Regulatory Science Program

Overview

The Problem

The tools and methodologies available to the pharmaceutical industry for drug development programs are outdated, inefficient, and non-informative. Regulatory agencies have identified gaps in regulatory science that should be addressed in order to accelerate medical product development.

In 2021, the European Medicines Agency (EMA) published a report on Regulatory Science Research Needs which outlines the gaps in regulatory science to improve medicine development and evaluation. This EMA report identified over 100 topics, such as validation of surrogate endpoints and biomarkers, predictive analytics to inform risk management, development of novel approaches and modeling to reduce animal testing, and utilization of real-world data, artificial intelligence and machine learning in regulatory decisions, with the intention of stimulating researchers and funding organizations to work towards addressing these challenges and accelerating medical product development.

Similarly, in 2022 the US Food and Drug Administration (FDA) published the Focus Areas of Regulatory Science (FARS) with the goals of modernizing development and evaluation of regulated products, strengthening post-market surveillance and labeling of regulated products, and invigorating public health preparedness and response. The FARS Report identified nearly 50 key regulatory science topics, including Women’s Health, Pediatric Health, Rare Diseases, Regenerative Medicine, Model-Informed Product Development, Patient-Reported Outcomes and others, of high relevance to supporting the FDA’s mission to protect and advance public health.

The Solution

The RegSci Program leads regulatory strategy development and brings together interdisciplinary scientific expertise from academia, industry, regulatory authorities, and patients to advance the adoption of DDTs in clinical trials and drug development programs. The Regulatory Science Program advances global regulatory policies by coordinating responses to FDA draft guidance documents and EMA public consultations. In addition, the RegSci Program supports global regulatory submissions of actionable solutions and seeks endorsements of new tools, standards, and methodologies that may be used to assess safety and efficacy of novel therapies.

The Impact

Through cross-functional collaborations with the Data Collaboration Center, Rare Disease Cures Accelerator-Data and Analytics Platform, Quantitative Medicine, and Regulatory Science, C-Path develops novel tools and methodologies, based on science and data-sharing, that modernize clinical trial design, increase efficiency of drug development, and inform regulatory decision making. 

Since its inception in 2005, C-Path has demonstrated itself to be a global leader in regulatory science by developing novel tools and methodologies that have been adopted by the pharmaceutical industry in the development of new medical therapies. Through collaborative efforts with regulatory authorities, pharmaceutical companies, and patients/patient groups, C-Path Consortia and Programs have led the development of novel trial simulation tools, disease progression models, novel biomarkers, endpoints, and outcome assessments.