The philosophy of pharmaceutical regulation-Paternalism or freedom of choice?

When assessing the value of new drugs regulatory authorities across the world frequently make different decisions even though their decisions are based on the same evidence package. In this perspective we argue that even in today's world regulatory and medical decision making is framed by conflicting philosophical schools of thought, namely the liberal tradition of the Anglo Saxon countries pioneered by the Scotsman Adam Smith and the continental European tradition of paternalism that roots back to the German philosopher Georg Friedrich Hegel. We outline the basics of these two philosophical theories and show that countries following the liberal tradition are more reluctant to reject new drugs due to weak evidence. Instead, they leave decisions to a greater extend to those who are affected, namely patients and their caregivers.

Survival outcomes of patients newly diagnosed with diffuse large B-cell lymphoma: real-world evidence from a German claims database.

PURPOSE: Diffuse large B-cell lymphoma (DLBCL) is the most common subtype of non-Hodgkin lymphoma with increasing incidence. Although the burden of disease is high, only limited current real-world data on survival analysis, especially survival time, of German patients with DLBCL are available. This retrospective claims-based analysis was conducted to describe real-world survival evidence and treatment patterns of patients with DLBCL in Germany. METHODS: Using a large claims database of the German statutory health insurance with 6.7 million enrollees, we identified patients between 2010 and 2019 who were newly diagnosed with DLBCL (index date) and had no other cancer co-morbidity. Overall survival (OS) from index date and from the end of each treatment line was plotted by means of the Kaplan-Meier estimator, both for the overall cohort and stratified by treatment regimen. Treatment lines were identified based on a predefined set of medications categorized by established DLBCL treatment recommendations. RESULTS: 2495 incident DLBCL patients were eligible for the study. After index date, 1991 patients started a first-line, 868 a second-line, and 354 a third-line therapy. In first line, 79.5% of patients received a Rituximab-based therapy. 5.0% of the of the 2495 patients received a stem cell transplantation. Overall, median OS after index was 96.0 months. CONCLUSION: DLBCL-associated mortality is still high, especially in relapsed patients and in the elderly. Therefore, there is a high medical need for new effective treatments that can improve survival outcomes in DLBCL patients.

Medication Adherence of People Living with HIV in Japan-A Cross-Sectional Study.

Long-term medical care for people living with HIV (PLHIV) is critical for treatment efficacy, and various studies have examined reasons for antiretroviral therapy (ART) non-adherence. In Japan, doctors assume patients maintain high adherence. However, little is known about real-world treatment adherence. We conducted an anonymous self-administered web-based survey asking about adherence for a total of 1030 Japanese PLHIV who were currently on ART. Adherence was determined using the eight-item Morisky Medication Adherence Scale (MMAS-8), for which scoring ranged from 0 to 8 and scores < 6 points were classified as low adherence. Data were analyzed based on patient-related factors; therapy-related factors; condition-related factors, such as a comorbidity with depression (utilizing the Patient Health Questionnaire 9, PHQ-9); and healthcare/system-related factors. Among 821 PLHIV who responded to the survey, 291 responders (35%) were identified as being in the low adherence group. A statistically significant relationship was found between the number of missed anti-HIV drug doses within the previous 2 weeks and long-term adherence, per the MMAS-8 score (p < 0.001). Risk factors for low adherence included age (younger than 21 years, p = 0.001), moderate to severe depression (p = 0.002, using the PHQ-9), and drug dependence (p = 0.043). Adherence was also influenced by a shared decision-making process, including treatment selection, doctor-patient relations, and treatment satisfaction. Adherence was mainly affected by treatment decision factors. Hence, support of care providers should be considered critical for improving adherence.

Healthcare Resource Utilization and Associated Costs of German Patients with Diffuse Large B-Cell Lymphoma: A Retrospective Health Claims Data Analysis.

