Patient-centered Decision Research Articles

Abstract OT1-15-01: SWOG 1904: Cluster-randomized controlled trial of patient and provider decision support to increase chemoprevention informed choice among women with atypical hyperplasia or lobular carcinoma in situ (MiCHOICE)

Abstract Background: Despite evidence of substantial breast cancer risk reduction, few high-risk women adopt chemopreventive medications such as selective estrogen receptor modulators (SERMs) or aromatase inhibitors (AIs). Women with benign breast disease, such as atypical hyperplasia (AH) or lobular carcinoma in situ (LCIS), have an increased risk of developing breast cancer and derive a greater benefit from antiestrogens compared to other high-risk women. Reasons for low uptake of chemoprevention include insufficient patient and clinician knowledge about antiestrogens, time constraints during the clinical encounter, and concerns about side effects. To address these barriers, we have developed patient and provider web-based decision support tools to improve informed choice about breast cancer chemoprevention among women with AH or LCIS. Study design: We are conducting a cluster-randomized controlled trial of clinical decision support to improve chemoprevention informed choice among women with AH or LCIS and their treating providers. Twenty-six U.S. sites through the SWOG Cancer Research Network were randomly assigned 1:1 to standard educational materials alone or in combination with the patient-centered decision aid (RealRisks) and provider decision support tool (BNAV). A total of 415 patients and 200 healthcare providers will be recruited from these sites. RealRisks consists of interactive modules to calculate personalized breast cancer risk and elicit preferences on chemoprevention. The modules are available in English and Spanish. BNAV is comprised of self-directed case-based learning modules on breast cancer risk assessment and chemoprevention. Patients complete questionnaires at baseline, 6 and 12 months. Providers complete surveys at baseline and after their enrolled patient’s 6-month clinical encounter. The primary endpoint is chemoprevention informed choice at 6 months, using a measure combining knowledge, attitude, and intention scales. Secondary endpoints include perceived breast cancer risk/worry, chemoprevention knowledge/intention, decision conflict/regret, shared decision-making, and chemoprevention uptake. For patients who begin chemoprevention, adherence and reasons for discontinuation are assessed annually for up to 5 years. Barriers and facilitators to implementing RealRisks and BNAV into clinic workflow will be assessed by conducting patient and provider interviews at baseline and mid-implementation. Eligibility criteria: Eligible patients include women, age 35-74 years, with AH or LCIS, no history of breast cancer, no prior use of SERMs or AIs, no bilateral mastectomies, English or Spanish-speaking, and access to the internet. Eligible providers include breast surgeons, medical oncologists, primary care providers, and physician extenders who see patients with AH or LCIS. Statistical methods: We have 90% power to detect a 15% increase in the frequency of chemoprevention informed choice with a 1-sided 0.025 level test, assuming an intraclass correlation (ICC) of 0.02 to account for clustering, roughly equal accrual at each site, 10% loss to follow-up, and ≤10% event rate in the control arm. Current/target accrual: The trial was activated on 9/1/2020. As of 7/7/2022, all 26 sites have been randomized, 157/200 providers and 184/415 patients have been enrolled. Discussion: Our hybrid effectiveness/implementation study seeks to evaluate the effectiveness of a multi-level intervention in promoting informed decision-making about breast cancer chemoprevention. Study results will provide valuable insights on how the decision support tools are integrated in diverse clinical settings. Citation Format: Katherine D. Crew, Garnet Anderson, Kathryn Arnold, Andrew Stieb, Jacquelyn N. Amenta, Cynthia Law, Ana Sandoval-Leon, Sarah Colonna, Tari King, Debra Mangino, Sandhya Pruthi, Maria Grosse Perdekamp, Christa Braun-Inglis, Stacy Krisher, Lisa Yee, Danielle Bertoni, Samantha Seaward, Kari B. Wisinski, Justin Floyd, Corrine Zarwan, Tarah J. Ballinger, Lindi VanderWalde, Masey M. Ross, Preston Steen, Shelly Lo, Alison Conlin, Kathleen Yost, John Ellerton, Erin Lin, Holly J. Pederson, Sagar Sardesai, Cheryl Jernigan, Dawn Hershman, Marian L. Neuhouser, Banu K. Arun, Rita Kukafka. SWOG 1904: Cluster-randomized controlled trial of patient and provider decision support to increase chemoprevention informed choice among women with atypical hyperplasia or lobular carcinoma in situ (MiCHOICE) [abstract]. In: Proceedings of the 2022 San Antonio Breast Cancer Symposium; 2022 Dec 6-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2023;83(5 Suppl):Abstract nr OT1-15-01.

