Values and evidence colliding: health technology assessment in child health

Keywords::

• child health
• comparative effectiveness research
• equity
• health technology assessment
• preferences

The call for evidence of the safety and efficacy of health interventions has grown louder with growing economic constraints in EU countries as well as the arrival of comparative effectiveness research (CER) and the Affordable Care Act in the USA, which directs new funding to CER. While CER is valuable for informed decision-making globally, it is not sufficient. Knowledge of the health system, the settings for care delivery and the information needs of decision-makers, such as cost–effectiveness and an understanding of societal preferences, all play a role in translating evidence into policy. Although in the USA, the Affordable Care Act prohibits cost–effectiveness ratios in coverage decisions [1], such evidence is useful and commonly employed in other jurisdictions. Health technology assessment (HTA) is a comprehensive evaluation framework that generates evidence of the value of new interventions, including medications, diagnostic tests, devices, medical and surgical procedures as well as programs and services [101]. In addition to assessing safety, clinical utility, costs and cost–effectiveness, HTA considers the social, legal and ethical factors that shape decision-making [101]. In so doing, HTA takes into account issues relevant to vulnerable members of society, such as children, that may not be captured with CER. The principle of harm prevention is particularly germane to child health in which others, usually parents, serve as proxy decision-makers and may be highly risk averse. In addition, the need to ensure access to care, when care is mediated by parents or teachers, or is required outside of traditional health settings, such as in schools, may require special programs and services.

HTA also considers equity, in terms of need for the technology and access to it, issues which are not typically considered in cost–effectiveness analysis. For example, health economic guidelines promote the use of quality-adjusted life years (QALY), which weigh life expectancy by health-related quality of life [2]. As a universal measure to guide decision-making across therapeutic areas, patient populations and age groups, the QALY is appealing. But QALY are hard to measure in children, especially young children. A utilitarian perspective that assumes QALY gains are equal across a population may penalize children if the quality of evidence is poorer or scanter than in adults. Such an approach also fails to consider that society may value health gains in children more highly because of their vulnerability [3].

Another equity issue relates to the latency of health benefits which may be deferred for years, even decades in children, such as with vaccinations against infectious diseases. Economic evaluation guidelines stipulate that deferred costs and benefits be discounted by applying a constant discount rate to determine the present value. When upfront costs are high or annuitized, but health benefits are deferred, which is the case for many pediatric prevention programs, the perceived benefit may be unfairly reduced. Careful consideration of when, where and to whom inequities occur is required so that decision-makers understand potential equity-efficiency trade-offs. In addition, HTA in child health is not complete without considering spillover effects and non-health benefits, including changes in parent/caregiver productivity and earnings, family member quality of life and functioning, school performance, educational attainment and future employment and earnings. This is consistent with a patient-centered approach to valuing health outcomes in child health [4].

Although HTA takes into account equity issues, other important aspects including budgetary constraints, pragmatic and political considerations, population demographics, decision-makers’ and governments’ values and societal preferences, also contribute to how healthcare is allocated [5]. Rules of rescue and the desire to address the needs of vulnerable populations may also play a role.

Currently, HTA is caught between pressure on researchers to use increasingly sophisticated methods for synthesizing evidence and the demand by decision-makers for transparency. In the middle of this struggle rest members of society, and in particular parents, whose views cannot be ignored. Parents are vocal stakeholders in the health decisions made for their children and may consider HTA evidence as secondary to harm prevention. For example, despite evidence of the safety of vaccines for viral diseases, some parents, motivated by a zero tolerance for harm, will choose not to vaccinate their child [6]. Strong views regarding exposure to risk of harm, however remote, cannot be ignored by decision-makers. Personalized medicine is another area where parental preferences and social values are critical. Because of the ability to intervene early to diagnose and possibly treat disease, children are strong candidates for personalized medicine, including genotyping. Ethical concerns regarding when and how to disclose findings regarding rare mutations and incidental findings have been growing. Parents’ views regarding the disclosure of genetic findings that affect their children’s future health must be considered alongside HTAs of clinical utility and cost–effectiveness, even when those views may collide with the evidence.

In many ways, HTA, with its recognition of social, legal and ethical factors that influence decision-making and its emphasis on a broad set of outcomes, is the response to questions of the relevance of health economics. To date, child health HTA has lagged far behind adult health HTAs. The University of York Centre for Reviews and Dissemination in the UK maintains a set of databases that indexes HTA reports from around the globe. Examination of these records revealed that between January 2011 and November 2012, there were 959 HTAs of which 698 (72.8%) were devoted to adult health, 69 (7.2%) to child health and 192 (20.0%) to both child and adult health [102]. An approach that considers the HTA factors described above is used by the National Institute for Health and Care Excellence (NICE) in the UK [103] and by the Common Drug Review in Canada [104] for decisions on medications. It is also being applied by the US Advisory Committee on Immunization Practices to develop vaccine recommendations and by the US Secretary’s Advisory Committee for Heritable Disorders in Newborns and Children for newborn screening policy. Thus, HTA reflects a reality that is established in many global regions and importantly promotes greater transparency in decision-making. It is appropriate that we continue to refine and improve our methods for synthesizing evidence, as well as how we interpret, disseminate and incorporate evidence. As we do so, we must keep in mind the challenges in applying HTA to children and the need to elicit societal values and parental preferences. By doing so, we may evolve toward a reality in which not just individual decisions, but entire healthcare systems are based on rational and equitable principles, where all members of society may benefit.

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.