Evidence-Based Dentistry for the Dental Hygienist

Indiana

Evidence-based dentistry (EBD) is an approach to caring for patients that is intended to increase the likelihood that a patient will receive optimal care while reducing the variation in treatment that is prevalent in the profession. It represents a systematic method for evaluating published research so that the knowledge necessary for the treatment of individual patients can be gleaned and applied. While this knowledge has always been assumed to be present when a treatment decision is made by a dental practitioner, too often the knowledge is lacking, out of date, or inaccurate. To a great extent, this problem is related to how the specific and special knowledge that defines the profession—the dental knowledge base—has been accumulated and made available to dental practitioners. Therefore, it is useful to review the evolution of the dental knowledge base, the source of evidence for EBD.

How the Dental Knowledge Base Has Evolved

The dental knowledge base is simply the collection of all that is known about oral health and disease and treatment methods and outcomes. Its contents comprise all of the extant dental journal articles and textbooks, as well as the minds of all oral health practitioners. The dental knowledge base is the foundation of the dental professions and the principal determinant of how dentists and dental hygienists practice. It informs professional decision making, and portions of it comprise the content of predoctoral and postdoctoral dental school curricula. Obviously, the dental knowledge base has grown over time, as new information and understandings have been contributed by researchers, practitioners, and manufacturers. More importantly, however, the knowledge base has also evolved over time with respect to how the information it contains has been created, synthesized into knowledge, and disseminated. One way to understand this evolution is to delineate four eras, or ages, in the evolution of the knowledge base (Fig 1-1). A brief consideration of these ages may help put the profession’s current involvement in EBD into perspective.

Fig 1-1 The four eras in the evolution of the dental knowledge base.

The age of the expert

Evidence of the treatment of teeth extends far back into human prehistory,¹ and early writings discuss tooth worms,² the supposed cause of toothache at that time. Ancient Roman, Greek, Egyptian, and Asian cultures all contain examples of dental technology related to replacing, retaining, and crowning teeth.³ During the early Middle Ages, barber-surgeons and toothdrawers extracted teeth for pain relief. The knowledge and skills underlying all of this early activity was strictly experiential; practitioners learned by doing. Throughout this age, initial training was limited principally to the master-apprentice relationship, wherein experts passed on their experience to one or more apprentices. After training, apprentices were largely on their own because there were virtually no texts and limited opportunities for sharing knowledge among practitioners, who were mostly illiterate anyway and restricted in travel radius.

Thus, information creation was an individual activity, as each practitioner learned from his or her own experiences. Synthesis of information into knowledge was also an individual activity, as the sole source of information to be synthesized was the experience of the individual practitioner. Knowledge dissemination was spotty and cumbersome. Apprentices lucky enough to train under a master or expert who observed carefully, accurately synthesized these observations into new knowledge, and then disseminated this new knowledge to them may have benefited, but other apprentices did not. Under these arrangements, the dental knowledge base expanded slowly, because although individual practitioners may have made new observations and even synthesized observations into new knowledge, dissemination to other practitioners was not guaranteed. Unfortunately, incorrect knowledge was also slow to be discredited and discarded because of the same problem with dissemination. This method of knowledge dissemination, at the feet of the expert, contributed to a deeply entrenched tradition of respect for expert opinion in the profession, one that persists today.

The age of professionalization

Around the middle of the 18th century, the dental knowledge base entered its second era, as dentistry began to develop the elements of a profession. Pierre Fauchard had published his comprehensive textbook representing his observations, those of his mentor, those included in the few earlier books on dentistry, those of other dentists at the hospital where he practiced, and later, those of other experts in Paris.⁴ This textbook exemplified a new era in knowledge synthesis, enabled by better access to knowledge created by others. However, what was synthesized was still simply the experience or observations of other individuals. There was almost no protocol-based, controlled experimentation for the creation of new knowledge. What experimentation did occur focused on the development of new treatment techniques and was typically more like tinkering than carefully designed research.

