Understanding the patient experience and treatment benefits in patients with non-small-cell lung cancer with brain metastasis.

Background: Despite the high prevalence of brain metastases (BM) secondary to non-small-cell lung cancer (NSCLC) (NSCLC/BM), patients' experiences (symptoms and impacts) are not fully understood. This study sought to understand the patient experience with NSCLC/BM and identify a patient-reported outcome (PRO) measure fit to capture the most important NSCLC/BM symptoms and impacts.

Methods: A targeted literature review was completed; the National Comprehensive Cancer Network (NCCN)/Functional Assessment of Cancer Therapy-Brain Symptom Index, 24-item version (NFBrSI-24) was identified as a relevant measure that assessed the core symptoms and impacts associated with NSCLC/BM.

Health-related quality of life in women with breast cancer: a review of measures.

Background: To identify and describe the breast cancer-specific health-related quality of life (HRQoL) instruments with evidence of validation in the breast cancer population for potential use in patients treated for breast cancer (excluding surgery).

Methods: We conducted a systematic literature review using PubMed, Embase, and PsycINFO databases to identify articles that contain psychometric properties of HRQoL instruments used in patients with breast cancer. Relevant literature from January 1, 2009, to August 19, 2019, was searched.

Intra-articular Hyaluronic Acid for Osteoarthritis of the Knee in the United States: A Systematic Review of Economic Evaluations.

Background: The economic impact of intra-articular hyaluronic acid (IAHA) for the treatment of knee pain associated with osteoarthritis (OA) has been evaluated in the United States, but not systematically summarized.

Objective: We reviewed the literature to determine the economic impact of IAHA for pain associated with knee OA in the United States.

Methods: A literature review was performed in PubMed (including MEDLINE and MEDLINE In-Process), Embase, the Cochrane Database of Systematic Reviews, and National Health Service Economic Evaluation Database and was limited to English language human studies published from January 2000 to October 2020.

The Role of Noninvasive Endpoints in Predicting Long-Term Outcomes in Pulmonary Arterial Hypertension.

Background: Until recently, many clinical trials in patients with pulmonary arterial hypertension (PAH) evaluated exercise capacity with 6-minute walk distance (6MWD) as the primary endpoint. Common secondary endpoints include PAH functional class (FC), which assesses symptoms, and either brain natriuretic peptide (BNP) or the inactive N-terminal cleavage product of its prohormone (NT-proBNP), which assesses cardiac function.

Objective: Examine the relationships among 6MWD, FC, and BNP/NT-proBNP measured at baseline or follow-up with long-term outcomes in PAH studies.

Evaluation of the Feasibility of a Web-Based Survey to Assess Patient-Reported Symptom Improvement and Treatment Satisfaction Among Patients with Psoriatic Arthritis Receiving Secukinumab.

Background And Objective: Patient perspectives regarding treatment experience and satisfaction may be useful for clinicians when making treatment strategies. This US-based study assessed the feasibility of evaluating real-world, patient-reported narratives regarding symptom improvement and treatment satisfaction among patients with psoriatic arthritis treated with secukinumab.

Methods: A cross-sectional, web-based survey collected data on demographics, disease characteristics, symptoms before and after secukinumab use, and treatment satisfaction with secukinumab.

A Pilot Study to Assess the Feasibility of a Web-Based Survey to Examine Patient-Reported Symptoms and Satisfaction in Patients with Ankylosing Spondylitis Receiving Secukinumab.

Purpose: This real-world study evaluated the feasibility of assessing patient-reported symptom improvement and treatment satisfaction using a web-based survey among patients with ankylosing spondylitis (AS) treated with secukinumab.

Methods: This cross-sectional, web-based survey collected data on demographics, symptoms, treatment history, and treatment satisfaction from US patients with AS who were receiving secukinumab at survey participation. Patients reported AS symptoms experienced before and after secukinumab initiation, time to symptom improvement, and satisfaction with secukinumab treatment.

Psychometric Evaluation of the Patient's Knee Implant Performance Questionnaire.

Objectives: To evaluate the psychometric properties of Patient's Knee Implant Performance Questionnaire (PKIP), a new patient-reported measure of knee implant functional performance assessing patient experiences before and after primary total knee arthroplasty (TKA).

Methods: The psychometric analysis sample (n = 761) was based on an ongoing, multisite, prospective, noncomparative, longitudinal study of patients undergoing TKA. The PKIP was completed at three study visits: presurgery (visit 1), less than 1 year (visit 3, days 1-303), and minimum 1 year (visit 4, days 304-668).

A Review of Patient-Reported Outcome Labeling in the United States (2011-2015).

Background: A review of new drug approvals (NDAs) by the Food and Drug Administration (FDA) for 2006 to 2010 showed that 24.1% of new drugs had patient-reported outcome (PRO) labeling.

