In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: The Added Value of Patient Engagement in Early Dialogue at EMA: Scientific Advice as a Case Study.
Today’s pick is from Murphy et al on an assessment of the added value of patient engagement during the scientific advice procedure published in Frontiers In Medicine doi 10.3389/fmed.2021.811855.
Drug developers can go to the regulatory agencies to get scientific advice. The European Medicines Agency offers scientific advice (SA) since 1996 and is coordinated by the scientific advice working party (SAWP). SA is not mandatory but can increase the chance of obtaining marketing authorisation.
The EMA is involving patients in most of their activities, including SA. Who better than the patients can provide insight in living with a condition on a daily basis? Here authors wanted to evaluate the impact of patient involvement in the SA process at EMA between 2017 and 2020.
478 surveys were sent to patients, 371 responded (very high response rate). Patients contributed to multiple areas (see figure). Other is mostly the daily impact of the disease. In 52% of cases, the contribution of the patients resulted in further reflection.
In 20% of cases the proposed development plan was changed based on patient input – in the 80% this was not the case, patients agreed with the proposed plan in 90% of cases. Patients can contribute to SA by attending meetings or providing input in writing.
EMA offers support to patients who provide SA e.g. to guide them through complex material and allow them to focus on areas their input is relevant. Patients valued the SA experience and the support offered.
Note that EMA is also offering training days for patients. Furthermore, of course EURORDIS also trains patients to contribute to drug development and regulatory engagement in the EURORDIS Summer School. Authors conclude that patient engagement has enriched the SA procedure.
Authors also mention limitations. e.g. 1-2 patients are often involved and they may not always represent everyone with the disease. Patient preference studies and focus groups are additional tools EMA is exploring in addition to direct involvement of patients during SA.
I like the publication but it would have been nice to ask companies to see if they valued the input from patients. Those that changed their development plan clearly did. Also note that companies can talk to patients also directly through EURORDIS community advisory boards.
Pictures by Annemieke, used with permission.