SPECIAL AUTHORITY FORMS November 2018

Transcription

1 SPECIAL AUTHORITY FORMS Contents Panel Approvals... 4 Alimentary Tract Metabolism... 6 Blood Blood Fming Organs Cardiovascular System Dermatologicals Genito-Urinary System Hmone Preparations - Systemic Excluding Contraceptive Hmones Infections - Agents f Systemic Use Musculoskeletal System Nervous System Oncology Agents Immunosuppressants Respiraty System Allergies Sensy Organs Various Extempaneously Compounded Preparations Galenicals Special Foods National Immunisation Schedule List of Fms Index

2 THE SPECIAL AUTHORITY SYSTEM Special Authity is an application process in which a prescriber requests government subsidy f a particular person. Page 2 Criteria The criteria f approval of Special Authity applications are included below each pharmaceutical listing. F some Special Authity pharmaceuticals, not all indications listed on the data sheets are subsidised. Criteria f each Special Authity pharmaceutical are updated regularly, based on the decision criteria of PHARMAC. The appropriateness of the listing of a pharmaceutical in the Special Authity categy will also be regularly reviewed. Applications f inclusion of further pharmaceuticals in the Special Authity categy will generally be made by a pharmaceutical supplier. Applications from Specialists Specialist means, a doct who holds a current annual practising certificate who satisfies the criteria set out below. a) The doct s name appears in the Vocational Register of medical practitioners in accdance with Section of the Medical Practitioners Act 1995 who is making the application in the course of practising in that area of medicine; the doct s vocational branch sub-branch is one of those listed below: anaesthetics cardiothacic surgery dermatology diagnostic radiology emergency medicine general surgery internal medicine neurosurgery obstetrics gynaecology occupational medicine ophthalmology otolaryngology head neck surgery thopaedic surgery paediatric surgery paediatrics pathology plastic reconstructive surgery psychological medicine psychiatry public health medicine radiation oncology rehabilitation medicine urology venereology b) The doct is recognised by the Ministry of Health as a specialist f the purposes of the Pharmaceutical Schedule receives remuneration from a DHB at a level which that DHB considers appropriate f specialists who has written that Prescription in the course of practising in that area of medicine c) The doct is recognised by the Ministry of Health as a specialist in relation to a particular area of medicine f the purpose of writing Prescriptions who has written the Prescription in the course of practising in that area of medicine. d) The doct writes the Prescription on DHB stationery is appropriately authised by the relevant DHB to do so. Approval Special Authity applications are administered by the Ministry of Health. They were fmerly administered by Health Payments, Agreements Compliance (HealthPAC), a division of the Ministry of Health. All applications should be sent, in writing, to: Ministry of Health, Private Bag 3015, WANGANUI Fax: (06) free fax F inquiries, please call the Contact Centre on, free phone Each application must include: name date of birth of the patient (codes f AIDS patients applications) diagnosis brief clinical details name of the medicine required, the fm strength of the medicine duration of the course of treatment alternative therapies that have been tried The application must: be signed by the practitioner include the practitioner s printed name address show the practitioner s Medical Council registration number provide evidence of the criteria as per Special Authity conditions f medicine applied f Subsidy

3 Page 3 Once approved, health providers can obtain the Special Authity approval details f prescribing dispensing purposes by calling the Contact Centre on Specialists who make an application must communicate the valid authity number to the prescriber who will be writing the prescriptions. The authity number can provide access to subsidy, additional subsidy, waive certain restrictions otherwise present on the pharmaceutical. Some approvals are dependent on the availability of funding.

4 Page 4 PANEL APPROVALS Applications to be made on the approved fms which are available from the co-dinat of the relevant panel: Product Dasatinib Dimethyl fumarate Dnase Alfa Fingolimod Iiloprost Imatinib Mesylate Imiglucerase Ledipasvir with sofosbuvir Natalizumab Other Multiple Sclerosis Treatments (glatiramer acetate; interferon beta-1- alpha; interferon beta -1-beta) Taliglucerase alfa Teriflunomide ambrisentan epoprostenol Panel CML/GIST Co-dinat Multiple Sclerosis Treatment Committee Cystic Fibrosis Advisy Panel Multiple Sclerosis Treatment Committee Pulmonary Arterial Hypertension Panel CML/GIST Co-dinat Gaucher's Treatment Panel Hepatitis C Treatment Panel (HepCTP) Multiple Sclerosis Treatment Committee Multiple Sclerosis Treatment Committee Gaucher's Treatment Panel Multiple Sclerosis Treatment Committee Pulmonary Arterial Hypertension Panel Pulmonary Arterial Hypertension Panel Panel Co-dinat Pharmac PO Box Wellington Phone: Facsimile: ECPanel@Pharmac.govt.nz Product Panel Prof. Wayne Cutfield National Co-dinat New Zeal Growth Hmone Committee C/- Department of Paediatrics University of Auckl Private Bag 92019

5 Page 5 PANEL APPROVALS AUCKLAND

6 Page 6 Alimentary Tract Metabolism

7 Osteoposis where there is significant risk of fracture Severe acne following treatment with conventional cticosteroid therapy Histy of severe psychiatric problems associated with cticosteroid treatment Ministry of Health APPLICANT (stamp sticker acceptable) Page 7 Fm SA1155 Budesonide - Cap 3 mg Controlled Release INITIAL APPLICATION - Crohn's disease Applications from any relevant practitioner. Approvals valid f 6 months. Mild to moderate ileal, ileocaecal proximal Crohn's disease Diabetes Cushingoid habitus Histy of maj mental illness (such as bipolar affective disder) where the risk of conventional cticosteroid treatment causing relapse is considered to be high Relapse during pregnancy (where conventional cticosteroids are considered to be contraindicated) INITIAL APPLICATION - collagenous lymphocytic colitis (microscopic colitis) Applications from any relevant practitioner. Approvals valid f 6 months. Prerequisites (tick box where appropriate) Patient has a diagnosis of microscopic colitis (collagenous lymphocytic colitis) by colonoscopy with biopsies INITIAL APPLICATION - gut Graft versus Host disease Applications from any relevant practitioner. Approvals valid f 6 months. Prerequisites (tick box where appropriate) Patient has a gut Graft versus Host disease following allogenic bone marrow transplantation* Note: Indication marked with * is an unapproved indication. See also: RENEWAL p8 I confirm the above details are crect that in signing this fm I underst I may be audited.

