Real world research

The need for real-world evidence

Demand for data on the real-world use of medicines is increasing. Today’s medicines are increasingly sophisticated and powerful. Pressure on price, emerging delivery models and changing views of what is valued by society demand a greater insight into the value provided by treatment. These questions can only be answered by real-world research.

What is real-world research?

Real-world research studies medicines as they are used by doctors in the environment they were designed for.

It is called real-world research to differentiate it from the research carried out during drug development. In a clinical trial, the selection of patients and the trial conditions are set up to answer the critical safety and efficacy questions but they do not fully reflect real-world use.

Real-world research is diverse, using a wide range of approaches and study designs and collecting data from many different sources and its direction is to become increasingly eclectic.

RW Research vs. RCT?

While Randomised Controlled Trials (RCT) are considered to provide the most robust evidence of the efficacy of therapeutic options, they are affected by well-recognised qualitative and quantitative limitations that may not reflect how the drug of interest will perform in real-life (ENCePP Guide on Methodological Standards in Pharmacoepidemiology)

Real-world studies do not replace RCT but they complement them. RCT remains the gold standard to assess the efficacy of a medicine, device or intervention.

Real-world research in the product lifecycle?

Real-world research plays a key role in supporting activities throughout the product life cycle and supports the development of clinical practice guidelines, licensing, post marketing surveillance, HTA activities and improved patient care.

Data from disease registries help to understand populations prior to a drug being launched and can then show a post launch impact.

Pragmatic trials provide external validity and naturalistic data support expanded access programs and accelerated pathways. After approval, prospective studies of a variety of types provide safety and outcomes data post approval in many cases mandated as a condition of license.

Building up the evidence to support a medicine requires both the explanatory data on the product from clinical trials and external context that comes from real-world research.

The diversity of study types and design

There is not one type of study that defines real-world research, it is characterised by the diversity of study types and of data sources.

Studies can be prospective, retrospective or both and can mix data from hospitals, general practice and patients.

They have different frequencies of data collection and different implementation models depending on the objectives and scope of the study and population.

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Experience

Our expert team has delivered world-class solutions in a variety of study environments.

Post Approval Saftey

Registry Studies

Health Economic Outcomes

Health Technology Assessment

Longitudinal studies

Audit

Our experience:
making sense of complexity

Cisiv has worked with leading Global Pharma companies and CRO’s delivering both large global studies and small national studies. In addition, we have a long experience in varied therapeutic areas and with diverse study structures.

Baseline Plus was built for this environment and has the flexibility to support the wide range of designs and implementations whilst ensuring rigorous methodology and high quality data.

Definitions

Refers to clinical trials in Phase IIIb and Phase IV. These can be interventional, non-interventional, low-interventional, or pragmatic studies.

There are many, very varied, definitions of what constitutes ‘real life’ data: an ISPOR task force dedicated to investigating the collection of real life data has described it as everything that goes beyond what is normally collected in the Phase III clinical trials program in terms of efficacy. According to ISPOR, the sources of real-life data include:

  • Databases: These include cross-sectional and longitudinal databases which essentially provide retrospective data but increasingly offer the opportunity to have prospective add-ins, e.g. on quality of life.
  • Patient and population surveys
  • Prospective or retrospective patient chart reviews
  • Observational data from cohort studies
  • Pragmatic clinical trials: Whether these are strictly “real-life” studies is open to debate. In a way they are simple experimental trials, which raise questions regarding the extent to which they reflect what is happening in real life. Efforts are however made to mimic a real life situation as much as possible.
  • Registries

A study where the investigator does not intervene in the care of the patient, but simply records what happens.

From an EU regulation perspective, a study qualifies as non-interventional and does not fall under the scope of the clinical trials directive 2001/20/EC, provided the following requirements are cumulatively fulfilled:

  • Medicinal product is prescribed in the usual manner in accordance with the terms of the marketing authorization
  • Assignment of the patient to a particular therapeutic strategy is not decided in advance by a trial protocol, but falls within current practice and the prescription of the medicine is clearly separated from the decision to include the patient in the study
  • No additional diagnostic or monitoring procedures are applied to the patients, and epidemiological methods are used for the analysis of collected data. Of note, interviews, questionnaires, and collection of blood samples may be performed as part of normal clinical practice

Pragmatic trials refer to a methodology that enable the researcher to measure effectiveness of a treatment or drug in routine clinical practise and applied to general range of population. Pragmatic trials are used in contrast to explanatory trials. An explanatory approach recruits as homogeneous a population as possible and aims primarily to further scientific knowledge. By contrast, the design of a pragmatic trial reflects variations between patients that occur in real clinical practice and aims to inform choices between treatments.

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