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Forum on Regenerative Medicine: 2016 and 2017 Program Activities (2018)

Chapter: Forum on Regenerative Medicine: 2016 and 2017 Program Activities

Suggested Citation:"Forum on Regenerative Medicine: 2016 and 2017 Program Activities." National Academies of Sciences, Engineering, and Medicine. 2018. Forum on Regenerative Medicine: 2016 and 2017 Program Activities. Washington, DC: The National Academies Press. doi: 10.17226/26246.
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Suggested Citation:"Forum on Regenerative Medicine: 2016 and 2017 Program Activities." National Academies of Sciences, Engineering, and Medicine. 2018. Forum on Regenerative Medicine: 2016 and 2017 Program Activities. Washington, DC: The National Academies Press. doi: 10.17226/26246.
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Page 2
Suggested Citation:"Forum on Regenerative Medicine: 2016 and 2017 Program Activities." National Academies of Sciences, Engineering, and Medicine. 2018. Forum on Regenerative Medicine: 2016 and 2017 Program Activities. Washington, DC: The National Academies Press. doi: 10.17226/26246.
×
Page 3
Suggested Citation:"Forum on Regenerative Medicine: 2016 and 2017 Program Activities." National Academies of Sciences, Engineering, and Medicine. 2018. Forum on Regenerative Medicine: 2016 and 2017 Program Activities. Washington, DC: The National Academies Press. doi: 10.17226/26246.
×
Page 4
Suggested Citation:"Forum on Regenerative Medicine: 2016 and 2017 Program Activities." National Academies of Sciences, Engineering, and Medicine. 2018. Forum on Regenerative Medicine: 2016 and 2017 Program Activities. Washington, DC: The National Academies Press. doi: 10.17226/26246.
×
Page 5
Suggested Citation:"Forum on Regenerative Medicine: 2016 and 2017 Program Activities." National Academies of Sciences, Engineering, and Medicine. 2018. Forum on Regenerative Medicine: 2016 and 2017 Program Activities. Washington, DC: The National Academies Press. doi: 10.17226/26246.
×
Page 6
Suggested Citation:"Forum on Regenerative Medicine: 2016 and 2017 Program Activities." National Academies of Sciences, Engineering, and Medicine. 2018. Forum on Regenerative Medicine: 2016 and 2017 Program Activities. Washington, DC: The National Academies Press. doi: 10.17226/26246.
×
Page 7
Suggested Citation:"Forum on Regenerative Medicine: 2016 and 2017 Program Activities." National Academies of Sciences, Engineering, and Medicine. 2018. Forum on Regenerative Medicine: 2016 and 2017 Program Activities. Washington, DC: The National Academies Press. doi: 10.17226/26246.
×
Page 8

Below is the uncorrected machine-read text of this chapter, intended to provide our own search engines and external engines with highly rich, chapter-representative searchable text of each book. Because it is UNCORRECTED material, please consider the following text as a useful but insufficient proxy for the authoritative book pages.

Forum on REGENERATIVE MEDICINE 2016 and 2017 Program Activities

ABOUT THE FORUM The National Academies of Sciences, Engineering, and Medicine offer a variety of activities to address the science policy needs of federal agencies and others who are in need of credible, independent advice, or are seeking a neutral setting for consideration of challenging issues. Forums are convening activities that provide both a mechanism and a venue for interested parties from academia, industry, government, and other stakeholder groups to meet and discuss issues of mutual interest and concern in a neutral setting. The primary purpose of Forums is to foster dialogue across sectors and institutions and to illuminate issues but not necessarily to resolve them. The goal of these activities is to develop a mutual understanding of the relevant issues and to provide a mechanism that fosters collaboration among stakeholders in addressing these issues. Forums are self-governing, i.e., the membership identifies the topics that it wishes to address, and with assistance from staff, develops meeting agendas and identifies workshop topics. As a result, the topics are likely to span a broad range of issues in research, policy, and practice. The National Academies of Sciences, Engineering, and Medicine’s Forum on Regenerative Medicine (“the Forum”) was established in 2016 and provides a convening mechanism for interested parties from academia, industry, government, patient and provider organizations, regulatory bodies, foundations, societies, associations, and others. Members meet and discuss sensitive and difficult issues in a neutral setting in order to engage in dialogue and discussions that address the challenges facing the application of, and the opportunities for, regenerative medicine to improve health through the development of effective new therapies. The Forum identifies existing and potential barriers to scientific and therapeutic advances; discusses opportunities to assist in facilitating more effective partnerships among key stakeholders; examines the impact that current policies have on the discovery, development, and translation of regenerative medicine therapies; considers the unique challenges of identifying, validating, and bringing regenerative medicine applications to market; and explores the ethical, legal, and social issues posed by regenerative medicine advances. Over the past two years, the Forum has identified important questions and challenges in the field of regenerative medicine that have led the group to explore a broad array of issues ranging from the unique challenges of manufacturing regenerative medicine products, to the complex needs of people who are exploring regenerative medicine as a treatment, to the difficulties of regulating a rapidly evolving field. By bringing together experts and leveraging a wide range of perspectives and knowledge, Forum members have collaborated to identify strategic opportunities to advance the field of regenerative medicine through hosting public workshops, publishing perspective papers and workshop proceedings, and developing information resources for the broader regenerative medicine community to support the advancement of high-quality science, efficient and effective therapies, and the safety and health of patients. nationalacademies.org/RegenMedForum

