For the Media
Online Press Kit
Media inquiries should be directed to:
55 Kenosia Avenue
Danbury, CT 06813-1968
What is a rare disease?
NORD uses the same definition that the U.S. Food and Drug Administration uses: A rare disease is one that affects fewer than 200,000 Americans at any given time.
How many rare diseases are there?
According to the National Institutes of Health, there are between 6,000 and 7,000 rare diseases affecting from 25 to 30 million Americans. This adds up to 1 in 10 people.
How old is NORD?
It was established in 1983, the same year the Orphan Drug Act was passed by Congress and signed into law by President Ronald Reagan.
What is the Orphan Drug Act?
It provides incentives for companies to develop treatments for rare diseases.
Has this legislation been successful?
Yes. During the decade before 1983, only 10 new treatments were developed by industry for rare diseases. Since 1983, more than 3,000 products have been designated "orphans" (meaning they are in development as possible treatments for rare diseases) and more than 450 have been approved by FDA for clinical use.
What services does NORD provide?
- Information about diseases and referrals to patient organizations
- Patient assistance programs
- Research grants and fellowships
- Advocacy on public policy issues and information on legislation that impacts the rare disease community
- Education for patients, medical professionals, students and the general public
- Help in forming organizations and mentoring for patient advocacy groups
How can you find out if someone is doing research on your disease?
ClinicalTrials.gov is an excellent website that lists all studies receiving funding from the U.S. government. Patient organizations are also a good source of information on clinical trials.
How can people help a family member with a rare disease get the best possible treatment?
With rare diseases, it's even more important for the family to be involved than it is with more common diseases. This is because there may be only a few doctors, and only a few medical centers, with expertise on this particular disease.
Your physician may be able to help you find an expert on your disease. You won't be insulting him or her by asking. Doctors understand the importance of involving someone who has experience with the disease.
It's usually best, with rare diseases, to be treated at a teaching hospital affiliated with a university, rather than a smaller community hospital. The teaching hospitals would be more likely to have experience with rare diseases. We have a list of teaching hospitals in each state, and you can call us to ask for the ones near you.
Another very important thing to do is to find out if there is a patient organization for your disease. Search the Organizational Database on this website to find an organization specifically for people with your disease. These organizations can be tremendously helpful in many everyday ways, and they are also a wonderful way to network with other people who have the same disease.
Where can I go for information about rare diseases?
The Internet is a wonderful tool, and even if you don't have a computer you can get access to it through your local library, through a senior center, or through other social service offices. You can also contact us at NORD, and we will be happy to send information to you through the mail.
If you go online yourself at home, just remember that there is no guarantee that everything you read online is accurate. It's important to use web sites you can trust, and that includes ones from the federal government, from universities, from teaching hospitals, or from non-profit organizations like NORD and the March of Dimes. The National Institutes of Health or NIH has some excellent web sites with information about certain rare diseases. The CDC (or Centers for Disease Control and Prevention) also has some good information about some rare diseases.
Also, people can call us at NORD and talk to our genetic counselor. We will email information to them or send it through the mail. We'll also refer them to a patient organization specifically for their disease, if there is one.
Why does it take so long to get a diagnosis? How can we educate our doctors?
Getting a diagnosis is one of the problems that people contact us about frequently. It's difficult because there are nearly 7,000 rare diseases, and the first doctor you go to may never have seen the particular rare disease you have. NORD and other patient organizations are working to raise awareness among physicians. It’s very important for families to be involved in the diagnosis and treatment process, and to become advocates for their loved one with a rare disease.
Is there anything you can do if you or your loved one don't have a diagnosis?
This is a very difficult situation, and we get a lot of phone calls and emails from people who are having a hard time getting a diagnosis. It's important to see the right kind of doctor for your disease. People are always welcome to contact our genetic counselor to get their suggestions on the type of doctor who might be an appropriate starting point for them. Also, it's a good idea to keep a notebook at home, and jot down things that seem significant so that you won't forget to mention them when you see the doctor. You know your loved one better than the doctor does, so if something seems unusual or significant to you, trust your instincts.