The Role of Nurse on the Treatment Decision Support for Older People with Cancer: A Systematic Review.

Background: The number of older adults with cancer is increasing worldwide. The role of nurses in supporting patients' decision-making is expanding, as this process is fraught with complexity and uncertainty due to comorbidities, frailty, cognitive decline, etc., in older adults with cancer. The aim of this review was to examine the contemporary roles of oncology nurses in the treatment decision-making process in older adults with cancer. Methods: A systematic review of PubMed, CINAHL, and PsycINFO databases was conducted in accordance with PRISMA guidelines. Results: Of the 3029 articles screened, 56 full texts were assessed for eligibility, and 13 were included in the review. We identified three themes regarding nurses' roles in the decision-making process for older adults with cancer: accurate geriatric assessments, provision of available information, and advocacy. Nurses conduct geriatric assessments to identify geriatric syndromes, provide appropriate information, elicit patient preferences, and communicate efficiently with patients and caregivers, promoting physicians. Time constraints were cited as a barrier to fulfilling nurses' roles. Conclusions: The role of nurses is to elicit patients' broader health and social care needs to facilitate patient-centered decision-making, respecting their preferences and values. Further research focusing on the role of nurses that considers diverse cancer types and healthcare systems is needed.

What is "Opioid Stewardship"? An Overview of Current Definitions and Proposal for a Universally Acceptable Definition.

Opioid stewardship has been widely used to promote rational use, monitoring and discontinuation of opioid therapy; however, its definition and scope of practice remain unclear. To synthesize definitions of opioid stewardship proposed by clinical practice guidelines and professional societies, and to offer a proposal for a universally acceptable definition. Systematic literature searches were performed (earliest records toMay 2022) in six databases (MEDLINE, EMBASE, APA PsycINFO, Scopus, and CENTRAL) and grey sources guidelines development bodies and professional societies through Google. The conventional but widely applied content analysis and word frequencies were used to analyze the definitions and scope of practice. After removing duplicates, 449 articles were retrieved (439 databases and registers and 11 from other sources), 19 of which included a definition of "opioids stewardship". A total of 12 themes was identified in the definitions, including 1) improvement or appropriateness of prescribing opioids use, 2) mitigation of risk from opioids, 3) monitoring opioid use, 4) evaluation of opioid use, 5) judicious opioid use, 6) appropriateness of opioid disposal, 7) identification and treatment of opioid use disorder, 8) reduction in mortality associated with opioid overdoses, 9) appropriate procurement practices, 10) appropriate storage, 11) promoting better communications between patients and prescribers including education provision and 12) patient-centered decision-making. Opioid stewardship is inconsistently defined across professional and research literature. While there is a greater focus on appropriateness and need for improvement of prescribing and monitoring of opioid use, the importance of communications between patients and prescribers, and patient involvement in both prescribing and deprescribing decision-making remains sparse. A comprehensive definition has been proposed as part of the work. There is a need to develop and validate the proposed definition and scope of practice to promote rationale for opioid prescribing, use and attainment of favourable outcomes through international consensus involving practitioners, researchers, and patients.

