|Home | About | Journals | Submit | Contact Us | Français|
This paper discusses an important element that is missing from the existing algorithm of preconception care, namely, comparative effectiveness research (CER). To our knowledge, there has been limited assessment of the comparative effectiveness of diverse interventions that promote preconception health, conditions under which these are most effective, for which particular populations, and their comparative costs. CER can improve the decision making process for the funding, development, implementation, and evaluation of comprehensive preconception care programs, specifically by identifying the most effective interventions with acceptable costs to society. This paper will examine the framework behind preconception care and how the inclusion of comparative effectiveness research and evaluation into the existing algorithm of preconception care could foster improvement in maternal and child health. We discuss challenges and opportunities regarding the utilization of CER in the decision making process in preconception health, and finally, we provide recommendations for future directions.
Comparative effectiveness research remains underutilized in most strategic frameworks of clinical and public health interventions. The present paper presents a strategy to incorporate CER into maternal and child health programs, focusing on preconception care as a case example. Preconception care offers an important window of opportunity to address risk factors throughout the life span, which subsequently impacts other preventive programs in maternal and child health. Accordingly, the present paper focuses on the critical role of CER in preconception care and provides reasons for the lack of its incorporation in MCH frameworks. We conclude with a recommended strategy to incorporate CER into the existing framework of preconception care, which could also be applicable in other maternal and child health programs.
Preconception care refers to a set of interventions that aim to identify biomedical, behavioral, and social risks before conception in the first or a subsequent pregnancy and then modify these risk factors before pregnancy to reduce the likelihood of adverse health effects for the woman, fetus, and newborn through prevention and management . It involves: (a) risk assessment (reproductive life plan, past pregnancy history, past medical and surgical history, medications and allergies, family and genetic history, social history, behavioral and nutritional assessment, mental health, and laboratory testing); (b) health promotion (family planning, stress resilience, nutritional preparedness, immune allostasis, and healthy environment; and (c) medical and psychosocial interventions (individualized for indentified risks, preventive services and primary care) .
The idea of preconception care has its roots within the life course perspective (LCP) . The LCP conceptualizes birth outcomes not only as a consequence of what happened during pregnancy but as a result of the overall developmental trajectories across the life span of the mother , through the following mechanisms: (1) early programming- early life experiences could determine reproductive outcomes in the future by biological inculcation of adverse events during the sensitive developmental periods [4, 5]; (2) cumulative pathways- unrelieved stressors result in allostatic load in excess of what the body system could handle and result in poor health and function [6, 7]. Preconception care offers a unique opportunity to identify cumulative risks and intervene based on understanding of pathogenic pathways before pregnancy, and modification of women’s future health-related behaviors for the promotion of women’s health over the life span .
The clinical components of preconception care include : maternal risk assessment (e.g., family history, behaviors, obstetric history, general physical exam); vaccinations (e.g., rubella, varicella and hepatitis B); screening (e.g., HIV, STD, genetic disorders); and counseling (e.g., folic acid consumption, smoking and alcohol cessation, weight management). The recommendations to include specific interventions into the clinical components of preconception care were based on a systematic review process [9, 10], however, evidence on the comparative effectiveness of different interventions was lacking, as well as the most effective delivery mechanisms for a systematic implementation. For some included interventions, pieces of evidence for effectiveness were gathered from : (a) case series for preconception care participants (e.g., phenylketonuria, rubella immunization); (b) pregnancy data base/pregnancy guidelines (e.g., periodontal disease); (c) non-preconception care group [women who didn’t attend preconception care (e.g., smoking, alcohol, obesity)]; and (d) common sense (e.g., genetic counseling). Only certain components of preconception care (e.g., folic acid supplement, care of diabetes mellitus) have shown to be cost-effective [11, 12]. Interventions that target alcohol and tobacco cessation are effective in some groups of the population but not in individuals who abuse multiple substances [13–15]. These issues highlight the need to incorporate comparative effectiveness research into the existing preconception care algorithm.
The strategic framework of preconception care, known as the preconception care pyramid , has a missing link, namely comparative effectiveness research. CER, defined as “the conduct and synthesis of systematic research comparing different interventions and strategies to prevent, diagnose, treat and monitor health conditions” , carries the purpose of involving and informing patients, providers, and other stakeholders in the healthcare decision-making process . Hence, CER is charged with the tasks of generating evidence-based information on which interventions are more effective under real world conditions (as opposed to controlled settings) and for which group of the population, as well as the timely dissemination to patients, care givers, and decision makers.
