Previous Section: 3. Support New Approaches to Improve Product Manufacturing and Quality

We are at a critical moment where advances in science are leading toward fundamental changes in the way medical treatments and diagnostics are being developed and used. Groundbreaking discoveries in complex chemistry and biosynthesis have the promise of yielding new drug candidates, and cutting-edge electronics, nanotechnology and materials science have revolutionized medical devices. Emerging fields such as gene therapy, cell therapy, tissue engineering, optogenetics, high intensity focused ultrasound, and information technology are also yielding innovative approaches to improve our health. These novel and increasingly complex approaches to health, foods, and medical products present growing challenges to FDA’s readiness to evaluate new products. Regulatory science must be one step ahead to equip FDA with the necessary tools and methods to reliably assess the safety and efficacy of products derived from these new scientific developments, in order to bring the rewards of discovery safely forward to benefit patients.

Implementation Strategy

FDA will develop necessary expertise and infrastructure to evaluate new and emerging technologies through active research intramurally and collaboratively with external partners to address each of the following needs:

1. Stimulate development of innovative medical products while concurrently developing novel assessment tools and methodologies:
   1. Implement innovation strategies, such as the Center for Devices and Radiological Health (CDRH) Innovation Initiative ¹ to facilitate partnerships that create new device development and assessment tools. This initiative explores ways to initiate first-in-human studies earlier in device development, offers an expedited pathway for development, assessment, and approval of important devices, and encourages early conversations with developers to make sure their ideas are translated into technologies that both help patients and are proven safe and effective. Other ongoing initiatives to stimulate innovation include the Center for Veterinary Medicine’s (CVM) InnoVation Exploration Team (IVET) to facilitate the discovery of novel approaches and innovative products while maintaining safety, effectiveness, and quality standards; and
   2. Encourage promotion of, and participation in, research and other efforts to increase scientific understanding and help facilitate assessment of data needs for novel regulated products, including those relevant to emerging technologies such as nanotechnology ².
2. Develop assessment tools for novel therapies:
   1. Develop new approaches such as in vitro and in vivo methods to identify measurable characteristics of product safety, quality, and potency when evaluating new therapeutics (e.g., engineered tissues or cell therapy products, including stem cell-derived products, for clinical application in regenerative medicine);
   2. Evaluate and adopt new developments in the fields of cell biology, developmental biology, and materials science to help us better understand the impact of interactions between living cells and materials used in tissue-engineered medical products, as well as the interaction of cellular products with microenvironments in the patient;
   3. Develop new ways to evaluate gene therapy and antisense therapy products developed during this period of fast-paced scientific progress;
   4. Integrate an understanding of product quality and safety based on novel genomic, proteomic, metabolomic, and other -omic technologies;
   5. Explore the role of wireless and information technologies in new medical therapies and diagnostics; and
   6. Conduct periodic, comprehensive horizon scanning to identify emerging technologies.
3. Assure safe and effective medical innovation:
   1. Develop robust innovation strategies that include reaching out to manufacturers in areas of high public health need with few existing effective interventions and challenging scientific hurdles;
   2. Build upon the clinical research initiatives to ensure rapid and reliable testing of innovative medical products. This may include developing new strategies to detect novel interactions and toxicities posed by novel material and mechanisms of action; and
   3. Help stimulate the development, standardization, and validation of new techniques to assess safety and effectiveness.
4. Coordinate regulatory science for emerging technology product areas:
   1. Enhance the collaboration of multidisciplinary scientific expertise within the Agency when evaluating emerging technology product areas; and
   2. Develop mechanisms to promote cross-disciplinary regulatory science training and research to address scientific gaps and challenges posed by novel products.

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Nanotechnology

As medical products are being revolutionized by cutting-edge technologies like nanotechnology, it is more critical than ever for FDA to fully understand how nanomaterials are being used in these products. FDA has established Nanotechnology Core Centers to provide critically needed equipment and technical staff to conduct product assessment and safety research in support of regulatory decision making. These centers will facilitate investigations on the safety of products that use nanomaterials, establish methods to assess quality and effectiveness of products that use nanomaterials, and identify standards to be incorporated in the preclinical safety assessment of products that contain nanomaterials.

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Public Health Impact:

FDA’s efforts will help foster greater predictability in the Agency's regulatory processes, enable innovation, and enhance transparency while protecting the public health.

FDA will be better equipped to facilitate the translation of groundbreaking new products into effective innovative medical products, while still maintaining the high safety standards Americans depend on.

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1. CDRH Medical Device Innovation Initiative accessible at: https://www.fda.gov/about-fda/about-center-devices-and-radiological-health/cdrh-innovation.

2. FDA Nanotechnology Regulatory Science Research accessible at: https://www.fda.gov/science-research/science-and-research-special-topics/nanotechnology-programs-fda.

Table of Contents: Strategic Plan for Regulatory Science

Next Section: 5. Harness Diverse Data through Information Sciences to Improve Health Outcomes