HEOR First Report: A Deeper Look at the Benefits of Hemlibra

Karen Powell, PharmD, MS, Pharmacy Solutions Coordinator, Conduent, discusses a recent study in which she and her colleagues measured the cost effectiveness and utilizations rates associated with emicizumab-kxwh treatment for patients with hemophilia. 

My name is Karen Powell and I currently work for Conduent in pharmacy benefit management.

We work primarily with state Medicaid programs, helping them with their pharmacy utilization coverage, looking at the impact of new and existing drugs on utilization and resource management, reviewing the clinical efficacy and effectiveness. We take all of these factors into consideration for placing on formulary for prior drug release or preferred drug list, as well as whether there will be prior authorization criteria.

What is significant about your study's findings; was anything surprising?

When Hemlibra was first approved in late 2017, it was only approved for use in patients with inhibitors to factor.

Hemophilia A patients take factor to decrease bleeding. Some of them take it prophylactically, and some of them on-demand if it's a milder disease course. In 2018, the indication for Hemlibra was expanded to be used in patients with or without inhibitors and to a wider population from newborns to adults.

There was concern about what would this mean. Would existing patients on factor switch over to Hemlibra? How can we get our arms around appropriate Hemlibra utilization, and what would be the cost impact of this? It was significant that we found that there was a decrease in drug cost and medical utilization, in overall drug performance and utilization and medical utilization, for all the patients. 

We used a claims database and were therefore limited to diagnoses by the ICD 10. There isn't an ICD 10 that signifies whether a patient with hemophilia A has inhibitors or not, so we looked at the entire population. It was interesting that there is an estimate that 25% of hemophilia patients have severe disease, where they have to have inhibitors. They either have to take very high doses of factor or have to use a bypassing agent. 

When we looked in the database, 22% of patients that had hemophilia A switched over to Hemlibra. That was pretty close to that 25% of patients with severe disease.

I think it's significant that all the patients that were treated with Hemlibra—and assuming that they're with inhibitors or without—had decreases in the overall pharmacy cost and medical cost and utilization. We were surprised that the pharmacy cost decreased, because a lot of times, when you look at a new drug that is fairly expensive, you think, "Okay, you have to give a little on the pharmacy side, but the bigger impact is going to be that the medical is going to go down."

We actually saw a decrease in the pharmacy costs that they had before being treated with Hemlibra. Both the pharmacy and medical costs decreased, and that surprised us.

What are the unique benefits of using Hemlibra for hemophilia treatment?

Hemlibra's unique in a couple of ways. One, it can be administered subcutaneously, just like insulin is administered subcutaneously. Most, if not all, of the other factors for hemophilia are given by [intravenous] IV infusion—which is much more time consuming, of course, and there are risks with having to have venous access. You have to do it in a specific setting, whether it's home health, you go into the clinic, or go into a physician's office. So that is one benefit of Hemlibra. Also, because it's administered subcutaneously, patients or caregivers can be trained to give Hemlibra. 

After you do your four weeks of induction, your four weeks of initial therapy for Hemlibra, you can continue weekly, every two weeks, or even every four weeks. Even though there are some factor products that can be administered every two weeks that are, some are extended release, the ones that are the longer acting ones. To date, there aren't any others that are every four weeks. Decreasing the number of injections, especially when you're talking about a large portion of your population being children, the better, and the less invasive you can make the treatment is a huge benefit.

Those are the two main benefits, and of course that it can be used in patients with inhibitors. When we talked about inhibitors before, patients on inhibitors either have to use those large doses of factor, or they're taking factor, and they're taking a bypassing agent. With Hemlibra, there's just the one agent, the one injection, that they take.

How can payers use this information to guide their formulary decisions?

They can use this by seeing that this drug can be covered, and it can be of clinical benefit to your patient, as well as cost benefit to the entire enterprise. You can see some improvements in clinical efficacy, because the studies show—even in our study—medical resource utilization went down.

In our study, not only were patients not bleeding more, or having to go to the emergency room (ER) or the physician more, they were actually going to the ER or physicians less.

I think this drug can provide a way to better manage these patients without seeing any significant increases to their budgets. A hemophilia patient easily spends over a million dollars just in pharmacy costs.

I believe that is important for payers to consider this information in their formulary decisions. The way we looked at the research, the way we approached it, it's very easy for decision makers to replicate it with their own data to see if they're seeing the same things.

Then, if they are, then they can look at what utilization management programs they have around it or criteria for use. They may find that expanding its access can be a benefit to their overall program.

Do you or your colleagues have any plans to expand upon this research?

Not at this time. There is a new gene therapy that is with the FDA for approval. In fact, if they follow the PDUFA date, it should be out this quarter or second quarter. I think it initially had a June review date.

We may want to look at something around that. As of right now, we don't have a plan to do anything more with Hemlibra.

Reference:
Brink D, Moore J, Powell KM, Sheen JV, Williams A. Uptake of Emicizumab-kxwh (Hemlibra®) in the MO HealthNet Population and Impact on Medical and Pharmacy Cost and Utilization. Poster presented at the AMCP eLearning Days, April 20–24, 2020.