Studies that purposely compare the comparative effectiveness of alternative preconception care packages, programs, and delivery mechanisms are plainly missing in the preconception care literature. The authors of this article strongly believe that incorporating CER as an integral part of the preconception care pyramid will guide and strengthen its components. The preconception care pyramid , consists of four overarching goals (i.e., improve coverage, risk reduction, empowerment, and disparity reduction), and a set of ten recommendations with respective action steps and implementation strategies. In this context, CER can provide an evaluation framework for the action step and implementation strategies of the pyramid by examining the evidence on the relative effectiveness, benefits, and harms (if any) of different preconception care interventions or providing alternative ways to effectively deliver preconception care. CER can also strengthen the upper level components (recommendations, goals, and vision) by engaging patients and stakeholders in the decision-making process for the generation of patient-centered cost-conscious guidelines. Figure 1 illustrates the role CER could play as a tool to facilitate evaluation of components of preconceptional care.
Comparative effectiveness research can assist relevant stakeholders to make informed decisions that will improve preconception care implementation. CER assesses not only the effectiveness of alternative clinical treatments, but also best practices on service provision, which permits the selection of the best choices (most effective) for particular populations and different conditions. CER can serve as an evaluation framework to assess the health benefits for the mother-to-be, select and package interventions for specific populations of women according to risk profiles, to be delivered in ways that maximize health gains (and minimize human and monetary costs), and galvanize the continuous improvement of preconception care at the local and national level . Hence, the key role of CER in preconception care programs lies in the benefit of determining the most effective set of interventions within and between components of preconception care, a step that can help package clinical and community-based interventions for the achievement of greater health benefits across diverse groups of women.
The scope and blend of components of preconception care currently lack uniformity in diverse practice settings. By assessing each of the components and their relative effectiveness in real world practice, CER may lead to improved preconception care within diverse settings (e.g., Healthy Start, Private clinic, Health Department, and Hospital). As a step further, among the interventions with reasonable effectiveness, CER studies can be logically complemented with cost-effectiveness analysis; or those interventions with higher costs should be subject to more rigorous assessment of comparative evidence . The potential benefit of CER was summarized by Brown et al.  in the following: “Standardized comparativeness and cost-effectiveness data will give physicians an information system to identify the interventions that confer the greatest value to patients, and thus deliver higher quality care than possible with evidence-based data alone while allowing the most cost-effective care”.
Information on the comparative effectiveness of different interventions is often not available for all population groups, as information used to generate current guidelines was based on efficacy studies that looked at selected populations (e.g., women with recurrent neural tube defects, obese women, or diabetic women); those studies have limited applicability to the majority of the population that may benefit from preconception care. In contrast, CER studies are most optimal to inform the development of population-based guidelines because of the intentional inclusion of diverse socio-demographic and clinical subpopulations. In particular, CER can help identify some strategies that could work for one group of population but not for the other. Herein resides the utility of CER, a tool that is capable of customizing pre-conception care recommendations across individuals with different needs.
To incorporate CER into actual recommendations for improving preconception health, it needs to be held accountable under adequate principles and weigh out whether the process, irrespective of resource allocation recommendations, is fair and transparent. We argue that a framework of accountability for reasonableness , usually applied to evaluate a priority setting process against ethical conditions, can guide the incorporation of CER into the preconceptional health care delivery model. The principle of accountability for reasonableness encompasses publicity (all stakeholders involved in limit setting decisions and priority setting), evidence revision in light of further evidence, and support from funding agents. Stakeholders are empowered to study each step in the decision making process over time, and to assess whether the process has been done fairly across comparison groups. As a result, key stakeholders are more likely to buy-in to an inherently transparent and fair process.
The missingness of CER in the national discourse on preconception care is a consequence of the pervasive lack of CER studies both in medicine and public health. Although CER is an integral part of the evidence-based practice paradigm [21, 22], nonetheless, it has historically received considerable less attention and allocation of resources compared to other forms of clinical and epidemiological research. Indeed, recent reviews indicate that less than a third of clinical studies (i.e., randomized trials, observational studies, and meta-analyses), meet the criteria for comparative effectiveness research [23, 24]. Furthermore, only 2 % integrated cost-effectiveness analysis, an important component of CER.
