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Intravenous Enzyme Replacement Therapy (ERT) for Gaucher Disease
UnitedHealthcare® Commercial Medical Benefit Drug Policy Intravenous Enzyme Replacement Therapy (ERT) for Gaucher Disease Policy Number: 2021D0048K Effective Date: April 1, 2021 Instructions for Use Table of Contents Page Related Commercial Policy Coverage Rationale ....................................................................... 1 • Provider Administered Drugs – Site of Care Applicable Codes .......................................................................... 3 Background.................................................................................... 3 Community Plan Policy Benefit Considerations .................................................................. 3 • Intravenous Enzyme Replacement Therapy (ERT) Clinical Evidence ........................................................................... 4 for Gaucher Disease U.S. Food and Drug Administration ............................................. 6 Centers for Medicare and Medicaid Services ............................. 6 References ..................................................................................... 7 Policy History/Revision Information ............................................. 8 Instructions for Use ....................................................................... 8 Coverage Rationale See Benefit Considerations This policy refers to the following drug products, all of which are intravenous enzyme replacement therapies used in the treatment of Gaucher disease: Cerezyme® (imiglucerase) Elelyso® (taliglucerase) VPRIV® (velaglucerase) -
Drug Consumption in 2017 - 2020
Page 1 Drug consumption in 2017 - 2020 2020 2019 2018 2017 DDD/ DDD/ DDD/ DDD/ 1000 inhab./ Hospital 1000 inhab./ Hospital 1000 inhab./ Hospital 1000 inhab./ Hospital ATC code Subgroup or chemical substance day % day % day % day % A ALIMENTARY TRACT AND METABOLISM 322,79 3 312,53 4 303,08 4 298,95 4 A01 STOMATOLOGICAL PREPARATIONS 14,28 4 12,82 4 10,77 6 10,46 7 A01A STOMATOLOGICAL PREPARATIONS 14,28 4 12,82 4 10,77 6 10,46 7 A01AA Caries prophylactic agents 11,90 3 10,48 4 8,42 5 8,45 7 A01AA01 sodium fluoride 11,90 3 10,48 4 8,42 5 8,45 7 A01AA03 olaflur 0,00 - 0,00 - 0,00 - 0,00 - A01AB Antiinfectives for local oral treatment 2,36 8 2,31 7 2,31 7 2,02 7 A01AB03 chlorhexidine 2,02 6 2,10 7 2,09 7 1,78 7 A01AB11 various 0,33 21 0,21 0 0,22 0 0,24 0 A01AD Other agents for local oral treatment 0,02 0 0,03 0 0,04 0 - - A01AD02 benzydamine 0,02 0 0,03 0 0,04 0 - - A02 DRUGS FOR ACID RELATED DISORDERS 73,05 3 71,13 3 69,32 3 68,35 3 A02A ANTACIDS 2,23 1 2,22 1 2,20 1 2,30 1 A02AA Magnesium compounds 0,07 22 0,07 22 0,08 22 0,10 19 A02AA04 magnesium hydroxide 0,07 22 0,07 22 0,08 22 0,10 19 A02AD Combinations and complexes of aluminium, 2,17 0 2,15 0 2,12 0 2,20 0 calcium and magnesium compounds A02AD01 ordinary salt combinations 2,17 0 2,15 0 2,12 0 2,20 0 A02B DRUGS FOR PEPTIC ULCER AND 70,82 3 68,91 3 67,12 3 66,05 4 GASTRO-OESOPHAGEAL REFLUX DISEASE (GORD) A02BA H2-receptor antagonists 0,17 7 0,74 4 1,10 4 1,11 5 A02BA02 ranitidine 0,00 1 0,63 3 0,99 3 0,99 4 A02BA03 famotidine 0,16 7 0,11 8 0,11 10 0,12 9 A02BB Prostaglandins 0,04 62 -
NTSAD Web Page on Miglustat Fran Platt Substrate Reduction Therapy
NTSAD Web Page on Miglustat Fran Platt Substrate Reduction Therapy for LSDs Background Several lysosomal storage diseases (LSDs) involve the storage of fatty molecules within cells of the body that are called sphingolipids (1). This is because an enzyme that normally works to break these molecules down in the lysosome, the waste disposal/recycling center of our cells, does not work properly (2, 3). Some sphingolipids are modified by the cells of our bodies by adding sugars, creating a family of specialized sphingolipids called glycosphingolipids (GSLs) (1). For example, in Gaucher disease a glycosphingolipid called glucosylceramide is not broken down and is stored, whereas in Tay-Sachs and Sandhoff disease it is a glycosphingolipid called GM2 ganglioside that is stored (1). Glycosphingolipids are made in the cells of our bodies by a single metabolic pathway that begins with the addition of a sugar molecule called glucose to a sphingolipid molecule called ceramide (1). The fact that there is only a single major pathway to make most glycosphingolipids offers a potential way of treating these diseases using a small molecule drug (4). How would a drug work? The principle behind this treatment is called substrate reduction therapy (SRT)(4). The idea is to partially block the cells in our body from making glycosphingolipids, specifically stopping them from adding glucose to ceramide, which is the first step in this pathway. This would mean fewer glycosphingolipids are made, so fewer would require breaking down in the lysosome. The aim is to balance the rates of glycosphingolipid manufacture with their impaired rate of breakdown. -
