The Doctor-Patient Relationship, Partnership Theory, and the Patient as Partner: Finding a Balance Between Domination and Partnership.

It is perhaps most useful to approach the Doctor-Patient relationship (DPR) by admitting that it's complicated. We review some of the strategies that have been employed to mitigate this complexity, zeroing in on one that promises to capture the main features of the DPR without eliminating some of its more important, existential components; pieces of the puzzle that must be retained if we are to avoid oversimplification and the errors that can arise by ignoring important foundational properties. We believe that a useful way to look at the DPR and to capture essential features that must be balanced in the process is provided by Partnership Theory and its definition in terms of the so-called domination and partnership systems. We apply this theory to the DPR and investigate the implications of this application to health care. We see that in the absence of mitigating circumstances, adoption of the patient-as-partner model serves healthcare well and is flexible enough to accommodate circumstances that dictate modifications.

The transition from inquiry to evidence to actionable clinical knowledge: A proposed roadmap.

RATIONALE, AIMS, AND OBJECTIVES: We consider the question "What should we do?" in the context of clinical research/practice. There are several steps along the way to providing a satisfactory answer, many of which have received considerable attention in the literature. We aim to provide a unified summary and explication of these "steps along the way". The result will be an increased appreciation for the meaning and structure of "actionable clinical knowledge". METHODS: We review the literature to identify pertinent works dealing with evidence production and translation into actionable clinical knowledge. We draw from insights in this literature about various aspects of reasoning relevant to clinical questions and integrate these into a unified approach to the processes that lead to actionable clinical knowledge. RESULTS: We collect, collate, and integrate some of the work by Bauer, Carper, Goldman, Haack, McHugh and Walker, and Upshur and colleagues and obtain guidelines to aid in the evidence-to-actionable-clinical- knowledge transition. CONCLUSIONS: Clinical decision-making is not infallible, and the steps we can take to minimize error are context dependent. Medical evidence, produced as it is by human effort, can never be perfect. We will be doing well by assuring that the evidence we use has been produced by a reliable process and is relevant to the question posed.

Scientism recognizes evidence only of the quantitative/general variety.

RATIONALE, AIMS AND OBJECTIVES: McHugh and Walker introduced a model of knowledge to demonstrate that EBM is a form of scientism that ignores important sources of knowledge thereby impairing the practice of medicine. We study the development of this model and explore additional applications. METHODS: Review of the relevant literature and identification of possible areas for fruitful application. RESULTS: We show that the McHugh and Walker model is closely related to the model of evidence considered earlier by Upshur et al. We also indicate that the utility of this model is not limited to showing scientism distorts clinical practice. Several representative applications are identified, including psychotherapy, the Salk polio vaccine trial, and the placebo effect. CONCLUSIONS: Priority should be given to Upshur et al for the development of a model that has far-reaching application to medical epistemology. It is shown that all four of the types of evidence considered-qualitative/personal, qualitative/general, quantitative/general, and quantitative/personal-are required to adequately characterize epistemology in medical research and practice.

Schemata, CONSORT, and the Salk Polio Vaccine Trial.

In this essay, we defend the design of the Salk polio vaccine trial and try to put some limits on the role schemata should play in designing clinical research studies. Our presentation is structured as a response to de Freitas and Pietrobon (de Freitas, R. S. and R. Pietrobon. 2007. Whoever could get rid of the context of discovery/context of justification dichotomy? A proposal based on recent developments in clinical research. Journal of Medicine and Philosophy 32:25-42.) who identified the CONSORT statement as a schema that would have, had it existed at the time, ruled out the design of the Salk polio vaccine trial of 1954 in favor of a completely randomized controlled clinical trial (RCT). We argue that large-scale public health interventions often require evidence beyond simple efficacy, the limit of what an RCT can provide, and that the design actually adopted for the Salk trial represented a reasonable-albeit imperfect-compromise. This is of more than historical interest in that many contemporary studies are of the scale and scope to require a more pragmatic, rather than explanatory, approach to study design (Kowalski, C. J. 2010. Pragmatic problems with clinical equipoise. Perspectives in Biology and Medicine 53:161-73.).

The Ethics of Clinical Care and the Ethics of Clinical Research: Yin and Yang.

