Children with the rare genetic disease known as chronic granulomatous disease (CGD) have an immune system that fails at fighting off bacteria and fungi. They suffer frequent and life-threatening infections, as well as inflammatory problems in their tissues and organs caused by a build-up of granulomas, or hard lumps formed by white blood cells at the infection site.
One hope for these children is ACTIMMUNE, or interferon gamma-1b, a drug that lowers the risk of serious infection in people with CGD when combined with antibiotics and antifungals. In addition, it is often through treating rare diseases that medical teams can uncover a deeper understanding of the potential utility of a medicine in new areas.
Last fall, Horizon Therapeutics committed to donate through the FNIH more than 1,400 vials of ACTIMMUNE to treat patients participating in clinical trials at the NIH Clinical Center, with approximately half of the committed donation delivered to the Clinical Center in December 2022.
Horizon Therapeutics delivers medicines for rare, autoimmune, and severe inflammatory diseases and provides compassionate support to patients. They, as well as other pharmaceutical and biotechnology companies, have made donations through the FNIH-coordinated In-Kind Drug Donation Program to provide for effective care of diseases with no clear treatment guidelines, while freeing up NIH resources to support cutting-edge research.
When we come together, we have a stronger voice, make a bigger difference and leave a lasting impact in patient care,” said Jeffrey W. Sherman, MD, FACP, Executive Vice President, Chief Medical Officer, Horizon. “By partnering with the FNIH and supporting the Clinical Center In-Kind Drug Donation Program, clinical teams can allocate resources where they are needed most, ensuring patient care while having the opportunity to expand the scientific knowledge around other rare diseases. Many of us are a patient, know a patient, or have been touched by a patient’s story, driving our passion to collaborate in new ways because we know every day matters.”
About the NIH Clinical Center
The NIH Clinical Center is the nation’s largest hospital devoted entirely to clinical research, with approximately 1,600 clinical research studies currently in progress. Since opening in 1953, the Clinical Center has enrolled more than 500,000 research participants from all 50 states and around the world. The development of chemotherapy for cancer, the first use of an immunotoxin to treat a malignancy, the first gene therapy, and the first treatment for AIDS are just a few of the many significant medical breakthroughs that have resulted from research conducted at the Clinical Center.
A key tenet of the Clinical Center’s work is providing top-notch care, free of charge, for patients participating in clinical trials; and, regardless of patient levels, the Center must provide for patient needs within a fixed budget. The Clinical Center In-Kind Drug Donation Program, established in 2008 and coordinated by the FNIH, offsets some of the pharmaceutical costs for treating patients and frees up NIH resources to support its cutting-edge research. Many FNIH partners have supported the program since its inception.
Since 2019, Horizon has committed to making in-kind donations valued at a total of $19.2 million to the NIH Clinical Center.
Steve Holland, MD, Director, Division of Intramural Research, National Institute of Allergy and Infectious Diseases at NIH, whose work has centered on the pathogenesis and management of chronic granulomatous disease, believes that “the contribution of interferon-gamma-1b from Horizon has been an enormous boon to patients with rare diseases and refractory infections. We are very grateful for Horizon’s generosity and partnership in our shared mission of helping the severely ill.”
Drug donations from companies can make an immense difference in overall patient care at NIH and frequently contribute to improved patient outcomes. The FNIH works to match the Clinical Center’s needs with potential donors or specific donations. For more info about the program, see Clinical Center Drug Donations | The Foundation for the National Institutes of Health (fnih.org).
If your company would like to contribute to patient care through either pharmaceutical or monetary donations, please reach out to the FNIH Strategic Alliances and Advancement team at [email protected].