Completed high cost medicine reviews

The Statewide Formulary for High Cost Medicines aims to ensure that eligible patients will have equal opportunity to receive a high cost medicine based upon their clinical condition rather than where they live or what hospital they attend.

All public hospitals must comply with the SA Health Statewide Formulary for High Cost Medicines policy (PDF 281KB) to enhance equity of access within the South Australian public health system.

The following medicine reviews have been completed by South Australian Medicines Evaluation Panel:

Adalimumab

Non-infectious posterior, intermediate or panuveitis

Indication: non-infectious posterior, intermediate or panuveitis that is refractory to corticosteroids and at least two other corticosteroid sparing agents.

SAMEP recommendation: To list Adalimumab on the High Cost Medicines Formulary for adult patients with non-infectious posterior, intermediate or panuveitis that is refractory to corticosteroids and at least two other corticosteroid sparing agents.

Rationale: The patients eligible for this treatment have vision threatening disease. High quality evidence demonstrates that adalimumab delays time to treatment failure relative to placebo and can facilitate steroid taper in some patients. The SAMEP view is that control of disease without the requirement for long term corticosteroids is an important goal of treatment in this population, particularly given the risk of ocular complications associated with long term steroids.

Outcome: Listed on the high cost medicines formulary.

Checklist for patient eligibility: Eligibility form - Adalimumab for non-infectious posterior, intermediate or panuveitis (PDF 192KB).

Active non-infectious sight threatening uveitis

Indication: Active non-infectious sight threatening uveitis for paediatric patients who had an inadequate response to corticosteroid and failed or intolerant of methotrexate (15mg/m2).

SAMEP recommendation: To list adalimumab on the High Cost Medicines Formulary as first line biologic treatment for paediatric patients with active non-infectious sight threatening uveitis who had an inadequate response to corticosteroid and failed or intolerant of methotrexate (15mg/m2).

Checklist form for patient eligibility and clinical pathway: Adalimumab for paediatric uveitis (PDF 594KB).

Contact SAMEP for a copy of the review.

Outcome: Listed on High Cost Medicines Formulary.

Anagrelide

Indication: Second-line option for treatment of essential thrombocythaemia after prior cytoreductive treatment.

SAMEP recommendations: The Eligibility Checklist for anagrelide was removed in July 2023.

Alemtuzumab

Indication: B-cell Chronic Lymphocytic Leukaemia (B-CLL).

SAMEP recommendations: Alemtuzumab rapid review (PDF 1314KB).

Outcome: Not listed on formulary.

Bendamustine

Indication: Treatment of relapsed or refractory non-Hodgkin’s lymphoma (NHL) or chronic lymphocytic leukaemia (CLL).

SAMEP recommendations: Bendamustine recommendations (PDF 1524KB).

Outcome: No further IPU approvals recommended for specified patient population.

Bevacizumab

Indication: Recurrent glioblastoma multiforme.

SAMEP recommendations: Bevacizumab (PDF 1416KB).

Outcome: Not listed on high cost medicines formulary (no further IPU approvals recommended).

Delamanid

Indication: pulmonary drug resistant tuberculosis.

SAMEP recommendation: to list delamanid on the High Cost Medicines Formulary for patients with pulmonary drug resistant tuberculosis.

Rationale: delamanid is approved by the European Medicines Agency and is included in the World Health Organization guidelines on drug-resistant tuberculosis treatment. SAMEP concluded that it was useful to have an alternative antibiotic active against pulmonary drug resistant tuberculosis where one or more of the available treatments cannot be used due to resistance, intolerance, adverse effects or interactions.

Outcome: Listed on the high cost medicines formulary.

Checklist for patient eligibility: Eligibility checklist for delamanid (DOCX 572KB).

Denosumab

Indication: For the prevention of fracture and recurrence in postmenopausal women with early breast cancer.

SAMEP recommendations: Contact SAMEP for a copy of the review.

Outcome: Denosumab is not listed on formulary.

Defibrotide

Indication: Treatment or prophylaxis of veno-occlusive disease.

SAMEP recommendations: Defibrotide rapid review (PDF 1285KB).

Outcome: No further IPUs recommended.

Eltrombopag

Indication: Paediatric patients with severe aplastic anaemia.

SAMEP recommendations: That eltrombopag in conjunction with horse anti-thymocyte globulin (h-ATG) and cyclosporin (CsA) be available via a Streamline IPU form in treatment naïve paediatric patients.

Rationale: The evidence indicates that eltrombopag when added to first line immunosuppressive therapy increases the rate of robust hematologic response relative to h-ATG and CsA alone. This limits the risk of complications and may allow patients to become transfusion independent that in turn may avoid or delay the need for haemapoetic stem cell transplantation.

