RWE4Decisions ’s Post

Are you curious about the HTA collaboration in the Nordic Countries? And how Real World Data can support HTA? Find link below to RWE4Decisions interesting webinar which was hosted wednesday.

I really enjoyed the discussion on RWD/E we had this week regarding the pivotal role it already is and should play in HTA decision making in the Nordics countries. Many thanks to RWE4Desions for hosting the webinar! The rapid decline in rate of availability of innovative medicines in the Nordic countries is very concerning. Diseases and innovative medicines are increasingly being stratified and segmented into smaller entities with patient populations become increasingly smaller. This is particularly true for Personalized Medicines and ATMP’s in Rare Diseases. It is positive that we are becoming more precise in how we should treat patients optimally. But it also poses a challenge to accumulate the necessary data when patient populations are evidently shrinking. We need to find alternative avenues to provide data - and RWD/E is one of these. In the Nordics we have a strong legacy developing and leveraging register data and we should take leadership in using this to aid access to new innovative medicines faster. This also includes that we to an even higher degree are using data generated in one Nordic country in other Nordic countries HTA processes and decisions. 

Today, we at Cencora PharmaLex and SwedenBIO, hosted a Brain & Breakfast event on the EU joint HTA process that sparked engaging conversations and valuable insights! A big thank you to all participants for contributing to the lively discussions, and special appreciation to our esteemed panelists Niklas Hedberg and Hanna Zirath, Ph.D. from TLV and Sofia Heigis, CEO at Oncopeptides, for transparently sharing your invaluable insights. The discussion was excellently moderated by Herbert Altmann, head of Marcet Access and Pricing and Reimbursement Europe, Cencora PharmaLex. Thank you Jessica Martinsson, Johanna Assadi Rissanen, Marjo Puumalainen and Frida Eriksson at SwedenBIO for a great collaboration.   Here are some key takeaways from our enriching discussion: 👉 Prioritize Preparation: It's essential for pharma/biotech companies to familiarize themself with the available information and materials continuously published by the EC and other competent experts to be adequately prepared. 👉 Emphasize Communication: Effective communication, interaction, and knowledge-sharing are pivotal both internally within organizations and externally between companies and regulatory authorities, especially 2025/26 during the implementation phase. 👉 X-functional collaboration: Implementing and integrated launch plan supported by research and development (R&D), market access, regulatory and other departments focusing on evidence generation for the new EU HTA process. 👉 Engage with Joint Scientific Consultation (JSC): Engaging in early scientific consultations presents a valuable opportunity to consider an optimal and robust evidence generation plan satisfying both regulatory and HTA bodies' requirements. 👉 PICO Considerations: The Joint Clinical Assessment (JCA) relies on the Population, Intervention, Comparator, and Outcome (PICO) framework for assessments. However, variations in healthcare systems and treatment guidelines among different nations will significantly influence how PICO criteria are applied in drug assessments. High quality input from Swedish affiliates or SMEs is critical to ensure a good discussion with TLV in Sweden.   Let's continue to foster collaboration and knowledge sharing for a brighter future in the pharmaceutical landscape!   Want to learn more? This can be a start: https://lnkd.in/dqSij-rt #EUHTA #JCA #JSC #BrainAndBreakfast #PanelDiscussion #TLV #SwedenBIO #CencoraPharmalex #Collaboration #Pharmaceuticals

Today Cencora PharmaLex and SwedenBIO hosted a Brain & Breakfast event on the EU joint HTA process that sparked engaging conversations and valuable insights! A big thank you to all participants for contributing to the lively discussions, and special appreciation to our esteemed panelists Niklas Hedberg and Hanna Zirath, Ph.D. from TLV and Sofia Heigis, CEO at Oncopeptides, for transparently sharing your invaluable insights. The discussion was excellently moderated by Herbert Altmann, head of Market Access and Pricing and Reimbursement Europe, Cencora PharmaLex.   Here are some key takeaways from our enriching discussion: 💡 Prioritize Preparation: It's essential for pharma/biotech companies to familiarize themself with the available information and materials continuously published by the EC and other competent experts to be adequately prepared. 💡 Emphasize Communication: Effective communication, interaction, and knowledge-sharing are pivotal both internally within organizations and externally between companies and regulatory authorities, especially 2025/26 during the implementation phase. 💡 X-functional collaboration: Implementing and integrated launch plan supported by research and development (R&D), market access, regulatory and other departments focusing on evidence generation for the new EU HTA process. 💡 Engage with Joint Scientific Consultation (JSC): Engaging in early scientific consultations presents a valuable opportunity to consider an optimal and robust evidence generation plan satisfying both regulatory and HTA bodies' requirements. 💡 PICO Considerations: The Joint Clinical Assessment (JCA) relies on the Population, Intervention, Comparator, and Outcome (PICO) framework for assessments. However, variations in healthcare systems and treatment guidelines among different nations will significantly influence how PICO criteria are applied in drug assessments. High quality input from Swedish affiliates or SMEs is critical to ensure a good discussion with TLV in Sweden.   Let's continue to foster collaboration and knowledge sharing for a brighter future in the pharmaceutical landscape!   Want to learn more? This can be a start: https://lnkd.in/dhG3mhrX #EUHTA #JCA #JSC #BrainAndBreakfast #PanelDiscussion #TLV #SwedenBIO #CencoraPharmalex #Collaboration #Pharmaceuticals

