FDA with No Delay: The Food and Drug Administration’s Rush to Approve Treatments for Rare Disorders and Cancers


While many types of cancer, like breast cancer, leukemia, and lung cancer all have foundations, charity walks, and, probably most important, direct treatment– rare cancers have none of these things. Sure, they have the hand-me-down therapies from other diseases and an umbrella association, NORD (or the National Organization of Rare Disorders), but they didn’t have the same access to personalized treatments– that is, until recently.

In the last five years, the number of drugs that researchers have put past the CDC for rare diseases and cancers has nearly doubled. In 2017, 700 drugs per year of rare illnesses came before the CDC– a marked increase in the number that the CDC tested in 2012.

This is partly due to the new designations that the CDC have put forth regarding rare drugs. The new name for “orphan drugs,” or drugs that treat a small, “orphan” population, places them ahead of other drugs for testing. This impetus for the development and approval of these drugs is mainly due to the difficulty that diagnosing and treating these cancers and these diseases presents.

But What are Rare Diseases and Cancers?

According to the official Food and Drug Administration website, to be a rare disease, it must “affect less than 200,000 people in the United States.” Examples of one of the more than 1200 conditions on the list include Aceruloplasminemia, a disease that leads to the abnormal accumulation iron in the bloodstream, or Visual Snow Syndrome, a neurological disorder which has small particles that resemble snow floating in the vision of the patient.

This diagnosis of an “orphan drug,” or drug used to treat a rare disease, has gotten a recent update with some new distinctions. Orphan drugs are now able to receive financial benefits, with the creators offered access to new loans and waived fees. Orphan drugs also now enjoy a longer term of exclusivity of seven years, where the creators will have the sole production and distribution rights to these drugs for nearly twice as long as they could before. This change provides a sometimes-well-needed incentive for pharmaceutical companies to research and invest in drugs for orphan diseases and treat some of these people who need it the most.

What are your thoughts on these recent rule changes in the CDC? Do you know someone who suffers from a rare disease or cancer? We’d love to know your experience! Please let us know with a comment below what you think!

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