Real-world treatment patterns and overall survival among men with Metastatic Castration-Resistant Prostate Cancer (mCRPC) in the US Medicare population.

BACKGROUND: Real-world treatment patterns and survival in metastatic castration-resistant prostate cancer (mCRPC) have not been characterized for the full fee-for-service Medicare population. METHODS: Men newly diagnosed with mCRPC were identified in Medicare fee-for-service claims during 1/1/2014-6/30/2019. Men had evidence of mCRPC and continuous insurance coverage ≥1 year before and ≥6 months after diagnosis unless patients died. Treatment patterns after diagnosis were described. Survival from mCRPC diagnosis and from start of first-line (1 L) therapy was modeled using Kaplan-Meier analysis. RESULTS: Among 14,780 men with mCRPC, mean age was 76 and median follow-up after mCRPC was 17.0 months. 22% received no life-prolonging therapy after mCRPC, 78% received ≥1 line of therapy (LOT), 42% underwent ≥2 LOTs, and 20% had ≥3 LOTs. Median time from start of 1 L to next LOT or end of follow-up was 13.7 months, 10.9 months from 2 L start, and 8.9 months from 3 L start. The most common 1 L to 2 L treatment sequences among men with ≥2 lines were NHT followed by a different NHT (33%), chemotherapy followed by NHT (14%), and NHT followed by chemotherapy (13%). For those initiating 1 L treatment with NHTs, only 28% received subsequent treatment with a different class of therapy. Median survival was 25.6 months after mCRPC and 23.4 months following treatment initiation. CONCLUSIONS: More than 1 in 5 Medicare patients with mCRPC did not receive any life-prolonging therapy, and less than half received 2 L therapy. NHTs were the most common 1 L and 2 L therapies, with patients treated with NHT as 1 L followed by a different NHT for 2 L as the most common treatment sequence. Median survival from diagnosis for all patients was 25.6 months. These data highlight the dramatic undertreatment that occurs for mCRPC patients, particularly for therapies beyond NHTs as well as the common use of sequential NHTs in real-world data.

Healthcare Costs in Men with Metastatic Castration-Resistant Prostate Cancer: An Analysis of US Medicare Fee-For-Service Claims.

INTRODUCTION: To analyze healthcare resource utilization (HRU) and healthcare costs in men with metastatic castration-resistant prostate cancer (mCRPC) in the US Medicare population. METHODS: A published claims-based algorithm was used to identify men with mCRPC in the fee-for-service Medicare population between January 1, 2014, and December 31, 2019. Unadjusted all-cause HRU (days) and healthcare costs paid by Medicare (medical and pharmacy) per patient per year (PPPY) are described for the periods before mCRPC diagnosis, after diagnosis, and from the start of first-line (1L), second-line (2L), and third-line (3L) therapy with mCRPC life-prolonging treatments to the start of subsequent therapy or end of follow-up/death. RESULTS: A total of 14,780 men with mCRPC were identified. After mCRPC diagnosis, 11,528 men initiated 1L mCRPC therapy, 6275 initiated 2L, and 2945 initiated 3L. All-cause medical HRU (days PPPY) increased after mCRPC diagnosis and from 1L through 3L treatment, particularly for outpatient care (pre-diagnosis, 10.4; 1L, 16.2; 2L, 18.9; 3L, 22.0) and physician/other visits (pre-diagnosis, 30.1; 1L, 46.5; 2L, 50.2; 3L, 56.9). Similarly, mean all-cause healthcare costs PPPY were $27,468 in the year before mCRPC diagnosis and increased over four fold to $124,379 after mCRPC diagnosis and continued to rise from start of 1L ($148,325) to 2L ($160,118) to 3L ($165,186) therapy. CONCLUSION: HRU and healthcare costs increased substantially following mCRPC diagnosis, and continued to increase even further through progression from 1L through 3L mCRPC therapy. These findings help to quantify the economic burden of mCRPC and to contextualize the economic value of treatments that delay disease progression.

The cost-effectiveness of dacomitinib in first-line treatment of advanced/metastatic epidermal growth factor receptor mutation-positive non-small-cell lung cancer (EGFRm NSCLC) in Sweden.

