1. Chapter 7 HEALTH TECHNOLOGY ASSESSMENT

2. Introduction - The development and use of health technology (HT) are facing greater challenges due to increasing evidence requirements for new technologies. - Payers, politicians, government agencies and others increasingly insist that prior to the approval, acquisition, or payment for a healthcare technology, there must be well-founded data to support its use in a specific healthcare environment. - The US Food and Drug Administration (FDA) is not involved in this type of assessment, nor is it within its scope or mandate. - Other regulatory agencies (European Medicines Agency (EMA), the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA), and Sweden’s Medical Product Agency (MPA) are actively collaborating with HTA agencies and allowing joint scientific advice meetings with manufacturers or sponsors of HTs.

3. Continued - The definition of HTA by the International Network of Agencies for Health Technology Assessment (INHATA) is the systematic evaluation of properties, effects and/or impacts of healthcare technology. It may address the direct, intended consequences of technologies as well as their indirect, unintended consequences. Its main purpose is to inform technology-related policymaking in healthcare. HTA is conducted by interdisciplinary groups using explicit analytical frameworks drawing from a variety of methods. - The European network for Health Technology Assessment (EUnetHTA) provides a similar definition, stating that “HTA is a multidisciplinary process that summarizes information about the medical, social, economic and ethical issues related to the use of a health technology in a systematic, transparent, unbiased, robust manner” with the aim “to inform the formulation of safe, effective, health policies that are patient focused and seek to achieve best value.

4. Continued

5. - Methods for HTA vary by organization; while several organizations such as the National Institute for Health and Care Excellence (NICE) in the UK or the Canadian Agency for Drugs and Technologies in Health (CADTH) in Canada have been utilizing HTA methods for many years, other countries are still in the process of developing or refining methods for conducting HTAs and using HTAs in decision making. - A word of caution to the contents below: The process is explained with an introductory mind- set. The processes involved can be very complex and the system requires increasingly technical expertise.

6. Health Technology Assessment Process - Step 1 – Formulation of the Policy Question ◦Careful formulation of the policy question is a critical factor to ensure an appropriate, useful HTA is produced since the policy questions are the focus of the decision maker’s eventual conclusion. - Step 2 – HTA Question ◦The HTA question that follows the policy question must be phrased in such a way that the assessment will offer the information needed by the decision makers or the target group to come to a decision on the policy question. ◦To that end, HTA questions must be clearly formulated, defined and answerable. - Step 3 – Gather the Evidence and Complete the Analysis of the HTA Question ◦the gathering of evidence encompassing four basic areas related to the derived HTA question: ◦ technology ◦ organization ◦ patient ◦ economy

7. Continued - Technology ◦The properties of health technology include performance characteristics and conformity with specifications for design, composition, manufacturing, tolerances, reliability, ease of use, maintenance, etc. Safety is the assessment of the acceptability of risk associated with using a technology in a specific environment or situation - The health technology will have a certain degree of maturity and diffusion and this also becomes part of the HTA: ◦Future—a technology at the conceptual stage or at the earliest stage of development ◦ Experimental—the technology is tested in laboratories, e.g., in animal models or cell cultures ◦ Tested—the technology is tested in clinical studies, e.g., in patients with specific diseases or applied in a few institutions or in limited parts of the healthcare system as part of a study ◦ Established—the technology is considered to be standard treatment or routine practice and generally applied in the healthcare system ◦ Obsolete—new, safer or more effective technologies have surpassed or replaced older technology

8. Continued - Organization ◦Health technologies cannot merely be implemented since technologies introduced into an organization (e.g., hospital, health system) may influence organizational structures, tasks and the organization’s staff (people) and vice versa - Patient ◦Central research questions will therefore typically be aimed at: ◦ social aspects ◦ economic aspects ◦ ethical aspects ◦ individual aspects ◦ communicative aspects

