The Process Behind the Development of New Blood Disorder Medications

2014 has been a year filled with new medications being released to the public for use; especially in the world of bleeding disorders.  Have you ever wondered the process behind getting a new medication from the lab to your home for use?Here is a little background on how this is all accomplished.

This whole process does not happen overnight. In the United States alone, it is estimated on average that it takes 10-12 years for an experimental drug to go from the lab to your home and that is if it even makes it there. The odds for a new drug to actually make it to market is only 1 in 5,000. New drugs are evaluated by the Food and Drug Administration (FDA) to ensure that all drugs marketed in the United States are safe for human use. The company that has created the new medicine seeks approval to market the drug and conducts lab and animal tests on the safety and effectiveness of a proposed new drug.  The company can then submit that information to FDA for review by physicians, pharmacologists and other scientists.

These clinical trials are extremely important. They are studies that use people to figure out the safety of a drug and what side effects it may cause down the road. The clinical trials gather information about a drug that have not yet been proven to treat patients with a specific condition, like hemophilia or von Willebrand Disease. According to the U.S. Food and Drug Administration website, clinical trials of these new drugs provide information concerning the following:

• Whether the drug has the effect it is supposed to have.
• How much of the drug to give to a patient and how often.
• What side effects are associated with the drug and how they can best be managed.
• How a drug is broken down in the body, and how long it stays in the body.
• Which foods, drinks, or other drugs can be used at the same time or should be avoided.
• Clinical trial results allow the FDA to make decisions about whether or not a drug should be approved for marketing.

There are different types of drug applications a company can apply for with the FDA and the amount of time it takes for a new drug to be approved by the FDA varies. The different types of drug applications include Investigational New Drugs (IND), New Drug Applications (NDA), Abbreviated New Drug Applications (ANDA), and a Biologic License Application (BLA). The approval process consists of two tier reviews– Standard and Priority. Standard Review is applied to a drug that offers minor improvement over existing marketed therapies and sets a 10 month goal for a standard review. Priority Review designation is given to drugs that offer major advances in treatment, or provide a treatment where none existed and sets a 6 month goal for completion.

With all of the science involved, combined with the investigational research and clinical trials, it is clear to see that new drugs are not approved all that often.  It is a rigorous and time consuming process but the newer drugs and factor medications that do make it to market may end up making all the difference in the world to someone with a chronic blood disorder.

Photo Credit- Food and Drug Administration (FDA)

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