- Clinical Trials: What You Need to Know
- Why do we need clinical trials?
- What happens before clinical trials?
- Some facts about clinical trials: Important points to keep in mind
- What are the phases of clinical trials?
- Phase 0 clinical trials: Exploring if and how a new drug may work
- Phase I clinical trials: Is the treatment safe?
- Phase II clinical trials: Does the treatment work?
- Phase III clinical trials: Is it better than what’s already available?
- Submission for FDA approval: New drug application (NDA)
- Phase IV clinical trials: What else do we need to know?
- Who sponsors and runs clinical trials?
- Should I think about taking part in a clinical trial?
- Answers to some common questions about clinical trials
- Other questions you should ask your research team
- What protects the study participants?
- What’s out there? Finding clinical trials
- How do I figure out which study is for me?
- What about cost? Will my insurance cover it?
- What would it be like to take part in a clinical trial?
- What if I’m not eligible for a clinical trial?
- Summing it all up
What if I’m not eligible for a clinical trial?
Although some people may be too ill or have other problems that do not allow them to take part in clinical trials, most people will probably be eligible for some type of study. This is true even if they’ve had many different treatments already. Of course, not all studies you are eligible for are a good fit for you. It’s always important to understand the purpose of the study and to have a good idea of the possible risks and benefits for you.
Clinical trials offer the best access to experimental treatments. Study protocols, which are written based on the results of studies done before, are strictly followed and patients are watched carefully.
Some people may be interested in a certain treatment that’s only available in clinical trials, but may not meet the eligibility criteria outlined for the studies. In some of these cases, a person’s doctor may ask the study sponsor if they can get an eligibility waiver or special exception to allow the person into the study, even though they do not strictly meet all of the criteria. This decision is usually made by the study’s clinical investigator, who sometimes consults with others involved in the study about the request. If entered in the study, the person is treated according to the study protocol (the same tests, doctor’s visits, follow-up, etc.), but the results from that person are not included in the final study results.
In other cases, the studies may have already enrolled enough people and aren’t taking more participants.
At times, there may be ways to get access to treatments that are in late phase clinical trials but not yet approved by the FDA. These are usually referred to as expanded access or compassionate use programs. In recent years the FDA has broadened these programs to allow some patients who urgently need these treatments to be able to get them. For more information, see our document, Compassionate Drug Use.
But it’s not always easy to get access to these treatments. The company making the treatment is not required to offer it for expanded access or compassionate use. Some companies may decide not to for various reasons (manufacturing issues, excess demand, etc.). Because of the amount of effort and paperwork involved, the process of trying to get an unapproved drug for compassionate use can be slow (weeks to months). And, not all doctors are willing to manage the use of an investigational drug for patients in their care.
Some of these programs are described below. All require your informed consent, much the same as for any clinical trial.
Treatment use of an investigational new drug (treatment IND)
In some cases, if a treatment is showing promise in late phase clinical trials, the maker may apply to the FDA for a treatment IND (investigational new drug) status. This is much like setting up a new study, but it’s meant mainly as a way for patients with no other options to be able to get the treatment before it’s approved.
This is sometimes done when a person would not have met the eligibility criteria for the clinical trials or when the studies are already closed to further enrollment. The patient must have a life-threatening or severely debilitating condition for which there are no other treatment options.
Your doctor would need to get in touch with the treatment manufacturer to see if such a program exists and what would be needed for you to enter it. As with clinical trials, these programs have to have a protocol (written guideline or plan) that meets FDA approval, as well as approval by an institutional review board (IRB) in many cases.
The supplier may or may not charge for the treatment in question. It’s important to find out beforehand whether you or your insurance company would pay for the treatment.
Single patient and emergency use of an investigational new drug
A single patient IND is used to get access to an unapproved treatment for one person with a serious condition who is not eligible for a clinical trial. It’s much like a treatment IND in some ways. It does not require that the clinical trial protocol be followed, but it requires that your doctor spell out in detail your case, previous treatments, the proposed treatment plan, and more.
To get a single patient IND, your doctor would need to contact the manufacturer of the treatment to see if they would supply it. He or she would then need to have the proposed treatment protocol approved by the IRB and the FDA before treatment would be allowed to begin.
An emergency IND can be used when there isn’t time to get approval from the IRB. Your doctor would need to contact the manufacturer to see if you can get the treatment, and then file the needed paperwork with the FDA. While IRB approval is not needed before starting treatment, the IRB would have to be told of the situation and would have to approve future uses.
Last Medical Review: 09/21/2012
Last Revised: 09/21/2012