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. Results: Nine expert advisers achieved consensus on 11 AK-EPQ items that encompass patient and clinician perspectives of treatment-related local skin reactions, clinical and cosmetic outcomes associated with AK, and satisfaction with treatment; the AK-EPQ will be first implemented in the Patient-Reported Outcomes for Actinic Keratosis study (NCT05260073). Limitations: The AK-EPQ does not directly measure quality of life, although it can be used with validated quality of life instruments. Conclusion: The newly developed AK-EPQ elicits insights into the patient and clinician experience with AK treatments. Comparative probing of these perspectives may help optimize precision medicine in AK treatment.

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. RESULTS: The safety population included 300 patients; the full analysis set included 290 patients (278 patients at W24). At W8, a statistically significant difference (P<0.03) was observed for Skindex-16 domains in all assessed subgroups. Clinicians and patients reported high global satisfaction (mean [SD] scores of 74.9 [23.9] and 72.0 [24.6], respectively) at W24. Overall skin appearance improved from baseline to W24 (83.6% clinicians; 78.5% patients). IGA success (IGA score of 0-1) was achieved by 71.9% of patients at W24 with a similar % at W8 (73.8%) suggesting a stable effectiveness over time. About 5% of patients reported at least one adverse event, 4% reported at least one serious adverse event and no patients reported serious adverse drug reactions. At W8, the most frequently reported local skin reactions were mild/moderate erythema (47.6%) and flaking/scaling (49.6%). CONCLUSIONS: Treatment with tirbanibulin demonstrated effectiveness in the management of AK lesions and a favorable safety and tolerability profile. Furthermore, QoL was improved as early as W8, and both patients and clinicians reported high levels of treatment satisfaction, independently of patients' characteristics. J Drugs Dermatol. 2024;23(5):338-346. doi:10.36849/JDD.8264.

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. Demographic and clinical characteristics, and HRQoL measures were compared between patient groups, and multiple regression analyses were conducted. RESULTS: After differences in patient demographic and clinical characteristics were controlled for, significantly better scores were observed in the G551D/IVA group than in the F508del/SOC group on multiple domains of the validated Cystic Fibrosis Questionnaire-Revised and the EuroQol 5-dimensions 5-level questionnaire. CONCLUSIONS: G551D/IVA patients reported better HRQoL than F508del/SOC patients on generic and disease-specific measures in a real-world setting.

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. RESULTS: Female (68%) family members (94%) assisted care recipients with multimorbidity (mean 2.9 conditions) and polypharmacy (mean 3.2 prescriptions). On average, caregivers performed 3 visit communication behaviors (e.g., notetaking) and implemented 2.6 home medication management strategies (e.g., filling/monitoring pill boxes). Communication-related variables explained 17% of home medication management assistance; including caregivers' visit communication (std. beta 0.31), physicians' medication communication (std. beta 0.15) and pharmacist discussions (std. beta 0.10). The final model included recipients' multimorbidity and caregiver education (std. betas 0.21 and 0.13) explaining 22% of caregiver assistance with home medication management. CONCLUSION: Caregivers' assistance with safe and effective home medication use crosses care contexts and is facilitated by clinician and pharmacist communication. PRACTICE IMPLICATIONS: Support for caregiver engagement in healthcare conversations can contribute to patient adherence and family-centered, high quality care.

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

INTRODUCTION: 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. Areas covered: A targeted literature review of studies published from 2010-2016 assessed adherence to CF therapies. Nineteen studies qualified for inclusion. Adherence to CF therapies was sub-optimal, and varied by treatment, mode of treatment administration, age, season, time and method of adherence measurement. Adherence to ivacaftor and inhaled antibiotics were reported higher than dornase alfa or hypertonic saline, oral pancreatic enzyme and vitamin supplements, and airway clearance therapy. Several patient, healthcare provider and treatment related factors influenced adherence. Sub-optimal adherence was shown to impact clinical and economic burden of the disease. Expert commentary: Higher adherence to CF therapies can lower disease burden, and improve patient outcomes. Healthcare providers and policy makers should devise patient-centered and caregiver-enabled interventions to improve adherence. Research on long-term adherence and outcomes associated with promising oral treatments such as CFTR modulators is needed. Identifying ways to overcome key barriers to adherence can positively affect outcomes associated with CF.

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). Eligible participants completed three QoL instruments, EORTC QLQ-C30, the pancreatic cancer module of EORTC QLQ-PAN26, and the EQ-5D, during their clinical visits at 14 clinics across the USA. RESULTS: Demographic characteristics were similar among groups (no treatment, n = 29; nab-paclitaxel plus gemcitabine PR or SD, n = 26; ≥1L PD, n = 17). Patients in the nab-paclitaxel plus gemcitabine PR or SD group had lower mean pain scores by EORTC-QLQ-C30 (27.6 vs 47.1; P = 0.02) and lower mean pancreatic pain scores by EORTC-QLQ-PAN26 (27.9 vs 45.4; P = 0.02) compared with the no treatment group. The groups did not differ significantly in QoL as measured by the EQ-5D. CONCLUSIONS: Patients who experienced PR or SD with 1L nab-paclitaxel plus gemcitabine had improved general and pancreatic pain scores and no clinically meaningful deterioration in QoL compared with patients who had not yet initiated chemotherapy.

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. OBJECTIVES: To assess US oncologists' perspectives on and support for ASCO's payment reform proposal, and to determine use of quality-of-care metrics, factors influencing their perception of value of new cancer drugs, the influence of cost on treatment decisions, and the perceptions of the reimbursement climate in the country. METHODS: Physicians and medical directors specializing in oncology in the United States practicing for at least 2 years and managing at least 20 patients with cancer were randomly invited, from an online physician panel, to participate in an anonymous, cross-sectional, 15-minute online survey conducted between July and November 2014. The survey assessed physicians' level of support for the payment reform, use of quality-of-care metrics, factors influencing their perception of the value of a new cancer drug, the impact of cost on treatment decision-making, and their perceptions of the overall reimbursement climate. Descriptive statistics (chi-square tests and t-tests for discrete and continuous variables, respectively) were used to analyze the data. Logistic regression models were constructed to evaluate the main payment models described in the payment reform proposal. RESULTS: Of the 231 physicians and medical directors who participated in this study, approximately 50% strongly or somewhat supported the proposed payment reform. Stronger support was seen among survey respondents who were male, who rated the overall reimbursement climate as excellent/good, who have a contract with a commercial payer that reimburses for dispensed oral cancer drugs, or who practice in a hospital setting. The use of at least 1 quality-of-care metric was more common among respondents participating in an accountable care organization (ACO) than among those not participating in an ACO (92.6% vs 83.2%, respectively; P = .0380). The most common metric used by the physicians in their practice setting was patient satisfaction scores (60.1%). Accountability for delivering high-quality care was supported by 74.9% of respondents; those who practice in a hospital setting were twice as likely as those in private practice to support accountability for quality of care (81.3% vs 67.6%; odds ratio, 2.1; P = .0176). CONCLUSION: Support for ASCO's payment reform proposal is mixed among oncology physicians and medical directors, underscoring the importance of continuous and broader engagement of practicing physicians around the country via outreach and dialogue on topics that impact their clinical practices, as well as providing education or awareness activities by ASCO to its membership.