Diabetes. Asthma. Rheumatoid arthritis. All are chronic, prevalent diseases that affect millions of people. Fortunately for the patients impacted by these disorders, a variety of safe, effective,FDA-approved therapies are available to treat and manage these conditions. And, the media who cover health care tend to report new developments in these therapeutic areas precisely because the large patient populations are a big part of their reader- and viewerships.
It’s a different picture for patients with rare diseases. Take hereditary angioedema (HAE), a serious, scary and potentially deadly swelling disorder that affects at most one in 10,000 people. Or acromegaly, a pituitary disorder with another small patient population. Or congenital fibrinogen deficiency, an extremely rare bleeding disorder that impacts only one in one million people. If you’ve never heard of these diseases, it’s because the media tend not to cover them, at least not on a regular basis. Individuals (and their families) coping with these illnesses have not only unmet medical needs, but also unmet information needs.
The good news here is that companies like our client CSL Behring have made the investment to develop FDA-approved treatments for people with diseases like HAE and congenital fibrinogen deficiency. And many rare diseases have spawned active patient advocacy organizations whose raison d’etre is to spread the word about these disorders so that people who may have them will know the symptoms, seek care, be diagnosed and receive appropriate treatment.
At MCS we collaborate with both companies and patient groups in the rare disease space to educate professionals and the public about these issues. Patients with rare diseases are no different than patients with very common disorders: they need attention, information, resources and care.
-Jeff Hoyak, President