Compassionate Use for Investigational Drugs in Expanded Access Programs

The Reagan-Udall Foundation for the FDA (RUF), a non-profit organization created to help the FDA accomplish its mission, is in the process of developing an online Navigator to help patients and physicians understand the processes and programs associated with expanded access (EA).

What is “Expanded Access”?

According to the FDA, "expanded access, sometimes called ‘compassionate use,’ is the use outside of a "well-controlled" clinical trial of an investigational medical product (i.e., one that has not been approved by FDA)." Almost all compassionate use requests that FDA receives are approved. However, the difficulty is not receiving approval. Rather, many patients run into trouble with the fact that the patient’s treating physician and the drug maker must agree to proceed with a compassionate use application. Because this sometimes creates an issue for those seeking early access, many patient advocates are asking “the FDA to make it easier for terminally ill patients to access potentially helpful new drugs,” says an article from BioPharma Dive.

Proposed EA Navigator

In order to solve this issue, RUF proposes to develop an online navigator, which would provide details regarding FDA policies and points of contact, as well as additional information concerning the compassionate use request process.

Navigator Scope and Functions

According to RUF’s project proposal, the Navigator will provide educational content, including information on:

• Individual patient access to drugs and biologics--- where there is no established Expanded Access Program--- and the steps involved in making a single patient IND request.
• The roles of FDA, treating physicians, manufacturers and IRBs in the process.
• How healthcare providers can perform required reporting on safety and efficacy outcomes as well as the importance of conducting such reporting.
• How EA fits into the drug development, clinical trial, and FDA approval processes.
• The principles of EA eligibility criteria and risk-based decision-making.
• Challenges of translating early research findings into clinical outcomes in real-world patients.
• Potential resources for healthcare providers who do not have ready access to an IRB through a hospital or research affiliation.
• Directory of information on FDA and manufacturer’s policies, procedures, and points of contact.

RUF notes that its Navigator will not address ethics, policy, or advocacy.  Furthermore, it will have no effect on decisions regarding eligibility and access.

Goals and Outcomes

In creating the Navigator, RUF’s goal is to increase the transparency and accessibility of information, thus increasing the overall understanding of compassionate use programs. RUF explicitly states that its goal is not to encourage or discourage people from requesting individual patient access. “By increasing the understanding of EA and the individual patient access request process, RUF hopes to achieve the following outcomes:

• Increased health literacy on EA, specifically individual expanded access (single patient INDs)
• Greater equity among patients and providers in accessing understandable information on EA
• Improved patient and healthcare provider experience
• More informed decision-making about EA and its risks/benefits
• Increased efficiency of the request process, reducing the resources needed by the FDA and manufacturers to educate patients and healthcare providers”

Public Workshop

On Monday, May 16^th, the organization held a public workshop in which it sought feedback on its proposed informational database.  The workshop was intended for patients, patient advocacy groups, healthcare providers, drug manufacturers, Institutional Review Boards (IRBs), and representatives from academia. In holding this workshop, RUF’s goal was “to discuss how best to disseminate information on the EA request process.”

For additional information, visit RUF’s website.

Are you seeking approval of a compassionate use request? We can help with this or any other type of FDA submission. To learn more about our services and how we can help you obtain FDA approval, contact us today.