Putting people with Fabry first: a talent that Amicus Therapeutics and FIN share. This commonality has ensured a strong partnership, spanning more than 16 years. Amicus Therapeutics is a pharma industry leader advancing the professional field of patient advocacy; it develops and delivers medicine for people living with rare diseases. Jayne C. Gershkowitz, Chief Patient Advocate, explains what this partnership means for the Fabry community today.
“We empower people living with a rare disease to become their own best advocates,” says Jayne
Understanding the disease
“At the beginning of this collaboration, we all sat around the table in Amsterdam at the first annual FIN Expert Meeting. It was an informal conversation, but a good opportunity to better understand Fabry disease. I don’t live with Fabry nor do my colleagues. The biggest challenge for me was to gain insight into what it’s like to live with this disease,” says Jayne; “Fabry International Network is our gateway to the community.” Through FIN, Amicus Therapeutics learns more about the challenges, needs and hopes of patients and their families.
Fabry Findings, a FIN initiative is a newsletter for the community with the latest research and tips including current research and scientific and clinical trends, and ideas for disease management written in lay language. Local organisations can translate these newsletters into their own languages. Amicus and FIN originally discussed the concept for Fabry Findings to make these scientific articles available and intelligible to the average person living with Fabry.
“This letter was a unified call to action from the community to the treatment centres around the world. A collective effort that directly could help people with Fabry and their families”
Dealing with emotional health, wellbeing and isolation
People affected by Fabry need information to better understand their disease. Information about the inheritance of Fabry, the physical and manifestations of the disease and the psychological effects. And COVID exacerbated this need. The Fabry community faced a lot of difficulties, including isolation and emotional health. During the pandemic, a different approach was needed to ensure the wellbeing of people with Fabry and people with other rare diseases was being addressed.
Telehealth visits offered a good solution, but people needed help to understand more about these alternative medical appointments and how they function. During the roundtable meeting, an agreement was established between the different Fabry organisations to address the need for continuous access to care for people living with Fabry in their countries. This resulted in a letter, signed by those organisations who participated in the roundtable, and sent to Fabry expert centres around the world. “This letter encompassed everybody’s concerns because it stated the Fabry community’s need for care. The group wanted to make sure that medical professionals were aware that there was a community waiting to be seen and heard and to be helped with their disease management,” says Jayne.
The roundtable meeting in late 2020 emerged from this need to understand isolation. The first roundtable series is an initiative developed jointly by FIN leadership and Amicus Patient & Professional Advocacy (P&PA) to bring Fabry leaders from different countries together. “We brainstormed about the needs of people with Fabry and about solutions that could be shared with local leaders. It helps to create that environment where people can share, and take good ideas and then model that behaviour,” recognises Jayne.
“We wanted organisations to leave the roundtable series feeling empowered with knowledge and tools to support the Fabry communities in their countries”
Today, the annual FIN Expert Meetings are now a larger scale conference today with an impressive schedule of speakers from both the patient and medical communities. “We’re even able to have simultaneous translations for people coming from different countries where English may not be as easily understood,” says Jayne. During the pandemic, meetings were virtual, leading to a successful shift: the geographical growth of FIN’s membership. The virtual meetings provided greater outreach and increased participation, but FIN and Amicus are also keen to return to face-to-face meetings. As a result, small regional meetings may emerge to ensure that all members have a chance to meet and to avoid long travel and expenses. FIN and all its partners are looking forward to being together at the April 2023 expert meeting in Amsterdam.
The Amicus P&PA team strives on making a difference. First, by advancing the professional field of patient advocacy within industry, which should help engagement with all patient communities, like FIN. Secondly, the biotech company champions patient-first thinking and ensures that the patient perspective is included in all aspects of their work. Finally, it is paramount that Amicus prioritises education and cooperation with a focus on mutual interests that help people with Fabry be more resilient. “We hope to make a difference in those three areas, and we believe collaborating with FIN will advance our work. But it is the individuals themselves who will determine how much difference we make. We will continue to work towards that,“ concludes Jayne.