Background and Meeting Objectives:
Recent years have seen both extraordinary opportunity and complex challenges in pharmaceutical innovation. New biomedical technology platforms are creating novel avenues for research and new opportunities for the discovery and clinical development of innovative diagnostics and therapies. Yet despite these advances, the pathway from basic science to new therapeutics faces challenges on many fronts. The translational divide results in only a small fraction of investigational new drugs reaching FDA approval and the patients who need them. New paradigms for discovering and developing drugs are being sought to bridge the ever-widening gap between scientific discoveries and translation of those discoveries into life-changing medications.
New collaborative approaches within the federal agencies, academia, and industry are directing focused attention on the advancement of the drug development enterprise. Among these initiatives is the Cures Acceleration Network (CAN), which was originally authorized in the Patient Protection and Affordable Care Act (P.L. 111-148) and was subsequently amended by the Consolidated Appropriations Act, FY 2012 (P.L. 112-74), which moved CAN to the newly authorized National Center for Advancing Translational Sciences (NCATS).
This is 1.5 day public workshop considered options and opportunities to maximize the usefulness and impact of the CAN program in order to advance translational sciences. In addition to providing suggestions to NCATS, the workshop was, in part, in response to Congressional interest in CAN expressed in the FY 2012 appropriations act conference report. The workshop informed NIH/NCATS in its efforts to implement CAN and advance translational sciences, and also informed the public, policy community, and other stakeholders as all of these parties continue to work to enhance the development and testing of therapies and diagnostics to patients. A summary was provided to NCATS and the newly established CAN Board to help it identify ways to accelerate and expand the number of cures.
Objectives
An ad hoc planning committee planned a public workshop that will explore ways to maximize the use-fulness and impact of CAN to advance translational sciences. Specifically, the workshop included consideration of the effective use of CAN authorities for the development of new tools and methods to enhance the development and testing of therapies and diagnos-tics to patients. The workshop featured invited presentations and discussions that will:
- Identify and catalog potential tools, methods, and approaches that hold promise foraccelerating translational science.
- Consideration of such promising approaches will draw from the experiences of existing activities at other federal agencies related to the goals of CAN (e.g., FDA, CDC, AHRQ).
- Discuss the authorities conferred to CAN and identify strategies for effectively using those authorities.
- Consideration of the CAN authorities will specifically explore the flexible research, or “other transactions”, authority and will reference existing efforts in which such authority is currently applied across other federal agencies (e.g., DARPA, DTRA, BARDA).
- Explore promising models for public-private collaborations that could be strengthened or facilitated by activities under CAN.
- Discuss barriers to such collaborations and identify opportunities and potential solutions for moving past the identified barriers.
- Discuss the respective roles of multiple sectors, including, e.g., biopharma, biotech, venture capital/private equity, and patient/disease advocacy.
- Identify barriers and potential solutions to facilitate coordination of activities under CAN with the FDA regulatory review process and timelines.
Planning Committee:
Carolyn Compton (Co-Chair), Critical Path Institute
Lous J. Degennaro (Co-Chair), The Leukemia & Lymphoma Society
Ann Bonham, Association of American Medical Colleges
C. Thomas Caskey, Baylor College of Medicine
William W. Chin, Harvard Medical School
Peter B. Corr, Celtic Therapeutics, LLP.
Paul R. Eisenberg, Amgen, Inc.
Kathy L. Hudson, National Institutes of Health
Ankit Mahadevia, Atlas Venture
Robert T. O'Niell, U.S. Food and Drug Administration
Barry Pallotta, Defense Advanced Research Protection Agency
Mary K. Pendergast, Pendergast Consulting
Ellen V. Sigal, Friends of Cancer Research
William L. Warren, Sanofi Pasteur
Below are materials for the workshop, including an agenda and presentations.
For additional information on the workshop, including the planning committee and sponsors, please visit our project page.