INTRODUCTION: Diffuse large B-cell lymphoma (DLBCL) is the most common form of non-Hodgkin's lymphoma with increasing prevalence. Although the disease burden associated with DLBCL is high, only limited data on healthcare resource utilization (HCRU) and associated costs of German patients with DLBCL is available. METHODS: Using a large claims database of the German statutory health insurance with 6.7 million enrollees, we identified patients who were newly diagnosed with DLBCL between 2011 and 2018 (index date). Treatment lines were identified based on a predefined set of medication. HCRU and related costs were collected for the entire post index period and per treatment line. RESULTS: A total of 2495 incident DLBCL patients were eligible for the analysis. The average follow-up time after index was 41.7 months. During follow-up, 1991 patients started a first-line treatment, 868 a second-line treatment, and 354 a third-line treatment. Overall, patients spent on average (SD) 5.24 (6.17) days per month in hospital after index. While on anti-cancer treatment, this number increased to nine (10.9) in first-line, 8.7 (13.7) in second-line, and 9.4 (15.8) in third-line treatments. Overall costs per patient per month (PPPM) increased from €421 (875.70) before to €3695 (4652) after index. While on a treatment line, PPPM costs were €17,170 (10,246) in first-line, €13,362 (12,685) in second-line, and €12,112 (16,173) in third-line treatments. Time-unadjusted absolute costs sum up to €59,868 (43,331), €35,870 (37,387), and €28,832 (40,540) during first-line, second-line, and third-line treatments, respectively. The main cost drivers were hospitalizations (71% of total costs) and drug acquisition costs (18% of total costs). CONCLUSIONS: The financial burden of DLBCL in Germany is high, mainly due to hospitalization and drug costs. Therefore, there is a high medical need for new cost-effective therapeutic options that can lower the disease burden and remain financially viable to support the growing number of patients with this aggressive disease.

Culture and COVID-19-related mortality: a cross-sectional study of 50 countries.

Using a cross-sectional sample of 50 countries we investigate the influence of Hofstede's six-dimensions of culture on COVID-19 related mortality. A multivariable regression model was fitted that controls for health-related, economic- and policy-related variables that have been found to be associated with mortality. We included the percentage of population aged 65 and above, the prevalence of relevant co-morbidities, and tobacco use as health-related variables. Economic variables were GDP, and the connectedness of a country. As policy variables, the Oxford Stringency Index as well as stringency speed, and the Global Health Security Index were used. We also describe the importance of the variables by means of a random forest model. The results suggest that individualistic societies are associated with lower COVID-19-related mortality rates. This finding contradicts previous studies that supported the popular narrative that collectivistic societies with an obedient population are better positioned to manage the pandemic.

Treatment Pathways and Health Outcomes of German Patients with Chronic Graft-Versus-Host Disease After Allogeneic Hematopoietic Cell Transplantation: A Retrospective Health Claims Data Analysis.

BACKGROUND: Although chronic graft-versus-host-disease (cGvHD) is an important long-term complication after allogenic hematopoietic cell transplantation (allo-HCT) and is associated with increased healthcare resource utilization, real-world evidence is scarce. OBJECTIVES: The aim of the study was to evaluate survival of patients with cGvHD in Germany and to analyze hospitalization and treatment patterns. PATIENTS AND METHODS: Based on a German claims database with 4.9 million enrollees, a retrospective longitudinal analysis covering a 6-year period between 2013 and 2018 was conducted. Patients with allo-HCT in 2014 or 2015 (index date) and no record of transplantation or documentation of GvHD 365 days prior to index were included. Patients who subsequently developed a cGVHD were compared with those who did not develop a cGVHD within 3 years after index date. cGVHD cases were identified based on documented International Classification of Diseases, Tenth Revision (ICD-10) diagnosis and treatment algorithms. Since the onset of cGvHD is defined at 100 days after allo-HCT, only those alive beyond day 100 were considered in the survival analysis. Patients who did not survive the first 100 days after allo-HCT were censored to prevent a selection bias due to early mortality within patients without GvHD. Survival rates were plotted using the Kaplan-Meier estimator. The number of hospitalizations and average lengths of stay as well as treatment patterns were descriptively examined. RESULTS: Overall, 165 cGvHD patients were identified and compared with 43 patients without cGVHD. Short-term survival rates were better for patients with cGvHD; the 6-month survival probability was 95.8% for patients with cGVHD and 83.7% for patients without cGVHD. However, long-term survival was better in patients without GvHD; The 30-month survival probability was 65.5% for patients with cGVHD and 76.7% for patients without cGVHD. While overall 90% of cGvHD patients were hospitalized at least once, the share was only half for patients without GvHD (44%). 78.2% of patients with cGVHD received corticosteroids in combination with other predefined immunosuppressants. CONCLUSION: Findings from this study reveal a high disease burden associated with cGvHD. This underlines the high medical need for new interventional strategies to improve survival and morbidity after allo-HCT.

