ISPOR HEALTH SCIENCE STRATEGY AD HOC GROUP:
Adrian Towse, MS Mphil, Office of Health Economics, London, England (Chair),
Karl Matuszewski MS, PharmD, University HealthSystem Consortium, Oak Brook, Ill
Louis Morris PhD, Melville, NY; Nicolaas Otten PharmD, Ottawa, Canada

GROUP OBJECTIVES

To determine:

  1. The role ISPOR should play in health policy;
  2. The committee structure needed to implement that role.
  3. ISPOR's role in policy making

BACKGROUND

The International Society for Pharmacoeconomics and Outcomes Research is an international organisation promoting the science of pharmacoeconomics and health outcomes research. The International Society is organised to act as a scientific leader relevant to research in pharmacoeconomics, health outcomes assessment, and related issues of public policy. The International Society represents health care researchers and practitioners including pharmacists, physicians, economists, and other health care professionals involved in pharmacoeconomic analysis and health outcomes assessment.

The mission of the International Society for Pharmacoeconomics and Outcomes Research is to translate pharmacoeconomics and outcomes research into practice to ensure that society allocates scarce health care resources wisely, fairly, and efficiently.

The society serves the public interest by the following means:
  • Provide a forum to facilitate the interchange of scientific knowledge in pharmacoeconomics and patient health outcomes,
  • Facilitate and encourage communications among the research community, health care professionals, and other research, regulatory, and educational groups, the communications media and the general public by educating public and private agencies on the usefulness of research in pharmacoeconomic and patient outcomes assessment,
  • Act as a resource in forming public policy relevant to the pharmacoeconomics, health care outcomes assessment, and related issues of public concern,
  • Promote the science of pharmacoeconomics by providing services as may be appropriate to advance pharmacoeconomic and outcomes research and educational activities,
  • Represent the science of pharmacoeconomics and outcomes assessment before public and governmental bodies by: a) seeking important issues concerning health care interventions as they relate to pharmacoeconomics and health care outcomes; b) making available the results of pharmacoeconomics analysis and health care outcomes studies concerning these issues; c) developing scientifically-based input (evidence) for public policy decisions dealing with these issues; d) providing this evidence and data to governmental bodies and other public fora, both domestic and international, upon request,
  • Provide for recognition of individual achievement in the field of pharmacoeconomics and outcomes research,
  • Foster career growth and the development of individuals involved in pharmacoeconomic and outcomes research and application of this research.

RECOMMENDATIONS OF THE AD HOC GROUP

The Health Science Strategy Ad Hoc Group recommendations that follow were approved by the ISPOR Board of Directors November 1999

Recommendation 1

ISPOR's role as a scientific society is to:
  • Assist in the development and dissemination of scientific policy (good practice in health outcomes research and its use in decision making).
  • Inform its membership of relevant public policy developments and, when appropriate, to seek to influence public policy making on the basis of good scientific practice to improve the use of scientific information (i.e. pharmacoeconomic information) in decision making.

Recommendation 2:

1A: To make a distinction between public policy (legal framework governing the provision of health care in a particular jurisdiction) and scientific policy (good practice in health outcomes research and its use in decision making).

1B: ISPOR should focus on scientific policy issues.

Discussion of Recommendation 2:

Scientific policies are more global such as good research practices or identifying research obstacles. The development of scientific policies will provide the basis for the later development of, or response to, health public policy issues. Health public policies are, for the most part, regional policies. Public policies are usually specific for a stated region. For example, the FDAMA Section 114 in the United States is specific to the United States or policies developed by the National Institute for Clinical Excellence (NICE) are applicable in the United Kingdom. There may be occasions when the public policy is specific to a certain region, such as the Patient Medical Record Confidentiality legislation in the US or the public policies identified above, but the underlying scientific issue (access to medical record information while assuring confidentiality, best practices in economic evaluations in promoting products / service, or best clinical practices) is applicable to any region. In these instances, a general scientific policy could be developed, and then regional policy positions developed to adapt the scientific policy regionally. The regional policy would take into account the unique rules in that region (country) and not reflect the Society as a whole.

