A comprehensive analysis of CBER experience with AAV gene therapy IND submissions  Nature

YouTube video by Jedediah Bila

Estudiantes de diversas localidades debatieron sobre eficiencia energética en el Centro Bonaerense de Energías Renovables. El Centro Bonaerense de Energías Renovables (CBER) fue el escenario de una jornada en la que participaron 160 estudiantes del ciclo superior de escuelas secundarias de La Plata, Berazategui, Melchor Romero, City Bell, Arturo Seguí y la Ciudad Autónoma de Buenos Aires. Uno de los puntos destacados fue la presencia de 13 estudiantes de la Escuela de Educación Secundaria Técnica N.º 2 de Berazategui, quienes están próximos a egresar de la Tecnicatura en Energías Renovables.

The voices of patients with a rare disease are sometimes heard by the FDA—at listening sessions and during advisory committee meetings—but for biologics head Karim Mikhail, it isn’t enough. These opportunities currently come late in the drug development cycle, as therapies are on the cusp of potential market entry. “We need to have sponsors, experts and patients engaged in the process from early on,” Mikhail, director of the Center for Biologics Research and Evaluation (CBER), told BioSpace in an interview—right from the time companies seek permission to test their assets in humans. Only then can substantive changes be implemented based on patient feedback. Today, when patients are heard during adcomm meetings, “that’s way too late,” Mikhail said. “The clinical development is already defined. Everything is already set.” Under the FDA’s new Rare Disease Framework Initiative, unveiled last month, drug sponsors should expect that the patient perspective will be an essential component of the regulatory submission, Mikhail said. Patient input will be used to define several elements of a rare disease development program, he continued, from a clinical endpoint that is “actually meaningful” to an understanding of the benefit-risk ratio. This is a question that should be answered by those who...