A recent 60 Minutes interview highlighted a reality that rare disease families live with every day: the extraordinary promise of gene therapies exists alongside extraordinary costs. Billions of dollars, years of research, regulatory hurdles, and immense scientific risk all funnel into therapies that may ultimately serve very small patient populations.
From an industry perspective, the interview underscored why these therapies carry such high price tags — the cost of failures, the complexity of manufacturing, and the long road to approval. But from the parent side, the conversation seemed incomplete if cost is discussed without context.
For Angelman families, these therapies are not abstract investments or line items on a balance sheet. They represent hope. Hope that our children might communicate more clearly, sleep more soundly, gain independence, or simply experience fewer medical crises. We are not chasing perfection or miracles; we are chasing quality of life.
What the interview touched on, but could not fully convey, is the role families and foundations play long before a therapy ever reaches a pricing discussion. Families enroll in natural history studies. They consent to trials. They travel, miss work, and shoulder emotional risk. Advocacy organizations fund early research, build clinical networks, create registries, and de-risk programs long before industry steps in. This ecosystem is built on partnership, trust, and shared urgency.
Sustainable innovation requires companies to survive and continue investing. Parents of individuals with rare diseases know that access cannot be an afterthought. A therapy that exists but is unreachable is not a success story for families. The real question we must keep asking is not whether gene therapies are expensive, but how we build systems that balance innovation with access, sustainability with equity, and profit with humanity.
Rare disease families are not naïve to the realities of drug development. We live them. What we ask for is transparency, partnership, and a seat at the table — early, consistently, and respectfully. Because behind every data point is a child. Behind every price discussion is a family. And behind every breakthrough is a community that has been waiting and working for decades.
This is exactly why advocacy is so critical. Policy decisions, reimbursement frameworks, and long-term access pathways do not happen on their own — they are shaped by informed, persistent voices.
Through Hope in Action, ASF and FAST are working together to ensure families are educated, engaged, and empowered to advocate for policies that support research, access, and sustainability across the rare disease ecosystem. To learn more and get involved, visit https://angelmanadvocates.org.
Watch the 60 Minutes segment below:
