Surgical procedures and devices should be evaluated in the same way as medical therapy

This paper is a personal essay that starts and ends with the message that surgical procedures and devices should be evaluated in the same way as medical therapies, namely, by randomized clinical trials (RCTs). I discuss, with particular attention to surgical procedures and devices, the objections raised against RCTs in medical decision-making, a schema for utilizing the traditional phases of RCTs in the evaluation of surgical procedures and devices, the importance of RCTs to FDA approval, financial compensation, and health care costs, the impact of RCTs on clinical practice, the role of RCTs in academia, teaching, and research, and the surgeon's obligation to participate in a leadership role in RCTs. The belief is expressed that national funding of health care should mandate allocations for RCTs and that such expenditures, as well as the spending of health care dollars on the basis of the outcomes of such trials, will not only improve patient management in the shortest time but will eventually reduce health care costs. The RCT, by selecting effective and safe surgical procedures and devices, as well as diets and drugs, is the best means science has to assess the validity of patient management.     

Is the randomized controlled trial overvalued as a basis for clinical decision-making? A review with comments

The randomized controlled trial (RCT) may have considerable limitations in clinical research. Lacking the possibility of blinding impairs the internal validity of the trials. The external validity is often impaired, as results of RCTs obtained in an ideal situation, may be difficult to generalize to a clinical routine situation. Pragmatic randomized trials move from ideal situations towards routine situations, and by modifying the design it is possible to reduce selection bias due to patient and physician preferences. Quasi-experimental studies have varying degrees of problems with internal validity but are necessary contributions to our knowledge of the effect of treatment in clinical routine situations. Limitations of the usefulness of RCTs as well as pragmatic and quasi-experimental studies in clinical research make it necessary to recognise that different methods complement one another. Research in development of RCTs and new methods in clinical research should be encouraged.     

Randomized controlled trials in the Journal of Psychosomatic Research; 1956-1993: a prevalence study

RCTs are the gold standard for the evaluation of mental health care [WHO Scientific Group on Treatment of Psychiatric Disorders, 1991]. All RCTs in the Journal of Psychosomatic Research to date (October 1993) were identified. A profile of the geographical origin, content, interventions under investigation, and methodological quality (as measured by reporting of randomization) of every trial is described. The prevalence of trials from Europe in the Journal is increasing. The frequency of trials regarding the psychotherapies in this journal is also increasing whilst that of drug trials is constant. High quality reporting of randomization is rare. This pilot study generates many disturbing questions regarding the content and quality of RCTs within psychosomatic medicine that urgently need to be answered. Trials in this journal may well not be representative of all RCTs relevant to psychosomatic medicine. If all such trials are to be made accessible to those interested in evidence-based practice, however, much methodical searching must be undertaken, and this study can then be repeated and the questions answered with more authority.     

Are results of randomized controlled trials useful to psychotherapists?

Two clinicians provided opposite answers to the title question: Persons argued that information from randomized controlled trials (RCTs) is vital to clinicians, and Silberschatz argued that information from RCTs is irrelevant to clinicians. Persons argued that clinicians cannot provide top quality care to their patients without attending to findings of RCTs and that clinicians have an ethical responsibility to inform patients about, recommend, and provide treatments supported by RCTs before informing patients about, recommending, and providing treatments shown to be inferior in RCTs or not evaluated in RCTs. Silberschatz argued that RCTs do not and cannot answer questions that concern practicing clinicians. He advocates alternative research approaches (effectiveness studies, quasi-experimental methods, case-specific research) for studying psychotherapy. (PsycINFO Database Record (c) 2010 APA, all rights reserved)     

A review of methodologies for assessing drug effectiveness and a new proposal: randomized database studies

The need to evaluate the effects of health technologies in clinical practice is increasingly important. In this article, we review the advantages and limitations of naturalistic randomized clinical trials (RCTs) and database analyses, the two primary methods for evaluating treatment effectiveness. Also, we comment on a newer research strategy, cross-design synthesis, which proposes the complementary use of both experimental RCTs and observational database methodologies to avoid the main weaknesses of each: respectively, the lack of external and internal validity. Finally, we propose a new strategy--randomized database studies--capable of generating results with an acceptable balance between internal and external validity. This strategy consists of the simultaneous use of both experimental and observational tools in the assessment of drugs' effectiveness. Randomization is essential to minimize comparison bias, and one possibility for such studies is that randomization modules could be included in computer-based patient records. Although we identify some of the difficulties in implementing the process, the progressive standardization of clinical practice and the development and widespread adoption of improved computer-based patient records could facilitate the use of randomized database studies as a new method of research.     

