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All rights reserved. 10% off print titles Custom notication of new releases in your eld of interest Special offers and discounts NATIONAL ACADEMY OF SCIENCES NATIONAL ACADEMY OF ENGINEERING INSTITUTE OF MEDICINE NATIONAL RESEARCH COUNCIL This PDF is available from The National Academies Press at http://www.nap.edu/catalog.php?record_id=12796 ISBN 978-0-309-14653-1 368 pages 6 x 9 PAPERBACK (2010) Priorities for the National Vaccine Plan Committee on Review of Priorities in the National Vaccine Plan; Institute of Medicine  Copyright National Academy of Sciences. All rights reserved. This summary plus thousands more available at http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 1 Summary The National Vaccine Plan is required by Title III in the 1986 National Childhood Vaccine Injury Act (NCVIA). 1 A plan was first released in 1994 and was updated by a draft plan issued in November 2008 (HHS, 1994, 2008). The National Vaccine Program Office (NVPO), located in the Of- fice of the Assistant Secretary for Health in the Department of Health and Human Services (HHS), solicited broad input from stakeholders, including the public, when drafting the plan. NVPO also asked the Institute of Medi- cine (IOM) to convene a committee to “prepare first a letter report 2 on its review of the 1994 plan” and then to prepare a “report with conclusions and recommendations about priority actions within the major components of the draft new plan” (see Box S-1). This report, Priorities for the National Vaccine Plan, aims to identify a set of actions the committee believes merit primary attention as NVPO and its partners finalize and implement the National Vaccine Plan. Strategic plans typically linked with budgets and resources are rarely sufficient to support every activity that planners may consider important and needed. Although the 2008 draft plan does not provide information about the potential costs of implementing its objectives and strategies, the committee defined “prior- ity actions” as actions that take precedence among many competing claims for resources. The committee made 18 recommendations about “priority actions” distributed among the plan’s five goals, and two additional recom- mendations, one of which refers to the scope of the National Vaccine Plan 1 See Appendix C. 2 The letter report was released in June 2008 and is available from the National Academies Press (http://www.nap.edu) and in Appendix D. Copyright National Academy of Sciences. All rights reserved. This summary plus thousands more available at http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 2 PRIORITIES FOR THE NATIONAL VACCINE PLAN and another that reflects NVPO’s role as a crucial ingredient in implement- ing the plan and ultimately ensuring that its objectives are achieved. CONTEXT Vaccination is a fundamental component of preventive medicine and of public health practice. The use of vaccines to prevent infectious diseases has resulted in dramatic decreases in disease, disability, and death in the United States and around the world. The contemporary national vaccine program 3 is extraordinarily complex in all aspects, from research and development of new vaccines to financing and reimbursement of immunization services. As a medical product, preventive vaccines occupy a unique niche because they are given to healthy individuals, they are purchased in large volume by the federal government as part of the Vaccines for Children entitlement pro- gram, and government public health agencies at the federal and state level make policy decisions about how best to use vaccines to protect the public’s health. Similar considerations inform policy for global vaccine efforts. In the latter part of 2009, the political, economic, and social environ- ment presents both opportunities for and challenges to strengthening the U.S. system for developing, manufacturing, regulating, distributing, funding, 3 In this report, the committee uses national vaccine program in lower case to denote the vast and complex network of actors and actions related to vaccines and immunization, and uses National Vaccine Program (per the 1986 act) when referring to the governmental agencies that have responsibilities related to vaccines and immunization. BOX S-1 The Charge to the Committee The federal government issued “Disease Prevention through Vaccine Devel- opment and Immunization, The US National Vaccine Plan” in 1994. The Institute of Medicine will convene an ad hoc committee to evaluate the 1994 National Vaccine Plan and then review and make recommendations regarding an update of this National Vaccine Plan. The committee will hold workshops a with national expert stakeholders in medicine, public health, and vaccinology to review a publicly available, draft update of the Plan. The committee will prepare a letter report of the evaluation of the 1994 Plan, and a report with conclusions and recommendations about priority actions within the major components of the draft Plan. a The IOM Committee on Review of Priorities in the National Vaccine Plan conducted its work between March 2008 and November 2009, including five information-gathering meetings with national stakeholders in Washington, DC, Chicago, Seattle, and Irvine. Copyright National Academy of Sciences. All rights reserved. This summary plus thousands more available at http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 SUMMARY 