Now, more than ever, we must unite for our future

Recent news headlines have sparked significant concern among those of us in the pulmonary hypertension (PH) and other rare disease communities. The massive budget and staff cuts across multiple federal agencies are particularly troubling, prompting a flurry of conversations both online and in private. As we navigate these challenging times together, we must remain vigilant and be proactive.

While I typically don’t pay much attention to the news, my advocacy work and having to manage PH every day make me both angry and concerned about the recent cuts, as well as others that are likely to come. And I’m not alone.

You’ve probably noticed an increasing amount of disappointment and stress being expressed on social media lately. One thing that prompted the ire of many was the postponement of FDA-NIH Rare Disease Day events, a decision made by officials at the U.S. Food and Drug Administration (FDA) and the National Institutes of Health.

“After much consideration, we are postponing FDA-NIH Rare Disease Day 2025 and will reschedule it to occur in the coming months,” a message on the FDA’s website states. “Rare Disease Day is important to all of us, and we want to make sure we can fully focus on the event to make it the best that it can be.”

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Some may wonder why we are so upset. One reason is that we patients never anticipated the additional stress of potentially losing our medical insurance, co-pay assistance to help cover the high costs of PH treatment, and other vital benefits. These unexpected difficulties would add additional layers to our already overwhelming challenges.

In light of this turmoil, I tracked social media posts in the chronic illness community and spoke with other members of the PH community to get their reactions.

‘We can’t afford to go backward’

Many parents of children with PH and other rare diseases shared on social media that they feared their children’s medical benefits were under threat. Medicaid and the Children’s Health Insurance Program, or CHIP, provide essential coverage for many children with rare diseases, ensuring that they receive affordable healthcare. These programs are vital for timely diagnoses and access to treatment.

Several older members of our PH community rely on Medicare. A close “PHriend” of mine, Carol Volkman, remarked, “Many of us, and many others, receive grants from organizations like The Assistance Fund to help with co-pays for our medications. The U.S. government partially funds these grants. I have not heard about losing my grant, but it occurs to me that they all may be at risk.”

Richard Channick, MD, a passionate PH specialist, said, “We can’t afford to go backward after we’ve worked so hard, collaborating closely with researchers and scientists to develop new therapies for PH. We are finally beginning to find new pathways to treating PH.”

Like Channick, I and many others had remained hopeful about new PH treatments, such as Winrevair (sotatercept-csrk), an injection therapy for adults with pulmonary arterial hypertension that was approved last year by the FDA.

I reminded those who share my concerns that our commitment to advocacy and healthcare storytelling remains unwavering. We have no choice. This situation will affect our future and the future of our loved ones, so we will continue to fight for the attention of policy makers.

The rare disease community is resilient and courageous. We’ll continue to advocate for ourselves and the 30 million others who live with rare diseases in the United States. Together, we can amplify our voices and ensure that the details of our struggles and victories are heard.

Our journey involves raising awareness and creating meaningful change that can impact the lives of millions of others who are affected by rare conditions. We stand united, and our collective efforts will not go unnoticed. We are dedicated to making a difference, one story at a time.

If you or a loved one has PH or another rare disease, please share your stories with your governmental representatives. Personal experiences resonate deeply and can have a genuine impact. Your voice matters and has the power to prompt change.

Note: Pulmonary Hypertension 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 Hypertension News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to pulmonary hypertension.