The content and methodology of research papers published in three United Kingdom primary care journals

BACKGROUND: With the expansion of academic departments, the National Research and Development initiative, and the Culyer report, United Kingdom (UK) general practice research is undergoing a period of investment and change. AIM: To examine the content and methodological quality of UK-published general practice research, and in particular to focus on the quantity and proportion of studies that were of high methodological quality, namely randomized controlled trials (RCTs). METHOD: We manually searched three UK-published journals over a five-year period: the British Journal of General Practice (BJGP), Family Practice, and the British Medical Journal (BMJ), which has a section devoted to general practice research. Studies were classified according to the International Classification of Health Problems of Primary Health Care (ICHPPC-2). RESULTS: Nearly half of published studies in UK primary care journals were concerned with either organization and administration issues in primary care or social problems (509 studies, 48%). Just over half were either qualitative studies or surveys of opinion or attitudes (528 studies, 50%). The overall number of RCTs was low (67 studies, 6%), and the proportion published has not changed over time (chi 2 for trend = 3.79, df = 1, P = 0.051). In contrast to surgical journals, nearly one-fifth of studies in general practice followed a longitudinal design (186 studies, 18%). CONCLUSIONS: The content and design of published general practice research in the UK is varied and broad. The most robust methodological design should be the aim of all prospective researchers in general practice.     

The effectiveness of surgery for stress incontinence in women: a systematic review

OBJECTIVE: To determine the methodological quality of studies evaluating surgery for stress incontinence, the effectiveness of different procedures and the frequency of complications associated with each procedure. MATERIALS AND METHODS: Eleven randomized controlled trials, 20 non-randomized trials/prospective cohort studies and 45 retrospective cohort studies were reviewed systematically. RESULTS: The methodological quality of the 31 prospective studies was generally poor. The considerable variation in inclusion criteria, surgical management and assessment of outcome precluded any statistical meta-analysis. Evidence as to the effectiveness of surgery for stress incontinence is weak; therefore, any conclusions are speculative. It appears that colposuspension may be more effective and the effect more long-lasting than that following anterior colporrhaphy and needle suspension. There is little information on the value of sling procedures. Comparisons of different ways of performing each procedure show no significant differences in outcome but this may reflect the methodological weaknesses of the studies. Valid and reliable data on the frequency of complications following surgery are lacking so the safety of the procedures is unclear. Repeat operations to correct stress incontinence are less successful than first procedures but this finding may be subject to confounding. CONCLUSIONS: There is an urgent need for some large, rigorous, prospective studies of high quality. Until such studies have been completed, recommendations as to the best clinical practice cannot be based on scientific evidence. Studies need to define cases according to widely accepted criteria, including standard measures of the severity of stress incontinence, and surgical terminology for the procedures performed needs to be standardized and outcomes need to be clearly defined, valid and reliable, not confined to short-term assessment and include patients' views along with the surgeon's assessments.     

Final reflections: wellness after obesity surgery

The articles presented in this symposium on treatment of clinically severe obesity cover most of the topics related to obesity surgery, from basic research about genetic causes of obesity to surgical techniques and clinical results on the severely obese patient. The most striking feature of those results observed after bariatric surgery for the clinically severe obese is not the remarkable weight loss and normalization of the body mass index but the amelioration of some of the metabolic and physiologic abnormalities related to obesity. In fact, carbohydrate and lipid metabolism show improvement, as do cardiac and pulmonary function. These observations justify all efforts devoted to the design and improvement of sound bariatric surgical techniques. Moreover, surgeons seek the well-being of the ex-obese patient, and in doing so they face special discriminatory attitudes from colleges, patients' families, and the society, mainly because of negative stereotypes related to the obese individuals and the lack of information on the real benefits of bariatric procedures. Bariatric surgeons will reach their goal only when an informed society, educated physicians, and properly oriented obese patients together give to bariatric surgery its real dimension. The objective of any surgeon is the wellness of his or her patient. This is important in bariatric surgery as well; beyond excess weight loss and normalization of biochemical parameters, the ex-obese patient should be able to live a normal, productive, happy life. Approaching this goal is a task that takes months or even years. Bariatric surgeons are committed to support and help their patients until they reach wellness after obesity surgery.     

