Approval of multi-product funding agreement with Novartis

Medicines

Decision

We’re pleased to announce a major funding package involving ten medicines in seven key therapy areas through an agreement with Novartis New Zealand Limited (Novartis).

What we’re doing

We’re pleased to announce a major funding package involving ten medicines in seven key therapy areas through an agreement with Novartis New Zealand Limited (Novartis).

Five new medicines will be funded, and access widened to three others, from 1 October 2018. The decision will provide substantial health benefits for up to 50,000 New Zealanders with a range of health conditions.

In summary, this decision will result in the following changes from 1 October 2018:

  • Funding of five new treatments:
    • Secukinumab (Cosentyx) for severe chronic plaque psoriasis
    • Sacubitril with valsartan (Entresto) for chronic heart failure
    • Vildagliptin (Galvus) for type 2 diabetes mellitus
    • Vildagliptin with metformin (Galvumet) for type 2 diabetes mellitus
    • Ruxolitinib (Jakavi) for myelofibrosis
  • Widening of access to three currently funded treatments:
    • Eltrombopag (Revolade) for idiopathic thrombocytopenic purpura contraindicated to splenectomy, and severe aplastic anaemia
    • Omalizumab (Xolair) for chronic spontaneous urticaria and severe asthma
    • Tacrolimus (Tacrolimus Sandoz) for non-transplant indications
  • Amended contractual terms (including pricing, rebates and protection periods) for:
    • Basiliximab (Simulect) for use in solid organ transplant
    • Fingolimod (Gilenya) for relapsing remitting multiple sclerosis

Any changes to the original proposal?

This decision was subject to a consultation in July 2018.

The proposed terms of listing, including commercial terms and Special Authority criteria, were approved as consulted on without any changes except for:

  • an amendment to the tacrolimus Special Authority criteria to include detail that non-transplant indications are not a Medsafe approved indication
  • the Wastage Rule will be applied to all strengths of ruxolitinib (Jakavi) and the Special Authority criteria have been amended to clarify the maximum dose of 20 mg twice daily
  • amendment to the omalizumab criteria for:
    • severe asthma, so that clinical immunologists would be able to apply in addition to respiratory physicians
    • chronic spontaneous urticaria, to better reflect current clinical practice, more clearly define the intended patient population, and response/stopping criteria.

This means that omalizumab will be funded from 1 October 2018 for patients with severe chronic spontaneous urticaria who meet the following Special Authority criteria (differences from criteria consulted on shown by additions in bold and deletions in strikethrough):

 

Initial application – (severe chronic spontaneous urticaria) only from a clinical immunologist or dermatologist. Approvals valid for 6 months for applications meeting the following criteria:

All of the following:

  1. Patient must be aged 12 years or older; and
  2. Either:

2.1.    Both:

2.1.1.  Patient is symptomatic with Urticaria Activity Score 7 (UAS7) of 20 or above; and

2.1.2.  Patient has a Dermatology life quality index (DLQI) of 10 or greater; and or

2.2.    Patient has a Urticaria Control Test (UCT) of 8 or less; and

  1. Any of the following:

3.1.    Patient has been taking high dose antihistamines (e.g. 4 times standard dose) and ciclosporin (>3 mg/kg day) for at least 3 months6 weeks; or

3.2.    Patient has been taking high dose antihistamines (e.g. 4 times standard dose) and at least 3 courses of systemic corticosteroids (>20 mg prednisone per day for at least 5 days) in the previous 6 months; or

3.3.    Patient has developed significant adverse effects whilst on corticosteroids or ciclosporin; and

  1. Either:

4.1.     Treatment to be stopped if inadequate response* following 4 doses; or

4.2.     Complete response* to 6 doses of omalizumab.

Renewal – (severe chronic spontaneous urticaria) only from a clinical immunologist or dermatologist. Approvals valid for 6 months for applications meeting the following criteria:

Either:

