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.

This session looks back at the effects the new ways of working have had on the Digital Regulatory Business Transformation in Europe and its impact on stakeholders´ resources and planning.

The session’s objective was to underscore the urgency of environmental change in healthcare.
In this session, we explored recent European policies and regulations that propel sustainability and examined the healthcare industry’s audacious measures to reduce carbon emissions in drug manufacturing, mitigate the environmental impact of clinical trials, and establish benchmarks for their initiatives.
The session concluded with a dynamic discussion on simple steps we can all take to shrink our environmental footprint.

Unmet medical need (UMN) is a provision in the pharmaceutical legislation for human medicines, but experience has shown that it can be difficult to apply (read more in the linked article below). In this session, the speakers presented the current perspectives of UMN, discussed new proposals for a definition that have emerged from the proposal for the revision of the pharmaceutical legislation, and debated options and challenges for their application.

The EU HTA Regulation will change the HTA decision making landscape in EU and preparation is in full speed by all stakeholders. This session will address this and other hot topics in the HTA arena. As the EU HTA Joint Clinical Assessment (JCA) will parallel the review process by Regulators, time for preparation of both dossiers, review, decision making and alignment with subsequent national reimbursement processes will provide new challenges for industry, HTA bodies and Regulatory Agencies. Patients and Physicians expect that access in innovation will be facilitated.

This session will outline the challenges from various stakeholder perspectives and provide a vision on how all can best interact during the procedure while keeping the remit of the various assessment bodies. As an outcome we expect recommendations - what gaps still need to be closed on our way to full implementation. Several implementing acts are still in drafting stage.

In this Session, the DIA team and the DIA Europe Programme Committee will go through the main outcomes from each Track, conclusions and identified next steps. Join us to gather fruitful outcomes and a general overview of the most important outcomes from DIA Europe 2024!

The EU landscape of policies and regulations governing medicines research, development and manufacturing becomes increasingly complex. Products become more complex and more often than not consist of components regulated by different policies. But there are also unintended consequences where there is a pre-existing link between, for example, food and pharmaceutical rules, or where such link is created when new rules are set up.

Developers of medicines must keep an eye not only on the legislation and guidance directly governing pharmaceutical products R&D and manufacturing but also on the interplay between other sectorial policies and legislations driven by considerations specific to food, device, diagnostics, chemicals, environment, data, etc. As a result, these other policies and regulations can either add to the evidence requirements increasing complexity and cost, or even contradict in some instances the principles on which medicines quality, safety and efficacy rely.

The session will bring together medicine developers, policymakers, national and European regulators from different domains, patients and academics to shed light on examples of such complexities, their impact, pragmatic mitigation measures and general principles that should drive policy development to avoid that complexity turns into complications that prevent R&D or manufacturing in Europe.

The session will focus on the Accelerating clinical trials in the EU (ACT EU) initiative, which aims to transform clinical trials in the European Union. The keynote speech will outline the European Medicines Regulatory Network’s vision for clinical research in the region, reflecting on the progress towards better, faster and optimised clinical trials and highlighting key milestones under ACT EU. A panel of stakeholder representatives will exchange views on the main challenges and opportunities for improving the clinical trial environment in Europe.

The concept of unmet medical need (UMN) plays an important role in investment and priority-setting decisions by a range of stakeholders, including regulators, HTA agencies, payers, academics and the pharmaceutical industry. Identifying a particular condition or disease area as an UMN is intended to signal its health policy significance, stimulate research activities and incentivise the development of innovative treatments, diagnoses or health technologies in these areas. Incentives associated with the identification of an UMN can take the form of preferential access to public research funds, access to alternative or accelerated regulatory pathways, consideration of UMN as a value element in HTA, and financial incentives or innovative payment models in reimbursing the health benefits a new treatment delivers.

As UMN should help to shape policy and action from early phase research, through clinical development to pricing and reimbursement to how a new medicine is used in practice, how the research and healthcare communities define and quantify unmet medical need is challenging as every patient’s perspective is very personal according to their own experience of living with disease and each constituency’s view of unmet need is formed by their own professional expertise and opinion.??

As the European Commission proposed to define the concept of UMN in the current legislative review, the concept is broadly discussed with divergent views. The session will bring together key players in pharmaceutical innovation from developers, health and regulatory authorities, patients to discuss Europe’s ambition for a definition of unmet medical need, what is needed and what are challenges.

