Center for Drug Evaluation and Research Perspective on Quality in Clinical Trials.

Clinical trial quality is essential to bringing effective treatments to patients as quickly as possible. Clinical trials that answer important questions, yield meaningful data, and protect trial participants can provide data that support both regulatory and clinical decision making. The Food and Drug Administration's (FDA's) Center for Drug Evaluation and Research (CDER) encourages stakeholders to improve clinical trial quality and efficiency. CDER believes that a systematic approach to clinical trial quality-one that builds in quality up front and focuses on the most critical aspects of study conduct-contributes to successful trials. Beyond FDA's regulatory requirements for clinical trial quality, CDER is an active participant in multiple efforts to advance clinical trial quality, including the addendum to ICH E6 (Good Clinical Practice) and the Clinical Trials Transformation Initiative project on quality-by-design for clinical trials. These efforts aim to move clinical drug development to a desired state that centers on efficient and agile clinical development programs that reliably produce high-quality data and adhere to important ethical standards.

Good Clinical Practice Training: Identifying Key Elements and Strategies for Increasing Training Efficiency.

Good Clinical Practice (GCP) is an international standard for the design, conduct, performance, monitoring, auditing, recording, analyses, and reporting of clinical trials. The goal of GCP is to ensure the protection of the rights, integrity, and confidentiality of clinical trial participants and to ensure the credibility and accuracy of data and reported results. In the United States, trial sponsors generally require investigators to complete GCP training prior to participating in each clinical trial to foster GCP and as a method to meet regulatory expectations (ie, sponsor's responsibility to select qualified investigators per 21 CFR 312.50 and 312.53(a) for drugs and biologics and 21 CFR 812.40 and 812.43(a) for medical devices). This training requirement is often extended to investigative site staff, as deemed relevant by the sponsor, institution, or investigator. Those who participate in multiple clinical trials are often required by sponsors to complete repeated GCP training, which is unnecessarily burdensome. The Clinical Trials Transformation Initiative convened a multidisciplinary project team involving partners from academia, industry, other researchers and research staff, and government to develop recommendations for streamlining current GCP training practices. Recommendations drafted by the project team, including the minimum key training elements, frequency, format, and evidence of training completion, were presented to a broad group of experts to foster discussion of the current issues and to seek consensus on proposed solutions.

Expediting drug development--the FDA's new "breakthrough therapy" designation.

The FDA's new "breakthrough therapy" designation for investigational drugs adds to the agency's portfolio of expedited programs for serious conditions. The designation requires preliminary clinical evidence demonstrating substantial improvement over existing therapies.