AIMS: Although the benefit of first-line epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors (TKIs) over chemotherapy in EGFR mutation-positive (EGFRm) non-small-cell lung cancer (NSCLC) has been demonstrated in clinical trials, the optimal treatment sequence remains unclear. The objective of our study was to evaluate the cost-effectiveness of dacomitinib in Sweden vs afatinib and osimertinib in first-line treatment of EGFRm NSCLC. MATERIALS AND METHODS: A partitioned survival model was developed with three health states: progression-free, post-progression, and death. Progression-free and overall survival curves were used to inform movements between states. Clinical data were taken from randomized trials, compared via a network meta-analysis (NMA). Utility data were taken from published studies and costs from national Swedish sources. The model used a 15-year time horizon and a Swedish healthcare payer perspective. Sensitivity and scenario analyses were performed. RESULTS: The base-case analysis showed that dacomitinib accrued a total of 2.10 quality-adjusted life-years (QALYs) at a total cost of Swedish krona (SEK) 874,615. The incremental cost-effectiveness ratio (ICER) for dacomitinib vs afatinib was SEK 461,556 per QALY gained. The ICER of osimertinib vs dacomitinib, where the small QALY gains of the former came at a high additional cost, was SEK 11,444,709. Deterministic and probabilistic sensitivity analyses confirmed the robustness of these results; changes to drug and medical resource use costs and overall survival had the greatest impact on ICER estimates. LIMITATIONS: This model is subject to uncertainty associated with extrapolating long-term treatment effects from shorter trial follow-up periods, although this would also be a limitation when using direct comparison or time-dependent hazard ratios. The NMA was limited by the use of indirect comparison, although sensitivity analyses supported the robustness of our findings. CONCLUSIONS: Our model demonstrated that dacomitinib is cost-effective for first-line EGFRm NSCLC treatment in Sweden vs afatinib and osimertinib.

Indirect analysis of first-line therapy for advanced non-small-cell lung cancer with activating mutations in a Japanese population.

Background: Five EGFR-tyrosine kinase inhibitors (EGFR TKIs) are currently available in the first-line setting for non-small-cell lung cancer (NSCLC) in Japan. The aim here was to compare the relative efficacy of EGFR TKIs in the Japanese population. Materials & methods: A systematic review identified randomized controlled trials examining the efficacy of first-line EGFR TKIs. A Bayesian network meta-analysis was used to assess these EGFR TKI comparisons for progression-free survival (PFS). Results: A total of seven randomized controlled trials were identified and considered for network meta-analysis. Dacomitinib showed a trend toward improved PFS versus all comparators. Conclusion: Dacomitinib demonstrated a trend toward improved PFS and therefore, should be considered one of the standard first-line therapies for Japanese patients diagnosed with EGFR+ non-small-cell lung cancer.

Network meta analysis of first-line therapy for advanced EGFR mutation positive non-small-cell lung cancer: updated overall survival.

Aim: To update overall survival (OS) results from a previous network meta analysis comparing the relative clinical efficacy of epidermal growth factor receptor-targeted tyrosine kinase inhibitors (EGFR TKIs) for EGFR mutation positive (EGFR+) advanced non-small-cell lung cancer (NSCLC). Materials & methods: A Bayesian network meta analysis was conducted using updated/mature randomized controlled trial OS results in response to first-line EGFR TKI therapies. Results: Dacomitinib showed a numerical improvement of OS relative to other EGFR TKIs: afatinib (hazard ratio [HR]: 0.87; 95% credible interval [CrI]: 0.61-1.24), erlotinib (HR: 0.79; 95% CrI: 0.44-1.42), gefitinib (HR: 0.75; 95% CrI: 0.59-0.95) and osimertinib (HR: 0.94; 95% CrI: 0.68-1.29). Conclusion: Dacomitinib should be considered as a first-line treatment option for patients diagnosed with advanced EGFR+ NSCLC.

Basal Insulin Initiation and Maintenance in Adults with Type 2 Diabetes Mellitus in the United States.

OBJECTIVE: A survey of US adults with type 2 diabetes mellitus was conducted to better understand patients' insulin initiation experiences and treatment persistence behaviors. RESEARCH DESIGN AND METHODS: Participants were recruited from consumer panels and grouped by basal insulin treatment pattern: continuers (no gap of ≥7 days within 6 months of initiation); interrupters (gap ≥7 days, resumed treatment); discontinuers (stopped for ≥7 days, not resumed). A quota of approximately 50 respondents per persistence category was set. RESULTS: A total of 154 respondents (52 continuers, 52 interrupters, 50 discontinuers) completed the survey. Mean age was 51.4 years; 51.9% male. Continuers were more likely to report their views being considered during initiation, and less likely to report a sense of failure. Concerns included insulin dependence (64.3% agree/strongly agree), frequent blood glucose monitoring (55.2%), costs/ability to pay (53.9%), fears of or mistakes during self-injection (52.6%), and weight gain (52.6%). Continuers were motivated by benefits of insulin therapy; experienced or potential side effects were notable factors for interruption/discontinuation. Healthcare provider instruction was indicated as a reason for continuing, stopping, and restarting therapy. CONCLUSION: Benefits of basal insulin therapy motivated continuers while side effects impacted interruption/discontinuation. Persistence on basal insulin is often influenced by provider actions. Earlier provider intervention upon signs of treatment discontinuation may promote persistence.

