No healthcare professional wants to hear that a drug being studied in a clinical trial did not meet the endpoints. It means that after countless hours of hard work, the therapy being examined will not be able to help people. But even though the drugs themselves will not be available, the knowledge gained through the trials will be. Researchers discover new information about diseases when they conduct a clinical trial, and this information can be applied to diagnosing people, improving treatment, or creating a better understanding of the disease. Dr. Kevin Williams talks about how a failed trial of rivipansel, a drug made for the treatment of sickle cell disease, actually helped to advance doctors understanding of the illness.
About Sickle Cell Disease
Sickle cell disease includes a group of disorders that are characterized by malformed red blood cells that look like sickles. The most common and severe form of this disease is sickle cell anemia. In sickle cell disease, deformed cells cause blockages and restricting blood flow as they get caught along the walls of blood vessels.
This disease is caused by a mutation in the gene responsible for producing hemoglobin, which carries oxygen throughout the body. This gene is inherited in an autosomal recessive pattern, meaning both parents must pass down a copy to their child. Sickle cell disease is most common in people of African and Hispanic descent.
Symptoms of sickle cell disease include pain crisis, swelling of the hands and feet, and symptoms associated with anemia like fatigue, jaundice, and delayed growth. Adults tend to constantly feel the effects of this disease, but children usually only experience them during pain crisis. Regardless of age, damage usually occurs to the organs that are affected by the blocked blood flow. The most commonly damaged organs are the brain, eyes, spleen, liver, kidneys, lungs, heart, skin, joints, and bones.
There is no cure for sickle cell disease. While some people qualify for bone marrow and blood transplants, not everyone is eligible for this procedure. Other forms of treatment are symptomatic and meant to prolong life.
About Dr. Kevin’s Study
Dr. Kevin Williams is the Chief Medical Officer for Rare Diseases at Pfizer. Pfizer recently concluded the third phase of their study of rivipansel, which did not meet all of their endpoints. While there was disappointment, Dr. Kevin spoke of the knowledge that was gained despite the failure of the trial. He spoke of the information about vaso-occlusive crises and sickle cell disease that was gained, and how it will help all future efforts towards creating treatments.
The Importance of Clinical Trials
Clinical trials are essential for the development of new treatments, which then better the lives of people. While these trials are needed in order to create a new therapy, they also teach researchers new information about diseases, especially rare diseases. Considering all of the regulations in place for clinical trials, they are a safe way for researchers to develop their drugs while also learning more about diseases.
While some trials do fail, they are still essential in learning more about diseases. A trial that does not meet its endpoints is never a complete failure, as researchers will always gain new knowledge through the course of the study.
Only 5% of rare diseases have FDA approved treatments, which stresses the importance of these trials. While many trials are not successful, the information and inspiration that they give to researchers is necessary for improving future studies. Pfizer and other companies will continue to conduct these trials to create treatments for rare diseases.
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