The increasing burden of chronic diseases presents challenges not only to the knowledge and expertise of the professional medical community, but also to the socioeconomic stability of society. Chronic disease—which includes chronic pain, diabetes, and heart disease, among others—represents 75% of health care spending in the USA; patients with five or more chronic diseases account for 76% of Medicare spending [1
Increasingly, treating patients' chronic illnesses requires complex interventions made up of various interconnecting parts [2
]. Especially in a combination of interventions and the possible interactions between them, a more personalized treatment plan is called for. Complementary and integrative medicine is among the options that can be tailored for more personalized/individualized medicine.
The National Center for Complementary and Alternative Medicine (NCCAM) defines complementary and alternative medicine as “a group of diverse medical and health care systems, practices and products that are not presently considered to be a part of conventional medicine. Complementary medicine is used together with conventional medicine, and alternative medicine is used in place of conventional medicine” [3
]. Its integration into health care has shifted more and more to an “integrative medicine” approach which was defined by the Consortium of Academic Health Centers for Integrative Medicine as “the practice of medicine that reaffirms the importance of the relationship between practitioner and patient, focuses on the whole person, is informed by evidence, and makes use of all appropriate therapeutic approaches, healthcare professionals and disciplines to achieve optimal health and healing” [4
In the USA, many patients now turn to interventions in complementary and integrative medicine to treat their illnesses. High usage of complementary and integrative medicine interventions, especially for patients suffering from chronic diseases, has been reported [5
]. In 2007, nearly 4 out of 10 American adults had used a complementary and integrative medicine therapy in the previous 12 months [6
As treatments become more complex (taking into account a patient's local context, subgroup membership, comorbidities, or other factors), the design of research assessing effectiveness requires flexibility in order to accommodate these various factors.
Unfortunately, many complementary and integrative medicine interventions lack the endorsement that evolves from high quality research studies; indeed, much of the evidence that supports their adoption has not made the transition from the expert level into accessible, widespread knowledge. Clearly, stakeholders (physicians, patients, payers, and others) need access to this evidence to make decisions about their treatment options.
Nevertheless, research studies in complementary and integrative medicine are on the rise. In the USA, the National Center for Complementary and Alternative Medicine at the National Institutes of Health and other funding agencies and foundations have supported clinical, translational, and basic research on the efficacy, safety, and mechanisms of action of diverse complementary and alternative medicine modalities. However, to date, the majority of clinical trials have assessed the efficacy of medical interventions rather than their effectiveness.
“Efficacy” refers to the extent to which a specific intervention is beneficial under ideal conditions. By contrast, “effectiveness” is a measure of the extent to which an intervention, when deployed in the field in routine circumstances, does what it is intended to do for a specific population [7
]. Therefore, effectiveness can often be more relevant to policy evaluation and the health care decisions of providers and patients. Unfortunately, some efforts to achieve rigorous methodological purity have resulted in clinical results that are only marginally meaningful, because patients, interventions, and settings are not comparable to the real world. This burden presents the research community with a mandate: to discover not only efficacious treatments, but also interventions that provide the evidence critical for decisions relevant to the treatment of usual care patients.
Drug research follows a clear hierarchical research strategy that establishes efficacy before effectiveness is evaluated. Because of its long history, complementary medicine treatments are often in widespread use before clinical research has been conducted. For complementary and integrative medicine, a reverse research strategy was recommended [8
]. Using a strategy that generates evidence on comparative effectiveness before determining component efficacy will help to focus on treatments that have relevance for practice and a potential for integration into health care while saving research resources.
Because studies in Comparative Effectiveness Research (CER) are designed to be carried out in settings that reflect usual care, they have considerable potential to help health care providers as well as patients and clinicians to choose among currently available therapeutic options in complementary and integrative medicine. The Institute of Medicine defines CER as “the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The purpose of CER is to assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve health care at both the individual and population levels” [10
]. (“Alternative” does not refer to “alternative medicine” but to “best care” options.)
Among other challenges confronting the US health care system is a paucity of information about CER [11
]. The current movement in conventional medicine towards more CER places strong emphasis on the evaluation of different treatment options by including more heterogeneous patients and by using less standardized treatment protocols and more patient-centered outcomes. Furthermore, stakeholder involvement is seen as highly relevant [12
]. Having patients, doctors, health plan managers, hospital executives, and other stakeholders participate in the design of CER can ensure that this vital research focuses on the evidence gaps most relevant to health care decision makers [13
CER offers a wide range of research designs and advanced techniques to distill and condense evidence from different types of studies [14
] and is not limited to randomized trials but includes, among other options, the possibility of using data from observational studies or registries. Additionally, the concept of pragmatic clinical trials has emerged to describe those randomized trials that are designed explicitly to meet the needs of clinical and health policy decision-making and gain increasing acceptance by decision-makers.
Because of the increasing and widespread use of interventions such as acupuncture, mindfulness-based interventions (yoga, meditation, etc.), and nutritional supplements to manage a variety of chronic disorders (chronic pain, cardiovascular disorders, etc.), and because there is a significant lack of evidence that supports decision-making regarding these interventions, the Institute of Medicine has identified them among its priorities for CER in complementary and integrative medicine [10
The aim of this project was to provide recommendations for a strategic framework for CER in the field of complementary and integrative medicine.