Patient Preference Information Research Articles

HTA community perspectives on the use of patient preference information: lessons learned from a survey with members of HTA bodies.

This research sought to assess whether and how patient preference (PP) data are currently used within health technology assessment (HTA) bodies and affiliated organizations involved in technology/drug appraisals and assessments. An exploratory survey was developed by the PP Project Subcommittee of the HTA International Patient and Citizen Involvement Interest Group to gain insight into the use, impact, and role of PP data in HTA, as well as the perceived barriers to its incorporation. Forty members of HTA bodies and affiliated organizations from twelve countries completed the online survey. PP data were reported to be formally considered as part of the HTA evidence review process by 82.5 percent of the respondents, while 39.4 percent reported that most of the appraisals and assessments within their organization in the past year had submitted PP data. The leading reason for why PP data were not submitted in most assessments was time/resource constraints followed by lack of clarity on PP data impact. Participants reported that PP data had a moderate level of influence on the deliberative process and outcome of the decision, but a higher level of influence on the decision's quality. Most (81.8 percent) felt patient advocacy groups should be primarily responsible for generating and submitting this type of evidence. Insights from the survey confirm the use of PP data in HTA but reveal barriers to its broader and more meaningful integration. Encouragingly, participants believe obstacles can be overcome, paving the way for a second phase of research involving in-depth collaborative workshops with HTA representatives.

Eliciting Exploratory Patient Preference Data: A Case Study in a Rare Disease.

Qualitative and quantitative methods provide different and complementary insights into patients' preferences for treatment. The aim of this study was to use a novel, mixed-methods approach employing qualitative and quantitative approaches to generate preliminary insights into patient preferences for the treatment of a rare disease-generalized myasthenia gravis (gMG). We conducted a mixed-methods study to collect exploratory qualitative and quantitative patient preference information and generate informative results within a condensed timeline (about 4 months). Recruitment was facilitated by an international health research firm. Study participants first reviewed a brief document describing six treatment attributes (to facilitate more efficient review of the material during the focus groups) and were then provided a link to complete an online quantitative survey with a single risk threshold task. They then participated in online focus groups, during which they discussed qualitative questions about their experience with gMG treatment and completed up to three quantitative threshold tasks, the first of which repeated the threshold task from the online survey. The study elicited both quantitative data on 18 participants' risk tolerance and qualitative data on their treatment experience, additional treatment attributes of importance, the reasoning behind their preferences, and the trade-offs they were willing to make. Most participants (n=15) chose the same hypothetical treatment in the first threshold task in the online survey and the focus groups. Focus group discussions provided insights into participants' choices in the threshold tasks, confirmed that all the attributes were relevant, and helped clarify what was important about the attributes. Patient preference information can be collected using a variety of approaches, both qualitative and quantitative, tailored to fit the research needs of a study. The novel mixed-methods approach employed in this study efficiently captured patient preference data that were informative for exploratory research, internal decision making, and future research.

Which body functions and activities matter for stroke patients? Study protocol for best–Worst scalings to value core elements of the International Classification of Functioning, Disability and Health

Background Stroke is a common, serious, and disabling healthcare problem with increasing incidence and prevalence. Following a stroke, identifying the factors associated with decisions about rehabilitation interventions is important to assess rehabilitation after stroke. The aim is to guide clinical staff to make patient-centered decisions. Fundamentally, decision makers cannot draw on evidence to consider the relevance of distinct functions and activities from the patient’s perspective. Until now, outcomes of rehabilitation are generally categorized using the International Classification of Functioning, Disability and Health (ICF). This can be seen as a conceptual basis for the assessment of health and disability. Since the ICF does not distinguish importance between these aspects there is a need to value the most important clinical factors as well as related activities from a patients and public perspective to help guide therapists in effectively designing post-acute rehabilitation care for individuals following stroke. The research question is which ICF body functions and activities are of value to stroke patients? Which trade-offs are patients willing to make within the core elements? Health preference research (HPR) answers the need to develop additional preference weights for certain ICF dimensions. Patient preference information (PPI) values health conditions based on the ICF from a patient perspective. Methods In this study we conduct three best–worst scaling (BWS) experiments to value body function and activities from patients’ and public perspective. Out of all ICF dimensions this research covers health conditions relevant to stroke patients in terms of body function, perception, and activities of daily living. Stroke patients as well as members of the general population will be recruited to participate in the online BWS surveys. Fractional, efficient designs are applied regarding the survey design. Conditional and multinominal logit analyses will be used as the main analysis method, with the best–worst count analysis as a secondary analysis. The survey is being piloted prior to commencing the process of data collection. Results are expected by the autumn of 2023. Discussion The research will add to the current literature on clinical decision-making in stroke rehabilitation and the value of certain body functions as well as related activities in neurorehabilitation. Moreover, the study will show whether body functions and activities that are currently equally weighted in international guidelines are also equally important from the point of view of those affected, or whether there are disconcordances in terms of differences between public judgements and patients’ preferences.

