The Pennsylvania Department of Public Welfare (DPW) is considering the implementation of a "Preferred Drug List" (PDL) for clotting factor concentrates for hemophilia patients on Medicaid. This proposed policy of the DPW, which may designate only a few factor products as "preferred products" and which requires a strict "fail-first" prior authorization, poses grave concerns for consumers with hemophilia and other bleeding disorders. The lack of therapeutic interchangeability of products, in terms of efficacy, tolerance, and adverse drug reactions, could result in life-threatening bleeding. Moreover, limitations recommended by Medicaid on products authorized by insurers, use of mail-order pharmacies, limitations on use of specialty labs, and limits on number of outpatient visits and inpatient hospitalization days may further threaten optimal health outcomes in those with bleeding disorders. Clotting factor products are licensed by the FDA based on specific indications. Implementation of a PDL could require evidence that such a policy results in no poorer medical outcomes. Finally, the lack of equity with privately insured patients will result in substandard access to treatment in a group of patients with substantial pre-existing economic barriers to optimal health care.

For these reasons, MASAC finds a PDL to be unethical and in direct conflict with good medical care. The characteristics of each product and the resultant product choice for an individual patient require a complex decision-making process, with the ultimate product being agreed upon by the patient and his/her healthcare provider. Institution of a PDL would lead to increased costs and predictable deterioration of health in a group of patients whose health and quality of life have improved in the last decade due to the availability of a variety of safe clotting factors which work best when prescribed on an individualized basis.