FDA Unveils Regulatory Science Progress Report

04 April 2017

FDA Unveils Regulatory Science Progress Report

April 4, 2017

The report, covering fiscal years 2015 and 2016, notes significant US Food and Drug Administration (FDA) progress in refining non-clinical and clinical tools to better evaluate medical products, advancing manufacturing and quality, and enhancing internal IT tools to support the scientific review of regulatory applications.


In terms of the non-clinical predictive models, the report notes that FDA researchers developed a wide variety of computational tools that now support nonclinical evaluation of medical products.

“These tools include sophisticated models to predict the carcinogenic effects of certain drug ingredients based on their structural attributes, mathematical representations of the human body (computational phantoms) that can be used to predict the effects of medical devices, such as exposure to radiation, and mechanistically informed pharmacokinetic models to help predict drug exposures in populations where clinical data are difficult to obtain,” the report says.

FDA also has developed in vitro methods and tools to better understand the properties of drugs and biologics, including vaccines, and new tools to support the evaluation and development of medical imaging technologies.

Genetic and transplantation approaches were also used to create animal models that seek to more closely predict human response to medical products, and novel physical methods and procedures were developed to support the evaluation of generic drug bioequivalence.

Clinical Evaluation

To better support the clinical evaluation of medical products, FDA is engaged in efforts to advance the development and evaluation of biomarkers to better predict long-term treatment outcomes or to assign certain patients to treatments more likely to succeed.

As an example, FDA notes that its statisticians have helped design trials of antibiotics that could evaluate factors related to the development of resistant organisms.

In addition, the agency has qualified new biomarkers to guide treatment decisions and to predict disease progression.

“A long-term research effort to improve prediction of cardiovascular risks contributed to the recommendation by the International Conference on Harmonisation that the costly and resource-intensive ‘thorough QT’ clinical study (required to evaluate most drug candidates) could be replaced with electrocardiogram-based measurements performed during early-phase clinical studies,” the report notes.

It also mentions FDA’s efforts around the use of real-world evidence, which staffers have expressed qualms with in the past.

And through initiatives such as the Patient-Focused Drug Development Initiative, the Patient Engagement Advisory Committee for medical devices and the Patient Preference Initiative, FDA is further incorporating the patient viewpoint into medical product development and evaluation.

Manufacturing and Quality

FDA medical product centers also address scientific issues related to new technologies critical for product manufacturing to improve the reliability of the drug supply, the characterization of complex products, quality standards, post-approval monitoring of product quality and understanding the interactions of regulated products with biological systems.

“We have developed in-house laboratory and computational capability for studying continuous manufacturing with an advanced process control system,” the report adds. “We collaborated with the Biomedical Advanced Research and Development Authority (BARDA) to leverage continuous manufacturing to minimize domestic vulnerability to chemical, biologic, and radiologic threats, and we spearheaded creation of a 3-D printing facility to understand factors contributing to the quality and performance of implantable medical devices, drugs, and combination products made with this new technology.”

In addition, FDA has developed automated approaches for predicting critical properties of human stem cell preparations, such as their ability to contribute to bone growth.


The agency has implemented its “JumpStart” service, which allows reviewers to organize, manage and verify the quality of the clinical data in product applications, and initiated Kickstart, a service that delivers individual training and user-driven support and analysis for non-clinical data.

“To make possible the secure deposition, retrieval, and analysis of the vast next generation sequencing data that will support personalized medicine, we continued to enhance our high performance scientific computing environments. We extended our laboratory capabilities and facilities for mission-critical areas, including advanced manufacturing, analytical methodology, and emerging infectious diseases,” the report says.

FY 2015-2016: Regulatory Science Progress Report

Source: RAPS