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

Neurofibromatosis type 1

Initial treatment

Patient must have plexiform neurofibroma(s) (PN) that is causing/likely to cause at least one of: (i) significant symptoms/morbidity, (ii) disability, (iii) disfigurement, (iv) impairment of normal body function; AND

Patient must have PN for which complete resection cannot be performed; AND

Patient must have either a: (i) Karnofsky, (ii) Lansky Performance Score of at least 70%; AND

Must be treated by a prescriber who is either: (i) a specialist physician with expertise in neurofibromatosis, (ii) a medical practitioner in consultation with a specialist physician with expertise in neurofibromatosis if attendance is not possible due to geographic isolation; AND

Patient must be aged between 2 to 18 years; AND

Patient must be able to swallow the whole capsule form of this drug.

At the time of the authority application, medical practitioners must request the appropriate number of packs of appropriate strength(s) to provide sufficient drug, based on the body surface area (BSA) of the patient, adequate for 4 weeks, according to the specified dosage in the approved Product Information (PI). A separate authority prescription form must be completed for each strength requested. Up to a maximum of 5 repeats will be authorised.

Confirmation of eligibility for treatment with diagnostic reports must be documented in the patient's medical records.

For the purpose of administering this restriction, significant symptoms/morbidity are defined as, but not limited to:

1. head and neck PN that can compromise the airway or great vessels;

2. paraspinal PN that can cause myelopathy;

3. brachial or lumbar plexus PN that can cause nerve compression and loss of function;

4. PN that can result in major deformity or significant disfiguring (e.g. orbital PN);

5. PN of the extremity that can cause limb hypertrophy or loss of function; and

6. painful PN.

The authority application must be made in writing and must include:

(1) details of the proposed prescription; and

(2) 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).

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

Neurofibromatosis type 1

Continuing treatment

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

Patient must be tolerating treatment; AND

Patient must have achieved either: (i) stabilisation of disease, (ii) adequate response to treatment, if have received at least 12 months of treatment with this drug; AND

Must be treated by a prescriber who is either: (i) a specialist physician with expertise in neurofibromatosis, (ii) a medical practitioner in consultation with a specialist physician with expertise in neurofibromatosis if attendance is not possible due to geographic isolation.

At the time of the authority application, medical practitioners must request the appropriate number of packs of appropriate strength(s) to provide sufficient drug, based on the body surface area (BSA) of the patient, adequate for 4 weeks, according to the specified dosage in the approved Product Information (PI). A separate authority prescription form must be completed for each strength requested. Up to a maximum of 5 repeats will be authorised.

Confirmation of eligibility for treatment with diagnostic reports must be documented in the patient's medical records.

For the purpose of administering this restriction, adequate response is defined as:

1. stability or improvement of the initial baseline measurements prior to initiating treatment with this drug;

2. relevant imaging has not shown an increase in tumour size of 20% or more.

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

Neurofibromatosis type 1

Transitioning from non-PBS to PBS-subsidised supply - Grandfather arrangements

Patient must have previously received treatment with this drug for this condition prior to 1 August 2024; or

Patient must have previously received treatment with another mitogen-activated protein kinase (MEK) inhibitor for this condition prior to 1 August 2024; AND

Patient must have met all other PBS eligibility criteria that a non-'Grandfather' patient would ordinarily be required to meet, meaning that at the time non-PBS-subsidised supply of a MEK inhibitor (including selumetinib) was commenced, the patient: (i) had PN that caused/was likely to cause at least one of: (a) significant symptoms/morbidity, (b) disability, (c) disfigurement, (d) impairment of normal body function; (ii) had PN for which complete PN resection could not be performed either: (a) safely, (b) without causing unacceptable morbidity; (iii) had either a: (a) Karnofsky, (b) Lansky Performance Score of at least 70%; (iv) was aged between 2 to 18 years; (v) was able to swallow the whole capsule form if received non-PBS supply with selumetinib; AND

Patient must be tolerating treatment; AND

Patient must have achieved either: (i) stabilisation of disease, (ii) adequate response to treatment, if have received at least 12 months of treatment; AND

Must be treated by a prescriber who is either: (i) a specialist physician with expertise in neurofibromatosis, (ii) a medical practitioner in consultation with a specialist physician with expertise in neurofibromatosis if attendance is not possible due to geographic isolation.

At the time of the authority application, medical practitioners must request the appropriate number of packs of appropriate strength(s) to provide sufficient drug, based on the body surface area (BSA) of the patient, adequate for 4 weeks, according to the specified dosage in the approved Product Information (PI). A separate authority prescription form must be completed for each strength requested. Up to a maximum of 5 repeats will be authorised.

Confirmation of eligibility for treatment with diagnostic reports must be documented in the patient's medical records.

For the purpose of administering this restriction, significant symptoms/morbidity are defined as, but not limited to:

1. head and neck PN that can compromise the airway or great vessels;

2. paraspinal PN that can cause myelopathy;

3. brachial or lumbar plexus PN that can cause nerve compression and loss of function;

4. PN that can result in major deformity or significant disfiguring (e.g. orbital PN);

5. PN of the extremity that can cause limb hypertrophy or loss of function; and

6. painful PN.

For the purpose of administering this restriction, adequate response is defined as:

1. stability or improvement of the initial baseline measurements prior to initiating treatment with this drug;

2. relevant imaging has not shown an increase in tumour size of 20% or more.

The authority application must be made in writing and must include:

(1) details of the proposed prescription; and

(2) 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).