Recommendation 3:

The HPD Ad Hoc Group identified five major areas in which the Society should take an active role in scientific and public policy development as follows:

  1. Medical information access and confidentiality
  2. Code of ethics in the area of pharmacoeconomic and outcomes research
  3. Good research practice in the area of pharmacoeconomic and outcomes research
  4. Use of health economic information for promotion of health care interventions
  5. Use of pharmacoeconomic and outcomes research information in health care decision making

Discussion of Recommendation 3:

Only five areas were identified to limit the scope of policy development to assure direction and importance to each policy issue undertaken. With limited resources, ISPOR must focus on policy issues, which most efficiently benefit the ISPOR member (researcher and user of health economic and outcomes information) and fulfil the mission of the Society, "to assure society allocates scarce healthcare resources wisely, fairly, and efficiently. It does not mean that the Society is limited to these five areas for time ad infinitum.

The basis for these 5 areas, in part, can be summarized in a presentation by Uwe Reinhardt PhD at the 2nd Annual Meeting, "Cost-effectiveness and cost-benefit analysis incorporate numerous assumptions and judgments by the analyst. These assumptions and judgements may reflect the analyst's own personal inclinations or perhaps that of the sponsor's. Whatever the drive, for these economic evaluations in health care to be taken seriously by the health care decision maker, the authors must be held accountable for the quality of their work.

A useful model in this respect is the accounting profession. The trust in and respect for the accountant's work rests on three major pillars.

  1. STANDARDS: The accounting profession has developed a standard body of account techniques that are shared worldwide. Example: "the ledger"
  2. CODE OF ETHICS: A widely shared set of principles guide the valuation of the asset and liabilities.
  3. WILLINGNESS FOR AUDIT: The accounting profession is willing to subject their work to deeply probing outside audit at a moment's notice.

Road to "respectability" for pharmacoeconomics outcomes research:

  1. STANDARDIZATION: The first step in attaining "respectability" is to seek a much greater standardization of pharmacoeconomic evaluations. The product of this standardization must take on the force of binding rules.
  2. CODE OF ETHICS: The second step is to develop a set of principles of conduct for analysts to follow. Empower professional health economic organizations to enforce the principles.
  3. AUDITS: Establish an audit process.

In the next century, decisions in health care increasing will be driven by sophisticated information systems available to both sides of the market. Health care research, in general, and pharmacoeconomic analyses, may be able to play a significant role if the studies can overcome certain obstacles and reach a new level of: respectability.

The rationale for the specific 5 areas is as follows:

  1. MEDICAL INFORMATION ACCESS: Since this issue is a "hot" issue in the US and Europe and ISPOR has already adopted the ISPE policy report: Data Privacy, Medical Record Confidentiality, and Research in the Interest of Public Health", the development of scientific and public policy in this areas has been initiated
  2. CODE OF ETHICS: ISPOR members have, on several occasions, requested the ISPOR Code of Ethics. This issue was discussed at the PE Issues Advisory Panel Meeting and the initial development of this issue was initiated and reported in Value in Health Vol. 2. No. 2.
  3. GOOD RESEARCH PRACTICES: A number of issues were identified in conducting economic evaluations in clinical trials, in economic modelling studies, and economic studies using retrospective analysis or large databases during the ISPOR-sponsored PE Issues Conference. The development of Good Research Practices relevant to these methodological issues is yet to be resolved. A 'How-to Manual', such as one has for the researcher needs randomised clinical trials. Scientific policies such as Good Research Practices for the pharmacoeconomic researcher will add credibility to the field as suggested by Dr. Reinhart.
  4. USE OF HEALTH ECONOMIC INFORMATION FOR PROMOTION OF HEALTH CARE INTERVENTIONS: the 1998-99 ISPOR Health Policy Committee in response to FDAMA Section 114 recently addressed this policy. Further development of this policy is warranted.
  5. USE OF PHARMACOECONOMIC AND OUTCOMES RESEARCH INFORMATION IN HEALTH CARE DECISION MAKING: The first step in attaining usefulness of health economic/outcomes studies to health care decision makers would be to seek a much greater standardization in presentation of information to health care decision-makers, than now exists, in principles and analytic techniques used in economic evaluations in health care. Examples would be rules on how to make economic studies transparent. This issue was also discussed at the ISPOR PE Issues Conference and recommendations were developed by the Advisory Panel and reported in ViH Vol. 2, No. 2.

