Haystack Project的动态

Biosimilars Policy Roundup for September 2024—Podcast Edition This month on Not So Different, we dive into the FDA's approval of a new biosimilar for retinal diseases, ongoing legal battles, market trends, and regulatory hurdles in the biosimilar space. Listen in for the full monthly recap! https://lnkd.in/ePvc5rWs

Rienk Pypstra will play an important role at BEAM Alliance's AMR Conference, a global gathering addressing the critical challenges of antimicrobial resistance. On Wednesday 26th February, Rienk will moderate an engaging panel discussion titled “Clinical Endpoints: Fit-for-Purpose?”. This session will delve into the current landscape and future requirements of clinical endpoints in antimicrobial development, featuring valuable insights from renowned experts in the field: Dr. Radu Botgros, European Medicines Agency, Marc Bonten, European Clinical Research Alliance on Infectious Diseases Jimin (Peter) Kim, U.S. Food & Drug Administration We’re looking forward to a great discussion! #AMR #AntimicrobialResistance #InfectiousDiseases

?? Clinical research on the frontlines isn't for the faint-hearted. Discover how Rubix LS is pushing boundaries in some of the world's most challenging environments. Pharma leaders, this is where innovation meets resilience. Dive into the full story here!?? #ClinicalResearch #Pharma #Frontlines https://lnkd.in/gXPdrsXY

First-to-market products play a pivotal role in shaping the orphan designation status of subsequent treatments in the EU. These regulatory nuances have significant implications for access, competition, and market strategies within the rare disease space. Our latest article looks deeper into these complexities, offering insights to help organisations navigate this evolving landscape.??? Read further into the regulatory nuances and how they shape market strategies in the rare disease space: https://lnkd.in/dSavjXbR #RareDiseases #OrphanDrugs #MarketAccess #RegulatoryStrategy

?? Early diagnosis and treatment can prevent complications and enable people to live as normal a life as possible. ?? Early diagnosis contributes greatly to improved quality of life and care for people with PI ?? Patient access to care and appropriate treatment can help prevent serious and life-threatening illnesses in people with PI With this in mind, the video campaign “AFFIANCO” ("walk together”) narrated the extraordinary and touching experiences of five patients and their referring doctors, who, from a certain moment of their existence, began to "walk together" in the difficult battle against Primary Immunodeficiencies (PID), rare diseases of the immune system. The initiative was conceived and developed by the Associazione Nazionale Immunodeficienze Primitive - AIP O.d.V. (Italian Association of Primary Immunodeficiencies - AIP O.d.V.) and supported by Kedrion with an unconditional grant. ?? Watch the stories: https://lnkd.in/dPyhpUYE [TIP: Click on the automatic subtitles on youtube for more languages] #WorldPIWeek #WorldPIWeek2024 #PIDs #PrimaryImmunodeficiency #RaiseAwareness

Accelerating Rare Disease Cures (ARC): Year Two Highlights As the FDA’s Accelerating Rare Disease Cures (ARC) Program wraps up its second year. Here are some key achievements from year two report: ?? 29 Rare Disease Drug Approvals: Through collaborative efforts with industry, researchers, and the FDA, we successfully approved new therapies that address critical unmet needs in the rare disease space. ?? Patient-Centered Initiatives: With 13 externally-led Patient-Focused Drug Development (PFDD) meetings and 15 Patient Listening Sessions, we ensured th at patient perspectives remain central to the development of new therapies. Hearing directly from patients and caregivers about their experiences helps shape clinical trial designs and therapeutic outcomes. ?? Innovative Regulatory Science: Our Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program launched in partnership with CBER, providing enhanced communication and regulatory support for rare disease drug developers. This program exemplifies our dedication to streamlining the development process for novel treatments. ?? Educational Outreach: Through initiatives like LEADER 3D, we are empowering rare disease drug developers with educational tools and resources, promoting a deeper understanding of the regulatory landscape to help navigate the complexities of drug development. ?? Scientific Advancements: The ARC Program has contributed significantly to rare disease clinical research by fostering collaboration on novel trial designs, innovative statistical methodologies, and advancements in clinical pharmacology. These efforts are vital to overcoming the challenges of developing therapies for small patient populations and rare diseases with limited data. ?? Expanding Access: With a focus on expanding access to therapies for conditions like ALS, our collaborations, including the ACT for ALS initiative, are breaking new ground in the development of treatments for neurodegenerative diseases. Looking ahead, ARC remains dedicated to further advancing rare disease treatments. The upcoming Rare Disease Innovation Hub will strengthen cross-sector collaborations and provide a centralized resource for stakeholders. #RareDisease #DrugDevelopment #PatientEngagement #RegulatoryInnovation #ARCProgram #FDA Compliance Architects?

Treatment is very difficult to access in Rare Diseases …! As BiotchPharma and BiotechCRO team, we have given our life, experience and ideals to this struggle together with Rare Patients in Türkiye Trust in our experience and join and support our struggle with us on this path... We will continue to be a breath of fresh air for patients together with all drug development companies, Turkish Physicians and TITCK Ministry of Health Fatih AKTA? at BiotchPharma / BiotechCRO #rarediseases #orphandrugs #compassionateuse #earlyaccessprograms #namedpatientprograms #managedaccessprograms

Last week, CBER’s deputy center director for strategy, policy and legislation Julie Tierney stated that the IRA could negatively impact R&D activities for rare diseases and small molecule development. Revisit our blog post about the IRA and how it puts #RWE front and center here: https://hubs.ly/Q02z0_Yr0 #raredisease #realworldevidence

Treatment is very difficult to access in Rare Diseases …! As BiotchPharma and BiotechCRO team, we have given our life, experience and ideals to this struggle together with Rare Patients in Türkiye Trust in our experience and join and support our struggle with us on this path... We will continue to be a breath of fresh air for patients together with all drug development companies, Turkish Physicians and TITCK Ministry of Health Fatih AKTA? at BiotchPharma / BiotechCRO #rarediseases #orphandrugs #compassionateuse #earlyaccessprograms #namedpatientprograms #managedaccessprograms

???The staggering potential for hepatitis C elimination: A call for political will ahead of World #Hepatitis Day (28 July)??? "The ability to eliminate a viral disease is pretty staggering. It's not something that has been done many times in our history," says Dr Joss O’Loan, General Practitioner and Co-Founder of the Kombi Clinic. "We just really need the political desire to achieve the 2030 goal." Despite making early headway towards the 2030 elimination goal, recent progress has stalled in Australia. "We've reached a stage where the prospect of elimination is real," highlights Dr Paul Slade, Country Medical Director at Gilead Sciences. "The challenge for us now is that we're in a steady state." "The time for talk is over, the time for action is now if we are to achieve the elimination goals by 2030," Dr O’Loan emphasises. Link to full podcast: https://lnkd.in/gqxbW8Y4 #WorldHepatitisDay #gilead #hepc #hepatitisc #pharmaceuticals #medicine #pharma #primarycare #generalpractice The Royal Australian College of General Practitioners (RACGP)