By Joseph Chiao, MD
February 23, 2017
As we look ahead to spring, which is always a busy time for all of us at CSL Behring, we join the hereditary angioedema (HAE) and rare disease communities around the world in celebrating the 10th international Rare Disease Day on February 28. Every year at this time, we proudly join with hundreds of patient organizations worldwide to raise awareness of rare diseases and their impact on people’s lives.
There are upwards of 7,000 rare diseases, with approved treatments for only 5% of these. HAE, a rare and potentially life-threatening genetic condition, occurs in about 1 in 10,000 to 1 in 50,000 people. People with HAE can have considerable swelling episodes referred to as angioedema, which can affect many parts of the body, including the face, abdomen, larynx and extremities.
This year’s Rare Disease Day theme is, “With Research, Possibilities Are Endless.” Working in the HAE therapeutic area, I fully subscribe to this mantra; I’ve seen what’s possible when we commit to R&D in HAE as well as in the other rare diseases. We are driven by our promise to unlock the potential of biotherapies.
With research, possibilities are endless.
A decade ago, HAE patients had no targeted treatment options. I feel fortunate and proud to have been on the front lines of helping to make available the first targeted therapy. Since then, more targeted therapies have been approved, giving people with HAE more options than ever to help prevent and treat attacks.
Our more than 1,100 research scientists around the world are dedicated to discovering and developing new therapies that save and improve lives, and we have invested more than $2 billion in R&D over the last five years. While we continue to research HAE with the hope of eventually conquering the disease, we also continue to introduce new innovations that address unmet medical needs or enhance current treatments for other rare and serious conditions.
Our innovations respond to human need, not market size.
For example, we introduced flexible dosing for patients with immunodeficiencies and are working to develop a family of novel longer-acting recombinant coagulation factor medicines to progress the care of people with hemophilia and other coagulation disorders. Our innovations respond to human need, not market size; one of our “super orphan” therapies treats a condition affecting approximately 300 patients in the U.S. and only one million worldwide.
I’m energized each day, in part, because of my excitement about the promise of new therapies in the management of HAE and other rare medical conditions. Imagine the improvement in a patient’s quality of life, if he or she is able to subcutaneously inject a therapy rather than having to infuse it intravenously.
With 1,100 research scientists around the world, we continue to research HAE with the hope of eventually conquering the disease.
We like to say innovation is in our DNA at CSL Behring, and I look forward to being part of the team that presents and publishes research around one of our latest innovations. In March we join thousands of health care professionals at the American Academy of Allergy, Asthma and Immunology (AAAAI) annual meeting to share the latest research and clinical management approaches in combating HAE. The meeting also provides us with an opportunity to connect with the HAE community, which informs our R&D, and to track our efforts in working with patients, advocacy organizations and healthcare providers.
Even with multiple treatment options available for HAE patients, we continue the important work of educating primary care physicians and allergy-immunology specialists about HAE, especially on the need for earlier diagnosis and setting realistic individualized management plans. Our focus this year is on educating the scientific community about HAE and our therapy options, and collaborating with them and HAE advocacy groups to provide the best possible care for people with the disease.
At CSL Behring, we believe that we’re just getting started, and our future has never been brighter.
Dr. Chiao is Medical Director, Therapeutic Area Head, Hereditary Angioedema at CSL Behring