11. Continued - Social Aspects ◦This covers whether, from a patient perspective, the technology will have or has, for example: ◦ a direct and/or indirect influence on or significance for: ◦work and training ◦family life and leisure time ◦lifestyle and quality of life - Economic Aspects ◦This covers whether, from a patient perspective, the technology entails, for example: ◦ direct and/or indirect costs in relation to ◦Work ◦family life ◦leisure time ◦lifestyle and quality of life

12. Continued - Individual Aspects ◦This covers whether, from a patient perspective, the technology entails: ◦ existential experiences, e.g., insecurity, worry, hope or anxiety ◦ patient roles and stigmatization ◦ courage to face life ◦ satisfaction ◦ use of one’s own resources (self-care, empowerment) - Communicative Aspects ◦This covers whether, from a patient perspective, the technology will have or has an influence on: ◦ exchange of information ◦ patients’ knowledge and understanding of the technology ◦ modified relations between the patient and health professionals ◦ involvement in decision making

13. Economy - Economic analyses may help determine how resources can be used best in the healthcare sector. - While economic analyses are not included in all HTAs, many agencies consider this a critical aspect of the decision-making process.

14. Assessment of Literature - Careful formulation of a literature search strategy, the literature search and the selection of studies to include in the analysis are the usual first steps in this stage of the HTA process. This step may in fact be circular in that the research offers up information that stimulates further requirements to investigate, such as when a reviewed article provides additional references that may be valuable. - A systematic approach to evaluating the literature is important in order to ensure an unbiased assessment of the available evidence, and the steps in the literature review should be fully documented to enable replicable results. Information from the “gray literature” or that is not available in peer-reviewed publications is often included as well to ensure a full review.

15. Continued - Information obtained for an HTA is often classified in terms of the hierarchy of evidence: (most rigorous to least rigorous): ◦ meta-analyses and systematic reviews (among others, Cochrane reviews8) ◦ randomized controlled trials (RCTs) ◦ non-randomized controlled trials ◦ cohort studies ◦ case control studies ◦ descriptive studies, limited series ◦ position papers, non-systematic reviews, leading articles, expert opinions

16. Data Generation, Analysis and Assessment - Various methods are available for the generation, analysis, assessment and use of data for an HTA. There are qualitative ethods involving interviews, participant observation and fieldwork and also procedures for questionnaire-based and registry studies. This entails descriptions of methods which ◦1) encompass both qualitative and quantitative approaches, and ◦2) are related to more than one HTA element.

17. Economic Analysis

18. Quality-Adjusted Life Years - An important concept in HTA is the Quality-adjusted Life Year (QALY). Health technologies have effects with more dimensions than can be measured in natural units. Cost:utility analysis should be employed if the use of a health technology is expected to lead to changes in healthrelated quality of life for patients or an extension of life with impaired health. - In cost:utility analysis, the effects are measured and valuated in QALYs. A unit of outcome of an intervention where gains (or losses) of years of life subsequent to this intervention are adjusted on the basis of the quality of life during those years. - Note: This parameter can provide a common unit for comparing cost-utility across different interventions and health problems. Disability-adjusted life years (DALY) and healthy-years equivalent (HYE) are QALY-analogous units

19. Generic Instruments as Utility Measures - Besides functioning as a profile measure for describing a patient’s self-assessed health, certain health status instruments also can be used as utility measures in economic evaluations. - In this context, the instrument must be able to generate a simple preference-based index score (on a scale of 0-1) for health status, e.g., for various patient groups or treatment alternatives. - Five generic instruments, have either been developed primarily as utility measures (EQ-5D, 15D, Health Utilities Index (HUI), Assessment of Quality of Life ((AQoL)) or aim at this on the basis of broad use as a profile measure (e.g., Short-Form 36 (SF-36)) Whereas the 15D and AQoL are relatively new, the other instruments are widely used and perform well with respect to validity and reliability.

20. Cost-Effectiveness Decision Matrix

21. Budget Impact Analysis - In addition to the analyses described above, many organizations also conduct a budget impact analysis to understand the overall financial impact of paying for a new technology on their organizations or on society as a whole. - Step 4 The Synthesis Process in HTA ◦The synthesis is designed to summarize and assess the findings while also providing one or more summary conclusions and, possibly, recommendations. It has been stressed that during this process great importance must be placed on transparency in terms of documents and methodology. Both advantages and disadvantages of various solutions also must be described.

