Esther Nzenza’s Post

At EUCOPE, we're passionately committed to building an innovative #RareDisease ecosystem and addressing critical gaps, such as the 95% of rare diseases currently without #treatment options. 🚫💊   #Innovation in rare diseases demands a holistic approach. Collaborating with the OD Expert Group, we've developed 14 forward-thinking policy recommendations across the entire rare disease lifecycle, from R&D to #access. These proposals encompass a coherent policy framework for #RWE, encourage iterative early dialogue for #EMA, #HTA bodies, and #OMP developers, and advocate for an #EU rare disease hub for large-scale collaboration (including public-private partnerships), data sharing and generation, #diagnosis.   🧬The revision of the #OMPRegulation marks a significant step in streamlining the regulatory framework and future-proofing it against scientific advancements. This is vital for translating research into the development of new #OrphanDrugs.   💡 However, this revision needs to be complemented by additional actions at both EU and Member States levels to fully leverage existing tools like the European Reference Networks (#ERNs). Improvements are necessary in areas like rare disease diagnosis, translational research, integrated care pathways and patient access.   🎯 Ultimately, an EU Rare Disease Action Plan would be a powerful statement of intent, setting ambitious goals for us to work towards. It would unify different policy areas, from research and industrial actions to regulatory initiatives and care pathways.   #RareDiseaseWeek #RDD2024 #RareDiseaseInnovation #OrphanDrugDevelopment #HealthcareCollaboration #PatientAccess

The diagnostic odyssey 🗺️ Improving access to diagnostic tools and services for patients with rare diseases is a key priority for EURORDIS. This includes advocating for the development and implementation of new diagnostic technologies, as well as working to reduce the cost and improve the availability of existing diagnostic tests. The Screen4Care project, which EURORDIS is a partner of, aims to harness the power of recent technological innovations to improve diagnostics for rare diseases and combat this phenomenon. Our role in the project is to direct and advise all partners on the priorities, needs, and perspectives of people living with rare diseases. Find out more about the project and our involvement: 👉 https://lnkd.in/eQSwrgXx #Screening4Rare #NewbornScreening #healthcare #RareDiseases #project #innovation #collaboration

We hold the record for enrolling the most patients in the shortest amount of time for any global trial in the last 50 years. That’s because Elixia has reinvented processes for repeated successes. https://elixiahealth.com #Elixia #clinicaltrials #clinicaltrialmanagement

💡 Join Anlitiks Inc. for an insightful webinar on the increasingly significant role of Real-World Comparator Cohorts (#RWCC) in clinical trials, especially in rare and complex disease populations where traditional randomized controlled trials (RCTs) may be infeasible or unethical. Featured speaker, Kitty Rajagopalan, will delve into methodological considerations surrounding the use of RWCCs in clinical trials! ➡️ REGISTER HERE: https://buff.ly/3SnjxLY #ClinicalTrials #ClinicalResearch #RandomizedControlledTrials #RealWorldData #RealWorldEvidence #ClinicalData #HEOR #RareDisease

AMR however threatens not just lives through illnesses that do not respond to treatment, but our entire healthcare system. As ‘SCIENCE’ puts it: "Antibiotic resistance undermines much of modern health care, which relies on access to effective antibiotics to prevent and treat infections associated with routine medical procedures" [17]. As The World Health Organization explains "The direct consequences of infection with resistant microorganisms can be severe, including longer illnesses, increased mortality, prolonged stays in hospital, loss of protection for patients undergoing operations and other medical procedures, and increased costs" [3]. Visit the Senzo website to request the full report... #AMR #AntimicrobialResistance #Superbugs #PostAntibioticEra #AntibioticStewardship #InfectionControl #GlobalHealth #AMRActionFund #Diagnostics #NewAntibiotics #InfectiousDiseases #DrugResistance #Healthcare #PublicHealth #Medicine #Pharma #Biotech #IDWeek #ASMmicrobe #ICAAC #ECCMID #microbiology

POCTA has been advocating for the Molecular Diagnostic Services Program (MoIDX) to clarify that CLIA-waived labs are not required to register and obtain a Z-code for CLIA-waived tests. After a request from POCTA, we are pleased to announce that MolDX has issued an updated FAQ in which they confirm that CLIA-waived labs are not required to register or obtain a Z-code for CLIA-waived tests. This is important for point of care (POC) testing because previously, many POC labs believed they would need to register and obtain a Z-code, which would have placed an additional administrative burden on these important health care providers. Read the updated MoIDX FAQ here: https://lnkd.in/eaDbj7td #pointofcaretesting

Developing and launching orphan medications for rare diseases is a complex process. In Europe the diverse healthcare environments, regulatory frameworks, and cultural contexts add many layers of additional complexity. Within this intricate landscape Patient Support Programs can add significant value, creating a bridge between innovative, life-changing therapies and patient-centric, holistic care. 📥 Download our guide to learn how to successfully set up a PSP in Europe: https://lnkd.in/eZGMD3R9 #RareDiseases #Biotech #PatientSupportProgram #PSPs #PatientOutcomes #OrphanDrugs #LifeSciences

About 230 million people worldwide have eczema, which can negatively impact their quality of life. Unfortunately, there's no cure for it. That's why our team is working on a couple of research studies to develop potential treatments. We're excited to share that our team had the first study participant in New Zealand and worldwide for one of those studies, and we've also brought in the first randomised patient in the ANZ region for the other. #AtopicDermatitis #Eczema #ClinicalTrials #OptimalClinicalTrials

Managing a clinical trial is inherently tricky. So why add headaches by using different vendors for individual safety and compliance reviews? Advarra provides IRB, IBC, DSMB, and clinical event adjudication.

Today, NICE - National Institute for Health and Care Excellence recommend the first therapy to treat #epidermolysisbullosa. Filsuvez® (birch bark extract), will be available to people in England and Wales (aged 6 months and older) living with dystrophic or junctional epidermolysis bullosa (EB). NICE recommended Filsuvez®, a topical, thixotropic gel, as part of their HST programme, after a single Evaluation Committee Meeting. Tolley Health Economics Ltd are delighted to have worked with Amryt Pharma since 2019 as they navigated the challenges of HTA in a rare disease, with the first licensed therapy for EB. Our team supported Amryt Pharma with many evidence generation activities, beginning with NICE early scientific advice and culminating in building the first, fit-for-purpose, HTA-ready cost-effectiveness model for EB. The availability of Filsuvez® marks a shift in the EB treatment paradigm and paves the way for future EB pipeline products, providing patients and their families hope for the future. Here https://lnkd.in/eKVRUZuu we describe the challenges encountered along the journey to gaining reimbursement, and the working solutions applied. #epidermolysisbullosa #fightEB #cureEB #raredisease #hta