8 APPLICANT (stamp sticker acceptable) Page 8 Fm SA1155 Budesonide - Cap 3 mg Controlled Release - continued RENEWAL Applications from any relevant practitioner. Approvals valid f 6 months. Prerequisites (tick box where appropriate) The treatment remains appropriate the patient is benefiting from treatment Note: Clinical trials f Entoct CIR use beyond three months demonstrated no improvement in relapse rate. I confirm the above details are crect that in signing this fm I underst I may be audited.

9 APPLICANT (stamp sticker acceptable) Page 9 Fm SA1329 Glyceryl trinitrate Oint 0.2% INITIAL APPLICATION Applications from any relevant practitioner. Approvals valid without further renewal unless notified. Prerequisites (tick box where appropriate) the patient has a chronic anal fissure that has persisted f longer than three weeks I confirm the above details are crect that in signing this fm I underst I may be audited.

10 APPLICANT (stamp sticker acceptable) Page 10 Fm SA1461 Rifaximin INITIAL APPLICATION Applications only from a gastroenterologist, hepatologist Practitioner on the recommendation of a gastroenterologist hepatologist. Approvals valid f 6 months. Prerequisites (tick box where appropriate) The patient has hepatic encephalopathy despite an adequate trial of maximum tolerated doses of lactulose RENEWAL Applications only from a gastroenterologist, hepatologist Practitioner on the recommendation of a gastroenterologist hepatologist. Approvals valid without further renewal unless notified. Prerequisites (tick box where appropriate) The treatment remains appropriate the patient is benefiting from treatment I confirm the above details are crect that in signing this fm I underst I may be audited.

11 APPLICANT (stamp sticker acceptable) Page 11 Fm SA1320 Diazoxide INITIAL APPLICATION Applications from any relevant practitioner. Approvals valid f 12 months. Prerequisites (tick box where appropriate) used f the treatment of confirmed hypoglycaemia caused by hyperinsulinism RENEWAL Applications from any relevant practitioner. Approvals valid without further renewal unless notified. Prerequisites (tick box where appropriate) The treatment remains appropriate the patient is benefiting from treatment I confirm the above details are crect that in signing this fm I underst I may be audited.

12 APPLICANT (stamp sticker acceptable) Page 12 Fm SA1603 Insulin Pumps INITIAL APPLICATION - permanent neonatal diabetes Applications only from a relevant specialist nurse practitioner. Approvals valid f 3 months. Patient has permanent neonatal diabetes A MDI regimen trial is inappropriate Has been evaluated by the multidisciplinary team f their suitability f insulin pump therapy Patient/Parent/Guardian has undertaken carbohydrate counting education (either a carbohydrate counting course direct education from an appropriate health professional) Applicant is part of a multidisciplinary team experienced in the management of type 1 diabetes care Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope RENEWAL - permanent neonatal diabetes Applications only from a relevant specialist nurse practitioner. Approvals valid f 3 months. Patient is continuing to derive benefit accding to the treatment plan agreed at induction Patient remains fully compliant transition to MDI is considered inappropriate by the treating physician It has been at least 4 years since the last insulin pump received by the patient, in the case of patients qualifying under previous pump therapy f the initial application; the pump is due f replacement Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope See also: INITIAL APPLICATION - severe unexplained hypoglycaemia p13, RENEWAL - severe unexplained hypoglycaemia p14, INITIAL APPLICATION - HbA1c p15, RENEWAL - HbA1c p16, INITIAL APPLICATION - Previous use befe 1 September 2012 p17 RENEWAL - Previous use befe 1 September 2012 p18 I confirm the above details are crect that in signing this fm I underst I may be audited.

13 APPLICANT (stamp sticker acceptable) Page 13 Fm SA1603 Insulin Pumps - continued INITIAL APPLICATION - severe unexplained hypoglycaemia Applications only from a relevant specialist nurse practitioner. Approvals valid f 3 months. Patient has type 1 diabetes has undergone a pancreatectomy has cystic fibrosis-related diabetes Has undertaken carbohydrate counting education (either a carbohydrate counting course direct education from an appropriate health professional) Applicant is part of a multidisciplinary team experienced in the management of type 1 diabetes care Has adhered to an intensive MDI regimen using analogue insulins f at least six months Has had four severe unexplained hypoglycaemic episodes over a six month period (severe as defined as requiring the assistance of another person) Has an average HbA1c between the following range: equal to greater than 53 mmol/mol equal to less than 90 mmol/mol Has been evaluated by the multidisciplinary team f their suitability f insulin pump therapy Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope See also: RENEWAL - severe unexplained hypoglycaemia p14, INITIAL APPLICATION - HbA1c p15, RENEWAL - HbA1c p16, INITIAL APPLICATION - Previous use befe 1 September 2012 p17 RENEWAL - Previous use befe 1 September 2012 p18 I confirm the above details are crect that in signing this fm I underst I may be audited.

