Source: Pharmac
What we’re proposing
We are seeking feedback on a proposal to widen access to three funded treatments, adalimumab, etanercept and tocilizumab, for the treatment of juvenile idiopathic arthritis (JIA). This is an inflammatory condition that affects the joints of children and young people.
From 1 December 2020 access criteria for adalimumab and etanercept would be widened to include funding for treatment of oligoarticular course JIA and treatment of polyarticular course JIA affecting 5 or more joints. Access criteria for tocilizumab would be widened for polyarticular course JIA affecting 5 or more joints at the same time.
Further details on this proposal, including how to provide feedback, can be found below.
Consultation closes at 5 pm on Friday, 25 September 2020 and feedback can be emailed to consult@pharmac.govt.nz.
What would the effect be?
From 1 December 2020 the current access criteria for adalimumab, etanercept and tocilizumab for treatment of polyarticular course JIA would be redefined to enable treatment of people who have 5 or more affected joints, which is lower than the current requirements. Funded access to adalimumab and etanercept would also be widened to include people with oligoarticular course JIA including extended oligoarticular (eoJIA), juvenile psoriatic arthritis (PsA) and enthesitis-related arthritis (ERA).
Widening access to these treatments would result in reduced joint damage and pain, improve mobility and quality of life and provide some patients with long-term remission. We estimate that approximately 65 patients each year would benefit from this proposal under the proposed criteria for funding.
Who we think will be interested
- People with JIA and their family, whānau or caregivers
- Paediatric rheumatologists, paediatricians, general practitioners, hospital and community pharmacists and other clinicians and health professionals involved in the management of people with JIA
- Consumer support groups for people living with rheumatic conditions
- Pharmaceutical suppliers and wholesalers
About JIA
JIA, formerly called juvenile rheumatoid arthritis, is a chronic inflammatory condition of unknown cause that affects the joints. For people with JIA, joint symptoms typically commence prior to the age of 16 and persist for at least six weeks. People with JIA experience joint pain and inflammation which can become severe and impact mobility, quality of life and puts them at risk of long-term joint damage.
Polyarticular course JIA affects five or more joints at any time and includes two subtypes: rheumatoid factor negative and rheumatoid factor positive.
Adalimumab and etanercept are both currently funded for the treatment of polyarticular course JIA, limited to patients with intolerable side effects from, or insufficient benefit from the alternate agent (adalimumab or etanercept); and for patients with severe active polyarticular course JIA for at least six months, who have not responded to at least three months of methotrexate in combination with either oral corticosteroids or a full trial of serial intra-articular corticosteroid injections, and persistent symptoms of poorly-controlled and active disease in at least 20 swollen, tender joints, or at least four joints of the: wrist, elbow, knee, ankle, shoulder, cervical spine and hip.
Tocilizumab has similar funding restrictions to adalimumab and etanercept in patients who have not previously been treated with adalimumab and etanercept; however, treatment with a tumour necrosis factor alpha inhibitor must be contraindicated. Tocilizumab is also funded for polyarticular course JIA where patients have trialled both adalimumab and etanercept and experienced either intolerable side effects or insufficient benefit.
Oligoarticular course JIA includes a number of different subtypes and encompasses extended oligoarticular (eoJIA), juvenile psoriatic arthritis (PsA) and enthesitis-related arthritis (ERA). The International League of Associations for Rheumatology classifies these subtypes of JIA as:
- eoJIA: where 1-4 joints are involved during the first six months of illness and more than four joints affected after the first six months
- PsA: where children have arthritis and psoriasis, or arthritis and at least two other specific features (e.g. dactylitis, psoriasis in a relative)
- ERA: where children have arthritis and enthesitis, or arthritis or enthesitis with two other specific factors (e.g. sacroiliac joint tenderness, presence of specific antigen).
Adalimumab, etanercept and tocilizumab are not currently funded for patients with oligoarticular course JIA, including eoJIA, PsA and ERA JIA subtypes.
About adalimumab, etanercept and tocilizumab
Adalimumab and etanercept
Adalimumab and etanercept are both tumour necrosis factor (TNF) inhibitors delivered by subcutaneous injection that reduce chronic inflammation and immune response activation. TNF is a naturally occurring cytokine that is involved in normal inflammatory and immune responses, playing an important role in the inflammatory processes of long-term conditions such as rheumatoid arthritis and juvenile idiopathic arthritis.
