HTA 101: Selected Issues in HTA

Quality of Care and HTA

The relationship between HTA and quality of care is often poorly understood. Although a thorough discussion of this subject is not possible here, the following are some definitions and fundamental relationships concerning these concepts.

Quality of care is a measure or indicator of the degree to which health care is expected to increase the likelihood of desired health outcomes and is consistent with standards of health care. HTA and quality assurance are distinct yet interdependent processes that contribute to quality of care.

HTA generates findings that add to our knowledge about the relationship between health care interventions and health care outcomes. This knowledge can be used to develop and revise a range of standards and guidelines for improving health care quality, including practice guidelines, manufacturing standards, clinical laboratory standards, adverse event reporting, architecture and facility design standards, and other criteria, practices, and policies regarding the performance of health care.

The purpose of quality assurance activities is to ensure that the best available knowledge concerning the use of health care to improve health outcomes is properly used. It involves the implementation of health care standards, including activities to correct, reduce variations in, or otherwise improve health care practices relative to these standards. Continuous quality improvement (CQI) and total quality management (TQM) (Gann 1994; Wakefield 1993) are among the contemporary systematic approaches to quality assurance that are being adapted for hospitals and other health care institutions. Such approaches include, for example, the identification of best practices and the use of benchmarking to develop improved clinical pathways or disease management for medical and surgical procedures, administrative operations, etc. (Kim 2003; Kwan 2002; Pilnick 2001). For example, CQI has been evaluated in a recent multicenter RCT as a means improve the adoption of two process of care measures for CABG: preoperative β blockade therapy and internal mammary artery grafting (Ferguson 2003). Notably, in this RCT, the intervention being tested was not those two health care interventions, but CQI.

Quality assurance involves a measurement and monitoring function, (i.e., quality assessment). Quality assessment is, primarily, a means for determining how well health care is delivered in comparison with applicable standards or acceptable bounds of care. These standards or bounds may be grouped according to the structure of care (institutional, professional and physical characteristics), the process of care (content or nature of the health care delivered) and the outcomes of care (health status and well­being of patients) (Donabedian 1988). Increasingly, quality assurance involves studies of effectiveness data, including health outcomes and the determinants of those outcomes from the perspectives of clinicians, patients, administrators, and policymakers (McDonald 2000). In detecting these differences between how well health care is delivered and applicable standards, quality assessment can also call attention to the need for further HTA or other investigations. In recent years, there has been further development and overlap of the fields of HTA and quality assurance, along with outcomes research, clinical epidemiology, and evidence-based medicine.  

In summary, HTA contributes knowledge used to set standards for health care, and quality assurance is used to determine the extent to which health care providers adhere to these standards (Lohr 1990; Lohr and Rettig 1988). Indeed, major reorganization of health care systems may be required to ensure that stronger evidence is generated systematically for setting standards of care, and that standards of care are broadly implemented (Institute of Medicine, 2001).

Outcomes Research and HTA

In principle, outcomes research concerns any inquiry into the health benefits of using a technology for a particular problem under general or routine conditions. In practice, the term outcomes research has been used interchangeably with the term effectiveness research since the late 1980s to refer to a constellation of methods and characteristics that overlap considerably with HTA. It has received increased attention in the US, particularly in the form of research funded by the AHRQ [http://www.ahrq.gov/] (formerly the Agency for Health Care Policy and Research). The attention given to outcomes or effectiveness research by government and, increasingly, the private sector (Mendelson 1998) reflects greater demand for data on patient and provider experience with technologies beyond what can be learned from the limited number of carefully circumscribed efficacy trials, e.g., premarketing clinical trials for new drugs and devices (McDonald 2000).

Outcomes/effectiveness research has emphasized health problem-oriented assessments of care delivered in general or routine settings; interdisciplinary teams; a wide range of patient outcomes including mortality, morbidity, adverse events and HRQL measures; the use of nonexperimental data (e.g., from epidemiological studies and administrative data sets); variations in practice patterns and their relationship to patient outcomes; and patient roles in clinical decision-making. The scope of outcomes/effectiveness research has expanded in recent years to include collection of experimental data on effectiveness, e.g., from large, simple trials conducted in general practice settings.

