Leonila F. Dans

Evidence for underuse of effective medical services around the world

Underuse-the failure to use effective and affordable medical interventions-is common and responsible for substantial suffering, disability, and loss of life worldwide. Underuse occurs at every point along the treatment continuum, from populations lacking access to health care to inadequate supply of medical resources and labour, slow or partial uptake of innovations, and patients not accessing or declining them. The extent of underuse for different interventions varies by country, and is documented in countries of high, middle, and low-income, and across different types of health-care systems, payment models, and health services. Most research into underuse has focused on measuring solutions to the problem, with considerably less attention paid to its global prevalence or its consequences for patients and populations. Although focused effort and resources can overcome specific underuse problems, comparatively little is spent on work to better understand and overcome the barriers to improved uptake of effective interventions, and methods to make them affordable.

Trade-off between benefit and harm is crucial in health screening recommendations. Part I: General principles

Health screening is defined as the use of a test or a series of tests to detect unrecognized health risks or preclinical disease in apparently healthy populations to permit prevention and timely intervention. A health screening strategy consists of the sequence of a screening test, confirmatory test(s), and finally, treatment(s) for the condition detected. The potential benefits of health screening are easy to understand, but the huge potential for physical and psychological harm is less well recognized. Thus, health screening should only be recommended when five criteria are satisfied: (1) the burden of illness should be high, (2) the tests for screening and confirmation should be accurate, (3) early treatment (or prevention) must be more effective than late treatment, (4) the test(s) and treatment(s) must be safe, and (5) the cost of the screening strategy must be commensurate with potential benefit. Direct evidence from screening trials is subject to less bias. In some instances, indirect evidence may be acceptable, e.g., when the condition screened for is a risk factor for a disease rather than the disease itself.

Standard 1: Consent and Recruitment

A 4-year-old boy with a serious metabolic disorder is eligible for a trial of a new enzyme replacement, the first potential option to treat this disease. His parents have little understanding of the disease or trial, even with careful explanation, but eventually they consent to entry in the trial. The treating clinician doubts whether their consent is valid. This and similar dilemmas face pediatricians in research every day. Many of the therapeutic options for children have not been tested with the rigor applied to similar treatments in adults. This highlights the need for research to improve the evidence base of childrens medicine, for more pediatricians to undertake research, and for more children and families to participate.1 … Address correspondence to Martin Offringa, MD, PhD, Senior Scientist and Program Head, Child Health Evaluative Sciences, Research Institute, The Hospital for Sick Children, 555 University Ave, Toronto, Ontario, Canada M5G 1X8. E-mail: martin.offringa{at}sickkids.ca

Development of a questionnaire for identification of the risk factors for osteoarthritis of the knees in developing countries. A pilot study in Iran and Bangladesh. An ILAR-COPCORD phase III study.

Background:  Knee osteoarthritis (OA) is one of the most prevalent rheumatic disorders in the Asia‐Pacific region. Identification of modifiable risk factors is important for development of strategies for primary and secondary prevention of knee OA.

Appraising a tool for guideline appraisal (the AGREE II instrument)

The AGREE II instrument has 3 goals e to assess the quality of clinical practice guidelines (CPGs), to provide a methodologic strategy for the development of guidelines, and to recommend how and what information should be reported in guidelines. Of these, the main purpose seems to be to assess guideline quality (i.e., ‘‘confidence that the potential biases of guideline development have been addressed adequately and that the recommendations are both internally and externally valid, and are feasible for practice’’) [1]. There is no doubt that this instrument is badly needed by health care providers and policy-makers around the world, who deal with a deluge of clinical practice guidelines in their day-to-day decisions. AGREE II assesses CPGs based on 6 domains: 1) scope and purpose, 2) stakeholder involvement, 3) rigor of development, 4) clarity and presentation, 5) applicability, and 6) editorial independence. Each domain is scored using several items. There are a total of 23 items in all to be scored on a 7-point Likert scale by at least 2 (preferably 4) independent observers. This is not a run of the mill appraisal guide, as users may take an average of 90 minutes to complete an evaluation. To learn more about the instrument, we decided to use it to evaluate Philippine Guidelines on Periodic Health Examinations [2]. Because we were personally involved in drafting these guidelines, the process took only 53 minutes. Below, we reflect on our experience in using AGREE II:

Clinical presentations and outcomes of Filipino juvenile systemic lupus erythematosus

