Innovation and Invention In Medical Devices

Workshop Summary

Kathi E.Hanna, Frederick J.Manning, Peter Bouxsein, and Andrew Pope

Editors

Based on a Workshop of the Roundtable on Research and Development of Drugs, Biologics, and Medical Devices

Board on Health Sciences Policy

INSTITUTE OF MEDICINE

NATIONAL ACADEMY PRESS
Washington, D.C.



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Innovation and Invention in Medical Devices: Workshop Summary Innovation and Invention In Medical Devices Workshop Summary Kathi E.Hanna, Frederick J.Manning, Peter Bouxsein, and Andrew Pope Editors Based on a Workshop of the Roundtable on Research and Development of Drugs, Biologics, and Medical Devices Board on Health Sciences Policy INSTITUTE OF MEDICINE NATIONAL ACADEMY PRESS Washington, D.C.

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Innovation and Invention in Medical Devices: Workshop Summary NATIONAL ACADEMY PRESS 2101 Constitution Avenue, N.W. Washington, DC 20418 NOTICE: The project that is the subject of this report was approved by the Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine. The members of the committee responsible for the report were chosen for their special competences and with regard for appropriate balance. Support for this project was provided by the U.S. Department of Health and Human Services and the Greenwall Foundation. The views presented in this report are those of the Institute of Medicine Committee on Assessing the System for Protecting Human Research Subjects and are not necessarily those of the funding agencies. International Book Standard No. 0-309-08255-2 Additional copies of this report are available for sale from the National Academy Press, 2101 Constitution Avenue, N.W., Box 285, Washington, D.C. 20055. Call (800) 624–6242 or (202) 334–3313 (in the Washington metropolitan area), or visit the NAP’s home page at www.nap.edu. The full text of this report is available at www.nap.edu. For more information about the Institute of Medicine, visit the IOM home page at www.iom.edu. Copyright 2001 by the National Academy of Sciences. All rights reserved. Printed in the United States of America.

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Innovation and Invention in Medical Devices: Workshop Summary Knowing is not enough; we must apply, Willing is not enough; we must do. —Goethe INSTITUTE OF MEDICINE Shaping the Future for Health

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Innovation and Invention in Medical Devices: Workshop Summary THE NATIONAL ACADEMIES National Academy of Sciences National Academy of Engineering Institute of Medicine National Research Council The National Academy of Sciences is a private, nonprofit, self-perpetuating society of distinguished scholars engaged in scientific and engineering research dedicated to the furtherance of science and technology and to their use for the general welfare. Upon the authority of the charter granted to it by the Congress in 1863, the Academy has a mandate that requires it to advise the federal government on scientific and technical matters. Dr. Bruce M.Alberts is president of the National Academy of Sciences. The National Academy of Engineering was established in 1964, under the charter of the National Academy of Sciences, as a parallel organization of outstanding engineers. It is autonomous in its administration and in the selection of its members, sharing with the National Academy of Sciences the responsibility for advising the federal government. The National Academy of Engineering also sponsors engineering programs aimed at meeting national needs, encourages education and research, and recognizes the superior achievements of engineers. Dr. Wm. A.Wulf is president of the National Academy of Engineering. The Institute of Medicine was established in 1970 by the National Academy of Sciences to secure the services of eminent members of appropriate professions in the examination of policy matters pertaining to the health of the public. The Institute acts under the responsibility given to the National Academy of Sciences by its congressional charter to be an adviser to the federal government and, upon its own initiative, to identify issues of medical care, research, and education. Dr. Kenneth I.Shine is president of the Institute of Medicine. The National Research Council was organized by the National Academy of Sciences in 1916 to associate the broad community of science and technology with the Academy’s purposes of furthering knowledge and advising the federal government. Functioning in accordance with general policies determined by the Academy, the Council has become the principal operating agency of both the National Academy of Sciences and the National Academy of Engineering in providing services to the government, the public, and the scientific and engineering communities. The Council is administered jointly by both Academies and the Institute of Medicine. Dr. Bruce M.Alberts and Dr. Wm. A.Wulf are chairman and vice chairman, respectively, of the National Research Council.

