As originally reported in StatNews, Mark Zuckerberg, founder of Facebook, along with his wife, Dr. Priscilla Chan, a former pediatrician and now head of the Chan Zuckerberg Initiative, made an ambitious goal in 2016: to “get all diseases under control by the end of this century.” This was an exciting goal to rare disease patients, but tackling all diseases? Diseases which already have doctors working round the clock to solve them without solutions, others that have no research or funding at all? It seemed far fetched.
The pair has begun this project by dedicating resources to groups advancing rare disease research in the ‘Rare As One’ program. The two founded the ‘Chan Zuckerberg Initiative’ or CZI in 2016 and dedicated $3 billion dollars of their personal wealth, accumulated by selling their Facebook shares. The CZI is multifaceted and aims to address justice, education, and opportunity; rare diseases are just one of their many projects.
They have announced their strategy: investing resources in patient-led groups to train them as part of their scientific initiative. After receiving 287 applications, the initiative decided to up the stakes; instead of funding the planned 10 groups, the project awarded $450,000 to 30 patient groups, administered over two years. Their method entails educating and empowering the patients to direct the mission themselves. Rather than trying to tick off solutions to thousands of diseases one by one, they hope the lessons of these patient groups will be contagious and in turn reach other rare disease patient groups. The selected rare disease groups can be models that influence the whole community and teach methods of action.
One of Patient Worthy’s Advocacy Partners, the Glut1 Deficiency Foundation, is one of the 30 chosen groups. Co-Founder and Executive Director Glenna Steele stated
“We are tremendously grateful for this opportunity, which we believe will bring help and hope to our patients in boundless ways. We look forward to learning, sharing, and growing alongside other rare disease organizations as each of us works to put the pieces in place to accelerate progress in our disease communities.”
Read their press release here.
The ‘Rare As One’ Grants
The Rare As One grant gives the recipients, mostly small patients groups run by patients themselves or parents of patients, leniency to appropriate the money as they see fit. The recipients are each required to host a relevant scientific convention in order to build community internationally and share ideas within the population. These patient communities are often underfunded. The initiative will help many of these beneficiaries fund paid staff for the first time and intends to organize a conversation as to how rare disease research is funded and how such funds are allocated. They have many methods to build a web between patients and researchers, including matching patient groups with scientific mentors, hosting monthly webinars, and promoting better technological infrastructure to aid their research. Having clean and manageable contact lists, databases, and tools are very important to organizing. They figure the more knowledgeable patients, doctors, and researchers put their perspectives and methods together, the more strategic the research will be.
Where Did the Idea Come From?
Priscilla Chan explained that her work as a pediatrician helped her come to understand the importance of listening to patients. She described to StatNews how learning this was a process. She turned her shame of not having enough personal expertise to help the peculiarities of each rare disease patient into a tangible way she could help. With over 7,000 rare diseases, becoming an expert on each case that came in wasn’t a possibility. Instead, she realized she needed a method to enable patients and their families to become the experts on their own health.
Chan said,
“Eventually, that was the smart way to work: to actually refer back, learn from the patients about how to best take care of them, and what worked and what didn’t work, because there was no central body of knowledge.”
So she quit her work as a full time doctor to head the CZI. The project’s mission isn’t easy, even with the funding the couple has accessible. Money isn’t the answer to all research problems, but it helps. $450,000 isn’t a ton when it comes to research and drug creation, but for these patient groups, that is no small number. The idea is that a small push can set off a chain reaction if it’s aimed well.
What are your thoughts about the Rare As One? Share your stories, thoughts, and hopes with the Patient Worthy community!
