🌟 New Episode Alert! 🌟 The latest episode of RARE, a blog by Laura Hulley, Principal Consultant at SciPro, features Tom Pulles, MD, a strategic medical leader with over a decade of experience in rare diseases. Tom has an impressive background with companies like Sanofi, Shire, Ultragenyx, and now Acadia Pharmaceuticals, focusing on Rett Syndrome in Europe. Tom shares his insights on the unique challenges of rare diseases, emphasising the importance of awareness, diagnosis, and collaboration. He discusses the rewarding nature of pioneering treatments and the need for a long-term, patient-focused strategy in this field. Discover Tom's journey and his passion for making a difference in the rare disease community by watching the interview now through the link below: https://lnkd.in/euKeUu46 Thank you, Tom, for your valuable contributions! #RareDiseases #PatientAdvocacy #MedicalInnovation
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Join us at the Rare Disease Day 2024 Summit as MORSE Consulting Inc.'s Sherry O'Quinn joins other panelists (Lindy Forte, Eversana; Alex Chambers, Bayer; Joan Paulin, PHA Canada; Bonnie Kam, Janssen; Christian Dong, PhD, MBA, Pfizer; Farah Jivraj, Biogen; Rute Fernandes, Takeda; Declan Hamill, IMC) in a compelling panel discussion on the Canadian Pathway to Rare Disease Drug Access. Scheduled for February 28th from 11:15 AM to 12:30 PM at the Ottawa Marriott Hotel, this session promises insights that aim to shape the future of rare disease drug access in Canada. Don't miss this opportunity to be part of the conversation at the forefront of rare disease treatment. Mark your calendars for February 28th and join us at the Ottawa Marriott Hotel for an enriching discussion. Learn more about the Rare Disease Day 2024 Summit here: https://ow.ly/mf6I50QHjhx #Canada4Rare #RareDiseaseDay2024 #RareDiseaseSummit #MarketAccess
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Antun Sablek presented a poster at #ISPOREurope titled “Adoption of precision health and precision medicine approaches in addressing population health needs in Europe”. The poster presented findings from a report commissioned by EFPIA - European Federation of Pharmaceutical Industries and Associations in collaboration with the EFPIA Oncology Platform. The poster shows the potential of precision approaches in addressing common diseases across patient care pathway and provides policy recommendations to improve their implementation. By embracing these recommendations, Europe has the opportunity to establish itself as a leader in advancing and applying PH/PM approaches to address the complex healthcare requirements of populations at large. For more information on CRA's involvement at ISPOR, click here: https://lnkd.in/gg6D2B3D #EFPIA #CRA #precisionmedicine #precisionhealth
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🌟 Exciting Kick off to RECON Conference! 🌟 🔬 Today, Peter Stein captivated audiences at the RECON conference with his enlightening presentation on the burgeoning field of orphan drugs. His talk, “Opportunities and Challenges in Creating a Winning Strategy from a Patient and Drug Developer Viewpoint” was a deep dive into the critical role of translational medicine in advancing rare disease treatments. 🚀 Key Highlights: 1️⃣ Unprecedented growth in orphan drug approvals, a testament to innovation and dedication in healthcare. 2️⃣ The transformative impact of translational medicine in bridging lab discoveries and patient care. 3️⃣ Insightful discussion on the FDA’s new guidance on “one adequate well-controlled trial” and its role in accelerating drug approval processes. 4️⃣ Peter’s engaging analysis of confirmatory evidence – a cornerstone in ensuring drug efficacy and safety and managing uncertainty 5️⃣ Fascinating case studies on surrogate endpoints – a mixed bag of successes and lessons learned. 🤝 This presentation not only shed light on the complexities of drug development for rare diseases but also sparked hope and inspiration for future breakthroughs. 💡 “We really have to think about assumptions when using surrogate. Test all the assumptions.” Peter remarked, emphasizing the importance of both successes and setbacks in the journey of drug development. 🔗 Stay tuned for more updates and insights from RECON! #RECONConference #OrphanDrugs #TranslationalMedicine #FDAGuidance #HealthcareInnovation
