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Clinical studies are critical to the drug development process

Through clinical studies, teams of medical professionals find new ways to diagnose, treat, and cure diseases. Treatments may include new drugs that are being used in humans for the first time, new surgical procedures, or new ways to use existing treatments. The goal of the study is to determine the safety and effectiveness of the therapy, device, or procedure.

Clinical studies can also simply observe patients — to improve the diagnosis process and the quality of life for people with chronic illnesses. Both healthy people and those with a disease or illness may participate in clinical studies.

Clinical studies offer many potential benefits to people with sickle cell disease, including:

  • Access to new treatments before they are widely available
  • Regular and careful monitoring of the disease by a team of medical professionals
  • Opportunities to learn more about advancements in sickle cell treatments

The drug development process

The journey from thousands of molecules (Stage 1) to one approved drug (Stage 5) can take more than a decade. Clinical studies—and the heroes who participate in them—are the key to finding new treatments that are both effective and safe.

 

More than 30 clinical studies related to sickle cell disease have been abandoned in the past ten years because of low enrollment.

$359 million

average amount of money it takes to develop a new drug.

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12 years

average time it takes to develop a new drug.

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5 in 5,000

number of drugs that make it to human testing.

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1 in 5 drugs

number of drugs approved for use by humans.

FAQs about Clinical Studies

Who runs clinical studies?

Government agencies do run clinical trials, but they are conducted by private pharmaceutical companies that are developing new treatments and medications. The National Heart, Lung, and Blood Institute website lists government-run clinical trials for sickle cell disease.

Private companies developing new treatments include:

Clinical trial treatment costs are often covered by your insurance — ask the research team for more information. Some studies offer participants stipends in the form of money to pay for expenses, like travel to the clinic or hospital.

Clinical trial treatment costs are often covered by your insurance — ask the research team for more information. Some studies offer participants stipends in the form of money to pay for expenses, like travel to the clinic or hospital.

What happens during clinical studies?

Before you agree to participate in a clinical trial, the researchers must tell you about any tests, procedures, and medications (including dosages) you will be given. This is called informed consent, and it’s a law that requires researchers in all clinical trials to provide you with complete information about the potential risks and benefits of participating in the study. Even after you agree to participate in a study, and after the study has begun, you have the right to ask for additional information and the right to withdraw from the study.

Before you agree to participate in a clinical trial, the researchers must tell you about any tests, procedures, and medications (including dosages) you will be given. This is called informed consent, and it’s a law that requires researchers in all clinical trials to provide you with complete information about the potential risks and benefits of participating in the study. Even after you agree to participate in a study, and after the study has begun, you have the right to ask for additional information and the right to withdraw from the study.

The medical research team running the trial is required to keep the identity of all participants confidential, so no one will know that you’re participating unless you tell them.

It is your right to withdraw from a clinical trial at any point. If you want to drop out, notify your research team immediately.

The length of clinical trials varies from study to study, and some require visits to a hospital or clinic during regular working hours. Ask the research team for details about the time commitment for the trial you’re considering.

Who participates in clinical studies?

Clinical trials are open to both healthy volunteers and those with an existing illness. All clinical trials have standards, called inclusion criteria, that define who can and cannot participate, including factors such as age, gender, treatment history, and stage of the disease. If you meet those criteria, you may be eligible to participate.

When you participate in a clinical trial, you provide sickle cell disease researchers with valuable information that may help them develop new treatments, or even a cure. Even if your participation doesn’t directly lead to new treatments or a cure, your data will help researchers understand what works and what doesn’t; both of which provide important information.

Aside from the satisfaction that you are directly contributing to sickle cell disease research, you may also:

  • Get access to new treatments before they are widely available
  • Have your disease monitored more regularly by a team of medical professionals who specialize in sickle cell disease
  • Learn more about advancements in sickle cell treatments

Feel like you have more control over your disease

African Americans are typically underrepresented in clinical trial, which means that the medical world is lacking information that could be helpful in finding the next treatment or cure, especially for conditions like sickle cell disease, which is much more common in African Americans than other groups of people. Reluctance and skepticism may be rooted in the history of African Americans’ relationship with medical researchers, and with good reason.  Visit our page on the History of African Americans and Clinical Trials to read about this history and the many advancements that have been in recent years.

What happens after clinical studies?

Results are posted on clinicaltrials.gov, though the medical information there can sometimes be difficult to understand. Talk with your provider or the person who referred you to the trial for more information.

It depends on the results of the study and the FDA approval status. You’ll need to discuss options with your doctor.

If the results of the study are successful, the treatment moves to the next study phase. A treatment must complete all phases and be approved by the FDA before it is made available to the public.

Researchers analyze all of the study data and report their findings to government agencies, at scientific meetings, and in scientific journals.

Have another question about clinical studies?

Talk to your doctor.
It's easy!

Take our “Ask Your Doctor” sheet to your physician to start a conversation about clinical studies. There are 5 questions that will help you learn more about studies in your area.

Want more information about clinical studies?

ClinicalTrials.gov is a database of privately and publicly funded clinical studies conducted around the world. Explore 259,383 research studies in all 50 states and in 201 countries.

ClinicalTrials.gov is a resource provided by the U.S. National Library of Medicine.

The National Institutes of Health (NIH), a part of the U.S. Department of Health and Human Services, is the nation’s medical research agency — making important discoveries that improve health and save lives.

FDA is an agency within the Department of Health and Human Services.

The FDA's organization consists of the Office of the Commissioner and four directorates overseeing the core functions of the agency: Medical Products and Tobacco, Foods, Global Regulatory Operations and Policy, and Operations.

CDC laboratories routinely work with some of the most deadly germs in the world – identifying health threats and conducting vital public health research. CDC constantly develops and reviews extensive laboratory guidelines and procedures to protect both the public and laboratory workers.

Clinical studies website made possible by a generous contribution from

Our vision is a world without sickle cell disease

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