Streamlining Drug Development and Improving Patient Care: CDER Quantitative Medicine Center of Excellence

CDER recently launched the new CDER Quantitative Medicine (QM) Center of Excellence (CoE). The goal of this CoE is to facilitate and coordinate the continuous evolution and consistent application of QM across CDER. QM involves the development and application of exposure-based, biological, and quantitative modeling and simulation approaches derived from nonclinical, clinical, and real-world sources to inform drug development, regulatory decision-making, and patient care. These approaches contribute to the totality of understanding of a drug's benefits and risks, helping to advance therapeutic medical product development and inform regulatory decision-making.

In this CDER Conversation, Rajanikanth Madabushi, lead for the QM CoE and associate director for Guidance and Scientific Policy in the Office of Clinical Pharmacology in the Office of Translational Science Super Office explains the purpose of the new CoE, provides an overview of current activities and resources, and shares ways the CoE can advance drug development and improve patient care.

What prompted CDER to establish the QM CoE?

For decades, CDER has been at the forefront of advancing QM approaches to inform premarket product review and post-market product assessment. Given the tremendous growth in QM, we see many opportunities to maximize synergies across CDER by centrally coordinating efforts through strategic planning and execution. By establishing this CoE, CDER is putting a stake in the ground to advance initiatives that spur innovation and promote integration of QM approaches in CDER. The CoE provides the organizational framework to foster collaboration and coordination of QM efforts across CDER and facilitate engagements with all stakeholders to advance therapeutic medical product development, inform regulatory decision-making, and promote public health.

What is the goal of the CDER QM CoE and what types of projects will fall under the CoE?

The primary goal of the QM CoE is to facilitate and coordinate the continuous evolution and consistent application of QM across CDER. CDER’s QM CoE is a center-wide effort and will:

• Spearhead QM-related policy development and best practices to facilitate the consistent use of QM approaches during drug development and regulatory assessment.
• Facilitate systematic outreach to scientific societies, patient advocacy groups, and other key stakeholders.
• Coordinate CDER’s efforts around QM education and training.

The CoE will take on key initiatives in the areas of Applied Science Policy, Strategic Planning and Coordination, and Multidisciplinary Education and Exchange.

How do Model-Informed Drug Development (MIDD) and the MIDD Paired Meeting Program fit into the new QM CoE?

As mentioned earlier, QM involves the development and application of exposure-based, biological, and quantitative modeling and simulation approaches derived from nonclinical, clinical, and real-world sources to inform drug development, regulatory decision-making, and patient care. As such, MIDD, an approach that involves developing and applying exposure-based biological and statistical models derived from preclinical and clinical data sources to inform drug development or regulatory decision-making, is a fundamental QM activity. As the use of QM approaches in drug development and regulatory assessment have increased, CDER has created new, dedicated forums for regulatory engagements with drug development stakeholders, including the MIDD Paired Meeting Program, Complex Innovative Trial Design (CID) Program, and the Model-Integrated Evidence (MIE) Pilot Program.

CDER has well-established processes for interactions through these forums. In general, the establishment of the QM CoE is not expected to alter any of the existing processes of these programs. As the central coordinating body, the CoE will facilitate sharing experiences and disseminate lessons learned across these forums to promote the consistent application of QM approaches throughout the life cycle of product development.

We recently noticed that the QM CoE shared an educational series related to MIDD. Can you share more about this resource? Who is the course meant for, and what can they expect to learn from the course?

One of the core focus areas of CDER QM CoE is multidisciplinary education and exchange, including the development, coordination, and dissemination of accessible education and training resources. As part of these efforts, CDER’s QM CoE coordinated the release of the educational series “Model-Informed Drug Development (MIDD): Methods Advancing Medical Products to Patients” aimed at increasing community-wide knowledge of these approaches.

The educational content was originally developed for regulatory scientists by experts in the field in partnership with the Critical Path Institute and funded by CDER’s Office of Translational Sciences. The series includes eight modules with 29 presentations and more than 10 hours of content. In this course, participants will learn the applications of MIDD approaches across the development of drugs and biological products. The course is intended as a foundational introduction to the applications of MIDD approaches for those involved in drug development and review.

How do you anticipate the QM CoE will advance drug development and, ultimately, improve patient care?

New drug development is a multifaceted, complex, high-risk endeavor involving a range of scientific, financial, and regulatory challenges. Innovative technologies, tools, and approaches have been proposed to increase drug development efficiency and optimize treatments reaching patients. QM is one of those innovations.

For example, QM approaches play a critical role in organizing diverse data sets, enhancing the mechanistic understanding of disease, exploring alternate study designs, informing dose selection and optimization, identifying subpopulations for therapy, evaluating critical regulatory review questions such as evidence of effectiveness, strategizing lifecycle plans in the post-approval setting, and supporting development of complex generic products. For more examples of how QM approaches have contributed to drug development and review, I encourage readers to visit the web page for the recording and slides of our recently held public workshop, Streamlining Drug Development and Improving Public Health through Quantitative Medicine: An Introduction to the CDER Quantitative Medicine Center of Excellence.

By continuing to foster the integration and broader adoption of QM approaches across CDER, the CoE can help advance drug development and inform regulatory decision-making. As a result, the QM CoE is anticipated to help streamline drug development and accelerate the delivery of safe, effective, therapeutically optimized medicines to the public.

Looking to the future, what are some upcoming projects under the QM CoE?

To advance the science and foster the use of QM methods in drug development, the CoE plans to:

• Identify and prioritize gaps in knowledge and determine areas of further research and development in CDER.
• Develop a strategic plan and create task forces to achieve its goals.
• Create additional opportunities to engage with the QM CoE in public forums.
• Provide additional education and training opportunities for all stakeholders involved in drug development and review.
• Develop a repository of case studies of when and how quantitative approaches helped streamline drug development, for example, by informing clinical trial design, optimizing drug dosages, helping determine patient populations, or improving benefit/safety profiles.

The QM CoE encourages all stakeholders to explore how they can help the QM CoE advance and integrate QM to maximize societal benefit and patient care. Interested parties can visit the CDER QM CoE web page or email CDERQuantMed@fda.hhs.gov for more information.