NOVARTIS
18.6.2020 01:03:14 CEST | Business Wire | Press release
STRICTLY FOR EUROPEAN MEDICAL AND PHARMACEUTICAL TRADE MEDIA ONLY
Many patients with SCD only live into their 40s and endure unpredictable pain crises which disrupt their lives physically, socially and emotionally.1 It is a genetic condition which is life-long and rising across Europe.2 Despite the World Health Organization recognising SCD as a global problem, knowledge about SCD among healthcare providers and the public is poor.3 SCD remains an invisible health issue in Europe and Novartis is dedicated to help make SCD a healthcare priority in Europe.
“SCD is a neglected chronic disease of increasing global health importance. There is a lot of work to be done to help the growing numbers of patients in Europe,” Professor Béatrice Gulbis, Co-coordinator of the European Reference Network EUROBLOODNET and specialist of inherited rare anemias. “It is important for healthcare professionals, policymakers, patients and the public-at-large to understand the severity of sickle cell disease and take a European-wide collaborative action on education, funding for research and awareness, and guidance and tools for screening and follow-up.”
SCD is a genetic blood disorders affecting millions of people worldwide. 4 It is a life-long and debilitating disease.5 It also has a substantial impact on patients’ emotional well-being and daily life affecting their ability to work and complete education.6 SCD financially affects the individual and their family; society and healthcare systems. 7 The burden of blood disorders in Europe is €23 billion per year, a level of cost that is not matched in current European haematology research funding.7
Early diagnosis and regular medical care can prevent complications and contribute to improved life expectancy and quality of life, however, a recent survey revealed that many patients don’t seek care despite symptoms and complications due to previous poor experience at hospital (39%) with some reporting that healthcare providers do not understand the disease (26%).6,8 Novartis is calling for a European-wide collaborative action on education, funding for research and awareness; and make sure that the patients receive equal and homogeneous healthcare access and treatment across Europe .
Tomorrow, a panel of EU experts and patients will be coming together for a one-hour webinar to highlight the lack of awareness and mis-conceptions of SCD amongst policy makers and the public in Europe. The meeting aims to highlight the action required to help improve the lives of those living with this debilitating disease. The experts will explore:
- SCD and the complexity of its diagnosis and management
- The burden of SCD on quality of life for patients and caregivers
- The impact of the COVID-19 pandemic on existing and emerging rare diseases in Europe
- Why there is a need for urgent strategy to address equal access to treatment in Europe
The expert panel comprises:
- Miriam Santos Freire, Patient Advocate
- Mariane De Montalembert, Healthcare Professional – (Eurobloodnet, ERN)
- Simone Boselli, Public Affairs Director – (European Organisation for Rare Diseases, EURORDIS)
- Yolande Adjibi, Head of Patient Relations in Sickle Cell Disease – (Novartis, Region Europe)
Yolande Adjibi, Head of Patient Relations in Sickle Cell Disease at Novartis comments: “For over 40 years, Novartis has been committed to understanding SCD and working towards treatment. We aim to support patients, healthcare providers and caregivers in their experiences with this debilitating disease. However, more needs to be done. We need to start conversations which will spark a positive change for SCD patients.”
In 2016 The European Hematology Association identified a need to improve treatment strategies for both acute and chronic complications of SCD to optimise patient care, in order to positively affect patient health and quality of life, and to reduce hospitalisation length, patient disability, and cost of care but limited progress has been made.7 As COVID-19 poses an additional threat to the lives of SCD patients and subsequently healthcare systems across Europe, urgent action is required to help improve the lives of those affected by SCD and to guide healthcare systems towards early diagnosis and equal access to treatment.
To join the conversation and pose questions directly to the panel please register here: https://us02web.zoom.us/webinar/register/WN_NB8ARtG2SMqJiCcM8MC9Kg
- END –
About Sickle Cell Disease
Sickle cell disease is a debilitating inherited genetic blood disorder that affects the shape of the red blood cells and can make blood cells and blood vessels stickier than usual.9 ,10 When blood cells stick to one another, they can form multicellular adhesion clusters in the bloodstream. These clusters can reduce and block the flow of blood and oxygen, which can cause damage to the blood vessels and lead to acute and chronic complications.6,9 These blockages also can lead to painful crises called VOCs, which are considered the clinical hallmark of the disease and the main reason why patients seek medical care in hospitals.9
About Novartis
Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 105 000 people of more than 140 nationalities work at Novartis around the world. Find out more at www.novartis.com .
References
1 Adegbola M, et al. 2012. Voices of adults living with sickle cell disease pain. J Natl Black Nurses Assoc. 23(2), pp.16-23.
2 Roberts I, de Montalambert M. 2007. Sickle Cell disease as a paradigm of immigration haematology: new challenges for hematologists in Europe. Haematologica 92(7)pp.865-71.
3 Mburu, Joy; Odame, I. 2019. Sickle Cell Disease: Reducing the Global Disease Burden. Int J Lab Hematol (41) 1:82-88.
4 Jain D, Lothe A, Roshan C. 2015. Sickle cell disease: current challenges. Journal of Hematology & Thromboembolic Diseases . 10. Doi: 10.4172/2329-8790.1000224.
5 Steinberg M. 1999. Management of sickle cell disease. N Engl J Med . 340(13):1021-1030.
6 Osunkwo I, Andemariam B, Inusa B, et al. Management Strategies and Satisfaction Levels in Patients With Sickle Cell Disease: Interim Results From the International Sickle Cell World Assessment Survey (SWAY). Poster presented at: The American Society of Hematology Annual Meeting; December 7-10, 2019; Orlando, FL.
