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 in 2023, where she produces written content covering multiple disease states.

She has a BA in journalism from Seton Hall University. You can connect with Brooke on 

Articles

Insurer-pharmacy benefit manager (PBM) firms control most of the Medicare Part D market, steering patients to their pharmacies through network exclusions and targeted marketing. 

Insurer-PBM Firms Dominate Medicare Part D Market, Steering Patients to Their Pharmacies

Maggie is an editor for The American Journal of Managed Care® (AJMC®) and produces written, video, and podcast content covering several disease states. She joined AJMC® in 2019, and has been with AJMC®’s parent company, MJH Life Sciences®, since 2014, when she started as a copy editor.

She has a BA in English from Penn State University. You can connect with Maggie on 

Generalized myasthenia gravis (gMG) is an autoantibody-driven chronic disease for which there is no cure.


Understanding gMG: Autoantibodies and Life-Altering Muscle Weakness

Mutations in one of the first human-isolated strains of H5N1 bird flu in the US were identified by the Texas Biomedical Research Institute.

New Insights Into H5N1 Variability in Human Mutations

The approval is based on findings from the phase 3, multicenter, open-label, randomized TROPION-Breast01 trial. 

FDA Approves Datopotamab Deruxtecan for HR-Positive/HER2-Negative Metastatic Breast Cancer

The American Journal of Managed Care provides insights into the latest news and research in managed care across multimedia platforms.

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Compendium

Patients with spinal muscular atrophy (SMA) type 2 showed improved motor ability when treated with intrathecal onasemnogene abeparvovec, an investigational gene therapy.  

Phase 3 STEER Study of SMA Gene Therapy Meets Primary End Point

Common measurements for modified adeno-associated viruses (AAVs), a gene therapy delivery method, showed substantial variation in accuracy and precision. 

Standardized Measurements Needed for AAVs in Gene Therapy, Study Finds

A phase 3 trial highlights betibeglogene autotemcel as a potentially curative gene therapy for severe transfusion-dependent β-thalassemia. 

Gene Therapy Demonstrates High Efficacy in Severe β-Thalassemia 

Check out this year's top coverage from The Academy of Managed Care Pharmacy (AMCP) Nexus 2024 meeting, which included relevant topics in health care policy, novel pharmaceutical developments, financial considerations across multiple conditions, and more. 

ICYMI: Highlights From AMCP Nexus 2024

Researchers study long-term safety of lentiviral gene therapy in primates, finding no evidence of somatic mutations or malignancy.

New Evidence Highlights Stability of Lentiviral Gene Therapies

Migvis Monduy on DMD Research and Gene Therapy

Migvis Monduy, MD, medical director of Neuromuscular and Movement Disorders Programs at Nicklaus Children's Hospital in Miami, Florida, discussed the most promising areas of research in

 (DMD), as well as the mechanisms of gene therapies for DMD.

What are the most promising areas of research in DMD right now? 

[There's] lots and lots of research, and a lot to keep up with—it's hard sometimes. But the gene therapies are a promising area of research. Different companies are trying to have different gene therapies developed for this particular indication. And oftentimes, [we're] trying to find a way of replacing the full length of the dystrophin gene, as opposed to micro-dystrophin or mini-dystrophin—so, finding alternative methods of having perhaps viral vectors that could replace parts of it, and then they'll assemble back together intracellularly again, or finding other ways of having gene therapy delivered without viral vectors that don't activate the immune system.

I think those will be areas for future research that are really, really promising, as well as other combinations of therapies—not just the steroids, but nonsteroidal things at the cellular level, working on regenerating muscle for the patients that have already had some of that muscle [loss] before they receive their gene therapy. I think in the long term, it's going to be a combination of different things and we're still trying to figure out what that will look like and what will work.



How is gene therapy being used to treat DMD, and how have advances in this area impacted the outlook for patients?

Gene therapy is really the latest development, with 1 particular gene therapy product already in the market since last year. It uses a viral vector to carry that micro-dystrophin, so a synthetic version of the gene, into the cells. So, you're infecting the cells in the body of the patient with this virus that is devoid of any actual viral DNA, but actually has the micro-dystrophin in there. We're hoping to create some dystrophin protein in the muscle cells by doing that, and then limit the problems with weakness and the progression of the disease. We can't say it's a cure, because you're not replacing the full length of the dystrophin protein, but hopefully, it will be enough to halt the disease progress and it can be sustained over the years. Again, this is all new, so that will have to be seen over time.


Videos

Migvis Monduy, MD, medical director of Neuromuscular and Movement Disorders Programs at Nicklaus Children's Hospital, discussed the most promising areas of research in Duchenne muscular dystrophy (DMD), as well as the mechanisms of gene therapies for DMD.

Duchenne Muscular Dystrophy

Dr Migvis Monduy on the Most Promising Areas of DMD Research

Landon Marshall, PharmD, PhD, of Prime Therapeutics, explains the benefits of gene therapy forecasting for health insurers and what future advancements can be expected. He discussed the topic further in the AMCP Nexus session, "Gene Therapies: Forecasting Potential Candidates Using Integrated Medical and Pharmacy Claims Data" on October 18.