Recommendation 4:

To this end ISPOR should, through its ISPOR Policy Development Committee:
  1. Monitor and develop scientific and public policy in the five major areas as defined above.
  2. Be a resource in these five major areas by:
    1. Compiling information for its membership
    2. Putting on educational events for its membership and others
    3. Bringing those involved in policy making together with other stakeholders (e.g. academics, industry, and health care decision-makers) to debate scientific policy and public policy issues with a scientific content relevant to ISPOR's scientific mission.
  3. On occasion, issue policy statements on scientific issues relating to these 5 major areas. These may include statements relating to both scientific and public policy issues. However, in order to ensure that the scientific mission of the society is not compromised, policy statements should be restricted to issues of good scientific practice, that, in principle, have international application, (i.e. beyond a particular jurisdictional debate) e.g. in relation to of pharmacoeconomic information in promotional claims, or the generalisability of clinical trial data results, or the use of observational databases in research.

Discussion of Recommendation 4:

The HPD Ad Hoc Committee identified several roles for ISPOR in monitoring and development of policy.

PROACTIVE ROLE: Develop standards/scientific policies and support regional (subgroup initiatives) in the five areas identified above.

ACTIVE REACTIVE ROLE: For issues other than those in the 5 areas identified above, the HP Ad Hoc Committee does not see a centralised monitoring or horizon scanning process either through the ISPOR office or through one or more of its committees.

PASSIVE REACTIVE ROLE: From time to time, there may be "hot" policy or scientific issues that may be identified by ISPOR members or regional subgroups. An operating procedure should be developed by the ISPOR Policy Development Committee that identifies what, when and how "hot issues" are processed.

Recommendation 5:

The HPD Ad Hoc Group recommends that initially there is one ISPOR Policy Development Committee to both monitor and develop policy in these five key areas. Policy or the organisation of events may then be developed by subgroups within the ISPOR Health Policy Development Committee. Regional (ad hoc) subgroups may be formed to develop regional positions if deemed consistent with the mission of the Society.

Discussion of Recommendation 5:

Initially, one central ISPOR Policy Development Committee would most effectively and efficiently co-ordinate the tasks needed to develop scientific policy in the areas defined above. Subgroups (such as the Code of Ethics Subcommittee or regional groups to address regional policy positions (such as the FDA Quality of Life Guidances or FDAMA Section 114) could be convened as necessary.

BACKGROUND INFORMATION

INCREASING THE IMPACT OF PHARMACOECONOMICS STUDIES
By Uwe Reinhardt PhD, James Madison Professor of Political Economy, Princeton University, Princeton, NJ USA
From: APOR NEWS, Vol. 3, No. 1 (January-February 1997)

Cost-effectiveness and cost-benefit analysis aside from their technical complexity usually incorporate numerous assumptions and judgements by the analyst. These assumptions and judgement may reflect the analyst's own personal inclinations or perhaps that of the sponsor's. Whatever the drive, for these economic evaluations in health care to be taken seriously by the health care decision maker, the authors must be held accountable for the quality of their work.