Burden of illness among patients with severe aplastic anemia who have had insufficient response to immunosuppressive therapy: a multicenter retrospective chart review study.

This study assessed treatment patterns and healthcare resource utilization (HRU) of patients with severe aplastic anemia (SAA) with insufficient response to immunosuppressive therapy (IST). A retrospective chart review was conducted at Dana-Farber Cancer Institute (DFCI), United States, and Hôpital Saint-Louis (HSL), France. Eligible patients were ≥ 18 years old, diagnosed with acquired SAA between January 1, 2006, and July 31, 2016, had insufficient response to IST, and had ≥ 12 months of follow-up post-diagnosis. Overall survival (OS) was estimated using the Kaplan-Meier method. Among the 40 patients, mean age at diagnosis was 44 years and 53% were women. Median follow-up time after SAA diagnosis was 48.3 months. Ninety-five percent of patients received antithymocyte globulin (ATG) as primary therapy prior to hematopoietic stem cell transplant (HSCT). Most common secondary SAA therapies prior to HSCT were eltrombopag (28%) and androgens (15%). Seventy-five percent of patients received HSCT. Prior to HSCT, patients received an average of 2.7 red blood cell (RBC) and 3.3 platelet transfusions per month; patients had 0.9 hospitalizations, 0.4 emergency room visits, and 12.8 office visits per year. Five-year OS was 75%, with infection as the primary cause of death. Additionally, this study provides information on the subgroup of patients receiving eltrombopag which was the most common secondary therapy. This study quantified transfusion and HRU burden associated with SAA and demonstrated high 5-year survival in a recently treated cohort.

Basal insulin persistence in Brazilian participants with T2DM.

OBJECTIVE: Real-world effectiveness of basal insulin therapy is affected by poor treatment persistence, often occurring soon after initiation. This analysis is part of an international cross-sectional study conducted in T2DM patients and is intended to describe the reasons behind non-persistence to insulin therapy in Brasil. METHODS: Responders to an online survey in seven countries were classified as continuers (no gap of ≥7 days), interrupters (interrupted therapy for ≥7 days within first 6 months, then restarted), and discontinuers (terminated therapy for ≥7 days within first 6 months, and did not start it again before the survey). We present the results from the Brazilian cohort. RESULTS: Of 942 global respondents, 156 were from Brasil, with a mean age of 34 years and a mean of 5.8 years since T2DM diagnosis. Reasons contributing to insulin continuation (n=50) were improved glycemic control (82%) and improved physical feeling (50%). Common reasons for interruption (n=51) or discontinuation (n=55) were, respectively, weight gain (47.1%, 43.6%), hypoglycemia (45.1%, 38.2%), and pain from injections (39.2%, 49.1%). However, not all patients who reported weight gain and hypoglycemia as a reason for interruption or discontinuation experienced these: 16/24 (66.7%) and 22/24 (91.7%) participants had weight gain, and 13/23 (56.5%) and 15/21 (71.4%) had hypoglycemia, respectively. The most important reason for possible re-initiation for interrupters and discontinuers, respectively, was persuasion by the physician/HCP (80.4%, 72.7%). CONCLUSION: The benefits of basal insulin therapy motivated continuers to persist with the treatment; experienced or anticipated side effects contributed to interruption and discontinuation. Physician and patient training is key in the treatment of diabetes.

Real-world treatment patterns among patients with advanced gastric cancer in Russia: a chart review study.

Objective: Little evidence is available on the management of patients with metastatic and/or unresectable gastric cancer (mGC) after the failure of first-line treatment. This study presents real-world data on characteristics and treatment patterns of patients with mGC in Russia. Methods: Eligible patients were ≥18 years old, diagnosed with mGC ≥ January 1, 2012, received first-line chemotherapy followed by second-line chemotherapy or best supportive care (BSC), had ≥3 months of follow-up after the start of second-line chemotherapy or BSC (except in cases of death), and had not participated in a clinical trial. Data were summarized using descriptive statistics. Results: A total of 88 physicians provided data from 202 charts. Mean age at mGC diagnosis was 53.7 (standard deviation: 11.2) years; 70.8% of patients were male. Reasons for first-line treatment discontinuation included disease progression (50.5%) and adverse events/toxicity (39.1%). There were 52 unique treatment regimens prescribed in second-line; capecitabine (14.5%), paclitaxel (9.3%), and capecitabine + oxaliplatin (8.7%) were the most frequent. Reasons for second-line treatment discontinuation included disease progression (39.8%) and patient refusal to continue (37.5%). During 2nd-line treatment, the most common treatment-related symptoms were nausea/vomiting (75.0%), while pain (73.8%) was the most common disease-related symptom. Antiemetics (63.4%), chemotherapy (61.6%), non-narcotic analgesics (48.3%), endoscopy (45.9%), and nutritional support (35.5%) were most frequently used as supportive care. Conclusions: Second-line treatment patterns for patients with mGC in Russia are heterogeneous. Results of this study indicate the need for more intensive implementation of the most active regimens in second-line treatment of mGC according to international and national guidelines.

