Accelerating the Development of New Drugs and Diagnostics: Maximizing the Impact of the Cures Acceleration Network: Workshop Summary (2012)

Appendix A

Workshop Agenda

Maximizing the Goals of the Cures Acceleration Network to Accelerate the Development of New Drugs and Diagnostics: An Institute of Medicine Workshop

June 4—5, 2012

National Academy of Sciences
Keck Building, Room 100
500 Fifth Street, NW
Washington, DC 20001

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 public workshop will consider 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 is, in part, in response to congressional interest in CAN expressed in the FY 2012 appropriations act conference report. The workshop will inform NIH/NCATS in its efforts to implement CAN and advance translational sciences, and will also inform 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. The summary will be provided to NCATS and the newly established CAN Board to help it identify ways to accelerate and expand the number of cures.

The workshop objectives are to:

• Identify and catalog potential tools, methods, and approaches that hold promise for accelerating 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.

JUNE 4, 2012
DAY ONE

Session I: Overview of NIH Translational Sciences and the Cures Acceleration Network

Session Objectives:

• Provide an overview of the translational science initiatives at NIH.
• Provide an overview and description of the Cures Acceleration Network program goals and authorities.

Session II: Approaches to Accelerating Translational Science

Session Objectives:

• Through discussion of case examples and other mechanisms, identify potential approaches that hold promise for accelerating translational science, highlighting approaches that could potentially benefit from the new CAN authorities.
• Discuss and identify attributes of success stories and failures.

Session III: Application of Matching Authority

Session Objectives:

• Explore existing efforts in which the matching authority, or similar match-type requirement, is currently applied across other federal and state agencies.
• Examine benefits and advances that have been achieved through use of this authority.
• Discuss how barriers to application and use of those authorities have been overcome.

Session IV: Application of Flexible Research Authority

Session Objectives:

• Explore existing efforts in which the flexible research (or similar) authority is currently applied across other federal agencies.
• Examine benefits and advances that have been achieved through use of these authorities.
• Discuss how barriers to application and use of those authorities have been overcome.

JUNE 5, 2012
DAY TWO

Session V: Situating CAN Within the Drug Development Ecosystem

Session Objectives:

• Identify potential approaches to facilitate coordination of activities under CAN with FDA regulatory science initiatives and activities.
• Discuss existing activities in multiple sectors and address ways to maximize CAN impact in the drug development ecosystem.
• 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.

Session VI: Concluding Panel Discussion: Principles for Deployment of CAN Authorities

Session Objectives:

• Discuss key themes from the workshop.
• Based on workshop presentations and discussions, identify principles for deployment of CAN authorities.