EMA, in collaboration with other parties, has recently published two scientific articles outlining the importance of early interactions as an opportunity to improve the generation of evidence required for bringing innovation to patients.
An article, published in the British Journal of Clinical Pharmacology, analyses the importance of early advice for the generation of robust evidence after authorisation, once a medicine is used in clinical practice. Post-licensing evidence is often requested by European regulators following the approval of a medicine, to complement the understanding of a medicinal product‘s benefit–risk profile. This includes approvals using early access tools such as conditional marketing authorisation, or products which target rare diseases or are advanced therapies. Health technology assessment (HTA) bodies also see a role for the generation of post-launch evidence to address uncertainties identified during the clinical development. Applicants should have a robust plan for post-licensing evidence generation when they file their marketing authorisation application to increase the confidence of decision‐makers that knowledge gaps can be filled in a reasonable timeframe.
The second article, published in Nature Reviews Drug Discovery, discusses how to best support developers who are using new digital technologies in their medicines development programme. Digital technologies are increasingly becoming part of the conduct of clinical trials. This includes continuous monitoring of clinically relevant parameters or the collection of patient-relevant outcomes. When it comes to using data from such sources as evidence for benefit–risk evaluation of medicines, questions often arise about the extent to which digital technologies can be considered to be in line with, or more or less reliable than, already established means of data capture. This is a fast-evolving field and EMA is open to discussing proposals with applicants, even at an early conceptual stage, and providing them with comprehensive advice on the necessary aspects to ensure safe and effective use of medicines. To guide developers through such qualification, a dedicated question-and-answer document has been published by EMA.
Both articles aim to stimulate developers to discuss with regulators early and prospectively their evidence generation plans, and are the result of discussions at the industry stakeholder platform on research and development support . They are available through open access:
- Regulatory and health technology assessment advice on postlicensing and postlaunch evidence generation is a foundation for lifecycle data collection for medicines
- Digital technologies for medicines: shaping a framework for success