1226 A MULTI-DISCIPLINARY APPROACH TO TRANSFORMING EYE CARE SERVICES FOR CARE HOME RESIDENTS

Abstract Introduction Care home residents can have variable access to eye care services and treatments. We developed a collaborative approach between optometrists, care homes, and primary and secondary care to enable personalised patient-centred care. Objective To develop and evaluate an integrated model of eye care for care home residents. Methods Small scale plan-do-study-act (PDSA) service tests were completed in three care-homes in Southwark (2 residential, 1 nursing) between November 2021 to May 2022. Processes were compared to historical feedback and hospital-based ophthalmology clinic attendances (Mar 2019-2020). Hospital-like assessments were piloted at two care homes for feasibility and acceptability. Further piloting utilised usual domiciliary optometry-led assessment with multidisciplinary meeting access (including optometrist, GP, geriatrician, ophthalmologist and care home nurse) to reduce duplication of assessments and to evaluate MDM processes and referral rates. Results Examination was 100% successful at home (visual acuity and pressure measurement) compared to hospital outpatients (71.7% success visual acuity, 54.5% pressures). Examination was faster than in hospital settings (16 minutes vs 45 minutes-1 hour). Residents were away from usual activities for 32 minutes vs 6 hours for hospital visits including transport. Residents were less distressed with home-based assessments. Did-Not-Attend (DNA) rates reduced (26.7% to 0%), secondary care discharge rates improved (8.4% to 32%). Hospital eye service referral were indicated in 19% -23%, half of which were for consideration of cataract surgery. Alternative conservative plans were agreed at MDM for nursing home residents who were clinically too frail or would not have been able to comply with treatments avoiding 33% unnecessary referrals. Conclusions Home-based eye care assessments appear better tolerated and are more efficient for residents, health and care staff. Utilising an MDM for optometrists to discuss residents with ophthalmologists and wider MDT members enabled personalised patient-centred decision-making. Future work to test this borough wide is in progress.

Surgical management for elderly patients with pancreatic cancer.

Pancreatic cancer, one of the diseases of the elderly, has dismal prognosis, demanding major surgery with high risk and life quality problems, especially in the elderly. Therefore, treatment selection, whether or not to undergo surgery, preoperative risk assessment, and perioperative management of the elderly are becoming critical issues. Although the elderly are expected to have higher morbidity and mortality and lower long-term survival outcomes, surgery is becoming safer over time. Appropriate surgical indication selection, patient-centered decision-making, adequate prehabilitation and postoperative geriatric care are expected to improve surgical outcomes in the elderly. Surgeons must have the concept of geriatric care, and efforts based on institutional systems and academic societies are required. If well selected and prepared, the same surgical principle as non-elderly patients can be applied to elderly patients. In this paper, the surgical treatment of elderly patients with pancreatic cancer is reviewed.

Derivation and validation of pragmatic clinical models to predict hospital length of stay after cardiac surgery in Ontario, Canada: a population-based cohort study.

Cardiac surgery is resource intensive and often requires multidisciplinary involvement to facilitate discharge. To facilitate evidence-based resource planning, we derived and validated clinical models to predict postoperative hospital length of stay (LOS). We used linked, population-level databases with information on all Ontario residents and included patients aged 18 years or older who underwent coronary artery bypass grafting, valvular or thoracic aorta surgeries between October 2008 and September 2019. The primary outcome was hospital LOS. The models were derived by using patients who had surgery before Sept. 30, 2016, and validated after that date. To address the rightward skew in LOS data and to identify top-tier resource users, we used logistic regression to derive a model to predict the likelihood of LOS being more than the 98th percentile (> 30 d), and γ regression in the remainder to predict continuous LOS in days. We used backward stepwise variable selection for both models. Among 105 193 patients, 2422 (2.3%) had an LOS of more than 30 days. Factors predicting prolonged LOS included age, female sex, procedure type and urgency, comorbidities including frailty, high-risk acute coronary syndrome, heart failure, reduced left ventricular ejection fraction and psychiatric and pulmonary circulatory disease. The C statistic was 0.92 for the prolonged LOS model and the mean absolute error was 2.4 days for the continuous LOS model. We derived and validated clinical models to identify top-tier resource users and predict continuous LOS with excellent accuracy. Our models could be used to benchmark clinical performance based on expected LOS, rationally allocate resources and support patient-centred operative decision-making.

S-10-5: OPTIMAL BLOOD PRESSURE AND CARDIOVASCULAR RISK MANAGEMENT IN PATIENTS WITH ATRIAL FIBRILLATION

Hypertension is one of the most common cardiovascular disorders. On a population basis, and high blood pressure (BP) is a major independent risk factor for incident atrial fibrillation (AF) and AF-related complications. Given the high prevalence and both disorders being commonly asymptomatic, opportunistic arrhythmia screening in hypertensive patients is needed. In terms of management, AF could be regarded as a manifestation of hypertensive target organ damage, and optimal BP control is recommended, ideally aiming for a target systolic BP of 120–129mmHg, and diastolic BP of < 80mmHg. There is a close link between hypertension and the risk of stroke in AF, as well as the risk of serious bleeds in anticoagulated AF patients. Also, hypertension burden can be related to other AF-related complications, such as dementia and end renal disease. BP control should also be complementary to lifestyle factors, such as weight reduction, smoking cessation, alcohol reduction and regular exercise, given the close association with incident AF and AF-related complications such as stroke, major adverse cardiac events (MACE) and heart failure. Contemporary management of AF has moved towards a more holistic or integrated care approach to AF management, summed up as the ABC (Atrial fibrillation Better Care) pathway: A/Avoid stroke with Anticoagulation; B/ Better symptom control with patient-centred symptom-directed decisions on rate or rhythm control; and C/Cardiovascular risk and comorbidity management, including lifestyle changes. Adherence to the ABC pathway is associated with a profound reduction in death, hospitalisation, stroke and bleeding in AF patents [Fgure]. In summary, increased awareness, diagnosis and proactive management of detected hypertension is crucial in patients with AF. Optimised blood pressure control is an essential component of such a holistic approach to AF care.

