Real World Data

At present, clinical trials remain the gold standard for assessing the safety and efficacy of investigational new drugs. However, global regulators are seeing the value in collecting data from additional sources outside of the clinical trial setting. Regulators are now recommending that Real World Evidence (RWE), generated through the collection and analysis of Real World Data (RWD) from patients, be incorporated into drug development and approval processes.

For example, new legislation in the US (21st Centuries Cures Act), will give the FDA the ability to review alternative measures of drug efficacy. The MHRA’s Early Access to Medicines Scheme in the UK also encourages the collection of RWD during early access programs.

RWD, collected from broader populations outside of clinical trials, can provide a number of benefits, including:

  • Further insight into drug safety and efficacy
  • Greater understanding of the varied patient journeys and treatment pathways
  • Generation of more data to support the drug approval process

  • Identification of populations with enhanced benefit/risk

  • Development of evidence to support expanding the indication for a drug

It is important to make the collection of RWD simple, quick and consistent, to reduce the burden on the physician and patient, which will result in more complete and accurate data being reported. In our experience, most physicians and patients are happy to provide this data. Generally, there is no payment to physicians or patients for the collection of RWD in an Expanded Access Program (EAP). However, it is also perfectly acceptable for Sponsors to make a small site payment to cover physician time, if they feel it is merited.

When considering data collection during an EAP, there are several important factors to take into consideration.

  • Data can be collected from the physician and patient directly

  • Minimal data reconciliation should be required, due to the types of information captured

  • Use intuitive field design to eliminate data entry errors

The graphic below shows examples of data that can be collected during an EAP: