(The comments in red are additional HTA issues identified by participants from the ISPOR HTA Roundtable – Europe ). As during the ISPOR HTA Roundtable – North America, the goal was to identify HTA issues, not to develop consensus.

Issue I: Need for HTA Information
  1. Make or buy HTA.  Should each HTA organization increase its capacity to produce its own HTA information or should HTA organizations network with each other for the information? (2) Most of HTA organizations are not big enough to do “everything”
  2. Identifying HTA networks.  so, where are the HTA networks in USA and other countries (like the EUnetHTA); HTA is a network of sciences too. EUnetHTA is developing a sharing system of core information using report cards in multiple languages (with Executive Summary in English, which requires a standard format for reporting and sharing information, a taxonomy of methods, and classification of diseases (i.e. ICH10, UMDNS (universal medical device nomenclature system)
  3. Identifying a “Who’s Who?” of HTA.  We need a directory of HTA agencies, HTA-related government groups, HTA assessors, and other NGOs to know ‘who to call’ for HTA information
  4. Creating a central resource of HTA information.  A knowledge base of all HTA information on an intervention is needed [‘One stop shopping on a health care intervention] We need to use / review the information that already exists more efficiently. Common elements of an HTA report are a) technology description, b) evidence extraction (literature searches (mostly published studies); c) uptake of technology by others. A single portal ‘window’ [at EU or US government level] for evidence review of a technology is optimum.
  5. Reducing duplication of HTA work. HTA organizations could have specific functions, thus eliminating duplication of work or confusion on conflicting assessments. Agencies can be experts on certain diseases/technologies.
  6. Adapting HTA to local policy. There is a need to take HTA reports from central agencies and adapt them for local use (where applicable), but beware of ‘cookbook’ HTA.
  7. Sharing information among HTA organizations,  industry and scientific organizations.  Since the health technology industry (drugs, medical devices, diagnostics, biotechnology products, and surgical procedures) has much information on technologies, HTA organizations or networks should collaborate with the industry and scientific institutions to share this information. The industry should have discussions (sit-downs) with HT assessors prior to licensing to improve the evidence. It is also important to share follow-up information, which can change health policies. For example, use of modeling for reimbursement decisions should include a quality assurance component to assure the technology was modeled appropriately.
  8. Ethical, legal, social information. Ethical, legal, social aspects of HTA are more emphasized in European HTA.
Issue II: HTA Scope & Technology Life Cycle
  1. Defining health care technology broadly.  The scope of health care technology should include drugs, medical devices, diagnostics, biotechnology products, surgical procedures, interventional procedures and organizational structures, as well as behavioral health interventions.
  2. Assessing a technology throughout its life cycle.  A health technology should be assessed throughout its life cycle; Assessment of a technology is a continuum in form (i.e. reports alerts mini reports); but HTA’ers may not have the authority to increase their scope to include assessment throughout its life-cycle.
  3. Defining criteria for obsolescent technologies.  All new technologies must have improved outcomes (to be future defined) or improved cost-effectiveness vs. existing technology to be a replacement for existing technology. Public health impact should have a major influence on prioritization. “Retiring” older technologies (technology obsolesce) is an important part of the technology cycle.
  4. Identifying appropriate data requirements throughout a technology’s life cycle.  For emerging technologies, there is a need for a clear definition of the research required throughout its life-cycle [RCTs, then observational studies retrospective data analysis, patient registries & records]
  5. Influencing use of a technology through information dissemination of HTA.  The diffusion of information on a technology takes time
  6. Emphasizing clinicians perspectives in designing HTA.  We need to look at disease states, not just technologies (e.g. cardiac arrest)] and incorporate probabilistic models; the manufacturer's perspective is, "I have this technology. What can I do with it?" The clinician asks, "I have a patient with this diagnosis. How can I treat?" We need to guide the research process so that it primarily answers the latter question.
  7. Incorporating post market surveillance of “off-label” drug use into HTA.  Drugs are often introduced to the public and then are used by the medical community primarily for a disease or disorder for which there is no assessment (‘off-label use’); Medical practice drives HTA especially on ‘off-label’ use of technologies; however, HTA should drive medical practice (i.e., evidence based best practice approach); Since RCTs continue to be done after drug approval to test off-label uses, there remains a need to encourage trial participation so that efficacy can be tested before these off-label uses diffuse into routine practice.
