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      RCTs and other clinical trial designs in Ayurveda: A review of challenges and opportunities

      review-article
      a , b
      Journal of Ayurveda and Integrative Medicine
      Elsevier
      Clinical trials, Ayurveda, RCTs, Non-hierarchical

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          Abstract

          Currently, there is a paucity of clinical trial designs that comprehensively evaluate the efficacy of most complementary and alternative systems of medicine (CAMs) like Ayurveda. Several factors such as complex interventions, individualized therapy, etc., make designing Ayurveda clinical trials challenging. The prevalent randomized control trial (RCT) designs largely involve symptomatology/pathology-based recruitment and standardized interventions in carefully monitored trial environments. The present paper critically reviews the suitability of the dominant RCT model to Ayurveda and argues for newer, more sensitive trial models including modified RCTs and other clinical trial designs. It also explores the merits of a non-hierarchical approach to clinical evidence generation.

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          Most cited references20

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          Understanding and misunderstanding randomized controlled trials

          Randomized Controlled Trials (RCTs) are increasingly popular in the social sciences, not only in medicine. We argue that the lay public, and sometimes researchers, put too much trust in RCTs over other methods of investigation. Contrary to frequent claims in the applied literature, randomization does not equalize everything other than the treatment in the treatment and control groups, it does not automatically deliver a precise estimate of the average treatment effect (ATE), and it does not relieve us of the need to think about (observed or unobserved) covariates. Finding out whether an estimate was generated by chance is more difficult than commonly believed. At best, an RCT yields an unbiased estimate, but this property is of limited practical value. Even then, estimates apply only to the sample selected for the trial, often no more than a convenience sample, and justification is required to extend the results to other groups, including any population to which the trial sample belongs, or to any individual, including an individual in the trial. Demanding ‘external validity’ is unhelpful because it expects too much of an RCT while undervaluing its potential contribution. RCTs do indeed require minimal assumptions and can operate with little prior knowledge. This is an advantage when persuading distrustful audiences, but it is a disadvantage for cumulative scientific progress, where prior knowledge should be built upon, not discarded. RCTs can play a role in building scientific knowledge and useful predictions but they can only do so as part of a cumulative program, combining with other methods, including conceptual and theoretical development, to discover not ‘what works’, but ‘why things work’.
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            The stepped wedge cluster randomised trial: rationale, design, analysis, and reporting.

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              Circular instead of hierarchical: methodological principles for the evaluation of complex interventions

              Background The reasoning behind evaluating medical interventions is that a hierarchy of methods exists which successively produce improved and therefore more rigorous evidence based medicine upon which to make clinical decisions. At the foundation of this hierarchy are case studies, retrospective and prospective case series, followed by cohort studies with historical and concomitant non-randomized controls. Open-label randomized controlled studies (RCTs), and finally blinded, placebo-controlled RCTs, which offer most internal validity are considered the most reliable evidence. Rigorous RCTs remove bias. Evidence from RCTs forms the basis of meta-analyses and systematic reviews. This hierarchy, founded on a pharmacological model of therapy, is generalized to other interventions which may be complex and non-pharmacological (healing, acupuncture and surgery). Discussion The hierarchical model is valid for limited questions of efficacy, for instance for regulatory purposes and newly devised products and pharmacological preparations. It is inadequate for the evaluation of complex interventions such as physiotherapy, surgery and complementary and alternative medicine (CAM). This has to do with the essential tension between internal validity (rigor and the removal of bias) and external validity (generalizability). Summary Instead of an Evidence Hierarchy, we propose a Circular Model. This would imply a multiplicity of methods, using different designs, counterbalancing their individual strengths and weaknesses to arrive at pragmatic but equally rigorous evidence which would provide significant assistance in clinical and health systems innovation. Such evidence would better inform national health care technology assessment agencies and promote evidence based health reform.
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                Author and article information

                Contributors
                Journal
                J Ayurveda Integr Med
                J Ayurveda Integr Med
                Journal of Ayurveda and Integrative Medicine
                Elsevier
                0975-9476
                0976-2809
                03 August 2021
                Jul-Sep 2021
                03 August 2021
                : 12
                : 3
                : 556-561
                Affiliations
                [a ]Consciousness Studies Programme, National Institute of Advanced Studies, Indian Institute of Science Campus, Bengaluru 560012, India
                [b ]Manipal Academy of Higher Education, Manipal, Karnataka 576104, India
                Article
                S0975-9476(21)00120-0
                10.1016/j.jaim.2021.06.012
                8377177
                34362605
                7b6db576-da29-4efb-826e-b188e09ab2f8
                © 2021 The Author

                This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

                History
                : 28 January 2021
                : 16 June 2021
                : 16 June 2021
                Categories
                Review Article

                Complementary & Alternative medicine
                clinical trials,ayurveda,rcts,non-hierarchical
                Complementary & Alternative medicine
                clinical trials, ayurveda, rcts, non-hierarchical

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