Areas of Interest The Forum members identified three areas of interest for further exploration and work that evolve to reflect developments in the field.    Communication Manufacturing, Cell/ Regulation The Forum’s working group on Tissue Sourcing, and The Forum’s regulatory working communications has identified several Tissue Engineering group has considered a wide array areas of interest, such as gaining a of regulatory challenges including The Forum’s working group on better understanding of how people developing regulatory standards, manufacturing and cell/tissue weigh and consider decisions related navigating the regulatory approval sourcing identified several areas to medical treatments and therapies, process, and learning about of interest, including defining and gather and collect information from regulation of regenerative medicine measuring critical quality attributes for different sources, and use resources to in other countries. This group follows regenerative medicine products and inform their treatment decisions. This current legislation and policy related materials, developing standards for group is interested in learning more to regulation of cellular therapies clinical trials and manufacturing, and about what types of resources are in with the goal of keeping members exploring challenges of manufacturing existence for use by those making current on recent developments. “living” therapies. The group treatment decisions about their The Forum also convenes regulatory published a perspective paper on disease/condition and where there are and legislative experts, patients, and these topics titled Manufacturing gaps in content and communication of other stakeholders on the topics Cell Therapies: The Paradigm Shift in those materials. of legislation and its impact on Health Care of This Century in June regulation. 2017 and hosted a public workshop titled Navigating the Manufacturing Process and Ensuring the Quality of Regenerative Medicine Therapies on June 26, 2017 (see page 3 for the links to these publications). The working group will continue exploring these topics in 2018, and plans to expand their focus to include challenges in the developing fields of tissue engineering and 3-dimensional scaffolding. 2016 and 2017 Program Activities | 1

2016 and 2017 Meetings JUNE 28, 2016 1st meeting Forum members discussed the function of a forum, defined the Forum’s purpose and goals, explored challenges and successes in the field, and outlined plans and areas for exploration. OCTOBER 13, 2016 Workshop OCTOBER 14, 2016 2nd meeting Exploring the State of the Science in the Field of Members explored opportunities for the Forum to Regenerative Medicine: Challenges of and Opportunities engage in activities related to regulatory issues such as for Cellular Therapies setting standards for potency and safety; manufacturing and clinical translation challenges such as the costs and technical barriers related to scaling up production MARCH 28–29, 2017 3rd meeting and defining critical quality attributes; and approaches Forum members discussed regulations and legislation to stakeholder education to improve understanding that impact regenerative medicine, as well as the issue about and inform realistic expectations for regenerative of unproven stem cell therapies being offered in U.S. medicine research. clinics. Members learned about various approaches to communicating science and educating patients, decision makers, and providers about regenerative medicine. Speakers also discussed the 21st Century Cures Act. JUNE 26, 2017 Workshop JUNE 27, 2017 4th meeting Navigating the Manufacturing Process and Ensuring the Quality of Regenerative Medicine Therapies Forum members discussed current regulations and legislation that impact regenerative medicine, learned about the psychological and sociological factors of patient decision making, and explored ways in which the Forum can continue to move the field of regenerative medicine forward in the areas of research, regulation, and manufacturing. OCTOBER 26, 2017 5th meeting Forum members discussed what resources patients need in order to make educated decisions about regenerative medicine therapies and what resources patient advocacy organizations have in place or need to develop to meet those needs. The group also learned about the state of the field in tissue engineering and 3-D scaffolding and explored new legislative and regulatory developments in regenerative medicine, both nationally and internationally. 2 | Forum on Regenerative Medicine

