Patient-Patient Centered Outcomes Research最新文献

Background: Emerging blood-based multi-cancer early detection (MCED) tests can detect a variety of cancer types across stages with a range of sensitivity, specificity, and ability to predict the origin of the cancer signal. However, little is known about the general US population's preferences for MCED tests.

Objective: To quantify preferences for MCED tests among US adults aged 50-80 years using a discrete choice experiment (DCE).

Methods: To quantify preferences for attributes of blood-based MCED tests, an online DCE was conducted with five attributes (true positives, false negatives, false positives, likelihood of the cancer type unknown, number of cancer types detected), among the US population aged 50-80 years recruited via online panels and social media. Data were analyzed using latent class multinomial logit models and relative attribute importance was obtained.

Results: Participants (N = 1700) were 54% female, mean age 63.3 years. Latent class modeling identified three classes with distinct preferences for MCED tests. The rank order of attribute importance based on relative attribute importance varied by latent class, but across all latent classes, participants preferred higher accuracy (fewer false negatives and false positives, more true positives) and screenings that detected more cancer types and had a lower likelihood of cancer type unknown. Overall, 72% of participants preferred to receive an MCED test in addition to currently recommended cancer screenings.

Conclusions: While there is significant heterogeneity in cancer screening preferences, the majority of participants preferred MCED screening and the accuracy of these tests is important. While the majority of participants preferred adding an MCED test to complement current cancer screenings, the latent class analyses identified a small (16%) and specific subset of individuals who value attributes differently, with particular concern regarding false-negative and false-positive test results, who are significantly less likely to opt-in.

Background: Patient-oriented research involves extensive collaboration with patients, their families, caregivers, clinicians and other relevant stakeholders to identify and investigate problems and outcomes relevant to patients. Patient-oriented research can help develop effective patient-centred interventions. Patient-oriented research is an increasingly used approach in high-income countries, but it is unclear how patients are engaged in research in low-income and middle-income countries (LMICs).

Objectives: The aim of this scoping review was to explore how patient-oriented research is conducted in LMICs. The objectives were to determine the levels of involvement of patients in the research, how studies have impacted healthcare and patient outcomes in these countries, the reported benefits of patient-oriented research on the research process and the reported challenges of conducting patient-oriented research in LMICs.

Methods: A scoping review was conducted using the methodological framework suggested by Arksey and O'Malley and the Joanna Briggs Institute guidelines for conducting scoping reviews. The eligibility criteria were any healthcare research using any research design that involved patients of any age group in the research process. Six databases were searched from their inception till January 4, 2022: MEDLINE, Embase, Cumulated Index to Nursing and Allied Health Literature, PsychInfo, Cochrane Central Register of Controlled Trials and EBM Reviews. The reference lists of relevant articles and Google Scholar were combed as well. Data extraction was performed with a self-developed data extraction guide. The findings were narratively summarised.

Results: Thirteen articles were included in this scoping review, representing eight LMICs in Africa and Asia. The majority of studies (38%, n = 5) focused on patients living with human immunodeficiency virus. More than half of the studies (n = 8, 62%) were conducted in the adult population, 31% (n = 4) of the studies involved children and/or adolescents. For most of the studies (92%, n = 12), the participants served as consultants; for one study, the authors identified the participants as co-researchers. Across the studies, information regarding patient-oriented research activities was not consistently reported in the same manner (i.e. different locations in the article), with very limited information in some cases. None of the studies used a patient-oriented research framework and the majority did not report on how patient-oriented research impacted healthcare and patient outcomes. Patient-oriented research was beneficial in identifying relevant patients' needs and improving collaboration among stakeholders, but it also led to extended research timelines and increased financial costs for the researchers and patients.

Conclusions: Researchers in LMICs are incorporating patient-oriented res

Background: Plain language summaries (PLSs) are intended for a non-expert audience in order to make health research accessible and understandable to the public. This is important because most research is written with jargon and at a high reading level. However, there is a high degree of variability in the instructions for writing PLSs, which may impede their usefulness as a tool for communicating health research to the public.