3 and administering safe and effective vaccines for all people. The Introduc- tion highlights key issues in the health care delivery system and in society, and also comments on the significance of the evolving 2009 novel H1N1 influenza pandemic. THE HISTORY OF THE PLAN The NCVIA called for the Secretary of Health and Human Services to serve as the director of the National Vaccine Program, 4 for a plan outlining the activities of the program to be updated annually, 5 an advisory commit- tee to provide guidance to the secretary and the program, and a budget to support specific types of program activities. The act also listed nine respon- sibilities for the program and its director (Public Law 99-660, Title XXI, Subtitle 1, Section 2102): 1. Vaccine research 2. Vaccine development 3. Safety and efficacy testing of vaccines 4. Licensing of vaccine manufacturers and vaccines 5. Production and procurement of vaccines 6. Distribution and use of vaccines 7. Evaluating the need for and the effectiveness and adverse effects of vaccines and immunization activities 8. Coordinating governmental and non-governmental activities 9. Funding of federal agencies Although the National Vaccine Program has had some great successes and there have been examples of effective coordination, neither NVPO (whose stated work is to provide “leadership and coordination among Fed- eral agencies, as they work together to carry out the goals of the National Vaccine Plan”) nor the plan have functioned as intended in the 1986 legisla- tion. This report includes several case studies that illustrate gaps or limita- tions in the program’s ability to perform important functions without the benefit of a strong, capable, and adequately resourced NVPO. These issues 4 Although the 1986 legislation did not specify the placement of NVPO and its relationship to the Secretary of HHS, delegation of authority by the secretary led to placement of the office in the Office of the Assistant Secretary for Health (ASH), and made the ASH the head of the National Vaccine Program. The National Vaccine Advisory Committee (NVAC) charter states that “Pursuant to the Statement of Organization, Functions, and Delegations of Authority for the Department of Health and Human Services (46 FR 61318, dated December 2, 1977; as amended in 52 FR 23502, dated June 22, 1987), the ASH shall serve as Director of the National Vaccine Program.” 5 In 1998 the requirement for annual updates of the National Vaccine Plan was repealed by Public Law 105-362, Title VI, § 601(a)(1)(H), Nov. 10, 1998, 112 Stat. 3285. Copyright National Academy of Sciences. All rights reserved. This summary plus thousands more available at http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 4 PRIORITIES FOR THE NATIONAL VACCINE PLAN and some of the reasons NVPO has never become what it was intended to be are discussed in Chapter 6. GENERAL COMMENTS ABOUT THE 2008 DRAFT PLAN The committee found that the lack of a coherent vision in the draft plan may be linked with an apparent sense of ambiguity about whether the plan should serve as (a) a collection of ongoing and planned activities that many agencies are already undertaking followed by an assessment of accomplishments at a later date; or (b) a list of critical needs and gaps that require coordinated attention by specific agencies or combinations of agencies and stakeholders; or (c) both a and b? A vision statement could resolve this ambiguity and guide the plan’s drafters, and the stakeholders who contribute to and will help implement the plan, in identifying the plan’s desired outcomes. It is understandable why the plan’s drafters chose to include both activities that are part of existing strategic plans and are certain to be ac- complished in the near future, and activities that are novel, not necessarily represented in any other planning document, and require multi-sectoral coordination and collaboration. However, the committee suggests that NVPO consider distinguishing between objectives or strategies that are likely to be accomplished regardless of their placement in the National Vaccine Plan and those that are unique to the plan and require coordina- tion among agencies and with non-government stakeholders in order to be achieved. 6 The committee’s recommendations about “priority areas within major components of the plan” refer to the latter type of objectives and strategies. Additionally, the forthcoming implementation plan NVPO will prepare after finalizing the strategic plan would be strengthened by a clear explanation of how the indicators in each goal relate to the objectives and strategies in that goal (Strikas, 2008). Below, chapter summaries and recommendations are provided in the order in which they occur in the report with one exception. In view of the importance of NVPO’s coordinating function, which is covered in the report’s final chapter (6), the overview of coordination appears first. CHAPTER 6: COORDINATION The history of NVPO and the National Vaccine Plan, and how it has influenced interagency coordination and coordination with stakeholders, is reviewed in the sixth chapter. The office’s authority and its human and financial resources have not matched its responsibilities, and the committee 6 W. Orenstein, 2007 NVAC meeting (NVAC, 2007). Copyright National Academy of Sciences. All rights reserved. This summary plus thousands more available at http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 