Neil B. Minkoff, MD: We’ve talked a fair amount about the different challenges to the patient in terms of getting access, and 2 hour drives to get monitoring, and so on. But from a provider perspective, what are some of the challenges that you’re facing in terms of managing or maintaining patients with SCD [sickle cell disease]?
Ahmar U. Zaidi, MD: I think for a long time, the issue has been lack of therapies. We have had hydroxyurea and blood transfusions for a disease that we’ve known about since 1910. And the problem is really the toolbox. We haven’t had tools in the toolbox to help us deal with patients. And thankfully we’re moving into the dawn of a new era where our toolbox is going to be replete with tools.
Neil B. Minkoff, MD: Hold that thought.
Ahmar U. Zaidi, MD: Sure.
Neil B. Minkoff, MD: From the payer point of view, what are the challenges you’re facing when it comes to maybe even identifying as well as helping to maintain a quality of life or the health of these patients?
John C. Stancil, RPh: I think the way we approach drug costs—because there are certainly very expensive gene therapies that are coming and are going to be impacting our budgets—is that we see the offset of the higher cost of hospitalizations and medical costs if we do allow access to those drug therapies. Again, most of these childhood diseases are very individualized in their approach, and so we don’t want to put restrictions to access to the provider or have the provider have to jump through hoops if they know this is the best choice or the best treatment option for that patient. We’re very collaborative with our provider groups, and try to partner with them on the standards of care that they want to implement within their institutions or centers of excellence.
Maria Lopes, MD, MS: I do think that there needs to be guidelines. There does need to be a standard without a doubt, as we think about gene therapy, and we now have 2 on the market. But we really are seriously wanting to know and understand what the durability of the effect is. Who are the good candidates for these gene therapies that promise innovation like no other in terms of a cure? But what happens if it isn’t? These patients still need to be monitored, they still need to be tracked, they still need to be followed. Most payers, especially in Medicaid, have such high churn that the issue of affordability and how we’re going to finance this gets us into innovative payment models that lead to some accountability for the pharmaceutical industry, the need for data and tracking to make sure these really are the cures that we all anticipate and value.