A useful model in this respect is the accounting profession. The accounting profession structures information that guides decision makers and uses fairly sophisticated techniques. Unlike health care researchers, accountants appear to have achieve far greater acceptance and respectability among decision makers for the structured information accountants provide, even though that profession constantly wrestles with troublesome problems of measurement — for example, the account for stock options. The trust in and respect for the accountant's work rests on three major pillars.

First, the accounting profession has developed a standard body of accounting techniques that are shared worldwide. Not only is there great uniformity in the structure of the "ledger" — the system of accounts used to rack revenues and expenses — but there is also great uniformity in the financial report submitted to owners, to the financial market and government around the world.

Second, a widely shared set of principles guide the valuation of the asset and liabilities. These set of principles demands that the accountant's report for a particular entity be developed "objectively" and "consistently" from year to year, and the entity's net worth (assets minus liabilities) be valued "conservatively." From this main set of principles follow a subset of principles on valuation of particular types of assets and liabilities, which in turn follow a subset of principles on measurement of the revenues and expenses attributable to a particular account period.

The third pillar of their profession is the willingness to subject their work to deeply probing outside audit at a moment's notice. These audits extend not only to the accounting techniques and valuation principles used in a given financial report. The go all the wary to the raw data used in the construction of the reports including a visual inspection of the assets the reporting entity claims to own. It is the nature of the auditing function, and the explicit code of profession ethics that govern both account and audit that sets accountant apart from economics and other health care researchers.

Presumably, the accountants well known standards, their code of ethics, and their willingness to be audited has earned accountants the respect among decision makers not now accorded the cost-effectiveness and cost-benefit analyses produced by the health care research community.

The first step in attaining "respectability" [i.e. usefulness of health economic/pharmacoeconomic studies to health care decision makers] would be to seek a much greater standardization, than now exists, in principles and analytic techniques used in economic evaluations in health care. The products of this standardization must take on the force of binding rules. The analogue in account for this standardization are the "accounting principles boards" such as the Financial Accounting Standards Board (FASB), which is composed of representative drawn from general industry and commerce, large auditing firms, and academia. The Board reviews the principles of valuation to be used for existing or newly evolving classes of assets. The Board proposes new rules, publishes them for comment, and issues binding rules that become part of the Generally Accepted Accounting Principles (GAAP) which bind the accounting profession.

A second step would be the establishment of the audit process and the development of an audit facility for policy-oriented, empirical health care research including economic evaluations of clinical practice. The audit process is not without its problems such as privacy and property rights of the researcher. Once again the accounting profession can provide guidance. Auditors who attest to the accuracy of a firm's financial reports have access to its most intimate trade secrets. Auditing has never been a major problem in financial accounting because auditors are constrained by a strict code of ethics and by legal strictures. Professional associations might be used to fulfil this function.

In the next century, decision in health care increasing will be driven by sophisticated information systems available to both sides of the market. Health care research, in general, and pharmacoeconomic analyses, in particular, may be able to play a significant role if the studies can overcome certain obstacles and reach a new level of "respectability."

Proceedings of the Advisory Panel Meeting and Conference on Pharmacoeconomic Issues: Executive Summary

From: Value in Health Vol. 2, No. 2. (March / April 1999)

Health care administrators, policy makers, and practitioners must balance the needs and desires of individual patients with the needs and desires of society at large, realizing that not all needs and desires can be met. Information comparing the expected gains of a medical intervention against the expected cost of that intervention versus other health care interventions are, many times, difficult to interpret or compare. The mission of the International Society for Pharmacoeconomics and Outcomes Research is to translate pharmacoeconomics and outcomes research into practice to ensure that society allocates scarce health care resources wisely, fairly, and efficiently. Toward this mission, ISPOR, co-sponsored with the U.S. Health and Human Services, Agency for Health Care Policy and Research, and the Health Outcomes Work Group of the Pharmaceutical Research and Manufacturers of America, convened an Advisory Panel Meeting and Conference on Pharmacoeconomic Issues, February 1998. This conference provided a forum for researchers and practitioners to communicate needs and concerns as consensus is developed on methodology, interpretation, and use of pharmacoeconomic information.

