If a patient is unable to gain access to an unapproved or investigational medicine through a clinical trial, they may be able to gain access to this medicine via an early access program, if this is available. Early access programs can include expanded access programs (EAP) or a compassionate use (CU) program.
Learn more about the criteria needed in order to request access to a Genentech investigational medicine through an EAP.
Medicine Must be in Active Clinical Development
The Genentech medicine must be part of an active clinical development program and being studied in patients. Once the FDA approves a medicine, neither existing expanded access programs nor compassionate use are available.
Granting Access Must Not Interfere with Clinical Trials and Potential Approval
Granting access to an investigational medicine must not interfere with the completion of important clinical trials that could support the FDA approval of the medicine or otherwise compromise the potential development of the investigational medicine.
As applicable, there is sufficient clinical data to identify an appropriate dose (amount and frequency of the medicine given).
Investigational Medicine Criteria
Genentech must consider the risk-benefit to be robust and persuasive and reasonably likely to be supported by the FDA.
Based on discussions with the FDA, Genentech must have reason to believe the FDA is likely to approve the medicine for use in this population of patients.
Expanded Access Programs allow a limited group of patients, who meet specific criteria, to have access to a specific investigational medicine. EAPs are studies managed by the company and follow a specific protocol, which is developed in consultation with the FDA.
Patient Seeking Access Must Not be Eligible for Existing Clinical Trials
Patients seeking to join an EAP must not qualify for any ongoing or upcoming clinical trials of the investigational medicine. Geographic limitations to participation in a clinical trial would generally not meet this criterion.
However, EAPs are limited to groups of patients who generally have the same stage, extent and type of disease or medical condition that was evaluated in the key study/studies that will be submitted to the FDA in support of an application for approval of the medicine.
A Potential Risk-Benefit Assessment for Patients
The potential benefit to the patient seeking access to the investigational medicine must always be considered to outweigh the collective potential risks to the patient of offering the medicine, including the outcome of the disease itself.
Additionally, patients with underlying medical conditions that may pose safety risks that have not been sufficiently characterized/studied would not be eligible to participate.
If an expanded access program is open, and if you meet the protocol inclusion criteria, you should work with your doctor to enter that protocol.
If you are seeking access to an investigational medicine via CU, follow these steps:
The request for access to a Genentech investigational medicine can only be considered if the patient's qualified treating doctor makes a formal request and is committed to, and supportive of, the requested treatment. We anticipate that it will take no longer than 3 business days to acknowledge receipt of the request.
Genentech is committed to a fair and impartial evaluation of each request for access to our medicines. Therefore, all decisions are based solely on clinical circumstances and are guided by the principles outlined above.
Whenever possible, patients will be referred to ongoing clinical trials as the primary way to access investigational medicines.
Decisions regarding potential access to investigational or unapproved medicines can only be made, in certain circumstances, after in-depth discussions between Genentech's clinical teams and the patient's qualified treating doctor acting on his or her behalf.
As described by the FDA, EAPs and CU are only available for specific patients with no other available treatment options, including clinical trials, and are not without risks.
These programs only apply to medicines not yet approved by the FDA. A doctor must decide whether the potential benefit outweighs the risk of receiving an investigational or unapproved medicine, based on the individual patient's medical history.
Additionally, the FDA requires Institutional Review Board (IRB) (ethics committee that approves and monitors clinical trials involving humans) at the patient's treating hospital or clinic to review and approve the use of the medicine before a company can provide it.