On 2 and 3 March, the European Medicines Agency (EMA) hosted a joint meeting of its Patients and Consumers (PCWP) and Healthcare Professional's (HCPWP) Working Parties. Held over two days, and attended by over 70 representatives of patient and HCP organisations, the meeting included updates on COVID-19 vaccines and therapeutics, advanced therapy medicinal products (ATMPs), personalised medicine and Big Data.
In November 2020, Emer Cooke began her mandate as the Executive Director of the EMA. Dr. Cooke kicked off day 1 of the PCWP/HCPWP meeting by welcoming all attendees and introducing herself to the assembled audience, emphasising that the EMA places great value on its interactions with PCWP and HCPWP members. Marco Cavaleri and colleagues then provided a detailed update on COVID-19 vaccines and therapeutics, noting that vaccine hesitancy has proved a particular challenge in recent months. In response, the EMA are prioritising public engagement and transparency for vaccine information, providing updates through their website, social media feeds and via public-facing webinars, including one to be held on 26 March. Dr. Cavaleri reported encouraging data on the immunogenicity of ChAdOX1, the AstraZeneca/University of Oxford vaccine that was approved by the EMA in January, and identified three key classes of COVID-19 therapeutics currently being discussed: SARS-CoV-2 antibody combinations, immunomodulators and anticoagulants.
Next, an update was provided on medicines development and authorisations, focusing on the European Reference Networks (which bring together patients, specialists and other stakeholders in specific rare or low-prevalence disease areas) and ATMPs such as gene and cell therapies. Martin Dorazil, deputy Head of DG SANTE at the European Commission, identified the key assets and achievements of the ERN portfolio, including greater coordination between dispersed stakeholders and the development of new clinical practice guidelines. In particular, discussions on ERNs and ATMPs emphasised the importance of real-world data, obtained through routine clinical practice, in supporting health policymaking and post-authorisation follow-up.
Day 2 of the PCWP/HCPWP meeting was primarily focused on personalised medicines and Big Data. Personalised medicine uses information about a person's genes or proteins to prevent, diagnose or treat disease. Anthony Humphries of the EMA and Ejner Moltzen of ICPerMed (the International Consortium for Personalised Medicine) outlined how discoveries from frontier research on the genetics and molecular basis of disease are slowly progressing towards implementation in healthcare, supported by the growing adoption of electronic healthcare records by EU Member States. Looking ahead, ICPerMed see greater patient involvement in personalised medicine as crucial to progress, and are working towards the launch of a European Partnership in personalised medicine (EP PerMed).
On the Big Data front, Peter Arlett (EMA) and Nikolai Brun (Heads of Medicines Agencies/HMA), co-Chairs of the Joint Steering Group on Big Data, reported on the activities of the group in 2020, which included the launch of a collaboration roadmap for real-world evidence, data protection workshops and the development of education programmes for Big Data upskilling. Looking ahead to 2021, much of the focus will remain on real-world evidence, with deliverables on data quality, workshops on AI and meta-data, and continuing pilot activities of the European Health Data Space. In particular, the Steering Group will continue to develop DARWIN EU (Data Analysis and Real-World Interrogation Network), a network of data, expertise and services designed to support better decision-making by EMA and other regulatory committees. Drawing the two-day meeting to a close, Fergus Sweeney provided an overview of an updated guideline for Good Clinical Practice (GCP), a process that ensures ethical and scientific quality standards for clinical research are met. The updated guideline will provide enhanced guidance on GCP for trials that use innovative designs and/or use novel data sources. A key principle for the guideline is respecting the rights, safety and wellbeing of trial participants, and ensuring that risks borne by participants are proportionate to the expected benefits and importance of the trial objectives.