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Resources for rare diseases and orphan drugs

Posted by on in Allergy
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Many people live with a constellation of symptoms that make up a rare disease that is difficult to diagnose, because it is just that—rare.

160531RareDiseases-325In the United States, the National Institutes of Health (NIH) considers a disease rare if there are fewer than 200,000 individuals affected. For reference, there are nearly 80 million people with high blood pressure in the U.S.

These patients often go from provider to provider seeking an answer to their health problems. In fact, most patients see up to eight different providers before getting a diagnosis, delaying treatment. It can sometimes take seven or more years to get an accurate diagnosis, if ever. Some diseases haven't even been named; they are known as SWAN, or Symptoms Without a Name.

If it is so difficult to get a diagnosis, imagine how challenging it is to find treatment.

In the United States, the Orphan Drug Act provides incentives for the creation and development of therapies for rare diseases. Orphan drugs are therapies that are developed for the treatment of rare diseases. The development of these drugs is slow going—there are several challenges, as you can imagine, because there are so few patients.

There are several rare diseases included in our own blogs, such as Hereditary Angioedema, Multiple Sclerosis (MS), Hodgkin Disease, and Marfan Syndrome. These brave patients and their families do everything they can to live a "normal" life, but all will say that they often just have to take things day-by-day or moment-by-moment and pray for effective therapies.

It is easy to take good health for granted. I remember when I'd greet my grandfather and casually ask how he was doing, he would always say, "Well, I've got my health." This became a joke among the grandchildren; however, as we have matured and seen those we love get diagnosed with serious health conditions, even some rare diseases, we appreciate his comment and now say it to each other.

Resources are available for those families and patients who are challenged to meet the needs of treatment and day-to-day life when living with rare disease. See the webpages listed below:

National Organization for Rare Disorders (NORD)
Medication & Financial Assistance for Patients & Families with Rare Diseases
Genetic and Rare Diseases (GARD) Information Center
U.S. Food and Drug Administration Orphan Drug Act

If you have questions about your own health, contact us.

Melanie Chala Wayne, MSN, FNP-BC is a nurse practitioner for Oak Street Medical and Oregon Allergy Associates.