The objective of this interdisciplinary conference was to identify the issues in conducting pharmacoeconomic studies, interpreting the results of these studies, and using pharmacoeconomic information in health care decisions. The specific goals of the conference were to:

  • Identify key contentious methodology issues in conducting health care economic evaluations with clinical trials
  • Identify key contentious methodology issues in conducting health care economic evaluations using modelling studies
  • Identify key contentious methodology issues in conducting health care economic evaluations using databases
  • Determine the education and skills needed for conducting pharmacoeconomic evaluations in health care decisions
  • Identify the issues in application of economic evaluations in health care intervention protocol development, formulary decisions, and practice guideline development and use
  • Identify the issues in addressing bias, credibility, and quality of pharmacoeconomic evaluations
  • Identify the issues in communicating and reporting health care economic evaluation information

During this conference, sixty-one pharmacoeconomics and outcomes researchers, clinical practitioners and healthcare decision-makers in the United States met to develop consensus on issues relating to pharmacoeconomic and outcomes research evaluations and the use of these evaluations in healthcare decisions. The results of this deliberation are as follows:

METHODOLOGICAL ISSUES IN CONDUCTING PHARMACOECONOMIC EVALUATION - CLINICAL TRIALS

Pharmacoeconomic methods used to assess cost alone or other measures of value often fall short of regulatory standards. Conversely, study methods used to demonstrate drug efficacy, such as randomised clinical trials, are insufficient for addressing the question of value in other applied settings. To overcome the limitations of using clinical studies data for health economic evaluations, researchers, decision-makers, policy-makers and consumers should be well versed in the appropriate use of clinical studies.

There are four key issues are as follows:

  1. When should randomised clinical trials be the primary approach to assessing questions of value?
  2. What modifications to randomised clinical trials would improve their usefulness as tools for health economic decision-making?
  3. When should observational studies be used to assess questions of value?
  4. What modifications to observational studies would improve their usefulness as tools for health economic decision-making?

The recommendations for these issues are:

  1. Define' usual care': As trials adopt more naturalistic designs, the use of "usual care" as comparator will likely increase and comparability will be necessary.
  2. Develop new methods to account for protocol-related costs
  3. Develop alternative methods for intent-to-treat analyses in usual care trials. Although the intent-to-treat analysis is important, there may be other research questions important to decision-makers that would benefit from alternate or additional analysis.
  4. Address problems of pooling economic data due to significant differences in clinical practice across settings and sites, especially in international studies.
  5. Establish the range for "acceptable" levels of certainty of study results to inform value-based decision-making.
  6. Improve statistical methods for adjusting for selection bias, for example, instrumental variables and propensity scores.
  7. Develop better methods to estimate variance around the components of value, such as bootstrapping and re-sampling techniques.
  8. Develop systematic comparison of randomised clinical trials and observational studies on the same interventions.
  9. Survey other disciplines (such as psychology, sociology, marketing research) for new approaches to methodology, particularly in the areas of data collection, analyses and instrumentation.
  10. Use large, simple trials to measure resource utilization such as early Phase IV studies,
  11. Develop better methods to measure direct medical costs not routinely captured, such as nursing time and telephone care (OMERACT panel).
  12. Develop better methods to measure relevant indirect costs such as caregiver time or lost productivity (ARAMIS). As electronic medical records evolve and expand, encourage inclusion of standardized outcome measures.

METHODOLOGICAL ISSUES IN CONDUCTING PHARMACOECONOMIC EVALUATION - MODELING STUDIES

The primary purpose of modelling is to inform the decision-making process. One considerable benefit of model formalization is that the uncertainties and assumptions in this process are made explicit and transparent. Currently there are two major obstacles confronting modelling methodology currently: a) how to optimise the production of useful information for health economic decision-makers and b) how to encourage its acceptance and use of information.

