• Regulatory NewsRegulatory News

    Global regulators promote platform trials to assess new COVID vaccines

    Regulators from the United Kingdom and Japan are promoting the use of platform trials to evaluate second-generation COVID-19 vaccines as the pool of unvaccinated patients for testing new vaccines grows increasingly smaller. They also proposed the creation of a working group to build consensus on the design of master protocols, particularly in the context of health emergencies.   These pronouncements emerged from a 24 June virtual workshop on aligning approaches for dev...
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    DIA: Woodcock, other top officials tout benefits of master protocols, want momentum to continue post-COVID

    Top officials from the US Food and Drug Administration (FDA) exhort the benefits of master protocols and hope the momentum of using these protocols continues in the post-COVID-19 era. Officials also say the pandemic has not dampened the enthusiasm for gene therapy development as the agency continues to receive a healthy number of investigational new drug applications (INDs) for these therapies.   These were some of the learnings imparted by agency officials in discussi...
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    FDA issues new COVID-19 master protocol guidance

    A new guidance from the US Food and Drug Administration (FDA) addresses how master protocols can be used in developing drugs to treat or prevent COVID-19.   “To meet the urgent demand for effective therapies, FDA has worked with clinical trial experts to rapidly advance best practices in the design and execution of clinical trials,” said acting FDA Commissioner Janet Woodcock in announcing the new guidance. “Master protocols that are well designed and executed can acce...
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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...