An Integrated Patient Advocacy Strategy With Transparent Communication

A roadmap for cohering patient perspectives.

The rare disease community is intuitive and sensitive, which is why those in this space need to be hyperaware of the pulse of patients because that will guide the next steps. As the liaison between Catalyst Pharmaceuticals, which focuses on the ultra-rare Lambert-Eaton Myasthenic Syndrome (LEMS), and the LEMS community, I know firsthand the importance of developing relationships early on with patients.

Rare disease patients are passionate, often because many are without treatment. When pharma companies that are developing drugs for rare diseases approach these highly aware, knowledgeable, and educated patient groups, they need to be transparent. The promise of a potential drug that is in development can be ecstatic for patients; it can also be difficult in the event the same drug fails. Managing expectations is key in forging a working relationship with the patient community.

The well-being of patients is the ultimate goal. To serve patients better, it is important to walk in their shoes, experience their vulnerabilities, and learn about every aspect of their lives. Educating the community and raising awareness of the diseases is crucial to establishing the foundation of relationships. At Catalyst, this is what we strive for.

For many diseases, great resources are available for the patients to understand their prognosis, but for most rare conditions next to nothing exists. This is the unmet need companies need to focus on—creating a safe space where patients can learn, not only about the condition, but from each other, voice their anxieties, and get clarity on the drug development process.

Patients become anxious when they do not fully understand the process. Helping elucidate their role in the ecosystem is incredibly important. While the pharmaceutical side understands where that line in the sand is, patients often do not. Informing them along the way with honest communication is imperative.

Transparency begets trust and mutual respect. Partnering with patients should not just be on paper, it should be reflected in all endeavors. This solicitation of feedback from the community on how to improve and provide patients with a stronger voice helps strengthen that trust. Partner and support, hold and elevate, sustain, and strengthen—that is how a patient community is empowered. The disconnect and isolation that a patient often feels from their friends and family because of their diagnosis, can be ameliorated by talking to another fellow patient who can provide guidance and insight.

With over 7,000 rare diseases, it takes a village, along with some perseverance, and a little luck, to bridge the gap of the unknown and obtain a rare diagnosis. Even then, access to treatment and support is crucial. This is where pharmaceutical companies should go beyond and offer the best support possible.

LEMS affects a small portion of the population. Until recently, there were hardly any resources available to patients; that changed when Catalyst came on the scene. In the past three years, we have accomplished an unprecedented level of LEMS education awareness and support for LEMS patient communities that was absent over the past 20 years. This was possible because we cared and were willing to fill the void, creating a haven for patients. Catalyst offers first-class, robust patient support programs, and an arsenal of tools and resources including an award-winning disease-specific website that was built with patient feedback in every aspect of its design. This website was created with passion. Not because we had to, but because we wanted to and respect the challenges of the LEMS community.

To move the needle ahead for all rare disease patients, the participation of everyone involved, across the board, is necessary. Physicians, researchers, patients, patient advocates, and pharmaceutical companies should come together at the table and work with each other. When the clock is ticking, priorities are varied and there are palpable desperations—cooperating is not easy. It’s daunting but possible; it has been done before. The biggest challenge is to be open to ideas, listen to options, and not be driven by an agenda.

Catalyst collaborates with multiple national and regional patient advocacy groups to understand and address the plights and concerns of our patients. Patients are our only priority. In the rare community, you cannot accomplish goals unless you're collaborating across teams.

This journey is ongoing, and as rare disease advocates, we have more to accomplish. Adversities are unavoidable in life, but in building and nurturing a relationship with a rare disease patient community, they are not allowed to dwindle. In our community, we face, conquer, and carry on.

Amy Grover, Senior Director of Patient Advocacy, Catalyst Pharmaceuticals