Decentralization of HTA

Although technology assessment originated as a primarily centralized function conducted by federal government agencies or other national- or regional-level organizations, HTA has become a more decentralized activity conducted by a great variety of organizations in the public and private sectors that make technology-related policy decisions (Goodman 1998; Rettig 1997). As noted above, an HTA done from a particular perspective may not serve the technology-related policymaking needs of other perspectives. Even for the same technology or clinical problem, there can be widely different assessment needs of politicians, regulatory agencies, health technology companies, hospitals, payers, physicians, and others. These needs are heightened with increased economic responsibilities and pressures on these different parties.

The growth in decentralized HTA activity has arisen less from a reduction in the level of centralized activity than expansion of HTA programs for particular decision-making needs. In the US, there remain multiple government centers with ongoing HTA responsibilities to fulfill particular purposes, e.g., drug and device regulation at the FDA, NIH consensus development conferences, Medicare coverage policies by the CMS, and the technology assessment program of AHRQ. There has been considerable expansion in activities elsewhere, particularly in the private sector, as well as greater reliance by centralized sources on HTA inputs from outside sources. Increasingly, large health care providers and major health care product companies are establishing units devoted to "technology assessment," "pharmacoeconomics," "clinical effectiveness," "health outcomes research," and related areas. More health plans (including various managed care organizations and insurance companies) have established formal programs to assess new procedures and other technologies in support of payment decisions. The number and magnitude of private firms and university centers involved in HTA is increasing. HTA committees (with various names) are now common among medical specialty and subspecialty societies. Hospital networks, managed care organizations and other large health care providers in the private sector have HTA programs to support acquisition and management of pharmaceuticals (e.g., P&T committees), equipment and other technologies and other technology-related needs throughout their systems (Kaden 2002; University HealthSystem Consortium 1996).  

Aside from the growth of HTA in the private sector, even HTA conducted by government agencies is drawing upon more decentralized resources. In the US, the FDA has long relied on advisory panels comprising outside experts to examine clinical trial findings and other evidence to provide recommendations regarding market approval of new drugs, biologicals, and medical devices. CMS has a large Medicare Coverage Advisory Committee (MCAC) [http://www.cms.hhs.gov/FACA/02_MedCAC.asp#TopOfPage], arranged into various panels, that provides recommendations for national coverage policies on new technologies and other interventions, based on review of the clinical literature, consultations with experts, and other data. AHRQ's Evidence-based Practice Centers (EPC) program [http://www.ahrq.gov/clinic/epcix.htm] has established contracts with 13 EPCs, mostly academic health centers and other institutions, including three in Canada, which generate "evidence reports" and technology assessments in support of clinical practice guidelines, coverage policies, and other practices and policies. Indeed, some EPC reports are conducted at the request, via AHRQ, of the NIH Consensus Development Program, CMS, and other government agencies; other requests are made by other organizations in the private sector, such as health professional organizations. In this manner, AHRQ provides a portal for decentralized HTA, via the 13 EPCs, on behalf of government and non-government organizations. AHRQ also administers the US Preventive Services Task Force [http://www.ahrq.gov/clinic/uspstfix.htm], an independent panel of experts in primary care and prevention that systematically reviews evidence of effectiveness and develops recommendations for a broad range of clinical preventive services.  

The Cochrane Collaboration [http://www.cochrane.org/], another highly decentralized, successful model, involves 50 workgroups of volunteer experts around the world, coordinated through about 14 centers based in 12 countries, who conduct systematic reviews of a diverse variety of health care interventions.  

Decentralization of HTA and related functions widens the expertise available to HTA and brings broader perspectives to the process and diminishes or balances potential conflicts of interest. Together, these generally add to the credibility of HTA processes and findings, and lessen any charges that assessments reflect narrow or self-serving interests of a particular agencies or organizations.  

Tracking changes in the locus and magnitude of HTA is confounded by a broadening connotation of the term. Rather than referring only to the comprehensive inquiries involving broad societal impacts envisioned for the field in the 1960s, HTA is now used to refer to almost any evaluation or analysis pertaining to health care technology. Much of the expansion of HTA concerns meeting focused, immediate needs such as a coverage decision for a particular procedure, determination of the cost-effectiveness of a new device, or an equipment purchase decision. Another shift in locus concerns professional responsibility. Whereas technology-related decision-making in health care organizations was largely the responsibility of physicians, it is increasingly shared or redistributed among a wider spectrum of managers and other professionals.

