Home | Orphan Drugs

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KNOW THIS:

Collectively, 350,000,000 people are affected by rare diseases around the world

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Is treatment the

first step to a cure?

In the United States alone, over 75% of all medication purchased in 2017 was non-orphan medication.

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ORPHAN

DRUG:

Medicine to treat patients who have rare diseases.

Typically, orphan drugs have been avoided by scientists and other researchers, because they are the least profitable (with the smallest consumer group).

Orphan Drug Dilemma

Many do not know what an orphan drug is, nor it's potential. A number of those who do know what an orphan drug is are concerned about its possible effects on the world of medicine.

In some cases, biotech companies may manipulate the financial policies of the Orphan Drug Act in order to rein in profits, without having the intent to help people as their main focus.

Kaiser Health News defines an orphan drug and goes into detail about this specific issue in the video on the right.

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For the few that do know what an orphan drug is, there are many misunderstandings that come along with the idea of orphan drugs and the Orphan Drug Act. To have a better understanding of orphan drugs, watch the National Organization for Rare Diseases' video on 5 Myths About Orphan Drugs and the Orphan Drug Act.

Visit the Issues page to learn more about the the problems regarding orphan drugs, or take a look at the Background page to discover the history behind orphan drugs.

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Pushing for the Orphan Drug Act

As one of the most influential people in getting treatments for people with rare diseases, Abbey Meyers had a lasting impact on orphan drugs today. She advocated for the passing of an orphan drug law with the assistance of Congressman Henry Waxman. Largely due to her influence, the law passed, it opened the door for and increase in the manufacturing of orphan drugs.

Watch the video on the left, and visit the Timeline or Innovators page below to learn more about Ms. Meyers.

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PNH & Soliris- An Overview

PNH, or Paroxysmal Nocturnal Hemoglobinuria is one of many rare diseases. To a person who has this disease, stem cells that are in the bone marrow acquire a gene mutation that causes them to produce abnormal blood cells instead of healthy red blood cells and white blood cells. The complent, part of the immune system, then destroys these cells. Before Soliris was an option, the only way to treat people was with a bone marrow transplant, which is incredibly risky.

Now, Soliris can be used to treat this disease. Below is a diagram of how complement destroys these cells.