AstraZeneca’s Post

What a privilege to have an eye-opening, up-close "encounter" with the KSA advances in rare disease. I had the honour of being invited to Riyadh the previous year when they launched the "Moonshot for Rare" program but had no idea they were not only going to fire the rocket but to land the ship and start building. Having a vision and commitment fuelled by investment in science is putting KSA at the forefront of realizing the possibility of a better life for rare disease patients and families. Cornerstones: integrated and comprehensive Rare Disease programs from screening/diagnosis to optimal care; patient data platforms for collecting, integrating and using real-world, real-time data; evidence-informed, multi-stakeholder, multi-criteria decision-making for efficient and cost-effective access to medicines and other therapies. Special kudos to @Ahmed Aljedai and @Ali Asheri for such willingness to share knowledge and expertise.

In the presence and under the patronage of H.E. Sheikh Nahyan bin Mubarak Al Nahyan, Cabinet Member and Minister of Tolerance and Coexistence, the MENA Congress for Rare Diseases has commenced in Abu Dhabi. The four-day event brings together 141 speakers, including 51 international experts, from 27 countries. BMC | Burjeel Medical City, the flagship facility of Burjeel Holdings, partners with the MENA Congress for Rare Diseases. The largest event in the field, the congress comprises 41 sessions covering a wide range of topics including recent research, advancements in diagnostics and therapeutics, regional experiences, and challenges related to rare diseases. Read More: https://lnkd.in/dkK_wD4A “I am delighted that BMC, with the guidance of Dr. Shamsheer Vayalil, is demonstrating its leadership in our country’s medical sector by hosting this congress. The launch of their project NADER here today also signals their determination to be the best in the field of treating rare diseases,” added Sheikh Nahyan. Rare diseases affect at least 5% of the population, and the MENA (Middle East and North African) region has a higher prevalence of rare diseases. Organizers crafted the congress agenda to be comprehensive and inclusive of the majority of rare disorders, focusing on highlighting key unmet needs and potential solutions through best clinical practices and research. Department of Health Abu Dhabi Professor Dr. Ayman El-Hattab, Professor Khaled Musallam, #mededgemea #BurjeelMedicalCity #menararediseases2024 #abudhabi

📢 Excited to share my latest publication: "Understanding Global Access to Topical Onychomycosis Therapy: A Systematic Review and Meta-Analysis," now published in *Mycoses*. 🔍 Our study delved into the global accessibility of topical treatments for onychomycosis, an infection where treatment choices are often complex due to dynamic changes in infective species. We examined the combination of infection prevalence, medicine efficacy, and regulatory availability to assess access to these essential medicines. 💡 Key Findings: - Access to effective topical treatments for onychomycosis is a global concern, with 90% of the assessed countries lacking access to the most efficacious products. - 22% of countries had no access to any broad-spectrum agents. - The problem is particularly severe in African countries. - Our research also highlighted the under-representation of Candida infections in clinical studies, which may be contributing to access issues. 🔗 You can read the full paper here:   https://lnkd.in/ea6mbDCh A big thank you to my co-authors and funding from the EPSRC, UK (grant number EP/V009567/1) for making this work possible. Let’s continue the conversation on improving access to essential medicines worldwide! 🌍💊 #Research #PublicHealth #Onychomycosis #HealthcareAccess

Exploring the Impact of Sickle Cell Disease in Saudi Arabia. A recent study highlights the significant economic and health-related challenges faced by patients with SCD in Saudi Arabia. Helping to get a better overview of SCD in KSA. Below the key findings: 1)High Financial Burden: Patients incur substantial out-of-pocket expenses, averaging USD 650.69 monthly, exacerbating financial strain. 2)Quality of Life: Frequent blood transfusions and comorbidities negatively impact patients’ health-related quality of life (HRQoL). 3)Educational Influence: Higher education levels are associated with better HRQoL among adult patients. 4)Urgent Need for Systemic Solutions: The study underscores the necessity for comprehensive approaches to address SCD’s challenges. These insights call for enhanced support and targeted interventions to improve the lives of those affected by SCD. https://lnkd.in/gu3AxcA7

📢As world leaders gather in New York to discuss the growing global health threat, the Fleming Initiative is poised to break down barriers to progress. Three new partners - LifeArc, Cepheid and Optum - have pledged additional funding and resources to the Fleming Initiative’s global effort to combat antimicrobial resistance (AMR), as leading medical expert Professor Lord Ara Darzi calls for tighter restrictions on the use of antibiotics. The Fleming Initiative brings together research scientists, policymakers, clinicians, behavioural experts, public and commercial partners to provide the networks and expertise to provide solutions to AMR at the global scale. The announcement comes at a critical moment. For only the second time, world leaders are preparing to gather for a United Nations General Assembly meeting on AMR in New York. Read more in this news piece: https://lnkd.in/ecZUdbtK #AMR #AntimicrobialResistance #GlobalHealth

