April 26, 2013: Partnering with Colleagues in Industry to Accelerate Therapeutics Development
My previous Director’s Messages have emphasized that translation is a team sport, describing my ongoing discussions with leaders from academia, government, industry and patient advocacy groups. The feedback from these conversations helps NCATS focus its priorities on the areas of greatest need and build the teams necessary to solve complex translational science problems that can prevent and delay the development of health-improving interventions.
Last year, NCATS reached out to the Biotechnology Industry Organization (BIO) for its advice on the top challenges and opportunities in therapeutics, device and diagnostics development. BIO leaders canvassed the organization’s members to identify the system-wide translational problems they find most difficult and most important for NCATS to address. Following are some of the recommendations that resulted, and how NCATS’ programs address each area of focus.
Identify biomarkers that better predict how patients will respond to therapies. NCATS is working with disease experts in academia and industry and with the Food and Drug Administration (FDA) on ways to identify biomarkers, show how they can predict response to an intervention, and demonstrate their effective and efficient use in the regulatory approval process.
Develop predictive pre-clinical efficacy and toxicity testing methods. The majority of drug candidates fail in early (phase II) clinical trials because they don’t prove effective enough to treat a specific disease. But these candidates still can be a source for new medicines if researchers demonstrate that the drugs are safe in people. Finding other potential diseases to treat with these partially developed drugs is the goal for NCATS' Discovering New Therapeutic Uses for Existing Molecules program — an alliance involving eight pharmaceutical companies.
NCATS leads two federal collaborations designed to improve ways to predict how effective or toxic a potential drug might be in humans:
- The goal of the Toxicology in the 21st Century (Tox21) program, which includes collaborators from the FDA, Environmental Protection Agency and National Toxicology Program, is to develop data-driven protocols that predict which chemical compounds have the potential to disrupt processes in the human body that may lead to adverse health effects.
- NCATS' Tissue Chip for Drug Screening program is a partnership with the Defense Advanced Research Projects Agency and the FDA. Through this program, researchers are developing 3-D human tissue models that mimic the structure and function of human organs. Once developed, researchers will use these models to predict whether a candidate drug, vaccine or biologic agent is safe and effective in humans in a faster, more cost-effective way than current methods.
Improve patient recruitment, clinical trial design and the institutional review board (IRB) process. Patient recruitment, novel clinical trial design and IRB harmonization are three areas of focus for the Clinical and Translational Science Awards (CTSA) program that NCATS leads.
Take a leadership role in working with the FDA to develop and validate novel approaches to drug development. From its inception, NCATS has worked closely with colleagues at the FDA across a wide range of regulatory science issues. For example, in both the Tox21 and Tissue Chip for Drug Screening programs, experts at the FDA help us explore how these technologies can be used to assess drug effects prior to approval for first-in-human clinical studies.
I want to thank each of you in the academic, nonprofit, biotechnology, pharmaceutical and venture capital communities who have and continue to contribute time, experience and advice to help NCATS focus on the most pressing issues in translational science. I look forward to continuing these discussions with both current and new stakeholders so that NCATS can align its resources most effectively with the public and private sectors to speed scientific innovation and improve human health.
Christopher P. Austin, M.D.
National Center for Advancing Translational Sciences
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