Barriers to Drug Access for Patients with Hemophilia A

EP: 1.Standard of Care in Treatment of Hemophilia A

EP: 2.Monitoring for Treatment Complications in Patients With Hemophilia A

EP: 3.Challenges Caused by Factor Replacement Therapy

EP: 4.Humanistic Burden Associated with Hemophilia A

EP: 5.Reimbursement Rates for Prophylactic Treatments in Hemophilia A

EP: 6.Evolving Treatment Landscape in Hemophilia A

EP: 7.Standard of Treatment for Hemophilia A

EP: 8.Prophylactic Treatment Options for Patients With Hemophilia A

EP: 9.The Impact of Extended Half-life Factor Prophylaxis on the Quality of Life

EP: 10.The Role of Inhibitors in Treatment Strategies for Hemophilia A

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EP: 11.Barriers to Drug Access for Patients with Hemophilia A

Lisa Schrade, PharmD: The financial aspect is one of the major barriers to access patients are experiencing. Making sure that the patient is enrolled in a plan that adequately covers their needs and that they have an employer that offers sick time if they need to use that for any kind of incidental issues. But another barrier is demographics. A lot of our patients live in very rural areas. They don’t have access to a hospital or a facility that has any knowledge, or the products themselves, to be available to assist the patient if there is an emergency. So they’re relying on their own education, and some of these patients come from very poor backgrounds so their knowledge base is very minimal. That’s another restriction that prevents their care.

And we’re living still through post-COVID-19 situations where they might be affiliated with a treatment center or a hematology office, but they can’t get in for proper evaluation because the appointments are backlogged, or the staffing is short. Besides dealing with their medical condition, there are all these other facets that are bombarding these individuals; it’s very stressful.

As far as the evolving treatment for hemophilia A, it’s an exciting time right now. It’s also scary because there’s so much stuff coming down the pipeline. Just this month, we had the approval of the first gene therapy for factor IX patients. And the one for factor VIII, or hemophilia A, patients, it’s on the horizon. Hopefully, maybe by the first quarter of next year, that one will be available for patients as well. So gene therapy is going to be another one of those game changers for certain individuals.

The screening criteria are very restrictive; not a lot of individuals will be eligible for participation. And even for those who are eligible for participation, there’s always the chance the therapy isn’t successful, so they’ll have to revert to previous therapies that they were on. The limitation with that, of course, is who’s going to pay for that? That’s always the question at the front of our minds. It already costs this much money for their factor replacement, and then to pay millions of dollars for a gene therapy treatment that may or may not take, and if it doesn’t, and they have to go back to the expensive factor replacement, was it worth it? But there are other therapies on the horizon.

The other therapy in the works is the tissue factor pathway inhibitor, which can be used for factor VIII and for factor IX patients, so lots of clinical trials going on. A lot of them are in the final stages, stage 3 research. There’s so much stuff in such a short amount of time. Considering we thought a lot of these extended half-life products started coming on the market superfast, and it was great, and now within the last 2, 3, 4 years there’s been all this new research and development. They talk about a cure for hemophilia, and it just seems like it’s more possible now than it ever was before. It’s a very exciting time. I’m so happy to be a part of this. It’s great to see the amount of research and dedication that are going into trying to help this very small group of individuals.

As far as additional points that I like to counsel my patients on when they’re initiating prophylaxis treatment, one is: listen to your body. I can’t interpret what you’re feeling. I’m going off your recommendation and your perspective about how you feel when you infuse, how you feel when you inject your medication. If your dosing is every third day, and by the second day your joints start to ache, and you have pain or difficulty getting out of bed in the morning, don’t just disregard that, because your therapy says that you’re only supposed to infuse every 3 days. Those are things that I tell the patients; open the line of communication, no detail is too small or too large for me to take into consideration so that I can coordinate the care with their provider to make sure that their needs are being met. With hemophilia patients, I can’t stress this enough they’re all special individuals. You can’t cookie-cutter their therapy.

Yes, there are guidelines that we can start with, but from there, it’s tailored specifically for that individual. So that’s one of the main things I try to do, build that relationship with my patients, so they understand that to us, they’re not just another prescription to fill, another claim to be paid. These are true individuals that could be one of my family members. And I want to make sure that they’re getting the proper care and treatment that they need, without having the burden and having to worry about: “How am I going to get my medication? Is it going to get delivered on time? Should I go without it because it’s Friday night at 5 before a holiday?” Those are things they shouldn’t have to worry about. They should be able to lead the same normal life as anybody else that doesn’t have a bleeding disorder. And that’s the way that therapy is migrating. Stay the course and continue to communicate with your pharmacist, your provider, and if you think something is off, just tell somebody. That’s one of the biggest things I can say.

Transcript edited for clarity.