Putting NICE guidance into practice. Resource impact report: Obeticholic acid for treating primary biliary cholangitis (TA443)

Transcription

1 Putting NICE guidance into practice Resource impact report: Obeticholic acid for treating primary biliary cholangitis (TA443) Published: April 2017

2 Summary Obeticholic acid is recommended as an option for treating primary biliary cholangitis subject to criteria in section 1 of this report. It is estimated that around 2,700 people with primary biliary cholangitis will be eligible for treatment with obeticholic acid each year. It is estimated that around 1,600 people will have obeticholic acid from year 5 onwards. Table 1 Estimated number of people in England having obeticholic acid 2017/ / / / /22 Uptake of obeticholic acid 9% 30% 41% 50% 59% Population having obeticholic acid each year ,100 1,300 1,600 This report is supported by a local resource impact template because the list price of obeticholic acid has a discount that is commercial in confidence. The discounted price of obeticholic acid can be put into the template and other variables may be amended. This technology is commissioned by NHS England. Providers are NHS hospital trusts. (April 2017) 2 of 8

3 1 Introduction 1.1 This report looks at the resource impact of implementing the NICE guidance on obeticholic acid for treating primary biliary cholangitis in England. 1.2 The guidance states that: Obeticholic acid is recommended, within its marketing authorisation, as an option for treating primary biliary cholangitis in combination with ursodeoxycholic acid for people whose disease has responded inadequately to ursodeoxycholic acid, or as monotherapy for people who cannot tolerate ursodeoxycholic acid. Obeticholic acid is recommended only if the company provides it with the discount agreed in the patient access scheme. Assess the response to obeticholic acid after 12 months. Only continue if there is evidence of clinical benefit. 1.3 The Department of Health and Intercept Pharma have agreed that obeticholic acid will be available to the NHS with a patient access scheme which makes it available with a discount. The size of the discount is commercial in confidence. It is the responsibility of the company to communicate details of the discount to the relevant NHS organisations. Any enquiries from NHS organisations about the patient access scheme should be directed to Ruth Nasr on or ruth.nasr@interceptpharma.com. 1.4 This report is supported by a resource impact template. The template aims to help organisations in England, Wales and Northern Ireland plan for the financial implications of implementing the NICE guidance by amending the variables. 1.5 This technology is commissioned by NHS England. Providers are NHS hospital trusts. (April 2017) 3 of 8

4 2 Background and epidemiology of primary biliary cholangitis 2.1 Primary biliary cholangitis affects around 17,000 people in England. Around half of people with primary biliary cholangitis will not have a diagnosis because it is not always symptomatic. 2.2 Standard treatment for primary biliary cholangitis is ursodeoxycholic acid. 2.3 People who cannot tolerate ursodeoxycholic acid, or their disease does not respond to it, currently have no other treatment options. Their disease usually progresses rapidly and they are likely to die from it within 5 to 7 years. Table 2 Number of people eligible for treatment in England Population Percentage Number of people Adult population 43,108,471 Prevalence of primary biliary cholangitis a ,200 People with a diagnosis of primary biliary cholangitis b 52 9,000 People who are receiving treatment c 92 8,200 People who have inadequate response to ursodeoxycholic acid (25% of 25 2,100 8,200) d People who cannot tolerate ursodeoxycholic acid (7.5% of 8,200) d Total eligible population 2,700 Total number of people estimated to have obeticholic acid each year from year 5 c a Carey EJ, Ali AH, Lindor KD. Primary biliary cirrhosis. Lancet ,600 b Kurtovic J, Riordan SM, Williams R. The natural history of asymptomatic primary biliary cirrhosis. QJM c Company submission d Corpechot C, Abenavoli L, Rabahi N, Chretien Y, Andreani T, Johanet C, et al. Biochemical response to ursodeoxycholic acid and long-term prognosis in primary biliary cirrhosis. Hepatology (April 2017) 4 of 8

5 2.4 It is estimated that around 2,700 people will be eligible for treatment with obeticholic acid each year. 2.5 From year 5 it is estimated that 1,600 people will have treatment with obeticholic acid each year, once uptake has reached 59%. 3 Assumptions made 3.1 The resource impact template makes the following assumptions: 25% (midpoint of 20%-30%) of people will have inadequate response to ursodeoxycholic acid 7.5% (midpoint of 5%-10%) of people will not tolerate ursodeoxycholic acid people who respond to ursodeoxycholic acid are excluded from the template because they are unaffected by the recommendations best supportive care has no cost in the resource impact template because it is assumed that all people have this. 4 Resource impact 4.1 The list price of obeticholic acid has a discount that is commercial in confidence. The discounted price of obeticholic acid can be put into the template to calculate the resource impact of the guidance. 4.2 The current treatment and future uptake figure assumptions are based on expert opinion and are shown in the resource impact template. Table 3 shows the number of people who we estimate will have obeticholic acid by financial year. (April 2017) 5 of 8

6 Table 3 Population estimated to have obeticholic acid in England using NICE assumptions 2017/ / / / /22 Uptake of obeticholic acid 9% 30% 41% 50% 59% Population having obeticholic acid each year ,100 1,300 1,600 5 Savings and benefits 5.1 Obeticholic acid reduces the requirement for people to have a liver transplant and therefore reduces the numbers on the waiting list for this procedure. 5.2 This drug meets a currently unmet need for people with primary biliary cholangitis who do not tolerate ursodeoxycholic acid, or whose disease does not respond adequately to it. 6 Implications for commissioners 6.1 Obeticholic acid falls into programme budgeting category 13C primary biliary cholangitis. 6.2 People with untreated primary biliary cholangitis will quickly die without a liver transplant. It is estimated that the introduction of obeticholic acid will reduce the number of liver transplants needed for people with primary biliary cholangitis, reducing the demand for donor livers and enabling more liver transplants to take place in other diseases. 7 Other considerations 7.1 Currently around 29% of people who do not respond adequately to ursodeoxycholic acid and 31% of people who cannot tolerate (April 2017) 6 of 8

7 ursodeoxycholic acid have no further treatment options and therefore require a liver transplant. 7.2 Around 5% of people who will undergo treatment with either ursodeoxycholic acid, obeticholic acid or a combination of both will require a liver transplant. 7.3 The addition of obeticholic acid as an option as either a monotherapy for people who cannot tolerate ursodeoxycholic acid or in combination with ursodeoxycholic acid for people who do not respond to ursodeoxycholic acid monotherapy has the potential to reduce the number of liver transplants performed for primary biliary cholangitis. 7.4 No costs or savings have been included in the template for any reduction in liver transplants as a result of implementing the guidance. The demand on donor livers is such that reducing the number of transplants performed for primary biliary cholangitis simply frees up donor organs to be used for transplants in other conditions and reduce waiting lists; the total number of liver transplants performed each year will not change. (April 2017) 7 of 8

8 8 About this resource impact report This resource impact report accompanies the NICE guidance on obeticholic acid for treating primary biliary cholangitis and should be read in conjunction with it. See terms and conditions on the NICE website. National Institute for Health and Care Excellence, All rights reserved. (April 2017) 8 of 8