What is the FDA's Compassionate Use program?

11,September 2018

The FDA has a Compassionate Drug Use program that is aimed at facilitating the administration of lifesaving drugs to patients in need of them by sidestepping the process of drug approval. In other words, when a patient is in a critical, life-threatening condition and there is no established or approved drug or therapy available in the market for it, the FDA can approve the administration of these tested, but unapproved drugs even if they have not gone through the usual approval process.

The reason such drugs are approved for administration is that the patient’s condition may not allow the usual process to take place, whereby only approved drugs are given. The FDA makes it possible for such unapproved drugs to be administered if it is convinced that the potential benefits of the drug, even it has not gone through the usual approval process, can overweigh the risks of trying an unapproved drug. Naturally, many factors have to be considered when the FDA uses the Compassionate Use Program:

The patient is in a critical condition and this is threatening her life
The regular, available medications are either not available or are not working
The patient’s condition prevents her from enrolling for a clinical trial and be a subject in this trial
This treatment will not interfere with or alter the course of the medication the patient already is under

The justification for the Compassionate Use Program

One grouse against the Compassionate Use Program relates to whether it is legal to administer a drug that has been only tested, but not yet approved. No law prevents its use, but the FDA has put in place a strict rider that only in exceptional cases it can skip the detailed and lengthy process of approval for the drug before administering it on the patient.

This is why such drugs are not allowed in normal patients. It is only in truly extraordinary cases, when it is completely convinced that the drug suited for the patient can possibly alleviate the condition, that too when all other options are foreclosed, that the FDA goes ahead in approving the Compassionate Use Program.

The process

The FDA prescribes a set of two processes through which the unapproved drug can be used on the patient:

Through the Expanded Access Program (EAP)
Through the single patient access process

Through the EAP

Through the Expanded Access Program, when the company that is sponsoring the clinical trial for the drug is at a late stage of the research and receives the application from the patient’s doctor, it can make this drug available to the patient under what is called the Expanded Access Program. The doctor should make an application to this effect with the drug company and should convince it of the possibility of improvement in the patient’s condition if the drug is given. The doctor should make out a case for the EAP and should comply with all the procedures that guarantee that the process will not be misused.

Through the single patient access process

This process is taken up for patients that don’t qualify for either the clinical trial or the EAP if it exists in the case of the drug the patient needs.

These steps need to be adhered to in case the single patient access process is being used:

The doctor must first seek the drug manufacturer’s permission to use the drug on the patient and enquire about its willingness to supply it
Once the permission is obtained, both the doctor and the company have to work together to request the FDA to approve the drug for use for just one single patient. The FDA requires the reason for which the request is being made, the treatment plan, and a consent form signed by the patient

Challenges relating to the Compassionate Use Program

The noble intention behind the principle of the Compassionate Use Program notwithstanding, there are a few challenges that exist with its implementation.

The most important challenge relates to the availability of the drug. Although the company could be developing the specific drug, it may not be in a position to guarantee its availability at the time at which the request comes to it. It may not be able to assure the efficacy of the drug considering the stage of its development at that time
Fixing the dosage is another challenge with the Compassionate Use Program. It is usually only approved drugs whose dosage is known. In the case of unapproved drugs, fixing the dosage could be quite a task
The process itself is a challenge. Even if the company is convinced of the need to administer the Compassionate Use Program, its approval process could be lengthy and bureaucratic. The FDA usually approves the Compassionate Use Program within 24 hours in cases of extreme urgency, but the pharma company, usually privately run and for-profit businesses, are not obliged to. If the company refuses to give the unapproved drug for treatment, there is not much anyone else, be it the patient or the doctor, or the FDA, can do about it
The process can drag many a time, during which time the patient’s condition may have further weakened. Drug companies may not necessarily be inhuman or heartless in denying access but could be up against their own hurdles. They may not have the sufficient quantities needed for the patient
Another issue with the Compassionate Use Program is that its procedure may be confusing. Different points in the system, such as the FDA, the doctor, or the company could be using different terms for the same item many times, compounding the confusion
The pharma company could also have another important challenge: it may find it impractical and expensive to part with this micro dosage for the sake of one or two patients from its sample of drugs
The doctor has to be completely aware of the drugs being developed at clinical trials all over the country to suggest the right one. Absence of this knowledge may prevent the Compassionate Use Program from taking off at all, even if it could eventually have made a difference to the patient if administered