Conceptional and structural conditions for successful clinical research

The efficiency of surgical research has again become the subject of debate. Clinical research is required to improve our understanding of surgical disorders and our ability to treat patients. This involves both experimental research (research in the test tube) and clinical research in actual patients. The surgeon must remain the expert because it is he who deals with the patient and is confronted with his problems. On the other hand, care for the patient, must always be the central issue. Here a new orientation is needed, evaluating the effectiveness of surgical research from the patient's point of view. Surgical treatment, particularly surgical research, must be adapted first to the individual patient and only secondly to the surgical disease - the problem must determine the method, not vice versa. While it is clear that a creative atmosphere, supportive structure and efficient organisation are enormously helpful, today's exaggerated attention to matters of structure and organisation are often poor substitutes for creativity and intuition. Surgical research does not refer solely to therapy research but includes methods for carrying out controlled clinical trials, establishing guidelines and scores and designing instruments for measuring outcome. Socioeconomic and analyses and ethical considerations are crucial for facing such conflicts as "quality versus quantity", "profession versus business", "patient care versus economics costs". Proposals for designing more effective concepts, structure and organisation for clinical research are presented here, and three models are introduced: the cooperation model, the integration model and a mixture of the two.     

Risk stratification in cardiac surgery: is it worthwhile?

Outcome analysis of many surgical procedures has become increasingly important to surgeons, institutions, and the public. Because there may be wide differences in case mix, outcomes must be evaluated in light of the patient's preoperative status. All relevant preoperative conditions must be identified and weighted, so that when risk factor scores are combined in some fashion, they will provide a single preoperative risk estimate for the individual patient, representing the likelihood of dying as a consequence of the operation. Comparing the mean risk adjusted score of a group of patients undergoing the same procedure with the observed mortality rate for the same group yields an index of the quality of care, provided all preoperative risk scores are calculated with reference to the same benchmark. We question the logic and wisdom of surgical outcome analysis because of the infinitely complex nature of biological and pathological processes, as well as the practical problems of reliable data collection. The assumption of true scientific accuracy may be illusory. Even though risk adjusted outcome analysis has merit in studying trends in therapy, it should be regarded with caution when used as a tool for evaluating quality of care. If publicized at all, the results should not be represented as "hard" scientific fact.     

Surgical treatment of obstructive sleep apnea syndrome. An evidence-based agreement

OBJECTIVES: To determine the current efficacy of type of surgery in the treatment of obstructive sleep apnoea. SEARCH STRATEGY: Electronic searches of the Cochrane Airways Group Sleep Apnoea RCT Register, MEDLINE, EMBASE and Cinahl. The references of relevant review articles were handsearched. Experts in the field were contacted to identify unpublished studies. SELECTION CRITERIA: The inclusion criteria were all randomised or quasi-randomised controlled comparisons of any surgical intervention for obstructive sleep apnoea versus other surgical or non-surgical intervention for obstructive sleep apnoea or no intervention. DATA COLLECTION AND ANALYSIS: No completed RCTs were identified by the search. MAIN RESULTS: A total of 666 articles were identified and assessed. No articles fulfilled the inclusion criteria. CONCLUSIONS: A thorough search with broad inclusion criteria yielded no evidence from randomised controlled trials to support the use of surgery in the treatment of obstructive sleep apnoea. Patients should be made aware of the lack of good trial-based evidence for the operations. An urgent need for high quality RCTs in this area was identified.     

