Novartis UK’s Post

❓Did you know that fewer than 5% of the known 10,000+ rare diseases have an approved treatment? The journey is long and complex, but patients, caregivers and advocates can play a powerful role in the drug development process. In an educational series created by the National Organization for Rare Disorders (NORD) with the FDA and Critical Path Institute, The Progeria Research Foundation (PRF) Co-Founder and Medical Director Dr. Leslie Gordon shares her journey in helping advance research toward treatments and the cure for Progeria. The Rare Disease Drug Development Series is a free, 3-part course that gives patients, caregivers and advocates the knowledge needed to actively participate in drug development. You’ll hear about PRF’s journey in helping advance a new therapy for Progeria, among other success stories. The course covers essential topics like: 💙 How personal stories can influence research 💙 Working with the FDA, the pharmaceutical industry and researchers 💙 Involvement in clinical trials https://lnkd.in/eqhw3Zk3 #clinicaltrials #findthecure #raredisease #research #DrugDevelopment #RareCommunity

Cystic fibrosis (CF) creates serious obstacles for patients, impacting their lungs and digestive systems. In a major breakthrough, NHS England has finalized a long-term deal with Vertex Pharmaceuticals, guaranteeing ongoing access to critical CF medicines like Kaftrio, Symkevi, and Orkambi for both children and adults. This agreement resolves a lengthy cost dispute with NICE, highlighting the transformative benefits of these treatments for patients. Explore over 5,000 agreements in Lyfegen’s comprehensive Agreements Library, the largest global resource of its kind: https://lnkd.in/euuJz8sC #HealthcareInnovation #CysticFibrosis #Pharmaceuticals #NHS #PatientOutcomes #AgreementsLibrary

Looking for a trusted CRO in Texas? Look no further than HGI Clinical Solutions! We are a relationship-focused #CRO, committed to providing personalized support for your clinical trials. With a broad range of therapeutic experience and expertise in investigational product types, study designs, and operational strategies, we bring the know-how to help your trials succeed. Our team, with an average of 15 years in the clinical trial industry, has participated in hundreds of studies across multiple therapeutic areas, including early phase and oncology-focused trials. We've supported numerous biotech and pharma clients, tackling even the most complex study designs. Partner with HGI Clinical and experience a dedicated approach that prioritizes your trial’s success. Learn more about our services at https://lnkd.in/e_DEw649 #ClinicalTrials #CRO #OncologyTrials #ClinicalResearch #Biotech #Pharma #EarlyPhaseTrials #TexasCRO #ResearchAndDevelopment #HGIClinical #ClinicalTrialManagement #DrugDevelopment #TherapeuticResearch

According to The European Federation of Pharmaceutical Industries and Associations (EFPIA)’s 2021 report, England and Scotland are jointly ranked 13th in Europe in terms of access to combination treatments. Slipping down 10 places!   If a comprehensive solution isn’t found, the UK clinical community will continue to face frustration if treatments, known to be beneficial in clinical trials, cannot be used routinely in the NHS. There is the risk that treatment standards could fall behind that of other countries.     As part of our video series, 'Combination Treatments: Why Access is Needed’, Helen M Taylor (Programme Access Lead at Takeda UK&I), Eric Low (Member of Takeda’s Advisory Group), and Vanessa Ohlmeyer (Head of Medical Affairs, Oncology at Takeda UK&I) discuss the implications for the life sciences industry and patients, as well as our proposed solution to tackle the access barriers.   For further information on our proposed solution visit here: https://lnkd.in/eY9c4UbD   #CombinationTreatments  

Within the ever-shifting domain of pharmaceutical research and development, there is a continual need to underscore the value of preserving the precision and relevance of an Investigator Brochure. This case study takes you deep into a pivotal task: the thorough refinement of an Investigator Brochure tailored for a groundbreaking investigational product, meticulously crafted to address respiratory diseases. Here, we will explore the nuances, obstacles, and moments of success encountered by a resolute team of experts devoted to advancing healthcare through exacting scientific exploration. View more………..https://lnkd.in/dNWkBMSd

Within the ever-shifting domain of pharmaceutical research and development, there is a continual need to underscore the value of preserving the precision and relevance of an Investigator Brochure. This case study takes you deep into a pivotal task: the thorough refinement of an Investigator Brochure tailored for a groundbreaking investigational product, meticulously crafted to address respiratory diseases. Here, we will explore the nuances, obstacles, and moments of success encountered by a resolute team of experts devoted to advancing healthcare through exacting scientific exploration. View more……….. https://lnkd.in/gYVkAuM6

Within the ever-shifting domain of pharmaceutical research and development, there is a continual need to underscore the value of preserving the precision and relevance of an Investigator Brochure. This case study takes you deep into a pivotal task: the thorough refinement of an Investigator Brochure tailored for a groundbreaking investigational product, meticulously crafted to address respiratory diseases. Here, we will explore the nuances, obstacles, and moments of success encountered by a resolute team of experts devoted to advancing healthcare through exacting scientific exploration. View more………..https://lnkd.in/dNWkBMSd

We’re proud to support the Our Common Goal campaign from the The Association of the British Pharmaceutical Industry (ABPI), uniting us all in a shared vision: a future where health conditions don’t define how we live.   This campaign emphasises the critical role of collaboration between the pharmaceutical industry, the NHS, patient organisations and other key partners. It’s a mission that deeply resonates with our commitment to innovative science, meaningful partnerships, and advancing health equity.   Together, we’re working to create new possibilities for those impacted by cancer and beyond, so that patients can enjoy a life well lived. UKI-UNB-0140 | November 2024

Many pharmaceutical companies are turning to patient support programs to ensure their products reach their full potential and improve patient outcomes. When deploying PSPs across Europe, they may encounter a unique set of challenges related to navigating the diverse healthcare landscape of the region. In our latest case study, we present how Sciensus successfully supported a US biotech in deploying a European patient support program for a pioneering therapy targeting an ultra-rare paediatric disorder. To learn more, visit: https://lnkd.in/eXPK7Vqd #PSP #PatientSupportProgram #RareDiseases #OrphanDrugs