Decision to widen access to treatments for juvenile idiopathic arthritis

Medicines Decision

We're pleased to announce a decision to widen access to medicines for the treatment of juvenile idiopathic arthritis.

What we’re doing

We're pleased to announce a decision to widen access to adalimumab and etanercept for the treatment of polyarticular course juvenile idiopathic arthritis (JIA) and oligoarticular course JIA from 1 December 2020. We are also widening access to tocilizumab for the treatment of polyarticular course JIA from 1 December 2020.

This change will widen access to include funding of adalimumab and etanercept for treatment of oligoarticular course JIA, and widen access of adalimumab, etanercept and tocilizumab for treatment of polyarticular course JIA affecting 5 or more joints. We estimate that around 65 patients each year will benefit from these treatments with reduced joint damage and pain, improved mobility and quality of life.

Any changes to the original proposal?

This decision was subject to a consultation letter dated 11 September 2020.

We thank all those who provided consultation feedback. Following review of the feedback received we have made some changes to the Special Authority criteria we consulted on. More information about these changes can be found below.

Who we think will be most 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

Details about this decision

Funded access to adalimumab, etanercept and tocilizumab for JIA will be amended in Section B of the Pharmaceutical Schedule from 1 December 2020 to the following criteria. Equivalent changes will also be made in Part II of Section H of the Pharmaceutical Schedule. No changes were made to the criteria for any of the other funded uses of these treatments.

ADALIMUMAB

Initial application – (polyarticular course juvenile idiopathic arthritis) only from a named specialist or rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:

Either:

  1. Both:
    1. The patient has had an initial Special Authority approval for etanercept for polyarticular course juvenile idiopathic arthritis (JIA); and
    2. Either:
      1. The patient has experienced intolerable side effects from etanercept; or
      2. The patient has received insufficient benefit from etanercept to meet the renewal criteria for etanercept for polyarticular course JIA; or
  2. All of the following:
    1. To be used as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
    2. Patient has had polyarticular course JIA for 6 months duration or longer; and
    3. Any of the following:
      1. At least 5 active joints and at least 3 joints with limited range of motion, pain or tenderness after a 3-month trial of methotrexate (at the maximum tolerated dose); or
      2. Moderate or high disease activity (cJADAS10 score of at least 2.5) after a 3-month trial of methotrexate (at the maximum tolerated dose); or
      3. Low disease activity (cJADAS10 score between 1.1 and 2.5) after a 6-month trial of methotrexate.

ADALIMUMAB

Initial application – (oligoarticular course juvenile idiopathic arthritis) only from a named specialist or rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:

Either:

  1. Both:
    1. The patient has had an initial Special Authority approval for etanercept for oligoarticular course juvenile idiopathic arthritis (JIA); and
    2. Either:
      1. The patient has experienced intolerable side effects from etanercept; or
      2. The patient has received insufficient benefit from etanercept to meet the renewal criteria for etanercept for oligoarticular course JIA; or
  2. All of the following:
    1. To be used as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance;
    2. Patient has had oligoarticular course JIA for 6 months duration or longer; and
    3. Any of the following:
      1. At least 2 active joints with limited range of motion, pain or tenderness after a 3-month trial of methotrexate (at the maximum tolerated dose); or
      2. Moderate or high disease activity (cJADAS10 score greater than 1.5) with poor prognostic features after a 3-month trial of methotrexate (at the maximum tolerated dose); or
      3. High disease activity (cJADAS10 score greater than 4) after a 6-month trial of methotrexate.

 

ADALIMUMAB

Renewal – (polyarticular course 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: 

  1. Subsidised as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
  2. Either:
    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. 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.

 

ADALIMUMAB

Renewal – (oligoarticular course 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: 

  1. Subsidised as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
  2. Either:
    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. 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.

 

ETANERCEPT

Initial application – (polyarticular course juvenile idiopathic arthritis) only from a named specialist or rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:

Either:

  1. Both:
    1. The patient has had an initial Special Authority approval for adalimumab for polyarticular course juvenile idiopathic arthritis (JIA); and
    2. Either:
      1. The patient has experienced intolerable side effects from adalimumab; or
      2. The patient has received insufficient benefit from adalimumab to meet the renewal criteria for adalimumab for polyarticular course JIA; or
  2. All of the following:
    1. To be used as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
    2. Patient has had polyarticular course JIA for 6 months duration or longer; and
    3. Any of the following:
      1. At least 5 active joints and at least 3 joints with limited range of motion, pain or tenderness after a 3-month trial of methotrexate (at the maximum tolerated dose); or
      2. Moderate or high disease activity (cJADAS10 score of at least 2.5) after a 3-month trial of methotrexate (at the maximum tolerated dose); or
      3. Low disease activity (cJADAS10 score between 1.1 and 2.5) after a 6-month trial of methotrexate.

