Caregivers' Perspectives on the Impact of Cannabidiol (CBD) Treatment for Dravet and Lennox-Gastaut Syndromes: A Multinational Qualitative Study.

Objective: To increase understanding of the impact of cannabidiol (CBD) on outcomes beyond seizure control among individuals with Dravet syndrome or Lennox-Gastaut syndrome.

Methods: Qualitative interviews were conducted with caregivers of individuals with Dravet syndrome or Lennox-Gastaut syndrome treated with plant-derived, highly purified CBD medicine (Epidiolex in the USA; Epidyolex in Europe; 100 mg/mL oral solution). Symptoms and impacts of Dravet syndrome and Lennox-Gastaut syndrome on individuals were explored, as were the effects of CBD.

Elicitation of Health State Utilities Associated with Progression from Bacillus Calmette-Guerin (BCG) Unresponsive Non-muscle Invasive Bladder Cancer (NMIBC).

Objectives: The treatments for high-grade non-muscle invasive bladder cancer (NMIBC) vary between bladder preserving intravesical approaches and radical cystectomy. The impact of these treatments on health-related quality of life may vary widely. The purpose of this study was to elicit the general public's perspective on quality of life, measured as utility scores associated with treatment for Bacillus Calmette-Guerin (BCG)-unresponsive NMIBC and disease progression, for supporting economic evaluation of newly developed treatments for NMIBC.

Patient Experiences, Satisfaction, and Expectations with Current Systemic Lupus Erythematosus Treatment: Results of the SLE-UPDATE Survey.

Objectives: To provide information on systemic lupus erythematosus (SLE) patients' experiences, satisfaction, and expectations with treatments and examine the association between treatment satisfaction and patient-reported outcomes (PRO).

Methods: A cross-sectional, non-interventional, online survey of US adult patients with SLE was conducted in 2019. The survey consisted of 104 questions about SLE and the following PRO instruments: LupusPRO™, Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue, Work Productivity and Activity Impairment (WPAI), an 11-point Worst Pain Numerical Rating scale (NRS), and an 11-point Worst Joint Pain NRS.

A health state utility valuation study to assess the impact of treatment mode of administration in Gaucher disease.

Background: This study aimed to obtain UK societal-based utility values for health states related to treatment mode of administration using Gaucher disease as the background condition.

Methods: A review of relevant literature and expert clinical input informed the development of five health states characterising the impact of Gaucher disease and its management on patients' lives. A base-state characterising the "controlled disease" was developed as well as four subsequent health states which varied in description of the method (intravenous versus oral) and frequency of treatment administration.

A utility valuation study assessing the impact of postprandial glucose control on quality of life of individuals with type 1 or type 2 diabetes.

Background: Consideration of health-related quality of life (HRQOL) in diabetes has been associated with long-term and short-term complications such as hypoglycaemia, but not with short-term glucose control. This study aimed to collect health utilities related to different degrees of poorly controlled postprandial glucose (PPG) and its impact on HRQOL in the UK and in Sweden.

Methods: Three health state descriptions were developed based on literature reviews and interviews with people with diabetes and healthcare professionals, characterising mild, moderate and severe impact of postprandial hyperglycaemic symptoms on HRQOL.

Development of a conceptual model to illustrate the impact of multiple myeloma and its treatment on health-related quality of life.

Purpose: Little qualitative research exploring the impact of multiple myeloma (MM) and its treatment on the health-related quality of life (HRQL) of patients has been published. This study aimed to explore the burden of MM symptoms and treatment and the impact of these on HRQL. A model was developed to illustrate key concepts and their interrelationships.

Health utilities in relation to treatment response and adverse events in relapsed/refractory Hodgkin lymphoma and systemic anaplastic large cell lymphoma.

New therapies for relapsing/refractory Hodgkin lymphoma (R/R HL) and R/R systemic anaplastic large-cell lymphoma (sALCL) have emerged. This study captured utility values for R/R HL and sALCL to support economic evaluation. Health state "vignettes" were developed describing states associated with R/R HL and sALCL: treatment response (complete or partial response), stable and progressive disease and treatment-related adverse events (B-symptoms, acute/chronic graft-versus-host disease [GVHD] and peripheral neuropathy).

Development of a disease-specific version of the EQ-5D-5L for use in patients suffering from psoriasis: lessons learned from a feasibility study in the UK.

Objectives: The EuroQol five-dimensional (EQ-5D) questionnaire is a generic measure widely used for the assessment of health status. Research has suggested that it may be insensitive to the burdens associated with particular conditions. This study was designed to explore the feasibility of developing and valuing a disease-specific "bolt-on" version of the EQ-5D questionnaire for use in psoriasis.

Patient-reported fatigue and its impact on patients with systemic lupus erythematosus.

Fatigue is a hallmark symptom of systemic lupus erythematosus (SLE), often associated with flares, side effects of treatment, and extensive organ damage and may have a significant impact on health-related quality of life (HrQoL). To date, the experience of fatigue in patients with SLE is underexplored. This study explored the experience of fatigue in patients with SLE and its impact on their lives through qualitative interviews.

A discrete choice experiment to determine patient preferences for injection devices in multiple sclerosis.

Objective: Relapsing-remitting multiple sclerosis (MS) is usually managed with disease modifying drugs (DMDs), most commonly administered via self-injection. The aim of this study was to estimate the influence that different treatment-related attributes have for MS patients on their choice of MS DMD device. By establishing the relative importance of these characteristics for patients it should be possible to better understand the acceptability of a given device and to optimize the development of future devices.

Measurement properties of the EQ-5D-5L compared to the EQ-5D-3L across eight patient groups: a multi-country study.