  8. Using HTA to drive public use of health technologies.  Drugs are often introduced to the public with limited knowledge on side-effects; other indications are determined by the public use of the drug
  9. Including in the HTA process conditional and time limited funding of interventions in exchange for PME. When ambiguity arises regarding funding for a new technology arising from an HTA, post-marketing comparative evaluation (PME) through conditional and time-limited funding should be increasingly employed as part of the HTA scope of activity. PME should be used to test generalisability and to address any uncertainty; while being governed by the same ethical principles that guide RCTs, PMEs should be designed not to impede access or uptake and should be based on effectiveness pragmatic studies rather than RCTs.
  10. HTA of Non-drugs. Assessment of non-drug interventions (medical devices, diagnostics) is different than drug assessment. Non-drug effectiveness depends on the ‘operator’, equipment, health care organization. Devices have short life cycle, more safety and beta trial issues as well as legal issues.
Issue III: HTA Data
  1. Requiring post-market data collection.  Data collection must continue after the approval of a health technology (e.g. patient registries). US has more HT management (e.g. DUR)
  2. Identifying sources for ongoing collection of real world data. Need data sources to allow us to collect and use data on an ongoing basis; Data needs to come from real world; There are new systems in development for collecting real data in real time. Use of ‘real world’ data is should be on an ‘as needed’ basis with the assessment indicating the type of ‘real world’ data needed. There is a need for good recording data practices including electronic medical records for the collection of ‘real world’ data.
  3. Accessing patient data for HTA. Data should be treated as a ‘public good’; Laws need to change & allow data access; Methods to access data need to be defined to provide protection of patients’ privacy and allow access to the data for HTA and other scientific issues.
  4. Identifying and using data on off-label uses.  There is limited quality information on the off-label use of a technology
  5. Using and understanding transaction data in HTA.  Transaction data (such as reimbursement/claims data) are being used as research data, but limitations of these data need to be considered as well as economic data.
  6. Using conditional coverage to address data uncertainty. With the uncertainty of evidence [efficacy, safety, cost information], is conditional approval of a technology the answer? [I.e. condition being more data is collected and analyzed on the technology in exchange for coverage]. Need to specify a specific research program with conditional coverage to validate the evidence.
  7. Identifying the value of evidence tables.  Are evidence tables (league tables) helpful to decision-makers and are they used in health care decisions?
Issue IV: HTA Methods & Studies
  1. Balancing information needs of decision maker—timeliness vs. quality and comprehensiveness.  Assessment of a new technology must be in time, since coverage and reimbursement decisions must be made quickly too, but is ‘quick & dirty’ the answer?  ‘Quick & dirty’ HTA assessment needs to change to ‘quick & clean’ (i.e. INATHA is working on a checklist that may assist). There must be a follow-up with a more thorough assessment later; At any time, HTA uses best “available” evidence & best “available” will change with time HTA over the life cycle of a technology should be an ongoing iterative process, reassessing as new science emerges.
  2. Standardizing the HTA process. There is a hierarchy of data (evidence) e.g. in therapeutic interventions from randomized clinical trial (RCT) of efficacy & safety of a technology to individual case studies. How can we deal with evidence in other areas like rehabilitation and diagnostics? How is this information consolidated? There are many systems for grading data (studies); The consolidation of data (evidence) should be transparent, since studies may be interpreted differently. Need guidance on what methods to use in HTA reports. Need to know questions to be answered. In assessing a technology, the assessor needs to know the questions (the framework) the assessment will answer.
  3. Applying experimental and nonexperimental data in HTA and policy making.  When do we use observational data versus RCT data; Observational study methodology needs to be made “respectable” “I need respect” says CMS representative; “I need respect too [DeCide data]” says AHRQ representative; we need good research practices/guidances for observational studies as well as cost-effectiveness analysis studies to get them accepted; For example, patient registries complement RCT information and reflect real world use of technologies. From these registry data, new research hypotheses may be generated [i.e. top to bottom research, then bottom to top research]. This ‘real world’ information is also useful for health needs assessment and health impact assessment.
