Wien, 17.05.2023 – Text: Catarina Carrão
The patient’s voice is essential in the different regulatory activities throughout a medical product’s lifecycle. An individual’s real-life experience as a patient living with a condition offers a crucial complementary perspective to understand the value of the scientific evidence necessary for the evaluation and management of medical products. The collection and use of evidence-based patient experience data not only helps benefit/risk-decision making but can also improve the quality of the medicines itself. This engagement fosters further trust among patients and consumers regarding regulatory decisions when new medical products are brought on to the market. Afterall, successful adoption of legally marketed medical products increasingly depends on the patient’s acceptance of that technology, and them being more engaged in the healthcare process – along with demonstrated public health benefits, of course.
At the moment, regulation (EC) No 726/2004 gives responsibilities to the European Medicines Agency (EMA), its Management Board and its various scientific Committees to develop contacts with patients and consumers1, 2. The Pharmaceutical legislation also defines the framework for providing them with clear and useful information, with a call for the implementation of specific forms of interactions with these stakeholders1. Furthermore, according to Murphy and colleagues3 published report “The added value of patient engagement in early dialogue at EMA: scientific advice as a case study” (Frontiers in Medicine, January 2022), patient input in early clinical development resulted in further reflection by EMA’s working party in 52% of the study cases in terms of endpoints, study population, feasibility and quality of life. Moreover, one in five scientific advice responses provided to the medicine’s developers were actually modified; and, in 90% of the cases where no modification was made, patients agreed with the proposed development plan. This analysis supports the continued involvement of patients in scientific advice and illustrates the importance of including this stakeholder group in an early dialogue between sponsors and regulators.
In January 2022, EMA defined four levels of stakeholder involvement aligned with the EC Better Regulation Guidelines [SWD (2015) 111 final]: (1) Inform, (2) Consult, (3) Consult and Involve, (4) Cooperate and Participate1. Additionally in January 2023, a concept paper was released by EMA on a EU multi-stakeholder platform for improving clinical trials4. The scope of the platform is to involve all aspects of clinical trials, including design, conduct, statistical analysis, proposal of revision of regulation(s), transparency of data and patient engagement. The aim is to mitigate some of the practical challenges associated with robust clinical studies, including challenges concerning study/research participant enrolment and retention in the study, particularly when protocols include lengthier follow-up periods (e.g., 2-years post-procedure), and/or frequent visits to the clinical site. Also, clinical studies prospectively designed with input from diverse patient advisors – including those from racially and ethnically diverse populations, may help address common real-world outcome challenges that clinical trials currently face.
A current complain from sponsors is that it is challenging to find patient advisors knowledgeable about clinical study methodology; and, to determine which patient advisors or patient organizations should be engaged – and if multiple patients’ advisors are engaged, how to reconcile the disparate perspectives. The European Innovative Health Initiative (IHI), the world’s largest Public Private Partnership in the life sciences, aims to address some of these issues and to include patients at every stage of its funded research projects5. Currently there are no open platforms for patient expertise supported by the initiative since the European Patient’s Academy on Therapeutic Innovation (EUPATI) recently closed6. Although, there is a recent open call from April 2023 for the re-opening of the pool of patient experts, that was previously supported by the Innovative Medicines Initiative (IMI). The deadline for applications is 8 June 2023 – so, if you are a patient and want to have your voice heard in the clinical trial processes, this is the right place to go.
1. EMA. Engagement Framework: EMA and patients, consumers and their organizations. EMA/649909/2021. 2022;Stakeholders and Communication Division(https://www.ema.europa.eu/en/documents/other/engagement-framework-european-medicines-agency-patients-consumers-their-organisations_en.pdf)(20 January 2022):https://www.ema.europa.eu/en/documents/other/engagement-framework-european-medicines-agency-patients-consumers-their-organisations_en.pdf.
2. EC. Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency (Text with EEA relevance). In: UNION TEPATCOTE, editor. Regulation (EC) No 726/2004. https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=celex%3A32004R0726: Official Journal L 136 , 30/04/2004 P. 0001 – 0033; 2004.
3. Murphy A, Bere N, Vamvakas S, Mavris M. The Added Value of Patient Engagement in Early Dialogue at EMA: Scientific Advice as a Case Study. Original Research. Frontiers in Medicine. 2022-January-20 2022;8doi:10.3389/fmed.2021.811855
4. EMA. Priority Action 3 concept paper: an EU multi-stakeholder platform for improving clinical trials. Accelerating Clinical Trials in the EU (ACT EU). 2023;https://www.ema.europa.eu/en/documents/scientific-guideline/priority-action-3-concept-paper-eu-multi-stakeholder-platform-improving-clinical-trials-accelerating_en.pdf(EMA/41656/2023)
5. IHI. European Innovative Health Initiative (IHI). https://wwwihieuropaeu. 2023;Assessed 16.5.23
6. IHI.eu. Patient engagement: display results. Innovative Health Initiative. 2023;https://www.ihi.europa.eu/site/search?keywords=patient+engagement(Assessed 16.5.23)