Study Of Real-world Outcomes Research Articles

Prospective Study of Real-World Outcomes in the Treatment of Neurogenic Orthostatic Hypotension With Droxidopa (P5.319)

Prospective Study of Real-World Outcomes in the Treatment of Neurogenic Orthostatic Hypotension With Droxidopa (P5.319)

The Who, How and What of Real-World Penile Implantation in 2015: The PROPPER Registry Baseline Data

To date, the published data on patients treated with penile implantation generally consist of small series of single surgeon, retrospective experiences rather than prospective or large, multicenter evaluations. This study establishes a baseline of data collection from the PROPPER (Prospective Registry of Outcomes with Penile Prosthesis for Erectile Restoration). The PROPPER is the first large, prospective, multicenter, multinational, monitored, and internal review board approved study of real-world outcomes for patients with penile implants. Data from the PROPPER study were examined to determine patient baseline characteristics and primary and secondary etiologies before treatment of erectile dysfunction. Data include type and size of implant received, surgical steps/techniques used during implantation, and duration of hospital stay. Through April 2, 2015 a total of 1,019 patients were enrolled in the study at 11 sites, with radical prostatectomy being the predominant etiology in 285 (28%). Of those 285 patients treated with radical prostatectomy 280 (98.2%) received an AMS 700™. Of these patients 65.0% (182 of 280) had placement of the reservoir in the traditional retropubic space vs 31.8% (89 of 280) in a submuscular location. Of those patients not treated with radical prostatectomy receiving an AMS 700, fewer underwent reservoir placement in the submuscular location (17.7%, 124 of 702, vs 80.9%, 568 of 702; p <0.001). Of those patients receiving an AMS 700, those treated with radical prostatectomy and those with diabetes had more outpatient admissions (less than 24 hours, 56.8% and 52.1%, respectively) compared to those with cardiovascular disease and Peyronie's disease (42.0% and 35.6%, respectively, p <0.001). This first-of-its-kind, large, prospective, multicenter study reveals most penile implant cases in North America receive an inflatable penile prosthesis and that radical prostatectomy is the most common primary etiology of penile implant surgery. Moreover, patients treated with radical prostatectomy were more likely to have the reservoir placed in a submuscular location, have a longer operating room time and be admitted to the hospital overnight compared with other patient groups.

Warfarin: Not Dead Yet

Warfarin: Not Dead Yet

Does Pen Help? A Real-World Outcomes Study of Switching from Vial to Disposable Pen Among Insulin Glargine-Treated Patients with Type 2 Diabetes Mellitus

The study was designed to evaluate real-world data on clinical and economic outcome differences between patients with type 2 diabetes mellitus (T2DM) who use insulin glargine with vial-and-syringe delivery and those who switch to pen administration. This retrospective study analyzed medical and pharmacy claims information from the national managed-care IMPACT(®) database (Ingenix Inc., Salt Lake City, UT). Adults with T2DM treated with insulin glargine were evaluated. Clinical and economic outcomes over 1 year were compared between individuals who had converted from administering glargine via vial-and-syringe to the SoloSTAR(®) (sanofi-aventis U.S., Bridgewater, NJ) pen (Switchers) and patients who continued to use vial-and-syringe administration (Continuers). Patients from each cohort were matched using propensity score matching for a comparison sample. In total, 3,893 eligible patients were identified (665 Switchers and 3,228 Continuers), with a matched cohort with 603 patients in each group. Baseline characteristics were similar between groups. One-year treatment persistence was significantly higher with Switchers versus Continuers (65.3% vs. 49.8%; P<0.0001). Medication possession ratio was also significantly higher among Switchers (0.79 vs. 0.76; P=0.0173). Insulin use and glycemic control were similar between groups. Healthcare utilization and total costs were also similar between groups. Higher prescription costs among Switchers were offset by lower overall and diabetes-related outpatient and inpatient costs. Switching from insulin glargine vial-and-syringe administration to pen delivery resulted in improved treatment adherence and persistence, with comparable clinical and economic outcomes.

VI1 Determining the Impact of Modeling Additional Sources of Uncertainty in Value of Information Analysis

The conditional reimbursement policy for expensive medicines in the The Netherlands requires real-world data collection on cost-effectiveness within a four years period (T=4) after the initial decision to reimburse a drug (T=0). This introduces new sources of uncertainty, which are less important in an RCT than in real life. This may affect the priorities for further (real-world) research as determined in a VOI analysis. Identifying and modeling types of uncertainty that are usually not parameterized at T=0 but may become relevant at T=4. Include them in the VOI analysis. We use a hypothetical model with four states and parameters related to transition and exacerbation probabilities, costs and utilities. Three additional uncertainties were parameterized: persistence, compliance and broadening of indication. Persistence refers to the duration of the treatment and it is determined by the probability of dropping out of the treatment. Compliance is characterized by the fraction of the treatment benefit obtained due to not taking the medication as it was indicated. The impact of indication broadening is modeled as the percentage of the RCT treatment effect realized in the outcome study. These extra parameters were included in the VOI analysis. Priorities change when new uncertainties are introduced in the model. Initially, the EVPPI was highest for transition probabilities followed by utilities; and it was very low for exacerbation probabilities and costs. After new uncertainties are included, compliance and broadening of indication (which is applied only to the new treatment) become as relevant as utilities. Persistence however has little impact in the model. VOI analysis at T=0 should anticipate and parameterize new types of uncertainty that may emerge during a four year outcomes study. This would help to focus the real-world outcomes study on those parameters that reduce uncertainty in the decision to continue the reimbursement most.