SUMMARY 5 found that this mismatch has resulted in missed opportunities for the Na- tional Vaccine Program, and in NVPO’s inability to fully meet its statutory duties. Coordination is at the heart of the plan’s purpose, which is “to promote achievement of the National Vaccine Program mission by providing strategic direction and promoting coordinated action by vaccine and immunization enterprise stakeholders” (HHS, 2008). For this reason, supported by a request from the National Vaccine Advisory Committee (see Appendix B), the committee considered NVPO’s coordinating role with regard to the plan, including intragovernmental coordination and coordination with external stakeholders, in addition to considering the individual elements of the plan. Although coordination is not always possible or even necessary, there are areas where it is critical. For example, using a vaccine research agenda to spur the efficient development of priority vaccines requires intersectoral coordination at a high level. Building a structured way of identifying and addressing emerging safety information where appropriate, useful, and real- istic, requires input from multiple agencies and external stakeholders. Each agency has its own fairly distinct responsibilities in the area of vaccines and vaccination. However, some areas require coordination to reduce inefficient duplication of effort, and in other areas, one agency’s efforts may not be enough to reach an important goal. Because vaccines and immunization constitute a major public health matter that involves multiple government agencies and has great importance to the public’s health, an effective coordinating entity is needed, and effec- tiveness is dependent on authority and funding commensurate with the task at hand. However, the committee finds that NVPO, which was envisioned by the 1986 statute to serve as this entity, currently lacks the authority, influence, and profile needed to do so. Recently, NVPO has been given and has seized the opportunity to play a crucial coordinating role with regard to H1N1 pandemic influenza vaccine safety (HHS, 2009; NVAC, 2009a; Vellozzi, 2009). This example illustrates NVPO’s potential as coordinating entity in the face of a major challenge to the National Vaccine Program. Recommendation 6-1: The Secretary of HHS should actively dem- onstrate the Department’s support for the National Vaccine Plan by: (1) clarifying its primacy as the strategic planning tool ap- plicable to all federal agencies with roles in the National Vaccine Program, and (2) allocating the resources necessary to assure robust planning and implementation, with coordination by the National Vaccine Program Office. Copyright National Academy of Sciences. All rights reserved. This summary plus thousands more available at http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 6 PRIORITIES FOR THE NATIONAL VACCINE PLAN CHAPTER 1: VACCINE DEVELOPMENT Developing and manufacturing most 7 vaccines involves using living organisms and presents unique technical and regulatory challenges. Both industry and regulators are risk averse, and progress in regulatory science in general has been slow; as a result a “tried and true” paradigm character- izes some aspects of vaccine development and regulation (Goldberg and Pitts, 2006; Poland et al., 2009). Furthermore, some barriers to innovation stem from administrative and communication challenges at the interface between regulators and industry, not from concerns about safety, efficacy, or immunogenicity. Recommendation 1-1: The National Vaccine Plan should incor- porate improvements in the vaccine regulatory process that reflect current science and encourage innovation without compromising efficacy and safety. Improvements include: • Strengthening communication with vaccine developers through more frequent workshops and guidance documents. • Revising procedures and standards for developing, licensing, and producing vaccines for infectious diseases that encourage flexibility and innovation. In order to ensure that the Food and Drug Administration (FDA) can promote vaccine development while protecting safety, the agency must have funding and staffing commensurate with its responsibilities to identify, develop, and apply the best and most current science to the regulation of vaccine products. 8 There currently is no ongoing, evidence-based process by which vaccine candidates are identified as priorities shared among various stakeholders. Such a process can accelerate the development of vaccines by identifying the need and the likely market, and should be accompanied by a concerted effort to employ modern techniques to reach the goal of new and improved vaccines. Recommendation 1-2: The National Vaccine Plan should incorpo- rate the development of an evidence-based approach for prioritiz- ing new and improved vaccine candidates by targeted disease and 7 Newer synthetic sub-unit vaccines are an exception. 8 “The non-user fee part of CBER’s budget request for FY 2009 is $158 million, an increase of just under $3 million, or a mere 1.9 percent over FY 2008” (Richards, 2008). Copyright National Academy of Sciences. All rights reserved. This summary plus thousands more available at http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 SUMMARY 7 develop specifications for high-priority vaccines to accelerate their development. Specifications, such as target population, will differ for each vaccine, and defining them would increase predictability for manufacturers, reduce financial risk, and perhaps cost. The evidence to be considered would in- clude disease burden and feasibility, and would incorporate data or guid- ance available from prior published work linking research and funding levels to national priorities. 