β-Blockade before Noncardiac Surgery: The Devil Is in the Details.

β-Blockade before Noncardiac Surgery: The Devil Is in the Details.

Implementing patient-centred decision making in peri-operative patient blood management.

Implementing patient-centred decision making in peri-operative patient blood management.

Drug Recommendation from Diagnosis Codes: Classification vs. Collaborative Filtering Approaches.

Over time, large amounts of clinical data have accumulated in electronic health records (EHRs), making it difficult for healthcare professionals to navigate and make patient-centered decisions. This underscores the need for healthcare recommendation systems that help medical professionals make faster and more accurate decisions. This study addresses drug recommendation systems that generate an appropriate list of drugs that match patients' diagnoses. Currently, recommendations are manually prepared by physicians, but this is difficult for patients with multiple comorbidities. We explored approaches to drug recommendations based on elderly patients with diabetes, hypertension, and cardiovascular disease who visited primary-care clinics and often had multiple conditions. We examined both collaborative filtering approaches and traditional machine-learning classifiers. The hybrid model between the two yielded a recall at 5 of 76.61%, a precision at 5 of 46.20%, a macro-averaged area under the curve of 74.52%, and an average physician agreement of 47.50%. Although collaborative filtering is widely used in recommendation systems, our results showed that it consistently underperformed traditional classification. Collaborative filtering was sensitive to class imbalances and favored the more popular classes. This study highlighted challenges that need to be addressed when developing recommendation systems in EHRs.

ASSESSING PURPORTED AGING THERAPIES IN THE CLINIC

Abstract Aging is a universal human experience that looms large in popular and commercial culture. It can be difficult for patients and providers alike to sort fact from unproven plausibility and from potentially harmful fiction. Geroscience is an emerging field, and while some interventions involve repurposed or over-the-counter drugs with early clinical data, it is important to note that none yet have a specific aging-related indication and none have a consensus recommendation for clinical use in an aging context. Even highly plausible links to mechanisms of aging do not ensure that an intervention will be effective or risk-free, and not all purported therapies align with preclinical science. Finally, some purported therapies fall into potentially problematic regulatory gaps. With all of these considerations in mind, attendees will gain insights into applying their existing skills to evaluate this field, and new tools for critically evaluating geroscience literature to assist in patient-centered decision making.

How Does the Public Evaluate Vaccines for Low-Incidence, Severe-Outcome Diseases? A General-Population Choice Experiment.

Because immunizing large numbers of healthy people could be required to reduce a relatively small number of infections, disease incidence has a large impact on cost effectiveness, even if the infection is associated with very serious health outcomes. In addition to cost effectiveness, the US Advisory Committee on Immunization Practices requires evidence of stakeholders' values and preferences to help inform vaccine recommendations. This study quantified general-population preferences for vaccine trade-offs among disease severity, disease incidence, and other vaccine features. We developed a best-practice discrete choice experiment survey and administered it to 1185 parents of children aged 12-23 years and 1203 young adults aged 18-25 years from a national opt-in consumer panel. The data were analyzed using exploded-logit latent-class analysis. Latent-class analysis identified two classes with similar relative-importance weights in both samples. One of the two classes represented about half the samples and had preferences consistent with well-structured, logically ordered, and acceptably precise stated-preference utility. Preferences for the other half of the samples were poorly defined over the ranges of vaccine and disease attributes evaluated. Both parents and young adults in the first class evaluated protection from a disease with 1 in 100 incidence and full recovery at home as having statistically the same preference utility as a disease with 1 in 1 million incidence requiring hospitalization and resulting in permanent deafness. The results suggest that vaccines that protect against low-incidence, severe-outcome diseases, provide 'peace of mind' benefits not captured by standard health-outcome metrics. The fact that half the respondents had poorly defined vaccine preferences is a reminder of the challenges of implementing patient-centric vaccine decision making.

