Development and Content Validation of the Patient's Qualitative Assessment of Treatment - Real-World (PQAT-RW): An Instrument to Evaluate Benefits and Disadvantages of Treatments in Real-World Settings.

Purpose: Quantifying patient-perceived benefits and disadvantages of treatments in a real-world setting is increasingly important in healthcare decision-making. The Patient's Qualitative Assessment of Treatment (PQAT) assesses patient-perceived benefits and disadvantages of treatment, and associated trade-offs potentially influencing patients' willingness to continue treatment. It has then been modified to capture patients' perceived magnitude of benefits and disadvantages of treatment quantitatively, as well as qualitatively (PQATv2).

Establishing clinically meaningful within-individual improvement thresholds for eight patient-reported outcome measures in people with relapsing-remitting multiple sclerosis.

Background: As disease-modifying therapies do not reverse the course of multiple sclerosis (MS), assessment of therapeutic success involves documenting patient-reported outcomes (PROs) concerning health-related quality of life, disease and treatment-related symptoms, and the impact of symptoms on function. Interpreting PRO data involves going beyond statistical significance to calculate within-patient meaningful change scores. These thresholds are needed for each PRO in order to fully interpret the PRO data.

Qualitative Patient Interviews to Characterize the Human Burden of Advanced Basal Cell Carcinoma Following Hedgehog Pathway Inhibitor Treatment.

Introduction: Evidence of patients' experiences of living with advanced basal cell carcinoma (aBCC) are limited, particularly after hedgehog pathway inhibitor (HHI) treatment. We explored the burden of aBCC on symptoms and patients' everyday lives post HHI treatment.

Methods: In-depth, semi-structured, approximately 1-h qualitative interviews of US patients with aBCC and prior HHI treatment were conducted.

Considerations to address missing data when deriving clinical trial endpoints from digital health technologies.

Digital health technologies (DHTs) enable us to measure human physiology and behavior remotely, objectively and continuously. With the accelerated adoption of DHTs in clinical trials, there is an unmet need to identify statistical approaches to address missing data to ensure that the derived endpoints are valid, accurate, and reliable. It is not obvious how commonly used statistical methods to handle missing data in clinical trials can be directly applied to the complex data collected by DHTs.

Enabling patient-reported outcome measures in clinical trials, exemplified by cardiovascular trials.

Objectives: There has been limited success in achieving integration of patient-reported outcomes (PROs) in clinical trials. We describe how stakeholders envision a solution to this challenge.

Methods: Stakeholders from academia, industry, non-profits, insurers, clinicians, and the Food and Drug Administration convened at a Think Tank meeting funded by the Duke Clinical Research Institute to discuss the challenges of incorporating PROs into clinical trials and how to address those challenges.

Post Hoc Health-Related Quality of Life Analysis According to Response Among Patients with Prostate Cancer in the PROSELICA and FIRSTANA Studies.

Background: The phase III PROSELICA (NCT01308580) and FIRSTANA (NCT01308567) trials investigated taxane chemotherapy among men with postdocetaxel metastatic, castration-resistant prostate cancer (mCRPC) or chemotherapy-naïve mCRPC, respectively. We present a post hoc analysis of patient-reported health-related quality of life (HRQL) among patients with or without a clinical (pain, tumor, or prostate-specific antigen [PSA]) response.

Materials And Methods: PROSELICA and FIRSTANA HRQL and pain data were collected and analyzed using protocol-defined Functional Assessment of Cancer Therapy-Prostate (FACT-P) and McGill-Melzack (Present Pain Intensity scale) questionnaires.

Determining Minimum Wear Time for Mobile Sensor Technology.

Part 1 in the DIA Study Endpoint Community Working Group on Mobile Sensor Technology (MST) series addresses considerations that may be useful when determining the minimum wear time associated with mobile sensor use to ensure reliable estimation of the clinical endpoint under consideration. What constitutes a minimum valid data set is a dilemma facing those using MSTs in clinical studies. If this alignment does not occur, the integrity of the data collected and conclusions drawn from these data may be in incorrect.

Aflibercept Plus FOLFIRI for Second-line Treatment of Metastatic Colorectal Cancer: Observations from the Global Aflibercept Safety and Health-Related Quality-of-Life Program (ASQoP).

Background: The objectives of this study were to evaluate the safety profile of aflibercept and health-related quality of life (HRQL) in patients with metastatic colorectal cancer (mCRC) provided with aflibercept access before marketing authorization.

Patients And Methods: Patients received aflibercept followed by FOLFIRI (fluorouracil, leucovorin, irinotecan) on day 1 of a 2-week cycle until disease progression, unacceptable toxicity, death, or patient/investigator decision to discontinue. Treatment-emergent adverse events (TEAEs) were evaluated, and HRQL was assessed at baseline, cycle 3, and every other cycle using the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30, EORTC QLQ-CR29, and EuroQol 5-Dimensions 3-Levels questionnaires (NCT01571284).

Impact of alemtuzumab on health-related quality of life over 6 years in CARE-MS II trial extension patients with relapsing-remitting multiple sclerosis.

Background: In CARE-MS II (Comparison of Alemtuzumab and Rebif Efficacy in Multiple Sclerosis; NCT00548405), alemtuzumab (12 mg/day; baseline: 5 days; 12 months later: 3 days) significantly improved health-related quality of life (HRQL) outcomes versus subcutaneous interferon beta-1a (SC IFNB-1a) in relapsing-remitting multiple sclerosis (RRMS) patients over 2 years. Patients completing CARE-MS II could enter a 4-year extension study (NCT00930553).

Objective: The aim of this study is to assess 6-year HRQL outcomes in alemtuzumab-treated CARE-MS II patients, including those with highly active disease (HAD).

An observational, multicentre study of cabazitaxel in patients with metastatic castration-resistant prostate cancer previously treated with docetaxel (CAPRISTANA).

Objectives: To obtain routine clinical practice data on cabazitaxel usage patterns for patients with metastatic castration-resistant prostate cancer (mCRPC) and to describe physician-assessed cabazitaxel effectiveness, health-related quality of life (HRQoL) and safety.

Patients And Methods: CAPRISTANA was an international, observational cohort study examining cabazitaxel use for the treatment of patients with mCRPC. Effectiveness was assessed by overall survival (OS), progression-free survival (PFS), time to treatment failure (TTF) and disease control rate.