Basal insulin persistence in Brazilian participants with T2DM

SUMMARY OBJECTIVE Real-world effectiveness of basal insulin therapy is affected by poor treatment persistence, often occurring soon after initiation. This analysis is part of an international cross-sectional study conducted in T2DM patients and is intended to describe the reasons behind non-persistence to insulin therapy in Brasil. METHODS Responders to an online survey in seven countries were classified as continuers (no gap of ≥7 days), interrupters (interrupted therapy for ≥7 days within first 6 months, then restarted), and discontinuers (terminated therapy for ≥7 days within first 6 months, and did not start it again before the survey). We present the results from the Brazilian cohort. RESULTS Of 942 global respondents, 156 were from Brasil, with a mean age of 34 years and a mean of 5.8 years since T2DM diagnosis. Reasons contributing to insulin continuation (n=50) were improved glycemic control (82%) and improved physical feeling (50%). Common reasons for interruption (n=51) or discontinuation (n=55) were, respectively, weight gain (47.1%, 43.6%), hypoglycemia (45.1%, 38.2%), and pain from injections (39.2%, 49.1%). However, not all patients who reported weight gain and hypoglycemia as a reason for interruption or discontinuation experienced these: 16/24 (66.7%) and 22/24 (91.7%) participants had weight gain, and 13/23 (56.5%) and 15/21 (71.4%) had hypoglycemia, respectively. The most important reason for possible re-initiation for interrupters and discontinuers, respectively, was persuasion by the physician/HCP (80.4%, 72.7%). CONCLUSION The benefits of basal insulin therapy motivated continuers to persist with the treatment; experienced or anticipated side effects contributed to interruption and discontinuation. Physician and patient training is key in the treatment of diabetes.

RESUMO OBJETIVO Dados de vida real sobre como a eficácia da terapia com insulina é afetada pela baixa persistência ao tratamento que ocorre logo após o início da terapia. Esta análise é a parte brasileira de um estudo transversal internacional conduzido em pacientes com DM2 que teve como objetivo descrever as razões relacionadas à não persistência ao tratamento com insulina. METODOLOGIA O estudo realizado em sete países por meio de questionários on-line classificou como pacientes continuadores (aqueles que não apresentaram intervalo ≥7 dias sem uso da insulina), interrompedores (interromperam a terapia por ≥7 dias nos primeiros seis meses de uso, depois recomeçaram) e descontinuadores (interromperam a terapia por ≥7 dias nos primeiros seis meses de uso e não retornaram). Nesta análise descrevemos os dados da coorte brasileira. RESULTADOS Dos 942 pacientes incluídos, 156 eram do Brasil, com idade média de 34 anos e média de seis anos desde o diagnóstico de DM2. Razões que contribuíram para o uso contínuo da insulina (n=50) foram a melhora do controle glicêmico (82%) e a melhora no estado geral (50%). Razões para a interrupção (n=51) ou para a descontinuação (n=55) foram, respectivamente, ganho de peso (41,7%, 43,6%), hipoglicemia (45,1%, 38,2%) e dor à aplicação (39,2%, 49,1%). Entretanto, nem todos os pacientes que reportaram ganho de peso e hipoglicemia como possível razão para interrupção ou descontinuação realmente apresentaram esses eventos: 16/24 (66,7%) e 22/24 (91,4%) dos participantes apresentaram ganho de peso e 13/23 (56,6%) e 15/21 (71,4%) apresentaram hipoglicemia, respectivamente. A razão mais importante para o possível recomeço entre os interrompedores e descontinuadores foi a persuasão de médicos/profissionais de saúde (80,4% e 72,7%, respectivamente). CONCLUSÕES Os benefícios do tratamento com insulina basal motivaram continuadores a persistir com a terapia; a experiência ou a antecipação de eventos adversos contribuíram para a interrupção e descontinuação. O treinamento de médicos e pacientes é um dos pilares fundamentais do tratamento do diabetes.

Key factors for overcoming psychological insulin resistance: an examination of patient perspectives through content analysis.

