Expanded Access Programs

Last week, at the 2022 Annual Meeting of the American Society for Clinical Oncology a question we were commonly asked was to define an Expanded Access Programs (EAP) and compare and contrast with a compassionate use program (CUP). An EAP allows physicians and patients to access investigational medicines outside of a clinical trial setting. The term EAP is often used interchangably with 'compassionate use" or early access program. Globally, the details surrounding the availability of medicines outside of a clinical trial will vary by region. Local laws and regulations drive specific details, however in all, these types of programs are for patients diagnosed with a serious or life-threatening condition where standard of care treatment options have failed, or the patient is intolerant, or there is no available treatment options.

In all cases, EAPs ofter treatment options for the patient and physician, as an approved mechanism to obtain a promising pre-approval drug. Patients who wish to access the investigational drug must have a physician willing to act on their behalf. Physician's are responsible for:

• Assessing available treatment options in the country where the patient resides
• Determine clinical justification for the use of the investigational drug
• Complete required paperwork with the relevant regulatory agency (e.g. FDA, MHRA, etc.) to provide approvals to import the product
• Contact relevant local ethics committee (or Investigational Research Board, IRB) to gain approval to use the investigational drug to treat the patient
• Ensure local requirements for privacy
• Obtain informed consent for the use of the medicine while still investigational
• Contact the Sponsor/Manufacturer to assess willingness to provide investigational drug
• Oversee the patient's treatment, and is responsible for patient care and reporting

All manufacturers with an investigational drug in phase 2 or 3 clinical trials in the U.S. are required to have an expanded access policy on their website, but these may be difficult to find. In the U.S., one good source to identify the procedure a manufacturer willing to provide drug through an EAP can be found on the Reagan-Udall Expanded Access Navitator. The Navigator provides manufacturer links and contact information. In the EU, a good source to identify whether a drug is available for compassionate use is via the EU Clinical Trials Register.

Sponsor / Manufacturers

EAPs offer a regulatory approved program that allows a sponsor/manufacturer to make their product available while investigational outside of clinical trials. There are benefits to offering innovative therapies via EAPs:

• Provide physicians with experience using the new medicine, becoming familiar with proper dosing and safety monitoring at an earlier timepoint
• Building relationships with physicians at an earlier timepoint than pre-launch
• Manage risk in the pre-launch setting with appropriate adverse event data collection, including Adverse Events of Special Interest
• Collect real-world data (RWD) which can inform how the drug will be used in clinical practice
• Access for oncology patients with serious or life-threatening diseases who have no other alternatives and are willing to accept greater risk
• Patient autonomy over healthcare decisions
• Bridge the gap between the latter stages of investigational development and approval by making a drug widely available during that period
• Submit annual report on patient status to the FDA or other Health Authority

Early Access Care can help you navigate this landscape and mature a best strategy for EAP and CUP programs. Contact us (anne.cropp@earlyaccesscare.com) to assist your program. This journey doesn’t need to remain mysterious and there is plenty of opportunity.