Compassionate Use Backgrounder

What is “compassionate use”?
Medical professionals use the term “compassionate use” to refer to the treatment of a seriously ill patient with an experimental medicine or product when there are no other treatments available outside of a clinical trial.¹ Other terms used to refer to compassionate use include “expanded access” or “pre-approval access.”

The U.S. Food and Drug Administration (FDA) describes compassionate use as “a pathway for patients to gain access to investigational drugs, biologics and medical devices for serious diseases or conditions. Such investigational drugs/devices have not yet been approved by the FDA and they have not been proven to be safe and effective.” ²

Most commonly, compassionate use is the term used when a doctor is requesting access for a single patient outside of other company-sponsored programs, like clinical trials or an expanded access program. At Janssen, we use the term “Single Patient Request.”

How does it work?

For an individual patient to receive an investigational medicine or product in the U.S., his or her doctor must request it from both the company developing the therapy and the FDA. FDA regulations impose certain requirements for such requests to be granted.[1] For example, when reviewing such a request, FDA must determine that:

• The patient has a serious or immediately life-threatening disease or condition, and there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the patient’s disease or condition.

• The potential patient benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or condition to be treated.

• Providing the investigational drug for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the expanded access use or otherwise slow the pace of drug development.

• The patient cannot obtain the drug under another clinical trial or expanded access program.

The patient’s physician must also determine that the probable risk to the person from the investigational medicine is not greater than the probable risk from the patient’s disease or condition. Beyond these requirements of FDA regulation, companies developing therapies may consider other factors in considering whether to grant requests for individual access, such as the availability of supplies of the investigational medicine.

How can a patient request access?
In addition to the patient, there are three key people or groups involved in the compassionate use process: the patient’s doctor, the FDA, and the company investigating the medicine.

• The doctor must review all of the FDA requirements for expanded access to the investigational treatment with the patient and obtain informed consent. ³
  

• The doctor may need to get Letter of Authorization (LOA) from a representative of the company investigating the treatment. ⁴

• The doctor will need to complete an application with the FDA requesting access to the investigational medicine. ⁵ Including an LOA as part of this application allows the FDA to refer to the official investigational new drug (IND) application for that medicine when reviewing the doctor’s request.

For investigational medicines Janssen is developing, the best and fastest way to get information about whether or not a patient may be able to gain access is to have the treating physician contact Janssen Medical Information (in the U.S.: 1-800-Janssen or janssenmedinfo@its.jnj.com).

What are the ethical considerations for companies when they review compassionate use requests?
Ensuring patient safety is always the top priority for pharmaceutical companies and the FDA. There are a number of additional considerations beyond safety that pharmaceutical companies take into account, including:

• All treatments treated fairly and equally

• That we conduct thorough scientific studies to understand the risks and benefits of any investigational medicine. These studies provide the information needed to seek approval from government health authorities and bring new medicines to those who need them.

• That we are not putting patients at risk of unnecessary harm.

How is the FDA addressing compassionate use?
FDA statistics show that few requests for expanded access are rejected once they are sent to the agency. According to the FDA,⁶ more than 99 percent of expanded access requests have been approved since 2009.

The Agency recently announced draft guidance to streamline the process doctors must follow to request access to investigational medicines. ⁷ As part of this guidance, in February 2015, the FDA shared a simplified application form, entitled “Individual Patient Expanded Access Applications: Form FDA 3926.”

The goal of the simplified form is to accelerate the process by which a doctor can request permission from the FDA to treat a patient with an investigational medicine and the FDA estimates the time needed to complete the new form is an hour or less, compared to approximately 100 hours required to complete the current form.

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