A basic introduction to medical research. Part II: An overview of different research methods

Though health research has long been equated with the randomised control trial (RCT), there are, in fact, many different forms of research. These complement one another as different pieces in a jigsaw of evidence. Qualitative research can provide valuable insights into meaning, motivation, social significance and other aspects of patients' and practitioners' experience. Such data can be useful in the design of more quantitative research, such as surveys of attitudes or RCTs. Audits monitor and evaluate the quality of health care delivery and are an important means of ensuring the value of RCTs in everyday clinical practice. Laboratory research provides data on the mechanisms of a treatment or on its therapeutic effect in controlled conditions. This can provide a valuable background to subsequent clinical research. A prospective researcher should choose a methodology depending on the particular research question he or she wishes to ask: a good question immediately suggests the appropriate type of research.     

Controversies and realities of therapy for varicosis

Proponents of surgical eradication of varicose veins claim a high cure rate. Proponents of the conservative methods of multiple injections of sclerosing agents claim that this money-saving ambulatory method avoids hospitalization and loss of working time, that the end results are quite satisfactory and that any recurrences can be easily dealt with by reinjections. Both methods are effective. Both have their indications. Many surgeons use sclerotherapy after surgery as complementary therapy. Radical views recommending surgery or injections exclusively are contrary to the principles of good medical care and do injustice to the patient. A simple venographic technic demonstrating the status of the greater saphenous veins is described.     

How tumor stage affects surgeons' surveillance strategies after colon cancer surgery

BACKGROUND: The factors that influence decision making among surgeons are not well understood. This study sought to evaluate how the tumor stage of patients subjected to potentially curative surgery for colon cancer affects the follow-up strategies used by practicing surgeons. METHODS: Hypothetical patient profiles and a detailed questionnaire based on these profiles were mailed to 2733 members of two major surgical societies. The effect of TNM Stage on the surveillance strategies chosen by the respondents was analyzed. RESULTS: Seven of the nine most commonly used surveillance modalities all were performed significantly more frequently with increasing TNM Stage. This effect persisted through 5 years of follow-up. The other two modalities (computed tomography and bone scan) were performed too infrequently for meaningful analysis. CONCLUSIONS: Surgeons performing surveillance after potentially curative surgery for otherwise healthy patients with colon cancer modify their strategies according to the patient's TNM Stage. These data should help in the design of prospective trials related to this topic.     

Conservation surgery for breast cancer: selection criteria and technical considerations

Patients with stage I or II breast cancer are candidates for either modified radical mastectomy or breast preservation therapy involving limited resection of the primary tumor, axillary dissection, and breast irradiation. The overall survival rates of both these approaches are comparable according to retrospective reviews and ongoing clinical trials, and long-term follow-up confirms the earlier findings. Thus, patients should be given the choice between these two options by surgeons, radiation therapists, and other physicians involved in their care. However, not all breast cancer patients will choose breast preservation surgery, and because of tumor-related and other factors not all patients are candidates. The patient selection criteria are discussed herein and the optimal surgical techniques are reviewed.     

Surgical management of locally recurrent rectal cancer

The surgical management of locally recurrent rectal cancer may involve major procedures and is not for the faint-hearted. Nevertheless, such treatment is preferable to chemotherapy and radiotherapy; the latter will fail over a period of months during which the patient is likely to experience intractable pain. Radical surgery offers good palliation and a better quality of life. Survival is prolonged by such operations which may be curative in up to one-third of patients. Nevertheless, surgeons must be realistic in their assessment of and discussions with patients.     

Neuropsychological outcomes in randomized controlled trials of antiepileptic drugs: a systematic review of methodology and reporting standards

PURPOSE: To systematically review the methodology and use of neuropsychological tests in randomized controlled trials (RCTs) of antiepileptic drugs (AEDs) in patients with epilepsy. METHODS: Trial reports were found by searching Medline 1966-1996 and searching through journals by hand. Inclusion and exclusion criteria were applied, and methodological and neuropsychological test data was extracted by using a proforma. RESULTS: 43 reports met our inclusion criteria, representing 40 RCTs. as three RCTs had generated two reports. Twenty-two were actively controlled, and 18 were placebo-controlled studies. Reporting of basic methods such as randomization method was poor. There has been no uniform approach to the use of neuropsychological tests, and a total of 87 has been used. The Stroop Colour Word Test and the Finger Tapping Test were most commonly used, at 13 times each, but were not used or reported in a uniform manner. CONCLUSIONS: Poor reporting of methods and the use of a plethora of neuropsychological tests create great difficulties for anyone wishing to make sense of currently available data. If we are better to understand the neuropsychological effects of AEDs, a more rational approach is needed, for which recommendations are made.     