PP132 Health Technology Assessment Agencies’ Expectations Regarding Patient Experience Data in Australia, China, And Japan

IntroductionHealth technology assessment (HTA) agencies are increasingly embracing patient experience data (PED) to support reimbursement decisions. This study aimed to review HTA agencies’ expectations with regards to utilizing PED to support drug reimbursement in Australia, Japan, and China.MethodsPublished HTA guidance documents were reviewed in 2021 to identify any PED-specific information. If available, recommendations related to the type of PED (e.g., generic vs. disease-specific clinical outcomes assessment (COA)); COA validation, analyses, endpoints and interpretation; and the interest in PED beyond COA in HTA decision-making (e.g., patient preference information) were reviewed. Literature review and semi-structured interviews with key opinion leaders (KOLs) were conducted to further explore these themes and future trends with regards to PED.ResultsAustralia’s Pharmaceutical Benefits Advisory Committee guidance document includes a dedicated section on patient-reported outcomes (PRO), providing details on preferred PRO instruments; validation expectations; and recommended methods to explore score interpretation, assess and report PRO results and handle missing data. While PED derived from non-PRO sources are not discussed in the guidance, the KOL noted that they should not be ruled out. Japan’s Center for Outcomes Research and Economic Evaluation for Health guideline includes a section dedicated to PROs without details related to instrument validation, analyses and interpretation, however, is focused on the use of PRO to inform health economic assessments. In China, the HTA center of China National Health Commission drafted two disease-specific technical guidance documents recommending the inclusion of PROs in efficacy assessments and use of instruments relevant in the Chinese population; these points were echoed by the KOL interviewed.ConclusionsThere are recommendations on PED use included in country-specific guidance documents, however their level of detail varies greatly. Knowing each agency’s expectations with regards to PED is key when submitting HTA evidence dossiers and should be considered early in clinical trial design to integrate market access perspectives and optimize drug development.

PP131 Health Technology Assessment Agencies’ Expectations Regarding Patient Experience Data in Europe

IntroductionHealth technology assessment (HTA) agencies are increasingly embracing patient experience data (PED) to support reimbursement decisions. This study aimed to describe the European Network for HTA (EUnetHTA) and HTA agencies expectations regarding PED to support reimbursement in France, Germany, Italy, Spain and the UK.MethodsPublished HTA guidance documents were reviewed to identify recommendations related to clinical outcomes assessment (COA) (including disease-specificity, validation, analyses, endpoints and interpretation) and other forms of PED (e.g., patient preference information) in HTA decision-making. Insights from guidance documents were supplemented with a review of literature and published HTA cases and interviews with key opinion leaders (KOLs) focused on current and future states.ResultsThe German and French guidance documents include PED recommendations focused on relevant COA and health-related quality of life data, without detailing preferred COA measures. However, key differences were noted between these two countries in the methodological approaches regarding responder definitions, acceptable missing data threshold and multiplicity analyses. These differences were reinforced by the case studies and the KOLs. UK’s sources also focused on COA, in general proposing specific use of the EQ-5D to derive utility values for modelling, but included limited details on other PED-related elements. The Italian and Spanish guidance documents do not detail COA or other PED expectations, but the Italian KOL described that COA is considered if submitted. The currently developed EUnetHTA21 guidelines include PED-related information that bear the signature of certain individual HTA bodies. Globally, there is limited interest in PED beyond COA across the agencies.ConclusionsThe level of expectations with regards to PED varies across EUnetHTA and several European HTA agencies. Interest in PED derived from non-COA sources is limited across the countries. Knowing each agency’s expectations with regards to PED is key when submitting HTA evidence dossiers and should be considered early in clinical trial design to integrate market access perspectives and optimize drug development. Global harmonization would help advancing PED measurement standards.