Adalimumab and etanercept are both currently funded for the treatment of polyarticular course JIA, as described above. This proposal to widen access would reduce the joint count requirement from 20 to 5 (at least 3 of these being joints with limitation of motion), remove the criteria regarding a full trial of serial intra-articular corticosteroid injections and require a trial of methotrexate for either 3 or 6 months depending on disease severity (moderate-high disease activity defined as clinical juvenile arthritis disease activity score (cJADAS) score of at least 2.5 and low disease activity defined as cJADAS score of 1.1 to 2.5).
Adalimumab and etanercept are not currently funded for patients with oligoarticular course JIA, including eoJIA, PsA and ERA JIA subtypes. This proposal would widen access to include oligoarticular course JIA in patients with at least 2 active joints with swelling or limited range of motion, and a trial of methotrexate for either 3 or 6 months depending on disease severity (moderate-high disease activity defined as cJADAS score greater than 1.5 and high disease activity defined as cJADAS score greater than 4).
Tocilizumab
Tocilizumab is a recombinant humanised monoclonal antibody of the immunoglobulin (Ig) IgG1 subclass, which binds to human interleukin 6 (IL-6) receptors, lowering IL-6 levels within the body. IL-6 is involved in the development of immunological and inflammatory reactions, similarly playing an important role in the inflammatory processes of long-term conditions such as rheumatoid arthritis and juvenile idiopathic arthritis. Tocilizumab is delivered by intravenous infusion in a hospital setting.
Tocilizumab is also currently funded for the treatment of polyarticular course JIA, limited to patients with intolerable side effects from, or insufficient benefit from, both adalimumab and etanercept; or for patients where treatment with a TNF inhibitor is contraindicated and the patient has severe active polyarticular course JIA for at least six months, has not responded to at least three months of methotrexate in combination with either oral corticosteroids or a full trial of serial intra-articular corticosteroid injections, and has persistent symptoms of poorly-controlled and active disease in at least 20 swollen, tender joints, or at least four joints of the wrist, elbow, knee, ankle, shoulder, cervical spine, and hip.
This proposal to widen access would reduce the joint count requirement from 20 to 5 (at least 3 of these being joints with limitation of motion), remove the criteria regarding a full trial of serial intra-articular corticosteroid injections, and require trial of methotrexate for either 3 or 6 months depending on disease severity (moderate-high disease activity defined as cJADAS score of at least 2.5 and low disease activity defined as cJADAS score of 1.1 to 2.5).
Why we’re proposing this
A funding application for widened access to adalimumab (Humira) and etanercept (Enbrel) to include patients with polyarticular course JIA with five or more affected joints and to include patients with oligoarticular course JIA (such as eoJIA, PsA and ERA) was reviewed by the Pharmacology and Therapeutics Advisory Committee (PTAC) in November 2019.
PTAC recommended(external link) that access for adalimumab and etanercept be widened for these patient groups with a high priority subject to certain criteria being met (described below). In making this recommendation, PTAC also recommended that the access criteria for tocilizumab be reviewed to enable funded consistency between treatments.
More information, including links to the PTAC records, can be found in the Application Tracker records:
Details about our proposal
Adalimumab, etanercept and tocilizumab would continue to be listed at the same price and subsidy in Section B and Part II of Section H of the Pharmaceutical Schedule.
Access to adalimumab (Humira) and etanercept (Enbrel) would be widened in Section B and Part II of Section H of the Pharmaceutical Schedule to include treatment for polyarticular course JIA in people with five or more affected joints and to include oligoarticular course JIA as follows (additions shown in bold, deletions shown in strikethrough). Note that only the relevant criteria are shown, the full current criteria for these treatments can be found here(external link).