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There are no FDA/EMA/etc approved therapies for mitochondrial disease. We need to work together to thoughtfully collect and integrate data to inform clinically meaningful endpoints and de-risk clinical trial design. This task force is dedicated to advancing the regulatory science needs so that individuals living with mitochondrial and inherited metabolic diseases can finally see some drug development they so desperately need and want. #missiondriven #collaboration #publicprivatepartnership #regulatory
C-Path today announced the launch of a task force focused on accelerating drug development for mitochondrial and inherited metabolic diseases. The task force will lay the groundwork for specific solutions, offering valuable insights that aim to contribute to regulatory decision-making. “C-Path is uniquely positioned to lead this new task force,” explained Amanda Klein, Pharm.D., C-Path’s Executive Director of the Transplant Therapeutics Consortium and lead for this task force. “We thank the communities for recognizing the importance of collaborative projects. We look forward to leveraging our core competencies to provide strategic and tactical guidance, engage relevant stakeholders, and bring diverse expertise to generate the solutions to help patients and their families.” Full details: https://lnkd.in/eevBsmjg Amanda Klein Alexandre Bétourné, PhD, PharmD, PMP Melody Kisor Cure LBSL Astellas Pharma Europe Dima Martini-Drew MD The Champ Foundation Cure Mito Foundation Sophia Zilber 🌺 Danielle B. mitoworld.org Alexander Sercel, PhD Midwestern University Volkmar Weissig Jon Brestoff Hope for PDCD Foundation Frances Muenzer Pimentel #CPath #taskforce #RDCADAP #NORD #FDA #raredisease #curelbsl #MitoWorld #Astellas #MitoFoundation #globalhealth #collaboration #drugdevelopment #datasharing
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💚 We are officially relaunched! 💚 We are driven by a mission to ensure that patients across Europe have access to the life-changing therapies they deserve. The path to developing life-saving treatments for rare diseases is complex, and securing commercial success is crucial to offset development costs. This is why ensuring global access to these therapies is essential. While North America and the Asia-Pacific (APAC) regions account for nearly 67% of all new rare disease therapies, navigating the intricacies of the European market can be a daunting task. Partner Rare stands as a beacon of expertise, housing a team of renowned industry leaders with decades of experience in drug development and commercialization in the rare disease domain. If you are based in North America or APAC and seeking EMA approval for your rare disease asset, or if you are a start-up seeking comprehensive support, Partner Rare is your trusted partner. We are eager to collaborate with you to shape and deliver strategic priorities that will pave the way for your success. Contact us today to schedule a consultation and embark on a journey of transformative change for patients with rare diseases. #raredisease #drugdevelopment #strategicadvisory #strategicadvisory #consultancy #europe #ema #raredisordertherapies #commercialization #startups
💚💚💚 WE ARE LIVE!! 💚💚💚 Partner Rare: Revolutionizing Rare Disease Treatment in Europe We're thrilled to announce the relaunch of Partner Rare, a leading provider of strategic advisory and consultancy services to the rare disease ecosystem, with a special focus on navigating the complexities of the European landscape. As we embark on this new chapter, we remain deeply committed to our mission of accelerating the development of life-saving treatments for rare diseases across Europe. Addressing Rare Disease Challenges in Europe The European rare disease landscape is vast and diverse, presenting unique challenges for companies seeking to bring innovative treatments to market. Partner Rare understands these complexities and is uniquely positioned to guide companies through the regulatory hurdles, market access strategies, and reimbursement pathways specific to the European region. Partner Forward: Accelerator for Rare Disease Innovation Partner Rare is proud to introduce Partner Forward, our exclusive accelerator program designed to foster the development of transformative rare disease therapies. Partner Forward provides early-stage rare disease companies with the resources, expertise, and connections they need to accelerate their path to clinical success. Through Partner Forward, we offer: Strategic guidance: We provide expert counsel on regulatory, clinical, and commercial strategies to ensure the successful development and commercialization of rare disease therapies. Access to funding: We connect early-stage rare disease companies with potential investors and funding opportunities to support their growth and development. Networking opportunities: We facilitate connections with key industry stakeholders, including potential partners, collaborators, and regulatory experts. Your Partner in Rare Disease Excellence Partner Rare is committed to revolutionizing rare disease treatment in Europe. We believe that by effectively navigating the European complexities and providing innovative solutions like Partner Forward, we can bring transformative therapies to patients across the continent and make a significant impact on patient lives. Visit our website to delve into our expertise and services: https://partner-rare.com/ PR💚 #raredisease #drugdevelopment #strategicadvisory #consultancy #europe #navigatingcomplexity #patientadvocacy #PartnerForward