Patient and Public Acceptance of Digital Technologies in Health Care: Protocol for a Discrete Choice Experiment.

Strokes pose a particular challenge to the health care system. Although stroke-related mortality has declined in recent decades, the absolute number of new strokes (incidence), stroke deaths, and survivors of stroke has increased. With the increasing need of neurorehabilitation and the decreasing number of professionals, innovations are needed to ensure adequate care. Digital technologies are increasingly used to meet patients' unfilled needs during their patient journey. Patients must adhere to unfamiliar digital technologies to engage in health interventions. Therefore, the acceptance of the benefits and burdens of digital technologies in health interventions is a key factor in implementing these innovations. This study aims to describe the development of a discrete choice experiment (DCE) to weigh criteria that impact patient and public acceptance. Secondary study objectives are a benefit-burden assessment (estimation of the maximum acceptable burden of technical features and therapy-related characteristics for the patient or individual, eg, no human contact), overall comparison (assessment of the relative importance of attributes for comparing digital technologies), and adherence (identification of key attributes that influence patient adherence). The exploratory objectives include heterogeneity assessment and subgroup analysis. The methodological aims are to investigate the use of DCE. To obtain information on the criteria impacting acceptance, a DCE will be conducted including 7 attributes based on formative qualitative research. Patients with stroke (experimental group) and the general population (control group) are surveyed. The final instrument includes 6 best-best choice tasks in partial design. The experimental design is a fractional-factorial efficient Bayesian design (D-error). A conditional logit regression model and mixed logistic regression models will be used for analysis. To consider the heterogeneity of subgroups, a latent class analysis and an analysis of heteroscedasticity will be performed. The literature review, qualitative preliminary study, survey development, and pretesting were completed. Data collection and analysis will be completed in the last quarter of 2023. Our results will inform decision makers about patients' and publics' acceptance of digital technologies used in innovative interventions. The patient preference information will improve decisions regarding the development, adoption, and pricing of innovative interventions. The behavioral changes in the choice of digital intervention alternatives are observable and can therefore be statistically analyzed. They can be translated into preferences, which define the value. This study will investigate the influences on the acceptance of digital interventions and thus support decisions and future research. DERR1-10.2196/46056.

PCR249 Advancing Equity, Diversity, Inclusion, and Belonging in Patient Centered Drug Development with Patient Preference Research

Regulators and pharmaceutical and medical device manufacturers acknowledge the role patient-preference research can play in patient-focused drug development (PFDD). The growing recognition of the value of equity, diversity, inclusion, and belonging (EDIB) initiatives has challenged stakeholders to identify opportunities to promote diverse healthcare perspectives from underserved populations. Applying EDIB principles to the collection, interpretation, and use of patient preference information (PPI) in PFDD can support more inclusive and equitable health outcomes. We present specific examples of how PPI can advance EDIB in PFDD, as well as challenges.

Technical points of human use experience of ethnic medicine

Ethnic medicine has a rich history of application. Because of the large number of ethnic groups, wide geographical distribution, and unique medical systems in China, the research on the human use experience(HUE) of ethnic medicine should combine the characteristics of ethnic medicine, be based on practical experience, and respect folk practice and tradition. The clinical positioning of ethnic medicine should consider three factors, i.e., population region, dominant diseases, and clinical demand. We should consider the development of traditional preparations that meet the needs of ethnic regions and encourage the development of new drugs that can be popularized and used nationwide for the dominant diseases of ethnic medicines. Attention should be paid to the problems such as a large number of customary articles or substitutes of ethnic medicinal materials, the phenomena of foreign bodies with the same name and different names for the same substance, the different standards of medicinal materials, and the poor processing standards. The name, processing method, source, medicinal parts, and dosage of ethnic medicinal materials or decoction pieces should be determined, and resources should be carefully evaluated to ensure the safety of medicinal resources and ecology. The preparation of ethnic medicine is mostly in the form of pills, powder, ointment, etc., with simple processing technology. The problems of low-quality stan-dards of some preparations, different prescriptions with the same name, and inconsistent processing technology should be overcome, and the process route and main process parameters should be clarified to lay the foundation for the subsequent empirical research on HUE. In the collection and analysis of the HUE data of ethnic medicine, the core guiding ideology of "patient-centered" should be established, and the experience data of patients should be collected. The problems of weak links existing in the inheritance of ethnic medicine should be solved, and flexible and diverse methods should be adopted. Meanwhile, on the premise of complying with the requirements of the principles of medical ethics, we should respect the religion, culture, and customs of ethnic areas to obtain the key HUE information of ethnic medicine. On the basis of the patient preference information and differences in regional disease epidemiology, population characteristics, and medical practice, whether the HUE conclusions of ethnic medicine can be extrapolated to patients outside the region is evaluated from the aspects of clinical benefits, risk tolerance, risk acceptance, etc. The HUE research on ethnic medicine is carried out in a clear way to guide the research and development of new ethnic medicines.

