MAPPs and New Benefit Risk Models: IMI2 Leads with Patient Engagement

The Innovative Medicines Initiative (IMI) yesterday launched its 5th Call for proposals under the IMI 2 programme (  Topic 1 addresses one of the key enablers of adaptive pathways – developing methods to incorporate the input of patients into the assessments of benefits and risks of new medicines at all stages of the drug development life cycle.

This is a vital piece of the MAPPs (adaptive pathways) puzzle in Europe. As MAPPs require a mutli-stakeholder agreement on the clinical endpoints and success metrics before the trial is launched, the current lack of a standardized approach and understanding of how to measure and quantify the patients understanding of benefit risk in a theoretical MAPPs trial is a very large constraint to their implementation.

According to IMI2 call 5, “There is an emerging consensus among stakeholders [that the] patients’ values and perspectives should inform the decisions taken during the development of medicines, as well as during the approval and post-approval phases. Stakeholders recognise that the robustness and transparency of pharmaceutical development, regulatory approval deliberations and health technology assessments (HTA) have been improved by information on patients’ values.”[1]

By addressing the need for broad agreement on how patient input into benefit risk assessments will be used in granting a MAPPs approval, IMI2 will help provide the necessary tools for improved HTA and reimbursement decisions across the EU member states. The call will facilitate a multi-stakeholder environment where new benefit risk models will be developed, tested, and applied with patients at the centre of the decision matrix.

To deliver on this IMI Call, the consortium will have to mobilise expertise in the area Patient Preference Research coming from various backgrounds – health technology outcome research, pharmaceutical and device development, biostatistics, epidemiology, behavioural research, experts in scientific communication to lay audiences, and experts in benefit-risk decision making to name but a few. Patient expertise is the key to success in this action.

The goal of any Applicant consortia is to enable the effective collaboration between key stakeholders, i.e. health-care professionals, regulators, and HTA bodies, as a prerequisite for success to facilitate the use of data gathered by the identified methodologies in the decision-making process. Therefore, the successful consortium will either include representatives of these key stakeholder groups and/or have the ability to bring the necessary stakeholders on board.

These are not typical IMI research consortia! We need your help to pass this information to all those listed above. More information on the call is available here:


Magda Chlebus

Magda Chlebus, Executive Director of Science Policy & Regulatory Affairs at EFPIA, is in charge of policy and...
Read Morechevron_right