RDI March Newsletter
RARE DISEASES INTERNATIONAL
A global alliance of Persons Living with a Rare Disease
We’re excited to share our March highlights with you! RDI has been actively preparing for the upcoming World Health Assembly in May, with growing momentum and engagement from an increasing number of organizations (over 240) joining the global rare disease coalition. Support for the rare disease resolution continues to strengthen, alongside expanding collaborations and knowledge-sharing, notably through our work on the Essential Medicines List (EML) and the Global Network for Rare Diseases (GNRD), and our participation in key events around the world. We are also delighted to welcome our new youth leaders.
RDI Youth Programme Holds Kickoff Meeting
On 12 March, RDI officially launched the Youth Leadership Programme (YLP) to empower young advocates in the rare disease community. The programme aims to engage young leaders in global decision-making and advocacy efforts.
The YLP will mentor an outstanding group of young leaders from around the world. This initiative aligns with the WHA Resolution on Rare Diseases, promoting innovation, collaboration, and active participation in global health forums. Sponsored by Fondation Ipsen, the YLP offers vital support to ensure young leaders are integrated into the rare disease policy landscape.
Global Survey Report Published
We’re excited to share the final report from our public survey on rare disease expertise, care-providing centers, and networks. The key findings provide an overview of the availability of expertise across countries,the characteristics of centers providing care, the criteria used (if any) to recognize them, and the collaborations in place. You can read the full report here.
WHA Resolution on Rare Diseases Update
The Coalition in support of the WHA Resolution continues to grow, with over 240 organizations now participating. The Coalition will launch a Call to Action on 3 April - check your inbox on Thursday or follow us on Linkedin to learn how you can participate.
We will also launch the next phase of our social media campaign on 7 April. Please check our website to learn how you can take part!
RDI Participation in Global Events
RDI Global Programme Director Chiu-Hui Mary Wang presented a poster at the RE(ACT) Congress 2025, held from 5-7 March in Brussels, Belgium. This event, organized by the BLACKSWAN Foundation, the International Rare Diseases Research Consortium (IRDiRC), and the European Rare Diseases Research Alliance (ERDERA), provided a platform for the exchange of knowledge and ideas in the field of rare disease research, bringing together scientific leaders, distinguished experts, and emerging talents from diverse disciplines. Mary also attended the IRDiRC Consortium Assembly alongside several RDI members from 3-4 March.
RDI CEO Alexandra Heumber Perry participated in a panel discussion on the “Global Commission to End the Diagnostic Odyssey: Policy and Practice” on 26 March as part of the Sanofi Rare Medical Forum in Cambridge, MA, USA. The panel discussions were incredibly insightful, featuring high-level expertise on critical themes for rare diseases, with a genuine focus on the ultimate goal, improving lives of Persons Living with a Rare Diseases. The discussion highlighted that we are at the cusp of a pivotal shift that could positively impact people living with rare diseases, provided that commitment remains strong, as political momentum, knowledge, and innovation are now aligned and mutually reinforcing.
Chiu-Hui Mary Wang, RDI Global Programme Director, and Kirsten Johnson, Chair of the RDI Council of Directors, participated in the Orphan Drugs and Rare Diseases Europe Global Congress 2025 Conference in London on 19-20 March. Mary gave a presentation on the Operational Description of Rare Diseases and Kirsten gave a talk entitled “Building the rare disease ecosystems through the global community of those living with rare conditions” and took part in the opening panel.
RDI Strategic Engagement Manager Debra Bellon and Global Policy Coordinator Alanna Miller participated in a panel discussion as part of the Fifth International Conference on Rare Diseases, organized by RDI Member Rare Diseases Greece. The panel illustrated the multi-stakeholder readiness to make the #Resolution4Rare a reality and to implement the Global Action Plan on Rare Diseases around the world in order to ensure that it drives meaningful outcomes for our community.
Network News
On 25 March, the Dominican Alliance of Patient Associations (ADAPA) submitted a draft bill to the Senate of the Dominican Republic calling for comprehensive support for people living with rare and complex conditions. This represents a landmark achievement for the PLWRD in Dominican Republic.
RDI Member FEPER successfully advocated for modifications to the law on Rare and Orphan Diseases, updating the list of rare conditions covered under the law, promoting the development of technological infrastructure and improving patient registries.
Global Albinism Alliance’s application to include sunscreen on the WHO Essential Medicines List (EML) in regard to persons with albinism was officially accepted for review by the 25th WHO Expert Committee. Aligned with this year's theme for International Albinism Awareness Day, "Demanding our rights: Protect our skin, Preserve our lives," this is a significant milestone in GAA’s mission to promote skin cancer prevention for persons with albinism globally. Learn more about the EML at the RDI webinar on 10 April.
RDI Announces New Partnership with Lundbeck
RDI is excited to announce a new partnership with Lundbeck . Lundbeck is a global pharmaceutical company specialized in brain diseases.