 

ETANERCEPT

Initial application – (oligoarticular course juvenile idiopathic arthritis) only from a named specialist or rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:

Either:

  1. Both:
    1. The patient has had an initial Special Authority approval for adalimumab for oligoarticular course juvenile idiopathic arthritis (JIA); and
    2. Either:
      1. The patient has experienced intolerable side effects from adalimumab; or
      2. The patient has received insufficient benefit from adalimumab to meet the renewal criteria for adalimumab for oligoarticular course JIA; or
  2. All of the following:
    1. To be used as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
    2. Patient has had oligoarticular course JIA for 6 months duration or longer; and
    3. Any of the following:
      1. At least 2 active joints with limited range of motion, pain or tenderness after a 3-month trial of methotrexate (at the maximum tolerated dose); or
      2. Moderate or high disease activity (cJADAS10 score greater than 1.5) with poor prognostic features after a 3-month trial of methotrexate (at the maximum tolerated dose); or
      3. High disease activity (cJADAS10 score greater than 4) after a 6-month trial of methotrexate.

 

ETANERCEPT

Renewal – (polyarticular course 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:

  1. Subsidised as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
  2. Either:
    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. 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.

 

ETANERCEPT

Renewal – (oligoarticular course 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: 

  1. Subsidised as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
  2. Either:
    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. 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.

 

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:

  1. Both:
    1. The patient has had an initial Special Authority approval for both etanercept and adalimumab for polyarticular course juvenile idiopathic arthritis (JIA); and
    2. The patient has experienced intolerable side effects, or has received insufficient benefit from, both etanercept and adalimumab; or
  2. All of the following:
    1. Treatment with a tumour necrosis factor alpha inhibitor is contraindicated; and
    2. Patient has had polyarticular course JIA for 6 months duration or longer; and
    3. To be used as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
    4. Any of the following:
      1. At least 5 active joints and at least 3 joints with limited range of motion, pain or tenderness after a 3-month trial of methotrexate (at the maximum tolerated dose); or
      2. Moderate or high disease activity (cJADAS10 score of at least 2.5) after a 3-month trial of methotrexate (at the maximum tolerated dose); or
      3. Low disease activity (cJADAS10 score between 1.1 and 2.5) after a 6-month trial of methotrexate.

 

TOCILIZUMAB

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:

  1. Treatment is to be used as an adjunct to methotrexate therapy or monotherapy where use of methotrexate is limited by toxicity or intolerance; and
  2. Either:
    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. 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.

You can view the changes to the Special Authority criteria compared with the criteria we consulted on here [PDF, 29 KB].

Our response to what you told us

We’re really grateful for the time people took to respond to this consultation. Responses were generally supportive of the proposal. A summary of the main themes raised in feedback, our responses to the feedback received, and changes we have made after listening to you are set out below.

THEME

PHARMAC COMMENT

Requests to consider changes to the Special Authority criteria and hospital indication restrictions to help clarify and simplify requirements: 

  • request to remove criteria requiring a trial of oral corticosteroids for patients with polyarticular course JIA, to be consistent with criteria for oligoarticular course JIA
  • request for a change in joint description and scoring system to better reflect current practice.
  • requested to remove required methotrexate dose. 

After carefully considering this feedback we have made changes to the Special Authority criteria and hospital restriction criteria to reflect these requests.

Noted there may be costs associated with this proposal in terms of clinician time when initiating a new medicine, infusion services for tocilizumab administration, and time supporting patient and/or care-giver administration of subcutaneous injection technique.

PHARMAC acknowledges the impact on clinical services involved in treatment initiation and support for patients receiving these treatments. We consider that there will likely be a small number of patients requiring intravenous tocilizumab, and consider that the overall cost and resource impacts on DHB services is expected to be low relative to the positive health impacts for patients. 

Noted support for further widening of access to these medicines and other ‘non-infusion’ treatment options for other inflammatory disease conditions.

We would welcome funding applications for relevant changes to funding criteria.

More information about how to make a funding application can be found here.

If you have any questions about this decision, you can email us at enquiry@pharmac.govt.nz; or call our toll free number (9 am to 5 pm, Monday to Friday) on 0800 660 050.