Expanded Access Treatment Options: Q&S with Johnson and Johnson Innovative Medicine’s Christine MacCracken

Christine MacCracken
Executive director of patient
strategies & solutions
Johnson & Johnson Innovative Medicine

Johnson & Johnson Innovative Medicine developed a guide to bring clarity around expanded access for terminally ill patients who have run out of treatment options and aren’t eligible for clinical trials. The Guide to Expanded Access was created based on requests from patients and patient representatives. Christine MacCracken, executive director of patient strategies & solutions and leader of the team that developed the guide, spoke with Pharmaceutical Executive about the guide and broader issues surrounding expanded access.

Pharmaceutical Executive: How difficult is it for patients to access expanded access treatment options?
Christine McCracken: One of the challenges that patients encounter, which can hinder their access to investigational medicines outside of clinical trials, is a lack of clear and transparent information. Expanded access varies by country, so it can be difficult for patients, caregivers, and physicians to navigate, especially without resources. Every country has a Health Authority with different requirements and processes for its expanded access programs. Understanding if expanded access is available in your country, the eligibility requirements, and the request process are all important when determining if it’s a potential treatment option. A lack of easily available information is a major obstacle for those exploring expanded access, making a resource like the Johnson & Johnson (J&J) Guide to Expanded Access vital.

PE: How many patients are in need of expanded access?
McCracken: This is difficult to assess for a variety of factors. However, any patient who is seriously or terminally ill, has a disease which has exhausted all available treatment options, and is not eligible for a clinical trial, is a potential candidate for expanded access. These programs are designed for this subset of patients who are unable to wait for the medicine to be available and are intended to be a bridge to when the medicine is available to them.

A common challenge for patients and their treating physicians is that expanded access is a just-in-time need during an already highly stressful time; therefore, access to readily available and transparent information is critical.

Because expanded access is an important potential option for patients, J&J built our expanded access program upon a bioethical framework that helps ensure we approach each request consistently and equitably. In 2015, we established two significant items which laid internal and external foundations to support those in need. First, we codified a policy and standard operating procedures to support the JNJ Innovative Medicines organization in considering expanded access as part of the development continuum. Second, in collaboration with New York University, we developed a first-of-its-kind committee to support our decision making, known as the Compassionate Use Advisory Committee (CompAC). We continue to evolve this external panel of ethicists, physicians, and patient representatives to support both development of our expanded access strategies as well as single patient requests. Since 2015, J&J has had the privilege of making investigational medicines available to more than 10,000 patients around the world.

PE: What led to the creation of the Guide to Expanded Access?
McCracken: In short, patient requests for information drove this effort. Information can be particularly difficult to find and, if available, may not always be in a format friendly to those not familiar with this option.And sometimes, even for those of us who are educated in this area, the requirements may not always be obvious. The Guide to Expanded Access is a comprehensive educational resource that was co-created with patient representatives, providing information in a people-friendly format for not only patients, but also their caregivers and treating physicians.

Anyone with a serious illness who has been through several lines of treatment and/or procedures can face many obstacles throughout the course of their treatment, but a lack of information should never be one of them. The power of having the right resources and information can’t be underestimated – something I learned as a nurse and firmly believe to this day.

As I mentioned earlier, expanded access processes vary by country, which can be very challenging to understand and navigate. The original Guide to Expanded Access, a first-of-its-kind resource released in 2021, was inspired by patient calls for greater clarity and resources and was designed to address this significant information gap. It was also important that this be geographically focused and company agnostic.

Now, three years later, we’ve released a revamped version after receiving positive feedback from advocates and patients alike. Patients not only led to the creation of this guide–their insights and experiences helped to bring it to life.

PE: What are the new elements in the updated version for 2024?
MacCracken: The revamped Guide to Expanded Access builds upon the original, maintaining key features while adding new ones to further support patients.

The guide now includes country-specific expanded access information for the United States, South Korea, and Switzerland, increasing the total countries covered to 30. Guided by patient and advocate feedback, we’ve also enhanced the language throughout for greater clarity.

Like the initial version, the updated guide is designed for easy navigation. It includes a table of contents map with all featured countries identified and linked to their respective pages, enabling patients to quickly locate country-specific information. Once on their desired country page, patients can find details spanning expanded access requirements, the request process, and more.

To increase patient understanding of the information presented, key terms are highlighted in red and linked to a glossary at the end of the guide. Links to external resources, such as a country’s regulatory agency site, are also included throughout.

The Guide is available for anyone to view at https://www.janssen.com/sites/www_janssen_com/files/guide_to_ea_june_2024.pdf.