Protocol for development of a core outcome set for clinical trials in melasma

INTRODUCTION: Melasma is a pigmentation disorder of the skin. Characterised by brown to gray-brown patches on the face and neck, the condition predominantly affects women and has been associated with pregnancy, hormonal variation and sun exposure. Melasma can be disfiguring and anxiety-provoking, and quality of life is often adversely impacted. Management includes sun protection, laser and energy device therapy, topical and oral skin-bleaching agents and chemical peels. While clinical trials of melasma exist, there is a lack of consistency in reported outcomes, which has been a barrier to the aggregation of data in systematic reviews and meta-analyses. This protocol describes a planned process for development of a minimum set of outcomes (ie, ‘core outcome set’) that should be measured in all clinical trials of melasma. METHODS AND ANALYSIS: An exhaustive list of potential outcomes will be extracted from four sources: (1) systematic literature review of outcomes in clinical trials; (2) semistructured patient interviews; (3) brochures, pamphlets, clinical trial registries, and other published and unpublished sources and documentation; and (4) interviews with non-patient, non-physician stakeholders, including federal regulators, industry scientists and non-physician providers. An international two-round Delphi process will then be performed to identify the outcomes deemed most important to patients and physicians. Subsequently, a consensus meeting will be convened to review and process the results, and to vote on a final set of core outcomes. ETHICS AND DISSEMINATION: Ethics approval was provided by the Northwestern University Institutional Review Board (protocol ID: STU00201637). This study is registered with both the Core Outcome Measures in Effectiveness Trials and Cochrane Skin-Core Outcome Set Initiative initiatives, and this protocol is in accordance with the guidelines for protocol development of both groups. All findings from the study described in this protocol will be disseminated to all stakeholders involved in the development process and will be submitted for publication in peer-reviewed journals. PROSPERO REGISTRATION NUMBER: CRD42020214189

Development of a core outcome set for clinical trials in squamous cell carcinoma: study protocol for a systematic review of the literature and identification of a core outcome set using a Delphi survey

Abstract Background Squamous cell carcinoma (SCC) is a common skin cancer that poses a risk of metastasis. Clinical investigations into SCC treatment are common, but the outcomes reported are highly variable, omitted, or clinically irrelevant. The outcome heterogeneity and reporting bias of these studies leave clinicians unable to accurately compare studies. Core outcome sets (COSs) are an agreed minimum set of outcomes recommended to be measured and reported in all clinical trials of a given condition or disease. Although COSs are under development for several dermatologic conditions, work has yet to be done to identify core outcomes specific for SCC. Methods/design Outcome extraction for COS generation will occur via four methods: (1) systematic literature review; (2) patient interviews; (3) other published sources; and (4) input from stakeholders in medicine, pharmacy, and other relevant industries. The list of outcomes will be revaluated by the Measuring PRiority Outcome Variables via Excellence in Dermatologic surgery (IMPROVED) Steering Committee. Delphi processes will be performed separately by expert clinicians and patients to condense the list of outcomes generated. A consensus meeting with relevant stakeholders will be conducted after the Delphi exercise to further select outcomes, taking into account participant scores. At the end of the meeting, members will vote and decide on a final recommended set of core outcomes. The Core Outcome Measures in Effectiveness Trials (COMET) organization and the Cochrane Skin Group - Core Outcome Set Initiative (CSG-COUSIN) will serve as advisers throughout the COS generation process. Discussion Comparison of clinical trials via systematic reviews and meta-analyses is facilitated when investigators study outcomes that are relevant and similar. The aim of this project is to develop a COS to guide use for future clinical trials

Development of a core outcome set for clinical trials in basal cell carcinoma: study protocol for a systematic review of the literature and identification of a core outcome set using a Delphi survey

Abstract Background Basal cell carcinoma is the most common skin cancer worldwide. Treatment options include both surgical and topical modalities. Although risk of metastasis is low, basal cell carcinoma can be invasive and infiltrate important underlying structures such as bone or cartilage. While many clinical trials examining therapies for basal cell carcinoma exist, the lack of consensus in outcome reporting across all trials poses a concern. Proper evaluation and comparison of treatment modalities is challenging. In order to address the inconsistencies present, this project aims to determine a core set of outcomes which should be evaluated in all clinical trials of basal cell carcinoma. Methods/design Outcomes will be extracted over four phases: (1) a systematic literature review, (2) patient interviews, (3) other published sources, and (4) stakeholder involvement. Potential outcomes will then be examined by the Steering Committee, who may add or remove outcomes. The Delphi process will then be performed to condense the list of outcomes generated. Two rounds of Delphi surveys will be performed with two groups of participants – physicians and patients. A consensus meeting with relevant stakeholders will be conducted after the Delphi exercise to further select outcomes, taking into account participant scores. By the end of the meeting, members will vote and decide on a final recommended set of core outcomes. For the duration of the study, we will be in collaboration with both the Core Outcome Measures in Effectiveness Trials (COMET) initiative and the Cochrane Skin Group – Core Outcome Set Initiative (CSG-COUSIN). Discussion This study aims to develop a core outcome set to guide assessment in clinical trials on basal cell carcinoma. The end-goal is to improve the consistency of outcome reporting and allow proper evaluation of treatment effectiveness

