Disease burden of Barrett's esophagus across the Paris metropolitan area.

INTRODUCTION: The prevalence of Barrett's esophagus (BE) in France is unknown. However, the management of dysplastic BE in expert centers is recommended and reduces the risk of developing invasive adenocarcinoma. Our aim was to determine the burden of BE patients in the Paris Region. METHODS: We performed a retrospective study using the data from electronic medical records from the data warehouse of the 39 Greater Paris public hospitals (Entrepôt de données de santé de l' Assistance Publique- Hôpitaux de Paris) for the year 2018, and used natural language processing to search for occurrences of Barrett's esophagus in endoscopy and pathology reports. RESULTS: we observed a 2.2 % prevalence of Barrett's esophagus. Patients with Barrett's esophagus were older, more frequently males, with a hiatal hernia, proton pump inhibitor users, and less frequently infected by H. Pylori. Gastro-esophageal reflux symptoms were not more frequently encountered in Barrett's patients. Eleven percent of patients with Barrett's esophagus had dysplasia or adenocarcinoma. DISCUSSION: Over 200 000 patients with Barrett's esophagus are expected in the Paris Region, of which 11 % harbor dysplasia or adenocarcinoma. This data should be taken into account to tailor healthcare offer in France.

Assessment of US Food and Drug Administration-Approved Digital Medical Devices for Just-in-Time Interventions: A Systematic Review.

Importance: Just-in-time interventions (JITIs) are a type of digital therapeutic intervention that combines remote monitoring tools and algorithms to personalize the delivery of specific interventions at the right time. The US Food and Drug Administration (FDA) regulatory approval documents are often the only available source of information on the effectiveness of therapeutic interventions based on these devices. Objective: To systematically review the publicly available information from the FDA on all recently approved medical devices used in JITIs to (1) assess how they operate to deliver JITIs and (2) appraise the evidence supporting their performance and clinical effectiveness. Evidence Review: Two reviewers systematically searched the Premarket Notifications (510(k)), Premarket Approvals, De Novo, and Humanitarian Device Exemption databases from January 2019 to December 2021 for all entries associated with devices that monitored patients' data over time to personalize the delivery of interventions to treat, prevent, or mitigate health conditions or events. They assessed whether the product summaries (1) enabled an understanding of how the device operated to deliver a JITI (eg, the nature, type, and frequency of the monitoring, the nature of the decision algorithm, and the nature and intended receiver of the intervention); (2) informed about the performance and effectiveness of the JITI; and (3) included information on data security and ownership. Findings: In total, 38 devices were included in this review. These were mainly intended for cardiac conditions (12 [31.6%]), diabetes (10 [26.3%]), and neurological diseases (4 [10.5%]). Monitoring devices ranged from wearable (18 of 28 [64.4%]; eg, smartwatches) to implanted sensors (6 of 28 [21.4%]; eg, inserted electrocardiographic sensors). Only 10 of 38 product summaries (26.3%) allowed a full understanding of how the device operated to deliver a JITI. Similarly, only 12 of 28 (42.9%), 12 of 36 (33.3%), and 5 of 38 (13.2%) reported the assessment of the performance of the monitoring device, assessment of the decision algorithm, and results of clinical studies assessing the effectiveness of the JITI, respectively. Finally, 14 of 36 product summaries (38.9%) included some information on data security, but none included information on data ownership. Conclusion and Relevance: The results of this systematic review suggest that the information publicly available in the FDA databases on the performance and effectiveness of digital medical devices used in JITIs is heterogeneous.

Important Issues to Severe Trauma Survivors: A Qualitative Study.

