January 19, 2022: The Decade Ahead: More Treatments for All People More Quickly
Happy New Year! Every new year offers an opportunity for reflection and for looking ahead. Our recent 10th anniversary celebration highlighted the many accomplishments of NCATS’ scientists, grantees, and collaborators across the translational science spectrum. It also underscored our collective efforts to advance treatments for all diseases, especially those that are rare and intractable. We heard personal stories of how NCATS tools and collaborations have aided families searching for treatment solutions and helped give one family 509 more days with their young son, who had a rare disease. These are the stories that inspire and motivate us every day.
The anniversary celebration allowed us to look back and take stock of where we have been. Now, with that solid foundation, we can look to the future. My vision is a future with more treatments for all people more quickly.
More Treatments: Right now, only 5% of diseases have a treatment. This statistic has been frustratingly static for too long, and I want to change that. NCATS is poised and determined to ratchet up this number. One audacious goal is to get that number up so that 25% of known diseases have a treatment in the pipeline in the coming decade. We will use tools such as 3-D tissue bioprinting and tissue chips to develop more reliable models of human physiology and pathology that better predict toxicity and efficacy and speed drug development. The Platform Vector Gene Therapy (PaVe-GT) program and the Bespoke Gene Therapy Consortium (BGTC) will lead to many more gene therapies for rare diseases.
All People: All aspects of the biomedical research enterprise — including everything from training and career development to clinical trials and implementation — should reflect the diversity of our country, so that everyone can benefit from the results. NCATS is developing programs and initiatives to tackle health disparities in all areas we support, while also building an organizational culture that supports diversity, equity, inclusion, and accessibility. Clinical and Translational Science Awards (CTSA) Program institutions, which have long prioritized engaging communities in the research process, are leading the way with strategies that increase participation among underrepresented and vulnerable groups in clinical research. Additionally, the rare diseases community is focusing on solutions for equitable delivery of therapies. Many challenges remain, and I am committed to our working together to overcome them.
More Quickly: Today, it takes 10 to 15 years for a drug to make it to market. I hope we will pick up the pace! Tools that harness real-world data — such as the National COVID Cohort Collaborative (N3C) — are already helping us quickly explore and test critical clinical research questions. We will employ NCATS laboratories’ core technologies to find new therapeutic uses for existing medications and develop drug candidates. We will leverage the Streamlined, Multisite, Accelerated Resources for Trials (SMART) IRB Platform and the Trial Innovation Network to bring tailored methods and processes that address critical roadblocks in clinical trials. Another key strategy is tackling many diseases at the same time by finding and leveraging commonalities through such initiatives as the Rare Diseases Clinical Research Network and the Biomedical Data Translator.
We can achieve this vision of bringing more treatments to all people more quickly by inspiring bold ideas, impactful programs, and new partnerships. In upcoming Director’s Messages, I will expand on this vision, outlining challenges and opportunities. Progress will depend on collaborations that embrace productive risk taking, creativity, scientific excellence, and an engaged community, and I am eager to get your reactions, input, and ideas. Please share your thoughts, and stay tuned.
Your partner in science and health,
Joni L. Rutter, Ph.D.
National Center for Advancing Translational Sciences