NEW THERAPIES IN PBC
Ocaliva (Obeticholic Acid, OCA), has now been approved by National Institute for Health and Care Excellence (NICE), who have recommended Ocaliva, within its marketing authorisation, as an option for treating primary biliary cholangitis in combination with ursodeoxycholic acid (UDCA) for people whose disease has responded inadequately to UDCA or as monotherapy for people who cannot tolerate UDCA.
NHS England has approved Ocaliva to be prescribed at a number of specialist centres, after each patient has been formally reviewed by a MDT team, against the national clinical treatment and management guidelines. Sites who have not been delegated to run a formal MDT and have not been selected to prescribe Ocaliva can refer potential patients with PBC to the prescribing centres.
UK-PBC would like to capture data on Ocaliva, and hopes to be able to do this by adapting the data collection requirements of the UK-PBC Genetics Study, which most sites are already taking part in. The MDT forms which are attached in the Download section are a template of the data UK-PBC would like to capture which University Hospital Birmingham NHS Foundation Trust has created and adopted. Prescribing MDT centres are welcome to use and adopt these forms.
Patients being referred to a prescribing MDT centre do not have to be enrolled to the UK-PBC Genetics study, although UK-PBC would be grateful if they are given the opportunity to do so. The recruitment process for UK-PBC Genetics Study for patients being considered for Ocaliva is exactly the same for any other patients with PBC. If you would like help or infomation regarding the UK-PBC Gentics study, please contact the UK-PBC Genetics Study Project Manager, Nikoletta Varvaropoulou.
FREQUENTLY ASKED QUESTIONS
No. Patients who are not taking part in UK-PBC Genetics Study, or have previously declined participation can still be referred for assessment at the prescribing MDT centres. UK-PBC enrolment status has no effect on Ocaliva eligibility.
UK-PBC simply ask referring centres to offer the patient the option to take part in UK-PBC Genetics study, as this will help with UK-PBC's academic goals. It will also allow UK-PBC to track the outcomes of Ocaliva, for future research.
NHS Engalnd have selected a number of regional treatment centres to set-up and form a formal MDT. The list of centres are available below.
You can access the official NICE recommendation on Ocaliva here.
NICE in its Technology Appraisal (TA 443) published 26th April 2017 has stated 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.
A official NHS referral should be made for the patient to the prescribing centre. The patient will need to attend the prescribing centre, in order to be assessed and prescribed Ocaliva. Clinical follow-up will predominantly remain with the patients local clinical care team. This will be discussed on a case by case basis.
No. Each prescribing centre will undoubtedly adopt their own process to facilitate a specialist MDT. The forms available on this section is provided as a example, and trusts are free to use and adopt these, should they wish to.
The list of specialist prescribing centres have been selected by NHS England. Please contact NHS England for any queries regarding this.
Now that Ocaliva is licensed, patients should be given the opportunity to have licensed drug. This is increasingly important as some patients may have been randomised to the placebo arm. Therefore, all patients on trial drug should be made aware that Ocaliva is now available on the NHS, and given the opportunity to come off trial drug and onto licensed drug, should the patient wish to. Please contact your CRA for the COBALT study for further information.
PRESCRIBING MDT TRUSTS
- Aintree University Hospital NHS Foundation Trust
- The Newcastle Upon Tyne Hospitals NHS Foundation Trust
- Leeds Teaching Hospitals NHS Trust
- Hull And East Yorkshire Hospitals NHS Trust
- Sheffield Teaching Hospitals NHS Foundation Trust
- York Teaching Hospital NHS Foundation Trust
- Royal Liverpool And Broadgreen University Hospitals NHS Trust
- Central Manchester University Hospitals NHS Foundation Trust
- Nottingham University Hospitals NHS Trust
- University Hospitals Of Leicester NHS Trust
- University Hospitals Birmingham NHS Foundation Trust (includes QEH)
- Cambridge University Hospitals NHS Foundation Trust
- Royal Free London NHS Foundation Trust
- Barts Health NHS Trust
- King's College Hospital NHS Foundation Trust
- Imperial College Healthcare NHS Trust
- Royal Surrey County Hospital NHS Foundation Trust
- Oxford University Hospitals NHS Foundation Trust
- University Hospital Southampton NHS Foundation Trust
- Portsmouth Hospitals NHS Trust
- University Hospitals Bristol NHS Foundation Trust
PRURITUS AND OBETICHOLIC ACID (OCALIVA): A PRACTICAL GUIDE FROM UK-PBC
Obeticholic Acid (Ocaliva) is the licensed second-line therapy recommended for use in patients with Primary Biliary Cholangitis (PBC) who show an inadequate response to treatment with the first line agent Ursodeoxycholic Acid. Patients showing an inadequate response to UDCA are at increased risk of death or needing liver transplant meaning, that enhancing their treatment is an important Part of clinical PBC management.