14 APPLICANT (stamp sticker acceptable) Page 14 Fm SA1603 Insulin Pumps - continued RENEWAL - severe unexplained hypoglycaemia Applications only from a relevant specialist nurse practitioner. Approvals valid f 3 months. Patient is continuing to derive benefit accding to the treatment plan agreed at induction of at least a 50% reduction from baseline in hypoglycaemic events HbA1c has not increased by me than 5 mmol/mol from baseline It has been at least 4 years since the last insulin pump was received by the patient The pump is due f replacement Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope See also: INITIAL APPLICATION - HbA1c p15, RENEWAL - HbA1c p16, INITIAL APPLICATION - Previous use befe 1 September 2012 p17 RENEWAL - Previous use befe 1 September 2012 p18 I confirm the above details are crect that in signing this fm I underst I may be audited.

15 APPLICANT (stamp sticker acceptable) Page 15 Fm SA1603 Insulin Pumps - continued INITIAL APPLICATION - HbA1c Applications only from a relevant specialist nurse practitioner. Approvals valid f 3 months. Patient has type 1 diabetes has undergone a pancreatectomy has cystic fibrosis-related diabetes Has undertaken carbohydrate counting education (either a carbohydrate counting course direct education from an appropriate health professional) Applicant is part of a multidisciplinary team experienced in the management of type 1 diabetes care Has adhered to an intensive MDI regimen using analogue insulins f at least six months Has unpredictable significant variability in blood glucose including significant hypoglycaemia affecting the ability to reduce HbA1 In the opinion of the treating clinician, HbA1c could be reduced by at least 10 mmol/mol using insulin pump treatment Has typical HbA1c results between the following range: equal to greater than 65 mmol/mol equal to less than 90 mmol/mol Has been evaluated by the multidisciplinary team f their suitability f insulin pump therapy Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope See also: RENEWAL - HbA1c p16, INITIAL APPLICATION - Previous use befe 1 September 2012 p17 RENEWAL - Previous use befe 1 September 2012 p18 I confirm the above details are crect that in signing this fm I underst I may be audited.

16 APPLICANT (stamp sticker acceptable) Page 16 Fm SA1603 Insulin Pumps - continued RENEWAL - HbA1c Applications only from a relevant specialist nurse practitioner. Approvals valid f 3 months. Patient is continuing to derive benefit accding to the treatment plan agreed at induction of achieving maintaining a reduction in HbA1c from baseline of 10 mmol/mol The number of severe unexplained recurrent hypoglycaemic episodes has not increased from baseline It has been at least 4 years since the last insulin pump was received by the patient The pump is due f replacement Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope See also: INITIAL APPLICATION - Previous use befe 1 September 2012 p17 RENEWAL - Previous use befe 1 September 2012 p18 I confirm the above details are crect that in signing this fm I underst I may be audited.

17 APPLICANT (stamp sticker acceptable) Page 17 Fm SA1603 Insulin Pumps - continued INITIAL APPLICATION - Previous use befe 1 September 2012 Applications only from a relevant specialist nurse practitioner. Approvals valid f 3 months. Patient has type 1 diabetes has undergone a pancreatectomy has cystic fibrosis-related diabetes Was already on pump treatment pri to 1 September 2012 had been evaluated by the multidisciplinary team f their suitability f insulin pump therapy at the time of initiating that pump treatment continues to benefit from pump treatment The patient has adhered to an intensive MDI regimen using analogue insulins f at least six months pri to initiating pump therapy The patient is continuing to derive benefit from pump therapy The patient had achieved is maintaining a HbA1c of equal to less than 80 mmol/mol on pump therapy The patient has had no increase in severe unexplained hypoglycaemic episodes from baseline The patient s HbA1c has not deteriated me than 5 mmol/mol from baseline It has been at least 4 years since the last insulin pump was received by the patient The pump is due f replacement Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope See also: RENEWAL - Previous use befe 1 September 2012 p18 I confirm the above details are crect that in signing this fm I underst I may be audited.

18 APPLICANT (stamp sticker acceptable) Page 18 Fm SA1603 Insulin Pumps - continued RENEWAL - Previous use befe 1 September 2012 Applications only from a relevant specialist nurse practitioner. Approvals valid f 3 months. The patient is continuing to derive benefit accding to the treatment plan has maintained a HbA1c of equal to less than 80 mmol/mol The patient s HbA1c has not deteriated me than 5 mmol/mol fromthe time of commencing pump treatment The patient has not had an increase in severe unexplained hypoglycaemic episodes from baseline It has been at least 4 years since the last insulin pump was received by the patient The pump is due f replacement Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope I confirm the above details are crect that in signing this fm I underst I may be audited.

19 APPLICANT (stamp sticker acceptable) Page 19 Fm SA1604 Insulin Pump Consumables INITIAL APPLICATION - permanent neonatal diabetes Applications only from a relevant specialist nurse practitioner. Approvals valid f 9 months. Patient has permanent neonatal diabetes A MDI regimen trial is inappropriate Has been evaluated by the multidisciplinary team f their suitability f insulin pump therapy Patient/Parent/Guardian has undertaken carbohydrate counting education (either a carbohydrate counting course direct education from an appropriate health professional) Applicant is part of a multidisciplinary team experienced in the management of type 1 diabetes care Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope RENEWAL - permanent neonatal diabetes Applications only from a relevant specialist nurse practitioner. Approvals valid f 2 years. Patient is continuing to derive benefit accding to the treatment plan agreed at induction Patient remains fully compliant transition to MDI is considered inappropriate by the treating physician Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope See also: INITIAL APPLICATION - severe unexplained hypoglycaemia p20, RENEWAL - severe unexplained hypoglycaemia p20, INITIAL APPLICATION - HbA1c p21, RENEWAL - HbA1c p21, INITIAL APPLICATION - Previous use befe 1 September 2012 p22 RENEWAL - Previous use befe 1 September 2012 p22 I confirm the above details are crect that in signing this fm I underst I may be audited.