Dive into the future of regulatory excellence with in this DIAmond session, on "Digital Transformation in the EU Regulatory Space."

This session will focus on the concrete plans that have been made for digital transformation in the regulatory space for the next ten years and gathers Healthcare regulators, European Commission representatives, Patient advocates, Technology disruptors and Industry leaders to explore the critical success factors driving digital evolution. Join us and be part of the conversation that shapes the next era of regulatory dynamics. Don't miss your chance to navigate the transformative journey ahead.

This session will focus on the global regulatory frameworks for enabling innovation in manufacturing and quality. Innovation includes the development and manufacture of new types of products, new manufacturing platforms, innovative technologies such as model-based controls, the wider scope of and new analytical technologies. The discussion comes at a critical time as the implementation of new digital tools and manufacturing technologies are key to the evolution of manufacturing, which can be enabled by tools such platform technology master files, The session will also reflect on how groups such as the new EMA Quality Innovation Group is seeking to lead the transformation of medicines’ manufacturing in Europe and how this is linked to fast moving initiatives outside of Europe, such as FDA FRAME and platform design technology programs, ICH, and ICMRA collaborative initiatives under the PQKMS umbrella, including collaborative assessments. The discussion will also focus on how collaboration and the identification of risk can already enable technology.

This session will focus on the emerging EU AI regulatory and policy landscape / ecosystem and how it relates to medicines development, with a specific focus on the EMA/HMA’s draft reflection paper on AI in medicines. With perspectives from regulators, legislators, patients and industry, we will discuss how these frameworks could impact the development of AI in medicines and explore practical recommendations to foster the development of reliable and responsible AI across medicines lifecycle development.

.

The session will provide multistakeholder views on ongoing activities happening in the landscape, including the first milestones in Collaborate, as well as highlights from ACT EU programme, including a wider European perspective on development of risk-based approaches.

Learning Objective

The objective of the session is to understand the progress and lessons learned during the first year of experience following full implementation of the EU CTR. It aims to give a voice to regulators, ethics committees, trial sponsors, and patients via presentations and active debate on the advantages of the system and ways to maximizing its potential for the benefit of patients and EU research.

This session will discuss the evolving global landscape for decentralised clinical trials, to (1) impact of recent and ongoing DCT initiatives and activities by various stakeholders (2) evaluate where we are in fulfilling the promise of DCTs for each stakeholder (3) debate what still needs to be done to advance the use of decentralised elements.

The session will focus on the impact of the Green Deal initiative as priority of the EU which will have an impact on the healthcare sector, particularly on the continuity of pharma R&D and manufacturing.

The session will bring together those who are engaging in developing relevant EU chemical and environment legislative and non-legislative dossiers (Commission, regulators), and those who will be impacted (industry, suppliers) to discuss three identified case studies and discuss opportunities. This track will provide insights into how manufacturers and regulators can collaborate to deliver this revolution without risks to the supply of medicines. Participants will be invited to share their concerns and ideas to progress innovation and concepts in moving towards a more sustainable future of the healthcare sector.

This session will focus on the regulatory framework for product Life-cycle Management. In particular, the participants will discuss the impact of the new ICH M4Q (R2) and the guidelines developed by the Expert Working Group; the new EU variation guideline and the opportunity for pragmatic approaches to PAC (Post Approval Changes) and the implementation of PAC using ICH Q12 principles, harmonization of regulatory requirements and the use of reliance to enable accelerated implementation of changes globally.

Key stakeholders including regulators, industry and patients are invited to discuss the impact of the current and new regulatory legislation on PACs.

This session will provide a platform for scientific exchange among the EMA, the EC DG Research and other global health authorities (e.g. FDA), patient advocates, and drug development innovators on the evolving global landscape for precision medicine

.

This session will provide stakeholders an in-depth information session on selected ICH guidelines (efficacy, quality and safety) and will give stakeholder the possibility to interact with the experts that have contributed to the harmonisation work.

This session will offer a platform for dialogue on the operationalization of AMA and will discuss proposals on improved collaboration in Africa on regulatory harmonisation and convergence, application of reliance pathways and digitalisation.

This session will offer a platform for dialogue on the operationalization of AMA and will discuss proposals on improved collaboration in Africa on regulatory harmonisation and convergence, application of reliance pathways and digitalisation.