The Belmont Report's distinction between research and the practice of accepted therapy has led various authors to suggest that these purportedly distinct activities should be governed by different ethical principles. We consider some of the ethical consequences of attempts to separate the two and conclude that separation fails along ontological, ethical, and epistemological dimensions. Clinical practice and clinical research, as with yin and yang, can be thought of as complementary forces interacting to form a dynamic system in which the whole exceeds the sum of its parts. Just as effective clinical practice cannot exist without clinical research, meaningful clinical research requires the context of clinical practice. We defend this thesis by triangulation, that is, by outlining how multiple investigators have reached this conclusion on the basis of varied theoretical and applied approaches. More confidence can be placed in a result if different methods/viewpoints have led to that result.

Beware Dichotomies.

This essay combines our thoughts concerning the generally destructive practice of dichotomization with a selective review of the literature supporting our critique. The apparent simplicity of dichotomous thinking encourages its use even when a dyadic representation is totally inadequate to understanding complex situations, and this "simpler is better" mantra continues to stymie our understanding of many of the world's complexities. The identification and naming of two distinct, opposing categories often results in their being seen as in opposition to one another, and that it is somehow incumbent upon us to choose one or the other. This either/or orientation reinforces the original split, confusing explanans and explanandum. We begin by considering dichotomization in general terms, and then turn to brief descriptions of several particular dichotomies. Some of these persist despite what might well be considered sufficient evidence to deny their usefulness, and this often deflects attention away from the more fertile, interesting, and important questions that may be directed to the points at which they intermingle.

Felicitometric hermeneutics: interpreting quality of life measurements.

The use of quality of life (QOL) outcomes in clinical trials is increasing as a number of practical, ethical, methodological, and regulatory reasons for their use have become apparent. It is important, then, that QOL measurements and differences between QOL scores be readily interpretable. We study interpretation in two contexts: when determining QOL and when basing decisions on QOL differences. We consider both clinical situations involving individual patients and research contexts, e.g., randomized clinical trials, involving groups of patients. We note the ethical importance of such understanding: proper interpretation and communication facilitate health care decision making. Communication that facilitates interpretation is of moral significance since better communication can attenuate ethical problems and inform choices. Much of what is communication worthy about QOL assessments is determined by the particular QOL instrument used in the assessment and how it is administered. In practice, these choices will be driven by the purpose of the assessment, but, it is argued, to maximize understanding, we should combine the information garnered from traditional standardized QOL instruments, from individualized QOL assessments, and from a recently proposed dialogic paradigm, where QOL is determined by shared conversation regarding the interpretation of texts. And, while some studies can surely succeed using abbreviated methods of administration (e.g., postal surveys may suffice for certain purposes), we will focus on methods of administration involving interviewer-respondent interaction. We suggest that during the QOL elicitation process, interviewer and respondent should engage in a two-way conversation in order to achieve a shared understanding of the "answers" to QOL "questions" and, finally, to reach a shared interpretation of the individual's QOL.

Pragmatic problems with clinical equipoise.

It is widely accepted that if one is to follow the ethical tenets of clinical equipoise, phase III controlled clinical trials must be designed pragmatically, to measure effectiveness rather than efficacy. This choice of a pragmatic rather than an explanatory approach to phase III clinical trial design has a number of consequences, some of which may be considered problematic. These include changes in what the trial is expected to accomplish, the way treatments are defined, the selection of subjects, the ways in which treatments are compared, and the assessment of the results. One also may end up challenging the real-world expectation that scientific results will be replicated before they are considered valid. This article discusses the connection between clinical equipoise and pragmatic trials, contrasts explanatory with pragmatic trials, points to the differences in the ways in which trial data are analyzed and interpreted, and discusses the power of replication, one of the defining hallmarks of the scientific method. Viewing clinical equipoise through a consequentialist lens reveals a number of problems, many of which are attributable to equipoise's insistence on a pragmatic approach to trial architecture.

Data and safety monitoring boards: some enduring questions.