ObjectiveJuvenile Systemic Lupus Erythematosus (SLE) varies by location and ethnicity. This study describes the clinical, laboratory profile and outcome of juvenile SLE seen at Philippine General Hospital (PGH) from 2004-2008.MethodMedical charts of all Filipino Juvenile SLE cases admitted at PGH during the 5-year period were reviewed collecting demographic profile, clinical and laboratory manifestations and treatment during disease course.ResultsSeventy-eight cases of juvenile SLE were reviewed. There were 7 boys and 71 girls. The mean age at diagnosis was 14 years (SD 2.7) with a range of 8-18 years. Fever (52.5%) and malar rash (41.0%) were the most common features at disease onset. At the time of diagnosis, the most common features were malar rash (65.3%), renal involvement (62.8%) and photosensitivity (55.1%). Mucocutaneous (92.3%), renal (71.7%) and hematologic (69.2%) involvement were the most common features during the entire course of illness. Infection (34.5%) and neurologic (19.0%) complications were observed most frequently. Corticocosteroid treatment was given in most of the patients in the form of prednisone (97.4%) and concomitant methylprednisolone intravenous pulses (29.4%). Nine patients died during the study period. The overall 5-year mortality rate was 11.5%. Infection (77.0%) was the most frequent cause of death.ConclusionMalar rash was a common feature at disease onset and at diagnosis among Filipinos with juvenile SLE. Throughout the disease course, renal involvement occurs in 71.7% of patients. Infection was the leading cause of complication and death. The clinical presentations of Filipinos with juvenile SLE were similar to juvenile SLE in other countries.

Trade-off between benefit and harm is crucial in health screening recommendations. Part II: Evidence summaries

Evidence on the effectiveness of health screening strategies may be direct (i.e., studies on screening vs. no screening) or indirect (i.e., studies that separately evaluate the screening test[s], the confirmatory test, or the treatment). Critical trade-offs in the balance between harm and benefit for many screening strategies mandate that advocates of health screening adhere to the ethical precepts of nonmaleficence, autonomy, confidentiality, and equity. In our first article, we pointed out five prerequisites to justifying a health screening program: (1) the burden of illness should be high, (2) the screening and confirmatory tests should be accurate, (3) early treatment (or prevention) must be more effective than late treatment, (4) the tests and the treatment(s) must be safe, and (5) the cost of the screening strategy must be commensurate with the potential benefit. As can be gleaned from these criteria, recommendations on screening must be tailored to specific populations. Recommendations in one country, no matter how authoritative, cannot be generalized to apply to all other countries. Although accuracy, effectiveness, and safety data may be global (criteria 2-4), burden of illness and efficiency (criteria 1 and 5) will always vary from country to country. Rather than review various national guidelines, in this last article of our two-part series, we present evidence summaries to illustrate health screening. Our examples were selected to address special issues related to four situations-screening for cancer, risk factors for disease, genetic disorders, and infectious diseases.

GRADE equity guidelines 3: considering health equity in GRADE guideline development: rating the certainty of synthesized evidence

Objectives The aim of this paper is to describe a conceptual framework for how to consider health equity in the Grading Recommendations Assessment and Development Evidence (GRADE) guideline development process. Study Design and Setting Consensus-based guidance developed by the GRADE working group members and other methodologists. Results We developed consensus-based guidance to help address health equity when rating the certainty of synthesized evidence (i.e., quality of evidence). When health inequity is determined to be a concern by stakeholders, we propose five methods for explicitly assessing health equity: (1) include health equity as an outcome; (2) consider patient-important outcomes relevant to health equity; (3) assess differences in the relative effect size of the treatment; (4) assess differences in baseline risk and the differing impacts on absolute effects; and (5) assess indirectness of evidence to disadvantaged populations and/or settings. Conclusion The most important priority for research on health inequity and guidelines is to identify and document examples where health equity has been considered explicitly in guidelines. Although there is a weak scientific evidence base for assessing health equity, this should not discourage the explicit consideration of how guidelines and recommendations affect the most vulnerable members of society.

The prevalence of rheumatic diseases in a Filipino urban population: A WHO-ILAR copcord study

OBJECTIVE To determine the point prevalence of musculoskeletal complaints and rheumatic diseases in a Filipino urban community. METHODS A descriptive cross-sectional 2 phase survey was conducted in an urban community in Metropolitan manila. Phase I (screening) used face-to-face interviews, while phase II (examination) involved case identification of the rheumatic diseases. We sampled 670 households (3065 adults) using a multistage cluster sampling method. A pilot study was conducted to pretest the questionnaire for cross-cultural adaptation and validation, field procedures, sampling design, and data management plan. Standardized translated COPCORD questionnaires (blind translation and blind back-translation) were administered by trained interviewers. Two weeks after Phase I, Phase II was conducted at local health centers. The COPCORD questionnaire screened the number of cases with musculoskeletal complaints. Identification of cases with rheumatic disease was based on American College of Rheumatology (ACR) criteria. RESULTS Respondents completed 3006 questionnaires (phase I response rate 98%). Of these 489 respondents had musculoskeletal complaints. Functional disability was reported in 25% among these respondents. We examined 353 (phase II response rate 72%), revealing 294 with rheumatic conditions. In 26 persons there were no abnormalities, while 32 had nonrheumatic conditions at examination. The most common rheumatic diseases were osteoarthritis (OA) (n = 124) and soft tissue rheumatism (n = 115). CONCLUSION The prevalence of musculoskeletal complaints was 16.3% (95% CI 8.6-24.0) of the adult population in a FIlipino urban community. The total prevalence of rheumatic disease is 9.8% (95% CI 8.2-11.4). The prevalence of OA was 4.1% (95% CI 3.3-4.9) and soft tissue rheumatism 3.8% (95% CI 2.9-4.8). The prevalence of rheumatoid arthritis, 0.17% (95% CI 0-9.36), was notably low compared to the prevalence in other developing countries.