From The Editor | September 23, 2019

The Role Of Patient Advocacy In Gene Therapy


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1

Jamie Ring

Why patient advocacy is a crucial component in the on-going successful treatment of rare diseases.

In the world of gene therapy, patient advocacy organizations are instrumental in helping people with rare diseases and complex healthcare needs by providing education, support, and services, and much more. I recently caught up with Jamie Ring, head of patient advocacy at Spark Therapeutics to better understand how patient advocacy can play a vital role in the field of gene therapy.  Jamie has dedicated her 20-year career to the field of patient advocacy in rare diseases and for the past 4 years has been part of the Spark Therapeutics team that brought the first gene therapy for a genetic disease to the United States.

What Patient Advocacy Is and What It Is Not

In the biotech and pharmaceutical industry overall, patient advocacy tends to be focused on serving as the voice of patients and patient needs inside the company, being a bridge to the patient communities a company is involved in and connecting employees to the patient experience. In addition, the function supports corporate awareness and patient engagement; for instance, corporate representation at advocacy events to offer education and to drive awareness of the company’s involvement with the disease.

“In a gene therapy company, patient advocacy means something different than it may in the rest of healthcare,” says Ring. “There is an unbelievable amount of misunderstanding when it comes to the public’s view of gene therapy. In the four years that I've been talking about gene therapy with various patient communities, gene therapy has long been viewed as science fiction. In addition, some patients affected by rare diseases are unaware that they have been living with a genetic disease all along, and so, patients start from scratch in terms of education about their disease/diagnosis and what gene therapy might then mean for them. That’s where patient advocacy driven education initiatives come in.” But patient advocates are not marketers. They do not promote individual products, and, even if patient advocates have medical backgrounds, they never provide medical advice.

In working with patient communities related to gene therapy, Ring and her patient advocacy colleagues start by explaining gene therapy to the patient and caregiver — what it is as well as the importance of understanding one’s genetic cause of disease. They educate patients either via company-driven programs, by supporting national and global patient advocacy organizations and the creation of patient education materials. They look for opportunities to bring patient insights into the company to better prepare for how to address common questions about gene therapy.  She also helps patient and families — as in the case with Spark and for most of the gene therapy companies — understand the on-going research in the field.

“For most of us who work in advocacy, our ultimate motivation is to make sure patients are empowered and educated, and as a result, they can make better health decisions for themselves or their loved ones,” she states. “There is a great amount of work to be done for people to really understand what goes into a potential one-time treatment. And for the majority of programs that are still in the clinical trial phase, as an industry and health care community, we need to make sure that trials have the right participants who understand the importance of their participation and what they’re signing up for, that communities know the questions to be asking about new treatments, and that they are empowered about their health care choices.”

“Here’s what we’re not doing — we’re not saying to patients, ‘Let me tell you about Spark’s specific gene therapy for this disease,’” says Ring. “Our goal is to explain the field of gene therapy holistically to patients. We help them understand gene therapy in the context of their specific rare disease and seek to empower them to ask the right questions of their health care provider or fellow community members. I often describe patient advocacy as being the macro-level of supporting an overall patient community and its members — disease awareness, connecting individuals to community resources and advocacy groups, etc. The micro-level of the patient journey may include their dialogue with their practitioner about an approved product, a clinical trial or individual patient support.”

Ring explains that patient advocacy can often be confused with the patient services function, which is usually a commercial function. “Patient services support reimbursement and access to therapies, and they’re the teams that help guide a family through the treatment access process,” she says. “As patient advocates, we aren’t involved at that level but rather are building relationships with advocacy groups in the hopes of better supporting patient care overall.”

Every Role Matters

For any patient advocacy program such as Spark’s to thrive, the senior leadership team must believe in and demonstrate a real commitment to patient needs, and, according to Ring, that starts with authenticity. “Senior leaders must believe in their core that what the company is doing is what’s best for the patient, and this is one of the areas where Spark excels,” says Ring. “You can have the best advocacy people around putting in the hours and doing the work, but the worst feeling in the world is standing in a room full of families and communicating something that you’re not actually sure the company is going to stand behind or champion,” shares Ring. “That’s about as hard as the job can get because as a cohort of professionals, we really are emphatic about serving as a champion for patient needs within our organizations.”

When the leadership team believes in the company’s mission, the positive effects resonate throughout every other department. Yet, it can still be difficult for employees in the various functional departments to always see how their day-to-day work directly impacts patients, especially when they aren’t frequently coming face-to-face with patients. “One of the things patient advocates in industry often hear is, ‘I don't interact with patients in my everyday job, so I don't really see how I can do my job in a patient-focused way,’” explains Ring. “Patient advocates work to help increase that understanding and provide opportunities for individuals working at a company to connect with the patient experience. Our goal is to help highlight how different roles impact the end user’s experience and that is often best done through patient visits to the company where employees can hear firsthand what it means to live with a disease and importantly, what the unmet needs are and how the company can potentially help to address those.

“All functions contribute to a healthy and productive organization and when the corporate vision resonates, in the case at Spark it is creating a world where no life is limited by genetic diseases, we all feel a sense of urgency and a responsibility to get that right.”

Patient Advocacy and the Bottom Line

Patient advocacy has demonstrated over time that it is a critical department to have and more and more companies are bringing in this role earlier in their life cycle especially in gene therapy companies because the learning curve of education is much greater and needs to start that much sooner.  With that said, can patient advocacy and its impact really be measured? “Delivering quantifiable metrics is one of the most difficult aspects of patient advocacy,” Ring says. “The common thread of any patient advocacy-oriented industry conference out there is always about defining the metrics that can be used to demonstrate that the function is creating some value, or that patient advocates are doing a good job.”

There are, in fact, metrics associated with patient advocacy. In some cases, metrics are associated with faster recruitment time and successful enrollment of clinical studies when advocacy groups have been engaged along the way to provide feedback and advice.  In that case, the people who end up enrolling tend to be more engaged and have a better understanding of what’s at stake and the trial itself may be more feasible because affected individuals have identified potential barriers in advance that the company is able to proactively address. “Companies where patient engagement has leadership’s support and accountability across the organization tend to ultimately see better, quantifiable results across the board. We can also measure perceptions from the community about engaging with the company which helps establish a baseline of metrics about how the organization is collaborating externally.

Interesting Facts about Patient Advocacy

When you peel back the layers of patient advocacy, you’ll find some interesting information. First, is the cross-functional nature of the program. “We’re one of the internal groups that has a reason to engage with almost every other functional group within the company,” says Ring. “For example, there are times when we might bring external advocacy leaders to our manufacturing facilities so that these groups can see first-hand and better understand the manufacturing process of a product.  Or we may work to bring colleagues to patient conferences to experience the nuances of a specific disease by spending days immersed in the community and hearing directly from affected families.”

Second, people are often surprised at the varied nature of professional backgrounds that make up any advocacy team. Patient advocates in industry can be genetic counselors, nurses, marketing professionals, former nonprofit leaders and so on.  “Patient advocacy is made up of a diverse group of professionals who have chosen to work in this space and do it because they feel that they are making a difference every day, with every conversation they have,” says Ring.

As a former employee of Genzyme, Ring attributes her nearly 20-year passion for advocacy to one of her mentors, Henri Termeer, that company’s CEO for decades and often considered the father of patient advocacy. Termeer often shared with Genzyme’s team, “if you do what’s best for patients, the business will follow,” and Ring has seen that to be true throughout her career. 

Ring firmly concludes that while rare disease drug development is difficult and challenging, it is also very collaborative. Patient advocacy offers valuable ways to engage patients and do right by the community while creating a sustainable business.