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    FDA Officials: Master Protocols Needed for Precision Medicine

    In an article published Thursday in the New England Journal of Medicine , two top officials from the US Food and Drug Administration (FDA) say that "master protocols" for studies involving multiple drugs or multiple diseases (or both) simultaneously are needed to efficiently generate evidence for precision medicines. "The standard approach to generating this evidence—a series of clinical trials, each investigating one or two interventions in a single disease—has become ...
  • Data Show Varied Use, Success of FDA's Expanded Access Program

    In recent years, much attention has been focused on the pharmaceutical industry and its use-and non-use-of the US Food and Drug Administration's (FDA) expanded access program. But how much success has it had? Background That program, sometimes referred to as the "compassionate use" program, allows patients to access experimental treatments they might otherwise not be eligible to receive. For a full explanation of the program, please see our Regulatory Explainer on ...
  • EMA Guide on Development of Novel Drug Development Methodologies Released

    The European Medicines Agency (EMA) released a new guidance on 26 January that addresses the development of novel methodologies in drug development, and how EMA develops qualification opinions and advice for scientific methods and protocols. The qualification process is a "new, voluntary scientific pathway leading to either a [Committee for Medicinal Products for Human Use (CHMP)] opinion or a Scientific Advice on innovative methods or drug development tools." The pr...