Special Authority for Subsidy
ADALIMUMAB
Initial application – (juvenile idiopathic arthritis) only from a named specialist or rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:
Either:
- Both:
1.1 The patient has had an initial Special Authority approval for etanercept for juvenile idiopathic arthritis (JIA); and
1.2 Either:
1.2.1 The patient has experienced intolerable side effects from etanercept; or
1.2.2 The patient has received insufficient benefit from etanercept to meet the renewal criteria for etanercept for JIA; or
- All of the following:
2.1 Patient diagnosed with Juvenile Idiopathic Arthritis (JIA); and
2.2 To be used as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
2.3 Either:
2.3.1 All of the following:
2.3.1.1 Patient has polyarticular course JIA for 6 months duration or longer; and
2.3.1.2 Either:
2.3.1.2.1 At least 5 swollen joints and at least 3 joints with limitation of motion, pain and tenderness; or
2.3.1.2.2 Moderate or high disease activity (cJADAS score of at least 2.5) after a 3-month trial of methotrexate (at a dose of 10-20 mg/m2 weekly or at the maximum tolerated dose); or
2.3.1.2.3 Low disease activity (cJADAS score between 1.1 and 2.5) after a 6-month trial of methotrexate; and
2.3.1.3 Patient has tried and not responded to a therapeutic trial of oral corticosteroids (prednisone 0.25 mg/kg or at the maximum tolerated dose); or
2.4 Patient has tried and not responded to at least three months of oral or parenteral methotrexate (at a dose of 10-20 mg/m2 weekly or at the maximum tolerated dose) in combination with either oral corticosteroids (prednisone 0.25 mg/kg or at the maximum tolerated dose) or a full trial of serial intra-articular corticosteroid injections; and
2.5 Both:
2.5.1 Either:
2.5.1.1 Patient has persistent symptoms of poorly-controlled and active disease in at least 20 swollen, tender joints; or
2.5.1.2 Patient has persistent symptoms of poorly-controlled and active disease in at least four joints from the following: wrist, elbow, knee, ankle, shoulder, cervical spine, hip; and
2.5.2 Physician’s global assessment indicating severe disease.2.3.2 Both:
2.3.2.1 Patient has oligoarticular course JIA for 6 months duration or longer; and
2.3.2.2 Either:
2.3.2.2.1 At least 2 active joints with swelling or limited range of motion; or
2.3.2.2.2 Moderate or high disease activity (cJADAS score greater than 1.5) with poor prognostic features after a 3-month trial of methotrexate (at a dose of 10-20 mg/m2 weekly or at the maximum tolerated dose); or
2.3.2.2.3 High disease activity (cJADAS score greater than 4) after a 6-month trial of methotrexate.
Special Authority for Subsidy
ETANERCEPT
Initial application – (juvenile idiopathic arthritis) only from a named specialist or rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:
Either:
- Both:
1.1 The patient has had an initial Special Authority approval for adalimumab for juvenile idiopathic arthritis (JIA); and
1.2 Either:
1.2.1 The patient has experienced intolerable side effects from adalimumab; or
1.2.2 The patient has received insufficient benefit from adalimumab to meet the renewal criteria for adalimumab for JIA; or
- All of the following:
2.1 Patient diagnosed with Juvenile Idiopathic Arthritis (JIA); and
2.2 To be used as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
2.3 Either:
2.3.1 All of the following:
2.3.1.1 Patient has polyarticular course JIA for 6 months duration or longer; and
2.3.1.2 Either:
2.3.1.2.1 At least 5 swollen joints and at least 3 joints with limitation of motion, pain and tenderness; or
2.3.1.2.2 Moderate or high disease activity (cJADAS score of at least 2.5) after a 3-month trial of methotrexate (at a dose of 10-20 mg/m2 weekly or at the maximum tolerated dose); or
2.3.1.2.3 Low disease activity (cJADAS score between 1.1 and 2.5) after a 6-month trial of methotrexate; and
2.3.1.3 Patient has tried and not responded to a therapeutic trial of oral corticosteroids (prednisone 0.25 mg/kg or at the maximum tolerated dose); or
2.4 Patient has tried and not responded to at least three months of oral or parenteral methotrexate (at a dose of 10-20 mg/m2 weekly or at the maximum tolerated dose) in combination with either oral corticosteroids (prednisone 0.25 mg/kg or at the maximum tolerated dose) or a full trial of serial intra-articular corticosteroid injections; and
2.5 Both:
2.5.1 Either:
2.5.1.1 Patient has persistent symptoms of poorly-controlled and active disease in at least 20 swollen, tender joints; or
2.5.1.2 Patient has persistent symptoms of poorly-controlled and active disease in at least four joints from the following: wrist, elbow, knee, ankle, shoulder, cervical spine, hip; and
2.5.2 Physician’s global assessment indicating severe disease.2.3.2 Both:
2.3.2.1 Patient has oligoarticular course JIA for 6 months duration or longer; and
2.3.2.2 Either:
2.3.2.2.1 At least 2 active joints with swelling or limited range of motion; or
2.3.2.2.2 Moderate or high disease activity (cJADAS score greater than 1.5) with poor prognostic features after a 3-month trial of methotrexate (at a dose of 10-20 mg/m2 weekly or at the maximum tolerated dose); or
