DDW Editor Reece Armstrong speaks to Christine MacCracken, Executive Director of Patient Strategies & Solutions at Johnson & Johnson Innovative Medicine, about expanded access strategies and the company’s decision to create a guide to help reduce barriers to investigational medicines for seriously or terminal ill patients.

RA: Why are expanded access pathways currently difficult to navigate? 

CM: This treatment pathway is needed at a time when someone is already dealing with a serious or terminal illness, they are not qualified for a clinical trial, and their disease has exhausted all available treatments.  

Adding to this, expanded access, also known as pre-approval access, is not routinely part of physician training, so it is essentially a ‘niche’ area of treatment that is not widely utilised on a routine basis.  

Generally, there are two main barriers: availability of the information and then interpreting it in each country. In some cases, even subject matter experts can face challenges, so imagine if you are a patient or physician new to this pathway.  

Access to information should not be an additional barrier, but unfortunately the question of whether expanded access is even allowed in a country may not always be readily available.

RA: Can expanded access strategies better inform how treatments can work and guide decision-making for seriously ill or terminal patients? 

CM: The purpose of expanded access is to be a bridge to when the medicine is hopefully approved and available, filling immediate unmet medical needs of patients. The patients who are potential candidates for expanded access have a just-in-time need and may not be able to wait for a medicine to be available.

Expanded access is a legal treatment pathway, but it is not research. This is an important distinction because clinical trials are the gold standard and primary way investigational medicines should be accessed by systematically collecting efficacy and safety data for submission to health authorities. 

RA: Given the varying regulatory landscapes for expanded access options, are patients at risk of missing out on potential treatments if they aren’t knowledgeable about this pathway?   

CM: This depends on multiple factors, one of which is a treating physician’s awareness of expanded access, which can vary by specialty and level of utilisation of the pathway. For instance, this may be more of a known potential option in oncology than dermatology due to the increased incidence of serious or terminal diagnoses. The treating physician also must be comfortable treating patients with investigational medicines outside of a clinical trial. 

RA: Why is it so important that patients have consistent and equitable access to investigational medicines?   

CM: First, access to investigational medicines outside of clinical trials is for a finite population for a finite time—this is not to treat the population of patients “at large.” These are patients who are not able to participate in a clinical trial, and their disease has been treated by every available option where they live. Second, providing expanded access must not compromise the ability to complete clinical trials, which allow medicines to be evaluated by health authorities with the goal of getting approval so the medicine is available to patients with the disease. Some patients cannot wait for that process to be completed and may receive potential benefit from investigational medicines.  

Consistent and equitable access for this group of patients is critical to help ensure that the medicine is being allocated fairly; every request is being evaluated against the same criteria and no request is given special treatment because of who the patient or their care team know. All requests must be handled the same. The Johnson & Johnson bioethical principles shape our Pre-Approval Access (PAA) Framework¹, our gateway to expanded access. They also support our work with the Compassionate Use Advisory Committee (CompAC), a first-of-its-kind committee that we created with New York University to support our decision making².

RA: Why did Johnson & Johnson decide to create this Guide to Expanded Access resource?

CM: Patients and patient representatives asked for it. They found information on this potential treatment path either difficult to find, difficult to interpret, or both.  In some cases, it was not feasible to determine easily if this was an option in a country. Bridging awareness and educational gaps in this area is a shared priority with the patient community, so we decided to make a Guide with them. It was also important that it was not specific to any company process, but country specific.  

RA: Could you talk about the process of engaging patients and their representatives to help create this and what some of the most important feedback you gained was? 

CM: Their involvement in the process was so important because patients not only led to the creation of this guide – their insights and experiences helped to bring it to life. When we created the first version, we first worked together to make sure the format was appropriate. Question and answer format is usually concise and easy for all of us to follow—that was the easiest part of the process. Next, we discussed the key questions that patients and caregivers may have, with a focus on what actions they can or cannot take and key aspects of the request process. And, of course, the Guide had to be easy to read and people-friendly—anyone who picked it up should be able to learn something and/or know where to go for additional information. This required the most effort because the information can be highly technical and while modifying it to be user friendly is critical, so too is making sure the spirit of the content doesn’t change. Essentially, we wanted to provide the fundamentals of what patients need to know but also be of service to their caregivers and healthcare providers.  

RA: How are you hoping the healthcare community engages with this guide?

CM: One of the commitments we have as part of our PAA Framework at Johnson & Johnson is to share best practices and learnings. This Guide³ is an educational resource for anyone which means it can, and has, been used by advocates, patients, healthcare providers, and other stakeholders. We created it for patients, but it is an educational resource for anyone interested in learning more about expanded access. Our second edition released in June of this year includes additional countries and encompasses feedback from users, and we hope the patient community will continue to share their thoughts and feedback with us!

From DDW Volume 26 – Issue 1, Winter 2024/25 Read the digital issue

References

1. https://innovativemedicine.jnj.com/how-we-work/compassionate-use-pre-approval-access/our-policy-and-principles 
2. https://innovativemedicine.jnj.com/how-we-work/compassionate-use-pre-approval-access/compassionate-use-advisory-committee 
3. https://www.janssen.com/sites/www_janssen_com/files/guide_to_ea_june_2024.pdf.

Biography:

As Executive Director, Patient Strategies & Solutions for Johnson & Johnson Innovative Medicine, Christine MacCracken leverages her expertise in expanded access, training as a paediatric oncology nurse, and experiences working across pharmaceuticals, nonprofit, and advocacy to implement patient-centred initiatives, driven by the guiding principle “every patient is a person first.”