In August, 2023 Pheo Para Alliance learned of the planned discontinuation of AZEDRA, the only therapy ever to receive FDA approval specifically for the treatment of pheochromocytoma and paraganglioma. In response to this devastating news, we formed an Advocacy Task Force comprised of patients, caregivers, allies, researchers and clinicians to create a strategy to support affected patients. As the Task Force nears its one-year anniversary, we’d like to share some updates on advocacy efforts in which Pheo Para Alliance and our volunteers have engaged over the past 12 months.
In May, we received word that the Retaining Access and Restoring Exclusivity (RARE) Act had made it through subcommittee with markup and was advancing to be taken up by the full Energy and Commerce Committee in the House of Representatives. This is good news for this piece of legislation, which featured heavily in the one-pager we created and passed out during 2024 Rare Disease Week on Capitol Hill.
In July, the Centers for Medicare & Medicaid Services (CMS) announced that it was proposing a rules change to unbundle radiopharmaceuticals from other hospital supplies. , effectively accomplishing what the FIND Act intended. This announcement was lauded by patient groups and the Society of Nuclear Medicine and Molecular Imaging alike! You may remember that several in our community went to Washington in April to talk to legislators about this.
To end the year, we received another piece of gratifying news: the Senate Appropriations Committee released the Defense Appropriations Report last week, which included neuroendocrine tumors in the list of diseases eligible to receive special funding through the Congressionally-Directed Medical Research Program (CDMRP) Peer-Reviewed Cancer Research Program (PRCRP)! This is another issue we discussed while in DC in April, and Major Lacey Koelling, USAF has been working closely with the SNMMI to reach out personally to key members in both the House and Senate. In addition to her duties in the Air Force, Maj. Koelling has metastatic paraganglioma and is participating in a clinical trial at the NIH.
Before we close, we would like to give special acknowledgement to those who have given their time to travel and talk to our national leadership in support of all patients with pheo para: Bessie Lewis, Jennie Rowen, Henry Rowen, Karen Murdock, Patricia Horton, Jen Marchetti, Paul Kaczmarek, and Maj. Koelling. Our thanks also go out to the Pheo Para Alliance Board of Directors and Alliance Task Force members for giving us a space to consider our community’s issues and how policy makers in Washington can help us address them.
It’s great to see our hard work pay off! We look forward to working on these and other issues over the next year!
