Of course we need to continue spotlighting the "broken R&D system" and need for a "new business model." After all, identifying the problem is half the battle. But it's time to shift our focus to real solutions.
Solutions can be tough to develop and tougher to implement. They invite second-guessing: Have we picked the right one? How can we hedge against risks? What about failure? Who's on board? Is this the right time to implement? It is clear that moving toward action will require bold vision, steadfast leadership and diverse support.
Thanks to our vitally important national investment in scientific discovery, we know more about disease and biology than ever before. Now we need systems in place that will allow these ideas and discoveries to be translated into effective products and therapies that will ultimately improve patients' health and quality of life. If indeed the scientific knowledge and possibilities are abundant but they're getting stuck in the translational pathway, we have to get moving.
The proposed NIH National Center for Advancing Translational Sciences (NCATS) is one way to jumpstart progress. Since there are no guarantees in the pursuit of treatments and cures, the NIH envisions creating an entity that would reengineer the process of developing diagnostics, devices and therapeutics to increase the odds of success. It would attempt to streamline the translational process to make it work better for all sectors and across all diseases.
- support broadly applicable rather than disease-specific target-validation approaches and investigate nontraditional therapeutic targets that are considered too risky for industry investment.
- encourage innovations in chemistry for drug delivery, such as nanoparticles, imaging agents for use as biomarkers, and detection technologies for use in diagnostics.
- aim to develop more reliable efficacy models that are based on access to biobanks of human tissues, use of human embryonic stem cell and induced pluripotent stem cell models of disease, and improved validation of assays.
- serve as an honest broker for matchmaking between compounds that have been abandoned by industry before approval and new applications for which these compounds might show efficacy.
- support innovative designs for testing combination therapies, because optimal treatment of many diseases is likely to require multiple therapeutic agents.
All of these strategies are essential to success, and the NIH has the ability to share the insights and system improvements emerging from the NCATS across every entity in the medical research system.
But the NIH can't go it alone. The entire medical research community must come together to ensure this proposed center is effective in achieving its mission. Patients need these improvements in translational research, and we urge congressional leaders to support the establishment of this important effort.