A31 Objectives: Many initiatives (e.g., PROTECT, EFSPI) are exploring quantitative methodologies to conduct benefit/risk assessments of medicines. Objectives of this study were to combine quantitative methodologies that can capture expert knowledge and decisionmakers insights to genuinely support real-world decisions. MethOds: Using the case study of efalizumab, approved by the EMA in 2004 for the treatment of plaque psoriasis and withdrawn in 2009, a pragmatic methodology was developed that combines advanced pharmacoepidemiology and MCDA for quantitative benefit/ risk assessment. Development involved application of: MCDA principles to ensure applicability to any therapeutic area, comparability across medicines, and portability over product cycle (re-evaluation); and advanced pharmacoepidemiology and Bayesian modeling to identify/generate most useful data. Overarching design was guided by ethical implications of criteria and data selection as well as applicability in real life settings including face validity, time constraints, complexity and transparency. Results: The hierarchical multicriteria model consists of two major domains: Benefits (favourable effects, covering the criteria Clinical efficacy/effectiveness and Patient Reported outcomes); and Risks (unfavourable effects-criterion Safety). The safety criterion is subdivided into three generic subcriteria (Adverse events, Serious AEs and Fatal AEs). The benefit criteria are subdivided into specific subcriteria that correspond to the most relevant outcomes for a treatment for plaque psoriasis. All performance are assigned relative to existing alternatives or placebo. Each subcriterion contributes to the output of the model, the Benefit/Risk Estimate, which is the sum of normalized weights for each subcriterion multiplied by the respective performance score. Pharmacoepidemiology data is provided in a standardized format for each subcriterion and includes meta-analytic comparative statistics based on clinical trials, observational data and Bayesian models. Uncertainty is explored in sensitivity analyses. cOnclusiOns: Integration of pragmatic MCDA modeling with advanced pharmacoepidemiology allows quantitative benefit/risk assessment that can be applicable and meaningful in real life regulatory settings.
OBJECTIVES:The use of intravitreal injection of vascular endothelial growth factor inhibitors is an effective treatment for AMD and trials have showed similar clinical effects of bevacizumab and ranibizumab. The aim of this study was to estimate the budget impact for Brazilian Ministry of Health (MoH) recommending ranibizumab instead of bevacizumab for AMD. METHODS: We did a deterministic budget impact analysis, with the MoH perspective, comparing the use of ranibizumab and bevacizumab for wet AMD. The target population was estimated by extrapolating epidemiologic data to the Brazilian population. Data about dosage, administration and fractioning were extracted from literature. Prices were obtained with the Brazilian regulatory agency, applying potential discounting benefits. This analysis did not consider the cost of the fractioning process because it will be assumed by the states and not by the MoH. RESULTS: The considered price of the ranibizumab vial was US$ 962.86 (fractioning is not an option). In contrast, a 4 mL vial of bevacizumab would cost US$ 410.86 (US$ 5.14 each 0.05 mL dose, resulting in 80 doses/vial). Therefore, the expenses of one year on ranibizumab would be about US$ 11,554.37 and about US$ 61.63 for bevacizumab (12 injections for both). Thus, the use of ranibizumab instead of bevacizumab for treating 467,600 people would be related with a US$ 5,374,007,960.48 budget impact. The sensitivity analyses also demonstrated a budget impact of US$ 3,097,416,007.65 and US$ 5,287,555,101.51 (1 dose/ vial and 20 doses/vial, respectively). CONCLUSIONS: Although not a label indication, bevacizumab has been widely adopted in clinical practice. As presented above, even with inefficient fractioning methods, the use of bevacizumab would bring substantial savings to MoH resources. Even the need of preserving the sterility of the solution being a real-world worry, stability studies have showed the maintenance of the solution characteristics through adequate handling and storage.
were conducted in South America (92%), particularly in Brazil (64%). The mean age was 60 ± 9 years and the mean ejection fraction was 36 ± 9%. Most studies evaluated more than one etiology (79%) but the etiology more studies exclusively was Chagas disease (13%). The incidence of HF ranged from 199 to 557 cases per 100,000 person-years and the pooled prevalence was 1%, being higher in older populations. Hospitalization rates in patients with HF ranged from 28 to 31% at different time points, and the median length of stay was 7.0 days. In-hospital mortality was 11.7%, being higher in patients with worse ejection fraction, with ischemic and with Chagas disease. Mortality at one year was 24.52% (95%CI 19.42 to 30.02). ConClusions: This SR of HF in Latin America, could help decision-makers to design better preventive strategies, and guide effective patient-centered care.