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Innovation and Invention in Medical Devices: Workshop Summary ROUNDTABLE ON RESEARCH AND DEVELOPMENT OF DRUGS, BIOLOGICS, AND MEDICAL DEVICES RONALD W.ESTABROOK (Chair), Virginia Lazenby O’Hara Professor of Biochemistry, University of Texas Southwestern Medical Center, Dallas ARTHUR L.BEAUDET, Investigator, Howard Hughes Medical Institute, and Professor and Chair, Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas (through February 1999) LESLIE Z.BENET, Professor and Chair, Department of Biopharmaceutical Sciences, School of Pharmacy, University of California at San Francisco D.BRUCE BURLINGTON, Director, Center for Devices and Radiological Health, Food and Drug Administration, Rockville, Maryland (through March 1999) ROBERT CALIFF, Director, Duke Clinical Research Institute, Durham, North Carolina MICHAEL D.CLAYMAN, Vice President, Global Regulatory Affairs, Lilly Research Laboratories, Indianapolis, Indiana RITA R.COLWELL, President, Maryland Biotechnology Institute, University of Maryland, College Park (through December 1997) ADRIAN L.EDWARDS, Private Practice, Internal Medicine/Cardiology, The New York and Presbyterian Hospitals, New York City DAVID W.FEIGAL, Director, Center for Devices and Radiological Health, Food and Drug Administration, Rockville, Maryland (from June 1999) STEPHEN GROFT, Director, Office of Rare Diseases Research, National Institutes of Health, Bethesda, Maryland ANNE B.JACKSON, Sarasota, Florida ROBERT I.LEVY, Senior Vice President, Science and Technology, American Home Products, Wyeth-Ayerst Research, Madison, New Jersey MICHAEL R.McGARVEY, Chief Medical Officer, Blue Cross and Blue Shield of New Jersey, Inc., Newark KSHITIJ MOJAN, Corporate Vice President for Research and Technical Services, Baxter Health Care Corporation, Roundlake, Illinois STUART L.NIGHTINGALE, Associate Commissioner, Health Affairs, Food and Drug Administration, Rockville, Maryland PAUL GRANT ROGERS, Partner, Hogan & Hartson, Washington, D.C. DANIEL SECKINGER, Group Vice President, Professional Standards, American Medical Association, Chicago, Illinois (through December 1997) WHAIJEN SOO, Vice President, Clinical Sciences, Roche Pharmaceuticals, Hoffmann-La Roche, Inc., Nutley, New Jersey REED TUCKSON, Group Vice President, Professional Standards, American Medical Association, Chicago, Illinois (from October 1998) JANET WOODCOCK, Director, Center for Drug Evaluation and Research, Food and Drug Administration, Rockville, Maryland

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Innovation and Invention in Medical Devices: Workshop Summary SUMNER YAFFE, Director, Center for Research for Mothers and Children, National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland KATHRYN ZOON, Director, Center for Biologics Evaluation and Research, Food and Drug Administration, Rockville, Maryland Liaisons to the Roundtable C.THOMAS CASKEY, Senior Vice President for Research, Merck & Co., Inc., West Point, Pennsylvania JAMES S.BENSON, Executive Vice President, Technology and Regulatory Affairs, Health Industry Manufacturers Association, Washington, D.C. BRIAN J.MALKIN, Associate Director for Patents and Hearings, Office of Health Affairs, Food and Drug Administration, Rockville, Maryland BERT SPILKER, Senior Vice President, Scientific and Regulatory Affairs, Pharmaceutical Research and Manufacturers of America, Washington, D.C. Study Staff JONATHAN R.DAVIS, Senior Program Officer VIVIAN P.NOLAN, Research Associate CHRISTINA THACKER, Research Assistant (to July 1998) NICOLE AMADO, Project Assistant Division Staff ANDREW M.POPE, Division Director LINDA DEPUGH, Administrative Assistant JAMAINE TINKER, Financial Associate (to October 1998) CARLOS GABRIEL, Financial Associate (from February 1999)