At Prime Therapeutics, Marshall is a principal health outcomes researcher, focusing on cell and gene therapy, as well as secondary data use to predict future spending trends. 

With the substantial price ranges of gene therapies, how does having a forecast of gene therapy benefit health insurers in managing financial risks and making informed business decisions?

Currently, the price range of the approved gene therapies that we're forecasting ranges from $750,000 up to over $3 million. Understanding within your book of business, within your membership, your population, how many individuals may be a candidate for that therapy is important for financial risk assessment. 

For underwriting, we do present actuaries, so they do use this information, but also, from a clinical standpoint, we want to understand member characteristics and potentially how this would impact current care for those members and also with the future cost and investment that these gene therapies come with.

In the future, what advancements or improvements to gene therapy forecasting can be expected?

We constantly think with the data that we're ingesting that we are improving the forecast. Having conversations with manufacturers really is a transfer of knowledge between the Prime Magellan colleagues and manufacturers.

As we enter additional gene therapies being approved, we'll start to see multiple gene therapies treating the same condition. We'll have to handle that and improve forecasting as we get product differentiation from these competitor products. Also, expecting expanded indications after early accelerated approvals for certain therapies. These are advancements that we're going to have to take in our forecast to maintain it and keep it relevant.

Landon Marshall, PharmD, PhD, of Prime Therapeutics, discusses the benefits of gene therapy forecasting for health insurers and what future advancements can be expected. 

Conference

Dr Landon Marshall Discusses Gene Therapy Forecasting Benefits, Future Advancements

Despite the long-term benefits of gene therapies, there still requires additional treatment and follow-up to ensure that disease remains under control, especially because most data are short term with less known about the long-term effects of these treatments, explained Michelle Booth, PharmD, senior director of specialty clinical solutions, and Andy Killpack, PharmD, director of specialty clinical solutions, both of Magellan Rx Management.

How should payers and providers balance the benefits of the largely one-shot nature of gene therapies—and their long-term benefits and reduced treatment burden—with the high cost of treatment?

I can see where there is a tradeoff. First and foremost, if I were a provider, I would want to assure that the right patients are getting the right medication. There really shouldn't be any wiggle room, and I do feel that providers should be responsive to what the study population was, as much as possible. Obviously, there are outliers that may be appropriate for the medication—I understand that that happens. But I really do feel that they need to be mindful of the study population.

From the perspective of continuing to see the patient, I would imagine they can't fall off and [think] “I don't have hemophilia anymore.” They’re still obviously going to need to be monitored, and I feel like it is potentially a different type of monitoring at this point: looking at not necessarily what you can see easily, like symptomatically, but what are we not seeing? What are the factor levels? Are those responding appropriately? Did they continue to respond appropriately? Are we starting to drop off? At what point are we going to need to start being concerned that somebody's going to need factor replacement?

I feel like it's uncharted waters. I’m really kind of unsure of how we're going to proceed with these patients, because we're at the forefront, and these are all new, and replacing somebody's gene and actually fixing them is a new territory. So, I think that we certainly are going to need to approach the patients that we're treating with gene therapies very differently—regardless of the vector and across the board. We are going to need to approach this a little bit differently.

Michelle summarized it well. We're really navigating uncharted waters. We've explored this new space of treatment types, and we have great hope that they're really going to work, they're going to have this durability, they're going to have long-term great effects. But we don't know that yet. We have some short-term data that look really good. We have some life-changing treatments for a lot of these diseases. But how long are they life-changing? That's the question that remains unanswered that we're trying to navigate.

Then tied to that is: What if they are and what if they aren't? Should it wear off over time, and you have to go back to the standard of care today, what does that mean for that treatment that was administered? What does that mean for the payer? What does that mean for the patient? What does that mean for the manufacturer? And those are questions that are unanswered today. Those are the things that ongoing discussions are going to have to take place. Some kind of agreement should be decided upon between the parties involved. And, as of yet, we don't have that. But those are the questions that are out there that we need to be talking about and looking to answer as more time comes on and more information is available with this real-world evidence.

Theoretically, should payers find these therapies attractive despite the high price tag because they lead to avoidance of other costs?

Yeah, because we're taking into consideration [emergency department] visits, hospitalization, home care. I'm envisioning a child with SMA [spinal muscular atrophy] and all the ancillary services. Caregivers coming in, overnight nursing or medical assistants coming in. Yeah, I agree. It certainly is something that would be attractive to insurers.

It's almost like you're paying up front. It's not a bad thing. It's just a lot of money. So, you worry about how am I going to pay for this? It's a budget buster.

But yes, I certainly agree that it's all encompassing. We’ve got to get out of the pharmacy silo. The budget goes beyond just, how much does the drug cost? I agree completely with that.

Many gene therapies promise life-changing effects, but without long-term data it remains to be seen how long the benefits last.

Gene Therapy

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