Open Consultation: HTA and Payers’ Use of Real-World Evidence in Decision-Making on Highly Innovative Technologies

A Brussels-based informal multi-stakeholder group, the “RWE4Decisions Task Force”, has been meeting over the past months to discuss the use of real-world data/evidence (sometimes referred to as RWD or RWE) and its potential uses in decision-making by payers and HTA bodies for assessing highly innovative technologies. those that use “ground-breaking, scientifically advanced mechanisms to target the underlying cause of a disease to deliver highly effective, potentially curative, or at least transformative, patient benefit. Examples include direct-acting antiviral therapies for hepatitis C, and the cell and gene therapies being developed for a range of conditions.

At stake are significant potential benefits for patients with high unmet needs. Such treatments may also go through regulatory assessment with limited data, using one of the accelerated pathways available under EU law. This creates a dilemma for HTA agencies and payers, who must balance the claims of effectiveness and benefit with often a high price. Value can be hard to determine based on limited data and time.

The present work, commissioned by the Belgian healthcare payer, explores ways in which RWE could be used to fill in such gaps in knowledge. It should be useful for wider discussions on the potential of RWE in assessing highly innovative new technologies.

The subject is clearly of great relevant to patients, whose contribution to HTA evaluations and reimbursement decisions on new treatments can be crucial. Patients’ perspectives must be fully integrated in assessment of the value of any new therapy. Patients are also the “owners” of their data so should have a say in how this data is used.

Recommendations for stakeholders – open for consultation 

The group has now published a draft paper that is open for consultation until 6 January 2020. The introductory elements of the paper will be developed into a formal publication, but the group is inviting comments from all international stakeholder groups on the proposed actions for stakeholders (sections 4 and 5).

We would encourage all patients/patient representatives to take a look and comment! If you have additional comments that you cannot submit via the online survey, please send them to EPF by emailing 

Is your patient organisation involved in using RWD?

The topic will be on EPF’s work programme in 2020 and we would like to hear from our members who are active in this area. Let us know what you are doing, and if you would be interested in contributing to EPF’s activities.

More information