  4. Emphasizing methodologic transparency.  Study selection bias, transparency, asking the ‘right’ questions, methodological rigor are all methodology issues. Trials should be listed in a public trial registry before patient enrollment begins, with public access to the study protocol. Results of all trials should be made available to HTA assessors.
  5. Assessing alternative interventions.  Assessment of complimentary & alternative therapies (e.g. acupuncture) is an issue.  How should assessment be approached?
Issue V: HTA Prioritization
  1. Meeting the needs of the decision-maker. Health policy decision-makers need HTA ‘yesterday’.
  2. Creating a defined, systematic approach to identifying and prioritizing HTA topics. The assessment of a technology has a systematic approach. A systematic approach to ‘what’ gets assessed is also needed.
  3. Educating end user on knowledge gaps. We need to focus on gaps & fill gaps on knowledge & educate end-user
  4. Identifying who is responsible for filling in knowledge gaps. We know what we know and we know what we do not know, now fill in the hole; For this knowledge ‘hole’ who is responsible to fill it?
  5. Emphasizing public health impact in prioritizing HTA topics. HTA should not only be driven by burden of illness, budget impact, but by public health impact.
Issue VI: Translating HTA information for or by Health Policy-makers
  1. Identifying the decision-makers - clinicians, patients, politicians?; Translating science (HTA) into ‘practice’ [i.e. the decision-maker] is the problem. ‘Practice’ is diverse; Decisions are made on multiple levels. The development of decision support tools, including those in patient-friendly language, is critical to the widespread acceptance of HTA. There is a strong cultural presupposition that "My doctor knows best." When developing ‘core’ HTA information, it is important to consider who the decision-maker will be to assure appropriate translation of the core information.
  2. Educating the end user. There is a lack of respect for research by policy-makers and the public; Policy-makers sometime ask “Am I going to get into trouble if I use this HTA report”; HTA is a tool for public health and we should educate via public health; We should educate the end-user; Publishing HTA reports and “hope someone will read it” is the status quo, but is not the answer; The EUnetHTA tool kit, which contains checklists and resources to aid in the adaptation of HTA reports to the policy or research questions is a great tool and needs to be adopted globally; When adapting HTA evidence to address a policy issue, they must be transparent. Beware of ‘not invented here’, so cannot use information mentality. Methodology for decision-making must be imbedded in the HTA.
  3. Educating the HT Assessor. Need to understand the health policy development locally, find generic elements of health policy development, and use tools such as transition modeling for communicating HTA to policy.
  4. Including key stakeholders and end users in HTA process.  Government & NGOs are also researching technology and health care systems. More interaction is needed between government & NGO policy-makers, researchers, industry, clinicians; For example counter detailing by health care organizations vs. industry drug detailing is an issue; We need to talk about HTA issues and policy makers issues to industry and have them help us solve them. HTA organizations need to discuss policy issues with policy-makers in a scientific and transparent way. However, in some countries, assessment of a technology must be independent from government or the users.
  5. Strengthening the interaction between clinical practice guideline development and the HTA process. Clinical practice guidelines (CPG) are tools, not rules; Summaries of HTA evidence translates to CPG; Use of these CPG are then assessed to produce revised HTA reports; Conflicting CPG is an issue; clinical treatment based on evidence is not always practiced; Rules are sometimes required to get things done; however there is always the compliance issue with rules. For example, clinical practice variation maps pioneered by John Wennberg are helpful to show practice is not perfect; and that clinical practice behavior needs to be modified; Other guidelines are directed to the patient such as the Drug (Patient Purchasing) Guidelines by DERP; For health care organizations, medical policies may be developed by disease, not by technology (i.e. ‘disease-based medical policy’); CPG are voluntary to use, but medical policy is more rigid [i.e. what you can & cannot do]; medical policy sub-classification is in development, however; Health care organization policies sometimes do not drive the use of the best technology (i.e. patient vs. outpatient reimbursement policies).

HTA Roundtables