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August 17, 2022: New Treatments Twice as Fast

This Director’s Message is one installment in a four-part series that lays out NCATS’ goals for bringing more treatments to all people more quickly.

More treatments to all people more quickly. These are NCATS’ audacious goals, and this Director’s Corner message focuses on the third one: more quickly. Picking up the pace of therapeutic development and delivery is essential to our mission — the sine qua non of translational science. We want to double the speed over the next decade.

A new drug’s journey from the lab to the medicine cabinet averages 10–15 years. Our Drug Discovery, Development, and Deployment Maps show more than 100 steps — and therefore 100 potential failure points — from target identification through clinical implementation. The examples below highlight long-standing crimps in the translational pipeline and some of our solutions to develop and deliver new treatments twice as fast.

Leveraging Big Data and Real-World Evidence

Traditional methods for accumulating and accessing data are restrictive and inefficient, creating a significant crimp in the pipeline. Real-world evidence approaches are increasing harmonization, interoperability, and interpretability to transform research data into health knowledge and clinical evidence faster than ever before.

The NCATS National COVID Cohort Collaborative (N3C) continues to demonstrate the power of using harmonized electronic health record data to explore emerging public health questions. For example, N3C-enabled work on long COVID found that vaccination before or after acute SARS-CoV-2 infection reduced the likelihood of needing care in a long COVID clinic and that difficulty breathing was one of the biggest risk factors for seeking specific long COVID care. The study was accomplished on a timeframe of weeks, delivering insights that would take months or years to gain with traditional study designs. We’re using the N3C and the speed and power of its analytics capabilities to ask new COVID-19 questions, such as Who’s experiencing clinical rebounds after taking Paxlovid, and How often are reinfections occurring?

The NCATS Biomedical Data Translator recently showed synthesizing data from a variety of sources quickly led researchers to therapeutic leads for drug-induced liver injury. Translator revealed that the drugs already had been tested in animal models and safely used in clinical trials for other diseases, spurring their advancement through the pipeline. This is one of many stories demonstrating Translator’s ability to bridge data silos to improve health across diseases.

Streamlining Regulatory Processes

Generating the preclinical and clinical data needed for new drugs to get regulatory approval is a critical and time-consuming step. Repurposing existing drugs, which have already undergone rigorous safety studies, serves to open an early crimp in the pipeline and can reduce therapeutic development time to 1–2 years. The NCATS Pharmaceutical Collection of approved drugs, for instance, has enabled researchers to find leads for rare and common diseases and move them quickly into preclinical and clinical testing (see this publication for examples).

A new area of interest for us is antisense oligonucleotides (ASOs), which hold great promise as a precision medicine therapy approach for many rare diseases. A significant bottleneck in ASO drug development is the animal toxicology experiments required by the U.S. Food and Drug Administration (FDA). We have established an RNA Therapeutics Laboratory at NCATS that is aimed at creating and validating a low-cost predictive assay platform that streamlines preclinical costs and time for toxicology studies. This program, along with the streamlined development of customized gene therapies, could allow us to quicken the pace of developing rare disease treatments over the next decade.

Standardizing Business Processes

All organizations involved in collaborative studies must negotiate and approve intellectual property arrangements, terms and conditions, and roles and responsibilities. These agreements create an underappreciated crimp in the pipeline when done de novo by each institution. Our Office of Strategic Alliances has streamlined the process by creating templated agreements. Using these templates, we facilitated 400 agreements in the last year alone.

The Clinical and Translational Science Awards Program has mastered this art to enable clinical translational science. Its master institutional review board reliance agreement for multisite clinical studies now has 1,000 participating sites and cuts the start-up time from months to weeks. Master protocols, which we use for our ACTIV trials and rare disease studies, remove another crimp in the pipeline because they provide a single, adaptive structure and design for multiple substudies running in parallel. They represent a coordinated, efficient approach that FDA leaders noted is a way forward as targets for new drugs become more precise.

Onward!

Smoothing out the crimps in the translational pipeline is a key strategy for tackling all of our audacious goals: (1) more treatments, (2) all people, (3) more quickly. Collaboration is critical to our success, and my next Director’s Corner message will turn to the important role of our stakeholder community — including YOU! If you or your organization would like to hear more about NCATS, please reach out.

Your partner in science and health,

Joni L. Rutter, Ph.D.
Acting Director
National Center for Advancing Translational Sciences

Last updated on October 12, 2023