The FDA’s Office of Clinical Pharmacology Staff Participate in Expert Working Groups Developing White Papers for Improving the Tools and Methods That Underpin the Development of Innovative Drugs to Treat a Variety of Diseases and Conditions

The FDA’s Office of Clinical Pharmacology (OCP) worked together with the Duke-Robert J. Margolis, MD, Center for Health Policy as a part of expert working groups on two white papers released in June 2016. These papers highlight the pressing need for stakeholders from across the biomedical innovation ecosystem to work collaboratively toward a more efficient, nimble, and targeted drug development process.

Improving Productivity in Pharmaceutical Research and Development: The Role of Clinical Pharmacology was developed in conjunction with a working group of experts that included OCP staff and was based, in part, on a workshop held in July 2015. It outlines key opportunities and priorities for making progress in the application of clinical pharmacology tools and methods to support stakeholder decision-making. Enhancing clinical pharmacology tools and methods has the potential for improving both the efficiency of the development process itself and the body of evidence on drug candidates.  These tools, such as quantitative and systems pharmacology (QSP) and model-informed drug development (MIDD), have the power to:

• Lower the risk inherent in early-stage decision-making for sponsors.
• Positively address the late-stage attrition trends in drug development.
• Better ensure that the right investigational compound is hitting the right target in the right patient

Facilitating Biomarker Development: Strategies for Scientific Communication, Pathway Prioritization, Data-Sharing, and Stakeholder Collaboration was developed in conjunction with a working group of experts that included OCP staff and was based, in part, on an expert workshop convened in October 2015. It highlights key priority areas where further multi-stakeholder collaboration is required and ideas for adopting a more cohesive national strategy to advance biomarker development and precision medicine.  Some of the challenges include:

• A lack of consistent and coherent set of definitions for biomarkers and their uses.
• Confusion surrounding the various regulatory pathways for biomarker acceptance.
• The need for data standards.
• Rigor in data practices.
• Improved collaboration in data sharing between interested stakeholders.
• The lack of coordination and prioritization of limited public and private resources.

Funding for these papers and foundational workshops were provided through a cooperative agreement with the FDA. Although OCP staff contributed significantly, the views expressed in these papers, written conference materials, or subsequent publications do not necessarily reflect the official policies of the Department of Health and Human Services nor does mention of trade names, commercial practices, or organizations imply endorsements by the U.S. Government.

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We always welcome your thoughts regarding the format, content, and utility of information you receive via this Burst email initiative. Comments may be sent via email to [email protected]. This burst was prepared by Office of Clinical Pharmacology, Office of Translational Sciences, CDER, FDA.