Patient organizations play a vital role in providing leadership, resources and access to care and support for patients and their families around the world. We know that supporting the work of patient organizations is critical to improving patient care and meeting the ever-growing needs of the rare disease community.
That’s why eight years ago, we launched the Patient Advocacy Leadership (PAL) Awards, a program that supports non-profit organizations that assist patients living with lysosomal storage disorders (LSDs), a group of rare, inherited disorders that can cause progressive and debilitating health problems.
Since the program was launched, we have awarded close to $810,000.00 to support 52 unique projects in organizations located in 28 countries across the world. We are currently reviewing applications that were submitted for our 2018 award cycle. We thank all those who applied for your ongoing work to support patients. We intend to announce our newest recipients this spring. In the meantime, you can read more about our 2017 winners below.
2016 PAL Award winners, The Associação Catarinense de Pacientes e Amigos de Gaucher and the Associação Gaúcha de Fabry, are working to provide informational materials and trainings at community health centers in Brazil. The trainings help healthcare professionals identify rare disease patients early in their visit to a health center, so they can offer personalized care that addresses the particular needs of their disease and reduces the risk of unnecessary procedures.
2017 PAL Award Recipients
In 2017, 36 patient organizations in 24 countries submitted proposals for collaborative and creative activities to support the LSD patient community across a variety of rare disease groups. An Independent External Review Committee reviewed all proposals and selected six PAL Award winners. Award recipients came from Ukraine, the United Kingdom, the United States, Romania, France, and New Zealand.
Each of the PAL Award winners created a unique approach to meeting the needs of the LSD community, clearly demonstrating that organizations are eager to support patients, raise awareness about LSDs in the public, and study trends in rare diseases.
“The PAL Awards put our mission into action,” said Sebastien Martel, Global Head of the Rare Diseases franchise. “The organizations that benefit are committed to empowering patients and addressing the unmet needs of patient communities.”
"Rare Diseases of Ukraine" - Ukraine
With their grant, “Rare Diseases of Ukraine” hopes to address unmet needs for adult rare patients. Since many health care providers in the country do not offer care for rare disease patients past the age of 18, their project will focus on advocacy trainings and creating regional groups of experts — lawyers, doctors, nutritionists — to support rare disease patients over the age of 18. As the project progresses, “Rare Diseases of Ukraine” plans to publish information about the rights of adult rare disease patients as well as host an awareness photo exhibit.
Romanian National Alliance for Rare Diseases - Romania
The Romanian National Alliance for Rare Diseases project will be an awareness campaign focused on the specific needs of people living with rare diseases. The group has proposed activities including helping Pompe patients establish a patient organization, launching a Pompe awareness campaign, organizing a marathon, and sharing patient stories through radio programming and literature. The group plans to share the project outcomes through an existing training course for journalists aimed at improving knowledge of rare disease patients.
Hermansky-Pudlak Syndrome Network - United States
The Hermansky-Pudlak Syndrome Network proposed an effort to educate patients and families on how to participate in scientific research. Through a steering committee they plan to develop an educational curriculum about informed consent, placebo trials, biospecimen collection, and donation of organs and tissues for research. The project will generate personalized plans for rare patients about how they can participate in scientific research.
Mucopolysaccharide & Related Diseases Society Aust. Ltd. - United Kingdom
Using information collected in the Red Book (the UK’s national standard record of a child's health and development) the Mucopolysaccharide & Related Diseases Society Aust. Ltd. will conduct a survey across all patients age 25 and under with Mucopolysaccharide and related disorders, to identify common patterns and trends. The organization hopes the data will reveal early indicators of the disease or commonalities among patients. The trends would then be shared with doctors, clinicians, and families, hopefully leading to earlier diagnosis and better outcomes.
Association de Patient de la Maladie de Fabry – France
The funding from this PAL Award will facilitate a 2-day event for 10 women with Fabry. The event will include a physician-led educational workshop, individual patient interviews with psychologists, and a writing workshop. Post-event, a booklet for patients and HCPs will be published, including information about how women experience Fabry, articles written by the participating psychologists, and patient testimonials. The booklet will be shared in-person and online with psychologists, patients, and physicians.
New Zealand Pompe Network - New Zealand
The New Zealand Pompe Network will use their PAL Award to fund the first-ever country-wide, multi-organization Pompe conference. Experts from countries with successful care and treatment for Pompe patients will be invited to share information, scientific knowledge, and best practices with New Zealand physicians and patients. Pompe patients will be surveyed in advance of the conference to determine what topics are of most interest to the community.
“Over the last eight years, we have been continually amazed by the innovative ideas proposed by patient organizations,” continued Martel “Their work has a real, global impact on the rare disease community, allowing small patient populations better access to the information and resources necessary for proper care.”