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Panel II - 'We have an opportunity: How to get a unified list of medicines and more efficient procedures?', opens questions to which many citizens also want answers. We talked about this very topic with experts Natas Grubis, director, of the Agency for Medicines and Medical Devices of BiH, Ana Petrović, director of the Association of Innovative Drug Manufacturers in BiH, Emina Budimlic, commercial director of BiH Takeda, prof. Dr. Timur Ceric, Head of the Day Oncology Hospital of the Clinic for Oncology, KCUS. "As for the medicines themselves, I would like to emphasize that the period of the pandemic led us to computerization and digitalization", said Grubiša, adding that today patients learn about certain therapies much faster, which is additional pressure for doctors. #pharmacy #pandemia #bloombergadria #conference
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Evidence strategies remains at the centre of the discussion of the EU wide new health technology assessment (HTA) system coming up in 2025 for medtech, cancer drugs and advanced therapies. An approach looking at patient population, intervention, comparator and outcomes (PICO) will be used to evaluate the benefits and risks of these treatments across all EU member states. However, how can companies ensure that their data meets the expectations of both the EU and the local authorities? How can they address the differences between clinical trial data and real world? And how can they use advanced methods to overcome the limitations of traditional comparisons? This is one of the topics that we look to unpack in the upcoming Baker Tilly webinar series on Evidence generation strategies for enhanced market access success. If you are interested in learning more about evidence strategies, don't miss this opportunity to join me in this webinar - March 19th at 15:00 GMT. I look forward to seeing you there! David Gregory Darren Jones Grace Macalino Keith Needham Peter Bannister Deepak Upadhyaya Alexandre Templier Xavier Mercadé Sanmartí Yogan A Patel #HTA #JCA #marketaccess #oncology #advancedtherapies #medtech #bakertilly
Evidence generation strategies for enhanced market access success
bakertilly.com
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Listen Here: https://lnkd.in/gmb8BHhj In this episode, Scott Becker is joined by Paige Twenter, Assistant Editor at Becker's Healthcare, to discuss the latest in healthcare pricing controversies, Mark Cuban's Cost Plus Drugs initiative, and ongoing drug shortages. Paige provides insights into the pricing debates surrounding GLP-1s, the expansion of affordable drug access, and the critical issue of oncology drug shortages affecting hospitals and patients nationwide. For more information about Becker's Healthcare, please contact Jessica Cole or Scott Becker. #podcast #healthcarenews #ozempic #drugshortages
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Listen Here: https://lnkd.in/ggqNkdys In this episode, Scott Becker is joined by Paige Twenter, Assistant Editor at Becker's Healthcare, to discuss the latest in healthcare pricing controversies, Mark Cuban's Cost Plus Drugs initiative, and ongoing drug shortages. Paige provides insights into the pricing debates surrounding GLP-1s, the expansion of affordable drug access, and the critical issue of oncology drug shortages affecting hospitals and patients nationwide. For more information about Becker's Healthcare, please contact Jessica Cole or Scott Becker. #podcast #healthcarenews #ozempic #drugshortages
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Medical Affairs / High-Performance Leadership / Strategy / Coaching / Education / Patient Advocacy / Rare Diseases
1moThank you very much Laura Hulley and SciPro for coordinating this and providing me the opportunity to share and discuss this important topic! I truly hope that this will help all the (ultra) rare disease communities around the globe 🙏