Monday, July 18, 2011

NCATS: Increasing the odds of getting to faster cures

By Margaret Anderson, Executive Director, FasterCures
This is old news: the drug development paradigm takes too long, it costs too much, and it’s laden with failures.

But for the thousands of people diagnosed each day with a disease for which there are no cures, or limited treatment options, the reasons – real as they may be – pale in comparison to the great need for living a healthy and productive life.

Of course we need to continue spotlighting the “broken R&D system” and need for a “new business model”. Identifying the problem is half the battle. But what are solutions to these problems? That is what matters most.

Solutions can be tough to develop, and tougher to implement. In the pursuit of solutions, we may second guess: Have we picked the “right” one? How can we hedge against risks? What about failure? Who is on board? Is it the right time to implement?

The road to success is paved with good intentions. It is clear that actions to address these vexing challenges will require bold vision, steadfast leadership, and diverse support. Thanks to our vitally important national investment in scientific discovery, we now know more about disease and biology than ever before.

The opportunity we must seize now is contingent upon having 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 we are in an era where the scientific knowledge and possibilities are abundant but they are getting stuck in the translational pathway, we have to get moving.

The proposed NIH National Center for Advancing Translational Sciences is one way to get moving. Knowing full well that there are no guarantees in the pursuit of treatments and cures, this proposed center was conceptualized to reengineer the process of developing diagnostics, devices, and therapeutics to increase our odds of success, and streamline a process to make it work better for all sectors and across all diseases.

In a paper published last week, NIH Director Collins laid out the possibilities that his proposed center could yield to reengineer the process of translational research . Among the specifics, he noted that the proposed NCATS will:

  • support broadly applicable rather than disease-specific target-validation approaches and the investigation of 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, as optimal treatment of many diseases is likely to require multiple therapeutic agents.

All of these are essential to all the sectors, and the NIH has the ability to share the insights and system improvements coming out of this work 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 does not become part of the road paved with good intentions. And we urge our Congressional leaders to support the establishment of this important effort.

Patients need these improvements in translational research, and that means you and me.

Relevant Links:1) Science Translational Medicine Commentary: Reengineering Translational Science: The Time Is Right
2) Podcast (MP3 - Dr. Collins summarizes the goals and functions of the proposed National Center for Advancing Translational Sciences)

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