In health care, rare disease patient communities are some of the most influential underutilized voices. With over 7,000 rare diseases affecting millions around the world, these patient networks offer valuable insights, experiences and perspectives. These voices shape the future of research and development, advocacy and patient care.
At CURA, we’ve had the privilege of working closely with rare disease patients through initiatives like advisory boards, summits, FDA listening sessions and Hill Days, and in the process, we’ve developed a proven set of best practices for fostering meaningful relationships with rare disease patients.
1. Prioritize authenticity over buzzwords.
Many organizations claim to be “patient-centric,” but few deliver on that promise in a tangible way. True patient engagement starts with listening—not just hearing the patient voice but ensuring that voice informs your decisions and actions. Establishing an ongoing dialogue with patients where they feel comfortable sharing their experiences and honest feedback is critical. From Patient Family Advisory Boards to listening sessions and in-depth interviews, every interaction should be driven by genuine curiosity about patients’ lived experiences.
It’s important to identify and engage a diverse, representative group of patients impacted by the disease state. For instance, though autoimmune conditions disproportionately affect women (80% of all cases) the CURA team identified and invited men and women of varied ages, backgrounds and races/ethnicities to an influencer summit held in San Francisco earlier this year.
Whether the focus is on pharma-driven learnings, legislative advocacy or raising awareness, centering on the patient perspective is non-negotiable. No two rare disease patient stories are the same—from diagnosis to treatment, the paths vary greatly. Understanding the breadth of experiences and how people communicate about their condition will create the most inclusive, accurate and appropriate strategies for engagement.
2. Meet patients where they are.
Engaging rare disease patients requires sensitivity and flexibility. These communities often face logistical challenges due to their unique care needs. Do your research on the symptoms and accommodations typically required by the disease state and ask the right questions. Whether it’s ensuring accessible transportation or accommodating medical equipment, small details make a significant impact on trust and engagement. For example, prior to in-person events or listening sessions, it’s essential to ask the right questions like “Do you require a power chair?” or “What accommodations would help you feel more comfortable?” Improper or ineffective accommodations can tarnish you and your client’s reputation among the very patients with whom they’re trying to foster trust.
Many patients living with rare diseases have a deep-seated distrust of the health care system due to past experiences. By showing authenticity, understanding and care, you build trust and foster open communication. We’ve seen that when patients feel respected and heard, they are far more likely to engage and share their experiences openly. By meeting these patients in their comfort zones—be it through digital platforms, local advocacy groups or in-person events—you can create a more inclusive and effective engagement strategy.
3. Empower patients through education.
Partnering with patients is a two-way street of information sharing. At CURA, we’ve seen firsthand the importance of providing clear, comprehensive information about available treatments and clinical trials, relevant advocacy initiatives and community programs. Our work with the congenital heart defect (CHD) community helped them advocate for research funding, secure corporate sponsorships and inform the research and development of novel treatments and therapeutics through disease registries and direct pharmaceutical engagement.
Educating patients also means preparing them for direct advocacy. Patients equipped with knowledge about their condition, treatment options and advocacy best practices are better prepared to be their own best advocates. We’ve successfully trained rare disease patients to effectively tell their story to legislators, the media and the public through direct campaign work and our flagship advocacy training program, Advocacy Academy. Providing patients with the resources to advocate not only helps them—it creates lasting waves of change in health care for years to come.
4. Support the entire care network.
Rare diseases don’t just impact the patient—they affect entire families and caregiver networks. Caregivers often face their own set of challenges, from physical strain to emotional burnout. Including family members and caregivers in your engagement efforts is key to a holistic approach. We’ve learned that when caregivers and family members feel heard and empowered, they become essential advocates for their loved ones and the broader patient community.
By incorporating family members and caregivers into advocacy events or summits, you foster stronger relationships with the entire care ecosystem. Their perspectives can lead to more comprehensive and compassionate policies and solutions, benefiting the broader rare disease community.
At CURA Strategies, we believe in the transformative power of the patient voice. Our experience working with rare disease communities has given us firsthand knowledge of how meaningful patient engagement can create lasting change. By following these best practices—authenticity, accommodation, education, and caregiver inclusion—organizations can establish stronger relationships with patients and drive real progress.
Learn more about CURA’s Advocacy Accelerator and how we help empower rare disease patients to advocate for themselves.
Sarah Farinelli
Account Supervisor
CURA Strategies
