NCATS sets disease treatment goals for next decade

Currently, only 5% of rare diseases have a treatment. Joni L. Rutter, PhD, Acting Director of the National Center for Advancing Translational Sciences (NCATS), wants to change that. Her vision is a future with more treatments for all people more quickly.

In January, NCATS held its 29th public advisory council meeting. In her Director's Report to Council, Rutter touched upon NIH and NCATS leadership changes, Rare Disease Day, the Advance Research Projects Agency for Health (ARPA-H), NCATS efforts to promote diversity, equity and inclusion (DEI), current NCATS research programs and the future of NCATS.

Setting three goals for the next decade, Rutter outlined the following:

More treatments in the pipeline

There are 7,000 rare diseases with more on the way, with only about 500 with therapies (5%). The goal is to go from 5% of rare diseases having treatments to 25%.

Dramatically increase inclusivity in all areas

Address health disparities among racial and ethnic populations, LGBTQ, and those in rural communities, prioritize development of strong community-engagement partnerships, train the next generation of translational scientists, DEIA emphasis in workforce, and improve commercial interest and market incentives for rare diseases.

Reduce by half the time for treatments to reach the market

Better predictive models, patient-centric designs, many-diseases-at-a-time strategies, train next-generation scientists to embrace team science, streamline and standardize administrative/operational processes, master protocols in clinical trials to test multiple drugs concurrently, drug repurposing, using data science and EHR data. N3C is showing how we can catalyze efforts for COVID, which is needed for other diseases as well.

Read Rutter's blog post, The Decade Ahead: More Treatments for All People More Quickly, for more details.