New therapeutic products and difficult decisions. The case of recombinant factor VIII in the management of haemophilia A.

New and expensive therapeutic products can place great pressure on health care purchasers. Often, evidence to support the inclusion of such products in the purchasing process is lacking or confusing, yet demand can be organized and forceful. In this paper we use the example of the introduction of recombinant factor VIII (rFVIII) in the management of haemophilia A, to highlight some problems purchasers face in deciding whether to fund its use. The introduction of rFVIII involves substantial extra funding requirements and the benefits afforded by its use are unclear. Although a case can be made for the use of rFVIII on the grounds of subjective furture theoretical risks and intangible benefits, the purchasers of health care are charged with maximizing the present and obtainable benefits of the community at large, given finite resources. Guidelines produced by the United Kingdom Haemophilia Centre Directors Organization state rFVIII to be its treatment of choice for all haemophilia A patients, but offer no insight into the benefits attainable through the use of rFVIII, in terms of health outcomes. We report summary findings of Purchaser Intelligence Groups examining the use of rFVIII and offer comment on the problems associated with the implementation of therapeutic products such as rFVIII.