20 APPLICANT (stamp sticker acceptable) Page 20 Fm SA1604 Insulin Pump Consumables - continued INITIAL APPLICATION - severe unexplained hypoglycaemia Applications only from a relevant specialist nurse practitioner. Approvals valid f 9 months. Patient has type 1 diabetes has undergone a pancreatectomy has cystic fibrosis-related diabetes Has undertaken carbohydrate counting education (either a carbohydrate counting course direct education from an appropriate health professional) Applicant is part of a multidisciplinary team experienced in the management of type 1 diabetes care Has adhered to an intensive MDI regimen using analogue insulins f at least six months Has had four severe unexplained hypoglycaemic episodes over a six month period (severe as defined as requiring the assistance of another person) Has an average HbA1c between the following range: equal to greater than 53 mmol/mol equal to less than 90 mmol/mol Has been evaluated by the multidisciplinary team f their suitability f insulin pump therapy Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope RENEWAL - severe unexplained hypoglycaemia Applications only from a relevant specialist nurse practitioner. Approvals valid f 2 years. Patient is continuing to derive benefit accding to the treatment plan agreed at induction of at least a 50% reduction from baseline in hypoglycaemic events HbA1c has not increased by me than 5 mmol/mol from baseline Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope See also: INITIAL APPLICATION - HbA1c p21, RENEWAL - HbA1c p21, INITIAL APPLICATION - Previous use befe 1 September 2012 p22 RENEWAL - Previous use befe 1 September 2012 p22 I confirm the above details are crect that in signing this fm I underst I may be audited.

21 APPLICANT (stamp sticker acceptable) Page 21 Fm SA1604 Insulin Pump Consumables - continued INITIAL APPLICATION - HbA1c Applications only from a relevant specialist nurse practitioner. Approvals valid f 9 months. Patient has type 1 diabetes has undergone a pancreatectomy has cystic fibrosis-related diabetes Has undertaken carbohydrate counting education (either a carbohydrate counting course direct education from an appropriate health professional) Applicant is part of a multidisciplinary team experienced in the management of type 1 diabetes care Has adhered to an intensive MDI regimen using analogue insulins f at least six months Has unpredictable significant variability in blood glucose including significant hypoglycaemia affecting the ability to reduce HbA1 In the opinion of the treating clinician, HbA1c could be reduced by at least 10 mmol/mol using insulin pump treatment Has typical HbA1c results between the following range: equal to greater than 65 mmol/mol equal to less than 90 mmol/mol Has been evaluated by the multidisciplinary team f their suitability f insulin pump therapy Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope RENEWAL - HbA1c Applications only from a relevant specialist nurse practitioner. Approvals valid f 2 years. Patient is continuing to derive benefit accding to the treatment plan agreed at induction of achieving maintaining a reduction in HbA1c from baseline of 10 mmol/mol The number of severe unexplained recurrent hypoglycaemic episodes has not increased from baseline Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope See also: INITIAL APPLICATION - Previous use befe 1 September 2012 p22 RENEWAL - Previous use befe 1 September 2012 p22 I confirm the above details are crect that in signing this fm I underst I may be audited.

22 APPLICANT (stamp sticker acceptable) Page 22 Fm SA1604 Insulin Pump Consumables - continued INITIAL APPLICATION - Previous use befe 1 September 2012 Applications only from a relevant specialist nurse practitioner. Approvals valid f 2 years. Patient has type 1 diabetes has undergone a pancreatectomy has cystic fibrosis-related diabetes Was already on pump treatment pri to 1 September 2012 had been evaluated by the multidisciplinary team f their suitability f insulin pump therapy at the time of initiating that pump treatment continues to benefit from pump treatment The patient has adhered to an intensive MDI regimen using analogue insulins f at least six months pri to initiating pump therapy The patient is continuing to derive benefit from pump therapy The patient had achieved is maintaining a HbA1c of equal to less than 80 mmol/mol on pump therapy The patient has had no increase in severe unexplained hypoglycaemic episodes from baseline The patient s HbA1c has not deteriated me than 5 mmol/mol from baseline Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope RENEWAL - Previous use befe 1 September 2012 Applications only from a relevant specialist nurse practitioner. Approvals valid f 2 years. The patient is continuing to derive benefit accding to the treatment plan has maintained a HbA1c of equal to less than 80 mmol/mol The patient s HbA1c has not deteriated me than 5 mmol/mol from initial application The patient has not had an increase in severe unexplained hypoglycaemic episodes from baseline Applicant is a relevant specialist Applicant is a nurse practitioner wking within their vocational scope I confirm the above details are crect that in signing this fm I underst I may be audited.

23 APPLICANT (stamp sticker acceptable) Page 23 Fm SA1739 Ursodeoxycholic Acid Note: Ursodeoxycholic acid is not an appropriate therapy f patients requiring a liver transplant (bilirubin > 100 micromol/l; decompensated cirrhosis). These patients should be referred to an appropriate transplant centre. Treatment failure -- doubling of serum bilirubin levels, absence of a significant decrease in ALP ALT AST, development of varices, ascites encephalopathy, marked wsening of pruritus fatigue, histological progression by two stages, to cirrhosis, need f transplantation. INITIAL APPLICATION - Alagille syndrome progressive familial intrahepatic cholestasis Applications from any relevant practitioner. Approvals valid without further renewal unless notified. Patient has been diagnosed with Alagille syndrome Patient has progressive familial intrahepatic cholestasis INITIAL APPLICATION - Chronic severe drug induced cholestatic liver injury Applications from any relevant practitioner. Approvals valid f 3 months. Patient has chronic severe drug induced cholestatic liver injury Cholestatic liver injury not due to Total Parenteral Nutrition (TPN) use in adults Treatment with ursodeoxycholic acid may prevent hospital admission reduce duration of stay INITIAL APPLICATION - Primary biliary cholangitis Applications from any relevant practitioner. Approvals valid f 6 months. Primary biliary cholangitis confirmed by antimitochondrial antibody titre (AMA) > 1:80, raised cholestatic liver enzymes with without raised serum IgM, if AMA is negative, by liver biopsy Patient not requiring a liver transplant (bilirubin > 100 umol/l; decompensated cirrhosis) INITIAL APPLICATION - Pregnancy Applications from any relevant practitioner. Approvals valid f 6 months. Prerequisites (tick box where appropriate) The patient diagnosed with cholestasis of pregnancy See also: INITIAL APPLICATION - Haematological Transplant p24, INITIAL APPLICATION - Total parenteral nutrition induced cholestasis p24, RENEWAL - Chronic severe drug induced cholestatic liver injury p24, RENEWAL - Pregnancy/Primary biliary cholangitis p24 RENEWAL - Total parenteral nutrition induced cholestasis p24 I confirm the above details are crect that in signing this fm I underst I may be audited.

