In remarks at the Regulatory Affairs Professionals Society’s (RAPS) 2017 Regulatory Convergence Conference yesterday, U.S. Food and Drug Administration (FDA or the Agency) Commissioner Scott Gottlieb, M.D., spent considerable time addressing how “seamless” clinical trials and more widespread use of modeling and simulation could help combat the costs of both drug development and new drugs. This is not the first time that Gottlieb has addressed the challenges of bringing new products to market and drug costs (see here and here).
Commissioner Gottlieb remarked at RAPS that “[w]e’re on an unsustainable path, where the cost of drug development is growing enormously, as well as the costs of the new medicines. We need to do something now, to make the entire process less costly and more efficient.” Gottlieb suggested that combined-phase studies, or “seamless” clinical trials, could help combat these costs. He explained that, instead of the typical three-phased trial, “seamless trials encompass one adaptive study where the phases are separated by interim looks. By using one large, continuous trial, it saves money and reduces costs.” Among other things, seamless trials reduce the number of patients that have to be enrolled in a study, he noted.
Gottlieb suggested that this approach has become increasingly popular in oncology drug development. He went on to explain that the Agency has seen sponsors develop such drugs that forgo the usual three phases of development in favor of a seamless approach, adding cohorts to first-in-human trials to investigate doses and activities in a variety of cancers. Gottlieb also observed that this approach has been used with some newer immunological therapies, and that it may be used in other drugs targeted against specific molecular defects. “Seamless designs are particularly advantageous for drugs that work in a variety of diseases, allowing rapid evaluation of the drug and potential approval under our accelerated approval pathway,” he indicated. Per Gottlieb, FDA has identified more than 40 active commercial investigational new drug applications (INDs) for large, first-in-human oncology trials that use the seamless approach.
In addition to addressing trial design, Gottlieb also focused on steps the Agency is taking to modernize how sponsors can evaluate clinical information, including more widespread use of modeling and simulation, as well as high performance computing clusters inside FDA. While the Agency already has these clusters, Gottlieb shared that access to them is limited, and that he is directing an effort to try to increase FDA’s investment in these tools. As part of an effort to advance use of these tools, the Agency plans to convene a series of workshops, publish guidance documents, develop policies and procedures for translating modeling approaches into regulatory review, and conduct a pilot program on these approaches.
We will continue to monitor these and other regulatory developments and provide more information as it becomes available.