EDITORIAL BENCH TO BEDSIDE
Partnerships: Beyond the Buzzword
Maria C. Freire is President and Executive Director of the Foundation for the U.S. National Institutes of Health (FNIH), Bethesda, MD 20814, USA. E-mail: foundation@ fnih.org
CREDIT: FNIH
10.1126/scitranslmed.3009673
Downloaded from http://stm.sciencemag.org/ on November 15, 2015
Citation: M. C. Freire, Partnerships: Beyond the buzzword. Sci. Transl. Med. 6, 242ed15 (2014).
SPURRED BY INNOVATIONS IN TECHNOLOGY, BIOLOGICAL KNOWLEDGE, AND MEDI cal need, the research landscape has become—by design and necessity—increasingly multidisciplinary and complex (1). Multiple forces, including the complexity of science and the regulatory demands that allow new products to enter the market, are shifing precompetitive private-sector research from internal investment to external collaborations. And although we have seen a recent uptick in investment in the biotechnology sector, funding for earlystage, high-risk research remains scarce. Combined with tangible, shortsighted shrinking of federal support for the U.S. National Institutes of Health (NIH), which has experienced a dwindling of the pool of funds for research grants and training of the next generation of biomedical researchers, the stark economic reality and vastly promising scientifc horizon demand—indeed, propel—us to craf new ways of doing business. “Partnership” has become a buzzword in the biomedical research community, even to the point of some talk of “consortia fatigue” (2). But such collaborations, in fact, are essential for fostering innovation and the development of creative new biomedical research paradigms. Partnerships, particularly alliances between the public and private sectors, embody powerful mechanisms to address the changing funding landscape and to tackle biological questions that cannot be resolved by a single player. Tey allow for a framework to share resources, risks, experience, and expertise; help overcome knowledge fragmentation; generate a common language and scientifc consensus; enable shared decision-making; and align incentives for multiple stakeholders to support sustainable innovation. Partnerships are particularly valuable in translational science, because they address information gaps in the research and development (R&D) pipeline that prevent fundamental biomedical discoveries from evolving into new, improved, or more precise treatments and preventative interventions. Within the next decade, research partnerships will become more than useful tools: Tey will be necessary for timely, clinically meaningful progress. MODEL SYSTEM Given the growing necessity of partnerships, it makes sense to design initiatives in a strategic way. Features of efective, diverse, multistakeholder partnerships can be gleaned by studying mature but innovative initiatives, including many led by the Foundation for the NIH (FNIH; www.fnih.org). A 501(c)(3) not-for-proft corporation, FNIH was established by the U.S. Congress in 1990 to support NIH in its mission and to advance collaboration with biomedical researchers from universities, industry, and nonproft organizations. FNIH is one of a very few charitable organizations established by Congress and afliated with a government agency that afords fundraising and partnering capabilities to support the mission of the federal institution. FNIH’s contributions over the past 17 years demonstrate that partnership mechanisms can work. Programs span the scientifc spectrum from fundamental biomedical research through translational and clinical R&D, also reaching into the education, training, and policy realms. FNIH serves as a linchpin for numerous collaborative studies. Examples include the Sports and Health Research Program, a partnership of NIH, the National Football League, and FNIH that is accelerating research on traumatic brain injury and concussion; Lung-MAP, a multidrug clinical trial that targets specifc genetic mutations in lung cancer; and the Alzheimer’s Disease Neuroimaging Initiative (ADNI; www.adni-info.org), a naturalistic, longitudinal study of the progression of AD. Indeed, many of FNIH’s research programs have a distinctly translational focus because this niche provides a fertile environment for the development of innovative, adaptable processes and tools for partnership creation, management, and exit. Although the programs difer in size, scope, and duration, all FNIH programs incorporate several basic principles that provide the infrastructure upon which the Foundation builds and tailors its approach to meet the needs of each collaborative efort. First, the quality of the science is paramount and is the nucleus for the overlapping interests of multiple stakeholders. Second, FNIH assumes the role of independent convener and manager of the partnership, thereby promoting and ensuring an environment of trust (essential for a level www.ScienceTranslationalMedicine.org 25 June 2014 Vol 6 Issue 242 242ed15
1
EDITORIAL playing feld where partners disparate in scale, scope, and resources can interact); open, frequent communication among partners; agreement on the criteria for success at an early stage of program development, including creation of milestones; centralized government and project management; and the highest level of ethical standards. Tird, it is essential to secure the involvement of a strong scientifc champion or principal investigator who is a subject-area expert and a passionate advocate for the project with the ability to “rally the troops.” Last, fexible fnancing models are required that can adapt to myriad parameters, including the number and diversity of stakeholders and the timing of funding needs.