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Innovation and Invention in Medical Devices: Workshop Summary REVIEWERS This report has been reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise, in accordance with procedures approved by the NRC’s Report Review Committee. The purpose of this independent review is to provide candid and critical comments that will assist the institution in making its published report as sound as possible and to ensure that the report meets institutional standards for objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confidential to protect the integrity of the deliberative process. We wish to thank the following individuals for their review of this report: Clifford Goodman, The Lewin Group Robert Mann, Massachusetts Institute of Technology Richard A.Merrill, University of Virginia School of Law Daniel Wooten, Institute of Medicine Although the reviewers listed above have provided many constructive comments and suggestions, they were not asked to endorse the conclusions or recommendations nor did they see the final draft of the report before its release. The review of this report was overseen by Melvin Worth, Institute of Medicine. Appointed by the Institute of Medicine, he was responsible for making certain that an independent examination of this report was carried out in accordance with institutional procedures and that all review comments were carefully considered. Responsibility for the final content of this report rests entirely with the authoring committee and the institution.

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Innovation and Invention in Medical Devices: Workshop Summary Foreword Kenneth I.Shine, M.D. President, Institute of Medicine As a cardiologist I have had experience with many medical devices. The experience that had the most profound effect on me was as a medical student caring for a young man with what was called the Hufnagle valve, a “birdcage” valve that was placed in the descending aorta in patients who had aortic insufficiency. It was placed in the descending aorta because at the time Hufnagle developed it we did not have the techniques to allow placing the valve directly into aortic position. Thus, it was done through a thoracotomy. It did not affect the regurgitation in the upper portion of the body, only in the lower portion. What was remarkable about this device was that it made noise. It was located close to the trachea, and if a patient opened his mouth, people across the room could hear the clicking. As long as the patient was in sinus rhythm, they could tolerate the noise, but over time some of these patients developed atrial fibrillation, which produced a random clicking that was highly disturbing to them. The patient I took care of committed suicide because he could no longer tolerate the sound. Much progress has been made with prosthetic valves since that time. The Hufnagle valve was an extraordinary contribution at the time that it was first implanted, but it had unexpected limitations. Development of medical devices depends on innovation that moves the field safely forward in a way that continually improves over time. The Roundtable on Research and Development of Drugs, Biologics, and Medical Devices has provided a useful forum for the exchange of ideas and concerns among representatives of industry, government, and academia. This com-

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Innovation and Invention in Medical Devices: Workshop Summary munication has enhanced the performance of all three sectors. Institute of Medicine roundtables are relatively unique in that they provide an opportunity for industry, government, and academia to come together over time to confront important issues. The Roundtable has had two previous workshops, one exploring issues regarding the quality and validity of data generated by clinical trials, and the other examining how to improve the quality and quantity of therapeutics developed and dispensed to infants and children. In April 2000, the IOM launched the National Clinical Research Roundtable to bring together individuals concerned about the future of clinical research. This particular workshop focuses on medical devices at an exciting and challenging time, when the field is diverse, covering a range of implants, imaging equipment, surgical instruments, prosthetics, and orthotics. One of the challenges to the field derives from the rapidly expanding science base. This expansion is bound to continue or even accelerate given the kind of projected increases in the budgets of the National Institutes of Health and the National Science Foundation. The challenge is to make sure that the science base is reflected in the development of new devices and that we learn new ways to blend engineering and medicine. The flip side of that challenge is to make sure that newly developed devices are responsive to the most pressing needs of patients. There is a continuing debate over how much medical device innovation really is pioneering and how much is embellishing existing devices and making them more costly. I remind you of the fundamental changes taking place in the pharmaceutical industry, in which the more mature companies have determined that it is no longer profitable for them to develop “me-too” drugs. The types of drugs that will make substantial profits are those that come first to market. This change in approach is going to have implications for the device industry as well. A second major challenge is to ensure that beneficial innovations are made available to patients in need. It is extremely difficult to predict how the device field will develop over the next several years, let alone how insurance companies, managed care companies, and government programs are going to deal with these changes. Who is going to pay for what and in what manner? One thing I am fairly certain about is that patients, physicians, and payers are going to be looking for value. They are weary of the continued escalation in health care costs and the contributions to these costs made by medical devices. We cannot ignore the fact that the quality of the innovation must be measured not only by what it can do but also by its incremental costs and benefits. We must conduct rigorous evidence-based evaluations of the safety, efficacy, and relative effectiveness of health care technologies to ensure that our limited health care dollars are spent as effectively as possible in promoting overall health. In the field of devices, we need a better nosology, that is, the classification of devices, and we need to determine how each class of device should be evaluated for approval purposes and long-term follow-up. My concern here is that for most individuals and groups working on devices, evaluation tends to be very

