Patient voice: Shaping the future of medicine regulation

Our first article, by Mandy Daly, Director of Education and Research at the Irish Neonatal Health Alliance, gives an overview of the evolving landscape of public and patient involvement (PPI). In recent years, there has been a positive change in the importance of collaborating and partnering with patients, carers, service users and the public to ensure that the views of the patient community are included in research, academia, health and social care and policy-making. This powerful article uses the real-life experience from the author to demonstrate the importance of patient involvement.

Our second article looks at the level of patient involvement in regulatory processes. As well as being increasingly recognised as an essential part of research and development in medicines, patient involvement is also starting to be actively sought in the area of regulatory guidance generation. This article, by Maria Dutarte of EUPATI and Birka Lehmann, demonstrates how patients offer unique insights to consider during the review of new medicine applications and the generation of patient information. However, while the European Medicines Agency’s processes for involving patients are well documented, less is known about the practices of National Competent Authorities (NCAs) across different EU Member States. This article reviews the findings of a recent EUPATI European-wide survey which was set up to better understand the interaction of NCAs with individual patients and patient organisations. As a result, it calls for increased patient education, a higher volume of available data from patient groups, and the need to harmonise best practices for patient involvement across the EU.

Our third article reviews the importance of patient advocacy in the orphan drug designation (ODD) process. Catherine Moncad, Vice President of Regulatory Services at TMC Pharma Services Limited, examines the European Medicines Agency’s (EMA) evolving engagement framework and the tools available to help define unmet patient need. It also provides key points for regulatory teams to ensure we drive more patient-centred outcomes. This article contains key points for regulatory teams supporting orphan designation applications to consider around the value of patient inclusion at this early stage of development.

Our fourth article, by the European Federation of Pharmaceutical Industries and Associations (EFPIA), moves into the area of clinical trials. It summarises an EFPIA paper which discusses the potential opportunities of randomised pragmatic trials (RPTs) and how they bridge traditional randomised clinical trials and non-interventional studies. It highlights that this clinical study method integrates routine clinical practice with patient experience, leading to clinical data across a more diverse patient population and a better reflection of patient settings. This approach to clinical studies also links to the EMA’s aim to establish the value of real-world evidence in decision-making by 2025 and the Accelerating Clinical Trials (ACT) EU initiative. The summary covers an overview from the paper of the key challenges for potential sponsors of RPTs, as well as the regulatory considerations for this approach.

Our fifth article, by Heidi Stauffer-McDonald and Sean Schofield of Thermo Fisher Scientific, remains in the area of clinical research. It provides not only an overview of patient representation in clinical trials, but also emphasises the importance of including all clinically relevant populations, and the impact this has on regulatory compliance, market access and the understanding of benefit-to-risk ratios. It offers strategic approaches that can be used by sponsors to promote diversity in clinical trials and includes an overview of the regulations and guidance in this area from the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) and a number of markets across the world.

Looking at other areas, our final article gives a review of the US Food and Drug Administration (FDA) draft guidance for manufacturing changes and comparability of cellular and gene therapy products. Written by Laura Guijarro, Rajendra Kunda, Slobodanka Cirin-Varadjan and Hao Liu at Syneos Health, it compares the proposed changes in this new guidance against existing guidance available for this product type and identifies key changes that will need to be considered by manufacturers and regulatory teams moving forward.

I hope you enjoy this issue.