🌟 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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Out now: the updated 'MS Brain Health – time matters' report was launched at ECTRIMS yesterday! It highlights the importance of early diagnosis, timely treatment and holistic, person-centred care, along with the policy changes needed to improve long-term outcomes for those living with MS, NMOSD and MOGAD. #BrainHealthTM #BrainHealth #MSTimeMatters
Our new #BrainHealthTimeMatters is now available: https://lnkd.in/eDavWAg So many have made this work possible. Our thanks go to Alexion Pharma Germany GmbH and Merck Healthcare for sponsoring the launch of this report and helping bring this work to all; to Roche, MS Society and Horizon, now part of Amgen for their critical support towards the development of the report; to the panel of authors Helmut Butzkueven, Gavin Giovannoni, Sofia Arkelsten, Giancarlo Comi, Kathy Costello, Michael Devlin, Jelena Drulovic, Emma Gray, Jodi Haartsen, Anne Helme, Jana Hlaváčová, Elisabeth Kasilingam, Yaou Liu, Thomas Mathew, Saul Reyes, Jérôme de Sèze and Mitzi Joi Williams M.D., FAAN and to the many members of the Working Groups named in the report who brought expertise and evidence to shape and support the recommendations. We thank you and look forward the next steps - collaborating with you and further stakeholders to support the implementation of the recommendations. #BrainHealthTM #BrainHealth #MSTimeMatters, #NMOSDTimeMatters, #MOGADTimeMatters, #MSBH #OHPF, The Sumaira Foundation, Sumaira Ahmed, EMSP - European Multiple Sclerosis Platform, MS International Federation (MSIF), Can Do MS
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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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🌟 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 excited to have our work presented at the upcoming AMCP Nexus Conference in Las Vegas this October. The results of our collaboration with Sun Pharma and key opinion leaders will be showcased in a poster titled "A Systematic Literature Review and Meta-Analysis of the Real-World Effectiveness, Quality of Life, and Safety of Tildrakizumab for Moderate-to-Severe Plaque Psoriasis." This presentation highlights the practical benefits of tildrakizumab in real-world clinical settings, offering valuable insights into its efficacy and impact on patient quality of life. Our poster L17 will be displayed in The Expo – Marquee Ballroom on October 16 from 1:00 pm – 2:30 pm https://lnkd.in/g_VsFa7g
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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
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#BeyondCelebrations "Celebrating" Disease Days: A Misstep in Pharmaceutical Communication 🚫 It's crucial to choose our words wisely, especially in the pharmaceutical industry. Recently, I've noticed a concerning trend where the word "celebrating" is used in conjunction with disease days or weeks. As mentors and leaders, it's our responsibility to guide our teams towards more sensitive and appropriate language choices. For instance, in discussions about diseases like cancer, using the term "celebrating" can come across as insensitive and disrespectful to those battling the illness and their loved ones. Instead, let's focus on raising awareness, supporting research, and advocating for better treatments and care. I've had discussions with several team members, urging them to reconsider their language choices but unfortunately, this practice still persists in some global circles and with Great short term sales brains. Let's strive for greater empathy and professionalism in our communications, ensuring that every word we use reflects our commitment to patients and their well-being. #PharmaceuticalCommunication #LanguageMatters #EmpathyInHealthcare #cancercare #narendramodiji #cervicalcancer #PharmaMarketing
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Neuroscience - Brain health- Gender Medicine - Consultant- TED-x Speaker - Scientific Writer -Alzheimer Advocate - Editor -Privat Dozent - FEAN
This is the message I would like policy makers and the society at large to hear: #Alzheimer is different now, we need to rethink the whole system around it. A great honour (and a pleasure) to have led the writing of this report in the context of the Rethinking Alzheimer's Disease project, coordinated by European Brain Council in collaboration with EFPIA - European Federation of Pharmaceutical Industries and Associations. I would like to thank all the members of this project for such a great collaboration. So, what is different now? - Alzheimer is not a mystery anymore -> thanks to scientific research we have started to understand the biological underpinnings of the clinical symptoms - it can be diagnosed early -> science has allowed us to identify markers for early and precise diagnosis, as well as targets for pharmacological treatment - it is treatable -> new treatments (potentially disease modifying) are starting to be approved, meaning that in the future Alzheimer will be treatable Like in oncology, we are moving towards #precisionmedicine, with early diagnosis as key for disease management (would you wait until stage 4 cancer to treat? I don't think so - so we should also not wait for the cognitive decline to be in its dementia stage before acting). Like in the field of multiple sclerosis at the time of the interferons, we are entering an era of new treatments (and look today how many treatment options exist for MS - it is not cured, but it's a disease that, with state of the art treatment, one can live with). This is all very exciting for a scientist like me, but how about patients? and caregivers? I have realized that without a profound societal and political change this new understanding will not reach the millions of patients and caregivers in need. The truth is, we are not ready for it. We do not have resources and facilities for early diagnosis, less so for treatment. But not doing it is not an option. Therefore, we need action. In this report we highlight current gaps and issues in post-diagnostic journey, and suggest structural and organizational changes to facilitate the implementation of this new vision of Alzheimer. Treating Alzheimer is important at several level - from the personal one (alleviating the burden of so many individuals and families, including millions of female caregivers), to the political one - preserving the brain health economy of the European population. We can't afford to do this wrong. Please help me spreading this paper and its message of hope and urgency for Alzheimer.
📢 On the occasion of the #WorldAlzheimersMonth, we are proud to launch, in collaboration with EFPIA - European Federation of Pharmaceutical Industries and Associations, the Perspective Paper "Rethinking Alzheimer's Disease Pathway: From Diagnosis to Care." The aim of this report is not merely to document the status quo, but to challenge it—to rethink how we approach Alzheimer’s treatment, care and management. By offering a comprehensive review of current practices, identifying challenges, gaps and barriers, and proposing policy recommendations, this report seeks to reshape the Alzheimer’s disease healthcare pathway in ways that enhance outcomes and quality of life for all involved, and reduce the impact on society. 🔎 Read more here: https://lnkd.in/e_iSYZwN
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“Many of our pediatric patients are the most effective advocates. We are finding that often times, growing up, these kiddos are the only ones that they know affected by their disease, and that can be really isolating.” In a special podcast episode recorded for Rare Disease Day, Angela Wheeler, President of Insight US at Lumanity and Dr. Karin Hoelzer, Director of Policy and Regulatory Affairs at the National Organization for Rare Disorders (NORD), discuss opportunities for pharmaceutical companies to work with and support rare disease communities. Listen now: https://buff.ly/3Ih8qOV Discussion highlights include the importance of: - continued support for the development of rare disease treatments - making a push towards more equitable clinical trial populations - focusing on how we can reduce the barriers to access - bringing in the patient perspective and patient voice in every step of the drug development process Listen on our website, or wherever you get your podcasts: https://buff.ly/3Ih8qOV Learn more about NORD and how to get involved as an individual, through their Rare Action Network and Advocacy Taskforce, and for drug companies, through their corporate council: https://buff.ly/4bPJhsa #RareDiseaseDay #WeAreRare #RareDiseases
Supporting Rare Disease Communities
https://lumanity.com
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Listen Here: https://lnkd.in/g3ZtmMxQ 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
4moThank 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 🙏