  1. All of the following
  1. Patient has previously adequately responded* to 6 doses of omalizumab; or
  2. Patient’s current Urticaria Activity Score is greater than 6; and
  3. Patient’s current DLQI score is greater than 5.
  1. Both:

2.1.     Patient has previously had a complete response* to 6 doses of omalizumab; and

2.2.     Patient has relapsed after cessation of omalizumab therapy.

Note: *Inadequate response defined as less than 50% reduction in baseline UAS7 and DLQI score, or an increase in Urticaria Control Test (UCT) score of less than 4 from baseline. Patient is to be reassessed for response (50% reduction in baseline UAS7 and DLQI score) after 4 doses of omalizumab. Complete response is defined as UAS7 less than or equal to 6 and DLQI less than or equal to 5; or UCT of 16. Relapse of chronic urticaria on stopping prednisone/ciclosporin does not justify the funding of omalizumab.

Our response to what you told us

We’re really grateful for the time people took to respond to this consultation. All consultation responses received were considered in their entirety in making a decision on the proposed changes.

Most responses were 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 outlined below.

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 66 00 50.

Feedback theme Comment
Vildagliptin/vildagliptin with metformin for type 2 diabetes mellitus
The desire for other antidiabetic agents to be funded and concerns that the proposal would impact on the funding of other antidiabetic agents, particularly from the GLP-1s and SGLT-2s classes, which responders considered to have superior outcomes in terms of cardiovascular risk and renal outcomes.

The decision to fund vildagliptin and vildagliptin with metformin does not preclude the funding of another antidiabetic agent at any time.

PHARMAC is continuing to assess a number of other agents, including SGLT-2 and GLP-1s. More information about this can be found on the Application Tracker.

Queries regarding the assessment of the pharmaceuticals.

PHARMAC has a robust process to consider funding of medicines that includes obtaining clinical advice and undertaking health technology assessment.

The proposal for vildagliptin was informed by advice from our clinical advisory committees, including both the Pharmacology and Therapeutics Advisory Committee (PTAC) and the Diabetes Subcommittee of PTAC. More information about the clinical advice, including the relevant minutes, can be found on the Application Tracker.

As with other funding proposals, a health technology assessment was undertaken by PHARMAC staff which includes cost-effectiveness assessment.

Concerns regarding the appropriate prescribing of vildagliptin As with any pharmaceutical, prescribers will need to make clinical judgements about their patients who would benefit from treatment with vildagliptin. We will be providing information about vildagliptin to support prescribers in this.
Requests for this treatment to be included on the nurse prescribing list as nurses are managing more long-term conditions.

Vildagliptin and vildagliptin with metformin will be funded without restriction which means that any relevant health practitioner, including nurses with prescribing rights, can prescribe these treatments.

The medicines included on the Registered Nurse Prescriber Medicine List are managed by the Nursing Council.

Secukinumab for severe chronic plaque psoriasis
Request for amendment of the proposed criteria to allow Special Authority application ‘on the recommendation of a dermatologist’ to allow shared care with general practice. The access criteria for secukinumab are consistent with those for tumour necrosis factor (TNF)-alpha inhibitors (etanercept and adalimumab) that are funded for chronic plaque psoriasis, where treatment can only be initiated by a dermatologist, but renewals can be made by either a dermatologist or a medical practitioner on the recommendation of a dermatologist.
Request for the TNF-alpha inhibitor criteria for patients with severe chronic plaque psoriasis to be amended to align with the criteria proposed for secukinumab (i.e for the TNF-alpha inhibitor criteria to have the same lower Psoriasis Area and Severity Index (PASI) score requirement of >10 rather than 15). This is something we could consider if we reached an acceptable agreement with the suppliers of adalimumab and etanercept.
Sacubitril with valsartan for chronic heart failure
Requests for general practitioners and primary care physicians to be able to make Special Authority applications

Special Authority applications for sacubitril with valsartan can be applied for by ‘any relevant practitioner’ which includes general practitioners and primary care physicians.