There are seven key issues (areas of controversy) in modelling methodology as follows:

  1. Standardization
  2. Making choices
  3. Methodological development
  4. Extending clinical trials and data issues
  5. Effectiveness measures
  6. Model validation
  7. Peer review.

The recommendations for these issues are:

  1. Work towards general acceptance that modelling of both costs and effectiveness as a valid and often essential method to inform health care decision-making
  2. Assemble a consensus of opinion on standardized practices and policies
  3. Prepare and disseminate a reference text of these practices once standardization has been achieved.
  4. Permit pharmacoeconomic claims based on these generally accepted modelling approaches by regulatory agencies, and should always include transparency and appropriate disclaimers such as: "This economic analysis is based on assumptions and simulations concerning the efficacy of [drug name] that meet FDA criteria for claims of efficacy." Any model that relies on assumptions about a drug's efficacy that are not based on data from RCT must prominently disclose such limitation in any promotion.
  5. Initiate and assemble a balanced international panel of thought-leaders and end-users in the field of modelling to develop a package of generally accepted modelling practices
  6. Encourage all stakeholders, professional societies, manufacturing associations, journals, government agencies, regulatory agencies, payers and health care providers, to accept these as standards and to endorse their use once these practices have been documented.

METHODOLOGICAL ISSUES IN CONDUCTING PHARMACOECONOMIC EVALUATIONS - RETROSPECTIVE AND CLAIMS DATA STUDIES

Health care decision-makers require rapid access to information. The evidence that assists decision-makers to draw conclusions often has not been available. Both RCT, and retrospective methods using existing databases, provide such information, and typically answer different questions. Most RCT are designed to measure efficacy, not effectiveness. "Real world" data can be provided by database studies.

Eight key issues were identified as follows:

  1. What research questions can be answered by retrospective analyses?
  2. What data sources are available to answer these questions?
  3. How is cost-effectiveness measured using automated databases?
  4. How can data quality within a database be evaluated?
  5. What types of statistical methods can be utilized to control for treatment effects?
  6. What potential types of bias exist in retrospective database analyses?
  7. What alternative methods for assessing selection bias are available?
  8. How can transparency be ensured in retrospective database analyses?

The recommendations for these issues areas follows:

  1. Begin retrospective database analysis studies with a clear question and design, based on guidelines for good epidemiological practices.
  2. Ensure privacy of individuals at all times in retrospective database analyses.
  3. Use techniques that exist to address shortcomings of retrospective data sets
  4. Subject multivariate models to extensive specification testing.
  5. Examine age- or gender-adjusted utilization rates and annual per capita expense by payer, health plan, geographic region and country
  6. Augment administrative databases, frequently used for retrospective pharmacoeconomic studies, to include more clinical information
  7. Establish standard measures to deal with all areas of potential bias.

EDUCATION AND SKILLS NEEDED TO CONDUCT, INTERPRET AND USE ECONOMIC EVALUATIONS IN HEALTHCARE

Like other disciplines, to expand and grow as a mature area of research and application, the field of health economics requires experts and skilled professionals. Unlike many other scientific fields, there is no one background or training that prepares the researcher or the user of health economic information, who currently come from a diversity of educational and experiential backgrounds.

The key issues related to education and skills in the field of health economics are as follows:

  1. Multidisciplinary Programs - The structuring of multidisciplinary programs needs to be defined for people coming from a variety of backgrounds.
  2. "Real-World" Applications - Training must include "real-world" applications.
  3. Ideal Program: It is unlikely that an ideal program can be created in any one place within one institution or group without collaboration with others.
  4. Minimal Competencies - The usefulness of minimal competencies in the field has to be determined. Minimal competencies will be different for current and future practitioners by depth of involvement. For each level of involvement, the type of competencies will have to be defined.
  5. Who should be trained - Questions pertaining to who should be trained, how should training be performed and what level of training is required need to be answered.
  6. Credentialing - The necessity of credentialing needs to be assessed.
  7. Training Opportunities - There is needed to improve the way information about training opportunities is disseminated.