Outcome: Available via Streamline IPU form.

Checklist form for patient eligibility: Streamline IPU form: Eltrombopag (PDF 100KB).

Eltrombopag for ITP

Indication: Paediatric patients with immune thrombocytopaenia.

SAMEP recommendation: Eltrombopag is listed on the High Cost Medicines Formulary for paediatric patients with immune thrombocytopaenia diagnosed for at least 6 months in whom at least three courses of steroids or IVIG have failed to achieve satisfactory haematological response.

Rationale: Relative to placebo eltrombopag reduces both all bleeding and clinically significant bleeding and can induce a sustained platelet response. A sustained platelet response is associated with a near zero rate of clinically significant bleeding and mortality. The SAMEP view is that eltrombopag is an alternative to rituximab or splenectomy in this population.

Outcome: Listed on the high cost medicines formulary.

Checklist form for patient eligibility: Eligibility form: Eltrombopag for ITP.

Indication: Thrombocytopenia following allogeneic haematopoietic stem cell transplant.

SAMEP recommendation: Eltrombopag is NOT listed on the SA Medicines Formulary for thrombocytopenia following allogeneic haematopoietic stem cell transplant. Eltrombopag can be obtained with a streamlined Individual Patient Use (IPU). SAMEP will review the usage of eltrombopag and the clinical outcomes after one year of implementation of this recommendation.

Rationale: There is limited and uncertain evidence on the efficacy of eltrombopag for thrombocytopenia following allogeneic haematopoietic stem cell transplant (one small RCT and a meta-analysis of observational studies) with regard to improvement of platelet counts and independence of platelet transfusion.

Outcome: SAMEP recommend limited access via a streamlined Individual Patient Use and review of local usage and clinical outcomes after one year.

Streamlined Individual Patient UseStreamlined IPU eltrombopag post stem cell transplant (PDF 152KB).

Fampridine

Indication: Symptomatic improvement of walking ability in adult patients with multiple sclerosis.

SAMEP recommendations: Fampridine evaluation summary (PDF 1388KB).

Outcome: Not listed on formulary.

Gemtuzumab ozogamicin

Indication: de novo CD33-positive acute myeloid leukaemia

SAMEP recommendation: Gemtuzumab ozogamicin is listed on the SA
Medicines Formulary for de novo CD33-positive acute myeloid leukaemia
as per PBS restrictions.

Rationale: Gemtuzumab ozogamicin has been funded on the PBS for this
indication since May 2023. Inpatients from public hospitals are excluded
from this listing but the induction cycle is usually done as inpatients at SA
Health. SAMEP considered the main evidence assessed by the
Pharmaceutical Benefits Advisory Committee.

Outcome: SAMEP recommended inclusion of gemtuzumab ozogamicin on
the SA Medicines Formulary for previously untreated, de novo CD33-
positive acute myeloid leukaemia meeting PBS criteria:

  • for inpatient use for induction cycle OR
  • as per Pharmaceutical Benefit Scheme (PBS) criteria for
    consolidation cycle in outpatients.

Jack Jumper Ant Venom Immunotherapy

Indication: Anaphylactic / immediate generalised reaction(s) to jack jumper ant (JJA) stings, with ongoing exposure risk to JJA.

SAMEP Recommendation: To list jack jumper ant venom immunotherapy (JJA VIT) on the High Cost Medicines Formulary for adult patients with confirmed anaphylactic / immediate generalised reaction(s) to JJA stings, with ongoing exposure risk to JJA.

Rationale: The available evidence was sufficiently compelling to conclude that JJA VIT in severely allergic and highly motivated people is efficacious in reducing future risk of severe allergic reactions relative to placebo. SAMEP noted cost offsets would be derived from reduced frequency of hospitalisation and emergency department presentation, avoidance of anaphylaxis associated complications, and improved quality of life.

Outcome: List on the high cost medicines formulary.

Infliximab

Acute severe colitis refractory to intravenous corticosteroids

SAMEP recommendations: SAMEP review completed prior to PBS listing for ulcerative colitis. Contact SAMEP for a copy of the review.

Outcome: Listed on High Cost Medicines formulary.

hidradenitis suppurativa

SAMEP recommendations: Contact SAMEP for a copy of the review.

Outcome: Not listed on the high cost medicines formulary.

Acute severe checkpoint inhibitor mediated colitis refractory to steroids

SAMEP recommendation: To make infliximab available for severe, refractory checkpoint inhibitor-induced colitis via non-formulary streamlined Individual patient use (IPU) with a review of outcomes and utilisation in 12-24 months.