We often reiterate: Everything we do is for the patient’s benefit. A conducive environment for studies is vital, from which patients in Germany stand to gain particularly. To facilitate easy access and provide a comprehensive view of MSD’s clinical trials, we’ve developed the MSD Study Portal. Here, individuals with various health conditions can seek out studies using a straightforward keyword or medication search, and discuss participation with their doctors. This grants patients early and readily available access to new and innovative therapies, one of the most important benefits of a flourishing pharmaceutical industry.   This Portal is just one example of the profound impact pharmaceutical research can have on patient lives in Germany. It underscores the necessity of fostering a favorable and competitive environment for conducting clinical trials, including for research-based pharmaceutical companies, within Germany. This week, the Medical Research Act was discussed in an expert hearing of the Ministry of Health. While this news may not be of particular interest to most people, it is of great importance to us at MSD Deutschland and the entire research-based pharmaceutical industry in the country. With the Medical Research Act, Germany has an excellent opportunity to move even further into pioneering health research and care. The procedures could be accelerated so that Germany could once again take on a leading role in the recruitment of patients for clinical trials in comparison with other European countries. So this is good news for everyone.   We at MSD Germany are keenly anticipating the results of the hearing as part of the association participation and the subsequent steps, and we wholeheartedly back the government's initiative! For those wishing to explore the MSD Study Portal in more detail, the link can be found in the first comment.   #ForschungIstDeutschlandsBesteMedizin #Pharmastrategie #InventingForLife

📍 Live from the World Orphan Drug Congress (#WODC) in Barcelona! 👁️🗨️ This morning's discussions on the Real World Evidence Track provided valuable insights into the growing importance of Real-World Data (RWD) and Real-World Evidence (RWE). 🧬 📊 #RWE is proving to be a powerful tool for pharmaceutical companies and health authorities to enhance patient outcomes and positively impact the entire healthcare system. Chaired by Stefano Romanelli - EUCOPE Senior Government Affairs Manager, the Track explored: 🔹 How RWE can support HTA and market access decision-making 🔹 Challenges in utilizing real-world data effectively 🔹 The critical role of patients in generating and shaping RWD/RWE 🔹 How public-private partnerships (PPPs) can drive innovation in rare disease research 🔹 Strategy for Orphan Drugs in Spain A big thanks to the global and EU healthcare stakeholders who shared their expertise on how RWD/RWE is shaping the future of rare disease therapies and healthcare in general! #WODC2024 #RealWorldEvidence #RWE #RWD #InnovativePharma #HealthcareInnovation #PatientCentric #HTA

Would you like to learn more about how different stakeholders can help generate robust Real-World Evidence (RWE) that meets the needs of HTA bodies and Payers? Then don’t miss out on the Annual RWE4Decisions Symposium, where we will launch the new “Stakeholder Actions to Generate Better Real-World Evidence for HTA/Payers”, an essential set of recommendations for national and collaborative HTA/Payers, the pharmaceutical industry, clinicians, patient groups, disease registry holders, and Real-World Data (RWD)/Analytics groups. The panel, moderated by Ashley Jaksa, VP of Scientific Strategy and Partnerships at Aetion, will include insights from: - Karen Facey, RWE4Decisions Facilitator - Piia Rannanheimo, Chief Specialist, Finnish Medicines Agency Fimea - Lääkealan turvallisuus- ja kehittämiskeskus Fimea (FIMEA) - Shaun Rowark, Associate Director for Data Access and Analysis, NICE - National Institute for Health and Care Excellence - Andre Vidal Pinheiro, Vice-President and Head of Patient Value & Access - Europe & Canada, Takeda One of the key highlights of our work this year, building on our successful 2020 publication, “Real-world evidence to support Payer/HTA decisions about highly innovative technologies in the EU—actions for stakeholders”, the 2024 Stakeholder Actions have been updated with the help of the RWE4Decisions multi-stakeholder Learning Network. The new version accounts for the important changes in the decision-making environment, already-made progress, and remaining challenges.  🔗 We hope you can join us for the launch – register here: https://lnkd.in/eYdNXqxT 📅 Date & Time: 14 November, 09:00-13:00 CET 📍Location: Hybrid – attend in Brussels or join online from anywhere #RWE4Decisions #HTA #RealWorldEvidence #RWE #EHDS