7 European Hematology Association (EHA) Roadmap for European Hematology Research: A Consensus Document (2016), Available at: http://www.haematologica.org/content/101/2/115 (Accessed June 2020).
8 National Heart, Lung and Blood Institute. Sickle Cell Disease. Available at: https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease (Accessed June 2020).
9 Gutsaeva D, Parkerson J, Yerigenahally S, et al. 2011. Inhibition of cell adhesion by anti-P-selectin aptamer: a new potential therapeutic agent for Sickle Cell Disease. 13;17(2), pp.727-35.
10 Sparkenbaugh E, Pawlinski R. 2013. Interplay between coagulation and vascular inflammation in sickle cell disease. Br J Haematol. 162(1). Pp.2-14.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200617005698/en/
Link:
About Business Wire
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Datroway® Recommended for Approval in the EU by CHMP as First-Line Treatment for Patients with Metastatic Triple Negative Breast Cancer Who Are Not Candidates for Immunotherapy26.6.2026 13:00:00 CEST | Press release
Recommendation based on TROPION-Breast02 phase 3 trial where Daiichi Sankyo and AstraZeneca’s Datroway showed a statistically significant and clinically meaningful improvement for the dual primary endpoints of overall survival and progression-free survival If approved, Datroway has potential to be the first TROP2 directed antibody drug conjugate for patients in EU with a demonstrated overall survival benefit as first-line treatment Datroway® (datopotamab deruxtecan) has been recommended for approval in the European Union (EU) as monotherapy for the first-line treatment of adult patients with unresectable or metastatic triple negative breast cancer (TNBC) who are not candidates for PD-1/PD-L1 inhibitor therapy. Datroway is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca (LSE/STO/NYSE: AZN). The Committee for Medicinal Products for Human Use
DAYBU®(trofinetide) Recommended for Approval in the European Union by CHMP26.6.2026 12:49:00 CEST | Press release
-- European Commission decision expected in the coming months-- If approved, DAYBU®would become the first treatment for neurobehavioral symptoms of Rett syndrome in the European Union Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion following a re-examination procedure, recommending the granting of a marketing authorization for DAYBU® (trofinetide) for the treatment of neurobehavioral symptoms of Rett syndrome in adults and pediatric patients aged five years and older. If granted marketing authorization by the European Commission, DAYBU® would be the first therapy approved for this indication in the European Union (EU). “The CHMP’s positive opinion for DAYBU® is an important milestone in our mission to bring this innovative therapy to the EU, where there are no therapies specifically approved for the neurobehavioral symptoms of this devastating
HTEC Showcases Lakebase Branching at Databricks’ Data & AI Summit26.6.2026 11:18:00 CEST | Press release
HTEC, the global technology and AI engineering firm headquartered in Silicon Valley, last week showcased how it used Lakebase data branching to enable faster development, safer operations, and new possibilities for data-driven organizations operating in highly regulated environments. The Solution was developed in collaboration with a leading risk and compliance technology provider who serves financial institutions. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260626875859/en/ HTEC, the global technology and AI engineering firm headquartered in Silicon Valley, last week showcased how it used Lakebase data branching to enable faster development, safer operations, and new possibilities for data-driven organizations operating in highly regulated environments. Solving Critical Development Bottlenecks in Regulated Environments At the core of the collaboration was a shared challenge: how to enable modern data workflows in an envi
Stallergenes Greer Named One of Switzerland’s Best Managed Companies for 202626.6.2026 10:28:00 CEST | Press release
Stallergenes Greer, a global leader in allergy care, has been recognised for its overall business performance, company culture, and sustained growth by receiving the prestigious 2026 Switzerland’s Best Managed Companies award. The 2026 Best Managed Companies programme award winners are among Switzerland’s best private companies. The awards are derived from Deloitte’s global Best Managed Companies awards programme, an internationally recognised programme that evaluates businesses based on their leadership in the areas of strategy, culture and commitment, capabilities, innovation, governance and financial performance. Applicants are evaluated by an independent jury of experts. The evaluation of the Switzerland awards is based on more than 30 years of observed practice from the global awards programme that has been rolled out in 50+ countries worldwide. This recognition reflects Stallergenes Greer's consistent ability to deliver on its strategic vision while maintaining the operational di
Roboverse Reply Impresses at ELROB 2026 With Intelligent Robotic Systems for Critical Missions26.6.2026 10:00:00 CEST | Press release
Roboverse Reply, the Reply Group company specializing in robotics and automation, won the 1st Place in the “Reconnaissance” category and a special prize for “Best Team Effort” at the European Land Robot Trial (ELROB) 2026 in Thun, Switzerland. Teaming up with ELP, a specialist for technical equipment for defusing service, the company demonstrated outstanding performance alongside a field of international participants from June 15 to 19, proving that its autonomous solutions operate reliably under real-world conditions. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260626012237/en/ Roboverse Reply won 1st Place in the “Reconnaissance” category and a special prize for “Best Team Effort” at the European Land Robot Trial 2026, proving that its autonomous solutions operate reliably under real-world conditions. ELROB is Europe’s most demanding and established showcase for robotics and unmanned systems, where leading experts from
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.
Visit our pressroom