Thus, much incorrect knowledge, including a belief in tooth worms, persisted. Regardless of the quality of the knowledge synthesized, however, the availability of textbooks represented an extremely important improvement in dissemination of the dental knowledge base. Dissemination in this age of professionalization was further strengthened through the establishment of dental schools and the first dental society journals in the 1840s. For the first time, it was possible for practitioners to learn from the experience of others both during and after their training.

The age of science

The dental knowledge base entered its third era, the age of science, approximately at the dawn of the 20th century, presaging the profession’s gradual shift from proprietary to university-based educational institutions. Knowledge creation accelerated as protocol-based, controlled experimentation became more common, and the scope of inquiry broadened to more fully include the causes and prevention of dental diseases. Synthesis of knowledge evolved from simple statements of fact based on an expert’s experience and opinion to identification and consideration of the available information in the scientific literature. The vehicle for this synthesis was the traditional review of the literature. While the literature review partially supplanted expert opinion as the most popular method to synthesize and disseminate knowledge from the dental knowledge base, it is important to remember that the expert was still the key element of the review, selecting the studies to be included and providing a subjective interpretation of this literature. Knowledge dissemination enjoyed its most active period yet, with early rapid growth of university-based predoctoral and postdoctoral dental curricula, the proliferation of dental journals, as well as organized continuing dental education through both in-person sessions and journals.

The age of evidence

The dental knowledge base has now entered a fourth era, in which attention to the strength of the evidence is heightened. To date, knowledge creation in this era can be characterized by the dominance of the randomized controlled trial (RCT), which represents the research design most likely to produce an accurate and valid finding (see chapter 2). Because of their complexity and expense, RCTs have been employed only occasionally to answer questions related to dental treatments. Thus, much of the information in the dental knowledge base has been gleaned from observational studies, which are more likely to be inaccurate (see chapter 2). However, improved multivariable analytic techniques are beginning to increase the value of these observational study designs.

The hallmark of the age of evidence is the systematic review, described in more detail in chapter 2. This method of knowledge synthesis, which is rapidly replacing the traditional literature review, represents another important step in strengthening the evidence that supports treatment decision making. Systematic reviews represent a substantial change in the paradigm of knowledge synthesis by ensuring inclusion of all relevant evidence, de-emphasizing the role of the expert, and minimizing bias through strict protocols demanding objectivity and transparency in the review process.

The methods of knowledge dissemination prevalent in the previous era (ie, dental curricula, texts, scientific journals, and continuing dental education), continue to be prominent, but there are initial signs of change here as well. Evidence-based clinical guidelines promulgated by various agencies and societies are increasingly common, and evidence summaries, essentially abstracts of systematic reviews accompanied by critical commentaries, form the bulk of the content of two dental journals⁵,⁶ and appear occasionally in others. Perhaps the most significant change concerning the dissemination of the dental knowledge base in these early years of the age of evidence is the greatly enhanced access to the knowledge base available through the Internet. For the first time, both practitioners and the general public can, with little special effort, obtain the information that in theory drives diagnostic and treatment decisions.

Thus, dentistry is in the midst of a new era that is changing the definition of the special knowledge used to treat patients. That knowledge used to be the province of experts but is more and more becoming available to anyone with the skill and willingness to thoroughly search the scientific literature to find all the studies relevant to a particular problem or question, analyze those studies, and from them synthesize the best solution to the problem or answer to the question. Better yet, many of these problems and questions have already been addressed, and the syntheses are available as guidelines or summaries. This text will help you gain the skills necessary to search the literature and synthesize the relevant studies as well as evaluate the efforts of others who have done so.

How EBD Developed and How It Is Defined

EBD is a direct descendent and analog of a similar evolution of the medical knowledge base, termed evidence-based medicine (EBM). The basic concept of basing decisions concerning the treatment of patients on evidence is not new. However, as was described in the evolution of the dental knowledge base, what constitutes evidence has undergone substantial change over time⁷ (Fig 1-2). Personal experience of a single individual was replaced by the synthesized observations of multiple practitioners, which in turn was replaced by the results of simple, single research studies, which in turn was replaced by the synthesized results of several research studies (ie, literature review). Now the traditional literature review is being replaced by the systematic review.

