Publications by authors named "Andrew Yaworsky"
Article Synopsis
- CHAPLE disease is a rare and serious condition affecting fewer than 100 young people worldwide, characterized by symptoms like abdominal pain and protein loss from the intestines.
- A study used mixed methods, including interviews and clinical assessments, to evaluate the impact of pozelimab treatment on these symptoms over 24 weeks.
- Results showed that after treatment, patients experienced complete resolution of core symptoms, highlighting the value of mixed-methods in understanding patient experiences in rare disease trials.
Purpose Of Review: Based on shared decision-making (SDM) principles, a decision aid was previously developed to help patients, their caregivers, and physicians decide which peanut allergy management approach best suits them. This study refined the decision aid's content to better reflect patients' and caregivers' lived experience.
Recent Findings: Current standard of care for peanut allergy is avoidance, although peanut oral immunotherapy has been approved by the Food and Drug Administration for use in patients 4-17 years old.
Validation of the novel Eosinophilic Esophagitis Impact Questionnaire.
J Patient Rep Outcomes
November 2023
Background: Eosinophilic esophagitis (EoE) has a detrimental effect on health-related quality of life (HRQOL). The Eosinophilic Esophagitis Impact Questionnaire (EoE-IQ) is a novel patient-reported outcome (PRO) measure assessing the impact of EoE on HRQOL. To assess suitability of the EoE-IQ, its measurement properties were evaluated.
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- The study evaluates physicians' preferences and experiences with once-weekly somatrogon, a long-acting growth hormone, compared to the standard once-daily rhGH for treating pediatric growth hormone deficiency (pGHD).
- Survey results showed that 75% of pediatric endocrinologists preferred somatrogon, citing its convenience and lower burden on patients and caregivers.
- A majority found both treatment regimens equally effective for long-term growth outcomes, but significantly more were "very satisfied" with somatrogon.
Background: Daily injections of recombinant human growth hormone are the standard of care to treat growth failure due to pediatric growth hormone deficiency (GHD). While effective, daily injections are burdensome and can compromise adherence. In recent years, novel injection treatments requiring less frequent administration for growth hormone deficiency (GHD) have been developed.
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- The study focused on developing two questionnaires to assess the signs and symptoms of eosinophilic esophagitis (EoE) in children ages 1 to less than 12 years, taking into account that younger patients may struggle to self-report their symptoms.
- Researchers conducted literature reviews, expert meetings, and interviews with children and their caregivers to identify relevant symptoms and ensure the questionnaires were understandable and comprehensive.
- The result was the Pediatric Eosinophilic Esophagitis Sign/Symptom Questionnaire for self-reporting in older children (PESQ-P) and a caregiver-reported version (PESQ-C), which both showed strong content validity for use in clinical trials.
Article Synopsis
- - The study evaluates the psychometric performance of the NCCN-FACT Ovarian Cancer Symptom Index-18 (NFOSI-18) in patients with advanced ovarian cancer, using observational data from 897 patients undergoing treatment.
- - It assesses factors like internal consistency reliability and construct validity, finding that the NFOSI-18 scores show acceptable reliability and correlate well with other established quality of life measures.
- - The results indicate specific clinically important differences associated with various symptoms and treatment effects, which can help in interpreting treatment outcomes in clinical trials.
Background: Evolving regulatory guidelines recommend routine assessment of the acceptability of pediatric oral medicines throughout clinical development processes. However, such assessment is problematic owing to a lack of standard methods or criteria that define acceptability for children and their caregivers. This research aimed to identify the attributes of acceptability for targeted oral formulation types that are important to children, and to develop content-valid patient- and caregiver-reported outcome acceptability measures for use in the context of clinical drug development.
View Article and Find Full Text PDFBackground: Patients treated with peanut oral immunotherapy (OIT) may experience adverse reactions, particularly during up-dosing.
Objective: To develop the Side Effects of Peanut Oral Immunotherapy Diary (SEPOD), an electronic questionnaire assessing the daily side effects of peanut OIT in clinical trials.
Methods: Content and design of the SEPOD were informed by empirical literature review and meetings with 3 allergy-immunology experts.