Objectives: To review PRO labeling for NDAs for 2011 to 2015 and to compare key findings reported previously.

Psoriasis patients with psoriasis Area and Severity Index (PASI) 90 response achieve greater health-related quality-of-life improvements than those with PASI 75-89 response: results from two phase 3 studies of secukinumab.

Background: The emergence of new biological therapies showing high and sustained level of Psoriasis Area and Severity Index (PASI) 90 response has provided the possibility of both greater skin clearance and increased quality of life (QOL).

Objective: To evaluate the association of greater response in skin clearance with improvements in skin-related QOL up to 52 weeks.

Methods: Subjects achieving various levels of skin clearance (PASI 90-100 or PASI 75-89) and Dermatology Life Quality Index (DLQI) (0/1) response were compared using ERASURE and FIXTURE trial data.

Secukinumab sustains early patient-reported outcome benefits through 1 year: Results from 2 phase III randomized placebo-controlled clinical trials comparing secukinumab with etanercept.

Background: Psoriasis is a chronic condition with negative impact on patients' quality of life that most often requires lifelong effective and safe treatment.

Objective: This analysis focused on the effect of secukinumab treatment on patient-reported health-related quality of life as assessed by the Dermatology Life Quality Index (DLQI) in patients with moderate to severe psoriasis.

Methods: The proportion of subjects achieving DLQI score 0/1 response at week 24, time to DLQI score 0/1 response, and sustained DLQI score 0/1 response up to week 52 were compared between secukinumab and etanercept.

Secukinumab provides better relief from the impact of psoriasis on daily activities and personal relationships than etanercept: results of two phase 3 placebo-controlled randomized clinical trials in moderate-to-severe psoriasis.

Background: Psoriasis can greatly impact patients' lives, influencing what clothing they wear and impairing their sexual functioning.

Objective: To provide a detailed comparison of the impact of secukinumab vs. etanercept on enabling patients with psoriasis to have more normal lives with respect to daily activities (DA) (e.

Secukinumab improves patient-reported psoriasis symptoms of itching, pain, and scaling: results of two phase 3, randomized, placebo-controlled clinical trials.

Background: Secukinumab is a human interleukin-17A antagonist indicated for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy. The objective of this analysis was to measure the treatment response on psoriasis-related itching, pain, and scaling via the Psoriasis Symptom Diary (PSD)(©).

Methods: ERASURE (n = 738) and FIXTURE (n = 1306) were double-blind, multicenter phase 3 studies in adults randomized to secukinumab (300, 150 mg, n = 1144) or placebo (n = 574) (administered at Weeks 0, 1, 2, 3, and 4, followed by dosing every 4 weeks) or a biologic active control (FIXTURE only).

Psychometric validation of the Psoriasis Symptom Diary using Phase III study data from patients with chronic plaque psoriasis.

Objectives: This analysis aimed to confirm the reliability, validity, and responsiveness of the Psoriasis Symptom Diary (PSD) using data from two Phase III studies in patients with moderate to severe chronic plaque psoriasis.

Methods: Data from two randomized, double-blind, double-dummy, placebo-controlled, multicenter Phase III studies (n = 820) assessing the efficacy and safety of secukinumab were used. The PSD (24-h recall; 0-10 numeric rating scale) was electronically administered each evening.

Factors affecting the health-related quality of life of patients with cervical dystonia and impact of treatment with abobotulinumtoxinA (Dysport): results from a randomised, double-blind, placebo-controlled study.

Objective: To describe the health-related quality of life (HRQOL) burden of cervical dystonia (CD) and report on the HRQOL and patient perception of treatment benefits of abobotulinumtoxinA (Dysport).

Design: The safety and efficacy of a single injection of abobotulinumtoxinA for CD treatment were evaluated in a previously reported international, multicenter, double-blind, randomised trial. HRQOL measures were assessed in the trial and have not been previously reported.

Development of a scale to assess performance following primary total knee arthroplasty.

Background: Quantitative assessment of postsurgical knee motion provides sensitive measurements, but results are technical and may not be meaningful to patients. Although several knee-specific instruments exist, no patient-reported outcome (PRO) measure correlates function with improved stability, motion, satisfaction, and confidence.

Objective: To address both the above limitations by developing a PRO measure to assess the phenomenon of a "normal" knee after primary total knee arthroplasty (TKA).

Assessment of PRO label claims granted by the FDA as compared to the EMA (2006-2010).

Background: The US Food and Drug Administration (FDA) provides formal guidance for the use of patient-reported outcomes (PROs) in support of labeling claims, whereas the European Medicines Agency (EMA) offers insight in a reflection paper relating to health-related quality of life in lieu of formal guidance.

