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Kimberly Pang: Our lives depend on leadership of Hassan, Kuster



For the Monitor
Saturday, November 10, 2018

Recently, I had the privilege of attending and leading a workshop alongside the National Organization for Rare Disorders’ Rare Action Network (RAN) as the state ambassador for New Hampshire.

At the workshop, I was able to meet with other members of the rare disease community, present on advocacy efforts in our state, share what drives me to be an advocate for rare diseases, and, perhaps most importantly, hear from the staff of Sen. Maggie Hassan and Rep. Annie Kuster. Both expressed the continued importance of working together as a community to ensure all patients and families in the Granite State have access to appropriate and affordable care.

I was very appreciative that the staff members were able to attend and relay Congress’s priorities with members of the New Hampshire Rare Action Network. The rare disease community – patients, caregivers and medical professionals – counts on people like Hassan and Kuster to champion its cause. In fact, in many ways, rare disease patients’ lives depend on people like Hassan and Kuster.

Thirty-five years ago, members of Congress in the very same position drastically impacted our lives by enacting the Orphan Drug Act. The ODA is critically important to those affected by rare diseases and their families. The ODA gives patients and caregivers like us great hope for new treatments for our diseases. Before the ODA, only 34 therapies for rare diseases had made it through the drug development pipeline to be approved by the Food and Drug Administration. Today, there are over 700 orphan drug approvals.

In other words, before the ODA, children with cystic fibrosis, for example, had no hope for a cure; whereas, now, those children can realistically look forward to a better tomorrow. All of this is great news for the estimated 30 million Americans living with a rare disease, including myself, but much more work is needed to develop treatments for all of the approximately 7,000 known rare diseases. Currently, about 95 percent are without a single FDA-approved therapy indicated for their rare disease, including mine. While I could not be happier about the success of the ODA so far, our community still depends on Congress to ensure that it remains strong.

Sen. Hassan said it best: “In order to make a difference for patients with rare diseases and their families, we must continue to invest in critical biomedical research, including basic science research that helps bring treatments and cures to patients.”

I could not agree more and look forward to working together to find more treatments for the entire rare disease community.

Like the National Organization for Rare Disorders says: “Alone we are rare. Together we are strong.” When you live with a rare disease, it is easy to feel isolated, but getting to meet with so many others living with a rare disease and share my story was uplifting.

As the New Hampshire state ambassador for the Rare Action Network, I strive to bring together individuals in the rare disease community from around the state. I will follow in the footsteps of those who succeeded in advocating for the ODA and continue to advocate for policies that help our community so that each new generation can live in a world with even more hope.

As Rep. Kuster said, “Community-based awareness and education efforts about those affected by rare disorders will enable earlier recognition and better care.”

I will not stop advocating for the ODA until all 7,000 known rare diseases have a treatment, and I hope the whole of New Hampshire, including Sen. Hassan and Rep. Kuster, will join me.

(Kimberly Pang of Concord is the state ambassador for the National Organization for Rare Disorders’ Rare Action Network.)