24 APPLICANT (stamp sticker acceptable) Page 24 Fm SA1739 Ursodeoxycholic Acid - continued INITIAL APPLICATION - Haematological Transplant Applications from any relevant practitioner. Approvals valid f 6 months. Patient at risk of veno-occlusive disease has hepatic impairment is undergoing conditioning treatment pri to allogenic stem cell bone marrow transplantation Treatment f up to 13 weeks INITIAL APPLICATION - Total parenteral nutrition induced cholestasis Applications from any relevant practitioner. Approvals valid f 6 months. Paediatric patient has developed abnmal liver function as indicated on testing which is likely to be induced by Total Parenteral Nutrition (TPN) Liver function has not improved with modifying the TPN composition RENEWAL - Chronic severe drug induced cholestatic liver injury Applications from any relevant practitioner. Approvals valid f 6 months. Prerequisites (tick box where appropriate) The patient continues to benefit from treatment RENEWAL - Pregnancy/Primary biliary cholangitis Applications from any relevant practitioner. Approvals valid f 2 years. Prerequisites (tick box where appropriate) The treatment remains appropriate the patient is benefiting from treatment RENEWAL - Total parenteral nutrition induced cholestasis Applications from any relevant practitioner. Approvals valid f 6 months. Prerequisites (tick box where appropriate) The paediatric patient continues to require TPN who is benefiting from treatment, defined as a sustained improvement in bilirubin levels I confirm the above details are crect that in signing this fm I underst I may be audited.

25 APPLICANT (stamp sticker acceptable) Page 25 Fm SA1691 Methylnaltrexone bromide INITIAL APPLICATION - Opioid induced constipation Applications from any relevant practitioner. Approvals valid without further renewal unless notified. The patient is receiving palliative care Oral rectal treatments f opioid induced constipation are ineffective Oral rectal treatments f opioid induced constipation are unable to be tolerated I confirm the above details are crect that in signing this fm I underst I may be audited.

26 APPLICANT (stamp sticker acceptable) Page 26 Fm SA1593 Galsulfase INITIAL APPLICATION Applications only from a metabolic physician. Approvals valid f 12 months. The patient has been diagnosed with mucopolysaccharidosis VI Diagnosis confirmed by demonstration of N-acetyl-galactosamine-4-sulfatase (arylsulfatase B) deficiency by either enzyme activity assay in leukocytes skin fibroblasts Detection of two disease causing mutations patient has a sibling who is known to have mucopolysaccharidosis VI RENEWAL Applications only from a metabolic physician. Approvals valid f 12 months. The treatment remains appropriate f the patient the patient is benefiting from treatment Patient has not had severe infusion-related adverse reactions which were not preventable by appropriate pre-medication / adjustment of infusion rates Patient has not developed another life threatening severe disease where the long term prognosis is unlikely to be influenced by Enzyme Replacement Therapy (ERT) Patient has not developed another medical condition that might reasonably be expected to compromise a response to ERT I confirm the above details are crect that in signing this fm I underst I may be audited.

27 APPLICANT (stamp sticker acceptable) Page 27 Fm SA1598 Sodium phenylbutyrate INITIAL APPLICATION Applications only from a metabolic physician. Approvals valid f 12 months. Prerequisites (tick box where appropriate) the patient has a diagnosis of a urea cycle disder involving a deficiency of carbamylphosphate synthetase, nithine transcarbamylase argininosuccinate synthetase RENEWAL Applications only from a metabolic physician. Approvals valid f 12 months. Prerequisites (tick box where appropriate) the treatment remains appropriate the patient is benefiting from treatment I confirm the above details are crect that in signing this fm I underst I may be audited.

28 APPLICANT (stamp sticker acceptable) Page 28 Fm SA1599 Sodium benzoate INITIAL APPLICATION Applications only from a metabolic physician. Approvals valid f 12 months. Prerequisites (tick box where appropriate) the patient has a diagnosis of a urea cycle disder RENEWAL Applications only from a metabolic physician. Approvals valid f 12 months. Prerequisites (tick box where appropriate) the treatment remains appropriate the patient is benefiting from treatment I confirm the above details are crect that in signing this fm I underst I may be audited.

29 APPLICANT (stamp sticker acceptable) Page 29 Fm SA1622 Alglucosidase Alfa INITIAL APPLICATION Applications only from a metabolic physician. Approvals valid f 12 months. The patient is aged up to 24 months at the time of initial application has been diagnosed with infantile Pompe disease Diagnosis confirmed by documented deficiency of acid alpha-glucosidase by prenatal diagnosis using chionic villus biopsies / cultured amniotic cells Documented deficiency of acid alpha-glucosidase, urinary tetrasaccharide testing indicating a diagnostic elevation of glucose tetrasaccharides Documented deficiency of acid alpha-glucosidase, documented molecular genetic testing indicating a disease-causing mutation in the acid alpha-glucosidase gene (GAA gene) Documented urinary tetrasaccharide testing indicating a diagnostic elevation of glucose tetrasaccharides, molecular genetic testing indicating a disease-causing mutation in the GAA gene Patient has not required long-term invasive ventilation f respiraty failure pri to starting enzyme replacement therapy (ERT) Patient does not have another life-threatening severe disease where the prognosis is unlikely to be influenced by ERT might be reasonably expected to compromise a response to ERT Alglucosidase alfa to be administered at doses no greater than 20 mg/kg every 2 weeks See also: RENEWAL p30 I confirm the above details are crect that in signing this fm I underst I may be audited.

