Common interests unite rare disease advocates in a shared mission

Long before learning I had idiopathic pulmonary fibrosis (IPF), I supported the advocacy efforts of the National Ataxia Foundation (NAF). Like pulmonary fibrosis, some forms of ataxia are rare diseases. One of them, spinocerebellar ataxia type 3, also known as Machado-Joseph disease, took the lives of three of my four stepsiblings. My fellow columnists Sean Baumstark, Kendall Harvey, and Matthew Lafleur all have Friedreich’s ataxia and write for Friedreich’s Ataxia News.

When I was diagnosed with IPF in January 2017, I faced an intense learning curve. That year also presented me with the opportunity to become an engaged advocate for my rare disease. My experience with advocacy for the NAF provided the tools I needed.

Joining forces

Some rare diseases may present with similar symptoms, but have entirely different outcomes. In these cases, there may be some benefit to joint advocacy for a solution in their common interest. This Venn diagram illustrates how organizations A, B, and C each have their own rare disease concerns. The sweet spot is where advocacy issues overlap for the three organizations.

A traditional Venn diagram illustrates areas of overlapping concern. (Illustration by Sam Kirton)

Other rare disease organizations, including the National Organization for Rare Disorders (NORD), work with a variety of rare disease-specific organizations, including the Pulmonary Fibrosis Foundation (PFF) and NAF, on issues that affect more than one specific disease.

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A recent example of this is the 126 organizations, including NORD, that sought an extension of Title V programs, including the Orphan Products Grants Program, which provides “grants to clinical investigators to support the development of safe and effective medical products for patients with rare diseases.” Congress has yet to act on this request.

The power of numbers

Each of us should understand two things about advocacy work: It’s hard work, and it’s not always successful. I’m not pointing this out to discourage you, but to prepare you — because disappointment will be a part of the advocacy experience.

When multiple advocacy organizations join together on an issue, it increases the number of voices raising a specific concern. When you participate in these advocacy meetings with members of Congress, each of those voices for various diseases represents a constituent. When their shared concern is raised with multiple members of Congress, it’s more likely they’ll be heard.

A timely example

Last week in my column, “Why I do rare disease awareness and advocacy work on Capitol Hill,” I wrote that I would be part of PFF Hill Day 2023. During the day, my team met virtually with six congressional offices to advocate for issues important to the pulmonary fibrosis community.

One of the issues we spoke about was the need to ensure patients requiring supplemental oxygen receive an appropriate level of support. This PFF effort established four principles that will provide the foundation for legislative reform.

The four principles are as follows:

1. The need for oxygen is a patient-centric necessity that should be protected by creating a patient bill of rights to ensure that care will place patient needs first. Additionally, it calls for replacing the term “home oxygen” with “supplemental oxygen” to help people who require it live full lives outside their primary residence.
2. Ensure access to liquid oxygen for patients who need it.
3. Help provide adequate pay for respiratory therapists, to ensure patients who need such assistance will have access to their expertise.
4. Ensure proper reimbursement and protect against fraud and abuse by establishing national standardized documentation requirements that will rely on a template rather than prescriber medical records in supporting the claims of supplemental oxygen suppliers.

This is not a solo effort by the PFF. The foundation has joined forces with other rare disease organizations whose patients require supplemental oxygen as part of their standard care.

Why now?

Today, Feb. 28, is Rare Disease Day, which is recognized around the world by rare disease communities to raise awareness. On this day, my voice is one among many helping educate others.

The many voices of the rare disease community offer the lived experience of a rare disease. Raising awareness as one of them is how I can make every breath count.

Note: Pulmonary Fibrosis News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Pulmonary Fibrosis News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to pulmonary fibrosis.