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ALS Worldwide
October 05, 2015

Expanded Access to Experimental Drugs

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“What if I’m not eligible for a clinical trial?” is one of the most often-asked questions from ALS/MND patients and their loved ones. Patients may be interested in a certain treatment that’s only available through a clinical trial, but they don’t meet the eligibility criteria outlined for the studies. 

A patient’s doctor can ask the study sponsor for an eligibility waiver or special exception to allow you into the study, even though you don’t meet all of the criteria. This decision is made by the study’s clinical investigator, who may consult with others involved in the study about the request. If entered in the study, the person is treated according to study protocol (the same tests, doctor’s visits, follow-up, etc.), but results from that person are not included in the final study results. 

Whenever possible, use of an investigational drug or device by a patient as part of a clinical trial is preferable because clinical trials generate data that may lead to the approval of products and, consequently, to wider availability for the benefit of the ALS/MND community. However, when patient enrollment in a clinical trial is not possible (a patient is not eligible for any ongoing clinical trials or there are no ongoing clinical trials), patients may be able to receive the drug or device, when appropriate, through the U.S. Food and Drug Administration’s (FDA) Expanded Access Program, also called "Compassionate Use”.

In recent years, FDA has dramatically broadened these programs to allow more patients who urgently need these treatments to be able to get them.  To learn more, please visit the FDA's website.

Most people are likely eligible for some type of study. This is true even if they’ve had many different treatments already. Of course, not all studies for which you are eligible are a good fit. It’s always important to understand the purpose of the study and have a good idea of the possible risks and benefits. Talk with your neurologist or other healthcare provider to see if a specific investigational drug for your ALS/MND treatment is right for you.

Remember that the company or organization developing the drugs will need to provide the medicine. Without their cooperation, you will not be able to access the investigational product. Visit clinicaltrials.gov, a registry and results database, to find publicly and privately supported clinical studies focusing on ALS/MND conducted around the world.

If you still have questions unanswered or just want to understand the process more, please contact the FDA Office of Health and Constituent Affairs at 301-796-8460 or [email protected] or for quick, personalized support, email: [email protected]