Explaining the FDA Approval Process



How Real World Experience from Patients Waiting for a Drug’s Approval is an Important Element in the FDA Review Process



The FDA drug approval process can seem complex and arduous to people unfamiliar with the procedures that are in place. For people with rare diseases hoping for drugs to be approved, one of the toughest but most important virtues they need is patience.

Most drugs that undergo preclinical (animal) testing never even make it to human testing and review by the FDA. The drugs that do must undergo the agency's rigorous evaluation process, which scrutinizes everything about the drug--from the design of clinical trials, to the severity of side effects and even the conditions under which the drug is manufactured. 1 It can take a decade or more to go through the full clinical process and be considered for approval. 


This patience can truly be tested when the one waiting for the drug approval is a child with a rare disease. Children suffering from certain diseases are no different from any other group awaiting approval for promising drugs. See more.


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