Developing a questionnaire for assessing clinician- and patient-reported outcomes in actinic keratosis: Results from an expert panel.

Background: Current assessments on topical treatment attributes in actinic keratosis (AK) do not evaluate safety, effectiveness, and satisfaction from both clinician and patient perspectives, creating an unmet need for more comprehensive AK-specific measures that fully capture the patient experience.

Objective: To develop an actinic keratosis-specific expert panel questionnaire (AK-EPQ) of patient-reported outcomes and clinician-reported outcomes for use in research studies.

Methods: Using interviews of patients with AK and targeted literature reviews, a 9-person consensus panel of dermatologists with expertise in AK treatment was convened to develop the AK-EPQ to assess AK-specific patient-reported outcomes and clinician-reported outcomes.

Patient- and Clinician-Reported Outcomes for Tirbanibulin in Actinic Keratosis in Clinical Practice Across the United States (PROAK).

Background: The Patient-Reported Outcomes in Actinic Keratosis (PROAK) study evaluated patient- and clinician-reported outcomes (PRO; ClinRO) during 24 weeks of follow-up among adult patients with actinic keratosis (AK) on the face or scalp who were administered tirbanibulin 1% ointment in real-world community practices in the United States.  Methods: Quality of life (QoL) was assessed by Skindex-16 at week (W) 8. Additionally, effectiveness (Investigator Global Assessment [IGA]), PRO and ClinRO (Treatment Satisfaction Questionnaire for Medication and Expert Panel Questionnaire), safety, and tolerability were assessed at W8 and W24.

Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study.

Background: Clinical studies demonstrate that ivacaftor (IVA) improves health-related quality of life (HRQoL) in patients aged ≥6 years with cystic fibrosis (CF). The real-world impact of IVA and standard of care (SOC) in groups of patients with G551D and F508del mutations, respectively, was assessed using a survey comprising disease-specific and generic HRQoL measures.

Methods: Patients with CF aged ≥12 years, or aged 6-11 years with caregiver support, with either (1) a G551D mutation and receiving IVA (G551D/IVA) for ≥3 months, or (2) homozygous for F508del and receiving SOC before lumacaftor/IVA availability (F508del/SOC), were eligible to participate in a cross-sectional survey.

Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.

Introduction: ACT DMD was a 48-week trial of ataluren for nonsense mutation Duchenne muscular dystrophy (nmDMD). Patients received corticosteroids for ≥6 months at entry and stable regimens throughout study. This post hoc analysis compares efficacy and safety for deflazacort and prednisone/prednisolone in the placebo arm.

Family caregivers' facilitation of daily adult prescription medication use.

Objective: To describe ways family members assist adult patients with prescription medications at home, during medical visits and at the pharmacy.

Methods: Online survey of 400 adults (caregivers) who help another adult (care recipient) with prescription medication use. Regression modeled the contribution of caregiver communication during recipients' medical visits, evaluation of physicians' medication communication and discussions with the dispensing pharmacist on caregivers' assistance with home medication management.

Adherence to therapies in cystic fibrosis: a targeted literature review.

Cystic fibrosis (CF) is a life-shortening condition with no cure. Available therapies relieving the symptoms of CF are complex and time-consuming. A comprehensive review assessing adherence to different CF therapies, association of adherence with outcomes, and factors influencing adherence could inform optimal patient management strategies.

Health-Related Quality of Life in Patients with Metastatic Pancreatic Cancer.

Purpose: Due to its clinical course and often-late detection, many patients with metastatic pancreatic cancer (mPC) experience poor quality of life (QoL). This pilot project assessed real-world QoL in patients with mPC at different stages of treatment.

Methods: A cross-sectional survey was conducted in the following groups of patients with mPC: before initiation of first-line (1L) chemotherapy (no treatment); with partial response (PR) or stable disease (SD) upon receipt of ≥3 cycles of 1L nab-paclitaxel plus gemcitabine (nab-paclitaxel plus gemcitabine PR or SD); and with disease progression during ≥1L chemotherapy and not currently receiving nab-paclitaxel (≥1L PD).

Oncologist Support for Consolidated Payments for Cancer Care Management in the United States.

Background: The cost of cancer care in the United States continues to rise, with pressure on oncologists to provide high-quality, cost-effective care while maintaining the financial stability of their practice. Existing payment models do not typically reward care coordination or quality of care. In May 2014, the American Society of Clinical Oncology (ASCO) released a payment reform proposal (revised in May 2015) that includes a new payment structure for quality-of-care performance metrics.

Demonstrating the value of medicines: evolution of value equation and stakeholder perception of uncertainties.

It is important to evaluate how the value of medicine is assessed, as it may have important implications for health technology and reimbursement assessments. The value equation could comprise 'incremental benefit/outcome' (relative results of care in terms of patient health, comparing the innovation to best available alternative(s)) in the numerator and 'cost' (relative costs involved in the full cycle of care (or a defined period) for the patient's medical condition, incorporating the relevant cost-offsets due to displacement of best available alternative(s)) in the denominator. This 'relative value' combined with the overall net budget impact (of including the drug in the formulary or reimbursed drug list) at the concerned population level in the given institution/region/country may better inform the usefulness of the new therapeutic option to the healthcare system.

