Why the Name – “Rare Diseases Patients First!”

You may be wondering why the name “Rare Diseases Patients First!

The reason is that for so long patients with rare diseases have not been first. In fact, they have been last.  Even with the introduction of Orphan Drug acts in the US and Europe, to encourage the development of treatments for rare diseases, this is still an area that receives relatively limited attention.

While many smallcompanies are seeking to develop treatments for rare diseases, the treatments often focus on the repurposing of established medicines. These types of companies often lack the expertise to develop novel treatments and are seeking a short cut by starting with established medicines. Unfortunately, this approach also has many challenges and limitations. For instance, many of these companies have to rely on government funding in the form of grants.

Large pharmaceutical companies do have the expertise to develop new treatments for rare diseases. This area is certainly garnering more attention than it previously did. However, it was not until companies like Genzyme devoted themselves to the development of new treatments for patients with rare diseases that the pharmaceutical industry began to realize that there was genuine value in rare diseases, and that they could see a return on their investment. However, we have a long way to go. Although there are 7000 rare diseases, only about 200-400 treatments for rare diseases are available.

Rosa Parks is an iconic figure who challenged the unjust system that required her to sit at the back of the bus because of her color. One day she simply refused to give up her seat and move to the back of the bus. The name “Rare Diseases Patients First!” is certainly an appropriate name because like Rosa Parks we want patients with rare diseases to finally be able to move from the back of the bus to the front of the bus. When Rosa Parks was asked why she challenged the bus system, she said she just became tired of the way things were. At Rare Diseases Patients First! we would like to think that patients with rare diseases are tired of the way things are and want to move to the front of the bus.  We believe Rare Diseases Patients are, should be and come First!

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