Sickle Cell Disease and Clinical Trials
What is Sickle Cell Disease?
Sickle cell disease (SCD) is the most common inherited blood disorder in the U.S. It primarily affects African-American births (1 in 500) and Hispanic American births (1 in 36,000). It is chronic and can cause severe pain, organ damage, or even stroke.
Sickle cell disease is devastating for patients and their families. FDA understands that patients have medical needs that current treatments cannot meet, so new treatments are urgently needed.
FDA wants to raise awareness about clinical trials for sickle cell disease. The agency is developing educational materials to raise awareness about clinical trials. Some of these materials include a webinar for patients to learn how to find clinical trials for SCD and meetings with SCD stakeholder groups to determine the best strategies to raise awareness about clinical trials participation.
If you are a patient with sickle cell disease, talk to your doctor to decide if clinical trials are right for you.
FDA Talked to Patients with SCD
In February 2014, FDA hosted a Sickle Cell Patient-Focused Drug Development Meeting to learn what patients think about available treatments for SCD and how the disease impacts their lives. This was the most well attended patient meeting in FDA’s Patient Network’s history.
Patients said that they want more clear, transparent information to help them balance the benefits and risks of participating in clinical trials. Patients also need new and better treatments for:
- Acute and chronic pain
- Sleep disturbances
- Stroke
- Fatigue
Sickle Cell Disease Treatment Options
Current treatments for sickle cell disease are limited to preventing and managing a pain crisis, which is the most debilitating symptom of SCD.
- Hydroxyurea: Patients can take this oral medication to help reduce the frequency of pain crises and the need for blood transfusions.
- Pain Medications: Patients can manage pain with nonsteroidal anti-inflammatory drugs (NSAIDs), opioids, antidepressants, and anticonvulsants.
- Chronic Transfusion Therapy: Patients can get regular blood transfusions to help prevent complications.
- Bone Marrow or Stem Cell Transplants: Younger patients with severe SCD can consider transplants, but they are expensive and require a suitable bone marrow or stem cell donor.
More Treatment Resources for Sickle Cell Disease
What are Clinical Trials?
Clinical trials are very important to developing more and better treatments for sickle cell disease.
Before FDA can approve a new medical product, experimental medications and therapies must be tested in a controlled environment on the people who are most likely to use the medicine. This is so FDA and drug developers can ensure that the medical product is safe, that it works, and to determine how the medicine compares to other existing treatments.
Learn More About Clinical Trials
Why Participate in a Clinical Trials?
Patients are motivated to participate in clinical trials for many reasons. For example, clinical trials can provide another option when standard therapy have failed. Patients often join clinical trials because:
- None of the standard (FDA-approved) treatment options work for the patient.
- The patient is unable to tolerate certain side effects of the existing treatment.
- The patient wants to be a part of the research process that contributes to the advancement of SCD treatments.
Safety in Clinical Trials
There are several laws in place to ensure that clinical trials are as safe as possible. Some safeguards include:
- Study monitors: Studies are monitored at all times by doctors and trained staff to ensure the study is as safe as possible.
- Informed consent: Researchers are required by law to give you all the facts about a study, including potential risks and benefits, before you agree to volunteer for a clinical trial.
You can leave the study at any time for any reason.
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