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The following recommendation was approved by the Medical and Scientific Advisory Council (MASAC) on April 22, 2006, and adopted by the NHF Board of Directors on June 3, 2006.
Individuals with bleeding disorders are affected by rare diseases that are best cared for by a comprehensive team of specialized dedicated staff who provide a wide range of services with access to appropriate therapies and laboratory testing. The outcome of these individuals is optimized when they are followed in the network of federally recognized comprehensive hemophilia treatment centers (HTCs) throughout the United States.
Growing economic pressures within medical care systems may directly impact access to care for people with bleeding disorders. Such limitations include;
1. Limitation of access to certain medical care systems through creation of networks within managed care organizations which may exclude the federally recognized comprehensive HTCs.
2. Limitation of coagulation factor products and adjunctive medications to treat these disorders through creation of preferred drug lists (PDLs), restricted drug lists (RDLs), formularies, and other restrictive lists. Coagulation factor products are not generic and cannot be viewed as interchangeable (see MASAC Document #165).
3. Limitations on provision of coagulation factor products through pharmacy benefit managers and in-house specialty pharmacies created by payers. In this context, use of pharmacy benefit managers and specialty pharmacies may result in
a. Increased cost for coagulation factor products;
b. Difficulty of access to these products for emergencies, bleeding episodes, or unforeseen events commonly experienced by this population;
c. Required use of a product not prescribed by the treating physician;
d. Less than standard of care for provision and administration of replacement products and ancillary supplies for home based therapy for this population.
4. Limitations on reimbursement of coagulation factor products for either inpatient hospitalizations or long-term care facilities. Reimbursement based upon DRGs without consideration or inclusion in the calculation the cost of the coagulation replacement factor products required during hospitalization may lead hospitals to suffer unacceptable losses; hospitals may therefore limit or discontinue stocking of coagulation factor products to decrease their financial risk requiring transfer of these patients to alternate institutions. At this time, Medicare does not reimburse long-term care facilities for coagulation factor replacement products; this may lead them to refuse to provide these therapies or to refuse admission to patients requiring these therapies.
5. Limitations to timely access to coagulation laboratory testing/data necessary to monitor adequacy of replacement therapy and to make therapeutic decisions. These specialized coagulation assays may be influenced by the time from specimen acquisition to separation of the plasma from the anticoagulated whole blood, and the inability to quality control precisely the handling of the specimen and environmental variables. These issues including the transport time and delays in performance of the coagulation activity assays by the off-site laboratory arrangements mandated by payers or managed care reimbursement programs compromise timely diagnosis and therapeutic management of individuals with coagulation disorders.
All of these limitations may result in sub-optimal care for bleeding disorder patients. Decreased access to optimal care has been shown to increase morbidity and mortality, reducing the affected individuals’ ability to participate in the work force and in society as a whole. Anecdotal and emerging peer reviewed evidence show that these methods to reduce cost of care have in fact negatively affected this population through
1. Increased medical risk;
2. Increased utilization of Emergency Rooms;
3. Increased number and duration of hospitalizations.
Therefore, MASAC recommends that access to care at federally funded HTCs be preserved for this vulnerable population including:
1. Access to the federally recognized hemophilia treatment center;
2. Access to the full range of replacement therapies licensed to treat these disorders;
3. Establishment of a carve-out for coagulation factor products for presently utilized mechanisms when that agency is either less costly than the mandated mechanism or meets the network cost for provision of these therapies (any willing provider);
4. Reimbursement methodologies are adopted for inpatient hospitalizations or care through long-term facilities that cover the cost of these coagulation factor replacement therapies for these patients, thereby preserving access to these institutions when required for this population.
1. Soucie JM, Nuss R, Evatt B, Abdelhak A, Cowan L, Hill H, Kolakoski M, Wilber N. Mortality among males with hemophilia: relations with source of medical care. The Hemophilia Surveillance System Project Investigators. Blood 2000; 96: 437-442.
2. Soucie JM, Symons J 4th, Evatt B, Brettler D, Huszti H, Linden J; Hemophilia Surveillance System Project Investigators. Home-based factor infusion therapy and hospitalization for bleeding complications among males with haemophilia.
Haemophilia 2001; 7:198-206.