Cancer prevention in cancer predisposition syndromes: A protocol for testing the feasibility of building a hereditary cancer research registry and nurse navigator follow up model.

Monogenic, high penetrance syndromes, conferring an increased risk of malignancies in multiple organs, are important contributors to the hereditary burden of cancer. Early detection and risk reduction strategies in patients with a cancer predisposition syndrome can save their lives. However, despite evidence supporting the benefits of early detection and risk reduction strategies, most Canadian jurisdictions have not implemented programmatic follow up of these patients. In our study site in the province of Newfoundland and Labrador (NL), Canada, there is no centralized, provincial registry of high-risk individuals. There is no continuity or coordination of care providing cancer genetics expertise and no process to ensure that patients are referred to the appropriate specialists or risk management interventions. This paper describes a study protocol to test the feasibility of obtaining and analyzing patient risk management data, specifically patients affected by hereditary breast ovarian cancer syndrome (HBOC; BRCA 1 and BRCA 2 genes) and Lynch syndrome (LS; MLH1, MSH2, MSH6, and PMS2 genes). Through a retrospective cohort study, we will describe these patients' adherence to risk management guidelines and test its relationship to health outcomes, including cancer incidence and stage. Through a qualitative interviews, we will determine the priorities and preferences of patients with any inherited cancer mutation for a follow up navigation model of risk management. Study data will inform a subsequent funding application focused on creating and evaluating a research registry and follow up nurse navigation model. It is not currently known what proportion of cancer mutation carriers are receiving care according to guidelines. Data collected in this study will provide clinical uptake and health outcome information so gaps in care can be identified. Data will also provide patient preference information to inform ongoing and planned research with cancer mutation carriers.

PCR166 Online Elicitation of Personal Utility Functions (OPUF) – An Open, Modular Health Valuation Platform

Commonly used preference elicitation methods, such as TTO or DCE, usually require data from hundreds of participants. Conducting health valuation studies thus becomes time and resource intensive, and eliciting preferences from patient, alongside HTA, for example, is often deemed infeasible altogether. This severely limits the availability of relevant (patient) preference information to decision makers. Here, we report on the current state of the Online elicitation of Personal Utility Function (OPUF) method; a new approach for valuing health and well-being.

Development of a Patient Preference Survey for Wearable Kidney Replacement Therapy Devices.

Recent innovations have the potential to disrupt the current paradigm for kidney failure treatment. The US Food and Drug Administration is committed to incorporating valid scientific evidence about how patients weigh the benefits and risks of new devices into their decision making, but to date, premarket submission of patient preference information (PPI) has been limited for kidney devices. With input from stakeholders, we developed a survey intended to yield valid PPI, capturing how patients trade off the potential benefits and risks of wearable dialysis devices and in-center hemodialysis. We conducted concept elicitation interviews with individuals receiving dialysis to inform instrument content. After instrument drafting, we conducted two rounds of pretest interviews to evaluate survey face validity, comprehensibility, and perceived relevance. We pilot tested the survey with in-center hemodialysis patients to assess comprehensibility and usability further. Throughout, we used participant input to guide survey refinements. Thirty-six individuals receiving in-center or home dialysis participated in concept elicitation (N=20) and pretest (N=16) interviews. Participants identified reduced fatigue, lower treatment burden, and enhanced freedom as important benefits of a wearable device, and many expressed concerns about risks related to device disconnection-specifically bleeding and infection. We drafted a survey that included descriptions of the risks of serious bleeding and serious infection and an assessment of respondent willingness to wait for a safer device. Input from pretest interviewees led to various instrument modifications, including treatment descriptions, item wording, and risk-level explanations. Pilot testing of the updated survey among 24 in-center hemodialysis patients demonstrated acceptable survey comprehensibility and usability, although 50% of patients required some assistance. The final survey is a 54-item web-based instrument that will yield estimates of the maximal acceptable risk for the described wearable device and willingness to wait for wearable devices with lower risk.

