Clinical trials have been conducted for decades, and, although people might not know the intricate ins and outs of how trials actually work, most people have at least heard of them. In comparison, Expanded Access, also known as Managed Access, Compassionate Use, Named Patient, etc. is a relatively new phenomenon and is much less familiar to, not only the general public, but also to the people involved in providing medication or caring for sick patients.
Both clinical trials and Expanded Access Programs (EAPs) provide investigational new drugs (INDs) to seriously sick patients who have exhausted all viable treatment options. But, they are not one and the same thing. As EAPs become more commonplace in the global healthcare system, it will be important for healthcare providers, patients, and the advocacy groups that represent patient interests, to be are aware of the difference between an EAP and a clinical trial.
The biggest difference between an EAP and a clinical trial is the purpose that they serve. A clinical trial is conducted by a drug manufacturer that wants to get its IND approved and licensed for use. In order to receive approval, the manufacturer has to prove to the national health regulatory body that the drug is safe to use, and that it is as good as, if not better than, current medication being used on the market. Clinical trials allow a manufacturer to use its drug, in a controlled and regulated setting, to treat patients in order to collect this necessary safety and efficacy data.
An EAP is different. Although an IND manufacturer can still collect safety and efficacy data from patients enrolled in an EAP, these programs are not established to help get a new drug onto the market. Instead, these programs are set up solely to provide much needed medication to where there is an unmet medical need. The drug manufacturer usually provides the drug through an EAP because it is the patient’s last hope, not because it has a lot to gain.
Ease of Access
Another big difference between an EAP and a clinical trial has to do with the ease of access to an IND that they provide. In general, it is easier to enroll in a clinical trial, and receive a drug that way. The process of gaining access to a drug through an EAP is usually a lot more complex.
If a clinical trial is being conducted, a patient’s physician can contact the IND manufacturer, and request to be enrolled in the trial. As long as the patient meets the enrollment requirements, and is located, or can relocate, close enough to the trial site, he/she will usually be enrolled.
Accessing a drug though an EAP, is not so simple. Firstly, in order to be enrolled in an EAP, a patient must have a physician willing to manage the access to, and administration of, the EAP drug. Furthermore, the patient must also be unable, for whatever reason, to enroll in a clinical trial; a patient cannot decide that he/she would rather receive a drug through an EAP instead of through a trial.
Secondly, it is important to note that, just because there is a clinical trial in place, does not mean there will be an EAP in place alongside it. These programs are administered by the drug manufacturer, and a manufacturer is never legally required to provide its IND outside the clinical trial. If a sponsor receives enough requests, or has a surplus of product, it will usually agree to set up an EAP. However, if the sponsor cannot afford to supply the drug outside the trial, or does not want to take focus away from receiving regulatory approval for its drug, it can refuse to provide the drug through an EAP.
And, thirdly, even if a sponsor does agree to provide the drug, it is still not certain that a patient will be able to access the drug. The patient’s physician first has to prove to the national regulatory body in their home country, that the patient has exhausted all viable licensed treatment options. Access to an IND through an EAP will not be provided to patients who have licensed options available to them.
Furthermore, the cost of paying for an IND can also prevent patients from enrolling in an EAP. Oftentimes, trial sponsors will set up an EAP, but will decide to charge the patient for receiving the drug. Because insurance companies often refuse to cover the cost for investigational medication, patients are left to pay out of their own pockets if they can afford it.