Clinical trials of Ocaliva have suggested that its use can be complicated by pruritus (itching). Pruritus is itself a symptom of PBC, and where it is reported with Ocaliva use it has the same characteristics as the itch seen in other patients suggesting that the drug has the potential to worsen PBC itch.
Ocaliva itch can either occur de novo or worsen itch is patients with pre-existing itch. It should therefore be considered in all patients in whom Ocaliva may be a treatment option.
Ocaliva can be used safely and effectively in most patients with itch, and this document provides practical advice which will help with optimal use. The need to effectively treat patients with UDCA non-response because of their enhanced risk means it is important to not discount the use of Ocaliva if they have pruritus.
STEP 1: BEFORE PRESCRIBING OCALIVA
• All patients who are being considered for Ocaliva should have their pre-treatment pruritus status assessed. In addition to asking whether is present assess its severity (many patients have itch but it is not troublesome and does not warrant treatment). Severity markers include:
a) Severity of 5 or more on a scale of 1-10
b) Pattern of recent worsening
c) Patient feels it needs treatment (or if already treated treatment increased)
d) Sleep disturbance is a feature
• Record the pruritus treatment currently being received and history of response/non-response to agents
• If pruritus is present and significant: consider bringing pruritus under control before starting Ocaliva. Cholestyramine is not normally effective. The EASL CPG-recommended 2nd line agent rifampicin is the drug of choice (150mg daily with liver biochemistry checked at 2-4 weeks and titrated up to a maximum of 600 mg until control is achieved. If rifampicin is contraindicated, not tolerated or ineffective consider naltrexone therapy. Start the itch regime at least 4 weeks before starting the Ocaliva.
STEP 2: STARTING OCALIVA
• In patients with no or minor itch start as per the standard Ocaliva protocol and
• In patients with pre-exisiting signficant itch, or on treatment for pruritus start at reduced dose of Ocaliva 5mg 3 times a week (5mg once per week as per the standard protocol if advanced cirrhotic- Child Pugh A with portal hypertension; Child Pugh B or C)
STEP 3: AFTER STARTING OCALIVA
• All patients should have their itch assessed following Ocaliva commencement (Aim to have a telephone update by 4 weeks normally, 1-2 weeks if previous itch).
• If itch develops and is clinically not significant (using criteria above): Watch for worsening 2 weekly.
• If itch develops and is clinically significant (using criteria above): Take the following steps
a) If not on second line anti pruritic therapy add rifampicin using above protocol (naltrexone if unable to take rifampicin). If effective continue with OCA at current dose and tritrate up rifampicin as needed
b) If rifampicin/naltrexone is not effective reduce the OCA dose to 5mg 3 times per week (or if on 5mg 3 times per week to 5mg once per week)
c) If pruritus still an issue discontinue Ocaliva, bring pruritus in control using the approach outlined in STEP 1 then consider re-starting Ocaliva
• If Ocaliva was started at 5 mg 3 times per week because of concern about pruritus but no pruritus is seen tritrate Ocaliva up to 5mg daily after 4 weeks
STEP 4: IF YOUR PATIENT IS STILL HAVING PROBLEMS
• Refer the patient to a centre with a specialist interest in pruritus management.