Critical Path Institute (C-Path)’s Post

Upcoming Webinar: The Influential Role of Patient Advocacy Groups in Registry Data Efforts Join us on Thursday, July 18, at 12 p.m. ET for an insightful webinar hosted by C-Path's Mitochondrial and Inherited Metabolic Diseases Task Force. Discover how patient advocacy groups are pivotal in driving registry data efforts for mitochondrial and inherited metabolic diseases, ensuring that patient experiences and outcomes are central to research and regulatory decision-making. Our presenters include: Sophia Zilber 🌺Board Member and Patient Registry Director, Cure Mito Foundation. Sophia brings over 20 years of experience in drug development and clinical data analysis, leading the global Leigh syndrome patient registry at the Cure Mito Foundation. Elizabeth Reynolds, PhD, Co-Founder and Executive Director, The Champ Foundation. Dr. Reynolds has spearheaded over $2 million in research grants for Single Large-scale mtDNA Deletion Syndromes and co-leads a multi-site Natural History Study and patient-registry. Amanda Klein, PharmD, Executive Director, Critical Path for Lysosomal Diseases and the Alpha-1 Antitrypsin Deficiency Consortia. Dr. Klein leads initiatives to accelerate drug development for mitochondrial and inherited metabolic diseases and is a key figure in the RDCA-DAP initiative. Don’t miss this opportunity to learn from experts about the crucial role of patient advocacy in advancing research and healthcare outcomes. 🔗 Register now to secure your spot: https://lnkd.in/eNExmF9R #CPath #PatientAdvocacy #MitochondrialDiseases #Webinar #HealthcareInnovation #DrugDevelopment #collaboration #datasharing

Yesterday marked #RareDiseases Day, prompting reflection on the valuable insights shared in this article from last year. It underscores the profound impact of digital and analytics capabilities in transforming the diagnosis, treatment, and support of rare disease patients. Technology not only enhances patient care and experience by facilitating improved identification and remote monitoring but also enables personalized treatment plans. Despite notable advancements, challenges persist in diagnosing patients, addressing treatment burdens, and scaling therapies. Nevertheless, collaborative efforts among pharmaceutical companies, patient advocacy groups, and healthcare providers are instrumental in overcoming these hurdles and ultimately enhancing the lives of individuals with rare diseases. Thanks to the authors for this amazing piece: Simon Alfano Christian Amberg Nils Peters Pablo Salazar Marianne Renaud Vieux-Rochas Sam Welton Jeffrey Algazy Jan Ascher Anton Maucuer #RareDiseasesDay #DigitalHealth #PatientsFirst

Our mission is to inform, support, and empower those affected by cavernous malformation and drive research for better treatments and a cure. PATIENT INFORMATION: The Alliance provides extensive information on our website and in our newsletter. We announce new research findings as they are published. We host patient conferences with presentations by disease experts. You can view these at any time on our YouTube channel. PATIENT SUPPORT: We offer patient support through Facebook, facilitated video conferences, and through our local communities. At our patient conferences, we offer time for attendees to share their stories. GENETIC TESTING: We offer free genetic testing to qualifying individuals with multiple cavernous malformations who can’t get coverage through their insurance. OPPORTUNITIES TO PARTICIPATE IN RESEARCH: The Alliance has a patient registry where you can sign up to be notified about research studies, including clinical drug trials, that are recruiting. We also are collaborators in multiple projects that are exploring the impact of the illness on patient’s quality of life. To join our patient registry, type www.ccmregistry.org in your browser or click the link in our bio. CLINICAL CENTERS: The Alliance has a growing network of recognized Clinical Centers and CCM Centers of Excellence that provide expert multi-disciplinary care. AWARENESS EVENTS: We sponsor volunteer-organized awareness opportunities around the country including at major league sporting events and at community walks. In addition to these patient and family activities, we sponsor the annual International CCM Scientific Meeting, which brings together researchers from around the world. We provide consultations to assist with their clinical drug trial planning. www.alliancetocure.org #CavernousMalformation #ItsAboutThePatients #CCM #Cavernoma #CavernousAngioma #AccelerateTheCure #PatientResources