Certain changes in the health care market are prompting greater balance between centralized and decentralized HTA. Hospital networks, large managed care systems and other large systems such as the Department of Veterans Affairs (VA) continually seek to build economies of scale and buying leverage for health care products, ranging from surgical gloves to hip joint implants. With HTA units that are centralized yet responsive to needs of individual facilities, these large organizations can consolidate their HTA efforts and support system-wide acquisition of drugs, equipment, and services.  

As health care providers and payers realize the resource requirements for conducting well-designed evaluations of health care technologies, they weigh the tradeoffs of conducting their own assessments versus subscribing to assessment report series from outside assessment groups. Clearly, assessment requirements vary widely depending on the type of technology involved. Acquisition of commodity products such as most types of syringes and surgical gloves is largely based on price, whereas acquisition of the latest drug-coated coronary artery stent requires a more considered evaluation of safety, effectiveness, cost, and other attributes. Nearly all hospitals and health care networks in the US rely on group purchasing organizations (GPOs) that use economies of scale to acquire most of their health care products. These GPOs, particularly the larger ones, have their own technology evaluation or clinical review committees that examine available evidence on technologies such as implantable cardiac defibrillators and MRI units, whose acquisition is a matter of factors other than price alone. In turn, many GPOs also subscribe to technology assessment report services (Lewin Group 2002).  

Barriers to HTA

Although the general trend in health care is toward wider and improved HTA, several countervailing forces to HTA remain. Foremost, particularly in the US and other wealthy countries, has been a "technological imperative" comprising an abiding fascination with technology, the expectation that new is better, and the inclination to use a technology that has potential for some benefit, however marginal or even poorly substantiated (Deyo 2002). Some argue that the increased potential of technology only raises the imperative for HTA (Hoffman 2002). Another countervailing factor is the sway of prestigious proponents or a "champion" of a technology in the absence of credible evidence. A third impediment is the inertia of medical practice, e.g., in the form of reluctance to change long-standing practice routines, conservative payment policies, and quickly outdated education. This is complemented by lack of opportunities for, or encouragement of, scientific inquiry and skepticism in clinical education.

Ever more effective marketing and promotions, including short courses sponsored by medical product companies to train physicians in using these products, can divert attention from key concerns of HTA. Another obstacle is the limited level of investment, by government and industry sources in HTA and related evaluations of what works in health care. Although some assessment programs and certain HTA findings are nationally or internationally recognized, the resources allocated for HTA in the US are virtually lost in the rounding error of national health care expenditures. Finally, the impression persists in some quarters that the goal of HTA is to limit the innovation and diffusion of health care technology.

Political processes can circumvent or threaten evidence-based processes (Fletcher 1997). One of the higher-profile applications of HTA is in determining covered services for health programs that are provided or funded by governments as well by the private sector. While most of these health programs have HTA processes that support benefits determinations, they are also subject to legislation (laws) in their respective countries, states, provinces, and other jurisdictions. Legislative bodies at these levels can mandate, or require, that health programs provide certain services. For example, in the US, the Congress has mandated that the Medicare program (for the elderly and disabled) provide certain services (e.g., screening procedures) that are not included in the benefit categories under the original Medicare statute. State legislatures have mandated that their Medicaid programs (for people with low incomes) as well as private sector health plans operating in their states, provide certain services. Recent examples of mandated services include autologous bone marrow transplant with high-dose chemotherapy (ABMT­HDC) for advanced breast cancer, bone densitometry screening for osteoporosis, screening mammography, prostate cancer screening, and treatment for temporomandibular joint disorder. Such mandates, including the ones noted here, may or may not be based upon the types of evidence-based methods used in HTA processes. As is the case for other industries, these mandates can be affected by political influence brought, e.g., by "lobbying" or "pressure groups" representing patient advocate organizations, physician groups, health product makers, and others (Deyo 1997; Sheingold 1998).

