Editorial, J Pharm Sci Emerg Drugs Vol: 9 Issue: 12
Analyzing the improvement of patients in clinical trials
Doctors must comprehend the benefits of new medicines from the perspective of patients in order to assess the relative risks and benefits of providing them to patients. To satisfy this demand, the discipline of health status assessment has grown, and various patient-completed instruments that are valid, reliable, and sensitive to therapy have been established. Such tools can give patients critical insight into how a medication affects outcomes like function and quality of life, which are often more important to patients than survival. However, for some illnesses, disease-specific health status measurements are not accessible, thus global assessments of clinical change are utilized instead. Although proven procedures for measuring global change reported by patients are available, simple ad hoc assessments are frequently utilised and can be examined from the patient's or doctor's perspective. Evangelou and colleagues investigate whether patients and doctors judge global change following experimental therapies differently in an accompanying survey of trials included in systematic reviews. Introduction Given the reliance of studies on global assessments of clinical change and the convenience of the doctor doing the assessment, it is important to understand the correlation between patients�?? assessments and doctors�?? assessments. The studies included in Evangelou and colleagues�?? review were mostly from the fields of rheumatology and psychiatry. The authors found a close correlation between the two assessments, which suggests that either should suffice when measuring the benefits of treatment from the patient�??s perspective. However, although they performed a thorough literature review, and used the data in a methodologically rigorous fashion, their findings may provide false reassurance. In the field of cardiology, doctors�?? assessments have been shown to be biased and irreproducible. The New York Heart Association (NYHA) classification of functional status in heart failure is meant to measure patients symptoms and functional limitations on a four point scale. But inter observer variability is high�??agreement between independent assessors is only 55%.8 9 Similarly, clinical investigators�?? and objective interviewers assessments of the severity of angina have been shown to be biased.10 If reproducible assessments cannot be determined at a single point in time, it is unlikely that doctors will be able accurately to represent changes over time. Yet, as Evangelic and colleagues note, such formal investigation of doctors�?? ability to measure change has rarely been performed. Clinical trials should study patients' perspectives on their disease by the serial assessment of valid, reliable, and responsive disease specific health status questionnaires, rather than relying on such crude assessments. 11 The Kansas City cardiomyopathy questionnaire, for example, is more sensitive to clinical change than the NYHA, sixminute walk test, and other generic tests. 12 Furthermore, such assessments can reveal which areas (such as symptoms, physical function, or social function) are most changed by treatment. Patients can have a more realistic estimate of the benefits of a suggested treatment with this information. When disease-specific metrics are unavailable, generic health-status measures should be considered. Clinicians can only get an accurate assessment of how a treatment impacts a patient's illness process and successfully communicate that information to the patient if they employ psychometrically sound metrics. Materials and Methods The difference between Evangelou and colleagues' findings and the well-documented limits of doctors' estimates of patients' health state could be explained by a number of factors. To begin with, the authors had to rely on group means because they used summary data from reviews rather than raw data from clinical observations, and they may not have grasped the individual diversity of each doctor's evaluation. Second, the clinical symptoms of the disorders analysed may have a stronger relationship with a patient's quality of life than other conditions like cardiovascular disease. Finally, the various clinical change metrics utilised in the research included in the reviews may obscure major differences in clinical change perspectives that would have been more apparent with better assessment tools. Interestingly, failing to account for study length and the possibility of recollection bias about patients' initial health state is more likely to have introduced biases that result in bigger discrepancies between patients' and doctors' ratings. Finally, because patients are frequently at the lowest point in their disease when they enrol in a clinical study, a background tendency toward improvement may have unduly emphasised the similarities between the patients' and doctors' judgments. Alternative techniques of monitoring patients' clinical condition in other diseases and clinical situations should be investigated by researchers. Serial changes in patients' health state, as assessed by patients themselves, should be used to document the benefits of treatment more frequently. This would eliminate the need for global assessments from either the patient's or the doctor's perspectives. Given the importance of understanding the association between patients' assessments and doctors' assessments, given the reliance of studies on global assessments of clinical progress and the convenience of the doctor performing the assessment. The majority of the research reviewed by Evangelou and colleagues came from the domains of rheumatology and psychiatry. The scientists discovered a strong correlation between the two assessments, implying that either one should sufficient when assessing treatment benefits from a patient's perspective. However, their findings may provide false reassurance, despite the fact that they conducted a thorough literature analysis and utilised the data in a methodologically rigorous manner.
Abstract
Doctors must comprehend the benefits of new medicines from the perspective of patients in order to assess the relative risks and benefits of providing them to patients. To satisfy this demand, the discipline of health status assessment has grown, and various patient-completed instruments that are valid, reliable, and sensitive to therapy have been established. Such tools can give patients critical insight into how a medication affects outcomes like function and quality of life, which are often more important to patients than survival. However, for some illnesses, disease-specific health status measurements are not accessible, thus global assessments of clinical change are utilized instead. Although proven procedures for measuring global change reported by patients are available, simple ad hoc assessments are frequently utilised and can be examined from the patient's or doctor's perspective. Evangelou and colleagues investigate whether patients and doctors judge global change following experimental therapies differently in an accompanying survey of trials included in systematic reviews.