In our latest webinar, Sano Senior Project Manager Hayley Holt, PhD shares an in-depth look at Sano’s work on a clinical trial for Alpha-1 Antitrypsin Deficiency (AATD). Learn about the methodologies used and the impressive success metrics achieved. 📈 Read the recap or watch the webinar, available on-demand now. 👇 https://lnkd.in/emiGdShR #ClinicalTrials #PrecisionMedicine #RareDisease

Did you know that industry-sponsored clinical trial launches in the UK fell by over 40% between 2017-21? We explored how the UK government is changing the way it works with the life sciences sector to reverse this trend.   Industry-sponsored clinical trials are pivotal to the UK’s life sciences sector. In a bid to restore the country’s historic global leadership in such trials, the government commissioned ex-Health Minister Lord O'Shaughnessy to identify key bottlenecks and guide on ways to not only mitigate them, but to also to deliver internationally exemplary services at the benefit of both patients and industry. Since expanding our Axiom Think Tank ‘across the pond’ to the UK in 2022, these improvements to trial set-up are critical to clients engaged with our London team as they seek to launch studies for their innovative oncology and rare disease treatments in the UK. Learn more in the infographic below about key barriers and recommendations outlined in the review, what this means for different stakeholders, and the remarkable progress seen as early as 6 months after – including nearly tripling the average monthly patient recruitment into commercial trials!   #ClinicalTrials #PharmaceuticalIndustry #Biotech #LifeSciences #UKLifeSciences #Healthcare #PatientAdvocacy #NHS #AxiomThinkTank

Want to reduce sample size in your rare disease clinical trial design? Have a look at our recent business case, highlighting the multiple benefits of a more patient-centric approach to get there! One2Treat's approach was recently used for an acute promyelocytic leukaemia (APL) clinical trial design. Our innovative methodology incorporating multiple patient-relevant outcomes in the primary analysis managed to reduce the required sample size by 40%, compared to more conventional statistics methods. This significant sample size reduction not only made the trial feasible, considering the challenges in recruiting patients in this indication (and for rare diseases in general) but also played a crucial role in convincing funders of its viability. For a deeper dive into this business case, you can read the paper we published on it here https://lnkd.in/e9NbVQta Questions or interest? Reach out to us at info@one2treat.com or visit our website www.one2treat.com #PatientCentricDesigns #RareDisease #ReducedSampleSize

In the realm of #raredisease, the necessity of strong #evidence is still a challenge even when populations are small. And this is possible! In the context of a Randomized Control Trial #RCT dedicated to acute promyelocytic leukemia (APL), we were able to significantly reduce the required sample size by accounting for multiple outcomes. On top of reducing the number of patients being exposed, we provided a better representation of the benefits for the patients beyond survival, by accurately considering the role of other toxicities. Interested in discussing this further? Please contact me for more information! #one2treat #GPC #samplesizereduction #patientcentricity #treatmentdecision

Interested in a practical example on how the innovative One2Treat’s methodology was able to make a clinical trial in a rare disease feasible by reducing the required sample size? Here is the published paper on the design for this clinical trial in acute promyelocytic leukemia (APL). 

Melinta Therapeutics has awarded Dr. Gary Fong an Industry Grant for his proposal on the need for a novel long-acting antifungal in an underserved population, worth almost $20,000. Dr. Fong and his co-PI will investigate the evolution of candidemia and antifungal resistance to assess how rezafungin could reduce healthcare costs and improve patient outcomes, especially for vulnerable populations. #pharmacy #pharmacyresearch #antifungal #healthcare https://lnkd.in/gJWtfrM6

Do you want to reduce sample size or do you NEED to find an efficient way to address the challenges of small patient populations? One2Treat may be the best way for you to achieve your #clinicaltrial goals with a more #patientcentric approach.