Quicker Approval for Gene Therapy: US FDA Looking at Hemophilia

In order to keep up with the quick-growing field of gene therapies, the United States Food and Drug Administration (FDA) announced it will provide faster methods of approval for new treatments. The new regulations will likely come into effect regarding treatment for hemophilia first. Keep reading to learn more, and follow the original story here for additional information.

Hemophilia is a genetic condition that affects the clotting of blood. Normally, when a cut occurs, mechanisms within the body drive blood cells together to form a block, or clot, that stops the flow of blood. In hemophilia, one of the major factors responsible for this behavior is missing. Depending on the specific genetic factor affected, a person may be diagnosed with either hemophilia A, or hemophilia B. Click here to learn more about hemophilia.

Traditional therapies often must be administered multiple times. Furthermore, in cases of rare genetic conditions such as hemophilia, treatments are often only able to focus on symptoms. Root causes remain unaffected, and the person must learn to live with and around his/her condition.

Perhaps the reason gene therapy has seen so much growth is because it is different in both respects. Gene therapies are designed with the idea of administering treatment only one time. They target the disease at the ground level, and alter the body’s functions internally. Many researchers are working to reprogram viruses, which serve as a method of delivery and proliferation.

That’s also where regulation, however, becomes somewhat difficult. The field of gene therapy research and development continues to grow and experiment. It simply isn’t the same as traditional drug development and agencies are unprepared for its growth and concepts. All the while, patients continue to need treatment and if there exists a way to help them it would be cruel to withhold it. Yet in many cases, the long term consequences of gene therapies have yet to be observed.

With the treatments making changes to the body at cellular and genetic levels, many people are alert to the possible harm that could be done. The types of diseases these new therapies target, however, tend to be those that are most dangerous. Thus, though the potential cost of some treatments may seem high, the condition of patients is often guaranteed to be fatal with no current treatment to be seen.

In the case of hemophilia, drugs are often evaluated based on their ability to control and stop blood loss. Drugs gain approval based on their ability to lessen the number of bleeds a patient suffers, and decrease the number of infusions (replacement clotting factor) the patient needs over time.

New FDA standards for gene therapies, however, may decide upon approval based on the amount of clotting factor a person’s body is able to produce after treatment.

Evaluating the drug based on biological indications rather than on demonstrable effects is not new territory. This is what is known as a surrogate endpoint. Effectiveness of treatment may be determined after approval should biological and chemical markers indicate its effectiveness. For example, drugs may gain approval by lowering cholesterol, and later be proved to reduce the risk of heart attacks, and stroke.


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