Cost per responder analysis of guselkumab versus targeted therapies in the treatment of moderate to severe plaque psoriasis in Germany.

BACKGROUND: The fully human monoclonal antibody guselkumab is an effective treatment option for patients with moderate to severe psoriasis. OBJECTIVE: The objective of this study was to examine the cost per responder of guselkumab compared with other targeted therapies for the treatment of moderate to severe plaque psoriasis in Germany. METHODS: A one-year cost per responder model was developed based on efficacy and safety data from a published network meta-analysis. Drug, treatment administration, resource use, and adverse event costs were included in the analysis. The primary analysis assessed the cost per Psoriasis Area and Severity Index (PASI) 90 responder at week 16. Additional analyses were conducted at year 1. In the year 1 analyses, treatment response was assessed at the end of the induction period (week 16) to determine which patients continued onto maintenance therapy (responders) and which patients moved onto a subsequent adalimumab or secukinumab therapy (non-responders). RESULTS: At week 16, the cost per PASI 90 responder was lower for guselkumab than all comparators except adalimumab and brodalumab. Similarly, in the year 1 analyses, guselkumab had a lower cost per PASI 90 responder than all comparators except brodalumab. CONCLUSIONS: Guselkumab is a cost-effective therapy option in Germany.

Health Resource Utilization and Associated Health Care Costs of Biologic Disease-Modifying Antirheumatic Drugs in German Patients With Psoriatic Arthritis.

OBJECTIVE: To investigate health care costs associated with biologic disease-modifying antirheumatic drugs (bDMARDs) in a German real-world cohort of adult biologics-naive patients with psoriatic arthritis (PsA). METHODS: Claims data from the Institute for Applied Health Research Berlin (InGef) research database for patients with a PsA diagnosis and bDMARD claims record (index date) between January 1, 2014, and December 31, 2017, and no bDMARD prescriptions for 365 days before the index date were retrospectively analyzed. Primary outcomes were determination of health care resource utilization and associated annual health care costs for overall and individual bDMARDs in the 12-month pre-index and post-index periods. These outcomes were compared between persistent and nonpersistent groups. Nonpersistence was defined as treatment gap or switch to a bDMARD other than the index therapy. RESULTS: Among 10,954 patients with a PsA diagnosis, 348 were eligible. Although mean ± SD post-index costs were significantly higher in the persistent group than the nonpersistent group (€27,869 ± 8,001 versus €21,897 ± 10,600, P < 0 .001) due to higher bDMARD acquisition costs (€23,996 ± 4,818 versus €16,427 ± 9,033, P < 0.001), persistence reduced inpatient treatment costs (-€760), outpatient treatment costs (-€192), other drug costs (-€724), and sick leave costs (-€601). CONCLUSION: Although initiation of bDMARDs increased the total health care costs irrespective of persistence status, partial cost offsets were observed in the persistent patient population.

Bright line or lottery? On significance and value in medical decision making.

It is widely acknowledged that using p-value thresholds as the basis for making decision on health care spending is not appropriate. In the context of medical decision making, we argue that patient preferences need to be a stronger factor. Depending on attitudes to risk, patients might prefer a medical treatment that performs on average worse than a comparator but offers a small probability of a large gain such as a cure. However, what has been labeled 'value of hope' is not yet fully reflected in the decision-making process of drug approval and health technology assessment (HTA). Therefore, patient risk preferences should be formally incorporated within the decision-making framework for regulatory and reimbursement decisions.

Real-World Treatment Persistence with Biologic Disease-Modifying Antirheumatic Drugs Among German Patients with Psoriatic Arthritis-A Retrospective Database Study.