The recommendations for these issues are as follows:

  1. Develop three levels of expertise: awareness, application, and conceptualisation
  2. Develop access to detailed information about available educational programs in the field of health economics
  3. Utilize relevant educational resources outside of health economics to enhance the educational infrastructure
  4. Accommodate multidisciplinary participants through the availability of prerequisite trainings and flexible core course offerings for degree programs
  5. Balance didactic and experiential education
  6. Develop a credentialing process to establish standards for the field
  7. Standardize training and certification through a three step process: a) develop guidelines for post-professional degree training; b) accredit pharmacoeconomic residencies and fellowships; c) establish collaborations with other organizations to expand accreditation to other relevant residencies

APPLICATION OF HEALTHCARE INTERVENTION ECONOMIC EVALUATIONS IN HEALTHCARE DECISION-MAKING

Information about the impact of new therapies on costs within a healthcare system should be essential for making better healthcare decisions. However, the relevance of health economic information to decision-makers has not been demonstrated. There is little user-friendliness in the health economic data. There is a lack of consistency of approach and format that would facilitate comparison under review. Finally, much of the information presented lacks the transparency necessary for the user to determine the appropriateness of methods or the soundness of assumptions. A fundamental disconnect exists between a) the way decisions are made by healthcare decision makers, b) type of information presented to healthcare decision makers and c) the type of information required by health economic and outcomes researchers.

There are eleven key issues as follows:

  1. Potential users of health economic research data often weigh evaluative criteria differently for decision-making purposes such as formulary committees, providers, and health plan managers, patients or employers.
  2. Language and definitional barriers hinder effective communication between potential users and producers of the information may exist.
  3. Lack of treatment comparisons. There is little data on direct product or treatment comparisons that are of greatest interest to potential users.
  4. Lack of communication. Little dialogue exists between the potential users and the producers of information on what is relevant and what information can be generated by health economic research.
  5. Lack of understanding. Potential users of health economic research data may be hesitant to include health economic information in their decision-making process because it is different from their established clinical orientation.
  6. Conflicting study design. To conduct studies, which provide health economic information that meets users' needs, certain research designs for health economic and outcomes research studies may conflict with clinical design, causing regulatory and liability concerns.
  7. Research Funding. Some sources of research funding may present a barrier to the credibility and application of study results.
  8. Societal perspective vs. individual perspective. A conflict may exist between recommendations based on population data and the care of individual patients.
  9. Lack of quality criteria. When health economic research data is used in the decision-making process, there is no recognized approach for measuring the quality of the decision or the net result.
  10. Lack of experts. There are few skilled opinion leaders or other resources from which potential users can seek advice and assistance.
  11. Segregation of organizational finances and health outcome decisions. Decision-maker organizations segregate budgetary decisions for pharmaceuticals from those related to other medical technologies and services.
The recommendations for these issues are:
  1. Recruit a central organizer to coordinate the improvement of the application of healthcare intervention economic evaluations in healthcare decision-making.
  2. Create focus groups to provide a forum for dialogue between potential users, producers, and regulators of information Researchers and suppliers of health economic data must actively engage with decision-makers to determine the key health economic evaluation criteria for decision-making purposes and formulate ways to supply the information consistently.
  3. Determine a set of variables, which researchers can supply. Decisions are seldom made using a single variable.
  4. Develops a set of simple criteria for evaluation of these studies agreed upon by consensus of all parties involved and designed to recognize different types of perspectives and research design so that specified research questions and business needs are met.
  5. Seek to bolster the objectivity, reliability, and credibility of the health economic studies through various mechanisms, including working with sponsors, researchers and journal editors, to adopt protocols that will establish the independence of research and statements for the disclosure of funding sources.
  6. Offer training for decision-makers in using health economic research information for decision-making. A consortium of managed care and other purchasing organizations, academic researchers and one or more health economic research organizations should be formed to execute this recommendation.
  7. Develop a standard reporting format to allow flexible weighting of factors based on individual decision-making preferences. The presentation of the results of health economic analyses is modified to show the various components of effectiveness measures, service utilization measures and costs.
  8. Form a committee that would produce a standard format for Data Element Shells (DES). DES would be in the form of desegregated data from cost-consequence or cost-effectiveness analyses. For each outcome of interest, reference to the data source could be made to allow reviewers to discern the degree of scientific support for each data element. ISPOR, in collaboration with potential users and producers of information, could be responsible for creation and updating of a DES form. The ISPOR committee would decide on the level of specificity of the DES, perhaps either a general format for all drugs or a specific format for individual drug classes.
  9. Support an information clearinghouse of available thought-leaders and experts in the field. This should include development and maintenance of an Internet WEB site with links to expert's homepages and e-mail addresses. ISPOR as an organization brings together many of the researchers qualified to evaluate health economic research and interpret findings.
  10. Develop rosters of persons qualified to review studies, similar to editorial boards for journals, where the reviewers would agree to participate in reviewing documents or addressing queries to promote a better understanding of the field of health care economics.

