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Online administration of surveys has a number of advantages but can also lead to increased exposure to bad actors (human and non-human bots) who can try to influence the study results or to benefit financially from the survey. We analyze data collected through an online discrete-choice experiment (DCE) survey to evaluate the likelihood that bad actors can affect the quality of the data collected. We developed and fielded a survey instrument that included two sets of DCE questions asking respondents to select their preferred treatments for multiple myeloma therapies. The survey also included questions to assess respondents' attention while completing the survey and their understanding of the DCE questions. We used a latent-class model to identify a class associated with perverse preferences or high model variance, and the degree to which the quality checks included in the survey were correlated with class membership. Class-membership probabilities for the problematic class were used as weights in a random-parameters logit to recover population-level estimates that minimizes exposure to potential bad actors. Our results highlight the need for a robust discussion around the appropriate way to handle bad actors in online preference surveys. While exclusion of survey respondents must be avoided under most circumstances, the impact of \"bots\" on preference estimates can be significant.

Purpose An essential aspect of patient-centered outcomes research (PCOR) and comparative effectiveness research (CER) is the integration of patient perspectives and experiences with clinical data to evaluate interventions. Thus, PCOR and CER require capturing patient-reported outcome (PRO) data appropriately to inform research, healthcare delivery, and policy. This initiative’s goal was to identify minimum standards for the design and selection of a PRO measure for use in PCOR and CER. Methods We performed a literature review to find existing guidelines for the selection of PRO measures. We also conducted an online survey of the International Society for Quality of Life Research (ISOQOL) membership to solicit input on PRO standards. A standard was designated as “recommended” when >50 % respondents endorsed it as “required as a minimum standard.” Results The literature review identified 387 articles. Survey response rate was 120 of 506 ISOQOL members. The respondents had an average of 15 years experience in PRO research, and 89 % felt competent or very competent providing feedback. Final recommendations for PRO measure standards included: documentation of the conceptual and measurement model; evidence for reliability, validity (content validity, construct validity, responsiveness); interpretability of scores; quality translation, and acceptable patient and investigator burden. Conclusion The development of these minimum measurement standards is intended to promote the appropriate use of PRO measures to inform PCOR and CER, which in turn can improve the effectiveness and efficiency of healthcare delivery. A next step is to expand these minimum standards to identify best practices for selecting decision-relevant PRO measures.

Purpose Patient-reported outcomes (PROs) are increasingly used in clinical trials to provide patients’ perspectives regarding symptoms, health-related quality of life, and satisfaction with treatments. A range of guidance documents exist for the selection of patient-reported outcome measures (PROMs) in clinical trials, and it is unclear to what extent these documents present consistent recommendations. Methods We conducted a targeted review of publications and regulatory guidance documents that advise on the selection of PROMs for use in clinical trials. A total of seven guidance documents from the US Food and Drug Administration, European Medicines Agency, and scientific consortia from professional societies were included in the final review. Guidance documents were analyzed using a content analysis approach comparing them with minimum standards recommended by the International Society for Quality of Life Research. Results Overall there was substantial agreement between guidance regarding the appropriate considerations for PROM selection within a clinical trial. Variations among the guidance primarily related to differences in their format and differences in the perspectives and mandates of their respective organizations. Whereas scientific consortia tended to produce checklist or rating-type guidance, regulatory groups tended to use more narrative-based approaches sometimes supplemented with lists of criteria. Conclusion The consistency in recommendations suggests an emerging consensus in the field and supports use of any of the major guidance documents available to guide PROM selection for clinical trials without concern of conflicting recommendations. This work represents an important first step in the international PROTEUS Consortium’s ongoing efforts to optimize the use of PROs in clinical trials.

Objective To assess minimally important differences (MIDs) for several pediatrie self-report item banks from the National Institutes of Health Patient-Reported Outcomes Measurement Information System® (PROMIS®). Methods We presented vignettes comprising sets of two completed PROMIS questionnaires and asked judges to declare whether the individual completing those questionnaires had an important change or not. We enrolled judges (including adolescents, parents, and clinicians) who responded to 24 vignettes (six for each domain of depression, pain interference, fatigue, and mobility). We used item response theory to model responses to the vignettes across different judges and estimated MID as the point at which 50 % of the judges would declare an important change. Results We enrolled 246 judges (78 adolescents, 85 parents, and 83 clinicians). The MID estimated with clinician data was about 2 points on the PROMIS T-score scale, and the MID estimated with adolescent and parent data was about 3 points on that same scale. Conclusions The MIDs enhance the value of PROMIS pediatric measures in clinical research studies to identify meaningful changes in health status over time.