Objective: To understand participant perceptions about insulin and identify key behaviors of healthcare professionals (HCPs) that motivated initially reluctant adults from seven countries (n=40) who had type 2 diabetes (T2D) to start insulin treatment. Research design and methods: Telephone interviews were conducted with a subset of participants from an international investigation of adults with T2D who were reluctant to start insulin (EMOTION). Questions related to: (a) participants' thoughts about insulin before and after initiation; (b) reasons behind responses on the survey that were either 'not helpful at all' or 'helped a lot'; (c) actions their HCP may have taken to help start insulin treatment; and (d) advice they would give to others in a similar situation of starting insulin. Responses were coded by two independent reviewers (kappa 0.992). Results: Starting insulin treatment was perceived as a negative experience that would be painful and would lead down a 'slippery slope' to complications. HCPs engaged in four primary behaviors that helped with insulin acceptance: (1) showed the insulin pen/needle and demonstrated the injection process; (2) explained how insulin could help with diabetes control and reduce risk of complications; (3) used collaborative communication style; and (4) offered support and willingness to answer questions so that participants would not be 'on their own'. Following initiation, most participants noted that insulin was not 'as bad as they thought' and recommended insulin to other adults with T2D. Conclusions: Based on these themes, two actionable strategies are suggested for HCPs to help people with psychological insulin resistance: (1) demonstrate the injection process and discuss negative perceptions of insulin as well as potential benefits; (2) offer autonomy in a person-centred collaborative approach, but provide support and accessibility to address concerns. These findings help HCPs to better understand ways in which they can engage reluctant people with T2D with specific strategies.

Clinical burden of asynchrony in patients with asthma when using metered-dose inhalers for control.

Background: Asynchrony, or lack of coordination between inhalation and actuation when using a pressurized metered-dose inhaler (MDI), could theoretically impact the delivery of inhaled medications and treatment efficacy. Objective: To assess the real-world association between asynchrony and clinical outcomes among patients with asthma who receive controller therapy delivered by MDIs. Methods: A cohort of patients was assembled via electronic health records. The patients were aged ≥12 years, with one or more documentations of an asthma diagnosis, no diagnosis of chronic obstructive pulmonary disease, and two or more prescriptions for an inhalation aerosol corticosteroid alone or with long-acting beta-2-agonist delivered via MDI. Their inhaler technique, demonstrated by using a placebo MDI, was evaluated at a clinic visit by study nurses who used a standardized 10-step checklist. Asynchrony was defined as any gap in timing between inhalation and actuation. Clinical outcomes were assessed via electronic health records during the 6 months before the clinic visit and were compared between patients with and patients without asynchrony by using multivariable regression analyses adjusted for age, gender, asthma severity proxy, and baseline comorbidities. Results: Of the total 254 eligible patients, mean age of 49.3 years, 90 males (35.4%), 32 (12.6%) had asynchrony. Patients with asynchrony had higher odds of an asthma exacerbation (adjusted odds ratio, 2.99; p = 0.009), and lower odds of risk domain asthma control (adjusted odds ratio, 0.41; p = 0.04) compared with patients without asynchrony. Conclusion: This study provided real-world evidence that asynchrony in MDI use among patients with asthma who were treated with controller MDIs was associated with clinical burden in terms of asthma exacerbations and control.

Insulin non-persistence among people with type 2 diabetes: how to get your patients to stay on insulin therapy.

Continuing use of medication is key to effective treatment and positive health outcomes, particularly in chronic conditions such as diabetes. However, in primary care, non-persistence (i.e. discontinuing or interrupting treatment) with insulin therapy is a common problem among patients with type 2 diabetes. To help primary care physicians manage patients who are non-persistent or likely not to be persistent, this review aimed to provide an overview of modifiable and non-modifiable factors associated with insulin non-persistence as well as practical strategies to address them. Data were extracted from published studies evaluating factors associated with non-persistence among patients with type 2 diabetes. A targeted literature review was performed using PubMed to identify recent studies (2000-2016) reporting measures of non-persistence with insulin therapy. Practical strategies to identify and prevent non-persistence were based on the authors' direct experience in primary care. Non-modifiable factors associated with non-persistence included gender, age, prior treatments, and cost of therapy. Before/at insulin initiation, modifiable factors included patients' perception of diabetes, preference for oral medication, and concerns/expectations about treatment complexity, inconvenience, or side effects. After initiation, modifiable factors included syringe use, difficulties during the first week of therapy, side effects, and insufficient glycemic control. Open-ended and patient-centered questions and a blame-free environment can help physicians identify, prevent, and reduce non-persistence behaviors. Possible questions to start a conversation with patients are provided. Effective physician-patient communication is essential to the management of diabetes. Primary care physicians should be familiar with the most common reasons for insulin non-persistence.