Objective: The aim of this scoping review was to conduct a detailed analysis of the author instructions for PLSs provided by leading biomedical and health journals.

Method: We screened 534 health journals covering 11 categories selected from the InCites Journal Citation Reports linked to the top 10 non-communicable diseases. We included journals published in English that recommended the inclusion of a PLS (as defined by the National Institute for Health Research) and provided authors with text-based instructions on how it should be written. Two independent reviewers extracted data pertaining to common elements identified in author instructions, such as word count/PLS length, content, structure, purpose, wording to support plain language, and the use of jargon, acronyms and abbreviations. Other aspects of PLSs were recorded, such as the label used (e.g., plain language summary, lay summary, and patient summary), journal publisher, consumer involvement and whether the PLS is optional or mandatory. We recorded the frequency of each element and qualitative details of specific instructions. A consumer representative provided ongoing and iterative feedback on the methods, results, and reporting of this study RESULTS: Despite reviewing 534 journals across 10 non-communicable disease areas and 11 journal categories, we found only 27 (5.1%) contained text-based instructions for PLS. Of the 27 journals included in this review, most (70%) did not require a PLS. Approximately 70% of journals with PLS instructions included advice about the use of jargon, abbreviations, and acronyms. Only one journal recommended the use of a readability tool, however five noted that the reading level of the audience or readability of the PLS should be considered. Author instructions were highly heterogeneous between journals. There was inconsistency regarding the word count/PLS length (e.g., between 100 and 850 words), structure (e.g., paragraphs or bullet points), and varying levels of detail for other elements in the instructions. Although only one journal recommended consumer involvement in the development of PLSs, many recommended authors consult those who are not an expert in their field to review their summary prior to submission.

Conclusion: The development of consistent author instructions could enhance the effectiveness and use of PLSs. Such instructions should be developed with consumers to ensure they met the needs of a lay non-expert audience.

Background: Sexual violence (SV) is prevalent among US college athletes, but formal reports are rare. Little is known about adaptations to institution-level reporting policies and procedures that could facilitate reporting.

Methods: We conducted a discrete choice experiment (DCE) survey with 1004 student-athletes at ten Division I NCAA member institutions to examine how attributes of the reporting system influence the decision to formally report SV to their institution. Changes in utility values were estimated using multinomial logistic regression and mixed multinomial logistic regression. Importance scores were compared to understand student-athlete preferences.

Results: In order of relative importance, the two attributes most preferred by student-athletes were higher probabilities of students perpetrating SV being found in violation of code of conduct policies (relative importance score = 33), and the availability of substance use amnesty policies (relative importance score = 24). Student-athletes with prior SV experiences were more likely to opt out of formally reporting in the DCE paired choice, had lower estimated utility values for all attributes, and had less between-person heterogeneity. While anonymous reporting and survivor-initiated investigations were preferred by student-athletes on average, there was considerable valuation heterogeneity between student-athletes (sizeable deviations from mean estimated utilities). These two attributes also varied in relative importance; anonymous reporting had higher relative importance after interacting levels with prior SV experiences and competitive status, but lower relative importance after interacting levels with whether a student-athlete played on men's or women's sports teams.

Conclusions: Changes to reporting policies and procedures (e.g., transparency about SV reporting outcomes, implementing substance use amnesty policies) may be promising institution-level interventions to increase formal reporting of SV among student-athletes. More research is needed to understand preference heterogeneity between students and generalize these findings to broader student populations.