Public Workshops OCTOBER 13, 2016 This workshop highlighted opportunities and challenges associated with developing regenerative medicine cellular therapies and related technologies. Exploring the State of the Stakeholder groups, including research scientists, clinicians, patients, and Science in the Field of representatives from pharmaceutical and biotech companies, presented Regenerative Medicine: their perspectives and participated in discussions about new advances in Challenges of and the field and opportunities for using cell-based therapies for various cell and Opportunities for Cellular tissue types such as blood and immunological, skin and musculoskeletal, Therapies cardiovascular and lung, neurological and ophthalmological, and renal tissues. http://bit.ly/2cpKsVL JUNE 26, 2017 This workshop focused on exploring what measurements, characteristics, and technologies may be important in the development of new products and Navigating the therapies. A better understanding of which characteristics define a source cell Manufacturing Process or tissue can support the development of a quality product. Likewise, a deep and Ensuring the Quality understanding of the critical characteristics of the final regenerative medicine of Regenerative Medicine product can help ensure its consistency, safety, and potency. Workshop Therapies speakers explored the various challenges, opportunities, and best practices associated with defining and measuring the quality of cell and tissue products http://bit.ly/2r3PhGy and raw materials in the research and manufacturing of regenerative medicine therapies. Workshop participants learned about examples of manufacturing of early generation regenerative medicine products and examined ways to address how progress could be made in identifying and measuring critical quality attributes, designing and adhering to standards, and navigating the scale-up process from a research laboratory to the manufacturing environment. PUBLICATIONS NAM PERSPECTIVE Exploring the State of the Science in the Field Manufacturing Cell Therapies: The Paradigm of Regenerative Medicine: Challenges of and Shift in Health Care of Opportunities for Cellular Therapies: Proceedings of This Century: Discussion a Workshop (2017) Paper (2017) http://bit.ly/2xu5bRV http://bit.ly/2wvT0Um Perspectives, published by the National Academy of Medicine (NAM), are individually authored by Roundtable and Forum members and outside experts in health and health care. The views expressed in these papers are those of the authors Navigating the Manufacturing Process and Ensuring and not necessarily of the authors’ organizations the Quality of Regenerative Medicine Therapies: or of the NAM. Perspectives are intended to help inform and stimulate discussion. They have not Proceedings of a Workshop (2017) been subjected to the review procedures of and are not reports of the NAM or the National Academies http://bit.ly/2j0QOvI of Sciences, Engineering, and Medicine. 2016 and 2017 Program Activities | 3

Forum Membership CO-CHAIRS: Rusty Kelley, Ph.D. Roderic Pettigrew, Ph.D., M.D. R. Alta Charo, J.D. Burroughs Wellcome Fund National Institute of Biomedical University of Wisconsin-Madison Imaging and Bioengineering Walter Koroshetz, M.D. (until November 2017) Anne Plant, Ph.D. National Institute of Neurological Disorders and Stroke National Institute of Standards and Jay P. Siegel, M.D. Technology Johnson & Johnson Audrey Kusiak, Ph.D. Sharon F. Terry, M.A. U.S. Department of Veterans Affairs Griffin Rodgers, M.D., M.A.C.P. Genetic Alliance National Institute of Diabetes and (from November 2017) Robert S. Langer, Sc.D. Digestive and Kidney Diseases Massachusetts Institute of James C. Beck, Ph.D. Technology Stephen Rose, Ph.D. Parkinson’s Foundation Foundation Fighting Blindness Cato T. Laurencin, M.D., Ph.D. Sangeeta N. Bhatia, M.D., Ph.D. Institute for Regenerative Krishnendu Roy, Ph.D. Massachusetts Institute of Technology Engineering; Connecticut Institute Georgia Institute of Technology for Clinical and Translational Krishanu Saha, Ph.D. Lucie Bruijn, Ph.D., M.B.A. Science; University of Connecticut The ALS Association University of Wisconsin-Madison Health Center George Q. Daley, M.D., Ph.D. Elizabeth Smith John Lepore, M.D. Juno Therapeutics, Inc. Harvard Medical School; Boston GlaxoSmithKline Children’s Hospital & Dana-Farber Martha Somerman, D.D.S., Ph.D. Cancer Institute Terry Magnuson, Ph.D. National Institute of Dental and University of North Carolina, Chapel Craniofacial Research Cynthia Dunbar, M.D. Hill American Society of Gene Lisa Strovink, M.B.A. & Cell Therapy Michael May, Ph.D. The New York Stem Cell Foundation Centre for Commercialization of Brian Fiske, Ph.D. Regenerative Medicine The Michael J. Fox Foundation for Sohel Talib, Ph.D. Parkinson’s Research Richard McFarland, Ph.D., M.D. California Institute for Regenerative Advanced Regenerative Medicine Gary Gibbons, M.D. Manufacturing Institute National Heart, Lung, and Daniel Weiss, M.D., Ph.D. Blood Institute Jack Mosher, Ph.D. International Society for Cellular International Society for Stem Therapy Larry Goldstein, Ph.D. Cell Research Sanford Consortium for Regenerative Michael Werner, J.D. Medicine; UCSD School of Medicine Duanqing Pei, Ph.D. Alliance for Regenerative Medicine Chinese Academy of Sciences Celia M. Witten, Ph.D., M.D. Susan P. Howley Christopher & Dana Reeve Thomas Petersen, M.D., Ph.D. U.S. Food and Drug Administration Foundation United Therapeutics Corporation Jiwen Zhang, Ph.D. Seigo Izumo, M.D. Anna Pettersson, Ph.D. GE Healthcare Takeda Pharmaceuticals Pfizer Inc. Claudia Zylberberg, Ph.D. International, Inc. Akron Biotech (AS OF DECEMBER 2017) 4 | Forum on Regenerative Medicine