Data Safety and Monitoring Boards (DSMBs) have been referred to as a "growth industry," and this trend continues to be fueled by recent FDA guidance and the NIH's requirement that DSMBs be employed in virtually all phase III clinical trials. The widening role of DSMBs has been sporadically questioned on ethical grounds, but growth has continued, despite the fact that many of the questions endure, unanswered, save for repeated references to safeguarding the scientific integrity of trials. This may be about to change. The recently appointed director of the Office for Human Research Protections (OHRP), Jerry Menikoff, is on record as regarding current practices--where consent forms often promise what the DSMB has been assembled to specifically not provide--as constituting fraudulent behavior. That is, a subject may inherently rely on, to their detriment, information that has been misrepresented in the consent document. In this paper, we assemble some of the enduring questions and top them off with Menikoff's tour de force to present what we hope will be a compelling argument to require that consent forms fairly represent what the DSMB will do--and not do--with trial data as they accumulate. We argue that DSMBs should be used only in rare circumstances, and question the practice of precluding principal investigators from DSMB membership, but our main thrust is to ensure that DSMBs, when used at all, are properly described in trial consent forms.

Human histological research: is it necessary? Humane? Ethical?

A review of the dental literature in the United States over the past 60 years is replete with studies utilizing human histological evidence for research. The first reference found for the use of human tissue for histological study was published in 1941 in the Journal of Dental Research.(1) Much of this literature focuses on the use of block sections, in which teeth scheduled for extraction are removed along with portions of the surrounding soft tissue and bone in order to study the effects of various interventions. The tissue removed in no way facilitates the surgical extraction of the tooth. It is removed to establish the type of healing, repair, or regeneration by histological evidence. There is no compensating benefit to the patient who, in fact, is put at risk--the removal of the extra tissue may compromise the fit of a subsequent prosthetic restoration.

Muscle pain inhibits cutaneous touch perception.

The processing of noxious and non-noxious sensations differs between chronic pain syndromes, and we believe that studies of sensory processing in the presence of pain will help to clarify the aetiology of the conditions. Here we measured in humans the threshold-level mechanosensitivity in tonic experimental muscle pain. We found (1) that muscle pain induced by hypertonic saline reduced cutaneous threshold-level mechanosensitivity at the site of pain and at the mirror site in the contralateral face, (2) that this effect outlasted the sensation of pain, (3) that it was more pronounced when the painful area was reported to be large, and (4) that the loss of mechanosensitivity was greater in males than females. Comparing our findings to results obtained with other pain models, all classes of nociceptors do not seem to have the same effect on cutaneous mechanosensitivity. The observed threshold-level hypoesthesia is consistent with the hypothesis that the increased mechanical thresholds found in clinic cases of temporomandibular disorders and cervicobrachialgia are a direct result of the activation of muscle nociceptors.

Computation of Foulkes and Davis' nonparametric tracking index using GAUSS.

Foulkes and Davis (1981) define tracking as the maintenance of relative rank over a given time span. This paper outlines the development of their statistic, based on a set of individual growth profiles, which estimates the degree of tracking observed in a one-sample longitudinal data set and shows how confidence intervals for the corresponding population parameter may be constructed. An example using a measure of skeletal growth is given and a GAUSS program to do the computations is provided. (Information on obtaining the GAUSS program is provided in the Appendix.) Properties of this statistical approach to tracking are contrasted with another non-parametric method based on Cohen's kappa statistic. © 1992 Wiley-Liss, Inc.

PC program for obtaining orthogonal polynomial regression coefficients for use in longitudinal data analysis.

Much of longitudinal data analysis begins with dimensionality reduction, i.e., the replacement of the T observations x1 , x2 , …, xT on an individual taken at times t1 , t2 , …, tT (not necessarily equally spaced) by a smaller number, P, of parameters which are then used to describe and compare growth processes. We focus on the class of polynomial growth curve models for one-sample data matrices in which the P regression coefficients are estimated by an equation of the form \documentclass{article}\pagestyle{empty}\begin{document}$ \hat \tau = ({\rm W'W}) $\end{document} 1 W'x and consider the choice of the design matrix W. The case in favor of using orthogonal polynomials to comprise the elements of W and provide a PC program, written in GAUSS, for obtaining them is presented. This program can be used instead of existing tables of orthogonal polynomials in the case of equally spaced time points, and to avoid laborious hand-computation to obtain them when the time points are not equally spaced. The program also computes the corresponding orthogonal polynomial regression coefficients \documentclass{article}\pagestyle{empty}\begin{document}$ \hat \alpha = (\Phi '\Phi)^{ - 1} \Phi '{\rm x} $\end{document}, where Φ consists of orthogonal polynomials, which may then be input into other programs for subsequent analysis, e.g., to compare the growth profiles of several groups of individuals. Examples of the use of the program are given. Information on obtaining a copy of the program is provided in Appendix A. © 1992 Wiley-Liss, Inc.