Rationale: Severe colitis that is refractory to the gold standard treatment, corticosteroids, can be life-threatening and in some cases surgery to remove part of the colon (colectomy) may be indicated. The SAMEP considered that effective pharmacologic options in these patients is important. According to the evidence available, treatment with infliximab is associated with improvement in steroid-refractory colitis in a substantial proportion of patients (between 30 and 100%). The SAMEP considered that infliximab has a role in the treatment of severe refractory checkpoint inhibitor induced colitis, particularly with a view to avoiding colectomy.

Outcome: Available on Streamline IPU request.

Checklist form for patient eligibility: Infliximab form for steroid refractory colitis secondary to checkpoint inhibitors (PDF 167KB).

Pyoderma gangrenosum

SAMEP recommendations: Infliximab should be available for patients with pyoderma gangrenosum (PG) with moderate to severe disease or mild PG that has failed topical preparation and who are being treated with topical treatments as adjunct to systemic therapy guided by physician judgement meeting the criteria specified in eligibility form. Contact SAMEP for a copy of the review.

Outcome: Listed on High Cost Medicines formulary.

Checklist form for patient eligibility: Infliximab for pyoderma gangrenosum: patient eligibility form (DOCX 476KB).

Clinical pathway: Infliximab for pyoderma gangrenosum: clinical pathway (PDF 124KB).

Active non-infectious sight threatening uveitis

Indication: Active non-infectious sight threatening uveitis for paediatric patients who had an inadequate response to corticosteroid and failed or intolerant of methotrexate (15mg/m2).

SAMEP recommendation: To list infliximab on the High Cost Medicines Formulary as second line biologic treatment for paediatric patients with active non-infectious sight threatening uveitis who had an inadequate response to corticosteroid and failed or intolerant of methotrexate (15mg/m2).

Checklist form for patient eligibility and clinical pathway: Infliximab for paediatric uveitis (PDF 594KB).

Contact SAMEP for a copy of the review.

Outcome: Listed on High Cost Medicines Formulary.

Third line treatment for refractory sarcoidosis

Indication: for adult patients with severe biopsy proven pulmonary, cardiac or ocular sarcoidosis who are refractory to standard treatment including at least 0.5mg/kg of prednisone (or equivalent) with the addition of at least ONE (preferably two) immunosuppressive steroid sparing agent for at least three months at target dose.

SAMEP recommendation: to list infliximab on the High Cost Medicines Formulary as Third line treatment for refractory sarcoidosis. Contact SAMEP for a copy of the review.

Outcome: Listed on High Cost Medicines Formulary.

Checklist form for patient eligibility and clinical pathwayInfliximab for refractory sarcoidosis (PDF 254KB).

Increased dosage in inflammatory bowel disease

SAMEP recommendation: to list increased dosage of infliximab on the High Cost Medicines Formulary.

  • for adult patients with moderate to severe Crohn’s disease or complex fistulising Crohn disease or moderate to severe ulcerative colitis
  • for paediatric patients (aged 6-17 years inclusive) with moderate to severe refractory Crohn’s disease, or moderate to severe ulcerative colitis
  • who are experiencing a loss of response to PBS-subsidised standard 8 weekly 5 mg/kg IV infusions of infliximab, and
  • who have low therapeutic infliximab levels and antiinfliximab antibody levels.

Rationale: dose escalation of infliximab supported by therapeutic drug monitoring (TDM) and monitoring of anti-infliximab antibody levels is a long-established practice in this setting and recommended in several clinical guidelines. Increased dosage of infliximab may result in a substantial albeit variable regain of response in patient who are experiencing a loss of response to PBS-subsidised standard dosing.

Outcome: listed on the high cost medicines formulary.

Checklist and clinical pathway for patient eligibility: Eligibility checklist and clinical pathway for increased dosage of infliximab in inflammatory bowel diseases (DOCX 556KB).

Ledipasvir 90 mg/sofosbuvir 400 mg

Indication: Hepatitis C infection (Genotype 1).

SAMAC recommendation: To list ledipasvir 90 mg/sofosbuvir 400 mg on the Statewide High Cost Medicines Formulary for Hepatitis C infection (Genotype 1) as per the PBS criteria in adults for adolescents aged 12 to 17 years.

Rationale: Treatment with 12 weeks of ledipasvir 90 mg/sofosbuvir 400 mg in adolescent patients with genotype 1 resulted in sustained virological response (cure) in 98 per cent of treated patients.SAMAC took the view that it was not appropriate to delay treatment for adolescents when a safe and curative treatment exists, particularly in view of uncertain future evaluation at the national level and public health benefits of access.

