February is a rare month in our calendar year. It is shorter than the other months and it has an ad hoc day every four years to get the Earths revolution around the sun back in line. So it is clearly a fitting month in which to celebrate Rare Disease Day (RDD), which takes place on the last day of February each year to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients’ lives.
RDD is organised by EURORDIS-Rare Diseases Europe, a patient-driven alliance representing over 900 rare diseases patient organizations from 73 countries that work together to improve the lives of the 30 million people living with a rare disease in Europe.
From its early days, Galapagos’ motto has been to pioneer for patients. That is because at Galapagos we want to make a meaningful difference not just in the way diseases, including rare diseases can be treated but also on how people are living with these diseases, day-to-day. That is why we are delighted to have joined the EURORDIS Round Table of Companies (ERTC), which fosters a long-term educational relationship and a constructive dialogue between EURORDIS and companies addressing rare diseases. As Patient Advocacy Lead at Galapagos, I have the privilege of working closely with various patient communities and organisations, such as EURORDIS, with the aim of learning from and demonstrating our support of patients and their families. It is a great opportunity, as I get to better understand patient needs, support the patient agenda, and partner to find new solutions for the challenges faced every day.
An example of how we want to ensure our work will make a meaningful difference for patients’ lives and allow us to be recognised as a purposeful partner by the patient community and beyond, we are developing a Patient Partnership Charter. This will be our reference document internally and externally, defining our mission and vision for patient partnership and engagement, our values and principles as a company committed to putting the needs of patients first in all aspects of our activities, from discovery, through development and into delivery of new medicines for the patient community. Most importantly, the Charter will be a tool that will create value for patients, with patients. It will be developed together with patient organisations, patient advocates and experts who will be involved in all consultations and development stages.
So, in living up to the principles that will direct the development of our Charter, I am delighted to have had the opportunity to talk to Celine Schwob, Corporate Relations Senior Manager at EURORDIS, who provided me with insight into the significance of RDD and why it’s important for the whole rare community and its supporters to come together on this day. Read below to find out more.
Marina Sardone: Can you tell us a little about EURORDIS and your organization’s mission?
Celine Schwob: Founded in 1997, EURORDIS-Rare Diseases Europe is a not-for-profit alliance of 956 rare disease patient organisations from 73 countries around the world. We work together across borders and diseases to improve the lives of the 30 million Europeans living with a rare disease.
We work toward our vision of enabling better lives for people living with rare disease by advocating for them, helping them to be part of the decision-making process and uniting the rare disease community to empower them. This helps ensure policies, services and research all take into account the real needs of people affected by a rare disease. We have, for example, been advocating for a long time to ensure the participation of patient representatives in the establishment of the European Reference Network (ERN) for rare diseases – these are networks for clinicians and researchers to share expertise, knowledge and resources across the EU.
Marina: What is Rare Disease Day and why it is important to have this awareness day?
Celine: Building awareness of rare diseases is so important because 1 in 20 people will be impacted by a rare disease at some point in their life. Rare Disease Day provides an opportunity to improve knowledge of rare diseases amongst the general public while encouraging researchers and decision makers to address the needs of those living with rare diseases.
Rare Disease Day was launched in 2008 on 29 February, a ‘rare’ date that happens only once every four years. Ever since then, Rare Disease Day has taken place on the last day of February to unite the rare disease community and raise awareness. It has always taken place in collaboration between EURORDIS and our Council of National Alliances. These are umbrella organisations who group together several rare disease organisations in a given country or region and they have been crucial to the success of the campaign over the last 13 years.
When we started Rare Disease Day in 2008, we had about 18 countries participating in the campaign. Thanks to our work in partnership with National Alliances, we now have over 100 countries and in 2020, we saw for the first time, countries like Gabon, Iraq and Rwanda joining the campaign as well.
Marina: The Rare Disease Day campaign message is ‘Rare is many. Rare is strong. Rare is Proud’;. Why did you choose this message and what do you hope to achieve with it?
Celine: There are over 300 million people around the world that are living with a rare disease, each supported by their family, friends and carers. Even though rare diseases might seemingly affect only a handful of people scattered across the world, collectively the number of people directly affected is equivalent to the population of the world’s third largest country! This is a significant number and by collaborating across borders and across the 6000+ rare diseases, we hope to make the rare disease community stronger as it works around a single cause, advocating for equity. People living with a rare disease have the right to equal opportunity to realise their potential.
Marina: Together, you and your member organizations represent the needs of more than 300 million people living with one or more of over 6,000 identified rare diseases. How do you plan to make a difference with Rare Disease Day?
Celine: Through this year’s message, ‘Rare is Many. Rare is Strong. Rare is Proud’, we want to shine a light on the global and diverse community of people living with a rare disease. To do this, this year’s campaign features stories of six individuals across different continents – all of them facing similar challenges. Through their stories, we want to show that while each rare disease is in fact rare, as a community, we share a lot in common.
Our long-term goal for the Rare Disease Day campaign is to achieve equitable access to diagnosis, treatment, health and social care and social opportunity for people affected by a rare disease. At EURORDIS, we are continuing to join forces for universal health coverage (UHC), part of the United Nation’s Sustainable Development Goals (SGDs) to advocate for equitable health systems that meet the needs of people affected by rare diseases in order to leave no one behind.
Marina: What activities have you planned to mark RDD?
Celine: A suite of high quality, high impact digital materials have been developed to effectively tell the story of Rare Disease patients. These materials are free to download and adaptable on https://www.rarediseaseday.org, allowing the global patient community to relay patient stories from around the world across diseases, borders, cultures and languages.
We also ran a series of activities leading up to Rare Disease Day. One such event I want to highlight is the Rare 2030 policy conference organised by us on 23 February, where we shared with the community our work on a Foresight study we started two years ago in collaboration with a wide group of stakeholders, including industry, policy makers, patients, healthcare providers and researchers. This will feed into policy recommendations for a new rare disease policy framework and lay the foundation of the kind of environment we want to create for people living with a rare disease from a policy perspective.
Marina: Why are initiatives, such as RDD important for the community?
Celine: Working in collaboration with as many stakeholders as possible is at the core of what we do – From individuals, patients, patient organisations, health professionals, researchers, drug developers, public health authorities.
We want to encourage industry leaders to help us amplify the patient voice, especially for diseases that may not be known and to raise awareness about unmet medical needs by highlighting diseases for which there is no treatment available and by sharing the stories of people living with serious, underserved conditions. Together, we can help strengthen voice of the rare disease community and help them lead better lives.
Galapagos is supporting voices of the rare diseases patient community on Rare Disease Day. Visit https://www.rarediseaseday.org/ to find out how you can show your support for the millions of people living with a rare disease.
INTERVIEW BY Marina Sardone
Patient Advocacy Lead at Galapagos
