By Richard Faris, Chief Commercial and Clinical Officer
Why does the hardest part of the journey often begin after a diagnosis?
I found myself reflecting on this question during my time at the TSC Alliance World Conference, one of the most important gatherings of patients, caregivers and advocates affected by tuberous sclerosis complex (TSC), a rare genetic disorder that often requires lifelong, multidisciplinary care. While the conference included discussions about research, emerging therapies, and future treatment options, what struck me most was the number of families in attendance and the stories they shared.
I spoke with parents coordinating care across multiple specialists, caregivers balancing treatment management with the demands of everyday life, and advocates who had become experts in navigating the healthcare system out of necessity. Those conversations reinforced an important reality: families are often expected to navigate insurance requirements, prior authorizations, and care coordination while also managing a complex medical condition. Most never expected to become healthcare experts, yet many are forced into that role. Even people who work in healthcare can find the system overwhelming when the patient is their own loved one.
Listening to those experiences reinforced what I’ve long believed: meaningful patient advocacy begins with truly hearing patients and caregivers. Hearing about access barriers in everyday work is very different from hearing the same frustrations voiced again and again by patients and caregivers who are living them. When you hear firsthand stories about months-long delays, repeated insurance denials, and the uncertainty of not knowing whether a prescribed treatment will ever reach the patient who needs it, the issue becomes much more than a reimbursement challenge. It becomes a reminder that patients should not have to navigate this complex system alone.
Understanding the Access Journey
Another reality that came up throughout the conference is that insurance requirements are becoming increasingly complex, creating additional barriers for families trying to obtain and maintain access to therapy. In fact, a recent PANTHERx-Morning Consult survey found that 90% of rare disease patients reported feeling that they have to be their own advocates.1
Patients and caregivers are frequently asked to navigate a maze of benefits investigations, prior authorizations, appeals, copay assistance programs, and other requirements before beginning treatment. Unfortunately, many families are forced to learn this process while simultaneously managing complex medical conditions. Through my discussion, I wanted to help simplify this journey.
Many attendees told us they had never received a clear explanation of how these programs work or how they affect access to therapy.
I spent time defining terms patients frequently hear but may not fully understand, including benefits investigation and verification (BIV), prior authorizations, appeals, copay cards, patient assistance programs, bridge programs, accumulators, and maximizers.
My hope was that by better understanding the process, patients and caregivers would feel more empowered to advocate for themselves and their loved ones.
Don’t Accept No for an Answer
One of the messages I shared with attendees was that patients should expect support from the organizations involved in their care, particularly their rare disease or specialty pharmacy.
Rare disease and specialty pharmacies are uniquely positioned to help patients navigate access barriers. We work with providers, manufacturers, payers, and patient support programs every day, and we should be advocating on behalf of the patients we serve.
I encouraged attendees not to accept vague answers when challenges arise.
If coverage is denied, ask why. Request specifics, including the rejection code the pharmacy is receiving from the insurance company. The more informed patients and caregivers are, the better equipped they are to work alongside their healthcare team to find a path forward.
Ultimately, patient advocacy isn’t just about speaking up. It’s also about understanding the process well enough to ask the right questions.
Why This Work Matters
One of the things I always take away from patient conferences is perspective. When you spend time with families living with rare diseases, you gain a deeper appreciation for what they face every day. You see the resilience of parents advocating for their children. You hear stories of uncertainty, perseverance, and hope.
Every patient deserves to focus on their health, rather than navigating insurance hurdles. The conversations at the TSC Alliance World Conference reinforced why advocacy matters: helping patients spend less time navigating barriers and more time focusing on the moments that matter.
1. Morning Consult. “The State of Rare Disease Care.” Custom survey commissioned by PANTHERx Rare Pharmacy and conducted January 27-February 11, 2026, among 226 rare disease stakeholders