INTRODUCTION: To investigate drug survival for biologic disease-modifying antirheumatic drugs (bDMARDs) in a real-world cohort of German adult biologic-naïve patients with psoriatic arthritis (PsA). METHODS: Claims data for patients with a diagnosis of PsA, a bDMARD claims record (index date) between 1 January 2014 and 31 December 2017, and no bDMARD prescription for 365 days before the index date were retrospectively analyzed. The primary outcomes were the overall and individual bDMARD persistence rates over 12 months. Nonpersistence was defined as a treatment gap exceeding the days of supply plus 60 days or switching to a bDMARD other than the index therapy. Sensitivity analysis was performed, wherein the treatment gap was found to vary depending on the bDMARD regimen. Kaplan-Meier curves were plotted to determine persistence; the log-rank test was used to evaluate differences in the persistence rate. Factors associated with treatment discontinuation were evaluated using Cox regression analysis. RESULTS: Among 10,954 patients with a PsA diagnosis, 348 were eligible. The overall bDMARD persistence rate was 57.5%; individual bDMARD persistence rates were 81.3% for ustekinumab, 66.7% for infliximab, and 60.0% for golimumab. The mean (SD) overall persistence with bDMARDs was 289 (103) days; the mean persistence was 334 (72) days for ustekinumab, 309 (82) days for golimumab, and 305 (92) days for infliximab. The main reasons for nonpersistence were switching to another bDMARD (15.8%) and treatment discontinuation (26.7%). Male gender was significantly associated with a lower risk of treatment discontinuation (hazard ratio 0.54, 95% confidence interval 0.39-0.77; P < 0.001). The sensitivity analysis yielded similar results. CONCLUSION: The one-year persistence rate for bDMARDs in German PsA patients is modest, although the persistence rate depends on the bDMARD considered.

Can adaptive clinical trials help to solve the productivity crisis of the pharmaceutical industry? - a scenario analysis.

AIM: The productivity of pharmaceutical research and development (R&D) investments is declining due to high failure rates in clinical research. Recently, the US Food and Drug Administration (FDA) acknowledged that adaptive designs can make drug development more efficient and less costly. Our objective is to simulate cost-saving effects and estimate the impact on global R&D expenditures as well as possible outcomes measured in life-years gained. METHODS: Based on published drug-development cost data we calculate potential cost savings derived from variations in clinical success rates that result from employing adaptive trial designs. In a subsequent step we estimate how those cost changes affect global R&D expenditures and outcomes. RESULTS: Our calculations indicate that an adaptive trial design with the potential to increase success rates of clinical trials by 4 percentage points could lower development costs for a new drug from 2.6 to 2.2bn USD. On a global scale, this cost reduction would free up an additional 4.2bn USD for investment into pharmaceutical R&D to bring about drug innovations that in turn would be capable of generating up to 3.5 million life-years. CONCLUSION: New clinical trial designs are crucial to improving productivity within the pharmaceutical industry and to fostering a sustainable health-care system.

Time to Treatment Discontinuation in German Patients with Schizophrenia: Long-Acting Injectables versus Oral Antipsychotics.

BACKGROUND AND OBJECTIVE: Long-acting injectable antipsychotics (LAIs) are associated with better treatment adherence and persistence than oral antipsychotics (OAPs) in patients with schizophrenia. However, real-world evidence assessing the impact of treatment with LAIs in Germany is limited. To fill this gap, we compared antipsychotic medication adherence and risk of treatment discontinuation (TD) among schizophrenia patients newly initiated on LAI or who switched their OAP regimen (overall cohort; OC). METHODS: Claims data of German schizophrenia patients who initiated LAIs or switched their OAP during 2012-2016 (index date) were retrospectively analyzed. Treatment switch was defined as add-on medication to existing prescription or terminating the existing prescription and initiating another OAP. Adherence and time to treatment discontinuation (TTD) were estimated. Determinants of treatment discontinuation were analyzed using two Cox regression models. Model 1 controlled for age, sex, and Charlson Comorbidity Index (CCI); model 2 also included insurance status, and medication, visit, and psychiatric inpatient stay costs. Sensitivity analysis on patients who terminated existing prescriptions and initiated new OAPs (complete switch cohort; CSC) was performed. RESULTS: In OC (n = 2650), LAI users had better adherence (35.4% vs. 11.6%), persistence (no 60-day gap; 40.7% vs. 19.8%), and longer TTD (median [95% confidence interval (CI)] 216 [193-249] vs. 50 [46-56] days) than OAP users. OAP usage (hazard ratio [HR] 1.89, 95% CI 1.73-2.06; p < 0.001) and greater CCI (HR 1.04, 95% CI 1.00-1.07; p = 0.023) were associated with greater risk of TD in model 1. Model 2 showed similar results. LAI users in CSC also had better adherence, persistence, and longer TTD. In CSC too, OAP usage and greater CCI were associated with greater risk of TD in model 1, but only CCI was significant in model 2. Higher pre-index psychiatric inpatient costs were associated with lower risk of TD (HR 0.99, 95% CI 0.98-1.00; p = 0.014). LIMITATIONS: Inherent limitations of claims data and lack of control on OAP administration may have influenced the results. CONCLUSION: This real-world study associates LAIs with better medication adherence and lower antipsychotic discontinuation risk than OAPs.

