Decision to fund oncology, multiple sclerosis and respiratory treatments

Medicines Decision

We’re pleased to announce a decision to approve funding for oncology, multiple sclerosis and respiratory treatments.

In summary, this will result in the following changes from 1 December 2019:

Any changes to the original proposal?

This decision was subject to a consultation letter dated 7 August 2019

The proposed terms of listing, including commercial terms and Special Authority criteria, were approved as consulted on without any changes except as a result of consultation feedback, the Special Authority criteria and restrictions for trastuzumab emtansine were amended to:

  • allow pre-treatment with trastuzumab and chemotherapy other than a taxane
  • remove the criterion for left ventricular ejection fraction (LVEF) (heart function)
  • clarify the intent regarding treatment of patients with brain metastases

This means that trastuzumab emtansine will be funded from 1 December 2019 for patients with HER-2 positive metastatic breast cancer who meet the following Special Authority criteria (differences from criteria consulted on shown by additions in bold and deletions in strikethrough):

Initial application - only from a relevant specialist or medical practitioner or on the recommendation of a relevant specialist. Approvals valid for 6 months for applications meeting the following criteria:

All of the following:

  1. Patient has metastatic breast cancer expressing HER-2 IHC 3+ or ISH+ (including FISH or other current technology); and
  2. Patient has previously received trastuzumab and a taxane chemotherapy, separately or in combination; and
  3. Either

3.1.    The patient has received prior therapy for metastatic disease*; or
3.2.    The patient developed disease recurrence during, or within six months of completing adjuvant therapy*; and

  1. Patient has a good performance status (ECOG 0-1); and
  2. Patient has left ventricular ejection fraction of 50% or more; and
  3. Patient does not have symptomatic brain metastases; and
  1. Either:

5.1.  Patient does not have symptomatic brain metastases; or
5.2.  Patient has brain metastases and has received prior local CNS therapy; and

  1. Treatment to be discontinued at disease progression.

Renewal – only from a relevant specialist or medical practitioner or on the recommendation of a relevant specialist. Approvals valid for 6 months for applications meeting the following criteria:

Both:

  1. The cancer has not progressed at any time point during the previous approval period whilst on trastuzumab emtansine; and
  2. Treatment to be discontinued at disease progression.

*Note: Prior or adjuvant therapy includes anthracycline, other chemotherapy, biological drugs, or endocrine therapy.

Who we think will be most interested

  • People who have or may develop lung cancer, breast cancer, multiple sclerosis or idioathic pulmonary fibrosis and their whānau

  • Oncologists, neurologists and respiratory clinicians

  • Community and hospital pharmacies

  • DHBs

  • Organisations with an interest in treatments for cancer, multiple sclerosis or respiratory diseases.

Our response to what you told us

We’re really grateful for the time people took to respond to this consultation. We had a large number of responses, most responses were really 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.

Feedback theme

Comment

Alectinib for ALK-positive advanced non-small cell lung cancer (NSCLC)

ALK testing

Clarification of the definition of an appropriate ALK test

It would be up to DHB services and clinicians to determine what an appropriate ALK test may be. The Lung Oncology Special Interest Group (LOSIG) supports the phrasing of the funding criteria. It considers the establishment of affordable and equitable access to testing is a DHB decision; and indicated it would help to develop a consistent national approach.

Concern regarding the costs associated with and availability of ALK-testing.

PHARMAC does consider the cost of testing in its assessment of impacts to the health sector, however notes that DHBs are responsible for the provision and funding of testing services.

Wider access

A request for amendment to the renewal criteria to remove the requirement to use RECIST criteria to measure response to treatment.

We note that the criteria are in line with clinical advice.

No changes have been made to the criteria consulted on for alectinib.

We consider that RECIST criteria provide an objective measure of response and disease progression which is well understood in the oncology community.

Trastuzumab emtansine for HER2-positive metastatic breast cancer

Wider access

Requests for amendment to the criteria to allow patients previously treated with vinorelbine rather than a taxane to be eligible.

 

The intent of the proposed criteria is to provide a second-line or beyond treatment option for all previously-treated HER2-positive advanced breast cancer patients.

Clinical advice from members of CaTSoP indicated that this would be an appropriate amendment. The criteria for trastuzumab emtansine have been amended to reflect this.

 

Requests for amendment to the proposed criteria regarding cardiac function to reduce to 40% or remove the requirement for left ventricular ejection fraction (LVEF) of 50% or more.

 

Our clinical advisors considered it is reasonable to remove this requirement from the Special Authority criteria.

The criteria have been amended to reflect this.

 

Requests for amendment to the proposed criteria to remove of the criteria which requires patients not to have symptomatic brain metastases.

 

Clinical advice from members of CaTSoP recommended clarifying the proposed criteria to detail that if brain metastases are present then these should be previously treated and at least stable prior to accessing trastuzumab emtansine.

The criteria have been amended to reflect this.

System and resource impacts

Feedback about resource impacts in terms of:

  • infusion and imaging services,
  • need for assessment of LVEF function,
  • health professional time to manage patients
  • support services time to update protocols and patient information

 

PHARMAC assesses and considers all additional costs to the health sector, however notes that DHBs are responsible for the provision and funding of services.

We note that the requirement for cardiac function assessment via LVEF measurement has been removed from the criteria based on consultation feedback.

 

Request for clarification of how patient numbers were estimated.

 

The eligible population of second-line HER2 positive advanced breast cancer of 60 patients per year was estimated based on:

  • the total number of patients seeking a second-line treatment following pertuzumab/trastuzumab in a first line setting, using rates from the trial evidence; and
  • the expected prevalent population of patients who would not have received pertuzumab (as were pre-treated prior to its listing for treatment naïve patients) and would be seeking additional treatment.

Request for clarification of the frequency of response assessment.

 

The frequency of assessment is a matter for clinical decision-making and should ensure the clinically appropriate management and monitoring of patients.

Ocrelizumab for relapsing remitting multiple sclerosis

Wider access

Respondents raised a number of aspects relating to entry and exit criteria. Requests for wider access included:

  • The stopping criteria be expanded to EDSS 6.0 or 6.5 irrespective of starting points
  • The gradient scale be removed from the stopping criteria
  • The starting criteria of EDSS 0-4 be removed
  • The starting criteria allow for treatment of those with clinically isolated syndrome (CIS) who meet the McDonald 2017 diagnostic criteria for RRMS

 

Funding applications for MS treatments have undergone extensive review by PTAC. PTAC considered that these treatments should be targeted to the patients likely to benefit most from such treatments. The entry and exit criteria are based on PTAC’s advice; that treatment should be targeted to patients with clinically definite RRMS with active inflammatory disease. Shou