Two individuals are credited as the principal drivers of the evidence-based movement, Archie Cochrane and David Sackett. Cochrane was a Scottish physician and epidemiologist who advocated the application of scientific methods, especially RCTs, to evaluate the effectiveness and efficiency of medical treatments. He is best known for his influential book, Effectiveness and Efficiency: Random Reflections on Health Services, published in 1972.⁸ The principles he clearly set out in this book were straightforward: Because resources would always be limited, they should be used to provide those forms of health care that had been shown in properly designed evaluations to be effective. Evidence from RCTs, he stressed, are likely to provide much more reliable information than other sources of evidence. The Cochrane Collaboration, named after Archie Cochrane, is an international network of volunteers that prepares and updates systematic reviews on a broad variety of topics as well as maintains the largest collection of records of RCTs in the world.⁹

Fig 1-2 Different concepts of what constituted evidence throughout the four eras of the dental knowledge base. The circles increase in size as they move from the age of the expert to the age of evidence.

Sackett is credited with creating the term evidence-based medicine. The term was first used at McMaster University in 1990 to describe an attitude of enlightened skepticism toward the application of diagnostic, therapeutic, and prognostic technologies in day-to-day patient management.¹⁰ The term was first published in 1991¹⁰ and reached widespread visibility in 1992 with the publication of a description of the concept in the Journal of the American Medical Association.¹¹

The classic definition of evidence-based medicine emerged a few years later from the same group at McMaster University who pioneered the movement: the conscientious, explicit and judicious use of current best evidence in making decisions about the care of the individual patient.¹² This definition focuses on the integration of individual clinical expertise with the best available external clinical evidence. This definition was refined a few years later to incorporate patient preferences and values.¹³

Both of these definitions emphasize the importance of best evidence, a term that from the beginning of the EBM movement has been associated with a rigorous process for finding the least biased information pertinent to an individual patient’s treatment.

As previously noted, EBD grew out of the EBM movement. Although the first systematic review addressing a clinical dentistry topic appeared in 1989,¹⁴ it took more than a decade before EBD progressed to a point where the most widely read dental journal published a paper explaining this approach to practice.¹⁵ Since that time, EBD has experienced steady growth, but for a variety of reasons, not all clinicians have adopted this approach to practice.¹⁶ The most commonly used definition of evidence-based dentistry is from the American Dental Association (Fig 1-3): An approach to oral healthcare that requires the judicious integration of systematic assessments of clinically relevant scientific evidence, relating to the patient’s oral and medical condition and history, with the dentist’s clinical expertise and the patient’s treatment needs and preferences.¹⁷

Fig 1-3 Definition of evidence-based dentistry as published by the American Dental Association.¹⁷

As described in Fig 1-4, and consistent with its definition, EBD has three components: the scientific evidence, the clinician’s expertise, and the patient’s needs and preferences. All are equally important in helping patients make individual decisions about their personal health care based on the current knowledge base in dentistry. The role of the clinician in this process is very important and includes identifying and appraising all of the currently available best evidence on a topic, ascertaining how the evidence may be applied to the patient’s personal needs, and helping the patient to make treatment decisions that incorporate this knowledge in consideration of their personal preferences.

Fig 1-4 The three components of EBD.

Evidence as the Hallmark of EBD

Practitioners are urged to use the current best evidence, best research evidence, and systematic assessments of clinically relevant scientific evidence in treating their patients. The majority of this textbook is devoted to describing what this evidence is and how it is obtained. Highlighted here are two basic concepts that shape the nature of the evidence in EBD: best evidence and systematic reviews.