2.3.2.2.3 High disease activity (cJADAS score greater than 4) after a 6-month trial of methotrexate.
Renewal for adalimumab and etanercept (amendments shown in bold and strikethrough):
Renewal – (juvenile idiopathic arthritis) only from a named specialist, rheumatologist or Practitioner on the recommendation of a named specialist or rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:
Both:
All of the following:
Either:
1.1 Applicant is a named specialist or rheumatologist; or
1.2 Applicant is a Practitioner and confirms that a named specialist or rheumatologist has provided a letter, email or fax recommending that the patient continues with [adalimumab/ etanercept] treatment; and
- Subsidised as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
- Either:
2.1 Following 3 to 4 months’ initial treatment, the patient has at least a 50% decrease in active joint count and an improvement in physician’s global assessment from baseline; or
2.2 On subsequent reapplications, the patient demonstrates at least a continuing 30% improvement in active joint count and continued improvement in physician’s global assessment from baseline.
Access to tocilizumab (Actemra) would be widened in Section B and Part II of Section H of the Pharmaceutical Schedule to include treatment for polyarticular course JIA in people with five or more affected joints as follows (amendments shown in bold and strikethrough):
Special Authority for Subsidy
TOCILIZUMAB
Initial application — (polyarticular juvenile idiopathic arthritis) only from a rheumatologist or Practitioner on the recommendation of a rheumatologist. Approvals valid for 4 months for applications meeting the following criteria:
Either:
- Both:
1.1 The patient has had an initial Special Authority approval for both etanercept and adalimumab for juvenile idiopathic arthritis (JIA); and
1.2 The patient has experienced intolerable side effects, or has received insufficient benefit from, both etanercept and adalimumab; or
- All of the following:
2.1 Treatment with a tumour necrosis factor alpha inhibitor is contraindicated; and
2.2 Patient has had severe active polyarticular course JIA for 6 months duration or longer; and
2.3 To be used as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
2.4 Both:
2.4.1 At least 5 swollen joints and at least 3 joints with limitation of motion, pain and tenderness; or
2.4.2 Moderate or high disease activity (cJADAS score of at least 2.5) after a 3-month trial of methotrexate (at a dose of 10-20 mg/m2 weekly or at the maximum tolerated dose); or
2.4.3 Low disease activity (cJADAS score between 1.1 and 2.5) after a 6-month trial of methotrexate; and
2.5 Patient has tried and not responded to a therapeutic trial of oral corticosteroids (prednisone 0.25 mg/kg or at the maximum tolerated dose)
2.3 Patient has tried and not responded to at least three months of oral or parenteral methotrexate (at a dose of 10-20 mg/m2 weekly or at the maximum tolerated dose) in combination with either oral corticosteroids (prednisone 0.25 mg/kg or at the maximum tolerated dose) or a full trial of serial intra-articular corticosteroid injections; and
2.5 Both:
2.5.1 Either:
2.5.1.1 Patient has persistent symptoms of poorly-controlled and active disease in at least 20 swollen, tender joints; or
2.5.1.2 Patient has persistent symptoms of poorly-controlled and active disease in at least four joints from the following: wrist, elbow, knee, ankle, shoulder, cervical spine, hip; and
2.5.2 Physician’s global assessment indicating severe disease.
Renewal — (polyarticular juvenile idiopathic arthritis) only from a rheumatologist or Practitioner on the recommendation of a rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:
Both:
- Treatment is to be used as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
- Either:
2.1 Following 3 to 4 months’ initial treatment, the patient has at least a 50% decrease in active joint count and an improvement in physician’s global assessment from baseline; or
2.2 On subsequent reapplications, the patient demonstrates at least a continuing 30% improvement in active joint count and continued improvement in physician’s global assessment from baseline
There are no proposed changes to other current Special Authority criteria for adalimumab, etanercept or tocilizumab.
To provide feedback
Send us an email: consult@pharmac.govt.nz by 25 September 2020.
All feedback received before the closing date will be considered by PHARMAC’s Board (or its delegate) prior to making a decision on this proposal.
Feedback we receive is subject to the Official Information Act 1982 (OIA) and we will consider any request to have information withheld in accordance with our obligations under the OIA. Anyone providing feedback, whether on their own account or on behalf of an organisation, and whether in a personal or professional capacity, should be aware that the content of their feedback and their identity may need to be disclosed in response to an OIA request.
We are not able to treat any part of your feedback as confidential unless you specifically request that we do, and then only to the extent permissible under the OIA and other relevant laws and requirements. If you would like us to withhold any commercially sensitive, confidential proprietary, or personal information included in your submission, please clearly state this in your submission and identify the relevant sections of your submission that you would like it withheld. PHARMAC will give due consideration to any such request.
Last updated: 11 September 2020