Purpose Patients with cancer experience acute and chronic symptoms caused by their underlying disease or by the treatment. While numerous studies have examined the impact of various treatments on symptoms experienced by cancer patients, there are inconsistencies regarding the symptoms measured and reported in treatment trials. This article presents a systematic review of the research literature of the prevalence and severity of symptoms in patients undergoing cancer treatment. Methods A systematic search for studies of persons receiving active cancer treatment was performed with the search terms of “multiple symptoms” and “cancer” for studies involving patients over the age of 18 years and published in English during the years 2001 to 2011. Search outputs were reviewed independently by seven authors, resulting in the synthesis of 21 studies meeting criteria for generation of an Evidence Table reporting symptom prevalence and severity ratings. Results Data were extracted from 21 multi-national studies to develop a pooled sample of 4,067 cancer patients in whom the prevalence and severity of individual symptoms was reported. In total, the pooled sample across the 21 studies was comprised of 62 % female, with a mean age of 58 years (range 18 to 97 years). A majority (62 %) of these studies assessed symptoms in homogeneous samples with respect to tumor site (predominantly breast and lung cancer), while 38 % of the included studies utilized samples with mixed diagnoses and treatment regimens. Eighteen instruments and structured interviews were including those measuring single symptoms, multi-symptom inventories, and single symptom items drawn from HRQOL or health status measures. The MD Anderson Symptom Inventory was the most commonly used instrument in the studies analyzed ( n  = 9 studies; 43 %), while the Functional Assessment of Cancer Therapy, Hospital Anxiety and Depression Subscale, Medical Outcomes Survey Short Form-36, and Symptom Distress Scale were each employed in two studies. Forty-seven symptoms were identified across the 21 studies which were then categorized into 17 logical groupings. Symptom prevalence and severity were calculated across the entire cohort and also based upon sample sizes in which the symptoms were measured providing the ability to rank symptoms. Conclusions Symptoms are prevalent and severe among patients with cancer. Therefore, any clinical study seeking to evaluate the impact of treatment on patients should consider including measurement of symptoms. This study demonstrates that a discrete set of symptoms is common across cancer types. This set may serve as the basis for defining a “core” set of symptoms to be recommended for elicitation across cancer clinical trials, particularly among patients with advanced disease.

Objective Tolerability and safety of treatments are important in oncology trials and should be informed by patient assessments. We identified the most relevant patient‐reported symptomatic adverse events (AEs) to measure in patients with non‐small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations. Methods This study selected relevant symptomatic AEs from 78 AEs available in the Patient‐Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO‐CTCAE) measurement system. Initially, symptomatic AEs were selected based on literature and product labeling reviews, and then core sets of symptomatic AEs were identified by patient and clinician interviews. Qualitative and descriptive analyses were performed using the data collected from three iterative rounds of patient interviews. Results During concept elicitation interviews involving 29 patients, 12 symptomatic AEs were identified and were then adapted into a 25‐item PRO‐CTCAE form for use in future clinical trials along with commonly used PRO measures. Cognitive interviews showed that the PRO‐CTCAE items were easy to answer and appropriate for assessing the patients' experience with symptomatic AEs. This study also assessed disease symptoms, impacts, and overall patient experience. Conclusions The 25‐item PRO‐CTCAE form captures the most relevant symptomatic AEs in this patient population, and it is available for future studies. Baseline characterization of AEs associated with this distinct patient group contributes to our broader knowledge about NSCLC and through platforms like Project Patient Voice will expand our understanding of treatment tolerability and safety for NSCLC. Ultimately, this data collection will help inform decision‐making for patients, caregivers, healthcare providers, and regulators. This figure shows the multiple steps in the process of selecting, testing, and refining a set of PRO‐CTCAE items specific to patients with NSCLC EGFR exon20 insertion mutations. The inputs included literature review, clinical expert opinion, several rounds of patient input and testing, and label review.

The use of item libraries for patient-reported outcome (PRO) measurement in oncology allows for the customisation of PRO assessment to measure key health-related quality of life concepts of relevance to the target population and intervention. However, no high-level recommendations exist to guide users on the design and implementation of these customised PRO measures (item lists) across different PRO measurement systems. To address this issue, a working group was set up, including international stakeholders (academic, independent, industry, health technology assessment, regulatory, and patient advocacy), with the goal of creating recommendations for the use of item libraries in oncology trials. A scoping review was carried out to identify relevant publications and highlight any gaps. Stakeholders commented on the available guidance for each research question, proposed recommendations on how to address gaps in the literature, and came to an agreement using discussion-based methods. Nine primary research questions were identified that formed the scope and structure of the recommendations on how to select items and implement item lists created from item libraries. These recommendations address methods to drive item selection, plan the structure and analysis of item lists, and facilitate their use in conjunction with other measures. The findings resulted in high-level, instrument-agnostic recommendations on the use of item-library-derived item lists in oncology trials.

