Iyar Mazar

Patient-reported preferences for oral versus intravenous administration for the treatment of cancer: a review of the literature

Objective The emergence of various modes of administration for cancer treatment, including oral administration, brings into focus the importance of patient preference for administration. The purpose of this research was to evaluate the administration preferences of cancer patients, specifically between oral and intravenous (IV) treatment, as well as the factors contributing to preference. Methods A literature search was conducted in OvidSP to identify research in which the preferences of cancer patients for oral or IV treatment have been evaluated. Data were analyzed in two stages: 1) those articles that directly compared preference between modes of administration were tallied to determine explicit preference for oral or IV treatment; and 2) all attributes associated with patient preference were documented. Results Of the 48 abstracts identified as part of the initial OvidSP search, eight articles were selected for full-text review. One article was removed following full-text review, and seven additional articles were identified through a gray literature search, yielding a total of 14 articles for evaluation. In Stage 1, 13 of the 14 articles compared preference, of which eleven articles (84.6%) reported that patients preferred oral treatment over IV, while two (15.4%) stated that cancer patients preferred IV treatment over oral. In Stage 2, the most frequently reported attributes contributing to preference included convenience, ability to receive treatment at home, treatment schedule, and side effects. Discussion Evidence suggests that oncology patients prefer oral treatment to IV. Rationale for preference was due to a number of factors, including convenience, perception of efficacy, and past experience. Further evaluation should be conducted, given the limited data on patient preference in oncology.

Patient-Reported Outcomes in Metastatic Breast Cancer: A Review of Industry-Sponsored Clinical Trials

Introduction Patient-reported outcome (PRO) measures serve to capture vital patient information not otherwise obtained by primary study endpoints. This paper examines how PROs are utilized as endpoints in industry-sponsored metastatic breast cancer clinical trials. Methods A search was conducted in the clinicaltrials.gov web site for trials involving common treatments for metastatic breast cancer. Thirty-eight clinical trials were identified which included a PRO endpoint in the study, and data were extracted and summarized. Results Overall, 17 unique PRO questionnaires and 14 concepts of measurement were identified as secondary or exploratory endpoints. The Functional Assessment of Cancer Therapy—Breast was the most frequently utilized questionnaire, commonly implemented to assess quality of life. The EORTC QLQ-C30 was also frequently used to measure quality of life or pain. Conclusion This review shares insights into the role of PROs in trials for metastatic breast cancer from which treatment developers and other stakeholders can enhance successful implementation of the patient voice into future trials.

Clinical Trial Patient-reported Outcomes Data: Going Beyond the Label in Oncology

PURPOSE Patient-reported outcome (PRO) data are increasingly being implemented in oncology clinical trial research to evaluate treatment benefit, such as disease-related symptoms, treatment-related adverse events, and health-related quality of life impacts. However, only a small amount of PRO data collected is used to support labeling claims, leaving a substantial amount of data that could be shared by sponsors to further convey treatment benefit from the patient perspective. METHODS This paper describes how pharmaceutical sponsors can realize the value of PRO data derived from oncology trials with regard to the following stakeholders: payers, health care providers (HCPs), and patient advocacy groups. Further, ideas are presented for integrating PRO data and implementing PRO assessments within oncology, by stakeholder type. Finally, a summary is provided to describe how PRO data can benefit the patient by facilitating better, more symptom-focused care and enhancing treatment decisions. FINDINGS With the goal of motivating further use of PRO assessments in oncology, we present examples of how payers utilize PRO data to inform reimbursement decisions (eg, PRO data inform decisions made by Germany׳s Institute for Quality and Efficiency in Health Care and the United Kingdom׳s National Institute for Health and Care Excellence); how communication of results with patient advocacy groups can lead to a better understanding of what is important to patients; and how HCPs can use PRO instruments to inform patient treatment decisions through real-world application. IMPLICATIONS Integrating PRO data can enhance health care by allowing the patients voice to carry beyond regulatory decisions and into those made by payers and HCPs, which are crucial to quality care and assessing the value of care. Utilizing PRO assessments and communicating results to key stakeholders in the oncology space can allow sponsors to report treatment benefit and, more importantly, can provide valuable insight into the patient treatment experience.

Understanding Foreclosure Risk: The Role of Nativity and Gender

Drawing on qualitative interviews and fieldwork, this paper examines the risk factors that lead families into mortgage default and foreclosure risk. While neoliberal ideology and policy recognize lower-income families as at greater risk, in this paper we argue that immigrants and women experience particular risks – separation and divorce, the expense of remittances, poorer treatment in rental markets, and conflicting experience of ownership between the United States and country of origin. When these differences are ignored, social policies cannot respond adequately to the disproportionate risk that women and immigrants face to homeownership.