𝐊𝐞𝐲 𝐭𝐚𝐤𝐞𝐚𝐰𝐚𝐲𝐬 𝐟𝐫𝐨𝐦 𝐭𝐡𝐞 𝐖𝐨𝐫𝐥𝐝 𝐎𝐫𝐩𝐡𝐚𝐧 𝐃𝐫𝐮𝐠 𝐂𝐨𝐧𝐠𝐫𝐞𝐬𝐬 𝐄𝐮𝐫𝐨𝐩𝐞 🌍   As the World Orphan Drug Congress in Barcelona concludes, Sven Tops and Frank Vanderdonck reflect on some key takeaways from the conference. Attending the event was a great opportunity to listen to and contribute to discussions on evidence needs and standards for rare and ultra-rare diseases:   - Flexibility in evidence standards: Health Technology Assessment (HTA) bodies often have rigorous evidence thresholds, yet randomised controlled trials (RCTs) are often challenging or unfeasible for many rare or ultra-rare diseases. Pragmatic evidence approaches—such as case reports, registries, and natural disease history studies—should be recognised as viable alternatives.   - Adapting payment models : Linking pragmatic evidence generation directly to payment models could ensure faster access for patients who urgently need new and highly specialised treatments. This would support great access and timely care without compromising the evidence base.   - Incorporating patient and caregiver voices: It's essential that the patient and caregiver perspectives are actively included in evidence generation and data presentation, especially within HTA evaluations. Their insights are invaluable in ensuring a holistic understanding of disease impact. While data can show the efficacy of a treatment, patients and caregivers provide an important insight into how that treatment will impact daily life, whether it improves their quality of life or addresses unmet needs.   - Championing a European Action Plan for rare diseases: The keynote panel highlighted the need for political commitment and shared accountability across the EU to drive the plan’s success and ensure meaningful impact. There was a focus on the need to align existing regional, national, and supranational initiatives in order to promote continued innovation and access to new treatments.   We are looking forward to seeing how these perspectives can help shape the future of rare disease treatment in Europe, and greater patient access.     #WorldOrphanDrugCongress2024 #WODC #RareDiseases #HTA

Module 2 of our course is online!

More Exciting News from Medalis Ltd! We are thrilled to announce that our work on Hypereosinophilic Syndrome (HES) will be presented at DGHO 2024 on October 13th, 2024 in Basel, Switzerland! The poster, titled: "Consensus on the disease manifestations of hypereosinophilic syndrome" demonstrates the value of the Jandhyala Method in developing one of the most comprehensive lists of HES disease manifestations to date. This innovative, mixed-methods approach allowed us to systematically gather and analyse insights from healthcare professionals across multiple specialties and geographies, reaching consensus on key HES disease manifestations. Our study identified over 300 HES-related manifestations and, using the Jandhyala Method, refined these into a core set of consensus-driven items. This list represents a significant step forward in understanding the diverse presentations of this complex disease and highlights the need for a multidisciplinary approach in HES management. This work underscores the power of collaborative research and the value of structured methods like the Jandhyala Method in achieving clinically relevant insights. Thank you to all the HCPs who contributed to this project, and to DGHO for the opportunity to share our work. Let’s continue to advance the understanding and treatment of rare diseases like HES! #DGHO2024 #RareDiseases #HypereosinophilicSyndrome #HES #HealthcareInnovation #JandhyalaMethod #MedialisLtd

Every now and then I get a distinct sense of pride, usually around a new publication or presentation using #jandhyalamethod and #neutraltheory. This work underlines the utility of the method in generating a comprehensive of #HES disease manifestations which is free of reporting #bias. We mapped the disease #phenotype describing >300 separate indicators making this the most detailed and exhaustive research in HES to date. Every disease needs such a list, otherwise we are not seeing the full picture. We will miss important aspects of the disease and consequently miss many ['hidden'] benefits of treatments. If you would like help in develop a Neutral List of Disease Manifestations for your disease, get in touch on: [email protected] R

Highlights: The peak of the #COVID19pandemic had a negative effect on quality of life (#QoL) and was a challenging situation for both #Thalassemia patients and #healthcare systems. This study reports real-world data on quality of life in #transfusion-dependent thalassemia during the COVID-19 lockdown restriction measures in Greece The level of quality of life remained low, the supportive measures for the transfusion-dependent thalassemia patients failed to protect most of the significant quality of life subdomains, whereas older, married, and higher educated patients were the more vulnerable groups. The supportive measures for high-risk patients should aim for a more holistic approach, and vulnerable patient groups warrant early protective interventions. The universality of the #TranQoL questionnaire may allow the generalizability of our study’s results and the comparison of quality of life in transfusion-dependent thalassemia patients among other countries and healthcare systems.