Clinical Commissioning Policy Statement: Siklos In Sickle Cell Anaemia December 2012 Reference : NHSCB/B8/2
NHS Commissioning Board Clinical Commissioning Policy Statement: Siklos In Sickle Cell Anaemia First published: December 2012 Prepared by the NHS Commissioning Board Clinical Reference Group for Haemoglobinopathies Crown copyright 2012 First published December 2012 Published by the Commissioning Board, in electronic format only. 2
POLICY STATEMENT: Siklos In Sickle Cell anaemia Policy Ref: NHSCB/ B8/2 Treatment: For: Background: Siklos is a medicine that contains hydroxycarbamide as its active ingredient. Nordic Pharma UK. Sickle-cell disease (SCD) in adults and children. SCD is caused by a structural abnormality of haemoglobin resulting in deformed, less flexible red blood cells. Acute complications in the more severe forms include sickle-cell crisis, where infarction of the microvasculature and blood supply to organs results in severe pain. A sickle-cell crisis often requires hospitalisation, intravenous fluids, analgesia, and treatment of any concurrent infection. Chronic complications include skin ulceration, renal failure, and increased susceptibility to infection. Sickle cell disease (SCD) and its variants are genetic disorders resulting from the presence of a mutated form of hemoglobin, hemoglobin S (HbS). It is the most common genetic disorder identified by the newborn screening programme in England. There are an estimated 15,000 patients with SCD in England. Every year, an additional 361 children are born with SCD. About 60 70% of the individuals with SCD live in the London area. The gene responsible is found most often in people of African descent and with much less frequency in eastern Mediterranean and Middle East populations. The male to female ratio is 1:1. SCD causes significant morbidity and mortality. Approximately half the individuals with homozygous HbS disease experience vaso-occlusive crises. The frequency of crises is extremely variable. Some individuals have as many as 6 or more episodes annually, whereas others may have episodes only at great intervals or none at all. Each individual typically has a consistent pattern for crisis frequency. SCD is one of the most common causes of hospital admissions in London. In the literature, it has been reported to be associated with significant paediatric and adult healthcare utilization in particular, high rates of emergency department utilization as a result of painful crises and the need for blood 1, 2, 3, 4, 5. transfusions. 3
Commissioning position: Effective from: Evidence summary: Equality impact: Responsible CRG: Date approved by NHS CB Clinical Assurance Group: Date approved by NHSCB Board: The NHS Commissioning Board (NHS CB) has determined that the product Siklos will not be routinely commissioned by the NHSCB for the treatment of sickle cell disease. 1 April 2013 Siklos has been granted European Marketing Authorisation for both adults and children. It is produced by a different company to existing preparations. The evidence presented to the European Medicines Agency (EMEA) on the effectiveness of hydroxycarbamide was based on the evidence in the literature which used the existing product. New effectiveness studies were not carried out by the company seeking authorisation for Siklos. The EMEA document states: Because hydroxycarbamide is a well-known substance that is already used in other medicines, the company used data from the scientific literature to support the use of Siklos in adults and children with sickle cell syndrome. 6 Siklos comes in 100 mg and 1000mg tablet forms and is significantly more expensive than generic hydroxycarbamide (See tables 1 and 2 in Appendix 1) In summary, Siklos contains hydroxycarbamide as its active ingredient. There hasn t been any research to indicate Siklos is a more clinically effective drug. Its cost is significantly more than that quoted for hydroxycarbamide. The NHS Commissioning Board (NHS CB) is committed to ensuring equality of access and non-discrimination, irrespective of age, disability, gender reassignment, marriage and civil partnership, pregnancy and maternity, race, religion or belief, sex (gender) or sexual orientation. In carrying out its functions, the NHS CB must have due regard to the different needs of different protected equality groups. This applies to all the activities for which they are responsible, including policy development, review and implementation. Haemoglobinopathies CRG December 2012 4
Policy review date: Version: Supersedes: To be confirmed 1 N/A Responsible officer/contact: Distribution/Target audience: References 1. Steiner CA, Miller JL. Sickle cell disease patients in U.S. hospitals, 2004. In: HCUP Statistical Brief. Rockville, MD: Healthcare Cost and Utilization Project, Agency for Healthcare Research and Quality; 2006. 2. Bilenker JH, Weller WE, Shaffer TJ, et al. The costs of children who have sickle cell anemia: preparing for managed care. J Pediatr Hematol Oncol. 1998;20:528-533.. 3. Epstein K, Yuen E, Riggio JM, et al. Utilization of the office, hospital and emergency department for adult sickle cell patients: a five-year study. J Natl Med Assoc. 2006;98(7):1109 1113. 4. Yusuf HR, Atrash HK, Grosse SD, et al. Emergency department visits made by patients with sickle cell disease: a descriptive study, 1999 2007. Am J Prev Med. 2010;38(4 Suppl):S536 541. 5. Davis H, Moore RM Jr, Gergen PJ. Cost of hospitalizations associated with sickle cell disease in the United States. Public Health Rep. 1997;112:40-43. 6. European Medicines Agency. Siklos: hydroxycarbamide EMA/762197/2010. EMEA/H/C/000689. Available from: http://www.ema.europa.eu/docs/en_gb/document_library/epar_- _Summary_for_the_public/human/000689/WC500050498.pdf Accessed 04/12/12. 7. British National Formulary No 63, March 2012. Available from: http://www.medicinescomplete.com/mc/bnf/current/127938.htm#_127938. Accessed September 2012. 8. British National Formulary No 63, March 2012. Available from: http://www.medicinescomplete.com/mc/bnf/current/127938.htm#_1. Accessed September 2012. 5
Appendix 1 Table 1: BNF costs for Hydroxycarbamide and Siklos Drug Cost per 1000mg dose Generic Hydroxycarbamide 7 20.94p Siklos 8 16.67 Table 2: Comparative Costs of Siklos and Generic Drug Age/weight Usual maintenance dose Daily dose Daily cost generic ( ) Daily cost Siklos ( ) Annual cost generic ( ) Annual cost Siklos ( ) 10 years/30kg 15mg/kg 450mg 0.111 (based on dose from 500mg capsule and make into suspension) 8.33 (based on half a tablet; this does not exceed the max daily dose of 35mg/kg)) 40.52 3040.45 10 years/30kg 30mg/kg 900 0.222 (based on dose from two 500mg capsules and make into suspension) 16.67 (rounding dose up to 1000mg daily; this does not exceed mad daily dose of 35mg/kg) 81.03 6084.55 Adult/62kg (av. weight of male and female) 15mg/kg 930 0.222 (based on dose from two 500mg capsules and make into suspension) 16.67 (rounding dose up to 1000mg daily; this does not exceed mad daily dose of 35mg/kg) 81.03 6084.55 Adult/62kg (av. weight of male and female) 35mg/kg 2170 0.555 (based on dose from five 500mg capsules and make into suspension). 33.34 (rounding dose down to 2000mg daily) 202.58 If dose rounded down to 2000mg daily, annual cost of 162.06 12,169.10 6