Best evidence

Not all evidence is created equal. That is, some evidence is more likely to be valid than other evidence. Validity measures how accurately the evidence reflects what is true, and it is an essential characteristic of evidence. Some types of evidence are more vulnerable to bias than others, and bias is the principal enemy of validity. Bias is the existence of factors or processes that can influence the results or conclusions of a trial. Bias occurs when there are important differences in (1) the way in which subjects or groups of subjects are treated or observed or (2) how data is measured or analyzed. In other words, it is anything that causes systematic error to be introduced into the sampling, intervention, or measurement procedures, thus favoring one outcome over another. As noted in the evolution of the dental knowledge base, research designs (ie, knowledge development) have become increasingly sophisticated over time, starting with the single observer and culminating with the RCT. The purpose behind this maturation has been to reduce the risk of bias in the information, or evidence, that is created.

EBD demands that the evidence upon which treatment decisions are based have the lowest possible risk of bias. There is therefore a hierarchy of study designs that corresponds to the differing amounts of risk of bias (see Fig 2-11). Not surprisingly, this hierarchy mirrors the maturation of the dental knowledge base. At the top of the hierarchy is the systematic review, followed by the RCT and the nonrandomized controlled trial. Next are cohort studies, case-control studies, and cross-sectional studies. These study designs will be described in more detail in chapter 2. At the bottom of the hierarchy are case studies, and, in the absence of any formal observational data, expert opinion. Unfortunately, RCTs or other low-bias evidence are not available to support every decision a practitioner must make. Therefore, the term best evidence really means the best available evidence based on this hierarchy of study designs. If higher levels of evidence are not available (ie, systematic reviews or RCTs), then one must seek studies lower in the hierarchy while at the same time acknowledging the potential for increased bias. In some instances, little more than expert opinion may be the best evidence currently available.

However, another requirement of EBD is a thorough search for the best available evidence. It is not appropriate for a practitioner to rely on the first piece of evidence encountered in a cursory search or to alter his or her practice in response to a single article read in a professional journal. The search should always aim for the highest levels of evidence that are likely to be less biased. It is only when studies using these research designs are not available that one uses the lower levels of evidence.

Systematic reviews

The systematic review is quickly surpassing the traditional literature review as the preferred method for summarizing and synthesizing relevant research evidence. The systematic review offers the advantage of reducing the biases inherent in traditional literature reviews while providing clinically relevant information to aid in decision making. These reviews, which follow strict protocols similar to other research endeavors, assess the strength of the available evidence for a given clinical question, thereby identifying not only what is but also what is not known.

A systematic review is designed to minimize the biases that are usually present in traditional literature reviews.¹⁸ The most frequent sources of bias in traditional reviews involve not including all of the relevant studies and not combining the information from the studies in an objective manner that takes individual study weaknesses into account. In part, these biases arise because traditional reviews of the literature tend not to be well-focused on a specific problem. Systematic reviews, on the other hand, focus on specific clinical questions. The question is usually expressed in the PICO format, identifying the problem or disease of interest, the intervention or treatment in question, the comparison treatment (usually the alternative treatment), and the outcome through which the intervention and comparison treatments will be evaluated. This narrower focus permits a much more careful and complete search and selection process to identify and include all relevant studies that have addressed the question of interest. Because the topic is limited, the number of articles that contain information is also usually quite limited so that their careful analysis is feasible.

Perhaps the best way to envision a systematic review is to think of it as a scientific study that is guided by the development of a protocol that outlines all steps in the review process. Systematic reviews are not simple surveys of the literature; they are exhaustive searches for information on a narrow topic that is phrased as a clinical question. Because systematic reviews are designed to minimize bias, they require the prior determination of search methods, inclusion criteria, and evaluation criteria, which reduces the chance of bias in deciding what studies to include and in evaluating the strength of those studies.¹⁹ Thus, the results of a systematic review will represent the best, most current evidence available that addresses a specific clinical question (Fig 1-5). See chapter 2 for more detailed information on systematic reviews.

Fig 1-5 The advantages of a systematic review. A systematic review uses scientific methodology to systematically identify all relevant literature around a clinical question, critically appraise the studies, evaluate the data, and develop conclusions. A high-quality systematic review is a reliable source of valid information for clinical decision making.

