Underlining the tendency, a poll in one of the DIA sessions revealed that 30% of the industry delegates attending the meeting believe that RWD may actually be more relevant than clinical trials data. Clearly, we also see a trend towards much greater usage of RWD for licensing and pricing negotiations.

Valuable ‘small data’ from Pre-approval Access Programs

Charles Makin, Vice President and Global Head, RWE & Late Phase Research, ICON plc, gave a fantastic overview of ‘Big Data’ – linking Electronic Medical Records to claims data at a population level. Whilst Big Data is of great interest – and its potential is only starting to be realized – it is our viewpoint at Inceptua that there is an underestimated potential in ‘Small Data’ in the RWD spectrum, ultimately offering opportunities to optimize access to treatments for patients.

At Inceptua we are specialized in Pre-approval Access Programs, and it is our experience that these programs present the perfect opportunity to capture valuable Small Data.

Capturing robust data from a relatively small population of patients in real-time through a study embedded in a Pre-approval Access Program can generate meaningful clinical data from the first patients to access the medicine. An approach like this has the potential to be used in individual, national Health Technology Assessment submissions, and a further advantage is that it costs considerably less than an open-label extension study.

In conclusion, Small Data might be a niche area in the RWD landscape, but it is also underestimated and has the potential to generate big benefits.

Interested in learning more? Don’t hesitate to contact me directly: stuart.bell@inceptua.com or Inceptua: info@inceptua.com