Regulatory Science Initiative

Title of proposed idea: Regulatory Science Initiative  

Nominator: NIH Institutes/Centers

 Major obstacle/challenge to overcome: The rapid pace of scientific discovery coupled with development of new technologies presents a challenge to researchers, clinical investigators, and regulators as they work to translate basic scientific advances into approved medical products.  Basic and preclinical research has been performed in large part independent of regulatory issues. In addition, it is clear that novel technologies and approaches to medical research are outpacing the ability of our regulatory system to incorporate them into current review practices and guidelines. To overcome these obstacles, NIH should support strategic initiatives that are essential to the translation of NIH funded discoveries into diagnostic and therapeutics.

Emerging scientific opportunity ripe for Common Fund investment:   An investment in Regulatory Science will benefit all stakeholders by helping to advance and incorporate cutting-edge science into regulatory decision making and helping to develop improved tools, standards and approaches for assessing the safety, efficacy, quality and performance of medical products. Major advances in genomics and genomics-based medicine are also creating potential scenarios in the clinical setting that are relatively new to the FDA regulatory process. Moreover, the unprecedented partnership between the NIH and FDA through the Joint Leadership Council provides an extraordinary opportunity to coordinate therapy development efforts, including regulatory decision-making guidelines, between the two agencies. 

Common Fund investment that could accelerate scientific progress in this field: A number of scientific opportunities are ripe for investment in the area of Regulatory science and across the therapeutics development pipeline. For instance:

• Advances stemming from next generation sequencing technologies to help set the stage for personalized medicine, and other novel, genomics-based approaches to drug development, and treatments, such gene-editing, regulatory RNAs, gene –silencing or transcriptional activation
• Nanotechnology, microfluidics, and stem cell technologies to alter the way experimental agents are tested for safety and efficacy, and ultimately delivered to patients as therapies
• New approaches to predictive toxicology including high-throughput screening strategies/models, new in-silico approaches and computer-simulation models
• Development of bioinformatics tools and approaches for data mining and meta-analyses on safety and efficacy that capitalizes on the increasing volumes of clinical and medical product-related information in data repositories,
• Novel approaches of conducting clinical trials for rare and neglected diseases

Potential impact of Common Fund investment: Pre-clinical and clinical investigators, and other researchers who are engaged in the diagnostics and therapeutics development industries will benefit from having a more rapid integration of evidence-based knowledge into a regulatory framework, thereby quickening the pace at which basic science advances can move into the therapy development realm. For instance, in the area of stem-cell technologies, the NIH and FDA are working together to identify and define markers and characteristics of “stemness”, thus providing standards that the entire field can use for purposes of comparing studies and preparing for regulatory considerations.  The possibility of individualized, autologous utility of stem cell-derived therapeutics, organs, tissues, and other biomedical products are fast becoming a reality. Other emerging areas in regulatory science will advance, such as nanomedicine, personalized medicine, efficient and expeditious clinical trial designs, predictive toxicology, and biomimetic models that are able to simulate human conditions and better predict safety and efficacy.  The NIH-FDA joint efforts in these areas would help to pave a clearer and more transparent scientific and regulatory path for the scientific community that will impact therapeutics product development and clinical practice.