Lundbeck is dedicated to advancing brain health and transforming lives. To fulfill this purpose, Lundbeck is engaged in the research, development, manufacturing, and commercialization of pharmaceuticals across the world. For more than 70 years, Lundbeck has been at the forefront of neuroscience research. You can find out more about the RDI Alliance of Companies on our website.
Upcoming RDI Events
EML Webinar
Learn about the WHO Essential Medicines List (EML), a critical instrument for advancing Universal Health Coverage (UHC) by ensuring equitable access to essential medicines, at our upcoming webinar. However, its role in rare disease treatment remains under-recognized. This webinar will explore how the EML can serve as a powerful tool to improve access to treatments for persons living with a rare disease (PLWRD) at both global and national levels.
Webinar Objectives:
World Health Assembly Side Event
Save the date for our hybrid side event, co-hosted by World Federation of Hemophilia, Thalassaemia International Federation, Sant Joan de Déu Hospital, the Harriet Joyce Foundation, the Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease and Wilhelm Foundation, at the World Health Assembly 78. We will be celebrating 10 years of progress and milestones, and looking forward to 10 years of possibility! Registration is now open for in-person attendance or virtual attendance.
Upcoming Member & Community Events
EURORDIS will hold its Membership Meeting in Riga, Latvia from 22-24 May. The Meeting is open to anyone in the rare disease community and consists of two days of workshops exploring a range of topics from advocacy at both national and EU levels, to volunteer engagement in the medicines lifecycle and healthcare. Look forward to detailed discussions on shared experiences, strengthening collaborations through in-person exchanges, and hands-on workshops to put your skills into practice!
The MENA Congress for Rare Diseases will take place from 17-20 April in Abu Dabi, UAE. RDI CEO Alexandra Heumber Perry will speak about the WHA Resolution on Rare Diseases and the MENA region’s role in shaping the its impact. Register here.
Rare Disease Opportunities
AstraZeneca is looking for people from all corners of the world who are committed to advocacy for health equity with a specific focus on non-communicable disease prevention and rare diseases. Apply to join AstraZeneca’s Young Health Programme Impact Fellowship for the opportunity to receive:
Find out more and apply now: https://bit.ly/4bidAIo
IRDiRC’s Therapies Scientific Committee (TSC) is looking for three new members to expand its expertise in rare disease therapeutic development. The TSC plays a key role in achieving IRDiRC’s goal: 1,000 new rare disease treatments by 2027. They are seeking experts from diverse backgrounds (industry, academia, regulatory, clinical, health technology) with: ✅ A strong track record in rare disease therapy development (medicines and/or medical devices) ✅ Expertise across the therapeutic spectrum (prevention, management, quality of life, curative care) ✅ Commitment to active participation in meetings and committee activities Apply by April 6, 2025 by sending your CV, biosketch, and letter of motivation to: scientific.secretariat@irdirc.org
The Ataxia Charlevoix-Saguenay Foundation, which is dedicated to advancing research and developing a treatment for Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS), has launched its 2025 Call for Proposals to support innovative research on ARSACS. This funding opportunity aims to encourage scientific advancements by providing financial support to researchers working on projects related to the disease. Funding is up to $100,000 per project – Multiple projects may be funded! Deadline is 23 May, 2025. Apply now and share this opportunity.
Recommended Reading
Improving Care for Rare and Undiagnosed Diseases – The Role of Care Pathways Patient care pathways are key to managing rare diseases. These multidisciplinary frameworks ensure that every step—from diagnosis to treatment and care—follows a coordinated and structured approach. They integrate medical and non-medical disciplines to streamline care, reduce diagnostic delays, and improve health outcomes. In the context of rare diseases, where conditions are often complex and multisystemic, care pathways help bring together experts from various fields, ensuring people living with rare diseases receive the right care at the right time. These pathways also include mental health support, counseling, and patient education to help families navigate the complexities of the healthcare system.
Read the article by RDI’s Global Programme Junior Manager Sara Brambilla , along with Gareth Baynam , Roberto Giugliani , Holm Graessner , Carmencita Padilla and Chiuhui Mary Wang .
"Drug–device combinations in rare diseases: Challenges and opportunities", published in Drug Discovery Today. Drug–device combinations (DDCs) offer a groundbreaking approach to treating rare diseases by enhancing drug delivery, improving targeting, and minimizing side effects. However, their development is hindered by complex regulatory frameworks and the absence of specific incentives—unlike orphan drugs.
Welcome Sandy Laham
Meet Sandy Laham , RDI’s new Resource Mobilisation and Partnerships Manager! With over eight years of experience leading public health initiatives across Europe and LMICs, Sandy has worked on health system strengthening projects, immunization programs, cancer prevention and improving health inequalities.
She has managed initiatives funded by the World Bank, WHO, and the EU, and she’s experienced in bringing together key stakeholders to drive meaningful change. Her ability to forge strategic partnerships is fueled by her deep expertise and passion for creating lasting health solutions.
Originally from Lebanon, Sandy speaks four languages and holds a master's in public health from the French School of Public Health (EHESP). She made France her home in 2019 and became a mother in 2024.
During off-hours, you’ll find Sandy indulging in her love for reading, traveling, and painting.