30 APPLICANT (stamp sticker acceptable) Page 30 Fm SA1622 Alglucosidase Alfa - continued RENEWAL Applications only from a metabolic physician. Approvals valid f 12 months. The treatment remains appropriate f the patient the patient is benefiting from treatment Alglucosidase alfa to be administered at doses no greater than 20 mg/kg every 2 weeks Patient has not had severe infusion-related adverse reactions which were not preventable by appropriate pre-medication / adjustment of infusion rates Patient has not developed another life threatening severe disease where the long term prognosis is unlikely to be influenced by ERT Patient has not developed another medical condition that might reasonably be expected to compromise a response to ERT There is no evidence of life threatening progression of respiraty disease as evidenced by the needed f >14 days of invasive ventilation There is no evidence of new progressive cardiomyopathy I confirm the above details are crect that in signing this fm I underst I may be audited.

31 APPLICANT (stamp sticker acceptable) Page 31 Fm SA1623 Idursulfase INITIAL APPLICATION Applications only from a metabolic physician. Approvals valid f 24 weeks. The patient has been diagnosed with Hunter Syndrome (mucopolysaccharidosis II) Diagnosis confirmed by demonstration of iduronate 2-sulfatase deficiency in white blood cells by either enzyme assay in cultured skin fibroblasts Detection of a disease causing mutation in the iduronate 2-sulfatase gene Patient is going to proceed with a haematopoietic stem cell transplant (HSCT) within the next 3 months treatment with idursulfase would be bridging treatment to transplant Patient has not required long-term invasive ventilation f respiraty failure pri to starting Enzyme Replacement Therapy (ERT) Idursulfase to be administered f a total of 24 weeks (equivalent to 12 weeks pre- 12 weeks post-hsct) at doses no greater than 0.5 mg/kg every week I confirm the above details are crect that in signing this fm I underst I may be audited.

32 APPLICANT (stamp sticker acceptable) Page 32 Fm SA1695 Laronidase INITIAL APPLICATION Applications only from a metabolic physician. Approvals valid f 24 weeks. The patient has been diagnosed with Hurler Syndrome (mucopolysacchardosis I-H) Diagnosis confirmed by demonstration of alpha-l-iduronidase deficiency in white blood cells by either enzyme assay in cultured skin fibroblasts Detection of two disease causing mutations in the alpha-l-iduronidase gene patient has a sibling who is known to have Hurler syndrome Patient is going to proceed with a haematopoietic stem cell transplant (HSCT) within the next 3 months treatment with laronidase would be bridging treatment to transplant Patient has not required long-term invasive ventilation f respiraty failure pri to starting Enzyme Replacement Therapy (ERT) Laronidase to be administered f a total of 24 weeks (equivalent to 12 weeks pre- 12 post-hsct) at doses no greater than 100 units/kg every week I confirm the above details are crect that in signing this fm I underst I may be audited.

33 A disder of intracellular cobalamin metabolism Ministry of Health APPLICANT (stamp sticker acceptable) Page 33 Fm SA1727 Betaine INITIAL APPLICATION Applications only from a metabolic physician. Approvals valid f 12 months. The patient has a confirmed diagnosis of homocystinuria A cystathionine beta-synthase (CBS) deficiency A 5,10-methylene-tetrahydrofolate reductase (MTHFR) deficiency An appropriate homocysteine level has not been achieved despite a sufficient trial of appropriate vitamin supplementation RENEWAL Applications only from a metabolic physician. Approvals valid f 12 months. Prerequisites (tick box where appropriate) the treatment remains appropriate the patient is benefiting from treatment I confirm the above details are crect that in signing this fm I underst I may be audited.

34 Treatment with sapropterin is required f a second subsequent pregnancy to suppt management of their PKU during pregnancy Ministry of Health APPLICANT (stamp sticker acceptable) Page 34 Fm SA1757 Sapropterin dihydrochlide INITIAL APPLICATION Applications only from a metabolic physician. Approvals valid f 1 month. Patient has phenylketonuria (PKU) is pregnant actively planning to become pregnant Treatment with sapropterin is required to suppt management of PKU during pregnancy Sapropterin to be administered at doses no greater than a total daily dose of 20 mg/kg Sapropterin to be used alone in combination with PKU dietary management RENEWAL Total treatment duration with sapropterin will not exceed 22 months f each pregnancy (includes time f planning becoming pregnant) treatment will be stopped after delivery Applications only from a metabolic physician any relevant practitioner on the recommendation of a metabolic physician. Approvals valid f 12 months. Following the initial one-month approval, the patient has demonstrated an adequate response to a 2 to 4 week trial of sapropterin with a clinically appropriate reduction in phenylalanine levels to suppt management of PKU during pregnancy On subsequent renewal applications, the patient has previously demonstrated response to treatment with sapropterin maintained adequate phenylalanine levels to suppt management of PKU during pregnancy Patient continues to be pregnant treatment with sapropterin will not continue after delivery Patient is actively planning a pregnancy this is the first renewal f treatment with sapropterin Sapropterin to be administered at doses no greater than a total daily dose of 20 mg/kg Sapropterin to be used alone in combination with PKU dietary management Total treatment duration with sapropterin will not exceed 22 months f each pregnancy (includes time f planning becoming pregnant) treatment will be stopped after delivery I confirm the above details are crect that in signing this fm I underst I may be audited.