Downloaded from http://stm.sciencemag.org/ on November 15, 2015
NEW MEDICINES: FAST FORWARD Te Accelerating Medicines Partnership (AMP) is a newly developed, innovative FNIH translational science program that taps into the Foundation’s capabilities and lessons learned (www.nih.gov/science/amp/index.htm). Te NIH, the U.S. Food and Drug Administration, 10 biopharmaceutical companies, several not-for-proft organizations, and FNIH are focusing on the identifcation and validation of disease targets in three common disease areas: AD, type 2 diabetes (T2D), and autoimmune disorders [specifcally, rheumatoid arthritis (RA) and systemic lupus erythematosus (SLE)]. Te need for better validation of therapeutic targets is well documented; much of the failure rate of new drugs in late-phase clinical trials and the associated increase in costs and time to regulatory approval can be traced to an inadequate understanding of the biological basis of human disease, resulting in the selection of inappropriate molecular targets (3). Using a governance model tailored to meet program requirements, AMP is pooling the expertise and resources of its government and private-sector partners to execute projects that promise better characterization of therapeutic targets in its selected disease areas. Data and analyses will be made broadly available to the research community. Tus, AMP’s design includes many of the features of successful partnerships: clear unmet medical needs, diverse stakeholders, sharing with the wider scientifc community, and clearly defned milestones and deliverables that are relevant to all participating sectors. Research plan topics for AMP include validating biomarkers of disease within NIA-funded AD clinical trials, conducting targeted sequencing of highpriority targets of interest in T2D, and establishing pathway and network maps of RA and SLE. Trough membership on disease-focused steering committees and with representation on an executive committee, all AMP partners are integrated into the oversight and decisionmaking processes for AMP. Te increasing uncertainty in federal and corporate funding of biomedical research shows every possibility of becoming the new normal, as industry and government dollars will likely fuctuate for years to come. One important way to make the most of every research dollar is to support mechanisms that enable committed scientists from all sectors to unite around common research goals. Organizations like FNIH serve as vital conduits for identifying scientifcally and clinically relevant problems and opportunities, establishing agile mechanisms for efective collaboration and communication, and enabling the sharing of data and expertise to jointly address medical needs—all with the goal of transforming the way we conduct biomedical translation. – Maria C. Freire
1. M. D. Lim, Consortium sandbox: Building and sharing resources. Sci. Transl. Med. 6, 242cm6 (2014). 2. M. Papadaki, G. Hirsch, Curing consortium fatigue. Sci. Transl. Med. 5, 200fs35 (2013). 3. E. A. Zerhouni, Turning the Titanic. Sci. Transl. Med. 6, 221ed2 (2014).
www.ScienceTranslationalMedicine.org 25 June 2014 Vol 6 Issue 242 242ed15
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Partnerships: Beyond the Buzzword Maria C. Freire (June 25, 2014) Science Translational Medicine 6 (242), 242ed15. [doi: 10.1126/scitranslmed.3009673] Editor's Summary
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Visit the online version of this article to access the personalization and article tools: http://stm.sciencemag.org/content/6/242/242ed15 The editors suggest related resources on Science's sites: http://stm.sciencemag.org/content/scitransmed/6/242/242cm6.full http://stm.sciencemag.org/content/scitransmed/5/200/200fs35.full http://stm.sciencemag.org/content/scitransmed/5/186/186fs18.full http://stm.sciencemag.org/content/scitransmed/3/89/89cm16.full Obtain information about reproducing this article: http://www.sciencemag.org/about/permissions.dtl
Science Translational Medicine (print ISSN 1946-6234; online ISSN 1946-6242) is published weekly, except the last week in December, by the American Association for the Advancement of Science, 1200 New York Avenue, NW, Washington, DC 20005. Copyright 2015 by the American Association for the Advancement of Science; all rights reserved. The title Science Translational Medicine is a registered trademark of AAAS.
