Jayne Gershkowitz has been with Amicus since 2006 and serves as their chief patient advocate. She talks to RARE Revolution about how patient advocacy is embedded in their organisation and about how heartened it makes her to see it being elevated to a key business function in many companies
PEO series: meeting the beating hearts behind the rare brands
How new is the patient engagement role in your organisation, how has it evolved and what are your hopes for the role in the future?
The role of patient advocacy at Amicus is anything but new; it’s been ingrained in our culture since the company’s inception and is one of our foundational, core beliefs. Having a parent advocate at the helm in Chairman and CEO John Crowley has been vital in infusing the passion and the urgency felt every day by individuals and families living with a rare disease.
As Amicus has grown over the years, both in terms of numbers of employees and our global footprint, we are attracting people who either have or want to further their understanding of the patient. They want to understand the lived experience and be able to contribute to that by making a difference. This central function of patient advocacy attracts experienced people from other sized companies or other kinds of corporate cultures.
Over the years, the biggest change I’ve seen with patient advocacy is how it is becoming elevated as a strategic business function akin to other key business functions. Not everywhere, but at many companies. To me, this demonstrates how critical patient advocacy is in the life sciences. That’s what I have always advocated for—this evolution of the field.
What does a typical week entail for you, and are there any specific projects you can tell us about?
I don’t really have a typical day or a typical week. Variety is what’s typical, and that is part of the challenge, the joy and the excitement.
First and foremost, for my team and for me, is to be available when patients or their caregivers reach out. People with a new diagnosis reach out—and not always with a disease for which the organisation currently has a focus. We speak with parents whose children have been recently diagnosed, partners of affected adults, sometimes extended family and even friends. I also meet with leaders of patient advocacy organisations—it can be part of an international roundtable or a one-to-one conversation. One week might involve an internal executive committee meeting, a strategy session, or spearheading a cross-functional team review of requests for charitable contributions. Another might involve listening to one of our Patient Advisory Boards discuss disease experience, meaningful outcomes and clinical study designs. It might also involve working with my team to finalise an educational resource on gene therapy for rare diseases or presenting at an industry conference on advocacy in clinical trials. What keeps me engaged is that it's not the same thing all the time and that I continue to learn.
What were your personal motivations to taking up a role in patient engagement?
I started my patient advocacy career chapter after extensive work in nonprofit organisational management, marketing and fund development, including various health and social services, community programming and advocating for adults with disabilities and immigrant populations. Being executive director of a rare disease nonprofit, I embraced being out of my comfort zone—science was not my forte. It was a personal challenge and an extremely rewarding experience learning and becoming entrenched in an area that I was unfamiliar with.
What really motivated me, though, was seeing the effect advocating could have for both the youngest children and adults living with a life-limiting, degenerative disease. I worked hard to increase the size, the scope and the financial health of a long-standing organisation in an incredibly challenging disease area. We supported the families, the research and hope for the future, but had not yet hit a collective stride. It was still building a foundation with vision as the blueprints.
Moving to Amicus in its earliest days kept me in the same disease space but to a grouping of disorders that were more advanced therapeutically and for which more immediate help and hope were possible. We were looking at paradigm-shifting ways of thinking about what might cause - and how you might treat - a disease. It was a heavy educational lift for patients and families, for healthcare providers and for the company. It led to the understanding and shaping of the intrinsic role that advocacy can play in drug development and, initially, clinical-stage programs.
What makes the role of patient engagement officer important to your organisation?
Amicus takes patient advocacy very seriously; it has been embedded in our culture from the very beginning. The fact that my role as chief patient advocate is a C-suite position reporting directly to the CEO shows this. It helps drive our mission-focused behaviours of extraordinary patient dedication and patient-first thinking.
In your role, how do you ensure the patient voice remains central?
The work that we do is, fundamentally, helping people living with a rare disease become their own best advocates. Our credo in patient advocacy – at least how I view it – is being about people, not product.
We always talk about engaging with patient communities in terms of establishing and nurturing relationships and understanding needs. Empowerment is looked at through the lens of sharing education, information and resources where we might identify mutual knowledge gaps. We can then build solutions together to fill these gaps.
Understanding this lived experience helps people feel more empowered in who they are as a patient or caregiver and what is their objective for their relationship or partnership with their healthcare providers. It also helps to energise people to go out there and do more for themselves, for their organisations and for their communities.
How do you reconcile operational business needs with elevating the patient voice?
In my experience, I don’t think there have been many instances where the patient voice and business needs are mutually exclusive. That's partly because of the way we at Amicus have conducted business all these years. If they were mutually exclusive, then one would have a company that was out of touch with the people it is trying to help.
At Amicus, we've always tried to understand the person, the family and the disease community through our Patient & Professional Advocacy function. We have one of the longest standing Patient Advisory Board programs in the industry. We invite patients, families, and caregivers to the company – or, most recently, virtually – for Lunch & Learns and internal roundtables to educate our entire team, across all functions. The patient community is given a forum to have their voices be heard and to speak directly to the people who are developing the clinical studies, designing the support services and striving to ensure access. This minimises the chance to have contradictions between what the patient community wants and what we're trying to achieve on their behalf.
What are the most rewarding aspects of your role?
It has been incredibly rewarding to see what we have built as a company and seeing the difference that can be made to people's lives. To get there, we've done a lot of listening and a lot of learning. It is meeting these people and seeing how they live with their health challenges with such strength and such grace. Those are the lessons that are transferable across so many different aspects of one's life—professionally and personally. And it keeps us grounded. No matter what our successes are, we must think about what success is for each person in each family affected by rare disease and how can we help them achieve it.
What is your proudest moment in your career thus far?
I’ve been privileged to be part of the uptake and growth of patient advocacy as an important corporate strategic function in biotech, and to co-found Professional Patient Advocates in Life Sciences (PPALS), a nonprofit organisation that I’m confident will continue to advance education for people in or wanting to enter patient advocacy, particularly within industry. A particularly proud and probably the most emotional moment for me at Amicus was when we received approval of our first medicine. It proved that all the effort, tenacity and perseverance it took to make this medicine available to patients was well worth it.
What advice would you give someone considering working in the rare disease space?
Strap yourself in for the ride! It's a special place. Most people who start working in the rare disease space stay in it. For those who come to it later, it makes its way into their hearts, and they stay. Know that you're going to be shaken to your core, because of what you learn from people living with rare diseases, which is an honour to witness. If you don’t have the lived experience of those big health challenges, you're going to see things that may well change how you think about life and the needs of people with complex health issues. You also have to be sure that you stay open to all of that, but also keep your objectivity. This industry brings out the best in people. It takes an honest and genuine approach that is maintained in all aspects of our ecosystem. I think advocacy can help to influence and shape the way people think in every single function of the business.
If you weren’t Chief Patient Advocate of Amicus, what was Plan B? What did your 10-year-old self want to do as a job?
My Plan B would probably have been corporate communications or working at a PR agency. As a teenager, I always I wanted to do something with writing and communication. I knew I wanted to be a journalist; I got my degree in journalism and, for a short while did just that. I enjoyed using my curiosity to ask questions, write and convey tough topics. Letting somebody else tell their story or share their passion and then relaying that to others made it interesting for me and validating for them. I always enjoyed that, and I don't think I'm doing anything that's too far off from that now. Except now, as a patient advocate, I can echo their voice, make sure others hear it, and with dedication and resolve, help bring something back to them that potentially can improve their health and their lives.