35 APPLICANT (stamp sticker acceptable) Page 35 Fm SA1720 Vitabdeck INITIAL APPLICATION Applications from any relevant practitioner. Approvals valid without further renewal unless notified. Patient has cystic fibrosis with pancreatic insufficiency Patient is an infant child with liver disease sht gut syndrome Patient has severe malabsption syndrome I confirm the above details are crect that in signing this fm I underst I may be audited.

36 APPLICANT (stamp sticker acceptable) Page 36 Fm SA1036 Multivitamins (Paediatric Seravit) INITIAL APPLICATION Applications from any relevant practitioner. Approvals valid without further renewal unless notified. Prerequisites (tick box where appropriate) The patient has inbn errs of metabolism RENEWAL Applications from any relevant practitioner. Approvals valid without further renewal unless notified. Prerequisites (tick box where appropriate) Patient has had a previous approval f multivitamins I confirm the above details are crect that in signing this fm I underst I may be audited.

37 APPLICANT (stamp sticker acceptable) Page 37 Fm SA1546 Multivitamin renal (Clinicians Renal Vit) INITIAL APPLICATION Applications from any relevant practitioner. Approvals valid without further renewal unless notified. The patient has chronic kidney disease is receiving either peritoneal dialysis haemodialysis The patient has chronic kidney disease grade 5, defined as patient with an estimated glomerular filtration rate of < 15 ml/min/1.73 m² body surface area (BSA) I confirm the above details are crect that in signing this fm I underst I may be audited.

38 Rapid crection of anaemia is required Ministry of Health APPLICANT (stamp sticker acceptable) Page 38 Fm SA1675 Ferric carboxymaltose INITIAL APPLICATION - serum ferritin less than equal to 20 mcg/l Applications from any medical practitioner. Approvals valid f 3 months. Patient has been diagnosed with iron-deficiency anaemia with a serum ferritin level of less than equal to 20 mcg/l Patient has been compliant with al iron treatment treatment has proven ineffective Treatment with al iron has resulted in dose-limiting intolerance RENEWAL - serum ferritin less than equal to 20 mcg/l Applications from any medical practitioner. Approvals valid f 3 months. Patient continues to have iron-deficiency anaemia with a serum ferritin level of less than equal to 20 mcg/l A re-trial with al iron is clinically inappropriate INITIAL APPLICATION - iron deficiency anaemia Applications only from an internal medicine physician, obstetrician, gynaecologist, anaesthetist any other medical practitioner on the recommendation of a internal medicine physician, obstetrician, gynaecologist anaesthetist. Approvals valid f 3 months. Patient has been diagnosed with iron-deficiency anaemia Patient has been compliant with al iron treatment treatment has proven ineffective Treatment with al iron has resulted in dose-limiting intolerance Patient has symptomatic heart failure, chronic kidney disease stage 3 me active inflammaty bowel disease a trial of al iron is unlikely to be effective Rapid crection of anaemia is required See also: RENEWAL - iron deficiency anaemia p39 I confirm the above details are crect that in signing this fm I underst I may be audited.

39 APPLICANT (stamp sticker acceptable) Page 39 Fm SA1675 Ferric carboxymaltose - continued RENEWAL - iron deficiency anaemia Applications only from an internal medicine physician, obstetrician, gynaecologist, anaesthetist any other medical practitioner on the recommendation of a internal medicine physician, obstetrician, gynaecologist anaesthetist. Approvals valid f 3 months. Patient continues to have iron-deficiency anaemia A re-trial with al iron is clinically inappropriate I confirm the above details are crect that in signing this fm I underst I may be audited.

40 Page 40 Blood Blood Fming Organs

41 APPLICANT (stamp sticker acceptable) Page 41 Fm SA1469 Hypoplastic Haemolytic INITIAL APPLICATION - chronic renal failure Applications from any specialist. Approvals valid f 2 years. Patient in chronic renal failure Haemoglobin is less than equal to 100g/L Patient does not have diabetes mellitus Glomerular filtration rate is less than equal to 30ml/min Patient has diabetes mellitus Glomerular filtration rate is less than equal to 45ml/min Patient is on haemodialysis peritoneal dialysis Note: Erythropoietin alfa is indicated in the treatment of anaemia associated with chronic renal failure (CRF) where no cause f anaemia other than CRF is detected there is adequate moniting of iron stes iron replacement therapy. See also: INITIAL APPLICATION - myelodysplasia p42, RENEWAL - chronic renal failure p42 RENEWAL - myelodysplasia p43 I confirm the above details are crect that in signing this fm I underst I may be audited.

42 APPLICANT (stamp sticker acceptable) Page 42 Fm SA1469 Hypoplastic Haemolytic - continued INITIAL APPLICATION - myelodysplasia Applications from any specialist. Approvals valid f 2 months. Patient has a confirmed diagnosis of myelodysplasia (MDS)* Has had symptomatic anaemia with haemoglobin <100g/L is red cell transfusion-dependent Patient has very low, low intermediate risk MDS based on the WHO classification based prognostic scing system f myelodysplastic syndrome (WPSS) Other causes of anaemia such as B12 folate deficiency have been excluded Patient has a serum erythropoietin level of <500 IU/L The minimum necessary dose of erythropoietin would be used will not exceed 80,000 iu per week Note: Indication marked with * is an unapproved indication RENEWAL - chronic renal failure Applications from any specialist. Approvals valid f 2 years. Prerequisites (tick box where appropriate) the treatment remains appropriate the patient is benefiting from treatment Note: Erythropoietin alfa is indicated in the treatment of anaemia associated with chronic renal failure (CRF) where no cause f anaemia other than CRF is detected there is adequate moniting of iron stes iron replacement therapy. See also: RENEWAL - myelodysplasia p43 I confirm the above details are crect that in signing this fm I underst I may be audited.

