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# Clinical Development Modernization Urged by Gottlieb
- URL: https://www.fdaweb.com/clinical-development-modernization-urged-by-gottlieb/
- Published: 2017-09-11T12:00:00.000Z
- Updated: 2026-09-14T22:48:10.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5139589

A top priority for FDA commissioner **Scott Gottlieb** is to modernize how clinical information is collected. “FDA continues to advance the use of new tools and clinical trial designs,” he said in an [online post of a speech](https://www.fda.gov/NewsEvents/Speeches/ucm575400.htm?ref=fdaweb.com) to stakeholders. “For example, we’re seeing wider use of adaptive approaches, which allow scientists to enrich trials for patient characteristics that correlate with benefits, or that help predict which patients are least likely to suffer a certain side effect.” Another promising area, he said, is the use by industry of combined-phase studies (also known as seamless trials). “Instead of conducting the usual three phases of study, seamless trials encompass one adaptive study where the phases are separated by interim looks,” h said. “By using one large, continuous trial, it saves time and reduces costs. It also reduces the number of patients that have to be enrolled in a trial.”

To illustrate the promise, Gottlieb pointed to advances seen with oncology drug development programs. Under oncology trial design seamless approaches, sponsors “typically add cohorts to a first-in-human trial to investigate doses and activity in a variety of cancers. These methods have been used with some of the newer immunological therapies. It may also 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. These new approaches are also highly consistent with the goals of the 21st Century Cures Act and the recently passed FDA Reauthorization Act.”

Gottlieb said the approach has “allowed the rapid development of drugs in multiple different tumor types. If we had to stop and start formal Phase 2I trials in each different organ system where a cancer arose, it could have been a protracted process. This approach is well suited to the kinds of drugs that are being developed now, where drugs intervene on common elements found across multiple kinds of disease states. At FDA, we’ve identified more than 40 active commercial investigational new drug applications for large first-in-human oncology trials alone that use these seamless strategies.”

Other areas FDA wants to incorporate into more development programs include common control studies and the wider use of large simple trials. “We’re also advancing the use of ‘master protocols’ to enable more coordinated ways to use the same trial structure to evaluate treatments in more than one subtype of a disease or type of patient,” Gottlieb said. “This approach is particularly relevant when it comes to targeted drugs. These are drugs that may intervene on markers that are relevant across many different disease subtypes. We may, for example, want to evaluate these different targets simultaneously, as part of one large study. This could give us a better way to understand the comparative benefits of a drug across different settings. To enable these master protocols, it’s often important to do molecular patient screening. This can lead to the development of a diagnostic that can also be used to guide patient care.”

New development approaches also lead to new regulatory challenges, according to Gottlieb. “First-in-human trials with expansion cohorts may also encompass an entire drug development program in a single trial,” he said. “So potentially important regulatory interactions, such as the standard guidance meetings held at the end of Phase 2 and before the initiation of Phase 3 trials may not automatically occur. Comparable regulatory milestones need to be built into the new seamless clinical trial process. We need to ensure we provide comparable interactions and oversight. The agency also needs to engage in more communication between sponsors, investigators, IRBs, and other stakeholders involved in the development program.”

A separate area Gottlieb said FDA will focus on is modernizing how sponsors can evaluate clinical information, and how the agency reviews this data. “This starts with better use of more advanced computing tools, and more sophisticated statistical and computational methodologies, as part of the drug development and the drug review process,” he said. “This includes more widespread use of modeling and simulation, and high performance computing clusters inside FDA. FDA already has high performance computing clusters. These tools help us develop more sophisticated methods for evaluating the data that’s submitted to us from clinical trials. The computing tools also enable us to properly evaluate the more sophisticated components that are submitted to us as part of product review applications.”

Gottlieb said he is pushing an effort to increase its investment in computing tools. “Almost 100% of all new drug applications for new molecular entities have components of modeling and simulation,” he said. “Typically, these modules are focused on similar uses. They include modeling dose response as a way to better evaluate the safety and efficacy of different doses, and help select the optimal dose for the general population or subgroups. They also include methods to estimate a new drug’s effect size to develop the appropriate sample size for pivotal trials. Finally, modeling and simulation is also commonly used to evaluate the reliability of endpoints, such as helping to demonstrate a relationship between a biomarker and a clinical endpoint relationship.”

To advance these efforts, FDA is planning to hold workshops, publish guidance documents, and develop policies and procedures for translating modeling approaches into regulatory review. It will also launch a pilot program on advanced tools.