Clinical Trials

New drugs in the United States are tested in clinical trials (research studies) to determine that they are safe, that they can successfully treatment a particular medical condition, and what the suggested dosage should be. The US Food and Drug Administration (FDA) reviews the results of the clinical trial to determine if the drug is a safe and effective treatment.

Clinical trials are a key research tool for advancing medical knowledge and patient care. ECD Clinical trials are used to determine whether a treatment is safe and effective for ECD patients. Clinical trials produce the best data available for health care decision making.

Purpose
The purpose of clinical trials is research, so the studies follow strict scientific standards. These standards protect patients and help produce reliable study results. A clinical trial’s protocol describes what types of patients are able to take part in the research—that is, who is eligible. Each trial must include only people who fit the patient traits for that study (the eligibility criteria). Eligibility criteria differ from trial to trial. They include factors such as a patient’s age and gender, the type and stage of disease, and whether the patient has had certain treatments or has other health problems.

What to expect
During a clinical trial, doctors, nurses, social workers, and other health care providers might be part of your treatment team. They will monitor your health closely. You may have more tests and medical exams than you would if you were not taking part in a clinical trial. Some people will need to travel or stay in hospitals to take part in clinical trials. ECD Clinical trials are currently taking place in medical centers and doctors’ offices in the US and Europe.

Benefits
Taking part in a clinical trial can have many benefits. For example, you may gain access to new treatments before they’re widely available. If a new treatment is proven to work and you’re in the group getting it, you might be among the first to benefit. You also will have the support of a team of health care providers, who will likely monitor your health closely.

Help others
Even if you don’t directly benefit from the results of the clinical trial you take part in, the information gathered can help others and add to scientific knowledge. People who take part in clinical trials are vital to the process of improving medical care. Many people volunteer because they want to help others.

Clinical trials do have risks and some downsides, such as the following.

  • The new strategies and treatments being studied aren’t always better than current standard care.
  • Even if a new approach benefits some participants, it may not work for you.
  • A new treatment may have side effects or risks that doctors don’t know about or expect.
  • Health insurance and health care providers don’t always cover all patient care costs for clinical trials. If you’re thinking about taking part in a clinical trial, find out ahead of time about costs and coverage for the treatment and travel to the medical center.

You should learn about the risks and benefits of any clinical trial before you agree to take part in the trial. Talk with your doctor about specific trials you’re interested in. (For a list of ECD trials, see: here.)

Once the FDA is satisfied that the drug works and is safe, it and the maker of the drug create a report, called the drug label. This report provides very specific information about the drug. The FDA approves the report (label), which provides necessary information to health professionals who prescribe or sell the drug.

Off-Label Use

When a drug is used in a way that is different from that described in the FDA-approved drug label, it’s said to be an “off-label” use. This can mean that the drug is:

  • Used for a different disease or medical condition
  • Given in a different way (such as by a different route)
  • Given in a different dose than in the approved label

Currently, all drugs used to treat ECD are either used off-label or as part of a clinical trial. Off-label is also called non-approved or unapproved use of a drug. The off-label use of FDA-approved drugs is legal in the United States and many other countries. Off-label drug use is common in the treatment of rare diseases.

The biggest problem with off-label drug use is obtaining approval from a payer (insurance company) to reimburse for the off-label drug use. Many insurance companies will not pay for an expensive drug that’s used in a way that’s not listed in the approved drug label. They do this on the grounds that its use is “experimental” or “investigative.” This financial burden can be an enormous strain on patients and families, especially when the cost of off-label treatments runs very high.

In cancer treatment, these issues have been largely addressed through 1993 federal legislation that requires insurance to cover medically appropriate cancer therapies. This law includes off-label uses if the treatment has been tested in careful research studies and written up in well-respected drug reference books or medical journals. In 2008, Medicare rules were changed to cover more off-label uses of cancer treatment drugs.

Still, the health insurance coverage laws and regulations are complex. If your doctor is thinking about off-label drug use, you and your doctor should carefully check your health plan’s coverage. If you are denied coverage, it might help if the doctor sends the insurer copies of peer-reviewed journal articles or other respected sources that support the off-label use. The ECD Global Alliance is happy to help medical teams with this. (Please see our page Filing Payer Appeals.)

Last updated: December 3, 2015

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