Real-World Investigations

 

Descriptive Studies

Prior to product launch, we conduct epidemiologic studies and targeted literature searches to examine the natural history and burden of the disease/conditions for which our clients are developing new treatments.

Such studies allow to evaluate the background incidence rates or risk factors for adverse events or outcomes in the target population and help our clients position their product and develop their research agenda. Additionally, these types of studies provide our clients with data that can be used to reinforce the product’s value dossier by better positioning the investigated drug on the pharmaceutical market. This study type is often preceded by our database mapping turn-key solution.

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Drug Utilization Studies (DUS)

Drug utilization studies (DUS) aim to describe patterns of drug use in the real world (characteristics of users, off label use, adherence, persistence etc.). They also allow the evaluation of effectiveness of risk communication or risk minimization strategies.

Depending on the objectives, drug utilization studies can be qualitative or quantitative. Quantitative studies are used, for example, to estimate drug utilization (prevalence, dosage, duration) in populations according to demographic characteristics, to monitor specific therapeutic categories or to estimate drug expenditures. Qualitative studies, however, could provide insights on the appropriateness of drug usage.

The development of drug utilization studies requires to identify the most appropriate methodology and data sources to address specific research questions. Thus, YOLARX extensive expertise in real-world data access and analysis is a major advantage to support you all along the process, from protocol writing to data analysis and report to be submitted to regulatory authorities or payers.

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Comparative Effectiveness Studies

Comparative effectiveness studies aim to inform healthcare decisions by providing evidence on the effectiveness, benefits, and harms of different treatment options.

These studies can be conducted using either systematic reviews, network meta-analyses, or real-world studies. Thus, YOLARX’s expertise in both fields is key to support your projects. Indeed, developing study protocols for such studies requires to use a variety of data sources and methods in order to conduct timely research and disseminate the results to relevant stakeholders within the shortest period of time. Strategies to generate the evidence as soon as possible either in the pre-approval or peri-approval phase are proposed.

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Observational Database Studies

In the real-world setting, the majority of observational studies currently conducted collect data on both, drug exposure and health outcomes, from healthcare databases (electronic medical records or administrative claims databases).

YOLARX has extensive experience in the pharmacoepidemiologic methods for the conduct of database studies that address a wide scope of research questions, including the evaluation of safety and effectiveness of healthcare interventions/treatments, healthcare utilization patterns, drug utilization.

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Field Studies (prospective & retrospective)

All types of epidemiologic research designs including cohort, case-control, and cross-sectional studies can be conducted as field studies in order to collect primary data. We have previously conducted field studies in many countries across the globe including Canada, France, Mexico, Brazil, the Philippines and Malaysia to name a few. YOLARX also provides our clients with effective solutions to adequately select a valid and representative sample of study sites, optimize patient enrollment and data collection and propose rigorous and cost-effective methods to obtain the information needed to answer specific research questions.

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Health or Patient Reported Outcomes

Health or patient reported outcomes are essential to fully establish a product’s real-world economic and clinical value. YOLARX provides our clients with strategic solutions regarding the conduct of outcomes research to effectively capture patients’ experience with treatments and therapeutic interventions in the real-world setting. Such evidence is referred to as “value beyond the pill”. Outcomes such as pain level, quality of life, work productivity are increasingly being considered by stakeholders to support value differentiation of new products.  We have used a variety of validated scales and can guide you in the selection of the most appropriate instrument for your study. We also have the capacity to develop and validate new and specific scales.

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Pragmatic Trials

Pragmatic trials measure a wide spectrum of outcomes, mostly patient-centered. Indeed, whereas explanatory clinical trials focus on measuring efficacy by evaluating objective symptoms or markers (clinical or biological), the research question of pragmatic trials is whether an intervention actually works in real life. These trials are designed to evaluate and compare the effectiveness of treatment in routine clinical practice in order to maximize applicability and generalizability. Because conditions mimic as closely as possible everyday clinical settings, with the exception that patients are randomly allocated, policy makers have an active interest in pragmatic trials. For example, cost-effectiveness analyses can inform policy makers and healthcare providers of a treatment’s cost in real-life situations.

Through YOLARX’s longstanding collaboration with regulatory and reimbursement agencies, our team can provide you with valuable strategic insights when setting up your pragmatic study and assist you with developing protocols that will meet the required standards.

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Quasi-experimental Studies

Quasi-experimental studies aims at evaluating the effect of a program or intervention in the population. However, to accordingly reflect real life clinical practice setting, they usually lack random allocation of study participants to treatment or control group. This lack of control over experimental conditions requires the researcher to carefully consider threats to validity and uncontrolled variables that may account for the results. YOLARX is working with experienced researchers who understood these challenges. We put great emphasis on the statistical methods that are necessary to ensure analyses are run accurately.