Treatment patterns and healthcare resource utilization in palmoplantar pustulosis patients in Japan: A claims database study.

BACKGROUND: Palmoplantar pustulosis (PPP) is a chronic, relapsing, inflammatory autoimmune condition, characterized by sterile pustules on the palms and soles. The treatment patterns of PPP and total health care resource utilization in Japan are not well described. Investigating these areas is needed to understand current PPP management in Japan. OBJECTIVE: To describe the characteristics, medication treatment and health care resource utilization patterns, and associated costs of PPP patients in Japan. METHODS: A retrospective analysis of insurance claims data was conducted using the Japan Medical Data Center database. Adult Patients with at least two claims with a PPP diagnosis from January 1, 2011 to March 30, 2017 and six months of follow-up after the first diagnosis were included. Patient characteristics described include age, gender, and comorbid conditions. Treatment patterns assessed include the types of treatment, sequence of treatment, and rates of discontinuation, switching, persistence and use of concomitant medications. RESULTS: A total of 5,162 adult patients met all inclusion criteria. Mean (SD) patient age was 49.7 (11.6) years and 43.2% were male. A total of 2441 patients (47.8%) received systemic non-biologic drugs during the entire follow up period, 2,366 (46.4%) were prescribed topical therapy, 273 (5.4%) were prescribed phototherapy, while 18 (0.4%) of patients with other autoimmune comorbidities were eligible for prescribed biologics. For treatment-naïve patients with mild PPP, topical therapy was most commonly (77.1%) prescribed, whereas in moderate to severe cases of PPP, systemic non-biologic drugs (65%) were most often used. The frequency of switching was similar (64.3% to 75.3%) across various therapies and treatment lines. CONCLUSION: This study describes the treatment patterns and health care resource utilization for Japanese PPP patients using a large claims database, and highlights an unmet need to derive better treatment strategies for PPP and address disease burden in Japan.

Understanding the global measurement of willingness to pay in health.

Objective: To understand the different methodologies used to elicit willingness to pay for health and the value of a statistical life year through surveys. Methodology: A systematic review of the literature was undertaken to identify studies using surveys to estimate either willingness to pay for health or the value of a statistical life year. Each study was reviewed and the study setting, sample size, sample description, survey administration (online or face to face), survey methodology, and results were extracted. The results of the studies were then compared to any published national guidelines of cost-effectiveness thresholds to determine their accuracy. Results: Eighteen studies were included in the review with 15 classified as willingness to pay and 3 value of a statistical life. The included studies covered Asia (n = 6), Europe (n = 4), the Middle East (n = 1), and North America (n = 5), with one study taking a global perspective. There were substantial differences in both the methodologies and the estimates of both willingness to pay and value of a statistical life between the different studies. Conclusion: Different methods used to elicit willingness to pay and the value of a statistical life year resulted in a wide range of estimates.

Hospitalization Rates and Therapy Costs of German Schizophrenia Patients Who are Initiated on Long-Acting Injectable Medication: A Mirror-Image Study.