Clostridium difficile and other adverse events from overprescribed antibiotics for acute upper respiratory infection.

Guidelines widely recommend avoiding antibiotics for many acute upper respiratory infections (aURIs) to avert adverse events in the absence of likely benefit. However, the extent of harm from these antibiotics remains a subject of debate and could inform patient-centered decision-making. Prior estimates finding a number needed to harm (NNH) between 8 and 10 rely on patient-reported adverse events of any severity. In this analysis, we sought to estimate adverse events by only measuring comparatively severe events that require subsequent clinical evaluation. We constructed a retrospective cohort, including 51 million patient encounters. Using logistic regression models, we determined the adjusted odds ratio (aOR) of clinically detectable adverse events following antibiotic use compared with events among unexposed individuals with aURIs. Our outcomes included candidiasis, diarrhea, Clostridium difficile infection (CDI), and a composite outcome. From our analysis, 62.4% of the population received antibiotics in an aURI encounter. Observed adverse events in the antibiotic-exposed group were 54,279 and 46,936 for diarrhea and candidiasis, respectively, yielding an aOR of 1.24 and 1.61, and an NNH of 3,126 and 1,975. Observed events of CDI in the exposed group were 30,133, and aORs of isolated CDI and combined adverse events were 1.07 and 1.30, resulting in an NNH of 17,695 and 1,150, respectively. Females were more likely to be diagnosed with any adverse event. Overall antibiotics were found to result in 5.7 additional cases of CDI per 100,000 outpatient prescriptions following an upper respiratory tract infection. Despite higher NNH than previous methods of analysis, we find substantial iatrogenic harm associated with prescribing antibiotics in aURIs.

Utilization of The Mobile Application of National Health Insurance (JKN) To Increase The Effectiveness of Services BPJS Insurance In Sibolga

Currently, the government has developed several mobile applications for the National Health Insurance (JKN) to facilitate services for patients. The development of some of these applications aims to help consumers and support patient-centered decisions in choosing which benefits to choose (Bundorf et al., 2019). The digital transformation of the Badan Penyelenggara Jaminan Sosial (BPJS) is to provide convenience for BPJS users or the public, such as paying BPJS contributions, changing membership data, knowing family participant data, etc (Kipo-Sunyehzi, 2021). The aims of this study is to determine the use of the national health insurance application (JKN) to increase the effectiveness of BPJS health services in the city of Sibolga. The type of research taken is descriptive research with a qualitative approach. This study took as many as four resource persons. While the data collection technique is using the technique of observation, interviews and documentation. The results showed that through the national health insurance (JKN) mobile application, all administrative services at the Sibolga City BPJS office for patients became more effective and efficient. This shows that the benefits of the JKN mobile application have proven to be very helpful in accessing BPJS health services.

Influence of interdisciplinary frailty screening on perioperative complication rates in elderly ovarian cancer patients: results of a retrospective observational study

PurposeFrailty is a frequent and underdiagnosed multidimensional age-related syndrome, involving decreased physiological performance reserves and marked vulnerability against major stressors. To standardize the preoperative frailty assessment and identify patients at risk of adverse surgical outcomes, commonly used global health assessment tools were evaluated. We aimed to assess three interdisciplinary preoperative screening assessments to investigate the influence of frailty status with in-hospital complications irrespective of surgical complexity and radicality in older women with ovarian cancer (OC).MethodsPreoperative frailty status was examined by the G8 geriatric screening tool (G8 Score-geriatric screening), Eastern Cooperative Oncology Group performance status (ECOG PS-oncological screening), and American Society of Anesthesiologists Physical Status System (ASA PS-anesthesiologic screening). The main outcome measures were the relationship between perioperative laboratory results, intraoperative surgical parameters and the incidence of immediate postoperative in-hospital complications with the preoperative frailty status.Results116 consecutive women 60 years and older (BMI 24.8 ± 5.2 kg/m2) with OC, who underwent elective oncological surgery in University Medical Center Mainz between 2008 and 2019 were preoperatively classified with the selected global health assessment tools as frail or non-frail. The rate of preoperative anemia (hemoglobin ≤ 12 g/dl) and perioperative transfusions were significantly higher in the G8-frail group (65.9% vs. 34.1%; p = 0.006 and 62.7% vs. 41.8%, p = 0.031; respectively). In addition, patients preoperatively classified as G8-frail exhibited significantly more postoperative clinical in-hospital complications (27.8% vs. 12.5%, p = 0.045) independent of chronological age and BMI. In contrast, ECOG PS and ASA PS did not predict the rates of postoperative complications (all p values > 0.05). After propensity score matching, the complication rate in the G8-frail cohort was approximately 1.7 times more common than in the G8-non-frail cohort.ConclusionPreoperative frailty assessment with the G8 Score identified elderly women with OC recording a significantly higher rate of postoperative in-hospital complications. In G8-frail patients, preoperative anemia and perioperative transfusions were significantly more recorded, regardless of chronological age, abnormal BMI and surgical complexity. Standardized preoperative frailty assessment should be added to clinical routine care to enhance risk stratification in older cancer individuals for surgical patient-centered decision-making.

