Proposal to widen access to rituximab to include children with nephrotic syndrome

Medicines

Consultation Closed

PHARMAC is seeking feedback on a proposal to amend the funding restrictions for rituximab in DHB hospitals.

In summary, this proposal would result in rituximab being funded in DHB hospitals for children with steroid dependent, frequently relapsing and steroid resistant nephrotic syndrome from 1 July 2016; subject to HML restrictions.

Detail of the proposal and some background information is set out on the following pages.

Feedback sought

PHARMAC welcomes feedback on this proposal. To provide feedback, please submit it in writing by 5pm Wednesday, 25 May 2016 to:

Caroline De Luca
Senior Therapeutic Group Manager / Team Leader
PHARMAC
PO Box 10 254
Wellington 6143

Email: caroline.deluca@pharmac.govt.nz
Fax: 04 460 4995

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.

Details of the proposal

From 1 July 2016, the restrictions applying to rituximab in Part II of Section H of the Pharmaceutical Schedule would be widened as follows:

Steroid dependent or frequently relapsing nephrotic syndrome

Initiation – Steroid dependent nephrotic syndrome (SDNS) or frequently relapsing nephrotic syndrome (FRNS)

Nephrologist

Limited to 4 weeks treatment

All of the following:

  1. Patient is a child with SDNS* or FRNS*; and
  2. Treatment with steroids for at least a period of 3 months has been ineffective or associated with evidence of steroid toxicity; and
  3. Treatment with ciclosporin for at least a period of 3 months has been ineffective; and
  4. The total rituximab dose used would not exceed the equivalent of 375 mg/ m2 of body surface area per week for a total of 4 weeks.

Note: Indications marked with a * are Unapproved indications.

Continuation - Steroid dependent nephrotic syndrome (SDNS) or frequently relapsing nephrotic syndrome (FRNS)

Nephrologist

Limited to 4 weeks treatment

All of the following:

  1. Patient who was previously treated with rituximab for nephrotic syndrome*; and
  2. Treatment with rituximab was previously successful and has demonstrated sustained response for >6 months, but the condition has relapsed and the patient now requires repeat treatment; and
  3. The total rituximab dose used would not exceed the equivalent of 375 mg/m2 of body surface area per week for a total of 4 weeks.

Note: Indications marked with a * are Unapproved indications.

Steroid resistant nephrotic syndrome

Initiation – Steroid resistant nephrotic syndrome (SRNS)

Nephrologist

Limited to 4 weeks treatment

All of the following:

  1. Patient is a child with SRNS* where treatment with steroids and ciclosporin for at least 3 months have been ineffective; and
  2. Treatment with tacrolimus for at least 3 months has been ineffective; and
  3. The total rituximab dose used would not exceed the equivalent of 375 mg/ m2 of body surface area per week for a total of 4 weeks.

Note: Indications marked with a * are Unapproved indications.

Continuation - Steroid resistant nephrotic syndrome (SRNS)

Nephrologist

Limited to 4 weeks treatment

All of the following:

  1. Patient who was previously treated with rituximab for nephrotic syndrome*; and
  2. Treatment with rituximab was previously successful and has demonstrated sustained response for greater than 6 months, but the condition has relapsed and the patient now requires repeat treatment; and
  3. The total rituximab dose used would not exceed the equivalent of 375 mg/ m2 of body surface area per week for a total of 4 weeks.

Note: Indications marked with a * are Unapproved indications.

Background

Rituximab is a monoclonal antibody agent currently listed in Section H of the Pharmaceutical Schedule (the Hospital Medicines List - HML) for use in a variety of indications, including cancer, rheumatoid arthritis, autoimmune and haematological conditions, and organ transplant (see: Section H: Rituximab).

Rituximab is registered with Medsafe for the treatment of non-Hodgkin’s lymphoma, chronic lymphocytic leukaemia, and rheumatoid arthritis. We note that rituximab is not registered by Medsafe for the other currently funded indications or for the proposed widening of funded access to include nephrotic syndrome (i.e. these are unapproved indications); therefore, in these settings, it would need to be prescribed and used in accordance with section 25 of the Medicines Act 1981.

Nephrotic syndrome (NS) is a kidney disorder in which excessive protein is excreted in the urine which typically leads to low albumin, swelling and high cholesterol. The presentation, disease progression and prognosis of nephrotic syndrome is different between children and adults. NS is classified on the basis of microscopy and response to treatment. Failure to respond clinically to treatment results in a greatly increased risk of developing end stage renal disease, particularly for children with steroid resistant NS.

Following receipt of a clinician funding application, the Nephrology Subcommittee and PTAC recommended widening access to rituximab for children with steroid dependant, frequently relapsing or steroid resistant nephrotic syndrome. The minutes of Nephrology Subcommittee and PTAC’s discussions can be found at: Application tracker: rituximab

PHARMAC estimates there would be less than 10 children in New Zealand for whom clinicians would recommend treatment with rituximab in accordance with the widened funding restrictions. We note that PTAC considered there was insufficient evidence to support the use of rituximab in adult patients with NS.