Great news! Our abstract on Patient Access to Orphan Drugs got accepted! On 7 May, I will present as part of a team of experts a case study of an orphan drug at the RAPS Euro Convergence in Berlin, Germany.   We will speak about an orphan drug for the treatment of erythropoietic protoporphyria (EPP) from the perspectives of:     ▪ Patient Advocates, the International Porphyria Patient Network IPPN ▪ Regulatory, the European Medicines Agency EMA ▪ HTA, the Federal Joint Committee G-BA in Germany   During this session, we will discuss   ➤ Why different evidence requirements for an EU-wide marketing authorisation recommendation by EMA and for a national reimbursement decision can be a key obstacle for patients’ access to innovative new medicines. ➤ Interdependencies between the opinions of the Committee for Orphan Medicinal Products (COMP), the Committee for Medicinal Products for Human Use (CHMP) and the G-BA as well as the benefits of parallel EMA-HTA discussions. ➤ The involvement of the EPP patient in both regulatory and HTA discussions, including in the selection of endpoints and measurement scales. ➤ The implications of these approaches for the national benefit assessment in Germany in the light of the upcoming EU Joint Clinical Assessment.   Upon completion of this session, participants should be able to:   ✓ Describe which EU regulatory decisions affect the national HTA decision in Germany and the benefits of parallel EMA-HTA discussions. ✓ Adapt the clinical development strategy of orphan medicinal products to the patient needs, regulatory and HTA requirements. ✓ Explain the role of patient involvement in both regulatory and HTA discussions for this orphan medicinal product.   I feel blessed to be part of this Expert Session with PD Dr. Jasmin Barman-Aksözen and Karl-Heinz Huemer kindly moderated by Beate R. Schmidt and with Kristina Larsson as special panelist.   You can view the RAPS Euro Convergence Agenda below in the comments.   #PatientAccess #OrphanDrugs #RareDiseases #MarketAccess #PatientAdvocate #EMA #HTA #FederalJointCommittee #JSC #JCA #RAPSEuroConvergence #MBMFutureHealth #Gratitude

We are honored to contribute to the Joint Heads of Medicines Agencies (HMA)/European Medicines Agency (EMA) Multistakeholder workshop on Patient Registries, organized on 12 and 13 February in Amsterdam, the Netherlands. This joint two-day hybrid HMA/EMA workshop on Patient Registries follows on the successful disease-specific workshops held between 2017 and 2019, as well as the EMA multi-stakeholder workshop on the qualification of novel methodologies. The EMA workshop will bring together representatives of registry holders, regulatory agencies, pharmaceutical companies, patients, healthcare professionals, academia, and health technology assessment bodies to address the following objectives. Today on behalf of the HARMONY Alliance, Jesús María Hernández Rivas (Institute of Biomedical Research of Salamanca, IBSAL_IIS), will participate in Session 2: "Experience gained, and lessons learnt from initiatives aiming to leverage the use of registries". Dr. Hernández Rivas will talk about the Interoperability between registries and other data sources, and will explain about the HARMONY Big Data Platform. The HARMONY Alliance would like to thank the European Medicines Agency the European Hematology Association (EHA) for this opportunity. More information and agenda details can be found here: https://bit.ly/3UEJ2Kt #bigdataforbloodcancer #patients #registries #data #bigdata #multi-stakeholders

The five FIP regional pharmaceutical forums, which cover the African, Americas, Eastern Mediterranean, South-East Asian, and Western Pacific regions, are now officially part of FIP, the federation announced today. These forums were established over 20 years ago, to increase dialogue, understanding and activities to improve pharmacy services and health according to specific needs, and to be the liaising structures between the World Health Organization (WHO) Regional Offices and FIP. “In recent years, both the forums and FIP expressed a desire for a closer relationship and a sense of belonging, and a need for greater support from and alignment with FIP, both institutional and strategic. Initial meetings between the forums and FIP were held in 2019 to explore options for integration, and following our consultations in 2023, I am pleased that the regional pharmaceutical forums are now fully integrated within FIP. This is an important step in our ‘One FIP’ approach,” said FIP president Paul Sinclair. The integration will facilitate FIP in consolidating its presence, visibility and influence across the WHO regions, including enhanced work with the WHO regional offices and partnerships with other regional stakeholders. “The WHO Regional Officers are important stakeholders in the definition of health and pharmaceutical policies in regions, with significant influence on national policies. In addition to global WHO policies, the policy and technical documents of its regional offices are opportunities for collaboration. Furthermore, cooperation among FIP member organisations within a region — given their common geography, and often cultural, economic and political closeness — will be efficient and valuable,” he added. The forums will act as the regional arm of FIP with the WHO, with speaking rights at the FIP Council, but no voting rights so as not to duplicate the votes of FIP member organisations. “The integration of the forums is part of a wider body of strategic work to align multiple governance structures and create synergies. I am very pleased to officially welcome the forums as part of FIP and to optimise efforts towards achieving FIP’s vision and mission,” Mr Sinclair said. More information about FIP regional pharmaceutical forums can be found here: https://lnkd.in/dkzRyXdH.