PCT Regulatory Consulting

The Need for ‘Real World Evidence’

During the development of a new drug, the primary focus is on assessing whether it’s safe (i.e., the benefit to the patient outweighs the impact of the side effects) and whether it works (Ref – Explanatory Trial/ Study). In order to answer these questions, it’s important to remove as many variables as possible, which means testing on patients who have as few as possible medical conditions, other than the one being investigated, and who are taking a minimal number of other drugs (i.e., concomitant medications).

However, the drug will be used on ‘real people’, which usually means we have a multitude of medical conditions (that seem to grow exponentially as we grow older) and we are taking a variety of drugs in order to combat these conditions and the side effects of the drugs. There are also the over the counter (OTC) drugs that we use when needed, such as painkillers, anti-histamines, hang-over cures etc.

Real World Evidence’


The drug wasn’t tested in all these ‘real world’ scenarios; to do so would be impractical.

“In the good old days”, market access was ‘simple’. You just needed marketing authorization approval (MAA) and then you were good to go.  Now MAA is just the first step.  You also need to secure reimbursement for your product and to secure reimbursement you need to demonstrate its value e.g., why should a payer purchase your product rather than someone else’s?

This is why we’ve seen an increasing need to generate real world evidence to support value statements for medicinal products.

There is often a significant knowledge gap regarding the ‘real world’ benefit or value of medicinal products once they have been approved.

So how do we get this information/ fill this knowledge gap?

We assess the use of these drugs in situations identical to, or close to, routine clinical practice. These assessments can either be non-interventional (i.e., the patient is already receiving the drug and we collect information on how it’s being used, how well it’s working, and what side effects/ adverse reactions the patient experiences) or ‘real world’ clinical trials e.g., we dictate which patients receive which drug in a research protocol, but the drug is used according to it’s authorized purpose and the trial population is as close as possible to the routine care population. This is where pragmatic clinical trials fit.


How can CHCUK help you?

Pragmatic clinical trials are a hybrid of classic randomized clinical trials and non-interventional studies.  We have the edge here because of our extensive regulatory experience in both fields.

Here’s an overview how we can help.


Areas covered
  • Low intervention trials (clinical trials with similarities to NIS design)
  • Pragmatic clinical trials (comparative effectiveness trials)
Examples of consultancy work offered
(includes but not limited to)
  • Study classification
  • Regulatory compliance gap analysis/ healthcheck
  • Country-specific and study-specific regulatory requirements
  • Study conduct considerations and support
  • Study design
  • Protocol development/ review
  • SOP development/ review
  • Biosampling and biobanking
  • Genetic research
  • Use of technology e.g., BYOD
  • Training and Education
  • Subject Matter Expertise
  • Workshops