Objective Previous work in pediatric oncology has found that clinicians and parents tend to under-report the frequency and severity of treatment-related symptoms compared to child self-report. As such, there is a need to identify high-quality self-report instruments to be used in pediatrie oncology research studies. This study's objective was to conduct a systematic literature review of existing English language instruments used to measure self-reported symptoms in children and adolescents undergoing cancer treatment. Methods A comprehensive literature search was conducted in MEDLINE/PubMed, EMBASE, CINAHL, and PsycINFO to identify relevant articles published through November 10, 2016. Using pre-specified inclusion/exclusion criteria, six trained reviewers carefully screened abstracts and full-text articles for eligibility. Results There were 7738 non-duplicate articles identified in the literature search. Forty articles met our eligibility criteria, and within these articles, there were 38 self-report English symptom instruments. Most studies evaluated only cross-sectional psychometric properties, such as reliability or validity. Ten studies assessed an instrument's responsiveness or ability to detect changes in symptoms over time. Eight instruments met our criteria for use in future longitudinal pediatrie oncology studies. Conclusions This systematic review aids pediatrie oncology researchers in identifying and selecting appropriate symptom measures with strong psychometric evidence for their studies. Enhancing the child's voice in pediatrie oncology research studies allows us to better understand the impact of cancer and its treatment on the lives of children.

Background Advances in multiple myeloma treatment and a proliferation of treatment options have resulted in improved survival rates and periods of symptom-free remission for many multiple myeloma patients. As a result, health-related quality of life (HRQoL) concerns related to myeloma treatments have become increasingly salient for this patient population and represent an important consideration guiding patients’ treatment choices. To gain an understanding of patients’ experiences with choosing myeloma therapies and explore the HRQoL concerns that are most important to them, we interviewed a diverse sample of US-based multiple myeloma patients about their treatment considerations. Methods We conducted a qualitative descriptive study using in-depth interviews. Participants reflected on (1) the factors that were most important to them when thinking about multiple myeloma treatment and how these have changed over time, (2) how they might weigh the importance of treatment efficacy vs. side effects, (3) trade-offs they would be willing to make regarding efficacy vs. HRQoL, and (4) treatment changes they had experienced. Interviews were audio-recorded and transcribed, and narratives were analyzed using applied thematic analysis. Results We interviewed 21 patients, heterogeneous in their disease trajectory and treatment experience. Participants were 36 to 78 years, 52% female, and 38% Black. Efficacy was named as the most important treatment consideration by almost two-thirds of participants, and over half also valued HRQoL aspects such as the ability to maintain daily functioning and enjoyment of life. Participants expressed concern about potential treatment side effects and preferred more convenient treatment options. Although participants stated largely trusting their clinicians’ treatment recommendations, many said they would stop a clinician-recommended treatment if it negatively impacted their HRQoL. Participants also said that while they prioritized treatment efficacy, they would be willing to change to a less efficacious treatment if side effects became intolerable. Conclusions Our findings link to other reports reflecting considerations that are important to multiple myeloma patients, including the importance placed on increasing life expectancy and progression-free survival, but also the tension between treatment efficacy and quality of life. Our results extend these findings to a racially diverse US-based patient population at different stages in the disease trajectory.

Background Identification of exposures in patients with interstitial lung diseases (ILDs) is essential for diagnosis and management and can be facilitated through the use of exposure questionnaires. However, for most ILDs, a patient-focused questionnaire is lacking. Cognitive interviewing is a methodology used to evaluate sources of understanding and misunderstanding in a questionnaire and to provide evidence of content validity. We developed and refined a new exposure questionnaire for patients with fibrotic ILDs by using cognitive interviewing to establish its understandability and content validity. Methods An exposure assessment questionnaire was developed by a multidisciplinary team. Cognitive interviews with 24 patients with fibrosing ILDs were conducted by trained interviewers over the phone or Zoom using a semi-structured interview guide. The questionnaire was amended based on the interviewers’ interpretation of sources of misunderstanding. The revised questionnaire was tested in a second round of cognitive interviews with a different group of 24 patients. Results Among the 48 patients who completed interviews, mean age was 61 years, 58.3% were male and 75.0% were white. Based on the first round of cognitive interviews, the multidisciplinary team modified the questions, organization, and instructions of the questionnaire to facilitate recall, adjust for exposures that were frequently misunderstood or required clarification, and focus on clinically relevant exposures. The revised questionnaire performed well in the second round of interviews. Conclusion An exposure questionnaire, developed with input from patients, can be used to assess clinically relevant exposures in adults with fibrosing ILDs. This is the first questionnaire for all types of fibrosing ILD to have undergone content validation.