Every medication or medical device used today underwent extensive testing to ensure its safety and efficacy. The United States set the standard for clinical trials more than 60 years ago, and the process has not changed much since, except to become more complex and lengthy. It can take a decade or more for a drug candidate to reach the clinic, which for many people is too long to wait.
Fortunately, over the past decade, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have taken steps to expedite certain aspects of clinical trials to more quickly deliver important drugs to people in need. In the US, the expanded access pathway (also called “passionate use”) gives people with life-threatening illness early access to drugs that have not finished clinical testing and, in turn, have not yet been approved by the FDA. The EMA supports expanded access and the programs are operated independently by each member of the European Union.
The Conditions for Compassionate Use
Since compassionate use programs involve giving people drugs that have are not proven safe and effective, the FDA always prefers that people participate in a clinical trial first. The FDA and other regulatory agencies take careful steps to make sure they are not putting patients under undue risk. The FDA only qualifies drugs for expanded access available if:
- The people who would take it have a immediately life-threatening illness and there are no comparable or effective treatments already available to them
- These people cannot enroll in a clinical trial, whether it is because no trial is available or the are not eligible for any trials
- The potential benefits of taking the drug outweigh the potential risks
- Giving the drug to people will not interfere with an ongoing clinical trial
Expanded access comes in many forms. In some cases, the program is designed to bridge the gap between the end of a clinical trial and marketing approval for a large group of people. In other cases, the pathway is used for a smaller group of people who need a drug that is not actively being developed for clinical use. Finally, a doctor may get permission to give an individual an investigational drug in an emergency setting.
Expanded Access for People with ALS/MND
ALS/MND’s status as a severe, life-threatening disease with no effective treatments might qualify certain experimental ALS/MND drugs for expanded access programs.
For example, a drug normally undergoes three clinical trial phases in progressively larger groups of people before it is approved for clinical use, but Amylyx is trying to get their ALS/MND drug, AMX0035, to people sooner. AMX0035 slows down nerve cell death. In a phase 2 trial, the drug slowed ALS progression, and in a three-year open-label trial, the drug reduced the death rate by 50 percent. The company is going to apply to enter the drug into an expanded access program before it completes its phase 3 trial.
The Risks Associated with Expanded Access Programs
While the FDA and EMA fully support expanded access programs, they prefer that people enter clinical trials because clinical research offer people more protection if the treatment is unsafe or ineffective. Also, clinical trials are the best way to prove a medication’s safety and efficacy.
It’s important to remember that drugs made available as part of an expanded access program have not been approved for use in the clinic — researchers have not yet proven that it is safe and effective. These drugs make produce unexpected side effects. It is important to discuss the risks with your doctor.
Learn more about expanded access from the FDA here.