OBJECTIVE: Identify issues that are important to severe trauma survivors up to 3 years after the trauma. BACKGROUND: Severe trauma is the first cause of disability-adjusted life years worldwide, yet most attention has focused on acute care and the impact on long-term health is poorly evaluated. METHOD: We conducted a large-scale qualitative study based on semi-structured phone interviews. Qualitative research methods involve the systematic collection, organization, and interpretation of conversations or textual data with patients to explore the meaning of a phenomenon experienced by individuals themselves. We randomly selected severe trauma survivors (abbreviated injury score ≥3 in at least 1 body region) who were receiving care in 6 urban academic level-I trauma centers in France between March 2015 and March 2018. We conducted double independent thematic analysis. Issues reported by patients were grouped into overarching domains by a panel of 5 experts in trauma care. Point of data saturation was estimated with a mathematical model. RESULTS: We included 340 participants from 3 months to 3 years after the trauma [median age: 41 years (Q1-Q3 24-54), median injury severity score: 17 (Q1-Q3 11-22)]. We identified 97 common issues that we grouped into 5 overarching domains: body and neurological issues (29 issues elicited by 277 participants), biographical disruption (23 issues, 210 participants), psychological and personality issues (21 issues, 147 participants), burden of treatment (14 issues, 145 participants), and altered relationships (10 issues, 87 participants). Time elapsed because the trauma, injury location, or in-hospital trauma severity did not affect the distribution of these domains across participants' answers. CONCLUSIONS: This qualitative study explored trauma survivors' experiences of the long-term effect of their injury and allowed for identifying a set of issues that they consider important, including dimensions that seem overlooked in trauma research. Our findings confirm that trauma is a chronic medical condition that demands new approaches to post-discharge and long-term care.

Is My Patient Overwhelmed?: Determining Thresholds for Acceptable Burden of Treatment Using Data From the ComPaRe e-Cohort.

OBJECTIVE: To estimate the highest burden of treatment beyond which patients with chronic conditions consider their current investments of time and effort in health care unsustainable. PATIENTS AND METHODS: We used data collected between January 1, 2017 and October 1, 2018 in the Community of Patients for Research (ComPaRe), an ongoing e-cohort of adult patients with chronic conditions in France. We matched participants' answers to the Treatment Burden Questionnaire (TBQ) and to a Yes/No anchor question: "Think about all the things you do to care for yourself. Do you think you could continue investing the same amount of time, energy, and money in your health care lifelong?" We defined the Patient Acceptable Symptom State (PASS) for the burden of treatment as the TBQ score below which 75% of patients reported an acceptable burden state. RESULTS: We analyzed data for 2413 patients (1781 [73.8%] women, 1248 [51.7%] multimorbid, median age: 48 (interquartile range, 36-59] years) enrolled in ComPaRe. Of these, 38% (917 of 2413) reported that they would be unable to continue the same investment of energy, time, and money in health care lifelong. The PASS for the burden of treatment was at 39% of the maximal score (ie, TBQ score = 59/150; 95% CI, 52-64) Using these results, clinicians can detect patients at risk for becoming overwhelmed by their medical care by identifying patients with TBQ scores of 59 or higher. CONCLUSION: About 40% of patients with chronic conditions report being unable to sustain current investments of energy, time, and money in health care lifelong. The PASS for treatment burden provides a practical yardstick to help clinicians and researchers interpret scores for burden of treatment.

Adaptation and validation of a Spanish version of the treatment burden questionnaire in patients with multiple sclerosis.