Outcome: Listed on the high-cost medicines formulary.

Omalizumab

Indication: Severe idiopathic urticaria.

SAMEP recommendations: Omalizumab recommendations (PDF 1499KB).

Outcome: Not listed on the high cost medicines formulary (No further IPUs recommended for specified patient population).

Nelarabine

Indication: T-Cell leukaemia in paediatric patients.

SAMEP recommendation: SAMEP recommended to NOT list nelarabine for T-Cell leukaemia for paediatric patients on the SA medicines formulary.

Rationale: There is limited and uncertain evidence for efficacy of nelarabine and concerns about its neurotoxicity. There is no international nor Australian consensus on inclusion of nelarabine in the standard of care for T-Cell leukaemia in paediatric patients.

Although there may be a rationale to include nelarabine as part of standard of care in the Children Oncology Group (COG) trial proposal, SAMEP judged that it was outside its scope to assess the rationale for the trial.

Outcome: SAMEP recommended:

  • to NOT list nelarabine for T-Cell leukaemia for paediatric patients on the SA medicines formulary and
  • to allow access to nelarabine through the IPU process overseen by WCH-DTC in the context of the affiliation of the WCH’s Haematology and oncology group with COG.

Palivizumab

Indication: Prevention of serious lower respiratory tract disease caused by Respiratory Syncytial Virus (RSV) in infants at high risk of RSV disease.

SAMEP recommendations: Palivizumab for prevention of RSV evaluation summary (PDF 1470KB).

Outcome: Not listed on the SA High Cost Medicines formulary.

Pertuzumab

Indication: The neoadjuvant treatment of women with human epidermal growth factor receptor 2 (HER 2) positive locally advanced or inflammatory breast cancer.

SAMEP recommendation: That pertuzumab not be listed on the Statewide High Cost Medicines Formulary for the neoadjuvant treatment of women with HER 2 positive locally advanced or inflammatory breast cancer.

The SAMEP agreed that pertuzumab could be considered by streamline non-formulary approval request to enable surgery in the rare cases of inflammatory HER-2 positive breast cancer.

Rationale: The SAMEP considered the assumption that pertuzumab would improve disease-free or overall survival to be inadequately supported in the available evidence and therefore could not conclude that the addition of pertuzumab in the neoadjuvant period would result in improved outcomes.

Outcome: Not listed on the Statewide High Cost Medicines Formulary.

Streamline Non-Formulary Approval Request form: Pertuzumab in inflammatory HER-2 positive breast cancer (PDF 125KB).

Plerixafor

Indication: For use in combination with chemotherapy and GCSF to mobilise haematopoietic stem cells for collection and subsequent autologous transplantation in patients with lymphoma or multiple myeloma who are poor/failed mobilisers.

SAMEP recommendations: Contact SAMEP for a copy of the algorithm or review.

Outcome: Listed on High Cost Medicine formulary in compliance with PBS criteria only.

Rituximab

Since September 2022, rituximab 100mg and 500mg injection restrictions were removed from the Pharmaceutical Benefits Scheme (PBS). In response to this significant change SAFC and SAMEP have made changes to the SAMF listing of rituximab, which includes removal of previous SAFC and SAMEP restrictions and associated forms. Clinicians can prescribe rituximab according to their assessment of the risk-benefit to the patient. It is strongly encouraged that clinicians use quality use of medicines (QUM) resources referenced in the updated formulary listing when prescribing off-label rituximab to support efficacy, safety, and informed patient consent in absence of formulary restrictions.

Thiotepa

Indication: Standard treatment as part of conditioning protocols for allogenic haematopoietic stem cell transplantation in adults.

SAMEP recommendation: To list thiotepa on the High Cost Medicines Formulary as standard treatment as part of conditioning protocols for allogenic haematopoietic stem cell transplantation in adults.

Prescribing restricted to haematology consultants and registrars and only for patients undergoing haematopoietic stem cell transplantation.

Refer to the SA Health Approved Cancer Chemotherapy Protocol register for Allogeneic intermediate intensity conditioning protocol (cord) CYCLOPHOSPHamide fludarabine thiotepa and total body irradiation (TBI).

Outcome: Listed on the High Cost Medicines Formulary.

Indication: Standard treatment as part of conditioning protocol for autologous haematopoietic stem cell transplant (ASCT) in the treatment of CNS lymphoma.

SAMEP recommendation: To list thiotepa on the High Cost Medicines Formulary as part of conditioning protocol for autologous haematopoietic stem cell transplant (ASCT) in the treatment of CNS lymphoma.

