Listen Up, Pharma: C. diff Patient Advocate Shares Tips To Better Understand Patients
A conversation with Christian John Lillis, CEO, The Peggy Lillis Foundation
When opening a conversation, we often cycle through a few pleasantries, including the knee-jerk question, “How are you?” We say it because it shows we care how the other person is feeling and about what’s going on in their lives. Right? But rarely do we intend to listen to the answer. It’s more of a segue into the broader conversation.
The same paradox might also apply to the solicitation of patient input from pharmaceutical companies. They ask, but are they really listening? The exercise of incorporating the patient voice has certainly increased in frequency in recent years, and many companies do make a genuine effort and habit of including patient feedback.
But there’s still room for improvement down the line — from disease education to clinical research awareness to study design and execution, says Christian John Lillis, CEO of The Peggy Lillis Foundation (PLF).
In this Q&A, Lillis discusses his experience advocating for C. diff therapies, highlighting some successes as well as shortcomings in feeling heard and affecting change in patient care and drug development for the condition.
Clinical Leader: What do PLF patient advocates prioritize when talking with pharma companies about clinical research?
Christina John Lillis: One of our chief concerns is whether the trial design and protocol are patient-centric. Because the symptoms of C. difficile infections can leave patients afraid to leave their homes for fear of soiling themselves, having a trial designed with that in mind is very helpful. We’re working with a company now that has a decentralized trial arm where patients can do the entire trial from home through telehealth and a visiting nurse. For recurrent C. diff, we want the trial design to consider the physical symptoms and the socio-emotional impacts the infection has on patients.
Can we drill down to specifics on one of those priorities? Why is it a challenge, and what can pharma do to accommodate patients?
Due to the symptoms, primarily urgent diarrhea, decentralized trials are conducive to recurrent C. diff patients. Decentralized trials also benefit patients who live outside urban centers, far from most academic medical centers. If that’s not possible, clinical trial sponsors should take into account the primary symptoms, which also include abdominal pain, fever, nausea, vomiting, and dehydration, which can make travel stressful.
Can you share a time when PLF successfully shared the patient voice to impact drug development, trial design, or something else beneficial to patients?
We’ve taken several actions to improve patient input into development and design. In March 2023, we convened a Patient Listening Session with the FDA. PLF staff and volunteers have also advised companies and academics on patient inclusion in trials. This increased awareness of the need to include patient perspectives has led to more recent trials including measures to assess patients’ experience. In turn, the sponsors of these trials have been sharing what they have learned about patient experience in their trials at conferences and in academic journals.
What have been some hurdles in sharing the patient voice and feeling heard?
For C. diff, a significant hurdle is the lack of patient participation in guideline development and review. Clinical guidelines from the Infectious Diseases Society of America (IDSA) and American Gastroenterological Association (AGA) are very influential in the actual practice of medicine. Yet neither of their major C. diff guidelines has included substantive engagement with patients in their development or review. As someone who has worked on this disease for over 15 years, it’s obvious that the guidelines lack input from patients. Given the low awareness of C. diff and its acute nature, having patient input on guidelines is critical. Over the past few years, we have seen increased interest in patient engagement throughout the clinical trial process from companies working on C. diff.
What are the most common disconnects between how pharmaceutical companies design clinical trials and what patients actually need?
The biggest disconnect within the C. diff space is the inability to test for cure. This is not the fault of any company; it’s simply the limitation of our current diagnostic tools. Patients want confirmation that the treatment has worked. The other disconnect, which could possibly be improved, is the inclusion and exclusion criteria. Given the demographics of C. diff patients, exclusions based on age-related ailments can disproportionately exclude the most likely sufferers. Similarly, many trials exclude patients with inflammatory bowel diseases, even though they’re at higher risk for C. diff. While we know it's essential to have an initial otherwise healthy cohort to demonstrate the treatment’s efficacy and identify any side effects, I believe having arms with broader inclusion would benefit patients and our understanding of the disease and proposed treatments in susceptible subpopulations.
What can pharmaceutical companies do to be better partners in this exchange of information?
Some pharmaceutical companies spend a great deal of money advertising specific products, which is only legal in the United States and New Zealand. I believe that money would be better spent educating the public about disease awareness, prevention, and treatment. Unfortunately, most Americans lack a basic understanding of healthy nutrition, the difference between bacterial and viral infections, when antibiotics are necessary, and so on.
Low health literacy leaves all of us at a disadvantage. It disadvantages the public, who tend to fear diseases that are not threatening or fail to engage in preventative behavior. When people are sick, most disease knowledge comes from advertising, fictional television, friends and family, or TikTok. They do not understand the critical importance of clinical trial participation, so they are often reluctant. A more educated population might be more inclined to participate in trials.
What advice would you give to pharmaceutical companies looking to improve their engagement with patient foundations?
Patient organizations need reliable funding and strong partnerships. Companies should take a broader perspective regarding which organizations they support. Particularly in the infectious disease space, there simply aren’t many companies developing anti-infectives. There is little incentive to fund trials that will cost hundreds of millions of dollars to develop drugs without a clear patient population. Since most infections are acute and can impact any demographic, there’s no obvious constituency to advocate for funding of infectious disease development, using the “right drug for the right bug” (regardless of cost), and ensuring that Medicare, Medicaid, and private insurers cover new and narrow-spectrum anti-infectives.
Despite our advocacy, it has taken nearly 13 years for fidaxomicin, a narrow-spectrum agent for C. diff with a lower recurrence rate, to be recommended equally with vancomycin for first occurrence. This was primarily due to the much higher cost. But for each recurrence of C. diff, the likelihood of sepsis and death increases, along with the expenses of hospitalization and admission to the intensive care unit. Patients end up with C. diff primarily due to antibiotic use, and we cannot predict who it will impact. So, oncologists, dentists, surgeons, and every other medical specialty should care deeply about C. diff. Ideally, pharmaceutical and other healthcare companies will invest in smaller infectious disease nonprofits, so that our work can benefit the entire system.
About The Expert:
Christian J. Lillis is the co-founder/executive director of Peggy Lillis Foundation and a leader in the world of nonprofits. He is a brother, son, thought leader, lobbyist, advocate, and writer. Speaking on issues surrounding C diff. awareness and patient advocacy, Christian uses his platform to promote the ongoing research and funding needed in the antimicrobial resistance space. Christian’s drive for change is influenced by his mother, Peggy Lillis, and her battle with C diff., ending in her loss. Christian has been featured in many publications, such as USA Today and Reuters. He has made frequent appearances on numerous podcasts and made a name for himself on Capitol Hill through lobbying efforts and congressional testimonies.