Real World Evidence


Since around the turn of the millennium the biopharmaceutical and medical devices industries have been under increasing pressure to not only provide evidence that medicines and devices work (efficacy data) but to demonstrate that they do so in a cost-effective manner, with tangible benefit to the healthcare system and the patient experience of care. They also, increasingly, are required to provide evidence that medicines shown to be efficacious in controlled environments (randomised, controlled trials; RCTs), continue to demonstrate similar effectiveness in more heterogenous populations in the “real world”.

Beyond the Randomised, Controlled Trial

RCT conditions use highly selected populations and randomise subjects in an effort to minimise bias to provide robust evidence proving the investigational drug is having the effects seen (both efficacy and safety). They are used to provide proof of “cause and effect” by minimising confounders. Of course, in real prescribing situations, patients do not fit the same selection criteria as in RCTs and any number of factors can influence which medicine they are prescribed (e.g. route of administration, other comorbidities, side effect profile, prescriber’s preference).

In addition, direct head-to-head comparator trials are rare. This can make it difficult for clinicians to make fully informed prescribing decisions in the interest of patients, especially in a disease area like Multiple Sclerosis (MS), where new drugs with unique mechanisms of action are regularly entering the market.

In a bid to address some of these challenges, Biogen has supported many MS registry efforts around the world and has invested in research to help fill knowledge gaps. This ranges from supporting independent registries, such as MS Base, to developing bespoke data collection programmes for some of our medicines, such as our Tysabri® (natalizumab) Observational Programme (TOP) database. In addition, Biogen uses validated statistical methods to try to compare different medicines using their respective clinical trial data (e.g. matching-adjusted indirect comparisons). These various sources of evidence help to support the phase III clinical trial data and allow clinicians to make more informed decisions.

Real-World Data (RWD) & Real-World Evidence (RWE)

Real-World Data (RWD) refers to any data derived from sources beyond the RCT setting. This can include but are not limited to disease/ drug registries, healthcare insurance databases, prescribing databases, electronic medical records and wearable devices. It’s important to note that RWD is not restricted to drug information and can include patient reported outcomes (PROs), natural history of disease, social listening and many other sources depending on the type of research being carried out.

Real-World Evidence (RWE) refers to the insights we create using robust methodologies to analyse RWD. For example, a disease register may record the number of specific events seen, such as relapses – the data – and an appropriate analysis will allow us to ascertain relapse rates and compare between products/populations – the insights or evidence. This is a simple example but as we start to add layers to the data sources (e.g. costs of drugs, costs of disease progression, patient reported outcomes) we can start to build a wider picture of the disease and respective management options.

Looking Ahead

Biogen is committed to providing robust, accurate, fair and balanced information on its medicines and MS in general, in line with the requirements of global regulatory bodies and to provide clinicians with as much information as possible to prescribe the right drug, for the right patient, at the right time.

This is why we are also providing educational content to help better understand RWE in general.

Tecfidera® (dimethyl fumarate) Evidence Map


To learn more about the educational support we can provide in RWE, please contact your local Biogen representative.


MSP-PAN-0010l Date of prep: September 2018