Implications of EBD for Dental Hygiene Practice

Using EBD in clinical practice essentially involves identifying and using the best available scientific evidence in caring for patients. EBD also incorporates the clinician’s expertise and the individual patient’s needs and personal preferences during treatment decision making. These decisions are ultimately made by the patient and are very personal, and thus they will vary from patient to patient. However, accessing and using current best available evidence is at the forefront of the decision-making process. There are five fundamental steps in using EBD to help patients make individual decisions regarding the treatment that is right for them (Fig 1-6).

Fig 1-6 The five steps of using EBD in clinical practice.

The first step is to ask a clinical question that is relevant to the condition of the patient. The clinical question is frequently described in a PICO format, as described previously (see Fig 2-8). The second step is to systematically access the most current scientific evidence on the clinical question. This is described in more detail in chapters 2 to 6. The third step is to critically appraise the identified literature. This step provides insight into the strengths and weaknesses of the study, which is necessary when deciding if and how to use evidence from a study in practice. The fourth step is to actually apply the evidence in practice. This may necessitate a change in practice patterns, and if so, it is essential that any impact be assessed. This assessment is the fifth step in applying EBD and specifically looks at any changes in outcomes as a result of applying the evidence in practice.

The role of a dental hygienist is critical in helping the dental team implement EBD. Most important is the awareness and the expectation that scientific evidence will change over time and that it is essential to keep up with the new knowledge. The skills acquired through EBD are likely the most critical in helping you maintain a current knowledge base over the course of your career.

1. What is the dental knowledge base, and what are its four eras?

2. During the age of the expert, how was training conducted and knowledge gathered, and how did the dental knowledge base expand?

3. During the age of professionalization, how was training conducted and knowledge gathered, and how did the dental knowledge base expand?

4. During the age of science, how was training conducted and knowledge gathered, and how did the dental knowledge base expand?

5. During the age of evidence, what is the main source of knowledge creation?

6. What is the hallmark of the age of evidence, and how is it different from a traditional literature review?

7. What are some changes in knowledge dissemination prevalent in the age of evidence?

8. Who were the principal drivers of the EBM movement?

9. What is the Cochrane Collaboration?

10. Who is credited with creating the term evidence-based medicine, and what is its current definition?

11. What is best evidence?

12. What is the definition of evidence-based dentistry?

13. What are the three components of EBD, and what is the role of the clinician in implementing EBD?

14. What are validity and bias, and how are they related?

15. What is the hierarchy of study designs, and how is it impacted by bias?

16. What is a systematic review, and what are some advantages of systematic reviews?

References

1. Coppa A, Bondioli L, Cucina L, et al. Palaeontology: Early Neolithic tradition of dentistry. Nature 2006;440:755–756.

2. Ichord L. Toothworms & Spider Juice: An Illustrated History of Dentistry [electronic resources]. Brookfield, CT: Millbrook Press, 2000.

3. Guerini V. A History of Dentistry from the Most Ancient Times until the End of the Eighteenth Century. Philadelphia: Lea & Febiger, 1909. http://books.google.com/books?id=UMJpAAAAMAAJ&printsec=frontcover&source=gbs_ViewAPI#v=onepage&q&f=false. Accessed 7 October 2012.

4. Weinberger B. Pierre Fauchard, Surgeon-dentist: A Brief Account of the Beginning of Modern Dentistry, the First Dental Textbook, and Professional Life Two Hundred Years Ago. Minneapolis: Pierre Fauchard Academy, 1941.

5. Evidence-Based Dentistry website. http://www.nature.com/ebd/index.html. Accessed 7 October 2012.

6. Journal of Evidence-Based Dental Practice website. http://www.jebdp.com. Accessed 7 October 2012.

7. Claridge JA, Fabian TC. History and development of evidence-based medicine. World J Surg 2005;29:547–553.

8. Cochrane AL. Effectiveness and Efficiency: Random Reflections on Health Services. London: Nuffield Provincial Hospitals Trust, 1972.

