In 1983, Congress passed the Orphan Drug Act to help incentivize the development of medicine to treat rare diseases.2 Since this law was signed, hundreds of drugs for rare diseases have been approved by the FDA.2 Thanks to the work of advocates, caregivers and drug and device manufacturers, even more solutions are being tested via clinical trials today – and may one day make a positive difference to patients.2
Due in part to many rare diseases’ complex biology and unavailable or incomplete natural histories, 95% of these conditions have no associated treatment options.1 Small patient populations also make it difficult to conduct clinical trials on potential rare disease treatments.
Living with or having a loved one with a rare disease is easier with support. The National Organization for Rare Disorders (NORD) hosts patient listening sessions with the FDA’s Patient Affairs Staff that offer patient communities opportunities to share experiences about their disease or condition.2 Participating in patient forums or working with nonprofits to help elevate awareness around a condition provides community and may create a sense of connection to others.
For example, a group of parents whose children lived with Growth Hormone Deficiency, a rare condition, turned their grassroots support group into a nationally recognized non-profit, called the MAGIC FOUNDATION4 that provides support services for families of children afflicted with growth disorders. In June 2014, the U.S. Congress passed a bill nationally recognizing Children’s Growth Awareness Week4 to draw attention to the challenges faced by children with these conditions. Novo Nordisk is a proud sponsor and partner of the Magic Foundation and we are committed to advancing solutions for this patient community.
For many, being diagnosed with a rare disease can be both emotional and overwhelming. Joining support groups and utilizing reputable information sources to learn about a condition can improve mental health and help prevent the spread of misinformation.
At Novo Nordisk, we work hard to drive change for those living with
rare diseases. We have two education programs in the U.S. – Changing
Hemophilia® and More than
Height – that serve as resources for people affected by these
Improving medical solutions, advocating for research and providing education and community are all ways to help improve life for patients with rare diseases. This year on Rare Disease Day, let’s do more than raise awareness – let’s work together to make a difference.