Treatment of haemophilia has evolved dramatically in the past forty years. With the discovery in the 1960s that coagulation factor VIII was present in the cold-precipitable fraction of plasma proteins (cryoprecipitate), the possibility of effective treatment started to become a reality. With later development of more highly purified concentrates of factor VIII (haemophilia A) and factor IX (haemophilia B), health professionals and affected families began to grapple with the issues of stopping bleeding into joints, muscles and other critical sites and rolling back the many barriers to life opportunities that arise from this condition.

Heady progress in haemophilia treatment continued during the 1970s and early 1980s. Larger quantities of coagulation-factor concentrates became available and it was soon recognised that prevention of crippling joint disability would require home-based prophylactic treatment starting in early childhood, rather than treatment of acute bleeding after it had started. Serious joint bleeding episodes, even if few in number, typically lead to progressive joint deterioration as further bleeding occurs more readily in affected ‘target joints’.1 This early progress was abruptly slowed by the coincidental worldwide spread of HIV infection and recognition that the technology used to manufacture treatment products at that time was not adequate to the task; HIV, hepatitis C and other viral infections could potentially be transmitted by all of the therapeutic products then in use.2

Subsequent developments occurred on two fronts. Blood product manufacturers developed methods to disinfect coagulation-factor products and these provided the margin of safety needed for clinical use. Second, genetic engineering provided recombinant human factor VIII and IX using mammalian (non-human) cell culture systems to express the protein. Further development of genetic engineering approaches is likely to see gene transfer become available, though probably not before the end of this decade.

A concurrent development in the last thirty years has been the growth of influence of strong patient-support groups in the form of the World Federation of Haemophilia and various national haemophilia societies and foundations. These have provided a forum for information on haemophilia and good management programmes. They have also represented a strong lobby for access to financial and other resources for haemophilia. The NZ Government, in response to approaches from clinicians and the Haemophilia Society, convened a review of haemophilia treatment by Coopers and Lybrand in the mid-1990s. The review found that significant economic benefits would arise from introduction of prophylactic treatment for children as affected individuals would have a good chance of entering adult life with no or minimal joint disability. Subsequently, funding was made available for expanded prophylactic treatment within the framework of hospital-based haematology services. We are now able to see the results of this programme in that many affected children have limited or no joint disability.

The paper by Harper et al in this issue3 highlights an important issue that has arisen: the cost of haemophilia treatment is high and is likely to rise further as the current population of affected children grow physically. Treatment doses are based largely on patient weight, and a population of growing teenagers will require more treatment product if current treatment practices are maintained. Annual costs per teenage patient on regular prophylaxis treatment often exceed $100 000 per annum, and may be much higher for patients with inhibitor antibodies that affect coagulation factors.

There has been much sympathy for the plight of people with haemophilia. People with severe haemophilia have a condition that will cause disabling physical problems if poorly treated or if complications arise, some have suffered the consequences of iatrogenic HIV, most over the age of 20 years have chronic hepatitis C infection, and some are carriers of hepatitis B. However, they are not alone in experiencing potentially disabling health problems and competition for access to health dollars is more intense than ever as publicly funded health services are curtailed.

A key issue facing all concerned is that loss of access to effective treatment will result in long-term consequences – lost ground cannot be recovered. Reducing the quality of care in teenage and young-adult years may mean spending more later – in prophylaxis, surgery, unemployment or sickness benefits, and personal costs. Unilateral cost shifting to other areas of the health and social support structure may be attractive to managers trying to manage this year’s budget but will be disastrous for people with haemophilia if they develop target joints.

Currently, spending on haemophilia treatment is producing stress in most if not all of the major hospitals in New Zealand as the costs typically exceed the available budgets. Options to move to centralised purchasing of treatment products, and possibly a form of broader oversight of individual treatment programmes, may achieve savings but are unlikely to be effective in bringing budgets into line. These steps at best will tinker with the cost issue, at worst they may lead to more bureaucracy in the clinical process.

The treatment expectations of patients and the NZ Haemophilia Foundation have been derived from haemophilia treatment programmes reported from countries that have larger budgets than those available in NZ. Many of those countries are now also reporting significant issues in health resource allocation. In addition, even the more generous treatment programmes will not avoid joint disability in all patients. When the available treatment studies are evaluated, it is apparent that specific issues have been evaluated, but only a moderate understanding exists of the long-term effects of different dosage schedules and there is little understanding of the variation in need of individual patients.4 There is also a dearth of studies that have evaluated cost effectiveness in haemophilia programmes, although they are now starting to emerge.5–7 Finally, these expensive treatment products have relatively rapid clearance after injection, but prophylaxis is mostly being administered every two or three days. Should they be given in smaller, more frequent doses as a means of limiting costs? If so, what would be the personal stress issues for patients and families in the home environment?

For those who treat haemophilia bleeding problems there is no time available for further study as budget management pressures on haemophilia treatment are at a crisis point. Now, more than ever, there is a need for the various interests to work together to achieve an effective solution for this serious health issue. Without a good measure of leadership from the respective sectors, and cooperative trust in this process, much of the potential benefit for people with haemophilia may be lost.

Author information: Jim M Faed, Senior Lecturer, Department of Pathology, University of Otago, Dunedin

Correspondence: Dr J M Faed, Department of Pathology, University of Otago, P O Box 913, Dunedin. Fax: (03) 479 7136; email: jim.faed@stonebow.otago.ac.nz