BACKGROUND: The Treatment Burden Questionnaire (TBQ) is a self-reported measure of the effect of treatment workload on patient wellbeing. We sought to validate the TBQ in Spanish and use it to estimate the burden of treatment in Argentinian patients with multiple sclerosis (MS). METHODS: The TBQ was forward-backward translated into Spanish. Two focus groups and 25 semi-structured interviews focused on wording and possible item exclusion. Validation was performed in 2 steps. First, 162 patients across a range of MS severity completed the questionnaire. Confirmatory factor analysis assessed the dimensional structure of the TBQ. Construct validity was assessed by studying correlations with fatigue and quality of life (QoL). Then, in a second cohort of 171 patients, we evaluated the association between TBQ scores and patients' sex, age, education level, employment status, type of MS, disease duration, comorbidities, EDSS, pharmacological treatment and medication adherence. RESULTS: The questionnaire presented a 3-factor structure in which burden was related to pharmacological treatment; comprehensive health assistance; and psycho-social-economic context. Composite reliability was > 0.8 for all factors. TBQ showed positive correlation with fatigue (rs = 0.467, p = 0.006), negative correlation with QoL (rs - 0.446, p = 0.009). For the second cohort, total TBQ score was 43 (SD 29). Lowest scores were observed on self-monitoring (0.53, SD 1.3) and highest for administrative load (4.2, SD 3.4). Inverse association was found between the TBQ score and medication adherence (r 0.243 p = 0.001). TBQ scores also correlated with daily patient pill/injection requirements (r 0.175 p = 0.020). Individuals receiving injectable treatment scored higher than patients on oral drugs (total TBQ 51 (SD 32) vs 39 (SD 27) p = 0.002). CONCLUSIONS: The TBQ in Spanish is a reliable instrument and showed adequate correlation with QoL and adherence scales in MS patients. TBQ may benefit health resources allocation and provide tailor therapeutic interventions to construct a minimally disruptive care.

Patients' perspectives on how to decrease the burden of treatment: a qualitative study of HIV care in sub-Saharan Africa.

OBJECTIVE: Patients living with HIV infection (PLWH) in sub-Saharan Africa face an important burden of treatment related to everything they do to take care of their health: doctor visits, tests, regular refills, travels, and so on. In this study, we involved PLWH in proposing ideas on how to decrease their burden of treatment and assessed to what extent these propositions could be implemented in care. METHODS: Adult PLWH recruited in three HIV care centres in Côte d'Ivoire participated in qualitative interviews starting with 'What do you believe are the most important things to change in your care to improve your burden of treatment?' Two independent investigators conducted a thematic analysis to identify and classify patients' propositions to decrease their burden of treatment. A group of experts involving patients, health professionals, hospital leaders and policymakers evaluated each patient proposition to assess its feasibility. RESULTS: Between February and April 2017, 326 participants shared 748 ideas to decrease their burden of treatment. These ideas were grouped into 59 unique patient propositions to improve their personal care and the organisation of their hospital or clinic and/or the health system. Experts considered that 27 (46%), 19 (32%) and 13 (22%) of patients' propositions were easy, moderate and difficult, respectively, to implement. A total of 118 (36%) participants offered at least one proposition considered easily implementable by our experts. CONCLUSION: Asking PLWH in sub-Saharan Africa about how their care could be improved led to identifying meaningful propositions. According to experts, half of the ideas identified could be implemented easily at low cost for minimally disruptive HIV care.

Acceptable medication non-adherence: A crowdsourcing study among French physicians for commonly prescribed medications.

BACKGROUND: Achieving good medication adherence is a major challenge for patients with chronic conditions. Our study aimed to assess the Threshold for Unacceptable Risk of Non-adherence (TURN), defined as the threshold at which physicians consider the health risks incurred by patients due to medication non-adherence unacceptable, for the most commonly prescribed drugs in France. METHODS: We conducted an online study using a crowdsourcing approach among French general practitioners and specialists from September 2016 to August 2017. Physicians assessed the TURN for various levels of missed doses by evaluating a series of randomly presented clinical vignettes, each presenting a given medication with a given therapeutic indication. For each "drug-indication group" (i.e., all drugs from the same pharmacological class with a similar therapeutic indication): 1) we described the distribution of physicians' assessments, 2) we provided a summary estimate of the TURN, defined as the frequency of missed doses above which 75% of the physicians' assessments were located; 3) we computed the number of pill boxes reimbursed in France in 2016 to put our results into context. RESULTS: We collected a total of 5365 assessments from 544 physicians, each of whom evaluated a random sample among 528 distinct clinical vignettes. Estimates of the TURN varied widely across drug-indication groups, ranging from risk considered unacceptable with 1 daily dose missed per month (e.g., insulin for diabetes) to risk always considered acceptable (e.g., anti-dementia drugs). Drugs with an estimated TURN of over one missing daily dose per week represented 44.9% of the prescription volume of the medications assessed in our study. CONCLUSIONS: According to physicians, the impact of non-adherence may vary greatly. Patient-physician discussions on the variable consequences of non-adherence could lead to a paradigm shift by seeking to reach "optimal adherence" depending on drugs rather than unrealistic "perfect adherence" to all drugs.