In some instances, legislative mandates arise through frustration with slowed or delayed HTA processes. A notable instance was the mandate by the US Congress for Medicare coverage of dual energy x-ray absorption (DEXA) for bone mineral density measurement, which had been subject to an assessment involving two federal agencies over a seven-year period (Lewin Group 2000). However, these mandates often circumvent evidence-based coverage policy, by providing an alternative, political route to coverage of technologies. The apparently direct process of mandating coverage of a technology, rather than subjecting it to well-founded HTA, can mask more complex clinical consequences. In the 1990s, many health plans reluctantly agreed to cover HDC-ABMT in response to state legislative mandates brought about by intensive political pressure, and the threat of litigation (legal action in courts). It was not until 1999, after tens of thousands of women were subjected to the procedure, that results of five well-conducted RCTs demonstrated that the procedure conferred no benefit over standard-dose treatment for breast cancer, and caused unnecessary suffering in some women (Berger 1999; Mello 2001; Sharf 2001). Aside from barriers to conducting HTA are barriers to implementing its findings and recommendations, particularly by decision makers and policymakers for whom HTA reports are intended. Among these are: lack of access to HTA reports, complex and technical formats of HTA reports, questionable data quality, absence of real-world applications, and narrow focus (Henshall 2002).

HTA and Underused Technologies

When used properly, HTA can reduce or eliminate the use of technologies that are not safe and effective, or whose cost is too high relative to their benefits. As discussed above, HTA can also be used to remove technologies from the market that are harmful or ineffective. Less attention is given to the ability of HTA to identify technologies that are underused, and to help determine why they are underused (Asch 2000; McNeil 2001). Underuse is prevalent in preventive, acute, and chronic care (McGlynn 2003) and contributes to tens of thousands of deaths and billions of dollars of losses to the economy and unnecessary health care costs (National Committee for Quality Assurance 2003).

For example, there is overwhelming evidence that smoking cessation interventions, including nicotine replacement therapy, the antidepressant bupropion, and counseling, are safe, effective, and cost effective (Anderson 2002; Foulds 2002; Jorenby 1999; Woolacott 2002). However, in Europe, North America, and elsewhere, these interventions are used far less than is indicated. Underuse is attributed to various reasons, including: lack of insurance coverage, concerns about short-term costs without regard to cost-effectiveness in the short-term (e.g., for pregnant women and infants) and the long-term; lack of smoker awareness of effective interventions; insufficient demand by patients, physicians, and the tobacco-control community; and the influence of the tobacco industry on policymaking (Schauffler 2001).

Box 42 shows examples of health care technologies for which good evidence exists of effectiveness or cost-effectiveness, but that are used significantly less than is indicated, even where they are affordable. Although this list applies primarily to the US, many of these technologies are underused elsewhere in North America, Western Europe, and other of the wealthier countries. The reasons that worthy technologies are underused are diverse, and include the following.

Lack of awareness on the part of patients, physicians, and others

• Inadequate information dissemination
• Limited coverage and reimbursement
• Concerns about short-term cost without regard for cost savings and cost-effectiveness in the short- and long-terms
• Inappropriate or unsubstantiated concerns about improper use (e.g., pain therapy)
• Inconvenience and misperceptions on the part of clinicians or patients
• Clinical inertia
• Insufficient supply (e.g., organs for transplantation)
• Disproportionate concerns about adverse effects (e.g., warfarin to reduce risk of stroke)
• Concerns about patient compliance (e.g., polypharmacy for HIV/AIDS)
• Fear of stigma (e.g., treatment of depression)
•  Professional conflicts and "turf battles" on the part of physician specialists, provider institutions, industry, and others