Disease burden and patient reported outcomes among patients with moderate to severe psoriasis: an ethnography study.

Objectives: To assess the impact of psoriasis on health-related quality of life (HRQoL).

Methods: An ethnographic study of patients with moderate to severe psoriasis was conducted in the US, France, Germany, Italy, Spain, UK, Brazil, and Canada to explore patients' views on treatment and the impact of psoriasis on HRQoL. Anthropologists and ethnographers spent a minimum of 5.

Economic burden of systemic lupus erythematosus flares and comorbidities in a commercially insured population in the United States.

Objective: To estimate the medical and productivity-related cost burden of systemic lupus erythematosus (SLE) flares and comorbidities in a commercially insured population.

Methods: Using administrative data, annual medical costs and indirect costs because of work loss were calculated for adult SLE patients, including flare severity and SLE-related comorbidity subsets, and a matched control group without SLE.

Results: Adjusted annualized medical costs were $18,952, $4305, $914, and $441 greater for SLE patients with severe, moderate, mild, and no flares, respectively, during follow-up than those of the matched controls.

A longitudinal analysis of costs associated with change in disease activity in systemic lupus erythematosus.

Objectives: To estimate the economic consequences of changes in disease activity on healthcare resource utilization (HRU) and costs.

Methods: A retrospective longitudinal study of systemic lupus erythematosus (SLE) patients receiving care in a regional integrated health delivery system in the US from 01/2004 through 03/2011 was conducted using electronic health records, medical chart reviews, and claims. Eligible patients were ≥18 years old, with ≥1 rheumatologist-confirmed SLE diagnosis and ≥1 eligible rheumatology encounter.

Fall-related hospitalization and facility costs among residents of institutions providing long-term care.

Purpose: The purpose of this study was to estimate hospital and long-term-care costs resulting from falls in long-term-care facilities (LTCFs).

Design And Methods: The study used a retrospective, pre/post with comparison group design. We used matching, based on propensity scores, to control for baseline differences between fallers and non-fallers.

Factors influencing the acceptance of changes in antiretroviral therapy among HIV-1-infected patients.

Physicians routinely consider modifying antiretroviral therapy (ART) regimen for their patients with HIV. Little is known about the factors associated with patients' willingness to accept providers' recommended ART changes. This multicenter prospective observational study examined factors associated with willingness to accept ART changes recommended by their providers among HIV-infected adults from six urban outpatient HIV clinics.

Is drug therapy for urinary incontinence used optimally in long-term care facilities?

Objectives: To examine the management of urinary incontinence (UI) among nursing home (NH) residents in the United States, particularly drug therapy for UI in those who may be suitable candidates for such treatment based on their functional status.

Design: Retrospective analysis of admissions (between January 2, 2002, and December 31, 2003) to a total of 373 skilled nursing facilities and assisted living centers operated by a single provider of long-term care.

Participants: Residents identified as incontinent according to at least one Minimum Data Set (MDS) assessment during their NH stay who had adequate mobility and/or cognitive ability to toilet, as determined by a toileting score of < or =2 on the 5-point MDS scale, and/or a score of < or =3 on the 7-point scale, the Cognitive Performance Score (CPS).

Antipsychotic drug use among nursing home residents taking rivastigmine.

Objective: To evaluate whether or not rivastigmine use is associated with a decrease in the initiation of antipsychotic drug therapy among nursing home residents in the United States.

Methods: A retrospective analysis was performed using Minimum Data Set data and physician order files for newly admitted residents of 452 US nursing facilities from 2000 through 2002. The rivastigmine group included those who were diagnosed with Alzheimers disease (AD) and began rivastigmine treatment within 30 days of diagnosis.

Urinary incontinence in the nursing home: resident characteristics and prevalence of drug treatment.

Objectives: To provide a descriptive overview of the elderly, nursing home patient population with urinary incontinence (UI).

Methods: This study was a descriptive, cross-sectional database analysis (2002-2003) examining UI prevalence, demographic and clinical characteristics of UI patients, and UI pharmacotherapy prevalence in the nursing home setting.

Results: Of the 29 645 eligible subjects, 8995 experienced some level of UI at the time the minimum data set (MDS) was completed (30%).

Comparison of pressure ulcer treatments in long-term care facilities: clinical outcomes and impact on cost.

This study compared clinical outcomes and nursing labor costs associated with (a) balsam Peru, hydrogenated castor oil, and trypsin (BCT) ointment; (b) BCT + Other; and (c) Other treatments in 2014 wound episodes occurring in 861 patients (mean 2.34 wounds/patient). Treatment with BCT ointment or BCT + Other was associated with a higher healing rate (P < .

Asthma control and patient satisfaction among early pediatric users of montelukast.

Objective: To assess asthma control and patient satisfaction among pediatric users of montelukast in a clinical practice setting.

Study Design: A prospective study of 175 children with persistent asthma, 6 to 14 years of age, who initiated treatment with montelukast between Feb-1998 and Aug-1998, in primary care and pediatric offices across the United States. Data on asthma control and satisfaction with treatment was collected in physicians' offices after enrollment and by survey to the patients' homes at 1 month of treatment.