Efficacy-Effectiveness-Gap – Extent, Causes And Implications

Abstract

A567assess whether the drug costs stated in BIA matches to real costs. Methods: Twelve HID obtained CED in 2013. The drug costs were identified in BIA of eleven HID. Real costs of General Health Insurance Company (VZP) were found out for each HID in the first year of therapy. As VZP holds 60% of the health insurance market, the data were extrapolated to the whole population. The differences between estimated and real drug costs were analysed and a correlation between this differences and drug characteristics justifying their HID status were investigated. Results: The estimated costs were exceeded in five cases (overrun between 31-332%). In six cases real costs did not achieve the estimation (12-91% of estimated costs). Concerning effectiveness and safety characteristics of investigated drugs, within seven drugs granted as HID because of absence of an alternative drug, four exceeded the estimation. One of two drugs granted as HID because of adverse effects reduction compared with current treatment exceeded the estimation. Remaining two drugs did not achieve the estimation, one was granted HID because of mortality reduction, another because of higher effectiveness compared with current treatment. ConClusions: However HID costs estimated in BIA are submitted in order to predict costs of public health insurance, the analysis did not prove their validity and contribution to a reasonable decision making. Despite the fact that BIA is a mandatory requirement and it is cost and time consuming, its role is formal only and its premises have neither practical impact nor they are subject of further review.

Response to Tyrosine Kinase Inhibitors in Real-World Patients With Chronic Myeloid Leukemia

“…The difference in outcomes between phase 3 clinical trial patients and RWPs is known as the efficacy-effectiveness gap. 25,26 Accounting for individual factors that affect dose response can maximize effectiveness and minimize AEs in RWPs. 27 Given our study findings, we designed and executed a prospective study (NCT03885830) that seeks to associate effectiveness and toxicity outcomes with TKI plasma concentrations, with the long-term goal of guiding TKI dose selection based on RWP factors.…”

The Associations of Diuretics and Laxatives Use with Cardiovascular Mortality. An Individual Patient-Data Meta-analysis of Two Large Cohort Studies

“…However, a network meta-analysis of clinical trials did not show higher cardiovascular risks of low-dose diuretics as first-line antihypertensive treatment compared to β-blockers, angiotensin-converting enzyme inhibitors, calcium channel blockers, alpha-blockers, and angiotensin receptor blockers; rather the opposite was observed [41]. The divergent results can have several reasons: the efficacy-effectiveness gap of clinical trials [42], non-comparable study populations, or an insufficient control for confounding in observational studies. The last point is supported by the fact that our analysis was limited by insufficient control for confounders, such as heart failure and CHD both presenting a higher baseline cardiovascular risk for the affected patients.…”

Using the Problem Tree and Results Chain to Improve Uptake of Intermittent Preventive Treatment in Pregnancy Services—Case Study of the Malaria Action Program for Districts in Uganda

“…Overall, uptake of the IPTp in Uganda is encumbered by several barriers categorized under the six building blocks of the health system, namely leadership/governance, health nancing, medicines and technologies, health information system, human resources, and service delivery and health service user-related barriers. Use of the PTRC analysis tool provided the MAPD team with systematic insights into the barriers effecting program implementation and possible solutions to address them to close the e cacy-effectiveness gap for IPTp [22]. Use of the PTRC analysis tool identi ed similar barriers in a narrative synthesis by Olaleye and Walker (2020), which found that factors effecting the supply and demand for IPTp services involve all pillars of the health system across sub-Saharan Africa [23].…”