ADDRESSING QUESTIONS OF BIAS, CREDIBILITY AND QUALITY IN HEALTH ECONOMIC EVALUATIONS

Multiple published studies have criticized the rigor, relevance, objectivity, methods, and reports produced within the health economic research domain. Consequently, health economic research findings are not used as extensively as they could be and rational decision processes about the efficient use of health care resources may not be fully informed. Ultimately, care for patients and populations may be adversely affected. In this context, there is a need for continued improvement in the quality of economic research conducted.

There are three key issues as follows:

  1. Quality: are best methods being used?
  2. Bias: whether real or perceived, how do we deal with it?
  3. Credibility: do we have a problem with believability or with relevance?

The recommendations are as follows:

  1. Design and conduct studies using the best available practices consistent with the study objectives
  2. Disclose any financial relationships which authors and speakers have linking them directly or indirectly to the interventions under study
  3. Authorship should conform to generally recognized practices among the peer research community. Research data, given full disclosure, transparency, and sufficient information to replicate the study, should be judged on the merits of its content
  4. Develop a code of ethics for health economic researchers
  5. Develop study methodology practice standards
  6. Convene a conference similar to this conference in 2 years to evaluate progress and recommend next steps

COMMUNICATION AND REPORTING HEALTH ECONOMIC INFORMATION

Users of health economic information represent many different perspectives with various levels of expertise and information needs. To obtain most value from the resources invested in health economic re

  1. Relevance: Is it needed?
  2. Usefulness: Will the intended audience be able to make use of it?
  3. Credibility: Is it believable?

The recommendations are as follows:

  1. Identify the needs of users of health care economic information. A survey of all users of health economic information will provide a basis for standardization of communications.
  2. Establish standard communication formats based on predetermined relevance, information and credibility needs of users and on standard health economic performance standards that should be under development elsewhere. These should eventually include: a) uniform presentation, standard terminology, adequate disclosure; and a basis in previously published guidelines
  3. Adopt a Reporting Guidance (RGs) and apply to all publicly presented communications, as standardized formats are established
  4. Evaluate the use of Reporting Guidance and the quality of reporting on a biannual basis.
  5. Establish a principle of publicly accessible reports that adhere to ISPOR RGs. This would allow access to research reports that is not directly controlled by the researcher or the research organization. Once a report has been "filed" for public accessibility, all subsequent communications could refer to that report
  6. Institute an enhanced mode of peer review for all forms of health economic communications. This type of review would assure that there was compliance with ISPOR RGs and fair, full and adequate disclosure, allow for review of the underlying data and any model used, and confirm that all other ISPOR standards for the conduct of health economic studies have been met.

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