BACKGROUND: Long-acting injectable (LAI) antipsychotics can reduce relapse, hospitalization, and costs in patients with schizophrenia. However, real-world evidence assessing the impact of treatment with LAIs in Germany is limited. OBJECTIVE: To provide updated evidence on the impact of LAI initiation on hospitalization rates and therapy costs. METHODS: Using a mirror-image design, claims data of 850 German patients with schizophrenia who initiated treatment with LAIs during 2013-2015 was retrospectively analyzed. For the included patients, costs and resource utilization were compared for the 12 months before the index date (first initiation of LAI) and the 12 months after the index date. Annual treatment costs, hospitalization rates, ambulatory visits, sick leaves and medical aids were assessed. Two models were used to evaluate hospitalization and its costs. In model 1, hospitalization during the index date (first LAI prescription in 2013-2015) was allocated to the "pre-" time interval, while in model 2 it was neither attributed to the pre- nor to the post-index date. Regression analysis was performed to identify patients who benefited the most in terms of cost reduction from LAI initiation. RESULTS: Medication costs were significantly higher post-switching to LAI compared with pre-switching period (€3832 vs €799; p < 0.001). In model 1, number of hospitalizations, days hospitalized, and associated costs were significantly lower post-switching compared with pre-switching (2.3 vs 2.6; 59.2 vs 73.4; and €5355 vs €11,908, respectively; all p < 0.001). Similar results were obtained for costs in model 2 (€5355 vs €10,276; p < 0.001). Mean total costs reduced significantly from pre-switching to post-switching period in model 1 (€13,776 vs €10,418; p < 0.001). Patients with characteristics such as higher number of non-psychiatric and psychiatric inpatient stays during the pre-index period (all p < 0.05) benefited the most from cost reduction after LAI initiation. CONCLUSION: In this cohort of German patients with schizophrenia, treatment initiation with LAI resulted in reduced hospitalization rates and total costs.

Treatment patterns and medical cost of systemic lupus erythematosus patients in Japan: a retrospective claims database study.

Aim: To describe the characteristics and medication treatment patterns, healthcare resource utilization (HRU), and associated costs in Japanese patients with systemic lupus erythematosus (SLE).Methods: Claims data from the Japan Medical Data Center (JMDC) database were used to identify patients with SLE-related claims from 2010 to 2017. Algorithms were developed to retrospectively categorize patients by disease severity, treatment experience, and SLE-related manifestations such as lupus nephritis and central nervous system lupus. Descriptive and multivariate analyses were used to describe treatment pattern and estimate HRU and associated costs for the SLE cohort overall and by disease severity and complications.Results: Among 4,733 eligible patients, 2,072 (43.8%) were treatment naïve, 2,214 (46.8%) were previously treated for SLE, and 447 (9.4%) did not receive any treatment. Mean (SD) age of the total SLE cohort was 45.2 (13.1) years and mean (SD) follow-up duration was 1,137.3 (758.0) d. Based on disease severity, 1,383 (29.2%) patients had mild, 2,619 (55.3%) patients had moderate, and 731 (15.4%) patients had severe SLE. Patients on glucocorticoids (both oral and parenteral) received add-on medications the most (35.5%, p < .001). Mean annual cost per SLE patient in the post-index period, inclusive of hospitalizations, outpatient visits, and pharmacy was ¥436,836; ¥1,010,772; and ¥2,136,780 for patients with mild, moderate, and severe SLE, respectively.Limitations: The database only captured information on patients up to 75 years of age. Due to the nature of the database, biases regarding conditions that attribute to the spectrum of SLE severity, flare incidences, or individual physical status cannot be ruled out.Conclusions: This study describes the treatment patterns and economic burden experienced by Japanese patients with SLE. The results highlight an unmet need to derive better treatment strategies for patients with SLE to effectively address the disease burden in Japan.

Modeling the Choice Between Risperidone Long-Acting Injectable and Generic Risperidone from the Perspective of a Japanese Hospital.

INTRODUCTION: The Japanese government's current policy is to encourage hospitals to discharge hospital patients with schizophrenia earlier and provide them with community care. This study aims to analyze clinical and economic outcomes of different discharge strategies in psychiatric hospitals in Japan. METHODS: A simulation was conducted to compare patient relapse and hospital revenues for different discharge plans. We constructed a decision tree where each tree consists of a different Markov chain that models hospital revenue for four different discharge plans: discharge of the patient after 1, 2, or 3 months, or 4 months or more. The simulation also included variations in the medical treatment regimen in an outpatient setting as part of the discharge strategy. In particular, we looked at the choice between risperidone long-acting injectable (RLAI) and generic risperidone (RIS GE). RESULTS: The use of RLAI in an outpatient setting reduced the number of rehospitalizations compared to generic risperidone use under all discharge plans. Different discharge plans were associated with differences in economic outcomes as well. One of the key revenue drivers for the hospital was the continuation of treatment in the outpatient setting after discharge. CONCLUSION: The use of RLAI in an outpatient setting could help to prevent rehospitalization, thereby contributing to better community care. FUNDING: The Rapid Service Fee was funded by Janssen KK.

