Notification to transition funded access to dornase alfa to a standard Special Authority

Medicines Decision

We're pleased to announce a decision to transition the process for obtaining funded access to dornase alfa (Pulmozyme) from the Cystic Fibrosis (CF) Panel to a standard Special Authority.

What we’re doing

We're pleased to announce a decision to transition the process for obtaining funded access to dornase alfa (Pulmozyme) from the Cystic Fibrosis (CF) Panel to a standard Special Authority.

This decision takes effect from 1 December 2020.

This decision will:

  • Allow clinicians to apply for dornase alfa (Pulmozyme) funding through the standard Special Authority application process, instead of applying to the CF Panel;
  • Remove the requirement for spirometry to commence treatment with dornase alfa or to renew access to dornase alfa for patients older than 5 years;
  • Focus the criteria on exacerbations rather than hospitalisations for all patients; and
  • Have one set of criteria for all patients with cystic fibrosis.

Any changes to the original proposal?

This decision was subject to a consultation letter dated 2 October 2020.

No changes were made to the proposal following consultation feedback.

Who we think will be most interested

  • People with cystic fibrosis and their family, whānau or caregivers
  • Cystic Fibrosis NZ and any others who support people and families with cystic fibrosis
  • Respiratory physicians, paediatricians, respiratory nurses, and other clinicians and health professionals involved in the management of cystic fibrosis
  • Hospital pharmacies and DHBs
  • Pharmaceutical suppliers

Detail about this decision

From 1 December 2020 the following changes will be implemented:

  • Access to dornase alfa will transition from approval by the CF Panel to a standard Special Authority.
  • Patients with existing Panel approvals will be transitioned as follows:
    • All patients aged less than 5 years (ie aged 0 to 4 years) who have had at least one renewal approved will be automatically granted an approval which will be valid without further renewal unless notified.
    • All patients aged less than 5 years who have not had at least one renewal will be automatically transitioned to an initial standard Special Authority with a 12 month approval.
    • Any patient currently undergoing a one-month trial (aged 5 years and over) will be transitioned to a 12-month approval under the initial criteria.
    • Any patient with a current life time approval will have their approval transitioned to a standard Special Authority approval which will be valid without further renewal unless notified.
    • From 1 December 2020, dornase alfa (Pulmozyme) will be listed in Section B (the Community) and Part Il of Section H (the Hospital Medicines List) of the Pharmaceutical Schedule subject to the following eligibility criteria:

Special Authority for Subsidy

Initial application - (cystic fibrosis) only from a respiratory physician or paediatrician. Approvals valid for 12 months for applications meeting the following criteria:

All of the following:

  1. Patient has a confirmed diagnosis of cystic fibrosis; and
  2. Patient has previously undergone a trial with, or is currently being treated with, hypertonic saline; and
  3. Any of the following:
    1. Patient has required one or more hospital inpatient respiratory admissions in the previous 12 month period; or
    2. Patient has had 3 exacerbations due to CF, requiring oral or intravenous (IV) antibiotics in the previous 12 month period; or
    3. Patient has had 1 exacerbation due to CF, requiring oral or IV antibiotics in the previous 12 month period and a Brasfield score of <22/25; or
    4. Patient has a diagnosis of allergic bronchopulmonary aspergillosis (ABPA).

Renewal - (cystic fibrosis) only from a respiratory physician or paediatrician. Approvals valid without further renewal unless notified where the treatment remains appropriate and the patient continues to benefit from treatment.

Note that there are no changes to other initiation criteria for the indications of significant mucus production and pleural empyema in Part II of Section H of the Pharmaceutical Schedule.

Our response to what you told us

We’re really grateful for the time people took to respond to this consultation.

A summary of the main themes raised in feedback, and our responses, are shown below.

Theme

PHARMAC response

Responders were generally supportive of the proposal to simplify the criteria and transition the application process to a standard Special Authority

This was consistent with our view

Responders noted that not every centre has radiologists that use the Brasfield scoring system, and including it in the access criteria may beneficially encourage wider use of the scoring system.

PHARMAC staff understand from the CF Panel that the use of the Brasfield scoring system is more common as a measure of disease in younger children and that it is not commonly used in adults.

Responders noted that this change will:

  • give CF patients better access to a medication that improves mucus clearance, decreases lung infections, thus slowing down airway remodelling for better health outcomes.
  • streamline the process for all concerned – notes the former prerequisite of three lung functions within 6 weeks was taxing (work pressures and time off work)
  • noted that the time and fiscal burden of spirometry, would be removed from patients, their families, and clinical staff.
  • widen the range of patients eligible, and supported the introduction of criteria that simplify and widen access.

Noted

Noted a concern that it was important to ensure that no existing patients lose access to funding due to the transition.

PHARMAC staff are working to ensure that all patients with an existing approval are transitioned successfully to the new approvals.

Concerns regarding criterion 1.3.3 as their DHB doesn’t routinely score chest x-rays; suggests 1.3.3 be reworded as previously, ie “Chest x-ray showing clear mucus plugging, focal consolidation or a Brasfield score <22/25 done at a time of stability despite currently approved treatment, including repeated physiotherapy assessment and education, and having had at least one admission to hospital”

PHARMAC note the concerns regarding this previous criterion.

The Brasfield scoring system in cystic fibrosis is based on conventional chest x-ray findings of structural changes and damage to the lungs, with reportedly good correlation with pulmonary function, but intra- and inter-observer variability between radiologists.

PHARMAC staff understand from the CF Panel that the use of the Brasfield scoring system is more common as a measure of disease in younger children and that it is not commonly used in adults.

In addition, the proposed criteria have moved away from the requirement for hospitalisation. PHARMAC staff note however that there remains a criterion (3.1) in the proposed criteria that would enable access to treatment for such a patient who would have accessed treatment via the previous criteria (1.3.3), by virtue of their respiratory related hospital admission and therefore this change would not prohibit such a patient from accessing treatment.

Support the proposal, and noted that:

  • this proposal includes a simpler criteria and process for accessing funding for patients, clinicians and families of patients with CF
  • Patients that have derived a lot of benefit from dornase alfa such as; increased exercise tolerance and improved clearance have not met the criteria to receive treatment. For some, this has occurred on multiple occasions

Noted

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.