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

Severe Crohn disease

Initial 1 (new patient)

Patient must have confirmed severe Crohn disease, defined by standard clinical, endoscopic and/or imaging features, including histological evidence, with the diagnosis confirmed by a gastroenterologist or a consultant physician; AND

Patient must not have achieved an adequate response to a tapered course of steroids, starting at a dose of at least 40 mg prednisolone (or equivalent), over a 6 week period; AND

Patient must not have achieved an adequate response to prior treatment with a thiopurine at a clinically optimised dose for 3 or more consecutive months; or

Patient must not have achieved an adequate response to methotrexate at a dose of at least 15 mg weekly for 3 or more consecutive months; AND

Patient must have a Crohn Disease Activity Index (CDAI) Score greater than or equal to 300 as evidence of not achieving an adequate response to prior systemic therapy; or

Patient must have short gut syndrome with diagnostic imaging or surgical evidence, or have had an ileostomy or colostomy; and must have evidence of intestinal inflammation; and must have evidence of not achieving an adequate response to prior systemic therapy as specified below; or

Patient must have extensive intestinal inflammation affecting more than 50 cm of the small intestine as evidenced by radiological imaging; and must have a Crohn Disease Activity Index (CDAI) Score greater than or equal to 220; and must have evidence of not achieving an adequate response to prior systemic therapy as specified below; AND

The treatment must not exceed a total of 2 doses to be administered at weeks 0 and 8 under this restriction; AND

Must be treated by a gastroenterologist; or

Must be treated by a consultant physician [internal medicine specialising in gastroenterology]; or

Must be treated by a consultant physician [general medicine specialising in gastroenterology]; AND

Patient must be at least 18 years of age.

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

(a) details of prior systemic drug therapy [dosage, date of commencement and duration of therapy]; and

(b) if applicable, details (date, unique identifying number/code or provider number) of the pathology or diagnostic imaging test(s) nominated as the response criteria; and

(c) if applicable, details of intestinal inflammation marker/s; and

(d) if applicable, the CDAI score and the date of assessment; and

(e) the date of the most recent clinical assessment.

Evidence of not achieving an adequate response to prior therapy must include at least one of the following:

(a) patient must have evidence of intestinal inflammation;

(b) patient must be assessed clinically as being in a high faecal output state;

(c) patient must be assessed clinically as requiring surgery or total parenteral nutrition (TPN) as the next therapeutic option, in the absence of this drug, if affected by short gut syndrome, extensive small intestine disease or is an ostomy patient.

Evidence of intestinal inflammation includes:

(i) blood: higher than normal platelet count, or, an elevated erythrocyte sedimentation rate (ESR) greater than 25 mm per hour, or, a C-reactive protein (CRP) level greater than 15 mg per L; or

(ii) faeces: higher than normal lactoferrin or calprotectin level; or

(iii) diagnostic imaging: demonstration of increased uptake of intravenous contrast with thickening of the bowel wall or mesenteric lymphadenopathy or fat streaking in the mesentery.

Where fewer than 2 doses are requested at the time of the application, authority approvals for sufficient treatment based on the patient's weight to complete dosing at weeks 0 and 8 may be requested through the Balance of supply restriction.

If treatment with any of the specified prior conventional drugs is contraindicated according to the relevant contraindications or precautions in the TGA-approved Product Information, please provide details at the time of application. Patients must attempt to trial alternate conventional therapies that are not contraindicated.

If intolerance to treatment develops during the relevant period of use, which is of a severity necessitating permanent treatment withdrawal, details of this toxicity must be provided at the time of application.

Any one of the baseline criteria may be used to determine response to an initial course of treatment and eligibility for continued therapy, according to the criteria included in the continuing treatment restriction. However, the same criterion must be used for any subsequent determination of response to treatment, for the purpose of eligibility for continuing PBS-subsidised therapy.

A patient who does not demonstrate a response to treatment will not be eligible for further PBS-subsidised treatment with this drug for this condition within the same treatment cycle. The patient will be considered not to have responded to treatment if a response assessment is not completed. A serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment is not considered as treatment failure.

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

Severe Crohn disease

Initial 2 (change or recommencement of treatment after a break in targeted therapy of less than 12 months)

Patient must have received prior PBS-subsidised treatment with a targeted therapy for this condition in this treatment cycle; or

Patient must have been eligible for prior PBS-subsidised treatment with a targeted therapy for this condition in this treatment cycle; AND

Patient must have either: (i) not previously received this drug for this condition in the current treatment cycle, (ii) previously received this drug and demonstrated/maintained an adequate clinical response; AND

The treatment must not exceed a total of 2 doses to be administered at weeks 0 and 8 under this restriction; AND

Must be treated by a gastroenterologist; or

Must be treated by a consultant physician [internal medicine specialising in gastroenterology]; or

Must be treated by a consultant physician [general medicine specialising in gastroenterology]; AND

Patient must be at least 18 years of age.

