Compassionate Use and Expanded Access Programs
The term "Compassionate Use" is used by medical professionals to refer to the treatment of a patient with a serious or life-threatening illness with an investigational (unlicensed) medicine. The term is used most commonly when a physician is requesting access for a single patient outside of other company-sponsored programs, like clinical trials, post-trial use protocols, or expanded access programs. The U.S. Food and Drug Administration (FDA) describes compassionate use as "a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials, when no comparable or satisfactory alternative therapy options are available." The FDA programs for "compassionate use" are administered under regulations for 'expanded access".
Requesting Compassionate Use
A patient and their medical treatment team may discuss various treatment options, including a clinical trial. Participation in a clinical trial may be one option for an individual with a serious medical condition. When a clinical trial is not an option, either because a patient does not qualify or perhaps there is no clinical trial, a physician may be able to request access to an investigational drug through a process called expanded access in the U.S.. Other countries us different terminology. It's important to know that only a treating physician can submit a request for investigational drugs on behalf of a patient. Not all physicians are willing or able to take on the responsibilities for early access. So the first step is for a patient and their physician is to have a candid conversation about their willingness to make a request for compassionate use.
What's the Process?
The first step is for the physician to contact the pharmaceutical company that makes the investigational drug. One source to identify the right contact is to review the Reagan-Udall foundation for the FDA "Navigator" website, finding the company on the Company Directory. If a pharmaceutical company has an active compassionate use program it is likely to be listed on the Navigator Directory. There will be company contact information. Sometimes there are direct links to company programs, with specific instructions and in some cases forms to use for the request process. Another place to check for existing Expanded Access Programs is on ClinicalTrials.gov and type "expanded access" into the "other terms" field. When a pharmaceutical company agrees to reviewng the request, and if drug is available, they will ask the requesting physician to provide patient information to acertain eligibility for the drug. This may include information about medical history, a list of prior treatments and outcomes, and a proposed treatment plan. If the company agrees to the request they will provide the physician with a Letter of Authorization (LOA).
The next step is for the physician to obtain authorization from the FDA. Submitting a request has never been simpler. Along with the simplified FDA Form 3926, physicians may now submit the request electronically using the eRequest process. In the eRequest, the physician will need to know the FDA Center that should review the request. The FDA review will entail a review of available clinical trials and eligibility, reviewing physician CV and patient oversight, and the LOA. Following FDA approval, in the form of an email to the requesting physician, the physician forward the approval documentation to the pharmaceutical manufacturer. IRB approval will be required prior to patient treatment. The physician should request a template informed consent form (ICF) from the pharmaceutical manufacturer for adaptation to the institution-specific ICF. The pharmaceutical company will arrange for shipment of the unlicensed drug to the physician's institution in the most expeditious manner possible.
How Early Access Care Can Help Pharmaceutical Companies
Early Access Care can help pharmaceutical companies navigate this landscape with a best strategy for the receipt and management of expanded access / compassionate use programs. Contact us (email@example.com) to assist your program. This journey doesn't need to remain mysterious nor burden your development teams with the demands of expanded access / compassionate use.