Choosing primary endpoints for clinical trials of health care interventions

Farran

Journal of Clinical Epidemiology

et al. 2020

Background and Objective: There is a pressing need for evidence-based interventions to address the devastating clinical and public health effects of the coronavirus disease 2019 (COVID-19) pandemic. The number of registered trials related to COVID-19 is increasing by the day. The objective of this study was to describe the characteristics of the currently registered interventional clinical trials related to COVID-19. Methods: We searched the World Health Organization's International Clinical Trials Registry Platform on May 15th, 2020. We included any entry that is related to COVID-19. We abstracted and then descriptively analyzed the following characteristics of the registered trials: study design, status, phase, primary endpoints, experimental interventions, and geographic location among other qualifiers. Results: We identified 1,308 eligible registered trials. Most trials were registered with ClinicalTrials.gov (n 5 703; 53.7%) and the Chinese Clinical Trial Registry (n 5 291; 22.2%). The number of participants to be enrolled across these trials was 734,657, with a median of 110 participants per trial. The most commonly studied intervention category was pharmacologic (n 5 763; 58.3%), with antiparasitic medications being the most common subcategory. Although over half of the trials were already recruiting, we identified published peerreviewed results for only 8 of those trials. Conclusion: There is a relatively large number of registered trials but with very few results published so far. Although our findings suggest an appropriate initial response by the research community, the real challenge will be to get these trials completed, published, and translated into practice and policy.

Section: Interpretations Of Findingsmentioning

confidence: 97%

A large number of COVID-19 interventional clinical trials were registered soon after the pandemic onset: a descriptive analysis

Farran

Journal of Clinical Epidemiology

et al. 2020

“…The way of how patients perceive the benefit derived from an intervention could be captured by the use of PROM. A common issue of co-primary endpoints appears when the two co-endpoints respond differently to the treatment [37,38]. However, combining an objective primary endpoint such as physical performance with a subjective well-being endpoint such as quality of life reduces the risk for this scenario.…”

Section: Primary Endpointmentioning

confidence: 99%

Update on the ESCEO recommendation for the conduct of clinical trials for drugs aiming at the treatment of sarcopenia in older adults

et al. 2020

Background In 2016, an expert working group was convened under the auspices of the European Society for Clinical and Economic Aspects of Osteoporosis and Osteoarthritis (ESCEO) and formulated consensus recommendations for the conduct of clinical trials for drugs to prevent or treat sarcopenia. Aims The objective of the current paper is to provide a 2020 update of the previous recommendations in accordance with the evidence that has become available since our original recommendations. Methods This paper is based on literature reviews performed by members of the ESCEO working group and followed up with face to face meetings organized for the whole group to make amendments and discuss further recommendations. Results The randomized placebo-controlled double-blind parallel-arm drug clinical trials should be the design of choice for both phase II and III trials. Treatment and follow-up should run at least 6 months for phase II and 12 months for phase III trials. Overall physical activity, nutrition, co-prescriptions and comorbidity should be recorded. Participants in these trials should be at least 70-years-old and present with a combination of low muscle strength and low physical performance. Severely malnourished individuals, as well as bedridden patients, patients with extremely limited mobility or individuals with physical limitations clearly attributable to the direct effect of a specific disease, should be excluded. Multiple outcomes are proposed for phase II trials, including, as example, physical performance, muscle strength and mass, muscle metabolism and muscle-bone interaction. For phase III trials, we recommend a co-primary endpoint of a measure of functional performance and a Patient Reported Outcome Measure. Conclusion The working group has formulated consensus recommendations on specific aspects of trial design, and in doing so hopes to contribute to an improvement of the methodological robustness and comparability of clinical trials. Standardization of designs and outcomes would advance the field by allowing better comparison across studies, including performing individual patient-data meta-analyses, and different pro-myogenic therapies.

Journal of Foot and Ankle Research

“…In an attempt to encourage a value based health care approach in foot orthosis practice, professionals may encounter considerable challenges when selecting valuable endpoints or measures. To assist users of the VALUATOR model, the following three-domain classification system has been selected for its strong link with evidence based practice: (1) clinical measures, (2) non-clinical measures, and (3) surrogate measures [ 59 ].…”

Section: The Value Based Foot Orthosis Practice (Valuator) Modelmentioning

confidence: 99%

“…Clinically meaningful measures/endpoints represent outcome measures about how a person feels, functions or survives. These may be measured objectively or subjectively, and are either (i) based upon judgements or interpretations of clinical signs by the professional, (ii) reported by patients (so-called patient reported outcome measures, PROMS) or (iii) observer-reported, such as a parents’ feedback about daily activity level of a child [ 59 ].…”

Section: The Value Based Foot Orthosis Practice (Valuator) Modelmentioning

confidence: 99%

The biopsychosocial‐digital continuum of foot orthosis practice and research: the VALUATOR model

Deschamps

Nester

Newton

et al. 2021

Foot orthoses have been used for decades despite uncertainty surrunding their therapeutic efficacy. Orthoses have been used exclusively to affect neuro-biomechanical input and outcome variables, however, there is emerging evidence that therapeutic efficacy may be affected by a psychological stimulus. Critical appraisal of the literature highlights that there is no holistic model upon which foot orthosis practice is taught, practised nor investigated. This paper introduces a conceptual model of foot orthosis practice (Value Based Foot Orthosis Practice (VALUATOR) model) that embraces a broader range of factors that are pertinent to orthosis practice, incorporating contemporary health service behaviours and values into orthosis practice for the first time.Within the VALUATOR model, foot orthosis design and clinical value is considered along a bio-psycho-social-digital continuum that reflects the reality of foot orthosis practice. The model contextualises the variable outcomes that are observed in research and practice within 6 key areas: 1) value, 2) person-centered approach, 3) zone of optimal bio-psycho-social stress, 4) bio-psycho-social assessment, 5) monitoring, 6) primary and secondary clinical strategies.The VALUATOR model is targeted at students, lecturers, scientists and practitioners and includes carefully chosen terminology to support a robust basis for educational and scientific discussion. It is believed that it provides a contemporary viewpoint and a structured conceptual metaphor that builds on existing evidence from a wide range of sources, invites constructive intellectual debate, and is anchored in the experiences of practitioners too. Stress testing the VALUATOR model will help determine its model and support further developments and evolution of orthotic practice in a evidence based way.