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Partnerships: Beyond the Buzzword
Maria C. Freire is President and Executive Director of the Foundation for the U.S. National Institutes of Health (FNIH), Bethesda, MD 20814, USA. E-mail: foundation@ fnih.org
CREDIT: FNIH
10.1126/scitranslmed.3009673
Downloaded from http://stm.sciencemag.org/ on November 15, 2015
Citation: M. C. Freire, Partnerships: Beyond the buzzword. Sci. Transl. Med. 6, 242ed15 (2014).
SPURRED BY INNOVATIONS IN TECHNOLOGY, BIOLOGICAL KNOWLEDGE, AND MEDI cal need, the research landscape has become—by design and necessity—increasingly multidisciplinary and complex (1). Multiple forces, including the complexity of science and the regulatory demands that allow new products to enter the market, are shifing precompetitive private-sector research from internal investment to external collaborations. And although we have seen a recent uptick in investment in the biotechnology sector, funding for earlystage, high-risk research remains scarce. Combined with tangible, shortsighted shrinking of federal support for the U.S. National Institutes of Health (NIH), which has experienced a dwindling of the pool of funds for research grants and training of the next generation of biomedical researchers, the stark economic reality and vastly promising scientifc horizon demand—indeed, propel—us to craf new ways of doing business. “Partnership” has become a buzzword in the biomedical research community, even to the point of some talk of “consortia fatigue” (2). But such collaborations, in fact, are essential for fostering innovation and the development of creative new biomedical research paradigms. Partnerships, particularly alliances between the public and private sectors, embody powerful mechanisms to address the changing funding landscape and to tackle biological questions that cannot be resolved by a single player. Tey allow for a framework to share resources, risks, experience, and expertise; help overcome knowledge fragmentation; generate a common language and scientifc consensus; enable shared decision-making; and align incentives for multiple stakeholders to support sustainable innovation. Partnerships are particularly valuable in translational science, because they address information gaps in the research and development (R&D) pipeline that prevent fundamental biomedical discoveries from evolving into new, improved, or more precise treatments and preventative interventions. Within the next decade, research partnerships will become more than useful tools: Tey will be necessary for timely, clinically meaningful progress. MODEL SYSTEM Given the growing necessity of partnerships, it makes sense to design initiatives in a strategic way. Features of efective, diverse, multistakeholder partnerships can be gleaned by studying mature but innovative initiatives, including many led by the Foundation for the NIH (FNIH; www.fnih.org). A 501(c)(3) not-for-proft corporation, FNIH was established by the U.S. Congress in 1990 to support NIH in its mission and to advance collaboration with biomedical researchers from universities, industry, and nonproft organizations. FNIH is one of a very few charitable organizations established by Congress and afliated with a government agency that afords fundraising and partnering capabilities to support the mission of the federal institution. FNIH’s contributions over the past 17 years demonstrate that partnership mechanisms can work. Programs span the scientifc spectrum from fundamental biomedical research through translational and clinical R&D, also reaching into the education, training, and policy realms. FNIH serves as a linchpin for numerous collaborative studies. Examples include the Sports and Health Research Program, a partnership of NIH, the National Football League, and FNIH that is accelerating research on traumatic brain injury and concussion; Lung-MAP, a multidrug clinical trial that targets specifc genetic mutations in lung cancer; and the Alzheimer’s Disease Neuroimaging Initiative (ADNI; www.adni-info.org), a naturalistic, longitudinal study of the progression of AD. Indeed, many of FNIH’s research programs have a distinctly translational focus because this niche provides a fertile environment for the development of innovative, adaptable processes and tools for partnership creation, management, and exit. Although the programs difer in size, scope, and duration, all FNIH programs incorporate several basic principles that provide the infrastructure upon which the Foundation builds and tailors its approach to meet the needs of each collaborative efort. First, the quality of the science is paramount and is the nucleus for the overlapping interests of multiple stakeholders. Second, FNIH assumes the role of independent convener and manager of the partnership, thereby promoting and ensuring an environment of trust (essential for a level www.ScienceTranslationalMedicine.org 25 June 2014 Vol 6 Issue 242 242ed15
1
EDITORIAL playing feld where partners disparate in scale, scope, and resources can interact); open, frequent communication among partners; agreement on the criteria for success at an early stage of program development, including creation of milestones; centralized government and project management; and the highest level of ethical standards. Tird, it is essential to secure the involvement of a strong scientifc champion or principal investigator who is a subject-area expert and a passionate advocate for the project with the ability to “rally the troops.” Last, fexible fnancing models are required that can adapt to myriad parameters, including the number and diversity of stakeholders and the timing of funding needs.
