Rare Disease Advocate Recommends Patients Advocate for Their Disease — And Here’s How

Health writer
Rep. Gus Bilirakis, R-FL, and Candace Lerman in his office on Capitol Hill. Bilirakis is the sponsor of the OPEN Act. They met in Oct. 2017 to discuss to discuss their strategy for gaining more cosponsors for the act in both the House and Senate.
Candace H. Lerman

Candace H. Lerman is the 1 in 10 Americans with a rare disease. If you have multiple sclerosis, multiple myeloma, or a host of other diseases and conditions, you are that 1 in 10, too. With often limited funding for research into treating these diseases (which are considered “rare” in the U.S. if the disease affects fewer than 200,000 people) and close to 7,000 rare diseases with no treatment at all, Candace knows well the need for more patient awareness and advocacy. In addition to her rare disease diagnosis, Candace also has diagnoses of fibromyalgia, Sjögren’s syndrome, and a mild form of celiac disease.

That’s why she’s dedicated her life to helping others through advocating on Capitol Hill, running the popular advocacy blog Rare Candace, and now, as a lawyer in the state of Florida as of April 16, 2018, bringing awareness to health legislature that impacts all patients. Here’s how it happened.

Gus and Candace with other Florida rare disease advocates during Rare Disease Week in February, 2015.
Candace in Bilirakis’ Capitol Hill office with fellow Florida rare disease advocates during Rare Disease Week in February, 2015.
Candace H. Lerman

HealthCentral (HC): Can you tell us a little about your story – and how you got involved in patient advocacy?

Candace: I became a patient advocate after being diagnosed with a rare disease and discovering there was no Food and Drug Administration (FDA)-approved treatment to help me. In May 2014, I was diagnosed with a rare autoimmune blood disorder called immune thrombocytopenia or ITP, which caused my body to destroy platelets, and can lead to fatal internal bleeding. After months of testing, my doctors determined my immune system was dysfunctional, but because I had no FDA-approved treatment, I needed to look elsewhere. I took to social media and gathered data from my fellow patients, eventually determining I fell into a category of those with ITP who responded to immunosuppression. I did a four-week treatment of the immune-suppressing drug Rituxan (rituximab) and put myself into remission in December 2014.

Since remission, I became an outspoken advocate for rare disease patients, working on the 21st Century Cures Act, meeting with members of Congress, speaking at events around the country, and obtaining my law degree in 2.5 years and my health care compliance certification from Seton Hall Law School. I was recently sworn in as a member of the Florida Bar, something that would not have been possible without repurposing Rituxan.

I have been working with members of the U.S. House of Representatives and Senate to pass a bill called the OPEN Act, which would incentivize pharmaceutical companies to run clinical trials for rare diseases using products already on the market.

HC: Why should patients attend an advocacy day or even advocate for themselves alone on Capitol Hill?

Candace: Educating lawmakers and their staff is critical to making the world a better place for patients. If they don't know what problems you're facing, there is no way they can write legislation to change the current landscape. I recommend people attend an advocacy day to obtain training and have support of a group setting. Once you get the hang of advocating, then you will feel confident going at it alone. It is a very emotional experience, especially telling your personal story to a complete stranger. Practice makes perfect, and I have seen many advocates build solid relationships with their elected representatives.

HC: How do you recommend patients prepare themselves for advocating on Capitol Hill?

Candace: Read up on current legislation and familiarize yourself with the committees your elected officials are involved with. Practice telling your story in the mirror, bring notes if necessary. It is really helpful to reach out to other advocates who are active on Capitol Hill, they can give you some tips and tricks to maximize your experience. I am always reminding patient advocates that they should not be disappointed if they meet with a staff member in their elected representative's office. I have made some amazing friends over the years, and they have helped me accomplish some advocacy goals. The staff keep their bosses up-to-date on what constituents are concerned about. Don't be afraid to ask for their card so you can e-mail them for a follow-up.

Members of the House and Senate are extremely busy and can have tough schedules balancing dozens of issues, their staff are a valuable tool for patient advocates to help push for change.

HC: What was one thing that surprised you about advocating for your health?

Candace: I could not believe how far my advocacy has taken me. It has been a rewarding experience working on the 21st Century Cures Act, inspiring more legislation, and being able to help other people find their voice. I have met some really amazing people who are committed to changing the world for rare disease patients.

HC: How have policy changes, including the 21st Century Cures Act ($6.3 billion in funding, mainly to the National Institutes of Health, enacted by Congress in late 2016), impacted patient advocacy, and what should we know about these changes as patients?

Candace: The 21st Century Cures Act opened the door for patients to be more involved in the drug approval process, clinical trial design, and further discussions on legislative changes to health care policy. Members of Congress, administrative agencies, research institutions, and health care providers are all interested in including the patient perspective moving forward. There is no better time to become an advocate than the present. I encourage patients to stay informed about proposed rules at the FDA, keep up to date on the latest research for their disease, and create a network to share information with fellow advocates.

The 21st Century Cures Act was just the beginning of a movement for reforming an outdated system for medical research, clinical trials, and drug approvals. Patient advocates have a lot of work left to do, so I enthusiastically encourage people to find their voice and share their story.

See more helpful articles:

How to Be Your Own Best Advocate

What is a Health Advocate?

Boston Marathon Bomb Survivor Doesn't Run From Chronic Pain