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Innovation and Invention in Medical Devices: Workshop Summary much tailored to the particular device, which makes it extraordinarily difficult to figure out what types of data are necessary to be compelling. We need to do a better job with classification so that investigators and regulators have a clearer notion of the expectations for the approval and follow-up processes. This workshop explores the challenges, opportunities, and obstacles facing the field of medical devices.

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Innovation and Invention in Medical Devices: Workshop Summary Preface The Institute of Medicine Roundtable on Research and Development of Drugs, Biologics, and Medical Devices has evolved from the Forum on Drug Development, established in 1986. The importance of maintaining a neutral setting for discussions regarding long-term and politically sensitive issues was determined by sponsor representatives and the Institute of Medicine to justify the need for revising and enhancing past efforts. The new Roundtable is intended to be a convening mechanism for dialogue and exchange of information among individuals, including government officials, who represent all sides of public policy issues related to the development of drugs, biologics, and medical devices. Goals of the Roundtable include providing an environment for the exchange of information and the identification of high priority issues in the areas of product discovery and development. In order to achieve these goals, the Roundtable convenes twice annually in Washington, D.C., and holds at least one workshop each year. Members of the Roundtable bring expertise from clinical medicine, pharmacology, health policy, industrial management, and product development as they pertain to research and development of drugs, biologics, and medical devices. Each member’s participation adds a unique perspective to discussion topics. Members are responsible for identifying areas of Roundtable focus and issues that can be further elucidated in subsequent workshops. These workshops provide the opportunity to assemble a broader group of experts in the area of interest. The Roundtable identifies problems that are current and likely to be ongoing, or expected to arise within the next few years, and develops approaches to exploit opportunities or solve problems. Previous workshops have focused on

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Innovation and Invention in Medical Devices: Workshop Summary assuring data quality and review in the conduct and review of clinical trials and issues in pediatric drug development. The first two were Assuring Data Quality and Validity in Clinical Trials for Regulatory Decision Making and Rational Therapeutics for Infants and Children. This, the third workshop, focuses on innovation and invention in medical devices. To allow full and candid participation, through this workshop, the Roundtable’s goal was not to make recommendations or endorse specific courses of action, but to identify approaches that can or might be used to promote innovation in the medical devices area. The early stage of the innovation process involves matching technologies to needs, and sometimes the matching of technology to needs gets done through technology push. Sometimes it is achieved through demand pull. The different disciplines represented at this workshop are those that intersect at various points in the innovation process, and the challenge is the extent to which that intersection can be managed. Can it be productive without taking away the essential tensions required, yet allow synergistic and constructive progress? Traditionally the intersections of medicine, engineering, materials science, and electronics have been the focus of device development. That development is now growing. Molecular biology and immunology have entered the in vitro diagnostics area, as well as implants and prosthetics. These new intersections cause researchers to stop and ask whether these products are devices, drugs, or biologics? Thus, these advances challenge traditional paradigms. Researchers are also challenged by the need to evaluate the contributions that devices make to improved health and to costs. With drugs, there is a massive up-front cost but the incremental cost of the next pill is small. The relationship to health outcome is often more obvious for drugs than in the case of devices. The complications with the delivery and use of devices are very different. The unit cost of devices can be very low or extremely high. Because of the wide range of devices and their costs, developing a device economic model is not simple, although it is a worthy goal. At the end of all these intersections is one common goal; to find solutions and answers to mitigate the adverse human condition. Ronald W.Estabrook, Ph.D. Kshitij Mohan, Ph.D. Roundtable Chair Workshop Chair