Point of data saturation was assessed using resampling methods in a survey with open-ended questions.

OBJECTIVE: To describe methods to determine sample sizes in surveys using open-ended questions and to assess how resampling methods can be used to determine data saturation in these surveys. STUDY DESIGN AND SETTING: We searched the literature for surveys with open-ended questions and assessed the methods used to determine sample size in 100 studies selected at random. Then, we used Monte Carlo simulations on data from a previous study on the burden of treatment to assess the probability of identifying new themes as a function of the number of patients recruited. RESULTS: In the literature, 85% of researchers used a convenience sample, with a median size of 167 participants (interquartile range [IQR] = 69-406). In our simulation study, the probability of identifying at least one new theme for the next included subject was 32%, 24%, and 12% after the inclusion of 30, 50, and 100 subjects, respectively. The inclusion of 150 participants at random resulted in the identification of 92% themes (IQR = 91-93%) identified in the original study. CONCLUSION: In our study, data saturation was most certainly reached for samples >150 participants. Our method may be used to determine when to continue the study to find new themes or stop because of futility.

Frugal innovation in medicine for low resource settings.

Whilst it is clear that technology is crucial to advance healthcare: innovation in medicine is not just about high-tech tools, new procedures or genome discoveries. In constrained environments, healthcare providers often create unexpected solutions to provide adequate healthcare to patients. These inexpensive but effective frugal innovations may be imperfect, but they have the power to ensure that health is within reach of everyone. Frugal innovations are not limited to low-resource settings: ingenuous ideas can be adapted to offer simpler and disruptive alternatives to usual care all around the world, representing the concept of "reverse innovation". In this article, we discuss the different types of frugal innovations, illustrated with examples from the literature, and argue for the need to give voice to this neglected type of innovation in medicine.

Taxonomy of the burden of treatment: a multi-country web-based qualitative study of patients with chronic conditions.

BACKGROUND: Management strategies for patients with chronic conditions are becoming increasingly complex, which may result in a burden of treatment for patients. To develop a Minimally Disruptive Medicine designed to reduce the burden of treatment, clinicians need to understand which healthcare tasks and aggravating factors may be responsible for this burden. The objective of the present study was to describe and classify the components of the burden of treatment for patients with chronic conditions from the patient's perspective. METHODS: We performed a multi-country qualitative study using an online survey and a purposive sampling strategy to select English-, French-, and Spanish-speaking participants with different chronic conditions. Participants were recruited by physicians, patients' associations, advertisement on social media, and 'snowballing'. The answers were analyzed by i) manual content analysis with a grounded theory approach, coded by two researchers, and ii) automatic textual analysis by Reinert's method. RESULTS: Between 2013 and 2014, 1,053 participants from 34 different countries completed the online survey using 408,625 words. Results from both analyses were synthesized in a taxonomy of the burden of treatment, which described i) the tasks imposed on patients by their diseases and by their healthcare system (e.g., medication management, lifestyle changes, follow-up, etc.); ii) the structural (e.g., access to healthcare resources, coordination between care providers), personal, situational, and financial factors that aggravated the burden of treatment; and iii) patient-reported consequences of the burden (e.g., poor adherence to treatments, financial burden, impact on professional, family, and social life, etc.). Our findings may not be applicable to patients with chronic conditions who differ from those who responded to our survey. CONCLUSIONS: Our taxonomy of the burden of treatment, provided by patients with chronic conditions from different countries and settings, supports the development of tools to ascertain the burden of treatment and highlights potential targets for interventions to minimize it.