9 An approach to priority setting may include the following: • Supporting disease burden studies (e.g., morbidity and mortality) when needed for vaccine prioritization. • Employing outcome measures that capture both survival gains and quality-of-life improvements. 10 • Employing cost-effectiveness analysis. • Consider technical and scientific feasibility of vaccine development as a prioritization criterion. The committee found that the vast majority of National Institutes of Health (NIH)-supported peer-reviewed vaccine research is investigator- initiated and that coordination among federal agencies and with academic and private sector stakeholders could be strengthened. Furthermore, some examples of innovative and productive intersectoral collaboration come from a history of public-private partnerships, from the World War II era collaborations between the Department of Defense, industry, and academia, to contemporary development of vaccines for global health through product development partnerships. Recommendation 1-3: The National Vaccine Plan should incor- porate creation of a strategy for accelerating development of high priority vaccines that (a) engages all relevant institutes within NIH and the Department of Defense, academic investigators, and private sector partners; and (b) adapts lessons learned from past and pres- ent innovative public-private partnerships. 9 See, for example Gross et al., 1999; Neumann et al., 2005. 10 Quality-adjusted life years, or QALYs, have been suggested for use in the United States for priority setting in vaccine development (IOM, 2000). Disability-adjusted life years, or DALYs, have been suggested for use internationally. However, it is important to note that both measures have their proponents and critics, and that there are other measures of health outcome that could be used to inform a process of priority setting. Copyright National Academy of Sciences. All rights reserved. This summary plus thousands more available at http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 8 PRIORITIES FOR THE NATIONAL VACCINE PLAN This coordinated outcome-focused approach to vaccine development would need to be periodically reassessed to maintain appropriateness. The distinction between preventive vaccines against infectious disease and other preventive therapeutic vaccines apparent in the 1986 law is not a reflection of 21st-century vaccine science. The committee believes that as long as the statutory requirements are met by the National Vaccine Program, there is nothing that prevents the Secretary of HHS from expanding the program’s mission or finding other ways to link HHS policy and strategy across vaccine categories. Recommendation 1-4: Future iterations of the National Vaccine Plan should include classes of vaccines (such as therapeutic vac- cines and vaccines against non-infectious diseases) beyond those expressly enumerated in the statute, and the Secretary of HHS should explore how best to assign responsibility for coordination in this area. This broader view of vaccines recognizes the potential value of new vaccines beyond the “traditional” role of preventing infectious diseases and positions the federal government to support coordination on and encourage the broader application of scientific and technologic breakthroughs related to non-traditional vaccines. CHAPTER 2: VACCINE SAFETY Taking every step necessary to maximize vaccine safety is as important as endeavoring to derive the greatest disease-prevention benefits that vac- cines can provide. Because vaccines are given to large numbers of healthy people, safety is a great concern and is addressed through a system (con- sisting of many agencies and stakeholders) that collects vaccine safety data, generates hypotheses, and conducts studies to evaluate safety hypotheses. Recommendation 2-1: The National Vaccine Plan should establish a process to identify potential vaccine safety hypotheses for further basic, clinical, or epidemiologic research through annual review of data from the Vaccine Adverse Event Reporting System (VAERS), the Vaccine Safety Datalink (VSD) project, the Clinical Immuniza- tion Safety Assessment (CISA) network, and the Vaccine Injury Compensation Program, and from information available from sources outside the United States. There is no coordinated vaccine safety research agenda or a periodic, systematic process to prioritize a safety research agenda for the nation Copyright National Academy of Sciences. All rights reserved. This summary plus thousands more available at http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 SUMMARY 9 (Klein and Myers, 2006; NVAC, 2009b). Although the Centers for Disease Control and Prevention (CDC) Immunization Safety Office (ISO) has its own research agenda, what is also needed is a national vaccine safety re- search agenda to help guide and coordinate the efforts of all federal agencies and various stakeholders that conduct activities related to vaccine safety research. Recommendation 2-2: The National Vaccine Plan should emphasize the development and publication of a framework for prioritizing a national vaccine safety research agenda