Criteria for Consideration of Access

Medicine Must be in Active Clinical Development

The investigational medicine must be part of an active clinical development program (Genentech must be currently studying the medicine 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 FDA approval of the medicine or otherwise compromise the potential development of the investigational medicine.

Additionally, patients must not be eligible (i.e. do not qualify) for ongoing (or soon opening) clinical trials of the investigational medicine. Geographic limitations to participation in a clinical trial would generally not meet this criterion.

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.

Dosing

As applicable, there is sufficient clinical data to identify an appropriate dose (amount and frequency of the medicine given).

Expanded Access Program

For the patient

The program is 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.

For the investigational medicine

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.

Compassionate Use

For the patient

The patient has:

• A serious, life-threatening illness
• Exhausted all available therapies typically used to treat the disease and is no longer responsive to, or able to tolerate, these treatments
• No other viable therapy options, including participation in ongoing relevant clinical trials
• The request to Genentech for access to the investigational medicine comes from the patient's qualified doctor

For the investigational medicine

• Genentech must have adequate supply of the investigational medicine.
• In the United States, the FDA and the Institutional Review Board (IRB) (ethics committee that approves and monitors clinical trials involving humans) at the patient's treating hospital or clinic must review and approve the use of the medicine, in the patient, before Genentech can provide it.