Adaptation and validation of the Treatment Burden Questionnaire (TBQ) in English using an internet platform.

BACKGROUND: Treatment burden refers to the workload imposed by healthcare on patients, and the effect this has on quality of life. The Treatment Burden Questionnaire (TBQ) aims to assess treatment burden in different condition and treatment contexts. Here, we aimed to evaluate the validity and reliability of an English version of the TBQ, a scale that was originally developed in French. METHODS: The TBQ was translated into English by a forward-backward translation method. Wording and possible missing items were assessed during a pretest involving 200 patients with chronic conditions. Measurement properties of the instrument were assessed online with a patient network, using the PatientsLikeMe website. Dimensional structure of the questionnaire was assessed by factor analysis. Construct validity was assessed by associating TBQ global score with clinical variables, adherence to medication assessed by Morisky's Medication Adherence Scale (MMAS-8), quality of life (QOL) assessed by the PatientsLikeMe Quality of Life Scale (PLMQOL), and patients' confidence in their knowledge of their conditions and treatments. Reliability was determined by a test-retest method. RESULTS: In total, 610 patients with chronic conditions, mainly from the USA, UK, Canada, Australia, or New Zealand, completed the TBQ between September and October 2013. The English TBQ showed a unidimensional structure with Cronbach α of 0.90. The TBQ global score was negatively correlated with the PLMQOL score (rs = -0.50; p < 0.0001). Low rather than moderate or high adherence to medication was associated with high TBQ score (mean [SD] TBQ score 61.8 [30.5] vs. 37.7 [27.5]; P < 0.0001). The treatment burden was higher for patients who had insufficient knowledge compared with those who had sufficient knowledge about their treatments (mean ± SD TBQ score 62.3 ± 31.3 vs. 47.8 ± 30.4; P < 0.0001) and conditions (63.0 ± 31.6 vs. 49.3 ± 30.7; P < 0.0001). The intraclass correlation coefficient for the retest (n = 282) was 0.77 (95% CI 0.70 to 0.82). CONCLUSIONS: We found that the English TBQ is a reliable instrument in this population, and provide evidence supporting the construct validity for its use to assess treatment burden for patients with one or more chronic conditions in English-speaking countries.

Development and description of measurement properties of an instrument to assess treatment burden among patients with multiple chronic conditions.

BACKGROUND: Patients experience an increasing treatment burden related to everything they do to take care of their health: visits to the doctor, medical tests, treatment management and lifestyle changes. This treatment burden could affect treatment adherence, quality of life and outcomes. We aimed to develop and validate an instrument for measuring treatment burden for patients with multiple chronic conditions. METHODS: Items were derived from a literature review and qualitative semistructured interviews with patients. The instrument was then validated in a sample of patients with chronic conditions recruited in hospitals and general practitioner clinics in France. Factor analysis was used to examine the questionnaire structure. Construct validity was studied by the relationships between the instrument's global score, the Treatment Satisfaction Questionnaire for Medication (TSQM) scores and the complexity of treatment as assessed by patients and physicians. Agreement between patients and physicians was appraised. Reliability was determined by a test-retest method. RESULTS: A sample of 502 patients completed the Treatment Burden Questionnaire (TBQ), which consisted of 7 items (2 of which had 4 subitems) defined after 22 interviews with patients. The questionnaire showed a unidimensional structure. The Cronbach's α was 0.89. The instrument's global score was negatively correlated with TSQM scores (rs = -0.41 to -0.53) and positively correlated with the complexity of treatment (rs = 0.16 to 0.40). Agreement between patients and physicians (n = 396) was weak (intraclass correlation coefficient 0.38 (95% confidence interval 0.29 to 0.47)). Reliability of the retest (n = 211 patients) was 0.76 (0.67 to 0.83). CONCLUSIONS: This study provides the first valid and reliable instrument assessing the treatment burden for patients across any disease or treatment context. This instrument could help in the development of treatment strategies that are both efficient and acceptable for patients.