We will be providing information about sacubitril with valsartan to support prescribers.

Requests for the duration of Special Authority approval (initial or renewal) to be valid without further renewal i.e. lifelong approval rather than require reassessment after 12 months.

The proposed criteria are in line with our clinical advice and include the requirement for reassessment after 12 months to ensure that treatment remains appropriate and the patient is benefitting.

More information about the clinical advice, including the relevant minutes, can be found on the Application Tracker.

Requests for widened access to include patients with a left ventricular ejection fraction (LVEF) limit of <40% rather than 35% or less. The criteria are in line with our clinical advice to target treatment to patients with a higher health need, to improve the cost-effectiveness of this relatively expensive medicine.More information about the clinical advice, including the relevant minutes, can be found on the Application Tracker.
Request that the Wastage Rule be applied to all strengths of sacubitril with valsartan.

Application of the Wastage Rule for this product is not proposed at this time given the relatively large estimated number of patients eligible to receive treatment.

We acknowledge that some pharmacies may choose not to break packs without a Wastage Rule. However, it is expected that pharmacies receiving 30-day prescriptions would dispense 28-days’ worth (one whole pack) to avoid part packs being left over.  

PHARMAC is open to reviewing the application of the Wastage Rule to sacubitril with valsartan in future.

Ruxolitinib for myelofibrosis
Request for access criteria to be widened for selected intermediate-1 patients and some polycaethemia rubra vera (PRV) patients with resistant splenomegaly.  PHARMAC’s main clinical advisory committee, PTAC, recommended funding ruxolitinib for people with intermediate-1 disease with a lower priority due to generally lower health need, the lack of high-quality evidence (only a small population in a cohort study), and the comparatively smaller benefits expected. The proposal to fund ruxolitinib for this patient population will remain ranked as a potential option for investment at a later stage.We welcome a funding submission for widened access to ruxolitinib for any further populations or subgroups if supported by published evidence.
Concern regarding the high cost of treatment and additional DHB costs for monitoring and review.

We consider that these patients are likely to have a high health need and, therefore, would already be requiring reasonably frequent specialist care for symptom control.

Ruxolitinib is expected to aid in disease control and reduce the requirement to manage individual symptoms. The Haematology Subcommittee of PTAC noted that ruxolitinib withdrawal syndrome is likely to be rare, manageable with gradual tapering of the dose and would be unlikely to add additional resources compared to the majority of other treatments managed by haematologists.

PHARMAC’s assessment for ruxolitinib has taken into account the impact on the health sector.

Request that the Wastage Rule be applied to all strengths of ruxolitinib. The Wastage Rule has been applied to all strengths of ruxolitinib given the high Pharmaceutical Schedule price, low patient numbers and regular dose titration based on response.
Eltrombopag for idiopathic thrombocytopenic purpura contraindicated to splenectomy and severe aplastic anaemia
Request for amendment of the access criteria to allow a short course of eltrombopag for patients with idiopathic thrombocytopenic purpura (ITP) whilst awaiting a response to immunosuppressive treatments The eligible patient populations have been established based on funding applications and clinical advice received under the Named Patient Pharmaceutical Assessment (NPPA) pathway. Further advice on this broader indication raised during consultation will be sought from the Haematology Subcommittee of PTAC at its next meeting.
Omalizumab – general  
Requests for general practitioners to be able to make omalizumab Special Authority applications rather than restricting applications to specialists. Clinical immunologists or dermatologists (urticaria) or respiratory specialists (asthma) are able to make Special Authority applications. This is in line with our clinical advice that specialists would be best placed to initiate treatment with omalizumab. Once a patient has a Special Authority approval, general practitioners are able to prescribe funded omalizumab as ongoing treatment.
Omalizumab for chronic spontaneous urticaria
Requests for amendment of the proposed criteria to:
  1. include in the initial criteria a retrospective severity scoring method, reduce the length of trial with ciclosporin and clarify the requirements for trialling systemic corticosteroids.
  2. clarify renewal requirements for patients that have had a complete response to prior treatment with omalizumab and when treatment should be stopped; and with regards to ciclosporin and systemic corticosteroid use.
  3. include a Note to detail back-titration of dose after initial treatment on initial treatment.
  4. allow funded treatment of solar urticaria.