9. Cochrane Collaboration website. http://www.cochrane.org/about-us. Accessed 7 October 2012.

10. Guyatt G. Preface. In: Guyatt G, Rennie D, Meade M, Cook D. Users’ Guides to the Medical Literature: A Manual for Evidence-Based Clinical Practice, ed 2. Columbus, OH: McGraw-Hill, 2008.

11. Evidence-Based Medicine Working Group. Evidence-based medicine. A new approach to teaching the practice of medicine. JAMA 1992;268:2420–2425.

12. Sackett DL, Rosenberg WM, Gray JA, Haynes RB, Richardson WS. Evidence based medicine: What it is and what it isn’t. BMJ 1996;312:71–72.

13. Haynes RB, Devereaux PJ, Guyatt GH. Clinical expertise in the era of evidence-based medicine and patient choice. Evid Based Med 2002;7:36–38.

14. Bader J, Ismail A. Survey of systematic reviews in dentistry. J Am Dent Assoc 2004;135: 464–473.

15. Ismail AI, Bader JD; ADA Council on Scientific Affairs and Division of Science. Evidence-based dentistry in clinical practice J Am Dent Assoc 2004;135:78–83.

16. Bader J. Stumbling into the age of evidence. Dent Clin North Am 2009;53:15–22.

17. American Dental Association Center for Evidence-Based Dentistry website. http://ebd.ada.org. Accessed 4 October 2012.

18. Mulrow C, Cook D, Davidoff F. Systematic reviews: Critical links in the great chain of evidence. In: Mulrow C, Cook D (eds). Systematic Reviews. Philadelphia: American College of Physicians, 1998:1–4.

19. Egger M, Smith G. Principles of and procedures for systematic reviews. In: Egger M, Smith G, Altman D (eds). Systematic Reviews in Health Care, ed 2. London: BMJ Books, 2001: 23–42.

This chapter discusses common designs for dental research from the approach of a consumer of dental research literature. Appropriate study design is not only integral to answering clinical questions, but the design can also influence the level of evidence and the confidence that can be placed in the clinical outcomes.

When assessing a study, the prudent clinician will consider the appropriateness of the research methodology, the strength of the experimental design, and the control of potential bias. Understanding the strengths and weaknesses of a study within the larger context of the existing literature serves to inform the clinical decision-making process.

The type of question being asked determines the appropriate study design. Even though some designs are inherently stronger because of their control of potential bias, the design is largely determined by the nature of the investigation. Table 2-1 summarizes the types of questions often asked and the appropriate designs for each.

Table 2-1 Examples of questions and common study designs

To understand the differences in research design, it is helpful to place studies into one of two categories: quantitative research or qualitative research. Quantitative research is by far the more dominant type of research in the dental literature.

Quantitative Research

Quantitative research, as the name implies, generally looks at numbers of things (ie, quantifiable data), is based on measurements, and typically uses statistical analysis. This broad category of research may be further divided into observational studies and interventional studies.

Observational studies

In observational studies, the investigators gather information without intentionally controlling or manipulating the environment of the subjects. These may be in the form of surveys, or they could compare specific observed outcomes of interest among different groups of people from an historical perspective or prospectively. This chapter examines five examples of observational studies: cross-sectional studies, case-control studies, cohort studies, case reports, and case series. Three of these observational study designs—case reports, case series, and case-control studies—may be either quantitative or qualitative, meaning that the research is based on thoughts and opinions of participants.

Cross-sectional studies

A cross-sectional study attempts to gather information about a population, or a subset of a population, at one point in time. This basic design may be a population survey, a clinical study of selected patients, or a record review assessing characteristics of a population group. A diagram of a cross-sectional study is shown in Fig 2-1. A cross-sectional study would be appropriate when asking a question such as What are the incidence and prevalence of periodontal disease?

Fig 2-1 Example of a cross-sectional study design. Exposure and disease status data are gathered at one point in time.

There are several important issues with this type of study:

•  Careful selection of a representative sample from the population of interest is critical. If subjects are chosen based on the wrong criteria, the sample may not represent the population accurately.

