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There are questions surrounding the real-world effectiveness of chemotherapeutic treatments for pancreatic ductal adenocarcinoma. This literature review compared the clinical characteristics and outcomes of available real-world evidence (RWE) for liposomal irinotecan in combination with 5-fluorouracil (5-FU) and leucovorin (LV), a treatment regimen indicated for patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) who previously progressed on gemcitabine-based therapy. A targeted literature search was conducted in the PubMed Central® and Embase® databases to identify available RWE regarding patients with mPDAC receiving liposomal irinotecan published within the last 5 years (January 2014–September 2019). Data were extracted for prior lines of therapy, performance status, overall survival (OS), progression-free survival (PFS), duration of exposure, and adverse events. Six studies met inclusion criteria. A comparison of baseline patient characteristics and results with the included evidence reveals a clinically fragile, real-world patient population in terms of age (range: 61–68), prior lines of therapy with 34–61% of patients receiving ⩾2 lines of lines of prior therapy and performance status [49.8–100% of patients with Eastern Cooperative Oncology Group (ECOG) 0–1]. Studies observed wide OS (range: 5.3–9.4 months) and similar PFS (range: 2.3–4.1 months), with two studies measuring duration of exposure (7.3 weeks, 3.1 months). Patients analyzed by RWE studies tended to be older with significant disease progression, poor performance status, and more heavily pretreated compared with the phase III registrational trial (NAPOLI-1). Despite this, patients treated with liposomal irinotecan + 5-FU/LV therapy had similar outcomes as those in NAPOLI-1.

Supplemental material, Supplemental_Table_1 for Comparing real-world evidence among patients with metastatic pancreatic ductal adenocarcinoma treated with liposomal irinotecan by Jim Koeller, Andy Surinach, Steven R. Arikian, Marko Zivkovic, Patrick Janeczko, Paul Cockrum and George Kim in Therapeutic Advances in Medical Oncology

Transfusion dependence (TD) among myelodysplastic syndromes (MDS) patients negatively impacts survival and health-related quality of life. We evaluated cost patterns of MDS care during TD and transfusion independence (TI). MDS patients were identified from a US claims database (2008-2013). TD was defined as ≥2 consecutive 8-week periods with ≥1 claim during each, and no interim 56-day period without transfusion; TI as 8 subsequent transfusion-free weeks; and transfusion frequency as the mean interval between transfusions during the TD period. 13,741 patients were included; 19% were TD and 70% had a mean interval between transfusions of ≤28 days. During a 2-year period, TD patients incurred a mean total cost of $17,815/patient-month; 53% higher for those with ≤28 days ($19,498) vs. 28 days ($12,717) between transfusions. Among patients who achieved TI, mean total cost was $7874/patient-month. For TD-MDS patients, cost increases are proportional to transfusion frequency and achieving TI yields economic benefits.

Studies indicate children and adolescents with Type 1 diabetes need treatment with multiple daily insulin injections (MDI) or subcutaneous insulin infusion (CSII) for glycemic control. This research study explored the efficacy of MDI’s compared to CSII therapy in children and adolescents with Type I diabetes in an urban setting by examining their HgbA1C outcomes. Retrospective chart review examined 58 records of children and adolescents (ages 0-21) in two outpatient settings who transitioned from MDI's to CSII. HgbA1c values were recorded during MDI use over one year and during the initial year of CSII use and the means were compared. The difference in mean HbA1c values during MDI and CSII therapies were tested using paired t-tests. HgbA1c values differed between age groups under and over 13. Reduction in mean HbA1c of 0.79 (p=0.02) in the older group was statistically significant. The change in the younger group of -0.09 (p=0.75) was not significant. Results suggest that children over 13 have improved glycemic outcomes using pump therapy. Nurses can build a long-term relationship with the patient and family, teaching self-management and offering choices of insulin administration as this study revealed that there was no difference in how they received insulin.

