Developing a product to prevent or treat a rare disease involves unique challenges from hard-to-identify and dispersed patient populations for clinical trials through to undefined endpoints and regulatory requirements. While each condition individually has a small population there are over 30 million people affected by rare diseases in the US alone. It’s therefore encouraging to read today that the FDA is establishing a Rare Disease Innovation Hub to connect with the rare disease patient and caregiver community, to enhance collaboration across FDA centres and to advance regulatory science. We look forward to seeing how this new initiative can accelerate treatment and prevention options across rare disease populations #raredisease #fda #patientcentricity
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Recently, the FDA unveiled the Rare Disease Innovation Hub – a new model that aims to foster collaboration and advance outcomes for patients with rare diseases. We're grateful for the FDA’s commitment to this area and with our like-minded vision to improve the patient journey, we recognize the critical need to break down barriers and co-create solutions to increase equitable care for all patients – no matter who they are or where they live. Working to enhance patient outcomes is at the core of what we do and why we are committed to activating meaningful change and empowering innovation across the rare disease care paradigm. The establishment of the Innovation Hub signifies a step forward toward optimizing care for patients, and ultimately creating a brighter future for the rare disease community. Read the FDA’s full statement: https://lnkd.in/eT3ZEEge
FDA Rare Disease Innovation Hub to Advance Outcomes for Patients
fda.gov
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💡Did you know, in the past 5 years, we have supported >350 rare disease clinical trials & consulting projects? Download our fact sheet to learn more about how we can help you advance development and approval of rare/ultra-rare disease therapies 📩 https://bit.ly/3IY5yaj #RareDisease #UltraRareDiseases
Science-First Strategies to Advance Development and Approval of Rare/Ultra-Rare Disease Therapies
info.veristat.com
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Developing rare disease therapies is complex and challenging. Read this insightful report by Rare Revolution Magazine that includes perspectives from all stakeholders. https://lnkd.in/gtA-GZYR #lifesciences #raredisease #clinicialtrials #patientcentricity
Eight challenges in developing rare disease therapies
https://rarerevolutionmagazine.com
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As per this FDA article below, an estimated 10,000+ rare diseases affect more than 30 million people - that's a lot of clinical trials where it can become challenging to recruit/enroll eligible subjects, while there's often a delayed diagnosis/misdiagnosis. If you play a role in one of these trials, we should talk about how our Judi for Eligibility solution can support you by streamlining/automating the central review process to help achieve a final determination in a timely manner. https://lnkd.in/eYKX_8aA #judiforeligibility #raredisease #collaboration
FDA Rare Disease Innovation Hub to Advance Outcomes for Patients
fda.gov
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Important article in Pharmafile.com by our client Dr Dan Williams at SynaptixBio Ltd. Dan considers the ongoing challenges facing rare disease patients, and discusses developments in the search for therapies. Many thanks to Betsy Goodfellow. The full article is here: https://lnkd.in/e_58twVs.
Overcoming obstacles in rare disease treatment
magazine.pharmafile.com
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Conducting clinical trials for rare disease presents unique challenges and opportunities. Innovative approaches are emerging that promise to revolutionize how we study and development treatments. This article explores fresh ideas that are being implemented to enhance the efficiency and effectiveness of rare disease trials, ultimately bringing hope to patients and their families. https://lnkd.in/ef7hc938
Fresh Take on How to Conduct Rare Disease Clinical Trials
clinicalresearchnewsonline.com
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Many thanks to Betsy Goodfellow at Pharmafile.com for featuring an article by Dr Dan Williams. Dan considers the ongoing challenges facing rare disease patients, and discusses developments in the search for therapies. The full story is here: https://lnkd.in/e_58twVs
Overcoming obstacles in rare disease treatment
magazine.pharmafile.com
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“We understand chronic HBV profoundly impacts the quality of life for people living with the disease, and we are excited to share our ongoing research efforts with the overall goal of working towards a functional cure.” For #HCPs: It’s an important year for #TeamBarinthus at the EASL | The Home of Hepatology 2024 Congress, as we prepare to present two clinical data updates on our lead #hepatitisB asset, VTP-300. These data underscore our commitment to improving outcomes for people with #HepB, providing hope for a potential functional cure. Read more details about our poster and oral sessions here: https://lnkd.in/eVgQrZ8s #EASL2024 #liverdisease #HBV #innovation
Barinthus Bio to Present VTP-300 Clinical Data Updates at EASL Congress 2024
investors.barinthusbio.com
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Today the EMA rejects #Biogen/#Eisai application for the commercialization of Lecanemab for #Alzheimer's disease in Europe because they argue that the risks overweight the potential benefits. Right or wrong, this decision highlights the desperate need for #precisionmedicine in this field. Acknowledging the biological heterogeneity in AD is the key to unlock patient stratification and reach significant benefits with minimal risk.
Eisai, Biogen's Alzheimer's med Leqembi turned down in Europe—but analyst says it's merely a 'delay'
fiercepharma.com
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Rare diseases affect over 30 million individuals in the US. While innovative therapies offer hope, treating these conditions is complex. Pharmacy's pivotal role spans from diagnosis to ongoing support for patients, providers, and payers navigating treatment challenges. Collaborative efforts among stakeholders, including tailored care, value-based models, and outcome tracking, are crucial to ensuring access and optimizing outcomes for those with rare diseases. Read more here: https://lnkd.in/engGnfEM #RareDiseaseDay #PharmacyTimesxMJH Pharmacy Times MJH Thought Leaders
Pharmacy’s Role in Treating Rare Disease: Connecting Patients, Providers, Payers, and Pharma
pharmacytimes.com
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