Blog 6: Public Policy for Rare Diseases

Hello everyone! For those of you just joining in, my name is Jonna Crocker. I am a high school senior who is making a series of blogs for my philosophy class, discussing the injustices for rare disease patients and what we can do to help. This is my sixth and last blog in this series, so make sure to read my other blog posts as well to learn more!!

Today, I will be discussing the public policies that are in place for rare diseases, and ways in which there could be a better system for the country as a whole. There is a great foundation out there, but places that are not following along in their footsteps will have a hard time advancing changes on a local and broader level. 

Rare diseases are a global public health issue, as they affect a large number of people throughout the country and the entire world. There are many organizations that touch on the policy issues for rare diseases, and highlight the current issues so that they can be addressed and changed. The largest organization that does this is NORD, the National Organization for Rare Disorders. As part of their mission, they are working to advocate for public policy issues including the lack of funding and research, providing better access to coverage, medication and diagnostics, COVID-19 impacts for rare disease patients, and advisory councils. 

One of the main things that I would like to focus on today is the idea of a Rare Disease Advisory Council, also known as an RDAC. Straight from NORD’s website, “RDACS act as an advisory body that gives the rare disease community a stronger voice in state government and makes recommendations as to how their state’s programs and policies should be modified to benefit the rare disease community”. The first-ever Rare Disease Advisory Council was created in North Carolina in 2015, and now there are 21 states that have established councils since. Members from all different aspects of the rare disease world serve as members on the council, including patients, health care providers, health insurers, researchers, and state government officials including your local senators. 

These councils are incredible and are bringing awareness and advocating for the rights that each patient should undoubtedly have. The problem is that this is not as widespread as it needs to be. Having 21 states be a part of this movement is amazing, but that means more than half of the country does not have this system in place. And for those that do, it has not been properly and fully implemented because it is relatively new. The laws passed through the council can be life-changing, and by asking for direct feedback from those who see rare diseases every day, the core of the problem is exposed and differences are made. 

In my previous posts, I touched on the lack of research due to limited income and funding, the access to coverage and insurance problems, and the lack of medications available. The other thing that has been trending in the public advocacy for rare disease patients is the effect of COVID-19. MANY rare disease patients are immunocompromised, and if they were to get sick with COVID-19 it would be detrimental and extremely damaging to their immune systems. In addition to the fears of COVID-19 as it is, for these patients it could cause major health setbacks and potentially life-threatening illnesses. Patients have been attempting to advocate for safer waiting spaces in hospitals, telemedicine appointments, and even improving medication access as many pharmacies have been backed up for months. 

These are just some of the things that impact rare disease patients on a daily basis that can be fixed through laws, regulations, and even just better business practices. The hope with the rare disease advisory council is that each person can take both positive and negative feedback from others, and revamp their part of the system to make changes for the better. The important thing is to continue to advocate and fight for the proper rights that all rare disease patients deserve. Learn more about if your state has an RDAC. See what your senators and legislators are doing. Do not be afraid to advocate. Let us help be the voice for those who can’t, and fight for a better world for everyone.