Background: Specialty medications (SP-D) are high cost prescription medications designed for the treatment of complex chronic conditions, i.e. cancer and neurological diseases. Such medications require special handling and shipping, administration, and patient education. To achieve the desired treatment outcomes, including minimizing adverse events, medication adherence is key. There are barriers to poor medication adherence, some of which include high cost, poor understanding of disease and associated therapy, psychological status, forgetfulness, complex dosing regimens, and side-effects. The consequences of non-adherence include increased healthcare resource consumption and poor disease treatment outcomes, e.g. increased relapse, decreased survival time, and/or lack of patient satisfaction. Specialty pharmacy provided medication therapy management (SPMTM) can be important to achieving acceptable medication adherence and, therefore, better treatment outcomes. The specialty pharmacy is a community pharmacy focused on dispensing and providing clinical services pertaining to SP-D. It has pharmacy permits in 50 states as well as in Washington DC, Puerto Rico, and the US Virgin Islands. It is accredited by URAC and ACHC as a specialty pharmacy. Some of the key services provided by specialty pharmacy are: oncology, neurology, autoimmune disease, analgesia (non-controlled drug), investigational drugs, and other SP-D to treat rare/ultra-rare conditions. Aims: The primary objective of the study was to measure the adherence rate to the oncology and neurology SP-D dispensed by the specialty pharmacy. The secondary objective of this study is to compare the quality-of-life (QoL) of patients who voluntarily participated in the SPMTM program at the program’s start of care (SOC) assessment (before the SP-D had been started) and at the follow-up (F-U) assessment (after SP-D had been started). Methods: A retrospective, observational study of patient reported outcomes (PRO) was conducted at a specialty pharmacy among patients diagnosed with various forms of cancers and neurological issues who had prescriptions filled for SP-D from January 1, 2018 to December 31,2018. Patient education by the pharmacist was offered as part of the SPMTM program throughout patient participation in the program. Each patient was offered medication therapy assessment at SOC and 7 days prior to each refill dispense, using a proprietary clinical assessment instrument designed to capture patient reported data via telephonic interview with patients or their caregivers. Embedded in the assessment instruments were two QoL PRO metrics: (1) Number of days work/school missed; and (2) How have you been feeling? (1–10 scale, where 1 was feeling terrible and 10 was feeling wonderful). The means of the SOC (BEFORE SP drug state) and the F-U (AFTER SP drug state) data were captured monthly and annualized. The annualized means were compared, using the Mann-Whitney U-value (M-WU) two-tailed statistic to test for a statistically significantdifference between the annualized means of the respective QoL measures at the p

Few studies have addressed the cost patterns of patients with multiple myeloma (MM) before and after first relapse. This US claims analysis evaluated, from a US health plan perspective, patterns of total direct costs of care from treatment initiation to progression for patients with MM treated with novel agents, using time to next therapy (TTNT) as a proxy measure for progression.A retrospective study was conducted using a large US claims database, evaluating patients with claims for MM between 2006 and 2013. Patients with claims for stem cell transplant (SCT) were excluded. The analysis focused on patients receiving lenalidomide (LEN) or bortezomib (BORT) based treatment, for whom complete claim history was available through initiation of subsequent treatment. Average patient monthly direct costs were determined, including medical and pharmacy costs, and total cost patterns over quarterly time periods were calculated.The study population comprised 2843 patients with newly diagnosed MM (NDMM) and 1361 with relapsed MM. Total monthly cost for patients with NDMM declined steadily, from $15,734 initially to $5082 at 18+ months after therapy. Upon initiation of second-line therapy, total monthly costs rose to $13,876 and declined to $6446 18 months later. Although NDMM cost levels for individual ordinal months were similar between the LEN and BORT groups, TTNT was longer for LEN-based treatments (37 months). The BORT-treated cohort had higher average monthly total costs for NDMM and for the common time period through 37 months after initiation of therapy ($7534 vs $10,763 for LEN and BORT, respectively). Key limitations of this study, in addition to the lack of mortality and staging information available from claims data, include the definition of TTNT based on change in treatment or a defined gap in therapy prior to retreatment, which may differ from actual time of progression in some patients.For patients with NDMM receiving either LEN- or BORT-based treatment without SCT, followed until TTNT, total direct monthly costs (drug + medical) declined steadily over time. Monthly costs returned to near initial levels when patients began second-line therapy and then followed a similar pattern of decline. Due to the longer TTNT for patients initiated on LEN and the associated longer period of below-average costs, patients initiated with LEN-based treatments had mean monthly total costs$3200 lower than total costs for patients initiated on BORT during the first 3 years after starting treatment, cumulating to nearly $120,000 in lower costs for patients initiated on LEN.

Psoriasis and hidradenitis suppurativa (HS) exhibit distinct clinical features, but no studies have directly compared the health-related quality of life (HRQoL) in patients with moderate-to-severe manifestations of these conditions.To determine which disease is associated with more severe HRQoL impairment.Weighted averages of each of the following baseline HRQoL measures were determined and compared between HS and psoriasis populations from 5 clinical trials: Visual Analog Scale (VAS) for pain, Total Work Productivity Impairment, Dermatology Life Quality Index; EuroQOL 5D VAS, and Short Form-36 Health Survey.Compared with patients with psoriasis, patients with HS reported higher scores for VAS-pain (54.3 vs 36.1 [P .0001]), Dermatology Life Quality Index (15.3 vs 11.3 [P .0001]), EuroQOL 5D VAS (58.8 vs 50.8 [P .0002]), and Total Work Productivity Impairment (35.4 vs 18.2). Patients with HS had lower Short Form-36 Health Survey scores than did patients with psoriasis (physical, 39.6 vs 49.0; mental, 41.5 vs 47.5 [both P .0001]).This analysis was performed using published summary data rather than patient-level data, and weighted pooled averages were compared.Patients with HS have a higher HRQoL burden than patients with psoriasis. This study clearly documents the needs of patients with HS and the potential impact of medical, scientific, and societal consensus for the development of more effective HS treatments.