•  ACE inhibitors for treatment of heart failure
•  ACE inhibitors for prevention of renal deterioration in insulin-dependent diabetics
•  Ambulation aids (canes, crutches, walkers)
•  Antibiotics for gastrointestinal ulcers
•  Beta blockers for survivors of acute myocardial infarction
•  Cholesterol-lowering drugs for patients at risk of coronary artery disease
•  Cochlear implants for severe-to-profound deafness
•  Colorectal cancer screening
•  Corticosteroid inhalants for treating asthma
•  Corticosteroid therapy for fetuses at risk of preterm delivery
•  Depression diagnosis and treatment
•  Diabetic retinopathy screening
•  Hepatitis B virus vaccination of infants
•  Implantable cardioverter-defibrillators for survivors of cardiac arrest
• Incontinence diagnosis and treatment
•  Intraocular pressure screening for glaucoma
•  Oral rehydration therapy for dehydrated children
•  Organ transplantation
•  Pain management
•  Polypharmacy (with protease inhibitors) for HIV/AIDS
•  Pneumococcal vaccine for high risk patients
•  Prenatal care
•  Smoking cessation interventions
•  Thrombolytic therapy for acute myocardial infarction
•  Thrombolytic therapy for ischemic stroke
•  Warfarin to prevent strokes due to atrial fibrillation

Conflict of Interest

HTA should consider the potential for conflict of interest on multiple levels. One is on the part of investigators who conducted and reported on the clinical trials and other studies that comprise the body of evidence under review. A second is on the part of sponsors of the primary research, e.g., technology companies, who have varying degrees of control over what research is conducted, selection of intervention and control treatments, selection of endpoints and follow-up periods, and whether research results are submitted for publication. Another is on the part of the health technology assessors themselves, including analysts, panel members, or other experts involved in reviewing the evidence and making findings and recommendations.  

Interpreting the literature for an assessment should include consideration of the existence of potential conflicts of interest that may have affected the conduct of a study or presentation of results. For study investigators, conflicts of interest may arise from having a financial interest (e.g., through salary support, ongoing consultancy, owning stock, owning a related patent) in a health care company (or one of its competitors) that may be affected by the results of a study or being an innovator of a technology under study. A systematic review of research on financial conflicts of interest among biomedical researchers found that approximately one-fourth of investigators have industry affiliations, and two-thirds of academic institutions hold equity in start-up companies that sponsor research performed at the same institutions. Industry sponsorship of research also was associated with restrictions on publication and data sharing (Bekelman 2003). Clinical trials and cost-effectiveness analyses that are sponsored by industry yield positive results more often that studies that are funded or conducted by others (Chopra 2003; Friedberg 1999). Among the reasons suggested for this discrepancy are that industry's publication restrictions tend to withhold studies with negative results. Another is that industry is more likely to sponsor studies (particularly RCTs) in which the results are likely to be positive, i.e., where there is an expectation that one intervention (e.g., a new drug) is superior to the alternative intervention. In the case of RCTs, this latter tendency would undermine the principle of "equipoise" for enrolling patients in an RCT (Djulbegovic 2000).

Peer-reviewed journals increasingly require disclosure of information pertaining to financial interests of investigators and the source of funding of studies (International Committee of Medical Journal Writers 1993; Kassirer 1993; Lo 2000). Some journals have particular requirements regarding protection against conflict of interest for economic analyses that have been subject to considerable controversy (Kassirer 1994; Steinberg 1995). Information about investigators, sponsorship of a study, or other factors that suggests the potential for conflict of interest should be considered when interpreting the evidence. Studies that are subject to potential conflicts of interest may have to be discounted or dropped from the body of evidence accordingly.  

HTA programs should take active measures to protect against potential conflicts of interest among assessment teams and panelists (Fye 2003; Phillips 1994). A conflict of interest may be any financial or other interest that conflicts with one's service on an assessment group because it could impair that person's objectivity or could create an unfair advantage. Conflict of interest is not the same as bias among assessment teams and panelists, which may entail views or inclinations that are intellectually motivated or that would be expected to arise from having a given organizational or professional affiliation. HTA programs should take active measures to minimize or balance bias among assessment teams and panel members.

The following recommendations for managing conflict of interest in practice guidelines development (Choudhry 2002) may be relevant as well to panels involved in HTA and related evidence-based activities.

• A formal process should exist to disclose potential conflict of interest before the guideline development begins.
• All members of the guideline group should be involved in a discussion of conflicts of interest and how significant relationships will be managed.
• Participants who have relationships with industry, government agencies, health care organizations or specialty societies need not necessarily be excluded, but the group has to decide among itself a threshold for exclusion.
• There must be complete disclosure to readers of the practice guidelines of financial and/or other relationships with industry, government agencies, health care organizations and specialty societies.