Introduction

CHRISTINE LEE

Professor of Haemophila, Royal Free Hospital NHS Trust

Haemophilia is a rare and expensive condition. Overthe past 20 years, the amount of clotting factorconcentrate used per patient has increased, and boththe quality of the clotting factors and methods ofadministration have improved. In 1991 an internalmarket of `providing' hospitals and `purchasing'health authorities was set up in the National HealthService in the United Kingdom. At the Royal FreeHospital NHS Trust, 14% of the haemophilicpopulation of England and Wales is cared for andit became increasingly dif®cult to cover the escalatingand unpredictable costs of the expenditure on hae-mophilia treatment within the block contracts. As aresult, the hospital trust and purchasers were ®ndingit increasingly dif®cult to ensure that patients withhaemophilia received appropriate care whilst sus-taining the level of service in other specialities. Afurther strain resulted from the publication ofguidelines by the UKHCDO in January 1997, whichrecommended that `Recombinant factor VIII is thetreatment of choice for all patients' although priorityfor the introduction of recombinant factor VIII wasfor previously untreated patients and children. Thisresulted in a chaotic situation in the United Kingdomwhere the treatment afforded for patients withhaemophilia was related to where they lived ±`treatment by postcode'.

It was against this backdrop that a conference washeld at the King's Fund and was participated in byproviders of haemophilia care, purchasers of thatcare and the patients. This supplement contains thepresentations and a summary of the discussions. Asoften happens, the events have subsequently beenovertaken by the political situation and with theadvent of the new Labour administration on 1 May1997, the Minister of Health, Frank Dobson, veryrapidly became involved in issues of haemophiliacare. There was also a link with the vexed problem ofBSE and new variant CJD which has proved to be anenormous ®nancial and political liability within theUnited Kingdom. On 17 March 1998, a health

circular was published by the National HealthService Executive which stated that `Chief Executivesof NHS health authorities and trusts should take thenecessary steps to ensure that, as soon as possiblewithin the new ®nancial year beginning 1st April1998, new patients with haemophilia A (thosepreviously untreated with plasma-derived products),and patients with haemophilia under the age of 16,have access to recombinant factor VIII, where thepatient's clinician recommends that treatment.' Thisdecision was made `While fully recognising that therisk of new variant CJD transmission through bloodproducts is theoretical and unquanti®able, giventheir past experience of HIV and hepatitis C trans-mission through plasma-derived factor VIII, theSecretary of State considers this new fear had raisedparticular concerns for people with haemophilia A.Accordingly, in response to strong representationsfrom the Haemophilia Society, particularly in rela-tion to the effect which such fears have [sic] onfamilies with haemophilic children, the secretary ofstate decided that recombinant factor VIII should bemade available to all those under the age of 16 whoare not already receiving it and to new patients,(those previously untreated with plasma-derivedproducts).'

Thus the politicians have certainly removed manyof the stresses from the providing units in establish-ing a clari®cation of categories of patients whoshould be treated with the most expensive clottingfactor concentrate. However, the escalating andunpredictable costs mean that it may be necessaryto revert to some form of central funding togetherwith a careful audit of effectiveness and quality ofoutcome.

Although this supplement reports the experience offunding this high-cost, low-volume service within theNHS in the UK, the issues discussed are relevant toany health-care system and, therefore, we hopereaders will be interested to read the report of thismeeting.

Haemophilia (1998), 4, (Suppl. 1), iii

Ó 1998 Blackwell Science Ltd iii