Development of a core outcome set for basal cell carcinoma

Background: There is variation in the outcomes reported in clinical studies of basal cell carcinoma. This can prevent effective meta-analyses from answering important clinical questions. Objective: To identify a recommended minimum set of core outcomes for basal cell carcinoma clinical trials. Methods: Patient and professional Delphi process to cull a long list, culminating in a consensus meeting. To be provisionally accepted, outcomes needed to be deemed important (score, 7-9, with 9 being the maximum) by 70% of each stakeholder group. Results: Two hundred thirty-five candidate outcomes identified via a systematic literature review and survey of key stakeholders were reduced to 74 that were rated by 100 health care professionals and patients in 2 Delphi rounds. Twenty-seven outcomes were provisionally accepted. The final core set of 5 agreed-upon outcomes after the consensus meeting included complete response; persistent or serious adverse events; recurrence-free survival; quality of life; and patient satisfaction, including cosmetic outcome. Limitations: English-speaking patients and professionals rated outcomes extracted from English language studies. Conclusion: A core outcome set for basal cell carcinoma has been developed. The use of relevant measures may improve the utility of clinical research and the quality of therapeutic guidance available to clinicians. Keywords: basal cell carcinoma; core; measure; outcome; set; skin cancer

Development of a core outcome set for cutaneous squamous cell carcinoma trials: identification of core domains and outcomes

International audienceBackground: The lack of uniformity in the outcomes reported in clinical studies of the treatment of cutaneous squamous cell carcinoma (cSCC) complicates efforts to compare treatment effectiveness across trials. Objectives: To develop a core outcome set (COS), a minimum set of agreed-upon outcomes to be measured in all clinical trials of a given disease or outcome, for the treatment of cSCC. Methods: One hundred and nine outcomes were identified via a systematic literature review and interviews with 28 stakeholders. After consolidation of this long list, 55 candidate outcomes were rated by 19 physician and 10 patient stakeholders, in two rounds of Delphi exercises. Outcomes scored ‘critically important’ (score of 7, 8 or 9) by ≥ 70% of patients and ≥ 70% of physicians were provisionally included. At the consensus meeting, after discussion and voting of 44 international experts and patients, the provisional list was reduced to a final core set, for which consensus was achieved among all meeting participants. Results: A core set of seven outcomes was finalized at the consensus meeting: (i) serious or persistent adverse events, (ii) patient-reported quality of life, (iii) complete response, (iv) partial response, (v) recurrence-free survival, (vi) progression-free survival and (vii) disease-specific survival. Conclusions: In order to increase the comparability of results across trials and to reduce selective reporting bias, cSCC researchers should consider reporting these core outcomes. Further work needs to be performed to identify the measures that should be reported for each of these outcomes

Development of a core outcome set for basal cell carcinoma

BACKGROUND: There is variation in the outcomes reported in clinical studies of basal cell carcinoma (BCC). This can prevent effective meta-analyses to answer important clinical questions. OBJECTIVE: To identify a recommended minimum set of core outcomes for BCC clinical trials. METHODS: Patient and professional Delphi process to cull a long-list, culminating in a consensus meeting. To be provisionally accepted, outcomes needed to be deemed 'important' (score: 7-9, of maximum of 9) by 70% of each stakeholder group. RESULTS: 235 candidate outcomes identified via a systematic literature review and survey of key stakeholders were reduced to 74 that were rated by 100 health care professionals and patients in two Delphi rounds. 27 outcomes were provisionally accepted. The final core set of 5 agreed-upon outcomes after the consensus meeting was: complete response; persistent or serious adverse events; recurrence-free survival; quality of life; and patient satisfaction, including with cosmetic outcome. LIMITATIONS: English-speaking patients and professionals rated outcomes extracted from English-language studies. CONCLUSIONS: A core outcome set (COS) for basal cell carcinoma has been developed. Use of relevant measures may improve the utility of clinical research and the quality of therapeutic guidance available to clinicians