EUCOPE's Sounds of Science

EUCOPE's Sounds of Science - Episode 18 On this episode, we talk about the revision of the General Pharmaceutical Legislation (GPL). We discuss the reports adopted on 10 April by the European Parliament, less than a year after the Commission’s proposal was released, focusing on the incentives framework and the regulatory provisions. Not only we will discuss the proposals adopted by the Parliament, but we’ll compare them with the Commission’s approach. We will also share our thoughts on how the revised framework might impact small and mid-sized companies and their investments in innovative treatments in Europe.   The guests of this episode are: Victor Maerterns, EUCOPE’s Government Affairs Director. Dr Seán Byrne, EUCOPE’s Senior Manager Legal and Regulatory Affairs. Stefano Romanelli, EUCOPE's Senior Government Affairs Director.

SOUNDS OF SCIENCE - EPISODE 17 On this episode, we explore the topic of Real-World Data and Real-World Evidence – that is data that is collected in a real-world setting, such as in healthcare institutions rather than through controlled experiments. We examine how and why this information can be useful for biopharma companies and health authorities, in order to bring new innovative therapies to patients. We will also look into some of the challenges with using this type of data, and view how a multi-stakeholder initiative - RWE4Decisions - is tackling those issues. Our special guests include: Karen Facey, Senior Advisor HTA, RWE4Decisions Carlos Martin Saborido, Scientific Officer, Spanish Ministry of Health Alexander Natz, Secretary General, EUCOPE =================================================================================================================== For more information on EUCOPE’s initiatives with Real-World Evidence or how your organisation can contribute to it, please contact Matias Olsen [email protected] If your company would like to put forward a topic for us to discuss or to participate in a future podcast episode, please reach out to our producer, Dante Di Iulio.

EUCOPE's Sounds of Science - Episode 16 On today’s episode, we explore the revision of the Orphan Medicinal Products (OMP) Regulation as part of the Pharmaceutical Package, and its impact on small and mid-sized pharmaceutical companies, the key drivers of innovation in Europe. We’d like to deep-dive into the implications of specific Commission proposals, and how innovative pharmaceutical companies see the introduction of concepts such as high unmet medical need as the basis for a modulated incentive framework. EUCOPE is no stranger to proposals to modulate the orphan incentive, having worked with the multi-stakeholder Expert Group on Orphan Drug Incentives to establish an alternative model, and today’s guest played an important role in developing that approach. Building on the success of the past 20 years, and establishing a system that continues to drive research, and crucially address the 95% of rare diseases that have no established treatments is no small task. This requires creative and alternative solutions, and small and mid-sized innovative pharmaceutical industry is keen to play its role. To help us understand the impact of this review, and how the European Innovative pharmaceutical industry sees the current proposal, we’ve got two rare disease experts joining us today: Diego Ardigò, Head of Research & Development, Chiesi Global Rare Diseases Alexander Natz, Secretary General, EUCOPE ============================================================================================================= For more information on EUCOPE’s efforts on rare diseases and orphan drugs or how your organisation can contribute to it, please contact Victor Maertens [email protected]

SOUNDS OF SCIENCE - EPISODE 15 On today’s episode, we focus on the EU’s Medical Device Regulation (MDR) which came into full effect on 26 May 2021 and followed by the In Vitro Diagnostic Device Regulation (IVDR) the following year. The idea of both regulations is to balance the regulatory review and approval processes of medical devices and IVDs across all EU Member States, essentially harmonizing the European market for medical devices. However, the Medical Device Regulation is causing some problems, and stakeholders and politicians at both the national and EU levels are warning that the MDR is leading to potential medical device shortages, and even medical devices disappearing from the EU market as a whole. In short, the main problem is that under the MDR, all medical devices produced in Europe must be re-certified. Re-certification needs to be done by a notified body, which is a national organisation designated by an EU country to assess the conformity of devices placed on the market. The main reason for the shortages is the capacity of these notified bodies to recertify. There are currently only 36 notified bodies with 23,000 certificates that need to transfer very much at the same time. Also, most manufacturers are not prepared for the new and sometimes costly rules of the MDR. In light of all this, and primarily to prevent the imminent risk of shortages, the European Commission decided to amend the medical device and in vitro diagnostic regulations. To help us breakdown the recent changes and what it means for EU medical device companies, we’re joined by three experts in the medical devices space: Jörg Plessl, Head of Global Regulatory Affairs, Norgine Megan Doyle, Director, Global Regulatory and R&D Policy, Amgen Axel Korth, Sr. Legal and Regulatory Affairs Advisor, EUCOPE  --------------------------------------------------------------------------------- If you're a medical device company looking to add your voice to the ongoing debate and need help navigating the complex EU medical device market, please reach out to EUCOPE's Leander Vranken ([email protected]) to learn how to join our MDR/IVDR Focus Group.

SOUNDS OF SCIENCE - EPISODE 14 With the passing of the Inflation Reduction Act, significant changes are coming for drug pricing and payment in the United States. At the same time, the upcoming revision of the EU Pharma Package will have a significant impact on the competitiveness and predictability of the EU biopharmaceutical landscape. There are several gaping differences between the biopharma ecosystems of the US and the EU. For instance, it takes on average 150 days longer to get an innovative medicine approved in Europe than in the US – time that could be crucial for some patients. To understand what is happening in policy and regulatory developments in both the EU and US markets and how they will impact biopharmaceutical innovation for the foreseeable future, our latest podcast gathers first-hand perspectives from our American counterparts and details how we can work together to build trans-Atlantic bridges for innovation in life sciences. Our special guests include: Justin Pine, Senior Director, International Affairs – Global IP and Data Policy, Biotechnology Innovation Organization (BIO) Ben Bradford, Vice President of Economic Development & Workforce, MassBio Alexander Natz, Secretary General, EUCOPE ============================================================================== Join EUCOPE at the BIO International Convention from 5-8th June in Boston, Massachusetts to Stand up for Science. Register here

