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# Gottlieb: More Focus on Modernizing Clinical Trials
- URL: https://www.fdaweb.com/gottlieb-more-focus-on-modernizing-clinical-trials/
- Published: 2018-07-25T12:00:00.000Z
- Updated: 2026-09-15T00:21:37.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5141962

FDA commissioner **Scott Gottlieb** says more focus is being placed on modernizing clinical trials. [Testifying](https://www.fda.gov/AboutFDA/CommissionersPage/ucm614772.htm?ref=fdaweb.com) at a House Energy and Commerce Subcommittee hearing 7/25, Gottlieb said that the use of surrogate endpoints is getting much attention. He said that the agency had just released a [list](https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm606684.htm?ref=fdaweb.com) of biomarkers that have been used to support both accelerated and traditional drug and biologics approvals, as well as surrogate endpoints the agency believes would be acceptable to support approval.

In a tweet after the hearing, he noted that the agency is seeing advances in oncology with the use of innovative endpoints for accelerated and traditional approvals. Surrogate endpoints for cancer product development have delivered promising treatments to patients years before they would’ve been available using traditional endpoints, he said.

“While the acceptability of these surrogate endpoints for use in a product development program will be determined on a case-by-case basis, this list is intended to serve as a reference guide to help inform discussions of potential surrogate endpoints with the relevant CBER or CDER review divisions, with the goal of facilitating product development,” Gottlieb said at the hearing. “We are currently working towards developing and publishing several guidances required by the Cures Act to establish the process, taxonomy, and framework for \[drug development tools\] qualification.”

Gottlieb told lawmakers that the “current one drug, one trial process can be wasteful and inefficient. It can delay both access to innovative therapies and developing sufficient evidence about their performance. And it doesn’t leverage the clinical trials that we have underway. Approaches like basket trials, master protocols, and seamless trial designs allow us to learn more about new drugs, and even evaluate different drugs in the conduct of the same clinical trial.”

  
Escalating drug trial costs and complexity play a role in market competition and drug pricing, Gottlieb told the hearing. A new FDA study examined the number of drugs or biologics that CDER has approved in the same class. “We found that new competition isn’t entering the market as quickly for these drugs,” he said in his testimony. “In other words, when a novel sole source drug wins approval it faces no competition from other drugs in the same class. Follow-on drugs and biologics to compete with the first in class have been arriving more slowly.” He shared the following results from the study:

- For non-orphan pharmaceuticals, which treat conditions affecting larger patient populations, 41% of the first-in-class approved between the years of 1991 and 2000 had at least one competitor in the same class within five years. This rate dropped sharply over the next decade. For the years from 2001 to 2010, for the same kind of cohort of medicines — first-in-class products that were approved to treat patients with prevalent conditions — only 18% of these drugs had a within-class competitor after five years.
- Another way of interpreting the data is to describe the lag in any competition. For the older classes, where the first in class was approved in 1991 to 2000, nearly a quarter had a competitor within two years. For the cohort where the first-in-class was approved in 2001 to 2010, it took an additional five years for there to be nearly as much competition. By year seven, competition still lagged the previous cohort, with only 22% of classes having any competitor. Similar patterns are also seen in most rare disease treatments.

Gottlieb said it is important to understand the data. “Part of it has to do with the difficulty of running clinical trials with a second to market drug, especially after there’s available therapy for an unmet need,” he said. “It’s becoming harder and harder to be second. That’s a problem. Efficient, modern approaches to designing and conducting clinical trials can address some of these challenges.”

For example, Gottlieb said one approach is to use seamless trial designs that “compress the traditional three phases of trials into one continuous trial. Through these approaches, you run one continuous trial. And as you enroll new patients, you expand subsequent cohorts of enrolled patients using the information you learn about the features that help predict benefit from a new treatment.”

He said that FDA will soon release a guidance on seamless trials that will discuss how to expand cohorts as trials progress, and the clinical criteria that can be used to expand cohorts as trials advance. “We’ve already successfully used these approaches with some of the new immunotherapies,” he said. “For drugs that might already qualify for breakthrough therapy designation, expansion cohorts can be used to quickly expand enrollment in biomarker selected Phase 1 cohorts, and evaluate the drug in one seamless, continuous clinical trial.

“Multiple, concurrently accruing and individual cohorts can allow us to better assess a lot of information in one large trial, answering questions about safety, the pharmacokinetics related to how a drug is absorbed and distributed in the body, and anti-tumor activity,” Gottlieb continued. “These questions can be evaluated in cohorts that are specially designed to assess these different, important questions. With more efficient development using a seamless design, the whole trial can be completed with a few hundred patients.”