Objectives: PRO label claims granted for new molecular entities and biologic license applications from 2006 through 2010 were reviewed to evaluate consistencies and discrepancies in PRO label claims granted by the FDA and the EMA and to highlight trends in the acceptance of PRO claims across agencies.

Methods: Products approved by both the FDA and the EMA were identified.

Role of Patient-Reported Outcome Measures in the Assessment of Central Nervous System Agents.

This work aimed to provide an understanding of the current use and regulatory acceptability of patient-reported outcome (PRO) measures in labeling claims for central nervous system (CNS) agents. A subset of CNS agents was identified from all New Drug Approvals and Biologic License Applications for new drugs approved in the US from January 2006 to June 2012. Clinician-reported outcomes (ClinROs) (62%) and PROs (38%) were the most widely used primary outcome measures.

Potential of patient-reported outcomes as nonprimary endpoints in clinical trials.

Background: The purpose of this research was to fully explore the impact of endpoint type (primary vs. nonprimary) on decisions related to patient-reported outcome (PRO) labeling claims supported by PRO measures and to determine if nonprimary PRO endpoints are being fully optimized.This review examines the use of PROs as both primary and nonprimary endpoints in support of demonstration of treatment benefit of new molecular entities (NMEs) and biologic license applications (BLAs) in the United States in the years 2000 to 2012.

Evidence-based medicine, appropriate-use criteria, and sports medicine: how best to develop meaningful treatment guidelines.

We propose using appropriate-use criteria (AUC) as the methodology of choice for formulating and disseminating evidence-based medicine guidelines in sports medicine and arthroscopy. AUC provide a structured process for integrating findings from the scientific literature with clinical judgment to produce explicit criteria for determining the appropriateness of specific treatments. The use of AUC will enable surgeons to treat patients in a more consistent manner based on expert clinical consensus and evidence-based medicine.

Validation of the Knee Injury and Osteoarthritis Outcome Score subscales for patients with articular cartilage lesions of the knee.

Background: The Knee Injury and Osteoarthritis Outcome Score (KOOS) assesses acute and chronic knee injuries or early-onset osteoarthritis in young, active patients. The United States Food and Drug Administration guidelines recommend that patient-reported outcome instruments used to support clinical trial label claims should demonstrate content validity using patient input and have acceptable psychometric properties in the target population. To use the KOOS subscales in safety and efficacy trials assessing new treatments for patients with articular cartilage lesions, additional validation work, using input from patients with articular cartilage lesions, was necessary.

Development of a conceptual model of adherence to oral anticoagulants to reduce risk of stroke in patients with atrial fibrillation.

Background: Oral anticoagulant (OA) medication is the recommended therapy for reducing the risk of thromboembolic complications in patients with atrial fibrillation (AF), and warfarin is the medication most frequently used. However, nonadherence associated with OA medications may lead to considerable health risks. A conceptual model of OA medication adherence in patients with AF could clarify factors affecting adherence, thereby assisting in the development and structuring of adherence-promotion programs.

Reasons for rejection of patient-reported outcome label claims: a compilation based on a review of patient-reported outcome use among new molecular entities and biologic license applications, 2006-2010.

Objectives: Previous analyses of patient-reported outcome (PRO) label claims concentrated only on successful label claims. The goal of this research was to explore the reasons why PRO label claims were denied and to compile regulatory feedback regarding the use of PROs in clinical trials.

Methods: By using the Food and Drug Administration's Drug Approval Report Web page, all new molecular entities and biologic license applications approved between January 2006 and December 2010 were identified.

A review of patient-reported outcome labels in the United States: 2006 to 2010.

Objective: In 2004, Willke and colleagues reviewed the efficacy endpoints reported in the labels of new drugs approved in the United States from 1997 through 2002 to evaluate the use of patient-reported outcome (PRO) endpoints. Of the labels reviewed, 30% included PROs. Our study aimed to build on this work by describing the current state of PRO label claims granted for new molecular entities (and biologic license applications since February 2006 after the release of the US Food and Drug Administration (FDA) draft PRO guidance.

Clinical staff resource use with intravenous patient-controlled analgesia in acute postoperative pain management: results from a multicenter, prospective, observational study.

The purpose of this study was to evaluate hospital resource utilization associated with intravenous patient-controlled analgesia (IV-PCA), with a focus on nursing, pharmacy, and central supply/engineering time spent from a hospital perspective. Data were collected during a multicenter (29 sites), prospective observational study in the United States of subjects who underwent total knee replacement (TKR), total hip replacement (THR), or abdominal hysterectomy (AH) and were administered analgesia through IV PCA for the management of acute postoperative pain. Nursing staff recorded the IV PCA-related tasks they performed for a subject and the duration of time required to perform each task from initial IV PCA set-up to discontinuation.