Insufficient funds and governmental support could have contributed to the inertia in the CER field, but this situation has changed recently. In 2009 the US Congress passed the American Recovery and Reinvestment Act, in which $1.1 billion was allocated to support CER studies through Agency for Health Research Quality and the National Institutes of Health . Subsequently, the Patient Protection and Affordable Care Act of 2010, led to the creation of the Patient-Centered Outcomes Research Institute , an independent non-profit organization aimed at providing leadership in conducting and disseminating CER studies. In this context, researchers in the Maternal and Child Health field must not fall behind in taking advantage of these new developments, and should strive to address priorities for CER of preconception, interconception, antenatal, and postnatal care interventions.
Because CER is a relatively recent and rapidly evolving field, its conceptual basis and its role in healthcare decision making are just being recognized and integrated in all spheres of medicine and public health. For this reason, it is not surprising that CER is missing in the preconception care framework. In the MCH context, we must recognize that the lack of CER evidence represents a gap in knowledge of what works, for whom, and under particular conditions hindering the potential of maternal and child health interventions (including preconception care). In this regard, CER studies must be clearly differentiated from research assessing the efficacy and/or the effectiveness of a single intervention (e.g., comparing active treatment with inactive treatment) which are relatively abundant in the literature. Distinctively, CER encompasses comparison of two or more different interventions in regards to their real-world effects (relative effectiveness), with or without cost considerations, among different populations groups, and diverse settings (i.e., what works, for whom, and under what conditions) .
An important reason for the missingness of CER relates to the reluctance to embrace one of its components, namely cost-effectiveness. Conducting research on preconception interventions was one recommendation of the CDC/ATSDR Preconception Care Work Group , specifically highlighting the importance of designing and conducting analyses of costbenefit and cost-effectiveness. Despite its importance, there has been very limited integration of economic evaluations into the general framework of preconceptional care especially. This represents an especially acute challenge for the full realization and integration of CER into the preconception care framework . Contrary to other subtypes of CER studies (e.g., systematic reviews, observational studies, and pragmatic trials), there has been reluctance to use cost-effectiveness analyses (CEA) due to fear of rationing the health care system and ethical issues that may arise. At a time of economic difficulties, this idea needs reconsideration under the CER framework .
Myriad competing healthcare practices of unproven value continue to consume resources inefficiently, which points to the pressing need of considering cost data as part of the CER strategic implementation. Results from cost-effectiveness analyses can assist patients, physicians, and payers in the decision making process (although not as the main driving force) by identifying interventions that provide greater value (i.e., more population and individual health gains per unit of cost) and inform the allocation of constrained resources. Therefore, CER with inclusion of cost data could be an optimal tool in the decision/policy making process to maximize health and healthcare efficiency. Thus, obstacles associated with utilizing cost data need to be overcome to fully realize the potential of CER.
Over the past few years some researchers also identified other factors that lead to the missingness or underutilization of CER in the decision making process of health care systems, which include: lack of understanding and/or mistrust of methods , ethical reasons , limited availability of comparable MCH measures and instruments , methodological challenges ; and insufficient workforce development in CER.
Comparative effectiveness research is relatively new to health care professionals and most of them have no training in CER methods . Therefore, it could be difficult for the majority of health professionals to understand the analytical methods that CER provides and/or readily assimilate CER-related concepts, such as quality-adjusted life years . This has lead to considerable misconceptions, for example, the misunderstanding of the role of economic evaluation in CER for decision makers in the US . Lack of consistency in CER methods, particularly, in determining which types of study designs (RCTs vs. observational) would best provide comparative effectiveness data is one important challenge that further confounds the understanding of CER.
Clinicians argue that the inclusion of comparative effectiveness and comparative costs in the process of clinical decision making is unethical. This argument is flawed since rationing is part of what healthcare professionals do routinely when they assess the evidence and implement recommendations based on quality of the evidence. Also, cost represents adverse consequences upon others due to the decisions we make and ignoring this cannot be ethical . According to Williams , the most important ethical issues are rather related to which costs and/or benefits to count and how to count them. Furthermore, several authors have indicated ways to successfully apply equity values and rightfully use cost-effectiveness data, which include the application of social justice principles and the incorporation of participatory approaches. Since continuous stakeholders’ involvement is a cornerstone of CER , the shared decision making process will likely facilitate the resolution of ethical concerns.
