This is a golden age for biomedical research, with novel technologies and groundbreaking science revealing new pathways to diagnose, treat, prevent, or cure disease. Yet too many patients still lack lifesaving therapies. Last year, the US House Committee on Energy and Commerce launched the bipartisan 21st Century Cures initiative to consider opportunities to transform biopharmaceutical research and development. Now under review in Congress, the 21st Century Cures Act, proposes new ways to streamline clinical trials, incorporate patient perspectives into decision making, and speed the science of cures to the bedside. Are fundamental changes needed in how we invest in and oversee research, product development and review? Where along the arc of discovery, testing, approval, and marketing can we accelerate the pace of biomedical advances?