PC program for comparing tracking indices in several independent groups.

A method for computing a measure of tracking based on Cohen's kappa statistic for one-sample longitudinal data sets was previously described and implemented. This paper shows how one may test the equality of several kappas, each computed from an independent longitudinal sample. Thus, it is possible to formally compare groups of individuals with regard to stability in growth (or adaptive) patterns. Relative assessments of predictability in growth outcomes in different populations can be made with this approach. Also, when a common value of kappa is not contradicted by the data, a method to estimate this value and obtain a confidence interval for it is shown. A menu-driven GAUSS program for carrying out the procedure is described and made available. The method and program are illustrated with three samples of Guatemalan children. © 1992 Wiley-Liss, Inc.

PC program for analyzing one-sample longitudinal data sets which satisfy the two-stage polynomial growth curve model.

The two-stage polynomial growth curve model is described and a GAUSS program to perform the associated computations is documented and made available to interested readers. The two-stage model is similar to that considered by us earlier (Schneiderman and Kowalski: American Journal of Physical Anthropology 67:323-333, 1985; American Journal of Human Biology 1:31-42, 1989), i.e., it is appropriate for the analysis of one-sample longitudinal data collected at either equal or unequal time intervals. Here, however, the covariance matrix, Σ, instead of being considered arbitrary, is now assumed to have the special structure Σ = W A W' + σ2 I. We show the conditions under which this special structure may be expected to arise and how it may be exploited to produce sharper results in certain situations. The method and the program are illustrated and the results are contrasted to those obtained when Σ is arbitrary. It is suggested that the two-stage model is more efficient when the same degree polynomial is adequate to model the data in the two situations, but that, should a higher degree be necessary for the two-stage model, confidence intervals and/or bands may be wider than those corresponding to Σ arbitrary.

A GAUSS program for computing an index of tracking from longitudinal observations.

Tracking can be defined as the tendency of individuals or collections of individuals to stay within a particular course of growth over time relative to other individuals. Thus, tracking describes stability in growth patterns. This paper outlines a statistical procedure for examining tracking in a single sample of measurements made on humans or other animals. This nonparametric procedure, based on Cohen's (1960) kappa statistic, is suitable for equally or unequally spaced serial data that is complete and is appropriate for questions concerning growth as well as other time-dependent phenomena. It is a conceptually simple longitudinal method that affords insight regarding the predictability of growth within a population. For example, by tracking, one can ask if young children who are in the lowest height for age category are likely to end up in that category at an older age. A user-friendly GAUSS program is provided that generates overall as well as individual and track-specific statistics. High-resolution graphic representations of the data are also generated by the program. Examples are presented, including a tracking analysis of Guatemalan Indian children using quartiles.

Implementation of Hills' growth curve analysis for unequal-time intervals using GAUSS.

Longitudinal data are widely regarded as the most efficient and informative type of data with which to investigate growth. Paradoxically, appropriate statistical methods for analyzing longitudinal data have been unavailable; with the exception of a computer program for executing Rao's (Biometrika 46:49-58, 1959) one-sample polynomial growth curve analysis (Schneiderman and Kowalski, Am. J. Phys. Anthropol. 67:323-333, 1985) and another applying the Preece-Baines function (Brown and Townsend, Ann. Hum. Biol. 9:495-505, 1982), no programs for analyzing longitudinal data are generally available to the scientific community. Whereas much of the pediatrically oriented work has involved fitting growth curves for individual children, the concern here is the estimation of growth trends for populations. An Adequate understanding of average tendencies is a prerequisite to understanding the growth of individuals. The present paper implements Hills' (Biometrics 24:189-196, 1968) analysis, which is formally equivalent to Rao's but uses finite differences instead of orthogonal polynomials. This method is suitable for data collected at unequal time points and generates explicit measures of velocity and acceleration. The polynomial specification of the curve that best fits the data is also determined with this method. An additional advantage of this approach is that it is conceptually simpler than the classic model of Rao. An application of this method is given using the same craniofacial growth data as in our earlier (1985) paper for comparability. We provide an easy to use program written in GAU's (Edlefson and Jones, Kent, WA; Applied Technical Systems, 1985), a matrix programming language that runs on PC-compatible microcomputers. This implementation for PCs extends the accessibility to investigators who may not have access to mainframe computers.