In recent decades, scientists have made rapid progress both in understanding countless diseases and in generating technologies that offer unprecedented potential to advance the translation of basic science discoveries into new medical treatments. However, most diseases have little or no treatment approved by the Food and Drug Administration (FDA), and the process of developing new therapeutics is fraught with uncertainty and failure...

Developing interventions for better human health differs from developing other consumer goods in a fundamental way: Interventions to improve health generally are developed without direct input from the people they are meant to benefit. I believe this odd fact of history is responsible for much of the inefficiency and ineffectiveness of translational science...

I often talk about how NCATS’ mission and programs are different from other organizations in the research ecosystem. One of these differences is that NCATS is “disease-agnostic”: rather than focusing on a single type of condition or biological system, we look for what is common among diseases and the translational process...

Understanding the characteristics and course of diseases in people, investigating the effectiveness and safety of new treatments, and devising ways to get treatments to all the people who need them are just some of the issues addressed in the clinical phase of the translational process. Like the preclinical phase I discussed in previous Director’s Messages in April and June, clinical translation is inefficient and poorly understood, leading to many lost opportunities for health improvement...

I often cite the essential components of translational science — collaboration, public-private partnerships, innovative research models — as common denominators of all NCATS initiatives. One of the most exciting parts of our work is when research teams demonstrate that these elements can produce breakthroughs in translation...

For basic research scientists, defining the role of a particular protein in health and disease — an early translational hurdle called “target qualification” — often is easier said than done. A chemical “probe” that can increase or decrease the activity of the target protein can be invaluable for target qualification...

NCATS’ work to develop, demonstrate and disseminate innovative tools and research approaches already is enabling great strides in advancing translational science. Our achievements to date would not have been possible without the diligent work of the diverse group of individuals serving on the NCATS Advisory Council...

In order for a new drug to be approved by the FDA for use in patients, it must be shown to be effective in clinical trials. Currently, more than 80 percent of new drugs tested in clinical trials fail, preventing therapies from reaching the medicine cabinet and causing enormous loss of time and expense. To address this translational roadblock, NCATS is developing new technologies to better predict which drugs will be safe and effective in clinical trials...

For many diseases, there is no single “magic bullet” therapy. Instead, combining two or more drugs is the standard of care for diseases such as cancer, tuberculosis and HIV/AIDS. Combinations of drugs can work better than a single therapy, providing patients with a more effective treatment while also making it more difficult for diseased cells and microbes to develop resistance...

As many as 25 million people in the United States are suffering from one of more than 6,500 rare diseases. Although each rare disease affects fewer than 200,000 Americans, in total, these illnesses affect a large part of our population. With NCATS’ emphasis on identifying commonalities among diseases as a route to accelerating the translational process, we are tackling the problems of rare disorders in an integrated way...

The Human Genome Project revolutionized science, not only because it identified all 3 billion letters of the human genome sequence, but also because all data from the project were made freely available to the public — a revolutionary idea at the time. The availability of these data has enabled scientists to develop better ways of understanding, diagnosing, treating and preventing disease, and it led to the development of entirely new areas of science...