Real-world treatment patterns and effectiveness among patients with metastatic colorectal cancer treated with ziv-aflibercept in community oncology practices in the USA.

Routine clinical practice data often differ from clinical trials. This study describes real-world treatment patterns and effectiveness among patients with metastatic colorectal cancer (mCRC) receiving ziv-aflibercept in non-academic, community oncology practices in the USA. De-identified electronic medical records from Vector Oncology and Altos Solutions databases were analysed. We identified 218 patients diagnosed with mCRC who had received prior oxaliplatin therapy and initiated ziv-aflibercept as part of second-line or later-line therapy. Overall survival (OS) and progression-free survival (PFS) were estimated using Kaplan-Meier analysis. Mean age was 62.8 years at ziv-aflibercept initiation. Most patients (91.7%) received bevacizumab before ziv-aflibercept, 95.4% initiated ziv-aflibercept with FOLFIRI or another irinotecan-based regimen, and 59.6% had received prior irinotecan. Overall, 24.8% of patients initiated ziv-aflibercept in second line, 31.7% in third line, 21.6% in fourth line and 22.0% in later lines of therapy. Mean duration of ziv-aflibercept treatment was 5.3 months. For patients initiating ziv-aflibercept in second-, third- and fourth-line therapy, median OS was 11.9 (95% confidence interval 5.1-16.2), 11.1 (6.9-16.7) and 8.1 (5.2-11.4) months, respectively, and median PFS was 4.4 (2.8-6.5), 4.3 (2.9-6.3) and 3.4 (2.2-5.2) months, respectively. Common adverse events (AEs) (any grade) included gastrointestinal disorders (64.7%) and asthenia/fatigue (63.3%). In routine clinical practice, ziv-aflibercept was frequently initiated in third line or later lines of therapy. Although patients receiving ziv-aflibercept were more heavily pretreated and potentially less robust compared with the VELOUR trial, median OS for patients receiving second-line ziv-aflibercept was comparable. AE rates were similar to or lower than the VELOUR trial.

Basal insulin initiation use and experience among people with type 2 diabetes mellitus with different patterns of persistence: results from a multi-national survey.

BACKGROUND AND OBJECTIVE: People with type 2 diabetes mellitus (T2DM) often interrupt basal insulin treatment soon after initiation. This study aimed to describe the experiences during and after basal insulin initiation among people with T2DM with different persistence patterns. METHODS: Adults with T2DM from France, Germany, Spain, UK, US, Brazil, and Japan were identified from consumer panels for an online survey. Respondents who initiated basal insulin 3-24 months prior to survey date were categorized as continuers (no gaps of ≥7 days in insulin treatment); interrupters (first gap ≥7 days within 6 months of initiation and restarted insulin); and discontinuers (stopped insulin for ≥7 days within 6 months of initiation without restarting). RESULTS: Among 942 participants, continuers were older than interrupters and discontinuers (46, 37, and 38 years, respectively, p < .01). Continuers reported having fewer concerns before and after insulin initiation than interrupters and discontinuers, while interrupters had the most concerns. Continuers also reported fewer challenges during the first week of insulin use. Continuers were more likely to respond that insulin use had a positive impact on specific aspects of life than interrupters and discontinuers, for example on glycemic control (73.0%, 63.0%, and 61.8%, respectively; p < .01 vs. continuers). CONCLUSION: Among people with T2DM with different persistence patterns after basal insulin initiation there were significant differences in patient characteristics and experience during and after insulin initiation. Interrupters and discontinuers more frequently reported having concerns and challenges during the initiation process, negative impacts after initiation, and less improvement in glycemic control than continuers.

Value of transfusion independence in severe aplastic anemia from patients' perspectives - a discrete choice experiment.