One important challenge in the comparison of effectiveness measures is the lack of a uniform comparability yardstick among diverse interventions that consider different diseases or specific health effects (e.g., preterm births prevented, number of maternal complications, days of hospitalization, reduction of neural tube defects, etc.) . There is a growing trend to evaluate interventions using a standard measure, which permits comparisons across diverse interventions and diseases. One approach is to measure health effects in terms of life expectancy and health-related quality of life. For instance, a ratio for each alternative can be calculated to reflect the years of life gained (as in cost-effectiveness analyses). Life expectancy, however, does not account for the quality of years gained (for instance, an added year of life with pain is the same as an added year without pain). Quality-adjusted life years (QALYs) can compensate this limitation by combining health-related quality of life with life expectancy (1 QALY = 1 year in perfect health) . Nonetheless, the absence of valid and reliable MCH-tailored health-related quality of life scales is one of the biggest challenges of integrating CER into preconceptional care.
Comparative effectiveness research studies carry unique methodological challenges because of the focus on real-world effects and stakeholder participation . Particularly observational studies may be more vulnerable to random error or confounding variables such as other concurrent health interventions, co-morbid conditions, or other unmeasured effects. Rigorous study designs must be combined with adequate statistical techniques for generating robust CER data, which should not only mitigate these biases (e.g., propensity scoring methods and multivariate adjustment) but also the quantification of the impact of uncertainty in the decision analysis (e.g., probabilistic sensitivity analyses). Other logistical issues include the access, utilization, and proper linkage of population-based databases enhanced with cost data and clinical information. Because of the emphasis of participatory approaches in CER studies, these may be also more susceptible to systematic errors such as selection bias (bias in recruitment), and challenges in obtaining institutional review board approvals. Nevertheless, these challenges are not insurmountable with capable research teams and infrastructure.
A major roadblock is the lack of infrastructure particularly related to workforce development capable to implement and utilize CER findings . Fortunately, this roadblock is starting to be surpassed with the implementation of renewed support for funding in CER , which may propel several system-wide changes, such as the development of educational curricula in CER-related areas by academic institutions, increased demand for CER studies from health organizations, and greater involvement of the non-academic community into the implementation and utilization of CER studies [41, 42].
This paper highlights reasons why it is imperative to incorporate CER into the current efforts to promote positive maternal and infant birth outcomes. CER can help identify new and emerging clinical/community interventions to improve preconception health, review and synthesize current medical research relevant to women’s health of reproductive age, identify gaps between existing maternal and child health research and the needs of clinical practice, promote and generate new scientific evidence and analytic tools, identify needs for human development in preconception care, translate and disseminate research findings to diverse stakeholders, and reach out to diverse stakeholders. Given the importance of determining which preconception care packages are most effective under real world conditions, it will be helpful to:
The implementation and utilization of CER, not just limited to preconception care interventions but to a wider scope, will not be an easy task because of the current nature of the health care system in the United States. It is relatively easier to implement CER in countries where centralized sources of information and comparable financing/billing mechanisms exist. In the US there are many parties (interest groups) involved and it might not be easier to implement CER consistently under a societal perspective. However, some programs, such as those federally funded (e.g., MCH Bureau) could potentially adapt a more consistent CER implementation strategy. Since the impetus for preconception care is federally-supported for MCH programs, such as the Healthy Start program, this provides an opportunity for a more consistent CER integration into actual decision-making mechanisms. In this context, participatory approaches can be used to quantify the relative effectiveness of interventions using measures that matter most for patients (e.g., quality of life domains, quality adjusted life, well-being, etc.). To this end, innovative ways of interdisciplinary collaboration will need to occur between traditional fields involved in health services research (clinical sciences, epidemiology, biostatistics, and health care economics) and other fields from the social and behavioral sciences.
This work is supported by funding from the Agency for Health Care Research and Quality (AHRQ) through a grant on “Clinically Enhanced Multi-Purpose Administrative Dataset for Comparative Effectiveness Research” (Award#: 1R0111HS0 19997).
Publisher's Disclaimer: Disclaimer: The views expressed in this paper do not necessarily reflect those of the AHRQ.
Conflict of interest The authors have no any conflict of interest.