Building Infrastructure to Exploit Evidence from Patient Preference Information (PPI) Studies: A Conceptual Blueprint

Patients are the most important actors in clinical research. Therefore, patient preference information (PPI) could support the decision-making process, being indisputable for research value, quality, and integrity. However, there is a lack of clear guidance or consensus on the search for preference studies. In this blueprint, an openly available and regularly updated patient preference management system for an integrated database (PPMSDB) that contains the minimal set of data sufficient to provide detailed information for each study (the so-called evidence tables in systematic reviews) and a high-level overview of the findings of a review (summary tables) is described. These tables could help determine which studies, if any, are eligible for quantitative synthesis. Finally, a web platform would provide a graphical and user-friendly interface. On the other hand, a set of APIs (application programming interfaces) would also be developed and provided. The PPMSDB, aims to collect preference measures, characteristics, and meta-data, and allow researchers to obtain a quick overview of a research field, use the latest evidence, and identify research gaps. In conjunction with proper statistical analysis of quantitative preference measures, these aspects can facilitate formal evidence-based decisions and adequate consideration when conducting a structured decision-making process. Our objective is to outline the conceptual infrastructure necessary to build and maintain a successful network that can monitor the currentness and validity of evidence.

Measuring the Differences of Public Health Service Facilities and Their Influencing Factors

The equitable distribution of public health facilities is a major concern of urban planners. Previous studies have explored the balance and fairness of various medical resource distributions using the accessibility of in-demand public medical service facilities while ignoring the differences in the supply of public medical service facilities. First aid data with location information and patient preference information can reflect the ability of each hospital and the health inequities in cities. Determining which factors affect the measured differences in public medical service facilities and how to alter these factors will help researchers formulate targeted policies to solve the current resource-balance situation of the Ministry of Public Health. In this study, we propose a method to measure the differences in influence among hospitals based on actual medical behavior and use geographically weighted regression (GWR) to analyze the spatial correlations among the location, medical equipment, medical ability, and influencing factors of each hospital. The results show that Wuhan presents obvious health inequality, with the high-grade hospitals having spatial agglomeration in the city-center area, while the number and quality of hospitals in the peripheral areas are lower than those in the central area; thus, the hospitals in these peripheral areas need to be further improved. The method used in this study can measure differences in the influence of public medical service facilities, and the results are consistent with the measured differences at hospital level. Hospital influence is not only related to the equipment and medical ability of each hospital but is also affected by location factors. This method illustrates the necessity of conducting more empirical research on the public medical service supply to provide a scientific basis for formulating targeted policies from a new perspective.

MP23-05 ASSESSMENT OF PATIENT PERSPECTIVE ON BENEFIT AND RISK (B-R) ASSOCIATED WITH HIGH INTENSITY FOCUSED ULTRASOUND (HIFU) FOR THE ABLATION OF PROSTATE TISSUE IN MEN WITH LOCALIZED PROSTATE CANCER