A65patient charts were identified (mean age: 41.2yrs; female:67%; currently treated with DMTs: 601(85%); discontinued DMTs in past 3 months: 35(5%); DMT-naive: 35(10%). Of the 601 currently treated patients, current line of DMT: 1 st -line-62%, 2 nd -line-28%, > = 3 rd -line-10%. 1 st -line patient characteristics included-mean age:40.0yrs; female:69%; average time-to-initiation of DMT from diagnosis:7.5mo; time on current 1 st -line DMT:51.5months; JCV status: positive-1%/negative-7%/ don't know-8%/not tested-84%. Top-4 reason for 1 st -line DMT initiation: efficacy against relapses(25%), efficacy in early MS(25%), efficacy in slowing disease progression(18%), patient decision(12%). Mean EDSS-score:1.65; current disability (perphysician-judgment): mild-81%/moderate-17%/severe-2.2%. 2 nd -line DMT patient characteristics included-mean age:42.8yrs; female:66%; average time-to-initiation of DMT from diagnosis:3.7mo; time on current 2 nd -line DMT:31.2months; JCV status: positive-14%/negative-26%/don't know-8%/not tested-52%. Top-4 reason for choosing the 2 nd -line DMT: efficacy against relapses(34%), efficacy in slowing disease progression(34%), patient decision(8%), tolerability(7%); correspondingly, top-4 reasons for switch from 1 st -line DMTs were: lack of efficacy(38%), relapse(24%), flu-like symptoms(17%), injection-site reactions(11%). Mean EDSS-score:2.98; current disability (per-physician-judgment): mild-50%/moderate-44%/severe-7%. ConClusions: In this cohort, RRMS patients on 2 nd -line DMT appear to have higher disease burden compared to those on 1 st -line DMT. Time-to-initiation of first DMT after diagnosis differed significantly between these two groups. Further research is warranted to scrutinize the observed treatment patterns and treatment sequencing strategies to alleviate patient burden.
OBJECTIVES:To evaluate medical and pharmacy costs associated with breakthrough pain (BTP) in a commercially-insured population with chronic, cancer-related pain. METHODS: The National Breakthrough Pain Survey studied a large commercially-insured population using claims data and structured interviews to assess the prevalence, characteristics, and impact of BTP. Adult patients with ≥2 medical claims at an interval ≥3 months with an ICD-9-CM code indicating a chronic pain condition (cancer or noncancer) and ≥3 opioid prescription claims consistent with chronic use were eligible. Patients were called and interviewed after providing consent; those verifying cancer pain were included in this sub-analysis. All-cause medical and pharmacy costs in 2010 US dollars were determined from administrative claims data for the 12-month period before the survey date. Generalized linear models with gamma distribution were constructed because of the skewed nature of the cost data. RESULTS: A total of 2198 patients were interviewed, 1279 had controlled persistent pain, and 145 of the latter group had cancer pain. Of those with cancer pain, BTP was reported by 77.2% (BTP, 112; no BTP, 33). Mean (SD) total annual health care costs for patients with and without BTP were $84,049 ($129,279) and $77,926 ($98,785), respectively. Costs in patients with BTP were 28.6% higher than patients without BTP (P=0.3211) after controlling for health plan, patient demographics, comorbidities, history of prior surgery, neuropathic pain, baseline pain severity, treatment by a pain specialist, and patient-reported pain interference. Mean (SD) total annual pharmacy costs for patients with BTP were $20,088 ($35,406) versus $9,939 ($9,715) for patients without BTP. Patients with BTP had pharmacy costs that were 81.7% higher than patients without BTP (P=0.0265) after controlling for the above variables. CONCLUSIONS: In a commercially-insured population, cancer patients with controlled, persistent pain and BTP had higher total health care and pharmacy costs than cancer patients with controlled, persistent pain without BTP.
A65problems with cognitive functions and problems with emotional dysregulation. ADHD was seen to impact everyday activities, social interactions and emotional functioning. These impacts had implications for the achievements at school; self-esteem and indulgence in risky behavior. Variables that moderate these impacts were also identified. The interrelationships among variables will be presented. The model was used to inform a strategy to evaluate outcomes of pharmacological treatments in adolescents with ADHD. The plausibility of the model was confirmed based on discussions with clinicians and drug development experts. CONCLUSIONS: An ADHD disease conceptual model was developed based on information from literature and stakeholders interviews, to describe ADHD in adolescents. It will be used to develop a strategy for PRO development in adolescent ADHD and identify new outcomes.
Older antiepileptic drugs (AEDs) are known to have a narrow therapeutic index. As a consequence, switching between bioequivalent AEDs remains controversial in the management of epilepsy. We investigated the association between A-rated switching of each class of currently available AED medication and emergent treatment for a seizure-related event. METHODS: We used a case-control method and claims data from the 2010-2011 Truven Health MarketScan ® Commercial Claims Database to estimate the risk of seizure following a medication switch. Cases and controls with an epilepsy diagnosis were identified by emergency/inpatient or outpatient visit claims, respectively. Cases and controls (n=8,106) were matched 1:1 by age, seizure diagnosis category and seizure medication. The exposure was defined as a switch between A-rated AEDs during the 90 days prior to index date. Conditional logistic regression was used to estimate the association, adjusting for gender, baseline Deyo-Charlson Comorbidity Index (0, 1, 2, or 3+), region (Northeast, Central, South, and West), and total AED medications. RESULTS: A switch between A-rated AEDs occurred in 1053 (23.2%) cases and 818 (18.0%) matched controls. The unadjusted and adjusted odds ratios of a seizure-related event for switching were 1.39 (95% CI:
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