker Medical Inc. has received U.S. FDA approval to market its breakthrough-designated system for inserting tympanostomy tubes into the eardrum to treat recurrent ear infections. The Tubes Under Local Anesthesia (Tula) system is the first delivery system for tympanostomy tubes, commonly known as ear tubes, that can be performed in young children under local anesthesia in a doctor’s office. The Tula system consists of the ionic anesthetic Tymbion, Tusker Medical tympanostomy tubes and several devices for inserting the anesthetic and tubes into the ear drum. A low-level electrical charge delivers the anesthesia to the eardrum prior to tube placement, allowing for quick and needle-free numbing of the tympanic membrane.