You have accessJournal of UrologyHealth Services Research: Practice Patterns, Quality of Life and Shared Decision Making III (MP23)1 Sep 2021MP23-05 ASSESSMENT OF PATIENT PERSPECTIVE ON BENEFIT AND RISK (B-R) ASSOCIATED WITH HIGH INTENSITY FOCUSED ULTRASOUND (HIFU) FOR THE ABLATION OF PROSTATE TISSUE IN MEN WITH LOCALIZED PROSTATE CANCER Olufemi Babalola, Geoffrey Sonn, Minhaj Siddiqui, and Charles Viviano Olufemi BabalolaOlufemi Babalola More articles by this author , Geoffrey SonnGeoffrey Sonn More articles by this author , Minhaj SiddiquiMinhaj Siddiqui More articles by this author , and Charles VivianoCharles Viviano More articles by this author View All Author Informationhttps://doi.org/10.1097/JU.0000000000002014.05AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: The FDA cleared HIFU for prostate tissue ablation in 2015. This study aims to explore patient preferences regarding the known benefit and important adverse events associated with HIFU for prostate ablation in men with prostate cancer. METHODS: Eligible subjects were treatment-naïve men with a physician confirmed diagnosis of localized prostate cancer (Gleason 6 or 7). A Discrete Choice Experiment (DCE) was used to evaluate patients’ B-R tradeoffs for prostate HIFU outcomes via an online survey. The outcomes of interest were: erectile dysfunction (ED), urinary incontinence (UI), and biopsy result after treatment. Content was based on a literature review, as well as clinical and regulatory knowledge, of HIFU for prostate ablation. Subjects were asked to choose a preferred treatment option in 9 different choice tasks. Each choice task includes a pair of hypothetical treatment profiles that have similar attributes (outcomes) with varying levels (see figure). RESULTS: 165 subjects with a mean age of 65 completed the survey. Preliminary results show patients are willing to trade off among treatment outcomes. The maximum acceptable risk (MAR) indicates that respondents are willing to accept a 1.59%-point increase in ED for a 1%-point increase in favorable biopsy. The minimum acceptable benefit (MAB) indicates that patients are willing to accept a 0.63%-point increase in favorable biopsy for a 1%-point increase in ED associated with HIFU. In addition, the MAR for UI shows that respondents are willing to accept a 1.86%-point increase in UI for a 1%-point increase in favorable biopsy. The MAB indicates that patients are willing to accept a 0.54%-point increase in favorable biopsy for a 1%-point increase in UI. CONCLUSIONS: Incorporation of patient preference information into device regulatory decision making is one of the strategic goals of the Center for Devices and Radiological Health at the FDA. Our results may help inform design and regulatory decision making for current and future prostate tissue ablation devices with limited benefit data by providing information from the patient perspective. Source of Funding: This project was supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award (U01FD005946; U01FD005978) totaling $146,080 with 100 percent funding by FDA/HHS. The contents are those of the authors and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government © 2021 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 206Issue Supplement 3September 2021Page: e402-e402 Advertisement Copyright & Permissions© 2021 by American Urological Association Education and Research, Inc.MetricsAuthor Information Olufemi Babalola More articles by this author Geoffrey Sonn More articles by this author Minhaj Siddiqui More articles by this author Charles Viviano More articles by this author Expand All Advertisement Loading ...

Stratified psoriasis treatment plans: why is patient preference information needed?

Linked Article: Dalal et al. Br J Dermatol 2021; 185:978–987.

Assessing Patient Preferences in Rare Diseases: Direct Preference Elicitation in the Rare Chronic Kidney Disease, Immunoglobulin A Nephropathy.

BackgroundPatient preference information is increasingly being used to inform decision making; however, further work is required to support the collection of preference information in rare diseases. This study illustrates the use of direct preference elicitation methods to collect preference data from small samples in the context of early decision making to inform the development of a product for the treatment of immunoglobulin A nephropathy.MethodAn interview-based swing weighting approach was used to elicit preferences from 40 patients in the US and China. Attributes were identified through a background review, expert engagement and patient focus groups. Participants completed a series of tasks that involved ranking, rating and scoring improvements in the attributes to obtain attribute swing weights and partial value functions. The preference results were then incorporated into a benefit-risk assessment simulation tool.ResultsParticipants placed the greatest value on avoiding end-stage renal/kidney disease. Similar weight was given to short-term quality-of-life improvements and avoiding infections. Treatment burden (number of vaccinations) received the least weight. Heterogeneity in preferences was also observed. Consistency tests did not identify statistically significant variation in preferences, and qualitative data suggested that the elicitation exercise was sensitive to participants’ interpretation of attributes and that participants were able to express their preferences.ConclusionDirect preference elicitation methods can be used to collect preference data from small samples. Further work should continue to test the validity of the estimate generated by such methods.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40271-021-00521-3.

Legitimization and Incorporation of Patient Preferences: The Arrow that Hit the Achilles Heel of Status Quo Kidney Care.

