DIA Europe Series

Integrating patient voice in medicine development and decision-making has introduced more quantitative approaches to generating patient input and evidence. IMI PREFER has been instrumental in developing the approach to gathering patient preferences, and work has been ongoing globally to develop more patient-relevant clinical outcomes. FDA and ICH work on patient-focused medicines development, and more guidance is expected in the coming years.

Even if it is acknowledged that patients are central to development programs and regulatory decision-making, there remain challenges to implementing the systematic evidence generation and subsequent use of this evidence in benefit-risk assessment, as well as securing patient involvement as early as possible for this purpose.

During our DIA Europe's last edition, we brought together patients, regulators, and researchers from public and private sectors to discuss lessons learned from the specific example of interactions on patient-generated evidence, practical solutions found and how these can help progressing patient engagement in other parts of the R&D and medicines evaluation processes:

Challenges encountered by Industry when developing the evidence and interacting with the regulators to engage with patients and include patient preferences and patient-centric outcomes in the product information.

The Regulators’ perspective on progress and experience using patient evidence for benefit-risk decision-making.

The Patient perspective on the issues, proposed solutions, and their impact on patient groups.

All you Need to Know About HTAR

With the regulation on Health Technology Assessment (HTAR) entering into force in January 2022 and becoming applicable in January 2025, the next two years will be critical for preparing for its implementation. National Authorities and other Stakeholders are preparing to adapt to a new system that has the potential to improve the availability of health technologiesfor EU patients.

In this free webinar, speakers from the European Commission, EUnetHTA 21, among others, will walk you through the critical steps of the HTA process and help you understand the basics of the EU HTA Regulation and its implications.

This session highlighted practical experiences by regulators from around the world and industry, leveraging case studies showing different collaborative and reliance pathways and discussing critical factors to identify effective reliance models that enable efficient use of resources and faster access to medicines.

As a non technical stakeholder, ever felt giddy with the Artificial Intelligence and Machine Learning domain, lingo, terminology, applications etc.? This session provides an overview of "must knows" using everyday human language, examples, as well as provide hands on use case examples for lesson learnt discussions. This session will broadly outline various AI and Machine Learning approaches, and importantly as a business stakeholder, what are the touch points, and what a project “looks like” (that is how an AI project should be managed). It also has a short hands on session, using Deep Learning as a method to highlight what a data pipeline can look like, and “do’s and don’ts”.

Workshop Outline:
• Intro to AL and ML – why? What is the value?
• AL and ML fundamentals, what non technical stakeholders should know. Reframing AI and ML in everyday human language.
• What is Deep Leaning? A non technical stakeholder “need to know” overview.
• Use Cases
• What’s involved in an ML project, touchpoints for non-technical stakeholders.
• What can go wrong in a ML project, typical problems
Live example using real data
• Q&A and discussion

This session discusses the many different initiatives on Decentralised Clinical Trials (DCTs) ongoing globally and analyses their unique objectives and perspectives, and how they interconnect and collectively move towards same aim of advancing DCTs to bring benefits to patients.

This session covers what the future looks like for pharmacovigilance and whether or not we need to make bold and innovative changes to the established approaches from a scientific, technological and regulatory standpoint in order to better protect patients. Are we having enough impact on healthcare systems? Are we focusing on micro level adjustments when we should be looking for a paradigm shift? Are we using the right technology to enable better and more efficient access to safety information? If we were to start with a blank sheet of paper what would a global pharmacovigilance ecosystem look like? During this session, you hear far-reaching discussions from pharmacovigilance thought leaders who challenge the status quo and leave us thinking differently about the future of pharmacovigilance.

Safe use of medicines is a key objective of pharmacovigilance. However, risk minimisation measures (RMM) have shown to have some limitations in effectiveness. It is hence suggested to study and implement enablers for knowledge and behaviour adoption for risk minimisation in healthcare. Potential enablers may emerge from engagement of patients and healthcare professionals to inform tools and designs of RMM, or from digital health transformation.

