Use Of Real-world Evidence Research Articles

PCN230 - Innovative Contracting: Evolving use of Web-Based Managed Entry Agreements in Italy

As payers and manufacturers turn to innovative contracting methods to increase patient access to innovative - and expensive - treatments, Italy is considered to be a prime example of a prolific user of managed entry agreements (MEAs). Italy has increasingly been using web-based MEAs to provide access to new oncology drugs in a way that does not overwhelm the healthcare budget and healthcare administration. This study will examine trends in types of web-based MEAs used in Italy over the last decade. Using national web-based drug monitoring lists from 2005 to 2015, monitored oncology drugs under MEAs were isolated and analyzed for an annual breakdown by type of MEA: outcomes-based, financial and outcome/financial hybrid and reason for type of MEA. These agreements were then further analyzed by indication, to determine what types of web-based MEAs are used in which indication and why. Overall, web-based MEA use has grown steadily over the last decade, from 1 contract initiated in 2005 to 13 in 2015 (peaking with 20 in 2014). Of the 82 web-based MEAs initiated in the last decade, the majority most years have been outcomes-based ones, with outcomes-based MEAs making up 65% of total MEAs. In contrast, outcome/financial hybrid MEAs make up only 2% of total in the last decade. After reaching a nadir in 2012, financial MEAs have been on a rise in real and relative terms since. The MEAs span 27 oncology indications, with most indications favoring a specific type of MEA. Few indications included both major types of MEA. As the pharmaceutical industry increasingly looks to value-based pricing, the prevalence of technology has allowed Italy to cut administrative costs by monitoring some drugs online via web-based platforms. Outcomes-based MEAs are favored, with web platforms allowing for easy monitoring and use of real world evidence.

PHP57 - Real World Evidence In Decision-Making Processes On Public Funding Of Drugs In Poland

Real World Evidence (RWE) is data from actual medical practice related to efficacy, safety and costs in opposition to experimental settings. The aim of this study was to assess the use and impact of RWE in decision-making processes in Poland. The analysis was based on data from the Agency for Health Technology Assessment and Tariff System (AOTMiT) published between 01.2012-09.2015 in Verification Analyses (VA), Statements of the Transparency Council (STC) and Recommendations of the President of AOTMiT (RPA). RWE was identified in 53% of VAs, 21% of STCs and 35% of RPAs. Predominantly, RWE was included in reimbursement applications by MAH, however in 4% of VAs AOTMiT analysts found RWE themselves. Most of RWE related to safety (89%) and less to effectiveness (55%). In up to ¾ of RWE including processes, RWE was not evaluated. When RWE was assessed, AOTMiT analysts and the President slightly more often judged that it was insufficient than sufficient (10% vs 9% and 14% vs 12%, respectively), while the TC was nearly twice more restrictive (19% vs 10%). The longitudinal analysis of RWE in AOTMiT evaluations shows steady increase in the number of processes with RWE in 2012-2014 (31, 40, 64 processes) with apparent decline in 2015 to estimated 36 processes p.a. The share of processes with RWE had a peak in 2014 (59% vs 48% in both 2012 and 2013). The share for 2015 including September data is 36%, which is the lowest in the analysed period. Applications with RWE more often had a positive Statement/Recommendation/Decision compared to those without RWE. There is a considerable lack of consistency in identification of RWE on the three distinct levels of assessment and appraisal within the AOTMiT. A growing importance of RWE can be observed, however, the sustainability of this growth remains uncertain.

The Use Of Real World Evidence To Support Market Access Of Medical Devices – Implications For The German Setting

The Use Of Real World Evidence To Support Market Access Of Medical Devices – Implications For The German Setting

PHP145 - Use of real World evidence in German amnog applications

The statutory health insurance in Germany comprises 90% of the German population covering nearly all healthcare services with only little co-payments. German health insurance claims data therefore constitute an important basis for real world evidence (RWE) on epidemiology and cost information. Aim of this study was to investigate to which extent RWE was used for estimation of prevalence and incidence in German AMNOG assessments since introduction 4 years ago and also its impact on price discounts. German AMNOG assessments submitted until December 2014 were evaluated. They were screened for use of RWE in assessing prevalence and incidence and also target populations. After description and discussion of methods and data sources used, statistics were applied to explore a potential influence of use and quality of RWE data on price discounts. In total, 108 AMNOG dossiers were included. Real world evidence was used in 42.6% of these dossiers to assess prevalence and incidence as well as target populations. German claims data were employed in 8 dossiers (7.4%), registry data in 7 dossiers (6.5%), other data sources like Delphi panels in 37 dossiers (34.3%). The impact of quality of RWE evidence on negotiated discounts is inconclusive with limited data available. German claims data comprise comprehensive information such as demographics, outpatient and inpatient care, prescriptions, devices and aids, incapacity to work and sick leave payments. The routine documentation of diagnoses, procedures and prescriptions as well as the ability to evaluate patient histories are particularly useful for prevalence and incidence analyses, especially regarding the target population and cost estimations which are of paramount importance in price negotiations following the AMNOG assessment. German claims data constitute a valuable and valid data source for assessing epidemiologic evidence in German AMNOG assessments. Indication specific claims data analyses are a meaningful complement to literature research.