22. Continued - Step 5 The HTA Report ◦It has been stressed that an HTA is not a decision, but rather it provides input or recommendations for decision-making. Decision makers are therefore the primary target user group for an HTA. The HTA may go directly to the decision makers or pass through several administrative stages where it is adapted or supplemented before the decision is made. ◦ There is a distinction between conducting HTAs and making decisions on the basis of HTAs. HTA development is based primarily in a research domain, while the outputs of HTA are used in a decision- making domain. Examples of HTAs can be found on various websites such as CADTH in Canada (http://www.cadth.ca/), NICE in the UK (http://www.nice.org.uk/) or the UK’s National Health Service,which give researchers access to more than 10,000.

23. Public and Private Agencies Using or Producing HTA Reports in the US11 - The Agency for Healthcare Research and Quality (AHRQ) is the primary government agency in the US responsible for developing and funding HTA. AHRQ supports HTA research through three external research networks: ◦1. Evidence-Based Practice Centers (EPCs) ◦2. | Centers for Education and Research on Therapeutics (CERT) ◦3. Developing Evidence to Inform Decisions about Effectiveness (DEcIDE) Program - AHRQ also conducts technology assessments in-house. AHRQ assessments typically focus on a systematic review f the literature and data synthesis from multiple studies. The Centers for Medicare and Medicaid Services (CMS) is the primary user of AHRQ technology assessments which are used to inform coverage policies for the national and local Medicare programs

24. Medicare/Medicaid - In the US, the largest government-sponsored healthcare organizations are Medicare and Medicaid. Medicare provides coverage for US citizens aged 65 and over while Medicaid serves the young, indigent and disabled. HTA assessments within these two agencies can occur at both the national and local levels. CMS is the agency responsible for oversight, financing and implementation of both the Medicare and Medicaid programs. As mentioned previously, AHRQ performs most technology assessments used by CMS staff in developing coverage decisions. The Medicare Evidence Development and Coverage Advisory Committee (MedCAC) is a group of external experts who review and weigh the evidence from HTAs such as those produced by AHRQ in open public meetings. The dossiers, public submissions of evidence submitted by the manufacturers and the final decisions for the Medicare program by CMS are placed on the CMS coverage website (http://www.cms.gov/medicare-coverage-database/indexes/technologyassessments- index.aspx).http://www.cms.gov/medicare-coverage-database/indexes/technologyassessments- index.aspx

25. Drug Effectiveness Review Project - The state of Oregon started the Drug Effectiveness Review Project (DERP) in 2000 to manage increasing Medicaid program costs by assessing pharmaceuticals for the state Medicaid Preferred Drug List. - DERP is a collaboration between the Oregon Evidence-based Practice Center (EPC) at the Oregon Health Sciences University and several states. - Currently multiple state Medicaid programs and other organizations are part of DERP and utilize its assessments in their decision-making processes. Current participants includes CADTH (Canada), Oregon, Washington, Idaho, Missouri, Montana, New York, Arkansas, Colorado, Wisconsin and Wyoming.

26. Other Federal Programs -The Department of Veterans Affairs (VA) includes the VA Technology Assessment Program (VATAP) (http://www.va.gov/vatap/). VATAP produces technology assessments including systematic reviews and evidence-based reports targeted at informing decisions within the VA program.

27. Private Sector Health Technology Assessment - Private health insurers and pharmacy benefits managers (PBMs) in the US use evidence evaluation techniques similar to HTA as part of their decision-making processes. Pharmaceutical and therapeutic (P&T) committees review the available evidence for a product and make recommendations for coverage and formulary placement. - For example, the Blue Cross Blue Shield (BCBS) Association funds the BCBS Technology Evaluation Center (TEC), which produces technology assessment reports for the Blue Cross plans that are also available to the public (http://www.bcbs.com/blueresources/tec/press/).