Virtual Conversation: Gene Therapy and the Promise for Rare Disease in California


Eli Kirshbaum


We recently had the opportunity to host an informative virtual conversation on the promise of gene therapy for treating rare diseases with Anna Talaga, the Medical Director for Neurology, Gene Therapy, and Rare Disease at Pfizer, Lynne Kinst, the Executive Director of the Hemophilia Council of California, and Brenda Gleason, President and Founder of M2 Healthcare Consulting.

The panel discussed the potential gene therapy has for saving the lives of millions of people living with rare diseases every day, as well as how to address some of the obstacles standing in the way of making these therapies accessible to those in need.

“It’s an incredibly exciting time, but it’s also a huge shift for patients and providers and the community at large in terms of how we’re thinking about treatment. There are many questions still with these newly approved therapies as it relates to, what is access to these therapies going to look like? … They are very expensive therapies … How are payers going to look at paying for these treatments?”

— Kinst

“We have patients with some sort of diseases that probably have no treatment, or treatment that maybe ameliorates some condition but doesn’t cure it. So we’re looking for something brand new, and the policy hasn’t caught up with that. We’re very comfortable with treating people in hospitals, we’re very comfortable with the concept of surgery … But this concept that you could have a one-time injection, or one-time infusion, or one-time something—that’s just not how the system was built…”

— Gleason

“I think we’re thinking too much at the state level, when I think we need to be thinking about access and reimbursement at the national level. I think that for each rare disease … the patient population is small. So if you’re trying to do any type of innovative agreement, you’re going to have to devote, in each state, time and resources into that personnel for just a handful of patients. I think that hopefully some innovation will come at the federal level and not just the state level.”

— Talaga

Watch the full conversation above!