Economic Implications For One Rare Disease
In the fifth century B.C, Hippocrates described a terrible triad of iridocyclitis, genital and aphthous ulceration that swept through ancient Greece. (1) Consequently, this triad was reported by others, but it was not truly recognised in the medical world until it was described by Hulusi Behcet in 1937. (2) Today, this triad is known as Behcet’s Disease (BD). Now further characterised by several other clinical features, BD is considered a multifactorial condition associated with widespread inflammation. Inflammation may involve the eyes, gastro-intestinal tract, nervous system, cardiovascular system, joints and skin.(2) Despite, first being described in Greece, the Silk roads claim the highest prevalence of BD.(2) In the case of the UK, approximately 0.64 per 100,000 individuals are thought to have BD. (3) In this extract, I would discuss the impact a limited evidence base has on funding for expensive therapies to treat BD. I hope after reading, you the reader will gain an appreciation for the economic difficulties encountered by patients with BD and to an extent other rare diseases.
The evidence base for treatment of BD is quite limited. (28,29) In a 2014 paper by Barry et al they revealed a gap in the data, particularly for cardiovascular, neurological and gastrointestinal disease. (28) This is illustrated below by graph 1. (28)
In addition, a Cochrane review assessing the evidence for the use of ‘biologics, colchicine, corticosteroids, immunosuppressants and interferon alpha’ in the treatment of neuro-Behcet’s yielded no results supporting or opposing their use.(30) With regards to treatment of oral ulceration in BD there was a variety of low quality studies supporting therapeutic interventions.(31) The prognosis is BD largely dependents on the presence of ‘ocular, neurological and vascular manifestations.(18) It is therefore important that physicians have the relevant information to inform their practise; this however, is challenging as the research is either non-existent or of low quality.
The impact of having not only limited but low level evidence has far reaching implications, particularly with regards to funding. One of the main determining factors of whether funding would be made available is cost-effectiveness. (32) With regards to biologics, they are not currently indicated to treat BD. (33) Furthermore, if it was indeed indicated for use, it is unlikely that the cost-effective analysis would be in favour of providing funds. One of the reasons for this is that the National Health Service has finite resources which must be distributed to obtain maximum value for money.(32) BD being a rare disease and biologics such as infliximab and interferon alpha costing £16,000 per year and £4000 year respectively would result in a large diversion of funds to treat a minority of individuals.(29) Additionally, the evidence supporting the use of biologics is limited.(28,29) There is however, a ray of hope for funding, as considerations are made for rare diseases where deriving robust evidence may not be feasible.(32) Also, patients can receive funding for biologics if a drug request form and outcome-record baseline form are approved.(32) The implications for funding is indeed challenging; however, obtaining funding is not impossible.
With finite funding choosing the right drugs to fund is important. Currently, infliximab is preferred to interferon alpha when biologics are prescribed.(33) However infliximab cost £12,000 per year more and perhaps may have a similar or even worst prognosis for the patient.(29) Therefore, to ensure value for money a comparison should be carried out.(29) A gene polymorphism found in patients with hepatitis B and C predict a favourable outcome when interferon alpha is used.(29) Currently, a study to compare the effects of infliximab with interferon with regards to the presence of this polymorphism is being proposed.(29) This should hopefully pilot other studies and further inform both practise and allocation of funding.
