REAL WORLD DATA (RWD) AND REAL WORLD EVIDENCE (RWE)

RWE is becoming the foundation for new pricing strategies that are more explicitly linked to the therapeutic value for patients and health outcome benefits to health systems and risk-bearers. RWE provides information on real-life effectiveness and safety, describing product performance in a broader and more representative population over a period of time. It also provides information on comparators and outcomes that are not part of clinical trial protocols.

We design and perform RWE observational studies by identifying optimal data sources and applying advanced epidemiologic and analytical methods. For example, a retrospective study may be designed that uses an electronic platform for real-world data collection to assess various aspects of real-life effectiveness of a treatment. Other options may include:

• Evaluation of treatment effectiveness on different subpopulations;
• Assessment of impact of risk factors;
• Verification of dependency between endpoints;
• Gathering data on clinical practice, costs and resource utilisation.

This service contains the following elements:

• Analysis of the aim and scope of the study;
• Study Protocol development;
• Development of a Statistical Analysis Plan;
• Recruitment of researchers and research centres;
• Development of an Electronic Platform (personalised portal);
• Coordination of data collection process;
• Statistical analysis and (optionally) modelling;
• Final Study Report.

RWE also has an impact on the medical product’s registration, reimbursement and use. Clients could use RWE in the following ways:

• To evaluate past examples or opportunities for private-public partnership and for managed entry schemes;
• To evaluate scientific value, access schemes and characteristics of electronic medical records supplied by providers and available at RW databases with respect to a particular country;
• To identify and characterise the RWE evaluations of a specific health intervention/condition;
• To conduct external, independent hypotheses validation by KOLs.

RWD ELECTRONIC PLATFORM FOR DATA COLLECTION

INAR creates a personalised electronic platform for multi-language and international surveys. The aim of conducting RWD surveys is to gather data that may include, but is not limited to, current clinical practice, epidemiology, currently used therapeutic regiments, costs and/or resource consumption. The advantage of using the RWD electronic platform is that responses to surveys are used as a source of reliable data reflecting a country’s practice and allows for analysis to be included in HTA reports and other publications.

The RWD electronic platform allows INAR to create personalised surveys that can include elements such as images, graphs, video and audio, as well as various question and response formats, including multiple choice, fill in the blank, drop-down, etc. The platform is easy to use for respondents and analysts alike, allowing for quick data collection and analysis. Respondents are able to view their progress, which is tracked as percent completed. The platform saves answers of active surveys, allowing respondents to view previous questions and to go back to the survey at any time. Furthermore, our analysts are able to track individual survey progress in real-time, prompting reminders when needed. The electronic platform also allows for execution of multi-stage surveys, which may include different pathways for completion. An example of this is having patients respond to a survey, which is then sent to a medical professional for to respond to patient entries. Another feature of the surveys is that different question pathways may be created depending on respondent’s answer selection.

For a respondent to start a survey, an online link is sent by INAR, from which respondents are able to access the personalised electronic platform. The platform is compatible with all systems and browsers. In the case that respondents are without or have limited access to the internet, we send hard copy surveys. This allows us to reach our complete responder base.

Below is an example of a question presented on the RWD electronic platform.

In order to create a personalised RWD electronic platform, a study protocol containing all related assumptions and methodology is required. As the scope of a study depends on our Partner’s specific needs, together with our Partner we determine the type and number of centres and the amount of clinical experts needed to perform the survey. Experts may either be proposed by our Partner or selected by INAR.

BURDEN OF DISEASE

INAR offers to develop a report concerning burden of disease for a chosen country(ies). In such a report, we provide information regarding a specific disease and its symptoms, taking the following into account: epidemiology, current treatment, standards of care, resource utilisation and costs of treatment for adverse events.

As burden of disease refers to the impact of a health problem on individuals and society, criteria measured may include: potential productivity lost, costs of illness or social impact for populations with disease/illness. To determine the level of burden of disease the following questions may be asked:

• Does the patient pay for private care?
• Does the patient have to stop employment / education / school / go on sick leave due to disease?
• Does the patient have to go on the disability retirement?
• Do the relatives take care of the patient due to his/her unemployment?
• Does the relative have to stop working to take care of the patient?
• Does the unemployed patient receive social benefits?
• Does the patient hire a caregiver?
• Did the patient become divorced/separated because of the disease?
• Did the patient lose contact with his/her children because of the disease?

Relevant information on burden of disease is collected in two ways:

• Literature review, and
• Collection of retrospective data from patient medical records.