1 – This guidance is aimed at Sponsors who are planning to conduct clinical research to support a regulatory decision.
2 – For information about how patient data is collected and used, why research using patient data is essential for healthcare and benefits patients and the NHS, and the safeguards in place to protect patient privacy, visit Understanding Patient Data.
3 – There are vast amounts of data routinely being collected on patients, for example, in electronic health records (EHR), and disease and patient registries. Such data is commonly called real-world data (RWD), reflecting that it is routinely collected while patients go about their regular lives, as opposed to being specifically collected in a clinical trial. When such data is analysed to make inferences about the effects of different treatments, the information produced is similarly called real-world evidence (RWE). While extensively used for monitoring the performance of drugs and devices after approval, RWE is utilised much less frequently when it comes to demonstrating the efficacy or effectiveness of an intervention to gain an initial approval or an extension of an indication for an existing product…