1. Priming the Pump: Filling the Therapeutic Pipeline
There is almost universal concern in the biomedical research enterprise about the declining productivity of the system. While there seems to be no shortage of scientific opportunity, there are precious few new products reaching the market, and even fewer genuine breakthroughs. Is innovative science enough these days, or are there management and financial innovations that need to go hand-in-hand? What are some of the novel ways companies are seeking to share risk with other stakeholders, from the government to universities to nonprofit organizations? How are these partnerships affecting industry's priorities and decision-making?
2. Collaboration: Secrets of Success
Partnering with other organizations - from for‐profit companies to government agencies, from academia to nonprofits - is not for the faint of heart. But the rewards can be huge. What must collaborators bring to the table? Funding? Scientific knowledge? Data? Patients? How can potential minefields, such as intellectual property and information sharing, be effectively navigated? Are there operational management tools that can help ease the way? This panel will feature lessons from the front lines and highlight best practice outcomes.
3. Who Will Pay for Tomorrow's Medical Breakthroughs?
The high cost and low success rate of medical R&D are causing many investors and companies to throw up their hands and cry that "someone else" needs to fund the earliest and riskiest stages of research. Who might that "someone else" be? Are there other sources of capital that can make a difference? Even if we are successful at getting new treatments through the R&D and approval process, the question remains whether payors will be willing to pick up the tab for them. What role are insurance coverage decisions - and insurers themselves - playing farther upstream in the R&D and investment process?
4. NIH Advances into the Valley of Death
With productivity in medical research in decline, NIH is seeking to take on a more expansive role in helping promising basic discoveries bridge the "valley of death" between preclinical development and clinical research. In 2011, NIH is on a fast track to establish a new National Center for Advancing Translational Science (NCATS), intended to "generate innovative tools, technologies, and ideas that would transform translational efforts throughout the scientific community." Is this an appropriate role for NIH to play? Will it/how will it affect NIH's basic research activities? What other programs or new initiatives by government funders could support translation?
5. Staying Afloat in a Sea of Data There is an enormous opportunity emerging from the confluence of three transformative breakthroughs—the completion of the Human Genome Project in 2003, the slow but steady rise in the use of electronic health records, and the expansion of health information technology infrastructure. But how do we make the most of these developments? How can scientists and clinicians possibly begin to make sense of all the new data from the human genome, for example, without more efficient means of linking these molecular data to other types of data about medicine and health status? And even if we can make sense of it, are the right policies are in place to govern how it is used, whether and how it is shared, and who owns it?
6. Regulating Risk: Clearing New Pathways for Progress at FDACompanies want FDA to approve their products faster. Patients want access to new therapies now. Consumer groups and elected officials want products to be risk-free. Science is moving forward more rapidly than the agency can account for its impacts on their decisionmaking process. While it needs more financial and human resources, no amount of money can get FDA through this thicket. What innovative models and initiatives are being discussed and pursued to bring fundamental change to the regulatory process? Can/should patient groups play a more active role in the dialogue about risk and benefit?
7. Living and Working in the Precompetitive Space
Over the last year there has been increasing interest within the biotech and pharmaceutical industries in collaborating more across companies and sectors on pre-competitive areas of research. But the concept remains ill-defined. Where does "pre-competitive" end and "competitive" begin? How should intellectual property be treated, and can it be handled in a standardized way? Can we create a "marketplace" that will help facilitate the exchange of pre-competitive information? Is there a business model that can be built around open innovation? What risk-and cost-sharing strategies are working when it comes to turning knowledge into drug candidates?
8. Streamlining Drug Repurposing and ApprovalIt used to be that finding new uses for existing drugs or abandoned compounds occurred primarily through serendipity, but more and more, the process for identifying potential "hits" is becoming more deliberate, less expensive, and faster. Strategic collaborations between the public, private, and nonprofit sectors are springing up to shepherd research efforts around abandoned compounds that might work in other diseases, and ensure the policies, processes, and resources are in place to permit their appropriate reuse. This panel will look especially at models and ideas for streamlining the regulatory process for repurposed assets to save time in clinical trials and review.
9. Experiments in Financing Early-Stage R&D Can we cross the Valley of Death - and accelerate medical solutions - by increasing and more strategically directing funding to early stage research alone? What financing mechanisms have been (or could be) successful at directing capital to early-stage research? Or do we need to fundamentally restructure the R&D ecosystem? If so, can we be successful by just reorganizing the current players (i.e., NIH, academics and research institutes, nonprofits, biotech, pharma, and investors)? Or do we need models that create new kinds of entities to provide the financial and human capital needed to bridge basic science to clinical application?
10. Philanthropy As Risk Capital
A new class of medical foundations known as "venture philanthropies" - organizations of a philanthropic nature that operate in venture-capital mode - are bringing a sense of urgency to the rest of the medical research community. By taking financial and strategic risks to turn discoveries in the lab into cures for disease, they are having an outsized effect on the development of new drugs, devices and diagnostics patients so desperately need. This panel will discuss recent high-risk, high-impact efforts of this community that are breaking down the barriers to medical progress including testing the boundaries of data sharing, creating responsible approaches to intellectual property, collaborating with industry and academia to repurpose shelved compounds, and charting new regulatory pathways.
11. Business Is from Mars, Academia Is from VenusUniversities are showing increasing interest in stretching their work into the "Valley of Death" to move their discoveries towards treatments. Companies are rethinking their alliances with universities as sources of innovation to fill their pipelines, beyond traditional sponsored research agreements. Where is such rethinking taking place, and are these models replicable? What are the challenges of this new kind of relationship?
12. Intellectual Property: The Lawyers' DebateEveryone in medical research seems to agree that intellectual property is a significant issue slowing progress, from disagreements over the value of discoveries to the time it takes for agreements to be negotiated and the aversion to standardized contracting. And everyone also agrees that it's all the lawyers' fault. This panel will feature lawyers from industry, academia, government, and the nonprofit sector who have managed to streamline the process and facilitate cross-sector deal structuring rather than hinder it.
13. Back to Basics: Patient Activism Thirty Years After HIV/AIDSThirty years ago HIV/AIDS activists banded together to create an advocacy movement that changed how research is conducted, how drugs are approved, and how patients engage with all areas of the federal government and the private sector. How can today's advocacy community apply lessons learned from this movement to effect change in the medical research paradigm? How can others make a similar transition from agitators to informed partners with a meaningful seat at the table? Where are today's activists pushing the research envelope?
14. Convergence: The Death of Disciplinary ScienceThere is a budding belief in some pockets of academia that the merging of life, physical and engineering sciences will herald a "Third Revolution" in biomedical research, and speed the path to personalized, accessible, and affordable health care. Called Convergence, this new model emphasizes integration between disciplines that have traditionally been separate and distinct. With tangible outputs ranging from the creation of a CTC Chip for cancer metastases detection to the development of brain grafts to treat victims of traumatic brain injury, advocates are calling for a pipeline of convergence-oriented researchers to be created. Is this the future of medicine, or the latest scientific fad? What would it portend for the funding and conduct of the entire medical research enterprise?
15. The Future of Research: The Kids Are AlrightBy now the statistics are familiar: The average age of an investigator receiving his or her first NIH grant is 42 years old, while most Nobelists did their prize-winning work before they were 40. NIH is funding significantly more investigators over the age of 60 than under the age of 40. How are we going to attract and keep young people in the system? Who will fund their work? How is their way of working - the use of technology, social networking, collaborative work, data sharing, crowdsourcing, interdisciplinary science - going to change the research establishment of tomorrow?
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