SOUNDS OF SCIENCE - EPISODE 13 This year’s Rare Disease Day comes at a crucial time, weeks before we expect the publication of the pharmaceutical package which will revise the General Pharmaceutical Legislation, Paediatics, and OMP Regulations, which will set the tone for the EU rare disease ecosystem for at least the next 2 decades. The theme for this year’s Rare Disease Day is equity, so we wanted to take the opportunity to dive into some of these debates and asses what the EU can do in this space, especially against the backdrop of the legislative review. To breakdown this year’s Rare Disease Day and its importance for addressing the 95% of rare diseases with no treatment, we’re joined by: Simone Boselli, Director of Public Affairs, EURORDIS - Rare Diseases Europe Alexander Natz, Secretary-General, EUCOPE ______________________________________________________________________ For more information on EUCOPE’s efforts on rare diseases and orphan drugs or how your organisation can contribute to it, please contact Victor Maertens [email protected] or visit our Rare Disease Hub. 

SOUNDS OF SCIENCE - EPISODE 12 With the entry into force of the EU HTA Regulation in January 2022, the European Commission and EUnetHTA21, a consortium of Member States HTA bodies, have been racing to develop the procedural rules and the methodology for EU Health Technology Assessment (HTA). This is before the first medicinal products will be subject to the new procedure starting 12 January 2025. The new EU HTA procedure will significantly change the requirements before placing innovative medicinal products on the market, with certain new products having to go through a joint European assessment as a first step. On today’s episode, we’ll review what’s happened in the final drafting stages that took place over the course of 2022 and if this new procedure will replace the fragmented patchwork of national frameworks that currently exist, or if it will only represent yet another step in the process. It's crucial to get this procedure right at the first go in order to speed up patients' access to innovative therapies. To help us do so, we’re joined by two EU HTA experts: Ana Palma, Senior Director Global Patient Access & Access Policy, Sobi Alexander Natz, Secretary General, EUCOPE _____________________________________________________________________ EUCOPE's EU HTA Regulation Task Force has been set up to more closely coordinate with members on the engagement with the European Commission and EUnetHTA 12 for the development of the procedural rules and methodology respectively. For more information on how your organisation can contribute to it, please contact Matias Olsen [email protected]

SOUNDS OF SCIENCE - EPISODE 11 Throughout 2022, there have been successes, challenges, delays and change within the European pharmaceutical industry and we expect more to come in 2023. On the final episode of the year, hear the EUCOPE Policy team - Victor Maertens, Matias Olsen and Leander Vranken - unravel the past year in European pharma and biotech, looking at everything from rare diseases to ATMPs to EU HTA to Digital Health, sharing key highlights and what to expect in 2023. We will be back with more amazing episodes and insights next year, so stay tuned and stay healthy!

SOUNDS OF SCIENCE - EPISODE 10 Within the European Commission’s ongoing review of the General Pharmaceutical Legislation and Orphan Medicinal Products (OMP) Regulation, there are several potentially controversial policy options that aim to enhance access to medicine and overcome unmet medical need, including launch conditionality. Under the proposed system, companies would only receive their full Regulatory Data Protection if they meet a number of predefined conditions. Two conditions under consideration are that companies must launch the product in most, if not all, EU Member States within a fixed timeframe and address a yet unspecified unmet medical need. Today’s discussion focuses on the launch condition, which is far-reaching and creates additional risk for small and mid-sized companies to launch products – particularly orphan medicinal products (OMPs) – in the EU. Small and mid-sized companies are key drivers of biopharmaceutical innovation in Europe. They play an important role in the development of new and underserved medicines, so it is crucial that their needs and interests are looked after in order to reinforce an innovative and competitive biopharmaceutical ecosystem in Europe. The EU should avoid implementing policy that discourages innovation, disproportionally punishes small and mid-sized companies and might limit, not improve access. To understand the potential impact of the launch condition on the small and mid-sized health technology company, we’ve invited two of our members on the show today: Johanna Grames, Senior Manager for International Governmental Affairs and Health Economics at AOP Health Thomas Bols, Government Affairs Manager, PTC Therapeutics Alexander Natz, Secretary-General, EUCOPE

SOUNDS OF SCIENCE - EPISODE 9 On today’s episode, we will be exploring the topic of Advanced Therapy Medical Products, or ATMPs. This covers a wide range of therapies, but we’ll be using it as shorthand for gene and cell therapies for today’s conversation. ATMPs have been receiving significantly more attention in recent years as a growing number of them come to market. As a potentially one-off, transformative treatment, these therapies are different from many therapies currently available or in development. However, much like all other therapies, ATMPs will be impacted by the ongoing review of the General Pharmaceutical Legislation, and many cases, the review of the OMP regulation as well. Beyond the legislative and regulatory framework, interesting developments are also taking place with regard to HTA and P&R. This combination of commercial and legislative developments will have long-term implications on ATMPs and the competitiveness of the EU. Suffice to say, there is a lot happening when it comes to ATMPs, and with the help of today’s guests, we hope we can start to unpack a few of these discussions, and possibly look into the future as well. Today's guests are: Dr Lutz Bonacker, Senior Vice President and General Manager, Commercial Operations Europe, CSL Behring Dr Alexander Natz, Secretary General, EUCOPE ------------------ For more information about our work on ATMPs or how to join our Cell & Gene Therapy Working Group, simply visit our website or send an email to [email protected].