Development of the Rheumatoid Arthritis Symptom Questionnaire (RASQ): a patient reported outcome scale for measuring symptoms of rheumatoid arthritis

Abstract Background: Rheumatoid arthritis (RA), a chronic, progressive inflammatory, autoimmune disease, can substantially reduce health-related quality of life (HRQoL) and lead to severe disability and early mortality. Patient-reported outcome (PRO) instruments are used to assess the patient experience of RA symptoms and impacts, and can capture RA treatment effects. To address limitations in existing PRO instruments, this research aimed to establish the content validity of a new instrument, the Rheumatoid Arthritis Symptom Questionnaire (RASQ), to assess the signs and symptoms of RA. Method: The most important and relevant sign and symptom concepts for RA patients were identified through a targeted review of the published literature, expert opinion, and concept elicitation patient interviews. Cognitive interviews were conducted with patients to test the comprehensibility and comprehensiveness of the RASQ. Results: Seven symptoms emerged consistently across the conceptual research: joint pain, joint swelling, joint stiffness, joint tenderness, joint warmth, muscle pain, and tiredness. Draft item content was developed to assess these symptoms, in addition to a single impact item, resulting in three RASQ versions: two utilizing a 7 day recall period (one assessing symptoms at their worst, the other on average) and a third using a 24 hour recall period assessing symptoms at their worst. Cognitive interview results demonstrated patient understanding and ability to use the instrument. Conclusions: Content validity of the RASQ was established in accordance with instrument development guidelines. The RASQ fills a measurement gap by assessing the RA signs and symptoms most important to patients. Research evaluating the RASQ’s psychometric properties is underway.

Patient-reported outcomes for US oncology labeling: review and discussion of score interpretation and analysis methods

This paper describes ways to approach the conceptual and practical challenges associated with interpreting the clinical meaning of scores produced by patient reported outcome (PRO) questionnaires, particularly when used to inform efficacy decisions for regulatory approval for oncology products. Score interpretation estimates are not inherent to PRO questionnaires per se, instead, vary dependent upon sample and study design characteristics. Scores from PRO measures can be interpreted at the individual and group level, and each carries its own set of statistics for evaluating differences. Oncology researchers have a variety of methods and data analytic strategies available to support their score interpretation needs, which should be considered in the context of their a priori knowledge of the target patient population, the hypothesized effects of treatment, the study design and assessment schedule, and the inferences and decisions to be made from the PRO data.

The use of patient-reported outcomes in advanced breast cancer clinical trials: a review of the published literature

Abstract Objective: As a means to measure quantifiable signs, symptoms, and impacts of a disease or its treatment, patient-reported outcome (PRO) instruments can be applied to numerous settings, including use in drug development to support labeling claims. This research summarizes the use of PROs in trials for 16 commonly used regulatory approved treatments for advanced or metastatic breast cancer. Methods: For each treatment (n = 16), a literature search was conducted in MEDLINE, Embase, and PsycINFO. The primary criterion for selection was the report of studies that used PROs to evaluate treatment benefit and/or toxicity in advanced or metastatic breast cancer. From this, a sub-set of articles for each treatment were selected for full-text review where PRO-related information was extracted and summarized. Results: The searches yielded 1727 publications. Following abstract review, 1702 were excluded because they failed to meet criteria, or were duplicates or less relevant for PRO information reported. Thus, 25 articles were reviewed in detail for this evaluation. Eleven PRO instruments were identified from these publications. The European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire – Core (EORTC QLQ-C30) was utilized the most frequently (n = 13, 52.0%). Most publications reported PROs positioned as secondary endpoints (n = 20, 80.0%); described some of the statistical analyses applied to PRO data (n = 21, 84.0%); and specified PRO results (n = 23, 92.0%). Conclusions: While several of the publications provided some information on how PROs were utilized, many did not describe details for PRO administration, scoring, analyses, and results interpretation. While it is encouraging that PROs are often used in clinical trials for patients with metastatic breast cancer, they are not commonly used to support endpoints that establish the basis for label claims. Because they yield direct insight into the patient experience of a condition, PROs may be used to provide a more comprehensive perspective of the benefits and risks from treatment.

Patient-reported outcomes in advanced breast cancer: inside the label and approval documents

ABSTRACT Patient-reported outcome (PRO) measures are used in clinical research and practice for the assessment of disease-related symptoms and impacts as well as treatment-related side effects, from the patient perspective. However, a systematic examination of the role of PROs in metastatic breast cancer treatment approvals is lacking. A review of FDA labels and historical drug approval documents for metastatic breast cancer treatments was conducted to determine how PROs had been used or pursued to support labeling claims. In the historical drug approval documents, PROs were often being implemented by sponsors, and regulatory reviewers noted several issues limiting their suitability to support label claims. The findings suggest there is much room for improvement in how sponsors develop, implement, and report PRO measurement strategies as part of drug approval.