Regarding engagement, the Pharmacovigilance Risk Assessment Committee (PRAC) at EMA created in 2022 an innovative pilot forum for analysing options for additional RMM together with PRAC members representing patients and healthcare professionals, called the PRAC Risk Minimisation Stakeholder Alliance (PRISMA). This aims at adding knowledge on healthcare systems and processes, as well as informal input to PRAC for their decisions on additional RMM, with a specific focus on their implementability. Also, regarding potential digital support for RMM, EMA has established a multistakeholder drafting group for developing a reflection paper on opportunities and challenges.

Likewise, pharmaceutical industry has worked together to provide an overview of the existing experience with digital tools including the perspective from a multi-source/off-patent environment, in order to describe a more effective communication of additional RMM and propose solutions that embrace the advantages of digital health transformation.

The potential of above initiatives and the impact on RMM tool selection and design are discussed in this session with a wider multistakeholder audience.

The developing data landscape is changing both the way medicines are being developed and their continuous assessment. Real-world data/evidence (RWD/RWE) can bring value to every stage of a drug’s life cycle.This session provides an overview of how RWE has been used by regulatory and HTA agencies at pre and post approval stages; explores through case studies how RWD/RWE has or could be used in the review and reimbursement for rare diseases/early access medicines; discusses the use of RWD as part of integrated evidence generation plans by companies through lifecycle of medicines;, as well as the challenges that companies and agencies face in ensuring that RWD/RWE is fit for purpose.

This HTAi-DIA session presents and discusses guidance and an accompanying tool that may facilitate the identification, communication and mitigation of uncertainty in HTA and regulatory decision-making by global, collaborative stakeholder engagement.

In 2023, the European Commission unveiled a new legal proposal on EU Pharma Legislation, Orphan and Paediatric Regulation. Watch this captivating session to assess proposed "horizontal measures" in the new framework and see what the audiences' thoughts about their potential were to support innovation and enhance the EU's regulatory framework competitiveness.

Watch this exciting discussion on the field of Dynamic Regulatory Assessment (DRA) and witness this significant multi-stakeholder engagement in a pivotal year 2023 with new legislation under the Pharmaceutical Strategy.

In continuation of the dialogue at previous DIA Europe conferences, this session provides an opportunity for further reflections on lessons learned from the response to the COVID-19 pandemic. The success achieved in the CMC development, manufacture, authorization and supply of new therapies and vaccines for COVID-19 in very rapid timescales provides a rich set of experiences. It built significantly on the outcomes from the collaborative work between industry and regulators in recent years, on the role of prior knowledge and innovative science and risk-based CMC strategies to the development of new, high-quality products for unmet medical needs. The challenges for rapid development & commercialization, as well as various approaches to facilitate accelerated development and global supply post initial authorization, will be revisited from a “post-pandemic” point of view. This will include the role of dialogue between sponsors and agencies to facilitate early decision-making and alignment, the value of improving reliance/collaborative assessments and increased collaboration between regulatory authorities (e.g. at the level of ICMRA) to reduce differences in global regulatory requirements and ultimately achieve earlier and greater access to high-quality critical medicines for patients worldwide.

Objective: Learn how to navigate the Quality aspects of accelerated pathway/reliance & work-sharing initiatives and manage the “flood” of post-approval changes (PACs) appropriately. The session covers the entire lifecycle.

The recent implementation of the MDR in Europe, specifically Article 117 for drug-device combination products, still presents challenges for manufacturers, Notified Bodies, and EMA, as additional clarity is needed related to submission content and the review process of significant changes requiring a Notified Body Opinion. This could be further impacted by the upcoming revision of the EU Pharmaceutical Legislation, which may include new approaches to combination product regulation in the EU. This session describes current challenges, proposed criteria for determining significant changes, and calls for additional clarity and proposed solutions on these issues, including upcoming guidance on this topic.