This is part of a series recognizing the 25th anniversary of Sanofi Genzyme’s Humanitarian program. The primary goal of our Humanitarian program is to deliver enzyme replacement therapy, to the best of our ability, to patients with lysosomal storage disorders (LSDs) who have a demonstrated need in certain circumstances where treatment access is limited. This series highlights our humanitarian efforts to support the LSD community, and represents just one part of Sanofi and Sanofi Genzyme’s charitable work.
In its 25-year history, Sanofi Genzyme’s Humanitarian program has provided free drug to more than 1,700 patients in 70 countries. There are currently more than 650 patients in 55 countries, and on six continents, receiving free enzyme replacement therapies (ERT). Our Humanitarian program supports patients with five lysosomal storage disorders (LSDs): Gaucher disease, Fabry disease, MPS I, MPS II (available outside the U.S. in Sanofi Genzyme’s responsible territory), and Pompe disease.
The Humanitarian program delivers many important benefits in addition to access to our drugs. For healthcare providers, it provides expanded opportunities in clinical education, treatment experience and patient monitoring. Our Humanitarian program often serves as a physician’s first experience in treating an LSD. We also work with local officials to help establish sustainable healthcare systems to care for the needs of rare disease patients. Over the years, several patients in the Humanitarian program have become patient advocates themselves, starting patient associations, advocating for policy change and raising disease awareness in their countries. Roman, a Fabry patient from Poland, and Vesna, a Gaucher patient from Macedonia, are two such advocates.
Roman, Fabry disease, Poland
Roman founded the Polish Association for Families Affected by Fabry in 2002 after meeting other Fabry patients in his country when he went to receive treatment for the first time as an adult—though he’d had symptoms of the disease since childhood. The association won a Patient Advocacy Leadership (PAL) Award in 2012 for its “Where is Fabry?” campaign, which was produced in conjunction with the National Forum for the Treatment of Rare Diseases and is featured in this video. The project sought to improve public awareness and understanding of Fabry disease by using Facebook and other social media, as well as involvement by Polish celebrities, to encourage Warsaw residents to "find" Fabry in their city. The project culminated in a large event in a public square in Warsaw where they used props, like a giant blow up ball, to get the attention of the general public and share information about Fabry disease.
“Today, the Association brings together 61 members. We meet together annually, always in different regions of Poland,” said Roman. “Maybe it’s not too often, but our families are scattered all over the country. And this is why those meetings are so important to us. It is an occasion to exchange our experiences.”
Though the association has not yet reached its goal of achieving reimbursement in Poland, Roman is hopeful that through their continued efforts, the members of the association can make a difference.
Vesna, Gaucher disease, Macedonia
Vesna was diagnosed with Gaucher disease in 2001, but didn’t receive her first treatment until 2010. In 2009, she founded Life with Challenges, a rare disease patient association in Macedonia. The association won a 2012 PAL Award for its efforts to support a public education campaign in the Macedonian language, and to make information about rare diseases accessible to local physicians, government officials and the media.
Previously, no information about rare diseases existed in the local language. The campaign included translations of educational materials and several disease awareness events. The group capitalized on public events such as Rare Disease Day and local road races to seek media attention, and in partnership with other patient groups such as the European Gaucher Alliance (EGA), held ongoing meetings with the Ministry of Health, the Health Insurance Fund, the Ministry of Labor and Social Affairs, and the Commission for Rare Diseases, among others, eventually leading to reimbursement at the end of 2015.
“The only wish my parents had was for me to be healthy,” said Vesna. “They never dreamed that I would have a normal life, an education and a career. And that one day I would be having a family on my own.”