Effectiveness of exposure and ritual prevention for obsessive-compulsive disorder: Randomized compared with nonrandomized samples.

The efficacy of exposure and ritual prevention (EX/RP) for reducing symptoms of obsessive-compulsive disorder (OCD) has been demonstrated in several randomized controlled trials (RCTs). However, procedures used in these studies to maximize experimental control may have limited their generalizability to typical clinical practice. Treatment outcome data from 110 clinical patients receiving EX/RP on an outpatient fee-for-service basis were compared with findings from 4 RCTs of EX/RP. Adult patients in the clinical sample were not excluded because of treatment history, concomitant pharmacotherapy, psychiatric comorbidity, age, or OCD severity. Clinical patients achieved substantial and clinically meaningful reductions in their OCD and depressive symptoms following EX/RP, which were comparable with those reported in the RCTs. Findings indicate that EX/RP is a potent treatment for OCD, and its benefits are not limited to select patient samples. (PsycINFO Database Record (c) 2010 APA, all rights reserved)     

Definitive or exploratory periodontal experimentation: an overview of the literature

Definitive and exploratory randomized controlled trials (RCTs) have different goals as well as different design and analysis characteristics. The goal of definitive studies is to provide unequivocal evidence of a treatment's tangible benefit to the patient; a pre-trial-specified hypothesis is tested by use of a pre-trial-specified method. The goal of exploratory studies is to elucidate biological treatment mechanisms, to identify promising treatments, and to generate hypotheses for definitive studies: multiple hypotheses are evaluated to extract as much information from the data as possible. The purposes of this study were: (1) to survey selected design and analysis characteristics of randomized controlled periodontal trials published between 1988 and 1992 (n = 86), and (2) to classify trials as exploratory or definitive studies. The periodontal RCTs surveyed were typical of exploratory studies whose primary goal was to elucidate biological treatment mechanisms. Trial reports indicated the testing of multiple hypotheses (> or = 6 hypothesis tests in 70 of the 86 trials) on a variety of biological markers (86 out of 86 trials). The sample size (< or = 30 subjects in 67 out of 86 trials), duration (< or = 6 months in 65 out of 86 trials), and design and analysis characteristics (e.g., an absence of masking in 57 out of 86 trials) were also typical of exploratory studies which strive to obtain quick answers (short duration) at a low cost (small sample size; accept bias for increased efficiency and a lower cost). No definitive trials were identified. Promising, biologically active, treatments identified in exploratory trials should be evaluated in definitive studies where the primary goal is the procurement of unequivocal evidence of a treatment's tangible benefit to the patient. The costs and benefits of conducting definitive periodontal RCTs to provide such evidence should be investigated.     

Occurrence of arrhythmias in general practice

The recent inclusion of research criteria for premenstrual dysphoric disorder (PMDD) in the DSM-IV should help physicians recognize women with symptoms of irritability, tension, dysphoria, and lability of mood which seriously interfere with their lifestyle. PMDD can be differentiated from premenstrual syndrome (PMS) which is primarily reserved for milder physical symptoms and minor mood changes. The use of DSM-IV criteria in conjunction with prospective daily charting for at least two cycles is now accepted as common practice in confirming the diagnosis. Treatment options range from conservative lifestyle and stress management to treatment with psychotropic medications and hormonal or surgical interventions to eliminate ovulation for the more extreme cases. Results from several randomized placebo-controlled trials have clearly demonstrated that selective serotonin reuptake inhibitors as well as medical or surgical oophorectomy are very effective in treating PMDD. Taken together, these data indicate that treatment may be accomplished by either eliminating the hormonal trigger or by reversing the sensitivity of the serotonergic system.