Introduction: The daily injection burden of recombinant human growth hormone (r-hGH) replacement therapy to treat growth hormone deficiency (GHD) may reduce compliance and limit treatment benefit. Research is needed to evaluate patient preferences for GHD injection regimen and device features.
Objective: Quantitatively evaluate factors driving preferences for r-hGH injection regimen and device features among pediatric (3-17 years, and caregivers) and adult (≥25 years) patients with GHD using a discrete choice experiment (DCE) approach.
Article Synopsis
- Current r-hGH replacement therapy for growth hormone deficiency (GHD) involves daily subcutaneous injections, which are burdensome for patients and may lead to poor adherence to treatment.
- To improve treatment compliance and outcomes, qualitative research was conducted to develop the Life Interference Questionnaire for Growth Hormone Deficiency (LIQ-GHD) that captures patient experiences related to these injections.
- The LIQ-GHD was tested through interviews and a cross-sectional study to assess its content, reliability, and validity across different patient age groups, using expert insights and literature review to refine the questionnaire.
Article Synopsis
- - The study aims to provide evidence-based recommendations for determining sample size in qualitative concept elicitation (CE) interviews, focusing on the point of saturation where no new data is expected.
- - A retrospective evaluation of 26 CE interview studies revealed that most symptom concepts saturated around the 10th to 25th interviews, with 99% of concepts emerging by the 25th interview.
- - The findings offer practical guidance for researchers on how many interviews might be necessary for saturation in qualitative studies related to clinical outcome assessment (COA) instrument development, addressing a key gap in qualitative research.
Introduction: To establish clinical consensus on important and relevant quality-of-care (QoC) attributes in rheumatic disease (RD) treatment that may improve treatment outcomes and guide best practices.
Methods: Twenty-three QoC attributes were identified in a literature review. Fifteen European-based clinicians were selected based on their contributions to RD guidelines, publications, and patient care.
Background: Despite an increased use of patient-reported outcomes (PROs) in oncology clinical trials, integrating the patient perspective into drug approval decisions and documentation has been challenging.
Objectives: To review important regulatory and measurement terminology, and to provide oncology outcomes researchers and those involved with building oncology programs with tools to plan PRO data collection, particularly in relation to drug efficacy claims for drug labeling in the United States.
Discussion: When contemplating a PRO measurement strategy for oncology clinical trials, outcomes researchers are challenged in several ways.
Article Synopsis
- The paper discusses the importance of patient-reported outcome (PRO) data in oncology clinical trials and how it can be better utilized to showcase treatment benefits from the patients' viewpoints.
- It suggests methods for pharmaceutical sponsors to enhance the value of PRO data for key stakeholders such as payers, healthcare providers (HCPs), and patient advocacy groups, ultimately aiming for more patient-centered care.
- Real-world examples illustrate how PRO data can influence reimbursement decisions, improve communication with advocacy groups, and aid HCPs in making better treatment choices, emphasizing the need for integrating this data into health care practices.
Patient-reported outcomes for US oncology labeling: review and discussion of score interpretation and analysis methods.
Expert Rev Pharmacoecon Outcomes Res
July 2016
Article Synopsis
- The paper addresses challenges in interpreting patient reported outcome (PRO) scores, especially in the context of regulatory approval for oncology treatments.
- It emphasizes that score interpretation is not standardized and varies based on study design and sample characteristics.
- Researchers have multiple methods available for analyzing PRO scores, which should align with their understanding of the patient population and the specific goals of their study.
Background: Treatments for upper facial lines (UFL), the most visible sign of aging, are of interest to patients and clinicians alike. Patient-reported outcomes (PROs) are valuable in evaluating the impact of such treatments; however, regulatory recommendations have stipulated that the patient perspective be central in developing these assessments.
Objectives: (1) To evaluate the content validity of the Facial Lines Outcomes Questionnaire, a PRO instrument developed to assess upper facial line impacts, according to the regulatory guidance of the United States Food and Drug Administration and (2) assess whether it adequately measures the psychological impacts associated with crow's feet lines (CFL) (lateral canthal lines) from the patient perspective.