Usama Malik, VP, Head of Innovation, Pfizer
Adam Bosworth, CEO, Keas
Robert Shelton, CEO, Private Access

What? This session will focus on two case studies addressing the challenges of pursuing "big ideas" at a time when investment capital for startup companies is down significantly, and the role of for-profit companies (both large and small) and condition-specific organizations (CSOs) in bringing such ideas to market.

The New Thinking: The session will describe ways in which to foster greater collaboration between Fortune 50 companies, small start-up firms, CSOs and visionaries. The panel will address how complementary strengths and enlightened self-interests make it possible for these firms and non-profit groups to help address one another's needs - and the needs of the patients whom each serves. Participants will address how the world's largest pharmaceutical company, two visionary startup companies and condition-specific organizations work together; what each firm gets - and aspires to get - from the partnership; and the challenges involved in creating and maintaining a vibrant collaboration. Here's one secret: It's about a lot more than the money! Suggestions will be made for how other entrepreneurs with big ideas might consider approaching a firm such as Pfizer; and how other large companies seeking to fuel innovation might work in collaboration with leading-edge pioneers such as Keas and Private Access as well as leading CSOs to speed positive transformation of the system.

Steven Walkley, Albert Einstein College of Medicine
Jonathan Jacoby, CEO, The R.A.R.E Project
Chris Austin, Director, NIH Chemical Genomics Center

What? SOAR-NPC (Support Of Accelerated Research for Niemann Pick Disease, Type C) works to develop treatments for NPC.

The New Thinking: As science and technology advance, expectations of parents are raised for accelerated research success to help their children. At the same time, the potential for disappointment, frustration and "fickleness" increases, sometimes to tangential and too-risky attempts to further accelerate the process. The SOAR partnership of researchers and parent-funders seeks to provide researchers with a better understanding of the motivations and potential contributions from patient advocates/funders/parents, and visa versa. This has led to accelerated studies and a more comprehensive and strategic focus on therapy development.

Santa Tumminia, Senior Science Advisor for Translational Research,
National Eye Institute, NIH
Joan O’Brien, Chair, Department of Ophthalmology,
Scheie Eye Institute, UPENN
Brad Williams, Genetic Counselor, GeneDx

What? The eyeGENE® Network was established as a bidirectional genetics initiative whereby a clinical component for patients who have inherited eye disease, enables and fosters research into the causes and mechanisms of these ophthalmic diseases.

The New Thinking: NIH had to be convinced that this fell within the realm of the NIH, eye health care providers had to change the way they perceived Standard of Care for their patients. They also had to refer their patients to the Network and be responsible for providing a standard set of clinical criteria for each patient, gratis! Researchers and clinicians with patients with inherited diseases needed to be convinced that no one was going to "steal" patients from them.

Richard Aragon, Director, Innovative Molecular Analysis Technologies, NCI, NIH

What? The purpose of the IMAT Program is to empower translational research through targeted (and perhaps disruptive) innovation in an attempt to achieve this New Thinking:

The New Thinking: Fostering cross-collaborations between individuals from traditionally disparate disciplines such that common and pressing technical barriers that impede progress in cancer research are addressed, soliciting and supporting the maturation of highly innovative, unconventional, high-risk but also potentially high-payoff technologies from the scientific and clinical communities.

Understanding that we must all change what we are thinking and doing, participants will engage in breakout groups that will envision viable pilot projects designed to increase shared infrastructure, the commons, and/or resources, both general and specific. Four pre-planned breakouts are listed here, other spontaneous breakouts may arise and are welcome.

Picking Low Hanging Fruit
Led by Stephen Friend, Sage Bionetworks

If I were to found a group to impact a disease, identify a diagnostic or generate therapies, no one in their right mind would set it up using a RO1 based world where tenure is provided the way it is, or data is shared the way it is today. Starting from a long-term goal capable of empowering a group to impact their disease area what barriers can be identified that fracture or limit it from success. This session will focus on identifying what steps might be taken to speed how disease research is performed.

Launching the Global Genes Fund - A collaborative fundraising platform for accelerated R&D
Led by Nicole Boice, The R.A.R.E. Project and the Children's Rare Disease Fund and Jonathan Jacoby, The R.A.R.E. Project

We will generate a set of criteria for choosing—and helping to identify—the first group of research projects that will be promoted within the Global Genes Fund, the purpose of which is to support accelerated collaborative research that will bring effective treatments and therapies to children within their lifetimes.

Gateway to Rare and Neglected Disease Therapeutics (GRANDRx)
Led by Margaret Anderson, FasterCures and James O’Leary, Genetic Alliance with Terence Ross, Crowell and Moring and Claire Driscoll, National Human Genome

Research Institute
Rare and neglected disease research faces many challenges, including the transfer of skills and sharing of knowledge, technologies and products that could accelerate development of treatments across these diseases. We will focus on the challenges and opportunities inherent in technology transfer. In this session, together we will 1) identify and understand the key issues in technology transfer as it facilitates and impedes forward progress, 2) examine the recent culture shifts around open networks and collaboration and what makes them successful, 3) consider potential solutions to allow sharing a “win-win” for all parties, and 4) chose a collaborative activity that can mitigate the problems.

A multi-disciplinary initiative to develop a methodological framework for comparative effectiveness research (CER) in rare diseases
Led by Sean Tunis, CEO, Center for Medical Technology Policy

The new institute for comparative effectiveness research (CER) established in the health reform bill (the Patient-Centered Outcomes Research Institute) includes a provision that would establish an expert advisory panel to focus on the design and feasibility of CER for rare diseases. Very little thought has been giving to CER methods for rare conditions, and it would be useful to begin the work of developing a set of principles that could be provided to this expert advisory panel once it is constituted. This working session will begin to identify experts, publications, concepts, and a workplan to develop these initial principles. Special attention will be directed to considering how CER for rare diseases can be approached in ways that are genuinely “patient-centered”, and fulfill the primary purpose of CER, which is to assist patients and clinicians in making informed health care decisions.