43 APPLICANT (stamp sticker acceptable) Page 43 Fm SA1469 Hypoplastic Haemolytic - continued RENEWAL - myelodysplasia Applications from any specialist. Approvals valid f 12 months. The patient's transfusion requirement continues to be reduced with erythropoietin treatment Transfmation to acute myeloid leukaemia has not occurred The minimum necessary dose of erythropoietin would be used will not exceed 80,000 iu per week Note: Indication marked with * is an unapproved indication I confirm the above details are crect that in signing this fm I underst I may be audited.

44 Patient has a platelet count of less than equal to 10,000 platelets per microlitre Ministry of Health APPLICANT (stamp sticker acceptable) Page 44 Fm SA1743 Eltrombopag INITIAL APPLICATION - idiopathic thrombocytopenic purpura - post-splenectomy Applications only from a haematologist. Approvals valid f 6 weeks. Patient has had a splenectomy Two immunosuppressive therapies have been trialled failed after therapy of 3 months each ( 1 month f rituximab) Patient has a platelet count of 20,000 to 30,000 platelets per microlitre has evidence of significant mucocutaneous bleeding Patient has a platelet count of less than equal to 20,000 platelets per microlitre has evidence of active bleeding INITIAL APPLICATION - idiopathic thrombocytopenic purpura - preparation f splenectomy Applications only from a haematologist. Approvals valid f 6 weeks. Prerequisites (tick box where appropriate) The patient requires eltrombopag treatment as preparation f splenectomy INITIAL APPLICATION - idiopathic thrombocytopenic purpura contraindicated to splenectomy Applications only from a haematologist. Approvals valid f 3 months. Patient has a significant well-documented contraindication to splenectomy f clinical reasons Two immunosuppressive therapies have been trialled failed after therapy of 3 months each ( 1 month f rituximab) Patient has immune thrombocytopenic purpura* with a platelet count of less than equal to 20,000 platelets per microliter Patient has immune thrombocytopenic purpura* with a platelet count of 20,000 to 30,000 platelets per microlitre significant mucocutaneous bleeding See also: INITIAL APPLICATION - severe aplastic anaemia p45, RENEWAL - idiopathic thrombocytopenic purpura - post-splenectomy p45, RENEWAL - idiopathic thrombocytopenic purpura contraindicated to splenectomy p45 RENEWAL - severe aplastic anaemia p46 I confirm the above details are crect that in signing this fm I underst I may be audited.

45 APPLICANT (stamp sticker acceptable) Page 45 Fm SA1743 Eltrombopag - continued INITIAL APPLICATION - severe aplastic anaemia Applications only from a haematologist. Approvals valid f 3 months. Two immunosuppressive therapies have been trialled failed after therapy of at least 3 months duration Patient has severe aplastic anaemia with a platelet count of less than equal to 20,000 platelets per microliter Patient has severe aplastic anaemia with a platelet count of 20,000 to 30,000 platelets per microlitre significant mucocutaneous bleeding RENEWAL - idiopathic thrombocytopenic purpura - post-splenectomy Applications only from a haematologist. Approvals valid f 12 months. Prerequisites (tick box where appropriate) The patient has obtained a response (see Note) from treatment during the initial approval subsequent renewal periods further treatment is required Note: Response to treatment is defined as a platelet count of >30,000 platelets per microlitre. RENEWAL - idiopathic thrombocytopenic purpura contraindicated to splenectomy Applications only from a haematologist. Approvals valid f 12 months. The patient s significant contraindication to splenectomy remains The patient has obtained a response from treatment during the initial approval period Patient has maintained a platelet count of at least 50,000 platelets per microlitre on treatment Further treatment with eltrombopag is required to maintain response See also: RENEWAL - severe aplastic anaemia p46 I confirm the above details are crect that in signing this fm I underst I may be audited.

46 APPLICANT (stamp sticker acceptable) Page 46 Fm SA1743 Eltrombopag - continued RENEWAL - severe aplastic anaemia Applications only from a haematologist. Approvals valid f 12 months. The patient has obtained a response from treatment of at least 20,000 platelets per microlitre above baseline during the initial approval period Platelet transfusion independence f a minimum of 8 weeks during the initial approval period I confirm the above details are crect that in signing this fm I underst I may be audited.

47 APPLICANT (stamp sticker acceptable) Page 47 Fm SA1201 Prasugrel INITIAL APPLICATION - conary angioplasty bare metal stent Applications from any relevant practitioner. Approvals valid f 6 months. Prerequisites (tick box where appropriate) The patient has undergone conary angioplasty in the previous 4 weeks is clopidogrel-allergic* INITIAL APPLICATION - drug eluting stent Applications from any relevant practitioner. Approvals valid f 12 months. Prerequisites (tick box where appropriate) The patient has had a drug-eluting cardiac stent inserted in the previous 4 weeks is clopidogrel-allergic* INITIAL APPLICATION - stent thromobosis Applications from any relevant practitioner. Approvals valid without further renewal unless notified. Prerequisites (tick box where appropriate) patient has experienced cardiac stent thrombosis whilst on clopidogrel RENEWAL - conary angioplasty bare metal stent Applications from any relevant practitioner. Approvals valid f 6 months. Prerequisites (tick box where appropriate) The patient has undergone conary angioplasty had a bare metal cardiac stent inserted in the previous 4 weeks is clopidogrel-allergic* RENEWAL - drug eluting stent Applications from any relevant practitioner. Approvals valid f 12 months. Prerequisites (tick box where appropriate) had a drug-eluting cardiac stent inserted in the previous 4 weeks is clopidogrel-allergic* Note: * Clopidogrel allergy is defined as a histy of anaphylaxis, urticaria, generalised rash asthma (in non-asthmatic patients) developing soon after clopidogrel is started is considered unlikely to be caused by any other treatment. I confirm the above details are crect that in signing this fm I underst I may be audited.