Rare diseases are often severe, debilitating, life-limiting conditions, many of which occur in childhood. These complex conditions have a wide range of clinical manifestations that have a substantial impact on the lives of patients, carers and families and often produce heterogeneous clinical outcomes. Therefore, the evaluation of quality-of-life (QoL) impacts is important. In health technology assessment (HTA), patient-reported outcome measures (PROMs) and/or health state utility values (HSUVs) are used to determine QoL impacts of new treatments, but their use in rare diseases is challenging due to small and heterogeneous populations and limited disease knowledge. This paper describes challenges associated with the use of patient-reported outcomes (PROs)/HSUVs to evaluate QoL in HTA of rare disease treatments (RDTs) and identifies five recommendations to ensure appropriate interpretation of QoL impacts. These were derived from mixed methods research (literature reviews, appraisal document analyses, appraisal committee observations and interviews) examining the use of PROs/HSUVs in HTA of RDTs. They highlight that HTAs of RDTs must (1) understand the QoL impacts of the disease and of treatments; (2) critically assess PRO data, recognising the nuances in development and administration of PROMs/HSUVs, considering what is feasible and what matters most to the patient population; (3) recognise that lack of significant effect on a PRO does not imply no QoL benefit; (4) use different forms of evidence to understand QoL impacts, such as patient input; and (5) provide methodological guidance to capture QoL impacts on patients/carers.

Objectives: The goals of this formative research are to elicit attributes of treatment and desired outcomes that are important to individuals with major depressive disorder (MDD), to develop a stated preference instrument, and to pre-test the instrument.

Methods: A three-phase survey study design elicited the patient's journey with MDD to design and pre-test the discrete choice experiment (DCE) instrument. Participants were 20 adults aged ≥ 18 with MDD who did not also have bipolar disorder or post-partum depression. We engaged patient advocates and a multi-disciplinary stakeholder advisory group to select and refine attributes for inclusion in a DCE instrument. The DCE was incorporated into a survey that also collected depression treatment and management and sociodemographic characteristics. The DCE was pre-tested with ten adults with MDD.

Results: Six attributes were included in the DCE: mode of treatment (medicine only, psychotherapy only, all modalities including brain stimulation), time to treatment effect (6, 9, 12 weeks), days of hopefulness (2, 4, 6 days/week), effect on productivity (40%, 60%, 90% increase), relations with others (strained, improved, no impact), and out-of-pocket costs ($30, $60, $90/month). The DCE test led to the refinement of mode of treatment (medicine, medicine and psychotherapy, and all modalities); time to treatment effect (4, 6, 9 weeks); monthly out-of-pocket costs ($30, $90, $270).

Conclusions: MDD treatment preferences revealed trade-offs among mode of treatment, time to treatment effect, functional outcomes, and cost. The findings demonstrate the potential for meaningfully incorporating the patient experience in preference measures.

Background and objective: Patient support programs aim to provide solutions beyond the medication itself, by enhancing treatment adherence, improving clinical outcomes, elevating patient experience, and/or increasing quality of life. As patient support programs increasingly play an important role in assisting patients, numerous observational studies and pragmatic trials designed to evaluate their impact on healthcare have been conducted in recent years. This review aims to characterize these studies.

Methods: A systematic literature review, supplemented by a broad search of gray literature, was conducted following PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) and Cochrane recommendations. Observational studies and pragmatic trials conducted in Europe to evaluate the impact of patient support programs, published in English or Spanish between 17/03/2010 and 17/03/2020, were reviewed. Two patient support program definitions were applied starting with Ganguli et al.'s broad approach, followed by the European Medicines Agency definition, narrowed to Marketing Authorization Holders organized systems and their medicines. The quality of publications was assessed using the STROBE (Strengthening the Reporting of Observational Studies in Epidemiology) statement 22-item checklist.

Results: Of the 49 identified studies following the Ganguli et al. definition, 20 studies met the European Medicines Agency definition and were reviewed. Patient support program impact was evaluated based on a wide range of methodologies: 70% assessed patient support program-related patient-reported outcomes, 55% reported clinical outcomes, and 25% reported economic impacts on health resources. Only 45% conducted a comparative analysis. Overall, 75% of the studies achieved their proposed objectives.

Conclusions: The heterogeneity of the observational studies reviewed reflects the complexity of patient support programs that are built ad hoc for specific diseases, treatments, and patients. Results suggest that patient support programs play a key role in promoting treatment effectiveness, clinical outcomes, and satisfaction. However, there is a need for standardizing the definition of patient support programs and the methods to evaluate their impact.