Review of real-world evidence studies in type 2 diabetes mellitus: Lack of good practices

ObjectivesUnlike randomized controlled trials, lack of methodological rigor is a concern about real-world evidence (RWE) studies. The objective of this study was to characterize methodological practices of studies collecting pharmacoeconomic data in a real-world setting for the management of type 2 diabetes mellitus (T2DM).MethodsA systematic literature review was performed using the PICO framework: population consisted of T2DM patients, interventions and comparators were any intervention for T2DM care or absence of intervention, and outcomes were resource utilization, productivity loss or utility. Only RWE studies were included, defined as studies that were not clinical trials and that collected de novo data (no retrospective analysis).ResultsThe literature search identified 1,158 potentially relevant studies, among which sixty were included in the literature review. Many studies showed a lack of transparency by not mentioning the source for outcome and exposure measurement, source for patient selection, number of study sites, recruitment duration, sample size calculation, sampling method, missing data, approbation by an ethics committee, obtaining patient's consent, conflicts of interest, and funding. A significant proportion of studies had poor quality scores and was at high risk of bias.ConclusionsRWE from T2DM studies lacks transparency and credibility. There is a need for good procedural practices that can increase confidence in RWE studies. Standardized methodologies specifically adapted for RWE studies collecting pharmacoeconomic data for the management of T2DM could help future reimbursement decision making in this major public health problem.

Fosphenytoin: Current Place in Therapy

Fosphenytoin is a parenteral phosphate ester prodrug of phenytoin developed to overcome the limitations associated with parenteral administration of phenytoin. Despite potential clinical advantages, pharmacoeconomic concerns have prevented widespread substitution of parenteral phenytoin with fosphenytoin. The purposes of this descriptive review are to (1) highlight recent clinical and pharmacoeconomic data regarding the therapeutic decision to use phenytoin or fosphenytoin for the parenteral management of acute seizures, and (2) discuss the implications of fosphenytoin use in neonatal and pediatric patients. Supporting recent, multidisciplinary, consensus guidelines, it is our opinion that each patient should be evaluated individually to identify those who will benefit most from fosphenytoin. Such patients may include those without intravenous or enteral access, those requiring parenteral therapy with tenuous peripheral intravenous access, and pediatric and neonatal patients. Additionally, institution-specific cost analyses should be done to assure the most appropriate agent is being used, while being sensitive to the potential disparate risk profiles between patient populations. Until the issues of safety relative to cost are objectively ameliorated, individual clinicians will likely use their own experience to dictate the place of fosphenytoin in their respective practices.