TOPRA Symposium 2023: Patient engagement in medicines’ regulatory lifecycle

Part Three: Patient engagement in medicines’ regulatory lifecycle

Maria Mavris (Patient Relations Department, EMA) and Adrea Furia-Helms (Director of Patient Affairs, FDA) sparked a discussion on patient engagement in the regulatory lifecycle of medicines.

Interactions with patients and consumers have been a progressive journey, with many different initiatives and organisations having been set up since 1996.

Patient engagement is increasingly recognised as a key element of the product lifecycle, with patient input being sought during the pre-submission, evaluation, and post-authorisation phases.

The European Medicines Agency (EMA) facilitates patient interaction in various ways, including international and European organisations, eligible organisations, and the Patients’ and Consumers’ Working Party (PCWP).

PCWP activities encompass workshops, information sessions, and contributions to new endeavours or strategies via public hearings.

The U.S. Food and Drug Administration (FDA) also runs Patient Affairs Programs and Activities consisting of various initiatives designed specifically to enhance patient communications.

Patient Listening Sessions allow for a better understanding of patients’ lived experience, as patients and caregivers can speak directly with FDA scientific staff.

Meanwhile, the Patient Engagement Collaborative is a collaboration between the FDA’s Patient Affairs and the Clinical Trials Transformation Initiative (CTTI).

Modelled on the EU’s PCWP, it offers a platform for the discussion of topics that inform patient engagement in medical product development and regulatory discussions at FDA.

Established in 2016 by the EMA and FDA, the Patient Engagement Cluster is a workgroup created to advance and strengthen international collaboration on patient engagement by sharing best practices for involving patients throughout the medicines’ regulatory lifecycle.

Health Canada joined the workgroup in 2023.

The importance of the patient voice is increasingly recognised as a key element of medicines’ development, as it provides:

• Insights into patients’ and caregivers’ issues, needs, and priorities
• Diverse opinions and expertise
• Insights into clinical trials, rick tolerance and potential benefit
• Real-world evidence

Maren von Fritschen (Head of Regulatory Policy, EU at Moderna), Kieran Breen (Research Advisor, Parkinson’s Europe) and Dr Joao Oliveira (Instituto Português de Oncologia de Lisboa) presented their industry perspectives and experiences, adding further evidence that the patient voice is valuable at all phases of drug development and that very early patient engagement is crucial.

Rachael O’Kane is a Manager, Regulatory Affairs at G&L.