Following consideration of consultation feedback, the initial and renewal criteria have been amended to include the suggestions and clarifications summarised in points 1 and 2. We consider that the amended criteria may better reflect current practice of treating patients with chronic spontaneous urticaria and provide more objective and more well-define the requirements to help clinicians assess eligibility for funded treatment.

Regarding point 3, we note that Special Authority criteria are not intended to provide clinical guidelines. Prescribers can refer to the Medsafe datasheet for more information about dosing and administration of omalizumab.(external link)

Regarding point 4, we would welcome a funding application supported by published evidence for the use of omalizumab in the treatment of patients with less severe forms of chronic spontaneous urticaria or solar urticaria.

Omalizumab for severe asthma
Requests for immunologists to be able to make Special Authority applications as these clinicians would be making clinical decisions for these patients and administering omalizumab, and it would have resource impact if only respiratory physicians could apply. Following consideration of consultation feedback, the proposed criteria have been amended so that clinical immunologists can make Special Authority applications.
Requests for amendment of the proposed criteria to:
 
Responses to each point raised are as follows:
  1. Remove the term ‘life-threatening’ from the application as this term is observer dependent, based on a single point in time assessment, and it may create confusion for the applicant.
  1. Following consideration of consultation feedback, the criteria have been amended to remove the term ‘life-threatening’.
  1. Include the Child-ACT asthma control score, renewal to be based on an increase in ACT score of 3 rather than 5, and to remove the requirement for patients to be over 6 years old.
  1. The criteria in the approved Special Authority are in line with the clinical advice we received and with the Medsafe registered indication. We are open to considering further widening of access in future and would welcome a funding application supported by published evidence for the wider use of omalizumab for severe asthma.
  1. Require a trial of budesonide/eformoterol turbuhaler via the SMART regimen.
  1. As above, the criteria in the approved Special Authority are in line with our clinical advice and with the Medsafe registered indication. The Special Authority criteria do not prevent a trial of these agents prior to use of omalizumab, should the prescriber consider this to be appropriate.
Tacrolimus for non-transplant indications
Concern that there would be an impact on clinical services when initiating patients on tacrolimus due to increased requirement for patient assessment and lack of familiarity with tacrolimus.

PHARMAC’s assessment for tacrolimus has taken into account the impact on the health sector. We consider that impacts on clinical services would be minimal, noting that we understand that these patients would likely already be under the care of a specialist familiar with tacrolimus. In addition, use of ciclosporin is a pre-requisite for access to tacrolimus and has similar monitoring requirements to tacrolimus.

Please note that the tacrolimus criteria for patients with steroid-resistant nephrotic syndrome will be superseded by the new criteria for non-transplant indications. PHARMAC will arrange for patients with Special Authority approvals for steroid-resistant nephrotic syndrome to be transferred to the new authority for non-transplant indications.

Basiliximab for use in solid organ transplant
Consultation responses were supportive of the proposal and did not raise any specific issues.
Fingolimod for relapsing remitting multiple sclerosis
Concern that the reduced subsidy would mean that patients would incur additional costs for treatment. Fingolimod will remain fully funded for eligible patients.
Other general responses
Concern that due to the price decreases there is significant potential for losses to be incurred in the supply chain; and request for price support to be provided. This feedback was passed on to the supplier. PHARMAC staff understand that the supplier intends to provide a price support mechanism to ensure that no wholesaler is disadvantaged.