Avalere Analyst on How Payers Make Cell, Gene Therapy Decisions With Limited Data

Megan Olsen, MPH, principal at Avalere, discusses how the impact of small patient populations and other limitations may influence payer conversations about gene and cell therapies.

Transcript

Currently, data on gene and cell therapies shows promise but is limited to small populations and no long-term data exists. How does current data influence payer coverage decisions?

The limitations on data availability right now is certainly playing out. I think one of the inherent challenges in this space is you're dealing with small patient populations. And as a result, you're seeing products launch with more limited trial and data, smaller population sizes and the trials and not much data on the really long-term impacts of these treatments, especially given, you know, the hope. And anticipation is that these treatments have that durable benefit for many years, or potentially over a lifetime. But that really long-term data isn't yet available. So I think there are a few things that we're seeing playing out given this environment. One is that stakeholders, including payers, are looking closely at clinical trial data. Sometimes, in some cases, we've seen differences in FDA label relative to the clinical trial populations. And in those cases, payers are looking to the inclusion exclusion criteria or clinical trial populations to think about coverage decisions, given those data available on that population, whereas the FDA label could potentially be a bit a bit broader. And another one of the implications of this limited data is really driving interest in innovative contracting or innovative financing models. So how can we test through payment that this product is having those intended outcomes for the patient and in those outcomes are durable over a longer term time horizon? So I think this is going to be a theme throughout today's conversation, but a lot of interest in new ways to pay for and finance these treatments while collecting that data over the long term.