Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Chronic thromboembolic pulmonary hypertension (CTEPH)

Initial treatment

Patient must have WHO Functional Class II, III or IV CTEPH; AND

The condition must be inoperable by pulmonary endarterectomy; or

The condition must be recurrent or persistent following pulmonary endarterectomy; AND

The treatment must be the sole PBS-subsidised therapy for this condition; AND

Must be treated in a centre with expertise in the management of CTEPH; AND

Patient must be at least 18 years of age.

CTEPH that is inoperable by pulmonary endarterectomy is defined as follows:

(a) Right heart catheterisation (RHC) demonstrating pulmonary vascular resistance (PVR) of greater than 300 dyn*sec*cm^-5 measured at least 90 days after start of full anticoagulation; and

(b) A mean pulmonary artery pressure (PAPmean) of greater than 25 mmHg at least 90 days after start of full anticoagulation.

CTEPH that is recurrent or persistent subsequent to pulmonary endarterectomy is defined as follows:

RHC demonstrating a PVR of greater than 300 dyn*sec*cm^-5 measured at least 180 days following pulmonary endarterectomy.

Where a RHC cannot be performed due to right ventricular dysfunction, an echocardiogram demonstrating the dysfunction must be documented in the patient's medical records.

Applications for authorisation of initial treatment must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail.

If the application is submitted through HPOS form upload or mail, it must include:

(a) details of the proposed prescription; and

(b) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).

The following must be provided at the time of application and documented in the patient's medical records:

(a) the results from the 3 tests below, to establish baseline measurements, where available:

(i) RHC composite assessment, and

(ii) ECHO composite assessment, and

(iii) 6 Minute Walk Test (6MWT); and

(b) confirmation of evidence of inoperable CTEPH including the pulmonary vascular resistance (PVR) value, a mean pulmonary artery pressure (PAPmean) and the starting date of full anticoagulation; or

(c) confirmation of evidence of recurrent or persistent CTEPH including the PVR value and the date that pulmonary endarterectomy was performed; or

(d) confirmation of an echocardiogram demonstrating right ventricular dysfunction.

Where it is not possible to perform all 3 tests above on clinical grounds, the expected test combination, in descending order of preference is:

(1) RHC plus ECHO composite assessments;

(2) RHC composite assessment plus 6MWT;

(3) RHC composite assessment only.

In circumstance where a RHC cannot be performed on clinical grounds, the expected test combinations, in descending order of preference is:

(1) ECHO composite assessment plus 6MWT;

(2) ECHO composite assessment only.

Where fewer than 3 tests are able to be performed on clinical grounds, a patient specific reason outlining why the particular test(s) could not be conducted must be documented in the patient's medical records.

The test results provided must not be more than 2 months old at the time of application.

Prescriptions for dose titration must provide sufficient quantity for dose titrations by 0.5 mg increments at 2-week intervals to achieve up to a maximum of 2.5 mg three times daily based on the dosage recommendations for initiation of treatment in the TGA-approved Product Information. No repeats will be authorised for these prescriptions.

Approvals for subsequent authority prescription will be limited to 1 month of treatment, The quantity approved must be based on the dosage recommendations in the TGA-approved Product Information, and a maximum of 3 repeats.

The assessment of the patient's response to the initial 20-week course of treatment should be made following the preceding 16 weeks of treatment, in order to allow sufficient time for a response to be demonstrated.

Patients who fail to demonstrate a response to PBS-subsidised treatment with this agent at the time where an assessment is required must cease PBS-subsidised therapy with this agent.

Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Chronic thromboembolic pulmonary hypertension (CTEPH)

Continuing treatment

Patient must have previously received PBS-subsidised treatment with this drug for this condition; AND

Patient must demonstrate stable or responding disease; AND

The treatment must be the sole PBS-subsidised therapy for this condition; AND

Must be treated in a centre with expertise in the management of CTEPH; AND

Patient must be at least 18 years of age.

Response to this drug is defined as follows:

For patients with two or more baseline tests, response to treatment is defined as two or more tests demonstrating stability or improvement of disease.

For patients with a RHC composite assessment alone at baseline, response to treatment is defined as a RHC result demonstrating stability or improvement of disease.

For patients with an ECHO composite assessment alone at baseline, response to treatment is defined as an ECHO result demonstrating stability or improvement of disease.

Test requirements to establish response to treatment for continuation of treatment are as follows:

The following list outlines the preferred test combination, in descending order, for the purposes of continuation of PBS-subsidised treatment:

(1) RHC plus ECHO composite assessments plus 6MWT;

(2) RHC plus ECHO composite assessments;

(3) RHC composite assessment plus 6MWT;

(4) ECHO composite assessment plus 6MWT;

(5) RHC composite assessment only;

(6) ECHO composite assessment only.

The results of the same tests as conducted at baseline should be documented in the patient's medical record with each continuing treatment application (i.e., every 6 months), except for patients who were able to undergo all 3 tests at baseline, and whose subsequent ECHO and 6MWT results demonstrate disease stability or improvement, in which case RHC can be omitted. In all other patients, where the same test(s) conducted at baseline cannot be performed for assessment of response on clinical grounds, a patient specific reason why the test(s) could not be conducted must be documented in the patient's medical records.

The test results conducted for continuing treatment must be no more than 2 months old at the time of application.

The assessment of the patient's response to the continuing 6 month courses of treatment should be made following the preceding 5 months of treatment, in order to allow sufficient time for a response to be demonstrated.

The maximum quantity per prescription must be based on the dosage recommendations in the TGA-approved Product Information and be limited to provide sufficient supply for 1 month of treatment.

A maximum of 5 repeats will be authorised.

Patients who fail to demonstrate disease stability or improvement to PBS-subsidised treatment with this agent at the time where an assessment is required must cease PBS-subsidised therapy with this agent.

Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Chronic thromboembolic pulmonary hypertension (CTEPH)

Balance of supply

Patient must have received insufficient therapy with this drug under the Initial treatment restriction to complete a maximum of 20 weeks of treatment; or

Patient must have received insufficient therapy with this drug under the Continuing treatment restriction to complete a maximum of 24 weeks of treatment; AND

The treatment must provide no more than the balance of up to 20 or 24 weeks of treatment available under the above respective restriction; AND

The treatment must be the sole PBS-subsidised therapy for this condition; AND

Must be treated in a centre with expertise in the management of CTEPH; AND

Patient must be at least 18 years of age.