BACKGROUND
What is an orphan drug?
Orphan drugs are drugs that have been approved to treat rare diseases. The United States of America defines a rare disease as one that affects less than 200,000 people, or one that affects over 200,000 people but does not have sufficient funds to research, develop, an market a suitable cure.
Orphan drugs are "orphans," in the literal sense of the word. Many companies decided to avoid finding treatments to rare diseases because there were no financial benefits to doing so. This changed with the passing of the Orphan Drug Act in 1983.
With the simple definition of an orphan drug, and the background behind the Orphan Drug Act, review the video on the right for a visual demonstration.
About Rare Diseases
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In America alone, over 25 million people are affected by a rare disease. This is about 1 in 10 Americans.
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In Europe, it is estimated that nearly 30 million people have a rare disease.
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Collectively, 350 million people are affected by rare diseases worldwide.
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There are roughly 7,000 diseases that are classified as "rare"
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Only 5% of rare diseases have treatments.
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80% of rare diseases are genetic.
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There are over 600 orphan drugs that are approved by the FDA.
In 2017, the most common orphan drugs in the process of being developed were designated as treatments for a rare cancer or genetic disorder. Many categories in this list fall under a genetic disorder as well. The orphan drugs that were least common in being developed were growth disorders and blood disorders.
Read the following infographic to learn more.
Prevalence Around the World
Multiple countries have acknowledged rare diseases. These countries have specific criteria as to what defines an rare disease that is suitable to have an orphan drug as treatment, and these criteria include the prevalence of the disease in that country.
UNITED STATES OF AMERICA - 0.075%
EUROPE - 0.05%
SOUTH KOREA - 0.04%
JAPAN - 0.04%
TAIWAN - 0.01%
AUSTRALIA - 0.01%
The Development Process
Below is a diagram of the typical process that most drugs go through, from the beginnings steps of initial research and development to marketing and commercialization.
The development process is not much different for orphan drugs.
1. Research & Discovery
Research is the foundation of all new medicines. Naturally, research also has to be at the core of developing new orphan drugs. With orphan medicine, research starts with finding the right molecule.
In many cases, a hypothesis related to the disease is developed to begin research.
Biotechnology is crucial to the creation of orphan products as it allows scientists to gain a better understanding of the diseases being researched and ideas for a potential cure. Below is Gleevec, an important drug that has been discovered.
To the left is the molecular structure of Gleevec. This medicine has an orphan designation for treating leukemia, specifically Chronic Myeloid Leukemia. It was discovered in 1959, and was intentionally developed to target the Philadelphia chromosome, which is found in leukemia patients.
2. Development and Marketing
One of the major groups that plays a role in this vital part of the drug development process is the FDA OOPD, or the Office of Orphan Products Development. This group's goal is to assist companies in the evaluating their potential orphan products. The OOPD strongly relies on looking over data to do this, and has allowed for at least 600 biotech orphan products to be developed since the beginnings of the Orphan Drug Act of 1983.
3. Patient Access
Companies need to be completely sure that all of their orphan medicine is safe to consumers, and they must prove that their medicine is effective. In order to make their drug accessible to patients, these companies must also set their drugs at reasonable prices. It may even be decided that reimbursed patients will be granted new treatments.