Expanding Access to Gene Therapies: Addressing Patient Burden, High Costs

Gene therapies remain very labor intensive, so despite their life-saving potential, access to and utilization of these therapies remains a challenge, explained Kevin Niehoff, PharmD, BCMAS, associate director, Market and Financial Insights, IPD Analytics.

This transcript has been lightly edited for clarity.

Transcript

There is an equity aspect to sickle cell disease since it disproportionately affects Black individuals, and the new gene therapies have a host of burdens to them plus extremely high price tags. What steps can the US health system take to ensure these patients can actually access and utilize these therapies?

I think that, fortunately, CMS is a little ahead of the curve in that respect. They have their new Cell and Gene Therapy Access Model, which they just announced in January of this year, and they're using the sickle cell therapies to kind of spearhead that effort and pilot these programs where we talked about value-based care and outcomes-based agreements. And the biggest challenge, I think, in implementing those has been uncertainty about what metrics are we going to cover? Who's responsible for the reporting and the data analysis to actually implement these programs? Because they sound great, but they really are labor intensive, especially depending on the amount of monitoring that's required to put them in place.

CMS, I think, really is doing a great job in trying to expand access for a patient population that, as you said, has been historically underserved. A large proportion is covered under Medicaid services, and as a function of that, they're also geographically kind of concentrated in pockets around the country. You see some states that are also disproportionately affected and then they're bearing a disproportionate burden of that therapy cost, where they might be extremely interested and concerned with how can we implement a value-based agreement to help shield us to some degree from the large burden of cost? I think that Cell and Gene Therapy Access Model is going to help tremendously.

But I think a large barrier here is that this specific patient population really does struggle with access to care, and that could be something as simple as can they make it to that appointment? Can they get in touch with their doctor to do the prior authorization work? I think we've seen purposeful placement of those qualified treatment centers geographically, in regions that have higher proportions of patients. And I think that from a marketing and an access perspective, it makes a lot of sense. And I think they're moving in the right direction, in terms of the manufacturers trying to get access where it's needed, but I think, ultimately, these gene therapies are still very labor intensive.

It's a tremendous amount of coordination between the providers and the manufacturers, and I think that manufacturers like bluebird bio, they have experience with their other gene therapies, but there's still a lot of barriers that need to be overcome. One interesting point is that Vertex Pharmaceuticals has actually been lobbying tremendously. Presently, Vertex is allowed to provide support in terms of lodging and travel accommodations for patients who couldn't otherwise afford it, because they're opening a lot of these qualified treatment centers, but there is still going to be a limited amount geographically, and you might have to travel 100 or 200 miles to get to one. So, for publicly funded, government-funded programs, Vertex, the manufacturer, is allowed to help those patients get there and have support programs. But presently, they are not allowed to offer fertility benefits, and I think that's another interesting point where they're lobbying and actually suing CMS to try to get access to providing these benefits.¹

Presently, if you're a patient who's in their 20s or 30s or reproductive years, that's a big question where you might undergo this therapy and then have issues with fertility in the future. Commercially insured patients are allowed to get that benefit from the manufacturer and have the manufacturer pay for fertility preservation therapy, whereas right now, government-funded patients do not. And I think that's another, in terms of looking at access more broadly and that therapy and adverse effects and things like that, I think that's another really big question for patients to say, “Am I willing to forgo potentially having a family in exchange for this life-changing therapy?”

Reference

Dunleavy K. Vertex sues US for right to provide fertility assistance program to gene therapy patients. Fierce Pharma. July 16, 2024. Accessed December 2, 2024. https://www.fiercepharma.com/pharma/vertex-sues-us-right-provide-fertility-assistance-program-gene-therapy-patients