Promise of Real-World Evidence for Patient Centricity in Gulf Cooperation Council Countries: Call to Action.

Presently, Gulf Cooperation Council countries are lagging in the generation of real-world data and use of real-world evidence for patient-centered care compared with the global average. In a collaborative effort, experts from multiple domains of the healthcare environment from the Gulf Cooperation Council countries came together to present their views and recommended key action points for the generation of robust real-world data and leveraging real-world evidence in the countries. The opinions of the experts are presented, along with existing barriers to the effective generation of real-world evidence in the countries. The Gulf Cooperation Council countries are undergoing transformative changes paving the way for improved healthcare measures; however, the challenges in generating reliable, robust, accessible, and secure real-world evidence are persistent. Hence, ongoing public-private engagements, as well as collaborations between regulators, policymakers, healthcare professionals, insurance and pharmaceutical companies, and patients, are warranted. A few notable examples of real-world evidence studies highlighting the benefits of real-world evidence for gaining valuable insights into patient-centric decision making are also discussed. The actionable steps identified for successful real-world evidence generation would provide long-term, real-world evidence-based patient-centric benefits for the countries.

Investigating the Severity of Complications following Mastectomy and Prepectoral Implant-Based versus Autologous Reconstruction.

Several clinical studies have reported autologous breast reconstruction (ABR) to be associated with a higher postoperative complication rate; however, few have investigated the impact of reconstructive modality on complication severity. This study examines the impact of reconstructive modality on complication severity in a matched cohort of patients who underwent ABR versus implant-based breast reconstruction (IBR). A retrospective study of patients who underwent nipple-sparing mastectomy with immediate reconstruction was performed. Propensity score matching ensured adequate matching of patients who underwent ABR and staged prepectoral IBR, respectively. Patient demographics, breast measurements, and postoperative outcomes (including the incidence and severity of complications) were analyzed. Multivariable logistic regression analysis was performed. P < 0.05 was considered significant. One hundred twenty-eight patients (214 breast reconstructions) were included for analysis (ABR, n = 64; IBR, n = 64). No difference in overall complication rate was noted ( P = 0.61). However, a significant association of IBR with major complications was noted ( P = 0.02). In contrast, minor complications were significantly more frequent following ABR ( P = 0.04). Although the reconstructive modality did not appear to have an effect on the overall complication rate, it did significantly affect the severity of postoperative complications, with major and minor complications being associated with IBR and ABR, respectively. These findings are relevant to patient-centered decision-making, as they provide further granularity regarding postoperative complications and address the issue of complication severity. Therapeutic, III.

The reimbursement for expensive medicines: stakeholder perspectives on the SMA medicine nusinersen and the Dutch Coverage Lock policy