BACKGROUND: Aplastic anemia is a rare, serious blood disorder due to bone marrow failure to produce blood cells. Transfusions are used to reduce risk of bleeding, infection and relieve anemia symptoms. In severe patients, transfusions may be required more than once/week. It is unclear from the patient perspective the impact that transfusions have on quality of life. This study aimed to elicit patient preferences for attributes associated with severe aplastic anemia (SAA) treatment, including transfusion independence. METHODS: An online discrete choice experiment (DCE) was conducted among patients with SAA who experienced insufficient response to immunosuppressive therapy and transfusion dependence for ≥3 months in the past 2 years. Recruitment occurred through the Aplastic Anemia and Myelodysplastic Syndromes International Foundation and referrals from clinical sites in the US and France. Respondents chose between hypothetical treatment pairs characterized by a common set of attributes: transfusions frequency, fatigue, risk of infection, and risk of serious bleeding. Conditional logit model with effects coding was used to estimate part-worth utilities for different attribute levels and the relative importance of each attribute. Predicted utility scores for transfusion frequency levels were reported. RESULTS: Thirty patients completed the survey. Most were age ≥ 40 years (73.3%), female (70.0%), and from the US (86.7%). 33.3% underwent bone marrow transplant; 36.7% received iron chelation therapy. Patients largely agreed that transfusion independence would result in less burden on time and costs, greater control and quality of life, less fatigue (86.7% noted each) and less scheduling around medical appointments (83.3%). The DCE found highest relative importance for risk of bleeding (0.30), followed by risk of infection (0.28), fatigue (0.23), and frequency of transfusions (0.20). More frequent transfusions resulted in lower utility, particularly when increasing monthly transfusions frequency from 4 (0.57) to 8 (0.35). CONCLUSIONS: Our study showed that higher utility was associated with fewer transfusions in SAA patients with insufficient response to immunosuppressive therapy. While risk of bleeding, risk of infection, and fatigue were more important for patient treatment preferences, frequency of transfusions was also important.

Basal Insulin Persistence, Associated Factors, and Outcomes After Treatment Initiation: A Retrospective Database Study Among People with Type 2 Diabetes Mellitus in Japan.

INTRODUCTION: The objective of this study was to assess basal insulin persistence, associated factors, and economic outcomes for insulin-naïve people with type 2 diabetes mellitus (T2DM) in Japan. METHODS: People aged at least 18 years with T2DM with first claim for basal insulin between May 2006 and April 2013 (index date), no insulin use before index date, and continuous insurance coverage for 6 months before (baseline) and 12 months after index date were selected from the Japan Medical Center Database. On the basis of whether there were at least 30-day gaps in basal insulin treatment, patients were classified as continuers (no gap), interrupters (at least one prescription after gap), and discontinuers (no prescription after gap). A multinomial logistic regression model identified factors associated with persistence. Annual healthcare resource use and costs in the year after initiation were compared between continuers and interrupters and between continuers and discontinuers using propensity score-based inverse probability weighting to adjust for baseline differences. RESULTS: Of the 827 people included (mean age 50 years, ca. 71% male), 36% continued, 42% interrupted, and 22% discontinued basal insulin therapy in the year after initiation. Having at least one inpatient visit and using fewer classes of non-insulin antihyperglycemic medications during baseline were associated with lower likelihoods of continuing therapy. Relative to interrupters and discontinuers, continuers had lower hospitalization rates [continuers, 12.7%; interrupters, 25.4% (p < 0.001); discontinuers, 28.4% (p < 0.001)] and lower inpatient costs [continuers, ¥132,013; interrupters, ¥225,745 (p = 0.054); discontinuers, ¥320,582 (p = 0.036)], but higher pharmacy costs [continuers, ¥158,403; interrupters, ¥134,301 (p = 0.039); discontinuers, ¥121,593 (p = 0.002)] in the year after insulin initiation. Total healthcare costs were similar for the three cohorts. CONCLUSIONS: Substantial proportions of people with T2DM in Japan interrupt or discontinue basal insulin within the year after initiation, and they have higher rates and costs of hospitalizations than patients who continue with their insulin therapy. Further research is needed to understand reasons behind basal insulin persistence and the implications thereof to help clinicians manage T2DM more effectively. FUNDING: Eli Lilly and Company, Boehringer Ingelheim.

Basal insulin persistence, associated factors, and outcomes after treatment initiation among people with type 2 diabetes mellitus in the US.

