FYI: Compassionate Use Programs


The process for receiving compassionate use therapies is often very long, confusing and frustrating.  Three questions often presented to The ALS Association are:

  1. “What is compassionate use?”
  2. “How can I get access to a drug through compassionate use?”
  3. “Does The ALS Association support compassionate use?”

There is a significant amount of information on the subject of “compassionate use” through government websites, company-specific home pages and association links.  One of the most inclusive sites discussing the issues surrounding compassionate use is the Food and Drug Administration’s (FDA).  Included in this educational piece are excerpts from documents of the FDA and testimony from Robert J. Temple, M.D., Associate Director for Center for Drug Evaluation and Research Food and Drug Administration. The goal of this document is to explain “compassionate use” guiding principles and requirements.

What is compassionate use?

"Compassionate use," officially known as expanded access, is the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition which has no comparable or satisfactory alternative treatment options.

For a patient to receive an experimental drug through a compassionate use program, the patient’s doctor must contact the drug company and then submit an application to the FDA.  For the FDA to consider the request, the patient must meet certain criteria:

  • The disease is serious or immediately life-threatening.
  • No treatment is available or the patient hasn't been helped by approved treatments for the disease.
  • The patient is not eligible for clinical trials of the experimental drug.
  • The physician agrees that the patient has no other options and may benefit from the experimental treatment.
  • The company that makes the drug agrees to provide it to the patient.

FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial.  They also permit expanded access for large groups of patients who do not have other treatment options available, once more is known about the safety and potential effectiveness of a drug from ongoing or completed clinical trials.

Just as in clinical trials, these investigational drugs have not yet been approved by the FDA as safe and effective for all recipients. They may be effective in the treatment of a condition, or they may not. They also may have unexpected serious side effects.  It is important for people to consider the possible risks if they are interested in seeking access to an investigational drug.

What is the FDA’s role in compassionate use?

“Regarding Compassionate Use, it's a common misperception in the ALS community that the FDA decides who gets compassionate use exceptions in totality. That is only part true. The first thing that needs to occur is the drug company has to APPLY [make application to the FDA].”  Greg Merfeld, 2010 (ALS patient)

The FDA cannot compel a company to supply an individual patient with an investigational drug outside of its planned clinical trials. The manufacturer or sponsor makes the final decision to provide an experimental drug or therapy to a patient. The sponsor may consider many factors, including the amount of information available about the drug, the amount of drug available, and how best to use its resources to optimize development of the drug for marketing.  This maximizes the availability of the drug to patients who can benefit from it.  In some cases, the sponsor is unwilling to provide the product outside of clinical trials, especially relatively early in drug development. Patients are sometimes confused or angered by this situation and misinterpret the company's unwillingness to provide the product as an FDA action.

The FDA may decide not to allow compassionate use treatment because of safety concerns. Generally, however, if a physician makes a request for treatment of an experimental drug with a patient for whom no effective therapy exists while there is an ongoing study of the drug and a sponsor agrees to provide the product, FDA does not typically object to compassionate use treatment.

According to Dr. Temple, “The FDA believes that it is appropriate to make certain promising, but not-yet-approved, products available to patients with serious and life-threatening illnesses who lack alternative treatment. This should be done in a way that does not interfere with recruitment to the clinical trials needed to support the effectiveness and safety of the drug.  It should also be done fairly.”

Can any individual patient qualify for expanded access to any drug?

No.  In order for a patient to gain access to an investigational drug outside of a clinical trial, the patient must have a serious or immediately life-threatening disease or condition and no comparable or satisfactory therapeutic alternatives. Additionally, the drug manufacturer and the patient’s doctor must make special arrangements to obtain the drug for the patient. These arrangements must be authorized by the FDA. These safeguards are in place to avoid exposing patients to unnecessary risks.

What are some of the challenges in developing and accessing compassionate use programs?

  • Manufacturers may not always be willing or able to provide access to a drug outside of their clinical trials.  Companies are not required to make their drug available through expanded access, or to make more of a drug for that purpose. Companies manufacture investigational drug(s) for the purpose of testing them in clinical trials, since that is the most effective and efficient way to determine whether the drug(s) work, and whether they are safe to use.
  • Sometimes, even when an expanded access program has been established, there may not be enough of a drug available for all patients requesting access.  Some companies establish lotteries to determine which patients will have treatment access, while others make the determination on a case-by-case basis.
  • Physicians may not always be able to seek expanded access for patients, depending on a patient’s medical history and the risks associated with taking an investigational drug. The physician must determine that the probable risk from the drug is not greater than the probable risk from the disease. Not all physicians are willing to manage the use of an investigational drug for patients in their care.
  • There may be competition between expanded access programs and the regulatory programs that will lead to drug approval. Competition can be either for patients entering trials or for available company resources. There is significant concern that availability of all investigational drugs outside a formal protocol will decrease participation in the formal study. In fact, FDA rules allow expanded access programs to be put on hold if they are interfering with the conduct of clinical trials.
  • The use of an investigational drug in less controlled setting of expanded access programs, especially in patients with very advanced disease, could lead to adverse reactions that might raise difficult to resolve, but spurious, safety concerns about the drug.

Are there costs associated with expanded access?

Investigational drugs may be expensive to manufacture. Some companies provide the drug for free to patients. Other companies charge patients costs associated with the manufacture of the drug. Most insurance companies will not pay for access to an investigational drug. In addition, there may be additional costs associated with administration and monitoring of the investigational drug by healthcare professionals.

Does The ALS Association support compassionate use?

In our quest to create a world without ALS, The ALS Association strongly believes that people with ALS should have access to effective treatments as quickly as possible.  Our primary core value is that people with ALS and their families come first in everything we do.  When it comes to advancing therapies for ALS, we work with all stakeholders in an effort to find the best path forward.

“The ALS Association is committed to bringing treatments from the bench to the bedside as soon as possible,” said ALS Association Chief Scientist Lucie Bruijn.  “In the meantime, it is absolutely critical that people with ALS continue to participate in clinical trials.  Ultimately, patient enrollment and participation in clinical trials is the only way to determine the effectiveness of treatments for this devastating disease.”

We will continue to work closely with academic institutions and pharmaceutical companies, who are developing potential treatments for ALS, to facilitate clinical trials and accelerate the availability of treatments, including through compassionate use programs.  We are, however, cognizant not to undermine the science that could potentially produce drug(s) approved to treat ALS.  We must not let our push to make a treatment available to all result in it not being available to anyone. 

How can I find out whether I can access a particular investigational drug outside of a clinical trial?

The ALS Association’s website,, has a tremendous amount of information for the ALS communities.  More specifically, the website houses an in-depth listing of research activities, which can be located on

Some companies have established expanded access programs. People can visit the website to view a list of expanded access studies or search for specific expanded access programs.  In addition, a patient or their healthcare provider can call a drug company directly to inquire about their policies.

Patient advocacy groups can sometimes help patients find and explore expanded access options.

If a patient is interested in seeking expanded access to a particular drug, they should talk with their healthcare professional to see if it might be a reasonable option.

Additional information for healthcare professionals can be found at Physician Request for an Individual Patient IND for Compassionate or Emergency Use.