Purpose: The aim of this study was to assess the measurement properties of the 5-level classification system of the EQ-5D (5L), in comparison with the 3-level EQ-5D (3L).

Methods: Participants (n = 3,919) from six countries, including eight patient groups with chronic conditions (cardiovascular disease, respiratory disease, depression, diabetes, liver disease, personality disorders, arthritis, and stroke) and a student cohort, completed the 3L and 5L and, for most participants, also dimension-specific rating scales. The 3L and 5L were compared in terms of feasibility (missing values), redistribution properties, ceiling, discriminatory power, convergent validity, and known-groups validity.

Elicitation of health state utilities in soft tissue sarcoma.

Purpose: Soft tissue sarcomas (STS) are uncommon tumours with varying histological subtypes. There is a paucity of available data concerning the quality-of-life (QoL) impact of STS which could be used to support economic evaluation of future treatments. This study aimed to elicit societal utility values for health states that depict the impact of STS and its treatment.

Development of a conceptual model of health-related quality of life for systemic lupus erythematosus from the patient's perspective.

Aims: The aim of this study was to evaluate the impact of systemic lupus erythematosus (SLE) on the lives of patients in order to inform the development of a conceptual model.

Methods: Twenty-two participants with SLE (defined as meeting four of the 11 ACR criteria) were recruited for this study. Semi-structured, in-person interviews were conducted with each participant, exploring the symptoms experienced and the impact on the patient's life.

Elicitation of health state utilities in neuroendocrine tumours.

Background: Neuroendocrine tumours (NETs) are a rare form of neoplasm that can arise in most organs of the body and which share many common pathologic features. Although curative surgery can be conducted for patients with localised disease, once progression occurs and the disease becomes metastatic or un-resectable, treatment aims to extend life and maintain quality-of-life for as long as possible. The aim of the study was to elicit utilities for health state vignettes describing the burdens associated with receiving therapy for advanced NETs.

Patient preferences for first-line oral treatment for mild-to-moderate ulcerative colitis: a discrete-choice experiment.

Background: Patients with ulcerative colitis (UC) frequently require long-term therapy to prevent relapse. Treatments such as 5-aminosalicylic acid (5-ASA [mesalazine]) are efficacious and well tolerated, but adherence to treatment is often poor.

Objective: This discrete-choice experiment (DCE) was conducted to estimate differences in patient preferences for 5-ASA treatment in mild-to-moderate UC based on levels of self-reported adherence.

Can the functional assessment of multiple sclerosis adapt to changing needs? A psychometric validation in patients with clinically isolated syndrome and early relapsing-remitting multiple sclerosis.

Background: The Functional Assessment of Multiple Sclerosis (FAMS) is widely used in clinical trial programmes; however, it was developed before the rise in trials targeted at early stage multiple sclerosis (MS) and clinically isolated syndrome (CIS).

Objective: The aim of this study was to assess the psychometric properties of the FAMS within two clinically distinct populations, CIS and early relapsing-remitting MS (RRMS), and discern the appropriateness of the FAMS within these populations.

Methods: Secondary analysis was conducted on FAMS data from two clinical trials assessing interferon beta-1b in early RRMS and CIS.

Preferences for antimuscarinic therapy for overactive bladder.

OBJECTIVE • To examine patient preferences and strength of preferences for treatment for the various symptoms of overactive bladder and adverse events associated with the use of antimuscarinic treatments. PATIENTS AND METHODS • A discrete choice experiment (DCE) survey was developed that detailed treatment choices in terms of attributes relating to their efficacy in reducing symptoms and the likelihood of experiencing typical adverse events. Levels for each attribute were based on a literature review, qualitative interviews and a meta-analysis of clinical trial data.

A valuation of infusion therapy to preserve islet function in type 1 diabetes.

Objectives: Recent advances in monoclonal antibody therapies offer the prospect of the prevention or amelioration of type 1 diabetes mellitus (T1DM). The present study was designed to capture UK (English and Scottish) preference weights for the process of undergoing infusion therapy and the likely outcomes of treatment for children (8-12 years), adolescents (13-17 years), and adults.

Methods: Vignette descriptions of T1DM health states (describing infusion therapy and reduced insulin need) were constructed based on qualitative interviews with people with type 1 diabetes, clinicians and findings from a literature review.

Elicitation of health state utilities in metastatic renal cell carcinoma.

Objective: The aim of the study was to obtain United Kingdom societal preferences for receiving newly developed treatments for metastatic renal cell carcinoma.

Methods: Health states were developed based on a literature review and in-depth interviews with clinical experts. These states described the burden of both stable and progressive disease, and a variety of commonly encountered toxicities associated with first-line therapies (fatigue, diarrhoea, nausea/vomiting, mucositis, hand/foot syndrome, hypertension and anaemia).

Tenoxicam and paracetamol-codeine combination after oral surgery: a prospective, randomized, double-blind, placebo-controlled study.

We studied 90 adults undergoing surgical removal of at least both lower third molar teeth as day cases under standardized general anaesthesia. Patients were allocated randomly (with stratification for surgeon) to receive tenoxicam 40 mg, tenoxicam 20 mg or placebo i.v.

A blinded comparison of fluticasone propionate with budesonide via powder devices in adult patients with moderate-to-severe asthma: a clinical evaluation.

In Vitro and in vivo data have demonstrated that there are detectable differences between inhaled corticosteroids commonly used to treat asthma. However, controversy still remains as to whether these differences translate into clinical benefits. This 12-week, international, randomized, doubleblind, parallel-group study was undertaken to compare the efficacy and safety of fluticasone propionate (FP) 800 mug daily, administered as a powder via the Diskhaler((R)), and budesonide (BUD) 1600 mug daily, administered using the Turbuhaler((R)), in adult patients with moderate-tosevere asthma.