Around 25 to 30 million people in the United States are affected by one of 7,000 rare diseases. Many of them are children, and often, access to adequate health care is lacking. This will change if Jacob Fraker’s AB 2283 is voted into law.
Jacob works as the legislative aid for Assemblymember Susan Eggman at the California State Capitol. Additionally, the passionate young man lives with cystic fibrosis, a rare, genetic, and progressive condition, which affects the lungs, leading to continuous infections and breathing difficulties, as well as other serious complications. Based on his experiences as a rare disease patient and using feedback from many other advocates, Jacob drafted bill AB 2283, which seeks to create a rare disease advisory council and hire an ombudsperson to support rare disease patients in the entire state of California.
A rare disease council made up of people from the community.
“The bill would create a rare disease advisory council and an ombudsperson within the Department of Health and Human Services in California,” Jacob explains. This council would be a large board made up mainly of rare disease community members of all ages – pediatric, youth, and adults. “All three of those perspectives are very important. The challenges that come with being a child with a rare disease are different from when you are transitioning to an adult unit. We know that a lot of rare disease patients do their best to stay with their pediatric doctor for as long as possible, and we want to make sure that the transitions to other units are being talked about and being addressed. We also want to represent the adult perspective and the different, complex issues within health, education, and society.”
In addition to the patient representatives, two patient advocacy organizations will sit on the council, as well as a proxy of the State Department and one from the drug review board, a rare disease doctor, a researcher, and a rare disease nurse. “Especially when we are in the hospital, we tend to get very close to our nurses. Rare disease nurses have great insight into the more intricate day-to-day issues that rare disease patients face,” Jacob says. Moreover, a representative of a rare disease pharmaceutical company would be part of the board as well to ensure access to rare disease medication. “The relationship between pharma and the patient can be very adversarial, but we want to make sure that everyone who is part of the problem is also part of the solution. We need to talk about what they can do to make things easier for patients,” Jacob emphasizes.
Your personal patient advocate: the ombudsperson.
The ombudsperson would receive a seat at the table, too. This individual is planned to be the go-to-person for any rare disease issue that could arise. And every rare disease patient knows there are many: with schools, doctors, insurances, state programs, and more. Rare disease patients invest a lot of energy in their health management. “Many times, especially during elementary school and high school, I would be hospitalized for weeks, and I had to work with my teachers to get all my homework assignments, so I didn’t fall behind. Some teachers were great about that; others were not. Sometimes, we had to fight the education administration. In the rare disease community, you can be healthy on Monday and in hospital on Tuesday, right? So we need people to work with us and to be cooperative, but that doesn’t always happen. In this scenario, I would have been able to call the ombudsperson and explain my situation, and this person would be able to reach out to the department of education and my school district and work as an official liaison to ensure that my school is doing their part in supporting me because of my rare disease,” Jacob speaks out about his experience.
Overall, 16 people are planned to be part of the rare disease council, which will meet at least four times each year. No more than two representatives of the same rare disease will be allowed on the board to make sure that no monopoly or uneven power dynamic develops. “We want to ensure that regardless of how large the patient population is, you have a voice at the table,” Jacob explains.
Other states have already implemented rare disease councils.
Even though California is known as the leading state in many areas, it falls behind when it comes to rare disease councils. Other states, including Alabama, already have an advisory board with varying degrees of efficacy. According to Jacob, AB 2283 has been developed considering the knowledge of people on those councils. They have contacted the responsible people in other states to learn about what worked and what did not work. The California bill is crafted to avoid the pitfalls previously reported in other states. “We want to make sure California is the most successful it can be, and I think the legislation we have crafted does that,” Jacob says.
Education and awareness for rare diseases is the goal.
According to Jacob, when rare disease patients approach the legislation for an issue, they first have to educate the person they are speaking to about their condition before they can get to the stage of addressing the actual problem. “This can be draining and exhausting for advocates. We really want to start solving problems, but we have to go through this whole phase of ‘this is why I deserve humanity; this is why I deserve respect, and this is why I deserve attention.’” They hope the rare disease council might be able to normalize conversations about rare diseases at the capitol. Additionally, Jacob would want to create a detailed health insurance plan for rare disease patients, especially if the United States will follow the example of countries like Canada or Ireland in implementing a single-payer health system. “Single-payer health care systems are incredibly impactful and helpful, but for us rare disease patients, they often limit access to the high-cost medication that we need to survive. Single-payer health care systems aren’t designed with rare disease patients in mind. We want to make sure that this council is in place long before we get to a single-payer health system to have that intentional planning throughout,” Jacob says. But until this all can happen, the bill needs to pass many legal steps.
A long way to go before the bill is signed into law.
The regular process for a bill from the initial idea to becoming a law is complicated and works in a cycle. After the first idea, the language needs to be crafted. Once it becomes an official bill – and in this case, because it is related to health – it gets moved to the assembly health committee before moving on to appropriations. Appropriations is the place that distributes the funds for certain bills. If the bill passes this stage, it goes over to the senate, where it has to be approved by a health committee and appropriation again. After this procedure, the full body will vote on it and send it to the governor to be signed into law.
AB2283 is at the stage of being heard by the health committee. “We are hoping for as much support as we can get from patient advocacy organizations, to rare disease individuals, to the pharma industry. Everyone, who has a stake in the rare disease community, we want them to come out to support the bill,” Jacob says. He is confident the bill will make it through the health committee, but worries about appropriation. “It should have no problems in the health committee because we have an incredible argument here and an incredible need, but when it gets to appropriation committees, that’s where sometimes things can get stuck,” Jacob says the appropriation committee is known as the graveyard of the legislator. So that’s a step where community support is even more critical.
This is what you can do to help!
If you want to support Jacob’s work, you can send a letter to your legislator or specifically to the chair of the health committee. In your writing, you should state that you support bill AB 2283 and why. “It can be your own personal narrative or the story of your friend or family member you want to support,” Jacob says. Additionally, you can call your legislator and ask them to support or co-author the bill. Lastly, you should share the bill with support groups and patient organizations to spread the word, so that this bill becomes a law and the lives of all rare disease patients hopefully improve.
Find out more about AB 2283:
Or contact Jacob directly:
Jacob would love to hear your response regarding how the COVID-19 pandemic affected people living with rare diseases.
How would a central source of information and resources for the rare disease community impact your navigation of the crisis – specifically right now during the Coronavirus pandemic?
By Karina Ulrike Sturm