OBJECTIVE: To assess basal insulin persistence, associated factors, and economic outcomes for insulin-naïve people with type 2 diabetes mellitus (T2DM) in the US. RESEARCH DESIGN AND METHODS: People aged ≥18 years diagnosed with T2DM initiating basal insulin between April 2006 and March 2012 (index date), no prior insulin use, and continuous insurance coverage for 6 months before (baseline) and 24 months after index date (follow-up period) were selected using de-identified administrative claims data in the US. Based on whether there were ≥30 day gaps in basal insulin use in the first year post-index, patients were classified as continuers (no gap), interrupters (≥1 prescription after gap), and discontinuers (no prescription after gap). MAIN OUTCOME MEASURES: Factors associated with persistence - assessed using multinomial logistic regression model; annual healthcare resource use and costs during follow-up period - compared separately between continuers and interrupters, and continuers and discontinuers. RESULTS: Of the 19,110 people included in the sample (mean age: 59 years, ∼60% male), 20% continued to use basal insulin, 62% had ≥1 interruption, and 18% discontinued therapy in the year after initiation. Older age, multiple antihyperglycemic drug use, and injectable antihyperglycemic use during baseline were associated with significantly higher likelihoods of continuing basal insulin. Relative to interrupters and discontinuers, continuers had fewer emergency department visits, shorter hospital stays, and lower medical costs (continuers: $10,890, interrupters: $13,674, discontinuers: $13,021), but higher pharmacy costs (continuers: $7449, interrupters: $5239, discontinuers: $4857) in the first year post-index (p < 0.05 for all comparisons). Total healthcare costs were similar across the three cohorts. Findings for the second year post-index were similar. CONCLUSIONS: The majority of people in this study interrupted or discontinued basal insulin treatment in the year after initiation; and incurred higher medical resource use and costs than continuers. The findings are limited to the commercially insured population in the US. In addition, persistence patterns were assessed using administrative claims as opposed to actual medication-taking behavior and did not account for measures of glycemic control. Further research is needed to understand the reasons behind basal insulin persistence and the implications thereof, to help clinicians manage care for T2DM more effectively.

Budget impact model of a 5-grass sublingual immunotherapy tablet for the treatment of grass pollen-induced allergic rhinitis.

OBJECTIVE: Allergic rhinitis (AR) is a chronic disease with a substantial clinical and economic burden. This study estimated the potential budget impact (BI) associated with market entry of Sweet Vernal, Orchard, Perennial Rye, Timothy, and Kentucky Blue Grass Mixed Pollens Allergen Extract Tablet for Sublingual Use ('5-grass SLIT tablet') for patients aged 10-65 with grass pollen-induced AR. METHODS: A budget impact model was constructed to estimate the potential BI from a US payer perspective. The model calculated pharmacy, medical, and total (pharmacy + medical) costs per-member-per-month (PMPM) with and without market entry of the 5-grass SLIT tablet, considering a 3-year time horizon. The target population was determined using an epidemiological approach and existing literature. The treatment market shares without 5-grass SLIT tablet entry were derived from an analysis of de-identified insurance claims data. Pharmacy costs and medical utilization rates and costs were obtained from the claims data analysis and existing literature. One-way sensitivities were conducted for key model inputs. RESULTS: Using an illustrative example of a hypothetical health plan with one million members, the estimated target population of AR patients aged 10-65 was 26,320. On a PMPM basis, pharmacy costs increased by $0.36, $0.44, and $0.51, while total costs (after medical cost offsets) increased by $0.15, $0.18, and $0.22 in the first, second, and third years following entry of the 5-grass SLIT tablet, respectively. Results were most sensitive to changes in the compliance rate, treatment duration, and price. The BI will vary from the base case example when alternative, payer-specific inputs are used. CONCLUSIONS: Using base case inputs, use of the 5-grass SLIT tablet to treat grass pollen-induced AR increased the pharmacy budget for a hypothetical third-party payer. Higher pharmacy costs were partially offset by lower medical budget due to reduced resource use compared with existing treatments.

Healthcare resource use and costs of privately insured patients who switch, discontinue, or persist on anti-muscarinic therapy for overactive bladder.

OBJECTIVES: To compare the healthcare costs of patients with overactive bladder (OAB) who switch vs persist on anti-muscarinic agents (AMs), describe resource use and costs among OAB patients who discontinue AMs, and assess factors associated with persisting vs switching or discontinuing. METHODS: OAB patients initiating an AM between January 1, 2007 and March 31, 2012 were identified from a claims database of US privately insured beneficiaries (n ≈ 16 million) and required to have no AM claims in the 12 months before AM initiation (baseline period). Patients were classified as persisters, switchers, or discontinuers, and assigned a study index date based on their AM use in the 6 months following initiation. Baseline characteristics, resource use, and costs were compared between persisters and the other groups. Resource use and costs in the 1 month before and 6 months after the study index date (for switchers, the date of index AM switching; for persisters, a randomly assigned date to reflect the distribution of the time from AM initiation to switching among switchers) were also compared between persisters and switchers in unadjusted and adjusted analyses. Factors associated with persisting vs switching or discontinuing were assessed. RESULTS: After controlling for baseline characteristics and costs, persisters vs switchers had significantly lower all-cause and OAB-related costs in both the month before (all-cause $1222 vs $1759, OAB-related $142 vs $170) and 6 months after the study index date (all-cause $7017 vs $8806, OAB-related $642 vs $797). Factors associated with switching or discontinuing vs persisting included index AM, younger age, and history of UTI. CONCLUSION: A large proportion of OAB patients discontinue or switch AMs shortly after initiation, and switching is associated with higher costs.