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Innovation and Invention in Medical Devices: Workshop Summary Acronyms and Abbreviations AdvaMed Advanced Medical Technology Association (formerly Health Industry Manufacturers Association [HIMA]) APL Advanced Physics Lab ATD Advanced Technology Development BECON Bioengineering Consortium CBER Center for Biologics Evaluation and Research CDC Centers for Disease Control and Prevention CDER Center for Drug Evaluation and Research CDRH Center for Devices and Radiologic Health CIEBM Committee on the Interplay of Engineering with Biology and Medicine CIMIT Center for Innovative Minimally Invasive Therapy CMS Centers for Medicare and Medicaid Services (formerly Health Care Financing Administration [HCFA]) CPT Current Procedural Technology CTO Commercial Technology Office DHHS Department of Health and Human Services DPI Dry Powder Inhaler

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Innovation and Invention in Medical Devices: Workshop Summary EU European Union FDA Food and Drug Administration GMP Good Manufacturing Practice HCFA Health Care Financing Administration (now Centers for Medicare and Medicaid Services [CMS]) HIMA Health Industry Manufacturers Association (now Advanced Medical Technology Association [AdvaMed]) ICG Impedance Cardiography ICH International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use IDE Investigational Device Exemption IND Investigational New Drug IOM Institute of Medicine IRB Institutional Review Board ISS International Space Station LAVD Left Ventricle Assist Device MDI Metered Dose Inhaler MDN Medical Device Network MDR Medical Device Report MEMS Micro-Electromechanical Systems MIC Minnesota Impedance Cardiograph MIT Massachusetts Institute of Technology MWGC MultiWire Gamma Camera NAE National Academy of Engineering NDA New Drug Applications NIH National Institutes of Health NIST National Institute of Standards and Technology NSF National Science Foundation OCT Optical Coherence Tomography OPS Orthogonal Polarization Spectral PCR Polymerase Chain Reaction PCT Patent Cooperation Treaty PICAB Percutaneous in Situ Coronary Artery Bypass Grafting

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Innovation and Invention in Medical Devices: Workshop Summary PMA Premarketing Approval PQRI Product Quality Research Institute PTH Parathyroid Hormone SBIR Small Business Innovative Research SNP Single Nucleotide Polymorphism Ta-178 Tantalum-178 TIP Telemedicine Instrumentation Pack TMR Transmyocardial Revascularization

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Innovation and Invention in Medical Devices: Workshop Summary Contents 1   INTRODUCTORY OVERVIEW   1     Overview,   1     Challenges to Innovation,   2     Regulatory Issues,   3     Payment Issues,   4     Value and Consequences for Innovation,   7     Conclusions,   7 2   KEYNOTE SESSION   9     Historical Perspective on IOM’s Role in Providing a Forum for Discussion,   9     A Regulatory Perspective,   13     An Industry Perspective: Challenges in the Development and Regulation of Drug-Device Combination Products,   16     An Evaluator’s Perspective,   20     Innovation and Invention in Medical Devices: Implantable Defibrillators,   21     General Discussion of the Keynote Session,   25 3   THE NATURE OF MEDICAL INNOVATION   31     The Innovative Process for Medical Devices: A NASA Perspective,   31     Endovascular Devices,   36     Gene Arrays,   39

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Innovation and Invention in Medical Devices: Workshop Summary     Inhaled Insulin,   40     Imaging the Microvasculature,   41     General Discussion of the Nature of Medical Innovation,   43 4   SOURCES AND SUPPORT OF MEDICAL DEVICES INNOVATION   47     An Overview of Public and Private Factors Affecting Medical Device Innovation,   47     The Federal Research Role,   49     The Federal Regulatory Role,   50     The Academic Role in Innovation,   51     The Academic Health Center Environment,   52     The Role of Small Medical Companies,   54     The Role of Large Medical Companies,   54     The Role of Public and Private Capital,   56     General Discussion of Sources and Support of Medical Device Innovation,   57 5   THE CHALLENGES AHEAD   61     Unmet Clinical Needs: Cardiovascular Disease,   61     Unmet Clinical Needs: Clinical Trials,   63     Barriers and Issues in Device Innovation: Reimbursement,   64     General Discussion of the Challenges Ahead,   65 6   SUMMARY AND CONCLUSIONS   69     APPENDIXES     A   Workshop Agenda,   71 B   Speakers’ Biographical Sketches,   77 C   Registered Participants,   89