PHP98 - Comparative Effectiveness Research (CER) and its effect on the Health Care decision-making Environment

Track the perceptions of key CER stakeholders about the current and future effects of CER on evidence generation and application for health care decisions Internet and mail survey of health care stakeholders, including government, health plans, researchers, human resources specialists, employers, and trade organizations, that are influential in or affected by CER; telephone follow-up to maximize response. Two-thirds of CER stakeholders say that the current CER evidence base is insufficient to support treatment decisions, but 90 percent remain committed to the importance of CER. This fifth survey in a series begun in 2010 found fewer respondents reporting no short-term effects of CER, but more than half of respondents believe that substantial improvement in decision-making based on CER will occur over the next 5 years. Almost three-fourths of respondents recognize PCORI’s significance in funding and monitoring new research, nearly double the level five years ago. Respondents are more positive about research priorities; nearly 40 percent believe priorities somewhat/adequately reflect treatment choices faced by patients and providers, compared to only 22 percent two years ago. Just 9 percent believe that processes for interpreting evidence are fully transparent and objective, fewer than in the previous three surveys. Only about 25% of respondents report that real-world evidence or evidence about variability in individual patient response is being used to support treatment decisions. In successive surveys, CER stakeholders continue to look to a five-year horizon for significant effects from CER. They generally view the evidence base as incomplete, recognize the key players in generating new evidence, and understand the time needed for research in their projections of CER’s impact. The relatively scarce use of real-world evidence and evidence about patient variability reflects their views about the completeness of the evidence base to support treatment decisions.

Market Access and Reimbursement: The Increasing Role of Real-World Evidence.

Randomized controlled trials (RCTs) have historically been considered the primary source of evidence to support decision-making in market access and reimbursement. However, thanks to significant progress in capturing data through computerized systems and making these data available, real-world evidence (RWE) has been increasingly considered by manufacturers and payers. Despite initiatives and research worldwide to promote the use of RWE in healthcare decision-making, an overview of its use over a product lifecycle, its strengths and pitfalls, and how it is perceived from a regulatory and payer perspective will provide a better understanding of what RWE is and how it can support decision-making.

The “E” in Cost-Effectiveness Analyses. A Case Study of Omalizumab Efficacy and Effectiveness for Cost-Effectiveness Analysis Evidence

This article is a call for increased use of real-world evidence in health technology assessment and related policy and decision making. There is currently a disconnect between evidence used to guide regulatory approval of therapies and evidence used to inform therapeutic coverage and reimbursement decisions. Public and private payers need to understand not only whether an intervention works but also whether it offers good value compared with licensed alternatives (not placebo) as they are used in the real-world practice and population (not in a controlled trial environment). Addressing such concerns requires evidence to be drawn from a wide range of study designs, but with consideration and weighting given to their relative strengths and weaknesses, as well as their position on the pragmatic-explanatory (i.e., effectiveness-efficacy) continuum. The potential impact of using different types of evidence to inform cost-effectiveness analysis (CEA) is discussed for omalizumab, comparing and contrasting a CEA model informed by an omalizumab efficacy trial to a CEA model drawing primarily on evidence from effectiveness observational studies of omalizumab. There was reasonable agreement between the two omalizumab CEA models, although the incremental cost-effectiveness ratio generated by the effectiveness observational study-driven model was more favorable for omalizumab. Health technology assessment bodies and payers must use their judgment to determine which components of efficacy-based and effectiveness-based CEA evidence are most closely aligned with their goals. For each CEA evidence component, perhaps the two E's form bounds of the truth as well as a fuller picture of the uncertainty surrounding the truth.

MO2 - Use of Real-World Evidence (RWE) to Validate a Trial-based Health Economic Model

MO2 - Use of Real-World Evidence (RWE) to Validate a Trial-based Health Economic Model