What is a Rare Disease Company?

Rare Disease

Let’s answer the question of what a rare disease company is by first defining the term “rare disease.”

The Wikipedia definition of a rare disease is one that affects a small percentage of the population.  The Rare Disease Act of 2002 defines a rare disease to be one that affects fewer than 200,000 people in the United States, or approximately 1 in 1,500 people.  Similar definitions were made in Japan and the European Union.

Governments were driven to define rare diseases in order to craft policies and programs that would promote research and development of treatments.

Rare diseases are typically genetic by origin, so they are present throughout a person’s life. Symptoms can appear immediately after birth and usually become evident before adulthood, although some experience the onset of symptoms in early adulthood.  Another trait of rare diseases is that many are life-threatening. A chilling statistic is that approximately 30% of children with rare diseases die before their fifth birthday.

Rare Disease Company

Many pharmaceutical companies research and develop drugs and treatments for diseases that affect more people.  A larger potential consumer base implies greater sales.  Every company is in business to accomplish its primary mission and make money in the process to fund product manufacture, research and development, salaries, rent, etc.  Simple math shows that more sales revenue equals longevity, so several pharmaceutical companies compete with each other for effective treatments for diseases that affect millions of people annually.

A rare disease company focuses on treatments and therapies for rare diseases and therefore will not have the same volume of patients as larger pharma companies.  They must still fund research and development, testing, FDA and other global FDA-equivalent agency approvals, stringent manufacturing processes, etc, all of which are incredibly expensive. Since prices of treatments fund the company’s operations, it stands to reason that the per-unit cost of rare disease therapies is much higher, since far fewer patients are treated.

Another challenge facing a rare disease company is in marketing. The medical community-doctors, nurses, etc may not have heard of either the definition of a particular disease, or a treatment, if one exists. Many doctors learn of new treatments and therapies from sales reps that constantly travel from practice to practice, or at conferences.

There’ a lot of personal gratification and satisfaction to be had when working for a company that produces a medicine or treatment actually saving the lives of children or young adults, helping them live their lives as most “normal” people. This is true especially when the severity of the disease causes a great deal of pain and suffering before certain death.

The World’s Rare Disease Company dedicates itself to serving the smaller population of rare disease sufferers.

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