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

(a) if applicable, the CDAI score and the date of assessment; and

(b) if applicable, details (date, unique identifying number/code or provider number) of the pathology or diagnostic imaging test(s) used to assess response to therapy for patients with short gut syndrome, extensive small intestine disease or an ostomy; and

(c) if applicable, the date of the most recent clinical assessment; and

(d) details of the most recent targeted therapy in the current treatment cycle, including the date and duration of treatment.

If a patient was eligible for prior PBS-subsidised treatment with a targeted therapy for this condition but did not receive PBS-subsidised treatment at the time, the application must provide a declaration that the patient met all 'Initial 1' eligibility requirements when targeted therapy treatment commenced. The following information must also be provided at the time of application:

(a) the baseline CDAI score and the date of assessment; and/or

(b) details (date, unique identifying number/code or provider number) of the pathology or diagnostic imaging test(s) used to assess response to therapy for patients with short gut syndrome, extensive small intestine disease or an ostomy.

Where fewer than 2 doses are requested at the time of the application, authority approvals for sufficient treatment based on the patient's weight to complete dosing at weeks 0 and 8 may be requested through the Balance of supply restriction.

A patient who does not demonstrate a response to treatment will not be eligible for further PBS-subsidised treatment with this drug for this condition within the same treatment cycle. The patient will be considered not to have responded to treatment if a response assessment is not completed. A serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment is not considered as treatment failure.

A patient may re-trial this drug after a minimum of 12 months have elapsed between the date the last prescription for a PBS-subsidised targeted therapy was approved in this cycle and the date of the first application under a new cycle under the Initial 3 treatment restriction.

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

Severe Crohn disease

Initial 3 (recommencement of treatment after a break in targeted therapy of more than 12 months)

Patient must have received prior PBS-subsidised treatment with a targeted therapy for this condition; AND

Patient must have had a break in treatment of 12 months or more from the most recently approved PBS-subsidised targeted therapy for this condition; AND

Patient must have confirmed severe Crohn disease, defined by standard clinical, endoscopic and/or imaging features, including histological evidence, with the diagnosis confirmed by a gastroenterologist or a consultant physician; AND

Patient must have a Crohn Disease Activity Index (CDAI) Score of greater than or equal to 300 while off treatment with targeted therapy; or

Patient must have a documented history of intestinal inflammation and have diagnostic imaging or surgical evidence of short gut syndrome if affected by the syndrome or has an ileostomy or colostomy; and evidence of flared disease while off treatment with targeted therapy; or

Patient must have a documented history and radiological evidence of intestinal inflammation if the patient has extensive small intestinal disease affecting more than 50 cm of the small intestine, together with a Crohn Disease Activity Index (CDAI) Score greater than or equal to 220; and evidence of flared disease while off treatment with targeted therapy; AND

The treatment must not exceed a total of 2 doses to be administered at weeks 0 and 8 under this restriction; AND

Must be treated by a gastroenterologist; or

Must be treated by a consultant physician [internal medicine specialising in gastroenterology]; or

Must be treated by a consultant physician [general medicine specialising in gastroenterology]; AND

Patient must be at least 18 years of age.

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

(a) if applicable, current CDAI score and the date of assessment; and

(b) if applicable, details (date, unique identifying number/code or provider number) of the pathology or diagnostic imaging test(s) or details of intestinal inflammation marker/s used to assess response to therapy for patients with short gut syndrome, extensive small intestine disease or an ostomy; and

(c) the date of the most recent clinical assessment.

Evidence of flared disease must include at least one of the following:

(a) patient must have evidence of intestinal inflammation;

(b) patient must be assessed clinically as being in a high faecal output state;

(c) patient must be assessed clinically as requiring surgery or total parenteral nutrition (TPN) as the next therapeutic option, in the absence of this drug, if affected by short gut syndrome, extensive small intestine disease or is an ostomy patient.

Evidence of intestinal inflammation includes:

(i) blood: higher than normal platelet count, or, an elevated erythrocyte sedimentation rate (ESR) greater than 25 mm per hour, or, a C-reactive protein (CRP) level greater than 15 mg per L; or

(ii) faeces: higher than normal lactoferrin or calprotectin level; or

(iii) diagnostic imaging: demonstration of increased uptake of intravenous contrast with thickening of the bowel wall or mesenteric lymphadenopathy or fat streaking in the mesentery.

Where fewer than 2 doses are requested at the time of the application, authority approvals for sufficient treatment based on the patient's weight to complete dosing at weeks 0 and 8 may be requested through the Balance of supply restriction.

Any one of the baseline criteria may be used to determine response to an initial course of treatment and eligibility for continued therapy, according to the criteria included in the continuing treatment restriction. However, the same criterion must be used for any subsequent determination of response to treatment, for the purpose of eligibility for continuing PBS-subsidised therapy.

A patient who does not demonstrate a response to treatment will not be eligible for further PBS-subsidised treatment with this drug for this condition within the same treatment cycle. The patient will be considered not to have responded to treatment if a response assessment is not completed. A serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment is not considered as treatment failure.

A patient may re-trial this drug after a minimum of 12 months have elapsed between the date the last prescription for a PBS-subsidised targeted therapy was approved in this cycle and the date of the first application under a new cycle under the Initial 3 treatment restriction.