What is the Bleeding Disorders Advisory Board?
This is a proposal to create a statewide board (or committee) to provide long-term focus and specific expertise in guiding states so they may best serve the bleeding disorders community.
Why is there a need for an advisory board?
Inherited bleeding and clotting disorders are devastating health conditions that can cause serious financial, social and emotional hardships for patients and their families. Hemophilia, which occurs predominantly in males, is a rare but well-known type of bleeding disorder in which one of several proteins normally found in blood is either deficient or defective. Accordingly, the blood does not clot properly and patients may experience internal bleeds into joints and connective tissues resulting in severe pain, swelling and permanent damage to joints and muscles. The Center for Disease Control and Prevention (CDC) estimates that hemophilia affects about 18,000 persons in the United States. There is no known family history of the disorder in about one third of all cases. In such instances, it appears that the disease has developed after a new or spontaneous gene mutation.
Hemophilia is one of a spectrum of chronic bleeding and clotting disorders. von Willebrand disease (VWD), another type of bleeding disorder, is caused by a deficiency or defect in the von Willebrand protein. People with the disorder often bruise easily, have frequent nosebleeds, and may experience heavy bleeding after tooth extraction, tonsillectomy or other surgery. Women with VWD also tend to have prolonged menstrual bleeding. In addition to hemophilia and VWD, there are clotting disorders, a term used to define a class of inherited conditions that increases the tendency for blood clots. The CDC estimates that VWD affects between 1% and 2% of the U.S. population while about 5% to 8% of the population have thrombophilia, a clotting disorder often referred to as the reverse of hemophilia because of the propensity for clotting.
There is no cure for these genetic disorders. However, advancements in the ability to produce safer blood products are allowing persons affected with such disorders to have greater latitude managing their conditions and are minimizing chronic complications including damage to the joints and muscles, the transmission of blood-borne infectious diseases, and chronic liver diseases. As a result, individuals are living longer and are healthier and more productive. While treatment has improved the quality of life, persons with bleeding and clotting disorders today face many challenges as they live longer. One of the major challenges is paying for health care. The cost of clotting factor product, the medication used to treat hemophilia and some types of VWD, ranges between $60,000 and $150,000 per individual per year. The costs are often be much higher for persons with an inhibitor (i.e., immune intolerance to regular clotting factor products) or associated complications such as HIV, AIDS and hepatitis C. Such high costs have made these conditions a primary target of states seeking to manage growing deficits within tight budgets. For example, several states have imposed restrictions to limit their spending on prescription drugs for people enrolled in Medicaid and Medicare. Some of these actions have included:
- Developing preferred drug lists (PDLs)
- Enacting prior authorization requirements for non-PDL drugs
- Denying coverage for recombinant therapies
- Restricting prophylactic use of clotting factor
- Reducing funding for and access to high-risk insurance pools or other similar state insurance options
- Reducing state funding for hemophilia treatment centers (HTCs)
- Denying access to trained hemophilia specialists (particularly for states that mandate participation in managed care)
- Imposing legislation or regulations allowing for discrimination or reduced access to people with bleeding disorders enrolled in private health plans.
All of these limitations could potentially increase medical risk, emergency room visits, and increase the number and duration of hospitalizations. This will result in sub-optimal care for bleeding disorder patients; as such activities undermine the existing model of comprehensive care that has allowed people with bleeding and clotting disorders to live healthy, productive lives. An advisory board will therefore be the link to getting state officials to address the concerns of the bleeding disorders community.
What will the advisory board do?
The proposal suggests that the board review, comment upon, and make recommendations to the Commissioner of Health and the Director of Insurance with regard to:
- legislative or administrative changes to policies and programs, including access to appropriate health insurance or similar health coverage;
- best practices in standards of care and treatment approved by the Medical and Scientific Advisor Council (MASAC) of the National Hemophilia Foundation;
- the establishment of community-based initiatives to disseminate information on services and related activities to the medical and health care community, the academic community, primary caregivers, advocacy associations, and the public; and
- the coordination of public and private support networking systems.
Who will serve on the advisory board?
We recommend that the board consists of the Commissioner of Health or his or her designee and the Director of Insurance or his or her designee, who shall serve as nonvoting members. In addition, we recommend that the board consists of nine (9) voting members appointed by the commissioner in collaboration with the director. The voting members shall be selected from among the following member groups:
- a physician who specializes in the treatment of individuals with hemophilia or related bleeding or clotting disorders;
- a nurse who specializes in the treatment of individuals with hemophilia or related bleeding or clotting disorders;
- a social worker who specializes in the treatment of individuals with hemophilia or related bleeding or clotting disorders;
- a representative of a hemophilia treatment center in this state;
- a representative of a health insurer or other health benefit plan issuer that holds a certificate of authority issued by the state insurance entity;
- a representative of a volunteer or nonprofit health organization that serves the population of this state with hemophilia and related bleeding or clotting disorders;
- a person who has hemophilia or a caregiver of a person who has hemophilia;
- a person who has a bleeding disorder other than hemophilia or a caregiver of a person who has a bleeding disorder other than hemophilia; and
- a person who has a clotting disorder or a caregiver of a person with a clotting disorder.
The proposal also suggests that the board has up to five (5) additional nonvoting members as determined appropriate by the Commissioner and Director. We recommend that nonvoting members be persons with hemophilia, a bleeding disorder other than hemophilia, or a clotting disorder; a caregiver; or persons experienced in the diagnosis, treatment, care and support of people with hemophilia and other bleeding and clotting disorders.
How often will the board meet and how will decisions be handled?
The proposal suggests that the board meet at least four (4) times a year and its conclusions be made available to the public.
How can we get started?
Establishing a Bleeding Disorders Advisory Board in most states will need either legislative or regulatory action. To help with the process, NHF have prepared draft legislation that may be used to start the process.
Support from elected officials is very important. Thus, the most efficient way to start the process is to identify a legislative sponsor and discuss the possibility of him or her introducing the legislation. That individual can connect the group with the office of legislative services who would be able to tell whether the State requires legislative or administrative action to commission such an advisory board. If the he or she agrees to sponsor the bill, staff will be assigned to work with your group and the Legislature’s bill drafting team to prepare a formal draft of the bill. Once the bill language is agreed to by all parties, the bill may then be introduced by the sponsor and co-sponsors.
The first step is always the most difficult, but that's where the staff at the National Hemophilia Foundation can assist you.
Whom should I contact for information on the advisory board legislation?
For further information, contact Ruthlyn Noel, NHF Manager of Public Policy at 212. 328.3730 or firstname.lastname@example.org.