Downloaded from http://stm.sciencemag.org/ on November 15, 2015
NEW MEDICINES: FAST FORWARD Te Accelerating Medicines Partnership (AMP) is a newly developed, innovative FNIH translational science program that taps into the Foundation’s capabilities and lessons learned (www.nih.gov/science/amp/index.htm). Te NIH, the U.S. Food and Drug Administration, 10 biopharmaceutical companies, several not-for-proft organizations, and FNIH are focusing on the identifcation and validation of disease targets in three common disease areas: AD, type 2 diabetes (T2D), and autoimmune disorders [specifcally, rheumatoid arthritis (RA) and systemic lupus erythematosus (SLE)]. Te need for better validation of therapeutic targets is well documented; much of the failure rate of new drugs in late-phase clinical trials and the associated increase in costs and time to regulatory approval can be traced to an inadequate understanding of the biological basis of human disease, resulting in the selection of inappropriate molecular targets (3). Using a governance model tailored to meet program requirements, AMP is pooling the expertise and resources of its government and private-sector partners to execute projects that promise better characterization of therapeutic targets in its selected disease areas. Data and analyses will be made broadly available to the research community. Tus, AMP’s design includes many of the features of successful partnerships: clear unmet medical needs, diverse stakeholders, sharing with the wider scientifc community, and clearly defned milestones and deliverables that are relevant to all participating sectors. Research plan topics for AMP include validating biomarkers of disease within NIA-funded AD clinical trials, conducting targeted sequencing of highpriority targets of interest in T2D, and establishing pathway and network maps of RA and SLE. Trough membership on disease-focused steering committees and with representation on an executive committee, all AMP partners are integrated into the oversight and decisionmaking processes for AMP. Te increasing uncertainty in federal and corporate funding of biomedical research shows every possibility of becoming the new normal, as industry and government dollars will likely fuctuate for years to come. One important way to make the most of every research dollar is to support mechanisms that enable committed scientists from all sectors to unite around common research goals. Organizations like FNIH serve as vital conduits for identifying scientifcally and clinically relevant problems and opportunities, establishing agile mechanisms for efective collaboration and communication, and enabling the sharing of data and expertise to jointly address medical needs—all with the goal of transforming the way we conduct biomedical translation. – Maria C. Freire
1. M. D. Lim, Consortium sandbox: Building and sharing resources. Sci. Transl. Med. 6, 242cm6 (2014). 2. M. Papadaki, G. Hirsch, Curing consortium fatigue. Sci. Transl. Med. 5, 200fs35 (2013). 3. E. A. Zerhouni, Turning the Titanic. Sci. Transl. Med. 6, 221ed2 (2014).
www.ScienceTranslationalMedicine.org 25 June 2014 Vol 6 Issue 242 242ed15
2
Partnerships: Beyond the Buzzword Maria C. Freire (June 25, 2014) Science Translational Medicine 6 (242), 242ed15. [doi: 10.1126/scitranslmed.3009673] Editor's Summary
Article Tools
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Visit the online version of this article to access the personalization and article tools: http://stm.sciencemag.org/content/6/242/242ed15 The editors suggest related resources on Science's sites: http://stm.sciencemag.org/content/scitransmed/6/242/242cm6.full http://stm.sciencemag.org/content/scitransmed/5/200/200fs35.full http://stm.sciencemag.org/content/scitransmed/5/186/186fs18.full http://stm.sciencemag.org/content/scitransmed/3/89/89cm16.full Obtain information about reproducing this article: http://www.sciencemag.org/about/permissions.dtl
Science Translational Medicine (print ISSN 1946-6234; online ISSN 1946-6242) is published weekly, except the last week in December, by the American Association for the Advancement of Science, 1200 New York Avenue, NW, Washington, DC 20005. Copyright 2015 by the American Association for the Advancement of Science; all rights reserved. The title Science Translational Medicine is a registered trademark of AAAS.
Downloaded from http://stm.sciencemag.org/ on November 15, 2015
The following resources related to this article are available online at http://stm.sciencemag.org. This information is current as of November 15, 2015.