•  Variable response rates to surveys can introduce bias. People who do not respond to a questionnaire may be different from responders and may be more or less likely to exhibit the trait in question.

Consider a study designed to answer the following question: What is the association between tooth erosion occurrence and the consumption of acidic foods and drinks among undergraduate university students? To answer this question, researchers recruited participants, completed dental examinations, and asked the participants questions about their diet and personal oral hygiene habits.¹ One of the strengths of this study is its direct approach: The investigators asked a group of students about their food-consumption habits and attempted to correlate these data to the condition of the students’ teeth. However, this approach has its weaknesses:

•  Recruitment bias may influence the results. The students who were selected to participate may not be representative of the population they represent.

•  Inaccuracies in reporting may distort the exposure data. The students may not have correctly recalled their history, they may have misrepresented their habits to project a positive image, or they may have misunderstood the questions. The degree to which the participants gave reliable responses determines the validity of the study.

•  The examiners may introduce bias. The examiners who completed the Basic Erosive Wear Examinations may have introduced other biases such as observer error or correlation of appearance to wear rather than nontraumatic morphologic variation.

This study illustrates the survey or cross-sectional study design. The success of using this design to answer the clinical question is determined by how well it controls problems inherent to the design, that is, how closely the selected participants reflect the population of interest and how well the authors consider the response rate in developing their conclusions.

Case-control studies

The next step up in strength of design is the case-control study. As the name implies, a case-control study includes both cases and matched controls to answer a clinical question without an intervention. This study design starts with a disease or other outcome in the case group and healthy individuals in the control group and then looks into the past to determine if exposure to something is associated with the disease in the individuals in the case group. A schematic diagram of a case-control study is shown in Fig 2-2. This design is useful (1) for elucidating potential associations, (2) when an answer is needed quickly, or (3) for rare conditions or diseases. This study type is unlikely to prove cause and effect but may suggest associations, which can be further investigated.

Case-control studies are retrospective in nature, meaning that they look back in time to establish a picture of events and behaviors possibly leading to the current health presentation. Because the study is retrospective, it often relies on subjects’ memories of past events, which may introduce recall bias into the study. Recall bias is the phenomenon whereby patients with a disease or condition are more likely to recall or remember events that they feel may have been (rightfully or wrongfully) associated with their condition.

Fig 2-2 Example of a simple case-control study design. They are retrospective and look back in time.

Even with all of its weaknesses, the case-control study is actually a powerful tool and may be used when stronger designs would be inappropriate. When a rare disease is being investigated, it is often difficult to associate past events with appearance of the disease. An important example of such a disease is intracranial meningioma, a cancer of the tissues surrounding the brain. It would be unethical and require a long period of time to study possible causes in a prospective experiment, but a case-control study may ethically and quickly show associations if they exist.

Cohort studies

If the goal is to eliminate or limit recall bias, there is another study design in which a group of people are selected and observed for a period of time: the cohort study. A diagram of a prospective cohort study is shown in Fig 2-3. Though not all cohort studies are prospective, they all include two or more groups of subjects who have similar baseline characteristics except for one variable of interest. Instead of starting with a disease or other outcome, such as in the case-control study, the cohort study starts with groups or cohorts with a particular behavior, condition, or environmental exposure and observes them over time to determine if the variable causes or is protective for a disease.

Fig 2-3 Example of a simple cohort study design. They are prospective and look forward in time.

However, the elimination of recall bias does not mean that all is well because people are free agents and can change behavior over time, complicating exposure parameters. In addition, an apparently causative factor may just be associated with a hidden factor that is actually the cause. For example, imagine a study that concluded that coffee drinking was a risk factor for cancer but the investigators failed to account for the fact that in the study population more coffee drinkers were also smokers compared with non–coffee drinkers. In this example, it would appear that coffee consumption was responsible for higher cancer rates when it was actually the confounder of associated cigarette smoking that was the more likely associated risk factor.²

Case reports

Case reports are observational studies that do not involve sampling or comparisons. These