Commentary

Video

Dr Yuqian Liu Investigates the Future Benefits of Cell, Gene Therapies Using Real-World Data

Author(s):

Yuqian Liu, PharmD, Magellan Rx Management, explores the current space of cell and gene therapies based on real-world data and potential emerging therapies in the future, while expanding upon long-term efficacy concerns.

Yuqian Liu, PharmD, senior director of specialty clinical solutions, Magellan Rx Management, assesses the current landscape and pipeline for cell and gene therapies (CGTs). Dr Liu participated in a session at the Academy of Managed Care Pharmacy (AMCP) 2024 annual meeting titled, "The Future of Cell and Gene Therapy Management: There is More Than Value Based Contracting!," where she explained how real-world data are used to determine the effectiveness of these therapies while evaluating potential financial implications.

In her role as senior director of specialty clinical solutions, she has led the company through various initiatives across regional and national health plan clients. She assists clinical program development, specialty drug landscapes, pipeline analysis, and a multitude of clinical initiatives, including specialty drugs.

Transcript

Real-world data on the long-term effectiveness of CGTs is still emerging. How can payers use currently available data to make informed coverage decisions for these therapies?

We do the best we can with what we have. There are some long-term durability data that we have currently available. For example, we have some 7-year studies, some 5-yeas studies that are coming out with the real-world data. That's what we kind of use to gauge on the continued effectiveness of these therapies to see how we can apply those to our population.

What role do you see real-world data playing in future payer assessments of CGTs?

I see that being an increasingly important role for real-world data that we're going to be using for our decision-making process for the cell and gene therapy. Ultimately, the way that these products are positioned on the market today is that they're supposed to serve as a cure for our patients. We need the long-term real-world outcomes data to be able to show the value in the space that we're practicing today. Therefore, it's going to have an increasing importance as time goes on.

Considering the current focus on oncology and rare diseases, are there any emerging therapy areas within cell and gene therapy that excite you for the future?

Yeah, absolutely. There's a lot of excitement in the cell and gene therapy space. I presented the session earlier this morning. We anticipate, in the pipeline just this year, 4 more that are coming.

Over the next 3 to 5 years, we have 70 in phase 2 clinical trials. We have, I believe, around 30, in the phase 3 clinical trials space. It's just fascinating the amount of products that are coming out.

I am particularly excited for the rare and ultra rare disease space, because historically, those patients have nothing. We're talking about life-changing therapies for these patients to be able to receive a potential cure for the condition that they have, which historically would have been deadly.

How can we address concerns about potential long-term efficacy of some gene editing therapies?

That's a tough one. The gene therapies like I mentioned, in theory, are supposed to be a cure for our patients, but we are seeing gradually diminished effects, unfortunately, for some of these therapies over time.

Right now, the only way that we're managing it is with add-on therapies, following the diminished effect from the gene therapies. But long term, there needs to be better solutions. To date, we don't have another way to address.

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