The drug discovery, development, and regulatory approval process typically take up to 15 years. This lengthy time frame makes it hard to respond to emergencies requiring new therapies, deprives patients of faster access to life-saving medicines, decreases the profitability period of pharmaceutical companies, and contributes to the high cost of new medicines. Moreover, the COVID-19 pandemic has made clear the need for faster drug development and regulatory evaluation process.

To address this critical issue of national importance, we propose to organize a Pathfinding Workshop focused on two major goals:

1. To identify scientific, technological, and regulatory mechanisms capable of reducing this time frame without increasing patient risk, and

2. To integrate these mechanisms into a coherent strategy for accelerating the discovery, development, and regulatory approval process.

The proposed workshop will bring together a focused group of thought leaders from academia, industry, and regulatory agencies, to discuss the critical elements needed to accelerate drug discovery, development, and regulatory evaluation based on recent scientific and technical advances in medicine, biology, chemistry, materials science, biostatistics, and advanced manufacturing technologies. These discussions will be led by pre￾selected panelists with an established track record in their respective fields. Acceleration mechanisms will be examined from multiple perspectives, including the strength of the underlying science, technical feasibility, and regulatory acceptability. The systemic brainstorming and analysis with the participation of experts across the relevant disciplines will enable significant outcomes, which wouldn’t be achieved in any existing scientific communication forum or conferences.

Acceleration mechanisms deemed to be potentially effective will be selected for further analysis by a focused multidisciplinary team of workshop participants, which will be tasked with integrating these mechanisms into a strategic plan and an implementation pathway. These documents, which will be the main output of the workshop, will serve as a blueprint for the Federal Government, industry, and academia to direct efforts in this field.

Problem statement

The drug discovery, development, and regulatory evaluation process, from the point where a molecule is identified as a potential therapeutic agent, to the point where it is approved for commercialization as a drug product, can take up to 15 years. This lengthy process involves many stages, including the determination of efficacy, safety, toxicity, and therapeutic regime, identification of target populations, side effects, and interactions, selection of a feasible synthetic pathway, and development of a product formulation and a reliable manufacturing process.

Every step of this process is informed by science but it is also heavily laden with legacy practices. Industry and regulatory agencies are appropriately conservative and risk-averse. However, in recent years, many approaches have emerged that are capable of accelerating the discovery, development, and regulatory evaluation process while preserving patient safety, including bioassays that can determine tissue toxicity and tissue targeting, models that can predict individual absorption, distribution, metabolism, elimination, and toxicity, prediction of clinical response in diverse patient populations based on sub-clinical exposure, chemistry, manufacturing, and control strategies that can accelerate development, and advanced manufacturing methods that greatly accelerate product and manufacturing process development. In combination, these and other emerging approaches can substantially accelerate the preclinical and clinical development stages, and can also facilitate rapid and reliable regulatory evaluation.

However, these approaches are emerging in various disciplines and have not been integrated as part of an overall strategy for accelerating drug product development and regulatory evaluations.

The Pathfinder Workshop will achieve two goals:

Goal 1: To identify Scientific, Technological, and Regulatory barriers that prevent significant reductions in the drug discovery, development, and commercialization time frame.

Goal 2: To identify mechanisms capable of significantly reducing the time required for drug discovery, development, and commercialization, and integrate these into a coherent strategy and implementation plan with improved efficiency and reduced risks to patients.

Benefits of participating in the Workshop

The Workshop Steering Committee will use the material generated during the workshop to create a coherent Strategy and Implementation plan for accelerating Drug Development and Regulatory Approval. This document will be the main tangible output from the workshop. The workshop will lead to the creation of a blueprint for accelerating drug development and regulatory evaluation that will be enormously valuable to guide the selection of research activities by academics, resource allocation by federal agencies, and selection of strategies by Industry. The multidisciplinary discussions to be conducted before, during, and after the proposed workshop will synergize currently isolated efforts by workshop participants, potentially leading to the identification of opportunities for collaboration.