that spans all federal agen- cies and includes all stakeholders, including the public. The scientific criteria of such a framework for prioritization might in- clude, but are not limited to: (a) Assessment of the nature and extent of existing evidence for a pos- sible association of an adverse event with a vaccine. (b) Determination of the individual or public health burden of potential adverse events following immunization. (c) Consideration of the feasibility of scientifically rigorous study of a safety concern. (d) Assessment of biological plausibility of a causal association between an adverse event and a vaccine. A national research agenda would call on other agencies, such as NIH (which has historically played a limited role in vaccine safety research), CDC, and FDA, and non-federal stakeholders (such as providers who work with special populations, and vaccine manufacturers) to assume joint responsibility and work collaboratively on high-level challenges in vaccine safety research. An NVAC-affiliated advisory entity dedicated to vaccine safety has the potential to play a role both as an independent source of guidance on vaccine safety issues and by offering a forum for dialogue on the subject of vaccine safety. Recommendation 2-3: The National Vaccine Plan should include the establishment and scope of work of a permanent NVAC vaccine safety subcommittee to: (a) provide guidance on the activities described in Recommen- dations 2-1 and 2-2 in a public and transparent manner; [...]... http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 CONTENTS xi APPENDIXES* A Draft Strategic National Vaccine Plan B  Letter to the Committee from the National Vaccine Advisory Committee C 1986 National Childhood Vaccine Injury Act (Public Law 99-660) D  Initial Guidance for an Update of the National Vaccine Plan: A Letter Report to the National Vaccine. .. 20001 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... Safety Task Force 2009 Federal Plans to Monitor Immunization Safety for the Pandemic 2009 H1N1 Influenza Vaccination Program Washington, DC: HHS Copyright National Academy of Sciences All rights reserved This summary plus thousands more available at http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 16 PRIORITIES FOR THE NATIONAL VACCINE PLAN IOM... http://www.nap.edu/catalog.php?record_id=12796 PRIORITIES FOR THE NATIONAL VACCINE PLAN Committee on Review of Priorities in the National Vaccine Plan Board on Population Health and Public Health Practice Copyright National Academy of Sciences All rights reserved This summary plus thousands more available at http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 THE NATIONAL ACADEMIES PRESS... that span the entire National Vaccine Program Goal 4: Vaccine Use and Supply Goal 4 in the draft National Vaccine Plan ensure a stable supply of recommended vaccines, and achieve better use of existing vaccines to prevent disease, disability, and death in the United States—covers an extraordinarily broad set of issues The National Vaccine Plan does not provide a clear and coherent vision for Goal 4... carving from ancient Greece, now held by the Staatliche Museen in Berlin Suggested citation: IOM (Institute of Medicine) 2010 Priorities for the National Vaccine Plan Washington, DC: The National Academies Press Copyright National Academy of Sciences All rights reserved This summary plus thousands more available at http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796... http://www.nap.edu Priorities for the National Vaccine Plan http://www.nap.edu/catalog.php?record_id=12796 Contents Summary 1 Introduction Charge to the Committee, 17 Methods, 20 General Comments About the 2008 Draft Plan, 21 The Purpose of Immunization, 22 References, 29 17 1 Development of New and Improved Vaccines Context: The Current State of Vaccine Research and Development, 32 Themes from Information Gathering,... 1999 The relation between funding by the National Institutes of Health and the burden of disease NEJM 340(24):1881-1887 HHS (Department of Health and Human Services) 1994 1994 National Vaccine Plan Available: http://www.hhs.gov/nvpo/vacc _plan/ 199 4plan/ [accessed January 2008] HHS 2008 (November) Draft Strategic National Vaccine Plan Available: http://www.hhs gov/nvpo/vacc _plan/ 200 8plan/ draftvaccineplan.pdf... comprehensive assessment of the outcomes of national health reform and their implications for the nation’s vaccine and immunization priorities Specifically, NVPO, as “owner” of the plan, could contribute by: • Participating in implementation efforts related to the expanded health insurance access for the population • Participating in implementation efforts related to the design of health insurance coverage... communication strategy Recommendation 3-2: The National Vaccine Plan should incorporate a process for identifying research needs to inform the national communication strategy, including research on how the public obtains information about vaccines and immunization, perceives risks, and makes decisions concerning vaccination in the contemporary information environment 11 12 For further discussion see Chapter 3 See . (2010) Priorities for the National Vaccine Plan Committee on Review of Priorities in the National Vaccine Plan; Institute of Medicine  Copyright National. placement of the office in the Office of the Assistant Secretary for Health (ASH), and made the ASH the head of the National Vaccine Program. The National Vaccine