A Prospective Observational Study on Targeted Therapy for Unresectable or Metastatic BRAFV600E Mutant Melanoma Patients

Plain-language summary

1. Background The purpose of this study is to describe the profile of patients with BRAF-mutated melanoma treated with BRAF/MEK inhibitors combination and using the Tavie Skin application. TavieSkin app, a digital solution developped by Pierre Fabre, is dedicated to all BRAF-mutant unresectable or metastatic melanoma patients who are treated with "any" targeted therapies. 2. Study objectives The primary objective of the survey is to describe the demographics and clinical characteristics of patients with unresectable or metastatic BRAF-mutated melanoma treated with targeted therapy (BRAFi/MEKi) and using the TavieSkin application The secondary objectives include: * To assess the use of TavieSkin app in patients with unresectable or metastatic BRAF-mutated melanoma treated with BRAFi/MEKi combination; * To assess the treatment adherence of patients using TavieSkin app including treatment interruption or permanent discontinuation; * To assess the health-related quality of life of patients using TavieSkin app (FACT-M); * To assess work productivity and activity impairment over the treatment duration * To assess the patient satisfaction toward the TavieSkin application; * To assess the patient satisfaction toward the treatment. 3. Research methods 3.1 Study design This prospective, longitudinal, survey will be conducted in Europe to characterize BRAF-mutant unresectable or metastatic melanoma patients using TavieSkin app designed for accompanying patients treated with targeted therapies. To date, there are three combinations of BRAFi/MEKi available in routine practice for the treatment of BRAF-mutant unresectable or metastatic melanoma. The survey does not provide or recommend any treatment or procedure; all decisions regarding treatment are made at the sole discretion of the treating physicians in accordance with their usual practices. The patients initiating any BRAFi/MEKi combination will be invited to use the TavieSkin app by their healthcare provider (HCP) (i.e. oncologist, dermatologist, nurse…). Once the patient has installed and started to use the application, an e-survey will be proposed to the patient via the app. A detailed information letter about the data collection, data privacy and analysis will be displayed to the patient via the app along with an e-consent for data collection. The patient will be able then to provide an e-signature, if he/she accepts to take part of this survey. The survey will collect anonymized data about health status, QoL data and satisfaction. These data will be collected by the patient only. The physician will not be involved in this e-survey (including e-consent), nor in data collection. Only patients having given consent (e-consent) to data collection and analysis will be included. Data will be collected at baseline and at different subsequent timepoints during the BRAFi/MEKi treatment duration only. Only data reported by the patients in the application will be collected and analyzed. The patient will discontinue the study in case of definitive withdrawal of BRAFi/MEKi treatment, or if he/she decides to withdraw the study and to stop data collection. The target countries for patient enrollment will include Germany, Belgium, Portugal, France, Spain, Italy and Sweden with the additional possibility of including patients from other EU countries. At least, 400 adult patients (≥18 years) will be enrolled. 3.2 Population (see section: Eligibility) 3.3 Study outcomes (see section: Outcome measures) 3.4 Statistical considerations Statistical analyses will be fully described in a written statistical analysis plan (SAP). The study endpoints will be analysed overall and by country. Analyses will be descriptive in nature, as no hypothesis will be tested. The treatment patterns of patients, baseline demographics and clinical characteristics, and reasons for treatment discontinuation will be described using summary statistics. Categorical variables will be summarized by frequencies and percentages. Continuous variables will be summarized by descriptive statistics (mean, and standard deviation, median, 25th and 75th percentiles, minimum and maximum). The number of missing observations for each variable will also be reported. Change in health-related quality-of-life scores (i.e. (FACT-M) will be summarised at baseline and at each timepoints. The change from baseline will be assessed using a mixed model for repeated measures (MMRM). Time to event data (i.e. time to treatment discontinuation, time QoL deterioration) will be evaluated using Kaplan-Meier survival curves. Median survival estimates will be reported along with the 25th and 75th percentiles and corresponding 95% confidence intervals (CIs). Cox regression analysis may be performed to adjust for predefined (baseline) covariates. If the sample size is adequate, subgroup analyses using variables at baseline might be conducted.

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