Historically, hemophilia—a bleeding disorder caused by the absence of a vital clotting protein called factor—has only had one successful treatment method: infused therapy of factor VIII for hemophilia A, and factor IX for hemophilia B. As new, innovative therapies emerge, it’s helpful to understand the history of treatment and what the future holds for the hemophilia landscape.
A brief history of hemophilia factor replacement therapy
In 1965, Dr. Judith Graham Pool discovered that a product of thawed plasma (cryoprecipitate) contained a high concentration of factor VIII, the clotting protein that people with hemophilia A lack. Within the next decade, the development of freeze-dried factor allowed for home infusions, greatly improving the health outcomes and quality of life of patients with hemophilia. Factor replacement therapy soon became the primary treatment method for hemophilia, succeeding the inefficient blood and fresh plasma transfusions that had been previously used.
In 1992, the U.S. Food and Drug Administration (FDA) approved the use of synthetic factor products, known as recombinant factor concentrates. This significant scientific advancement helped ensure safety in the world of hemophilia care after the use of donor-derived plasma led to the widespread transmission of human immunodeficiency virus (HIV) and hepatitis C virus (HCV) among hemophilia patients in the 1980s. Around the same time, viral inactivation—removing or impairing viruses present in blood donations—and enhanced screening processes were introduced, re-establishing that factor from donated plasma could be safely used to treat hemophilia.
Even as new therapies have been approved and released, factor replacement remains safe, effective and the standard treatment for hemophilia patients today.
Evolving therapy innovation for hemophilia
Over the last decade, there have been important pharmaceutical drug developments that have simplified treatment and alleviated symptoms for patients with severe hemophilia.
In 2017, the FDA approved Hemlibra® (emicizumab) as a prophylactic treatment to prevent bleeding episodes in people with hemophilia A. Hemlibra is a bispecific antibody, a man-made protein derived from Chinese hamster ovary cells, that allows the clotting process to occur without the presence of factor VIII. While factor treatment is still necessary in response to episodic bleeds, using Hemlibra is less-invasive and less-frequent than the burdensome factor replacement these patients would otherwise require. Hemlibra provides patients with a higher quality of life, as it can be self-injected every one-to-four weeks in place of factor infusions, which may be administered multiple times per week.
The development of gene therapies to treat hemophilia represents another layer of innovation and hope for people with severe forms of the condition. Gene therapies directly edit or replace the genes that cause a patient’s disease—in the case of hemophilia, viral cells are manipulated to transport genetic material. These viral vectors are used to introduce a healthy version of the missing or defective gene that produces factor into a patient’s body. Hemgenix® (approved in 2022) and Roctavian® (approval expected summer 2023) are gene therapies designed to treat hemophilia B and A, respectively.
As this rush of innovative hemophilia treatments has taken place over a few short years, there’s promise for even more advanced and cutting-edge treatment options to emerge in the near future.
Opportunities for cost efficiency in hemophilia treatment
The arrival of these new therapies has also introduced complexities for stakeholders like plan sponsors, prescribers and physicians to consider. Selecting a specialty partner willing to respond effectively to pharmacological innovation can be critical in mitigating waste, driving savings and having the right controls in place.
Most gene therapies have been developed for ultra-rare diseases with limited treatment options. In contrast, patients with hemophilia have many viable and effective options including a variety of factor replacement products and Hemlibra. Uncertainty about the durability of hemophilia gene therapies (i.e. how long they are optimally effective), combined with their multi-million dollar price tags, means payers will have to make critical benefit design and utilization management decisions about who will be covered for these treatments and under what circumstances.
Hemlibra, which is less invasive and burdensome for Hemophilia A patients than factor, presents significant medication waste management opportunities since it is dosed by weight and comes in four vial sizes. This means it is imperative that the optimal dose and correct combination of vial sizes is prescribed for the patient’s needs to avoid wasted medication, unnecessary injections and potential administration mistakes.
To this end, Accredo created an algorithm for the Hemlibra care protocol which analyzes the patient’s weight, prescribed dosage and regimen to optimize vial selection and administration. Based on the output, clinicians engage with prescribers to modify patient treatment where the opportunity presents itself.
A recent study examined the application of Accredo’s proprietary dose and vial optimization protocols for Hemlibra in a sample of 360 hemophilia A patients. The study found:
- 64% of the initial prescribed regimens failed the protocol for suboptimal dosing or vial size, meaning a majority of physicians are writing for larger vials than necessary.
- In the 43% of cases where a prescriber accepted a pharmacist’s recommendation to adjust a Hemlibra regimen, plan sponsors with participants in the study achieved an aggregate annual savings of $1.8 million across 48 patients.
- 600mg of medication waste per dose was prevented by calculating more appropriate vial sizes for the patients.
This study highlights that when a specialty pharmacy takes the initiative to create protocols and tools to accurately dispense a complex drug like Hemlibra, it is able to significantly minimize waste and prevent unnecessary spend. In a treatment class that is continuously evolving, it’s crucial to partner with a specialty pharmacy that can help predict costs, optimize patient care, avoid substantial medication waste and save plan sponsor dollars.