Drug Survival of Biological Therapies for Psoriasis Treatment in Germany and Associated Costs: A Retrospective Claims Database Analysis.

INTRODUCTION: Our aim was to study drug survival and associated costs of biologic treatment in a German real-world cohort of biologic-naïve psoriasis patients. METHODS: We utilized a German claims database base with 3,682,561 enrolled patients between 2001 and 2015. Kaplan-Meier curves were plotted to show the persistence of different biologics. To determine factors that influence persistence, a Cox regression analysis was performed. In addition, associated costs were calculated 12 months before and after treatment start with biologics. RESULTS: Among 75,561 patients with a diagnosis of psoriasis, we identified 347 patients who received a biologic; 176 of them were biologic-naïve prior to initiating therapy. Overall, the 1-year persistence rate was 56%. The highest persistence rate was observed for ustekinumab (80%). Younger patients, and those with a high comorbidity index, had a numerically increased risk of treatment discontinuation. However, parameter values were not statistically significant. While the overall costs after treatment start increased due to the acquisition costs of biologics, we found a strong decrease (- 41%) in sick leave after treatment with biologics had been initiated. CONCLUSION: Observed drug survival rates in this real-world setting were relatively low. In line with previous studies, ustekinumab had a higher persistence rate than other biologics. FUNDING: Janssen-Cilag.

Shared Decision-Making and Patient Satisfaction in Japanese Rheumatoid Arthritis Patients: A New "Preference Fit" Framework for Treatment Assessment.

INTRODUCTION: We have developed a new framework to assess shared decision-making (SDM) as a tool to improve patient satisfaction. This framework is based on a "preference fit" index that relates SDM to patient treatment preferences and patient satisfaction in a sample of rheumatoid arthritis (RA) patients in Japan. METHODS: We surveyed 500 RA patients in Japan and explored the interactions between the treatment preference fit index, SDM, and overall patient satisfaction. RESULTS: Our new preference fit index reveals significant impact on patient satisfaction: the better the fit between SDM and patient preferences, the higher the patient satisfaction with the current treatment. Patients treated with biologic agents were more satisfied. Patients suffering from depression or migraines scored significantly lower both on our preference fit measure and for overall patient satisfaction. CONCLUSION: The association between depression and a low treatment preference fit suggests that depression may pose challenges to SDM and that doctors in Japan are less attuned to the SDM preferences of depressed patients. FUNDING: Janssen Pharmaceutical KK.

Co-insurance and health care utilization in Japanese patients with rheumatoid arthritis: a discontinuity regression approach.

BACKGROUND: Co-insurance rates in Japan decrease when patients turn 70 years of age. We aim to compare changes in medical demand for Japanese patients with rheumatoid arthritis (RA) at age 70 prior to 2014, when there was a reduction in co-insurance rates from 30 to 10%, with changes in medical demand at age 70 after 2014 when co-insurance rates decreased from 30% to only 20%. METHODS: We used administrative data from large Japanese hospitals. We employed a discontinuity regression (RD) approach to control for unobserved endogeneity in the data. RESULTS: We identified a total of 7343 patients with RA, 4905 (67%) turned age 70 before April, and found that a 20% decrease in co-insurance was associated with increased utilization of more expensive biologic RA drugs, more outpatient visits and higher total medical costs. However, a 10% decrease in co-insurance for patients who turned 70 after 2014 did not significantly change demand for medical services. CONCLUSIONS: For the younger cohort, we did not observe any changes in medical demand after a price decrease. We therefore conclude that the economic goal of cost sharing, namely a behavioural change towards lower health-care utilization, is not achieved in this particular cohort of chronic patients.