We hope that at the point that readers see this CJASN article, they will have already read the three accompanying CJASN articles on patient preference information and patient-centered innovation in KRT ([1][1][⇓][2]–[3][3]). These four articles represent a combined effort to chronicle the

Using Patient Preference Information to Inform Regulatory Decision Making: An Opportunity to Spur Patient-Centered Innovation in Kidney Replacement Therapy Devices.

Medical devices play an essential role in the health care system, often facilitating patients’ management of their own health. Such devices—items used for the “diagnosis…cure, mitigation, treatment, or prevention of disease that are not absorbed or metabolized by the body” ([1][1])—range

Patient Preferences in Rare Diseases: A Qualitative Study in Neuromuscular Disorders to Inform a Quantitative Preference Study.

IntroductionIt has become increasingly important to include patient preference information in decision-making processes for drug development. As neuromuscular disorders represent multisystem, debilitating, and progressive rare diseases with few treatment options, this study aimed to explore unmet health care needs and patient treatment preferences for two neuromuscular disorders, myotonic dystrophy type 1 (DM1) and mitochondrial myopathies (MM) to inform early stages of drug development.MethodsFifteen semi-structured interviews and five focus group discussions (FGDs) were held with DM1 and MM adult patients and caregivers. Topics discussed included (1) reasons for study participation; (2) disease signs/symptoms and their impact on daily lives; (3) top desired benefits; and (4) acceptability of risks and tolerance levels for a hypothetical new treatment. Data were analyzed following a thematic ‘code’ approach.ResultsA total of 52 participants representing a wide range of disease severities participated. ‘Muscle strength’ and ‘energy and endurance’ were the disease-related unmet needs most often mentioned. Additionally, improved ‘balance’, ‘cognition’ and ‘gut function’ were the top desired treatment benefits, while ‘damage to the liver, kidneys or eyes’ was the most concerning risk. Factors influencing their tolerance to risks related to previously having experienced the risk and differentiation between permanent and temporary risks. A few differences were elicited between patients and caregivers.ConclusionsThis qualitative study provided an open forum to elicit treatment-desired benefits and acceptable risks to be established by patients themselves. These findings can inform decisions for developing new treatments and the design of clinical trials for DM1 and MM.Electronic supplementary materialThe online version of this article (10.1007/s40271-020-00482-z) contains supplementary material, which is available to authorized users.

Treatment Satisfaction, Patient Preferences, and the Impact of Suboptimal Disease Control in a Large International Rheumatoid Arthritis Cohort: SENSE Study.

BackgroundPatients’ needs and perspectives are important determinants of treatment success in rheumatoid arthritis (RA). Assessing patients’ perspectives can help identify unmet needs and enhance the understanding of treatment benefits.ObjectiveThe SENSE study assessed the impact of inadequate response to disease-modifying antirheumatic drugs (DMARDs) on treatment satisfaction, disease outcomes, and patient perspectives related to RA disease management.MethodsSENSE was a noninterventional, cross-sectional study conducted in 18 countries across Europe, Asia, and South America. Adult patients with poorly controlled RA of moderate/high disease activity were eligible. Patient satisfaction was assessed by the Treatment Satisfaction Questionnaire for Medication (TSQM v1.4). Treatment adherence, healthcare resource utilization (HRU), quality of life (QoL), work ability, digital health literacy (DHL), patient preference information, and treatment strategy were also assessed.ResultsA total of 1624 patients were included in the study: most were female (84.2%) and middle-aged, and mean disease duration was 10.5 years. Mean TSQM global satisfaction subscore was 60.9, with only 13.5% of patients reporting good treatment satisfaction (TSQM global ≥80). The strongest predictor of good treatment satisfaction was treatment with advanced therapies. Most patients (87.4%) reported good treatment adherence. In general, patients had impaired QoL and work ability, high HRU, and 67.4% had poor DHL. Leading treatment expectations were “general improvement of arthritis” and “less joint pain”. Most patients preferred oral RA medications (60.7%) and rapid (≤1 week) onset of action (71.1%). “Increased risk for malignancies” and “increased risk for cardiovascular disease” were the least acceptable side effects. Despite suboptimal control, advanced therapies were only used in a minority of patients, and DMARD switches were planned for only half of the patients.ConclusionSuboptimal disease control negatively impacts treatment satisfaction, work ability, QoL, and HRU. Data collected on patient perspectives may inform shared decision-making and optimize treat-to-target strategies for improving patient outcomes in RA.