Guest Column | October 12, 2021

Co-Creating With Patients: The New Guide To Expanded Access

By Christine MacCracken, Janssen Pharmaceuticals

program software to intern mentoring concept-iStock-1248161455

In recent years, engaging with patients has increasingly been incorporated into various stages of drug development and related initiatives. Obtaining the “voice of the patient” is something that, fortunately, seems to be moving toward a standard; however, there is always room for improvement to ensure this is not a “check box” but rather a true collaboration where feedback is not only heard but heeded. 

Meaningful engagement, regardless of with whom we are interacting, is dependent upon essential communication skills we all should ideally be able to expect. Managing expectations, listening more than speaking, and closing loops require our ability to create an atmosphere where candor is welcome regardless of what we hear. The same holds true for engaging with patients. One of the best pieces of feedback I’ve ever received from a patient was, “That has too many words and makes no sense.” This patient advocate saw immediately what I couldn’t because I was too close to the item under development. The critique was 100% accurate. So, we worked together to make it sensible and, of course, fewer words. 

Recently, Patient Strategies & Solutions (PS&S) in the Janssen Pharmaceutical Companies of Johnson within the Office of the Chief Medical Officer (OCMO), had the privilege of working with patients and advocacy groups to co-create a resource about expanded access for patients and caregivers. Expanded access, also known as compassionate use or other terms depending upon where you live in the world, is a potential treatment pathway whereby a physician may request access to an investigational medicine on behalf of a seriously ill patient. In addition to being seriously ill, the patient must also have exhausted all available commercial treatment options and be ineligible to participate in a clinical trial, the preferred route for access to investigational medicines. 

Just as terminology may vary country to country, so, too, does the amount of information available on the expanded access pathway. Patients outside of the United States have shared with us their challenges in not only discerning if expanded access is an option in their country, but also how the request process works. If one is in the position of needing to find information on expanded access, it is likely that time may be a precious commodity. Fruitless searches for the information will only heighten a patient’s uncertainty at a time when speed and clarity are essential.

PS&S set out to fill that information gap and collaborated with Bionical Emas, patients, and patient representatives to create a first-of-its-kind Guide to Expanded Access. And, as with most items very much worth doing, our goals were straightforward but not necessarily easily accomplished. Identifying the most relevant information and then validating with patients what was indeed most important to them was the top priority. Once the key pieces of information were culled, we needed to make sure we maintained the intent and spirit of the language while making it people-friendly.

Together, we set to work identifying and extracting information for 28 European countries and Canada. It was a significant effort and we eventually focused on nine key questions and answers from patients. Europe has many health authorities with varying requirements regarding expanded access, so navigating this pathway can be tricky. The first question addressed for each country was, “Is expanded access an option for me in my country?” Because expanded access is not available in every country, the team thought it was critical to answer this question up front so patients could pursue other avenues, as appropriate. Understanding what is not available is equally, if not more important, as knowing what is available. 

The result is a tool we hope will begin to bridge the educational and information gap for patients, caregivers, and providers in need of information about expanded access. The Guide to Expanded Access has been shared with a wide array of patient groups and is available on to anyone interested in learning more. While we are proud of this tool, continued feedback from users is essential and we encourage the patient community to share their feedback on the Guide to Expanded Access at

As previously mentioned, meaningfully engaging with patients is moving toward being a standard practice which is mutually beneficial for patients and the pharmaceutical-biotech industry.  Remembering patients are people first helps us keep their needs front and center. Like any other relationship and discussion, we all want to be heard. Some readers in the United States and Canada may be familiar with Mr. Rogers, a beloved children’s television show host who taught kids how to be caring. Listening is a key skill he encouraged.

More and more I've come to understand that listening is one of the most important things we can do for one another. Whether the other be an adult or a child, our engagement in listening to who that person is can often be our greatest gift. Whether that person is speaking or playing or dancing, building or singing or painting, if we care, we can listen.” — Fred Rogers

We will continue to candidly discuss the Guide to Expanded Access with patients to ensure the tool remains useful and relevant. Even if the feedback is, “That has too many words and makes no sense.” We care. We are listening. Let’s talk. That’s what patient engagement is all about.

About The Author:

Christine MacCracken, MSHEd, BSN, is senior director and head of patient strategies & solutions in the Global Medical Organization, Office of the Chief Medical Officer, at Janssen Pharmaceuticals. In this role, she is responsible for developing and implementing patient-centered programs to support and elevate critical needs of people around the world. She is an advocate for patients and operates by the tenet that “every patient is a person first.”