BackgroundThe reimbursement for expensive medicines poses a growing challenge to healthcare worldwide. In order to increase its control over the costs of medicines, the Dutch government introduced the Coverage Lock (CL) policy in 2015. The CL postpones decisions regarding reimbursement of expensive medicines until detailed advice on i.e., cost-effectiveness has been given. The CL has been in place for six years, has raised many questions and concerns, but currently, no evaluation is known to the authors. A better understanding of the effects of the CL on all stakeholders involved may contribute to reflections on the CL process and help find ways to improve it. An evaluation of Dutch policy will also be relevant for other countries that aim to optimize reimbursement procedures for expensive treatments. To perform this evaluation, we focused on the CL procedure for the medicine nusinersen. Nusinersen is the first treatment for spinal muscular atrophy (SMA). Following EMA approval in May 2017, it was placed in the CL. The analysis of cost-effectiveness and added therapeutic value resulted in an advice for reimbursement limited to children younger than 9.5 years at the start of treatment; this was implemented from August 2018 onwards.MethodsQualitative stakeholder perspective analysis of the CL procedure focusing on nusinersen with 15 stakeholders.ResultsStakeholders raised key issues of the CL based on their experience with nusinersen: emotional impact of the CL, duration of the CL procedure, appropriateness of the CL procedure for different types of medicines, transparency of the CL, a wish for patient-centred decision-making and the lack of uniformity of access to expensive treatments.DiscussionStakeholders supported measures to control healthcare expenses and to ensure reasonable pricing. They considered the delay in access to therapies and lack of procedural transparency to be the main challenges to the CL. Stakeholders also agreed that the interests of patients deserve more attention in the practical implementation of the reimbursement decision. Stakeholders suggested a number of adjustments to improve the CL, such as a faster start with conditional reimbursement programs to ensure access and intensify European collaboration to speed up the assessment of the medicine.

Reliability, Validity and Responsiveness of the Polish Version of the Western Ontario Shoulder Instability Index (WOSI-PL) in Patients after Arthroscopic Repair for Shoulder Instability.

Apart from imaging and physical examination for shoulder instability (SI), medical history with patient feedback should be considered to assess the patient's condition and recovery. The aim of this study was to evaluate psychometric properties of the Polish version of Western Ontario Shoulder Instability Index (WOSI)-one of the most frequently used patient-reported outcome measures for SI. During examination 1, 74 patients after arthroscopic repair for SI (age x¯ = 30.01 ± 8.98) were tested. Examinations 2 and 3 involved 71 and 51 patients, respectively. They completed the Polish version of the WOSI, the shortened version of the Disabilities of Arm, Shoulder and Hand Questionnaire (QuickDASH), the Short Form-36 version 2.0 (SF-36 v. 2.0) and 7-point Global Rating Change scale (GRC). The WOSI-PL demonstrated high internal consistency (Cronbach's alpha for total = 0.94), and test-retest reliability (Total ICC2,1 = 0.99). High construct validity was found (89%) as the a priori hypotheses were confirmed. All domains and total scores of WOSI-PL showed a moderate to strong degree of responsiveness (ES = 0.37-0.44; SMR = 0.87-1.26). Minimal clinically important difference (MCID) for the Total WOSI-PL was 126.43 points/6% (95%CI 67.83-185.03) by the anchor-based method and 174.05 points/8% (95%CI 138.61-233.98) by the distribution-based method. The Polish version of the WOSI can be considered a reliable, valid and responsive PROM. It is recommended for assessing the quality of life in patients after arthroscopic repair for SI and can be applied in research and in the clinical setting for monitoring treatment and facilitating patient-centred therapeutic decisions.

Long-Term Management of Pulmonary Embolism: A Review of Consequences, Treatment, and Rehabilitation.

The concept of pulmonary embolism is evolving. Recent and emerging evidence on the treatment of specific patient populations, its secondary prevention, long-term complications, and the unmet need for rehabilitation has the potential to change clinical practice for the benefit of the patients. This review discusses the recent evidence from clinical trials, observational studies, and guidelines focusing on anticoagulation treatment, rehabilitation, emotional stress, quality of life, and the associated outcomes for patients with pulmonary embolism. Guidelines suggest that the type and duration of treatment with anticoagulation should be based on prevalent risk factors. Recent studies demonstrate that an anticoagulant treatment that is longer than two years may be effective and safe for some patients. The evidence for extended treatment in cancer patients is limited. Careful consideration is particularly necessary for pulmonary embolisms in pregnancy, cancer, and at the end of life. The rehabilitation and prevention of unnecessary deconditioning, emotional distress, and a reduced quality of life is an important, but currently they are unmet priorities for many patients with a pulmonary embolism. Future research could demonstrate optimal anticoagulant therapy durations, follow-ups, and rehabilitation, and effective patient-centered decision making at the end of life. A patient preferences and shared decision making should be incorporated in their routine care when weighing the benefits and risks with primary treatment and secondary prevention.