Forum Sponsors Advanced Regenerative Manufacturing Institute Juno Therapeutics, Inc. Akron Biotech The Michael J. Fox Foundation for Parkinson’s Research Alliance for Regenerative Medicine National Institute of Standards and Technology The ALS Association National Institutes of Health American Society of Gene & Cell Therapy National Heart, Lung, and Blood Institute National Institute of Arthritis and Musculoskeletal and Burroughs Wellcome Fund Skin Diseases California Institute for Regenerative Medicine National Institute of Biomedical Imaging and Bioengineering Centre for Commercialization of Regenerative Medicine National Institute of Dental and Craniofacial Research Christopher & Dana Reeve Foundation National Institute of Diabetes and Digestive and U.S. Department of Veterans Affairs Kidney Diseases National Institute of Neurological Disorders and Stroke U.S. Food and Drug Administration The New York Stem Cell Foundation Foundation Fighting Blindness Parkinson’s Foundation GE Healthcare Pfizer Inc. GlaxoSmithKline Takeda Pharmaceuticals U.S.A., Inc. International Society for Stem Cell Research United Therapeutics Corporation Johnson & Johnson (AS OF DECEMBER 2017) Foundations/ Associations/ 27% Societies Industry 27% 17% Patient Advocacy Groups 30% May not equal 100% due to rounding Government 2016 and 2017 Program Activities | 5

FORUM STAFF Sarah H. Beachy, Ph.D. Siobhan Addie, Ph.D. Senior Program Officer and Program Officer Forum Director Meredith Hackmann Ariel Markowitz-Shulman, M.S. Research Associate Associate Program Officer Rebecca Ray Senior Program Assistant (from August 2017) BOARD ON HEALTH SCIENCES POLICY STAFF Andrew Pope, Ph.D. Hilary Bragg Board Director Program Coordinator (until August 2017) ABOUT THE The National Academy of Sciences, National Academy of Engineering, and National Academy of Medicine work together as the National NATIONAL Academies of Sciences, Engineering, and Medicine (“the National ACADEMIES Academies”) to provide independent, objective analysis and advice to the nation and conduct other activities to solve complex problems and inform public policy decisions. The National Academies also encourage education and research, recognize outstanding contributions to knowledge, and increase public understanding in matters of science, engineering, and medicine. The Health and Medicine Division (HMD), formerly known as the program unit of the Institute of Medicine, is a division of the National Academies. HMD’s aim is to help those in government and the private sector make informed health decisions by providing evidence upon which they can rely. Each year, more than 3,000 individuals volunteer their time, knowledge, and expertise to advance the nation’s health through the work of HMD. Many of the studies that HMD undertakes are requested by federal agencies and independent organizations; others begin as specific mandates from Congress. While our expert, consensus committees are vital to our advisory role, HMD also convenes a series of forums, roundtables, and standing committees, as well as other activities, to facilitate discussion; discovery; and critical, cross-disciplinary thinking.

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