Prescribing restricted to haematology consultants and registrars and only for patients undergoing autologous haematopoietic stem cell transplantation.

Refer to the SA Health Approved Cancer Chemotherapy Protocol register for  Autologous conditioning protocol primary CNS lymphoma carmustine and thiotepa.

Outcome: Listed on the High Cost Medicines Formulary.

Trastuzumab emtansine

Indication: HER2-positive metastatic breast cancer.

SAMEP recommendation: Contact SAMEP for further information on the rapid review.

Outcome: Not listed on formulary. No further IPU requests to be funded by SA Health.

Ustekinumab

Indication: Hidradenitis suppurativa in patients who have failed multiple treatments including adalimumab.

SAMEP recommendation: Ustekinumab is NOT listed on the SA Medicines Formulary for Hidradenitis suppurativa in patients who have failed multiple treatments including adalimumab. Ustekinumab can be obtained with a streamlined Individual Patient Use (IPU). SAMEP will review the usage of ustekinumab and the clinical outcomes after one year of implementation of this recommendation.

Rationale: There is limited and uncertain evidence on the efficacy of ustekinumab for Hidradenitis suppurativa in patients who have failed multiple treatments including adalimumab (a few small observational studies and case reports).

Outcome: SAMEP recommend limited access via a streamlined Individual Patient Use and review of local usage and clinical outcomes after one year.

Streamlined Individual Patient UseStreamlined IPU Ustekinumab Hidradenitis suppurativa (PDF 258KB).


Indication: moderate to severe refractory Crohn’s disease, or moderate to severe ulcerative colitis in paediatric patients (aged 6 years and over).

SAMEP recommendation: Ustekinumab is listed on the SA Medicines Formulary for moderate to severe refractory Crohn’s disease, or moderate to severe ulcerative colitis in paediatric patients (aged 6 years and over):

  • with chronically active or steroid-dependent disease and
  • who are experiencing a loss of response to anti-TNF medicines despite dose optimisation after measurement of drug levels and antibody levels, and use of immunomodulator medicines.

Rationale: Ustekinumab is funded by the PBS for adult patients in these indications. Observational studies in children show efficacy results similar to those observed in randomized clinical trials in the adult population. Ustekinumab is currently recommended in paediatric clinical guidelines and may provide an effective alternative to surgery in the paediatric population.

Outcome: SAMEP recommend restricted access to ustekinumab via a Eligibility Checklist that will facilitate review of local usage and clinical outcomes.

Eligibility Checklist: Eligibility Checklist ustekinumab (DOCX 495KB)

Vedolizumab

Indication: steroid dependent checkpoint inhibitor induced colitis not meeting the criteria for infliximab.

SAMEP recommendation: Not to list vedolizumab on the high cost medicines formulary on the basis of insufficient evidence to validate the clinical claims.

Rationale: The evidence for vedolizumab consists of a single case series and one case report. The SAMEP considered that the case series including vedolizumab had poor applicability to the proposed indication and therefore that the evidence was insufficient to determine whether it has a benefit in checkpoint inhibitor induced colitis.

Outcome: Not listed on the high cost medicines formulary.


Indication: moderate to severe refractory Crohn’s disease, or moderate to severe ulcerative colitis in paediatric patients (aged 6 years and over).

SAMEP recommendation: Vedolizumab is listed on the SA Medicines Formulary for moderate to severe refractory Crohn’s disease, or moderate to severe ulcerative colitis in paediatric patients (aged 6 years and over):

  • with chronically active or steroid-dependent disease and
  • who are experiencing a loss of response to anti-TNF medicines despite dose optimisation after measurement of drug levels and antibody levels, and use of immunomodulator medicines.

Rationale: Vedolizumab is funded by the PBS for adult patients in these indications. Observational studies in children show efficacy results similar to those observed in randomized clinical trials in the adult population. Vedolizumab is currently recommended in paediatric clinical guidelines and may provide an effective alternative to surgery in the paediatric population.

Outcome: SAMEP recommend restricted access to vedolizumab via a Eligibility Checklist that will facilitate review of local usage and clinical outcomes.

Eligibility Checklist: Vedolizumab eligibility checklist (DOCX 495KB)

Zoledronic acid

Indication: For the prevention of recurrence in postmenopausal women with early breast cancer.

SAMEP recommendations: The formulary listing for zoledronic acid should extend to postmenopausal women with early breast cancer deemed to be at 10% or greater risk of breast cancer death within 10 years as estimated by the NHS PREDICT tool.

Outcome: Listed on formulary.