Treatment for haemophilia by postcodeBMJ 1997; 314 doi: https://doi.org/10.1136/bmj.314.7082.749 (Published 08 March 1997) Cite this as: BMJ 1997;314:749
- a United Kingdom Haemophilia Centre Directors Organisation, Haemophilia and Thrombosis Centre, Royal Infirmary, Edinburgh EH3 9YW
- b Haemophilia Centre, Royal Infirmary, Manchester
- c Haemophilia Centre, Queens Medical Centre, Nottingham
Editor—Recently the United Kingdom Haemophilia Centre Directors Organisation issued evidence based guidelines on therapeutic products for treating haemophilia. Before finalisation the draft guidelines were circulated to professional societies, royal colleges, and departments of health and modified accordingly. The final guidelines were unanimously approved at the organisation's annual geneal meeting and published.1
One recommendation was that the treatment of choice for haemophilia A was recombinant factor VIII concentrate (rFVIII). This is because plasma derived factor VIII concentrates (pdFVIII) still transmit viral infections—for example, those caused by non-lipid coated viruses such as hepatitis A and parvovirus—despite the incorporation of viral inactivation steps in the manufacturing process.2 3 At present the licenced rFVIIIs contain human albumin as a stabiliser but there are no reports of viral transmission by it and manufacturers are developing non-plasma derived stabilisers. Despite concern that rFVIII may contain neoantigens, the incidence of anti-factor VIII antibodies is probably similar to that after the use of pdFVIII concentrate.
rFVIII is more expensive than pdFVIII. Value added tax has been imposed recently, thus increasing the cost by a further 17.5%. As we recognised that purchasers would probably not want to change all patients immediately to rFVIII the guidelines suggested those patients who should be offered it first: those who had not been exposed previously to pdFVIII (mostly babies with severe haemophilia) and those not infected with hepatitis C virus (mostly children). This policy is in keeping with practice in Western Europe and North America, where about half of all therapy is with rFVIII concentrate.
The implementation of the recommendations on rFVIII by purchasers has been variable. For example, in the south of England, Scotland, and Northern Ireland rFVIII is becoming available for the high priority patients. In other areas some directors of public health have stated that rFVIII should not be prescribed, whereas others in neighbouring districts are funding rFVIII so that treatment choice depends on the patient's post code. There are thus widely divergent arrangements for treatment with a drug that is perceived by physicians, patients, and parents to be safer than the cheaper plasma derived concentrate. The consequence is likely to be that some patients will move to districts that do pay for rFVIII, thus increasing the financial burden further for the new purchasers. An intolerable situation will arise when a patient currently receiving rFVIII has to move to a district that does not fund this and he is consequently switched to pdFVIII.
Before the publication of the guidelines the department of health informed all directors of public health in England that purchasers should make their own evaluation of the benefits of rFVIII before allowing it to be purchased. As a result various consortia of purchasers have attempted to evaluate the complex medical, scientific and medicolegal issues related to treatment of haemophilia and to estimate the cost effectiveness of rFVIII in an arbitrary way. Despite a specific request, the department has provided no evidence that rFVIII should not be the treatment of choice nor has it criticised the priority given to different groups of patients. What is needed to help purchasers and trusts with haemophilia centres is for the department to engage in a constructive dialogue with our organisation and provide leadership on how the guidelines should be implemented. This will ensure patients are treated fairly. To leave the decision to the vagaries of local purchasers is to abrogate its responsibility for an important aspect of health care.