Hemophilia and other bleeding disorders are rare and highly specialized disorders. It is critical that access to physicians with specialized expertise in bleeding disorders are available in all health insurance plans, both private and public. Studies of this disorder group have shown that to receive high quality treatment, patients need access to a comprehensive team of health care specialists to formulate the best treatment regime at the appropriate site of care.

Individuals with bleeding disorders require access to products and treatments prescribed by their health care providers. This includes FDA-approved products as well as novel therapies and bypassing agents for individuals with hemophilia and inhibitors.

Clotting factor products fall in a class commonly known as "specialty drugs," which because of their properties, require specialized storage and handling. Given these special handling and storage requirements, someone with hemophilia cannot go to a traditional neighborhood pharmacy to obtain their life-saving medications.

Instead, they receive their medication from what is known as a specialty pharmacy.

A woman may go, on average, 16 years from the onset of symptoms to a bleeding disorders diagnosis. Bleeding disorders — especially hemophilia — have long been considered a boy’s disease but can also affect women. von Willebrand disease is extremely under-diagnosed. It is estimated that 1–2% of the total population has von Willebrand disease, and many suffer many years before diagnosis. We need to improve access to women’s healthcare. Many girls are put on birth control, women have unnecessary hysterectomies, or almost lose their lives.

Bleeding Disorders put a lot of stress on the entire family. In a study done in 2022, more than 1/3 of patients with a bleeding disorder reported a connection to a mental health condition. We know this number has continued to rise. Some patients require residential mental health care, and although their bleeding disorder treatment is stable and self-administered, they are denied admission because of their bleeding disorder. 

MEDICAID — Educating the community about the importance of keeping information current and reviewing Medicaid benefits.

PREFERRED DRUG LIST — April 24, 2026 — Drug Utilization Review.

It is essential that ALL hemophilia medications remain included on the Preferred Drug List.

In 2023, HB 999 eliminated copay accumulator programs. Now, insurance plans are implementing copay maximizers. Some health plans classify certain specialty medications as “non-essential” and exclude coverage for our life-saving drug; these “non-essential” medications are then placed into a copay maximizer program. The plan sets the patient’s copay at the maximum amount of available copay assistance, while the manufacturer’s copay assistance does not count toward the patient’s deductible or out-of-pocket maximum. As a result, patients take much longer to meet cost-sharing requirements and end up paying thousands of dollars more than the intended annual maximums.

The establishment of a Rare Disease Advisory Council would enhance outcomes for patients with numerous rare conditions, including bleeding disorders. A robust RDAC enables government officials and the rare disease community to collaborate in creating the resources needed to strategically prevent and address barriers, helping affected citizens thrive. A Council includes representation from patients, physicians, stakeholders, hospitals, public health officials, researchers, and patient-led organizations. It provides information on the provider–patient relationship, identifies best practices, and raises awareness to inform and improve public policy.