Vpriv-Pm-En.Pdf
PRODUCT MONOGRAPH INCLUDING PATIENT MEDICATION INFORMATION PrVPRIV® velaglucerase alfa Powder for Solution for Injection 400 U/vial, Intravenous Enzyme Replacement Therapy ATC code: A16AB10 Takeda Canada Inc. Date of Initial Approval: 22 Adelaide Street West, Suite 3800 October 1, 2010 Toronto Ontario M5H 4E3 Date of Revision: December 21, 2020 Submission Control No: 241844 VPRIV® and the VPRIV Logo® are registered trademarks of Shire Human Genetic Therapies, Inc. TAKEDA™ and the TAKEDA Logo® are trademarks of Takeda Pharmaceutical Company Limited, used under license. Page 1 of 24 RECENT MAJOR LABEL CHANGES Warnings and Precautions, Immunogenicity (8) 9/2020 Warnings and Precautions, Infusion-Related Reactions (8) 9/2020 Warnings and Precautions, Breast-feeding (8.1.2) 9/2020 TABLE OF CONTENTS PART I: HEALTH PROFESSIONAL INFORMATION ................................................................. 4 1 INDICATIONS .................................................................................................................4 2 CONTRAINDICATIONS ..................................................................................................4 4 DOSAGE AND ADMINISTRATION ................................................................................ 4 4.1 Dosing Considerations ..........................................................................................4 4.2 Recommended Dose and Dosage Adjustment ....................................................... 4 4.3 Administration .......................................................................................................4 -
Estonian Statistics on Medicines 2016 1/41
Estonian Statistics on Medicines 2016 ATC code ATC group / Active substance (rout of admin.) Quantity sold Unit DDD Unit DDD/1000/ day A ALIMENTARY TRACT AND METABOLISM 167,8985 A01 STOMATOLOGICAL PREPARATIONS 0,0738 A01A STOMATOLOGICAL PREPARATIONS 0,0738 A01AB Antiinfectives and antiseptics for local oral treatment 0,0738 A01AB09 Miconazole (O) 7088 g 0,2 g 0,0738 A01AB12 Hexetidine (O) 1951200 ml A01AB81 Neomycin+ Benzocaine (dental) 30200 pieces A01AB82 Demeclocycline+ Triamcinolone (dental) 680 g A01AC Corticosteroids for local oral treatment A01AC81 Dexamethasone+ Thymol (dental) 3094 ml A01AD Other agents for local oral treatment A01AD80 Lidocaine+ Cetylpyridinium chloride (gingival) 227150 g A01AD81 Lidocaine+ Cetrimide (O) 30900 g A01AD82 Choline salicylate (O) 864720 pieces A01AD83 Lidocaine+ Chamomille extract (O) 370080 g A01AD90 Lidocaine+ Paraformaldehyde (dental) 405 g A02 DRUGS FOR ACID RELATED DISORDERS 47,1312 A02A ANTACIDS 1,0133 Combinations and complexes of aluminium, calcium and A02AD 1,0133 magnesium compounds A02AD81 Aluminium hydroxide+ Magnesium hydroxide (O) 811120 pieces 10 pieces 0,1689 A02AD81 Aluminium hydroxide+ Magnesium hydroxide (O) 3101974 ml 50 ml 0,1292 A02AD83 Calcium carbonate+ Magnesium carbonate (O) 3434232 pieces 10 pieces 0,7152 DRUGS FOR PEPTIC ULCER AND GASTRO- A02B 46,1179 OESOPHAGEAL REFLUX DISEASE (GORD) A02BA H2-receptor antagonists 2,3855 A02BA02 Ranitidine (O) 340327,5 g 0,3 g 2,3624 A02BA02 Ranitidine (P) 3318,25 g 0,3 g 0,0230 A02BC Proton pump inhibitors 43,7324 A02BC01 Omeprazole -
2021 Formulary List of Covered Prescription Drugs
2021 Formulary List of covered prescription drugs This drug list applies to all Individual HMO products and the following Small Group HMO products: Sharp Platinum 90 Performance HMO, Sharp Platinum 90 Performance HMO AI-AN, Sharp Platinum 90 Premier HMO, Sharp Platinum 90 Premier HMO AI-AN, Sharp Gold 80 Performance HMO, Sharp Gold 80 Performance HMO AI-AN, Sharp Gold 80 Premier HMO, Sharp Gold 80 Premier HMO AI-AN, Sharp Silver 70 Performance HMO, Sharp Silver 70 Performance HMO AI-AN, Sharp Silver 70 Premier HMO, Sharp Silver 70 Premier HMO AI-AN, Sharp Silver 73 Performance HMO, Sharp Silver 73 Premier HMO, Sharp Silver 87 Performance HMO, Sharp Silver 87 Premier HMO, Sharp Silver 94 Performance HMO, Sharp Silver 94 Premier HMO, Sharp Bronze 60 Performance HMO, Sharp Bronze 60 Performance HMO AI-AN, Sharp Bronze 60 Premier HDHP HMO, Sharp Bronze 60 Premier HDHP HMO AI-AN, Sharp Minimum Coverage Performance HMO, Sharp $0 Cost Share Performance HMO AI-AN, Sharp $0 Cost Share Premier HMO AI-AN, Sharp Silver 70 Off Exchange Performance HMO, Sharp Silver 70 Off Exchange Premier HMO, Sharp Performance Platinum 90 HMO 0/15 + Child Dental, Sharp Premier Platinum 90 HMO 0/20 + Child Dental, Sharp Performance Gold 80 HMO 350 /25 + Child Dental, Sharp Premier Gold 80 HMO 250/35 + Child Dental, Sharp Performance Silver 70 HMO 2250/50 + Child Dental, Sharp Premier Silver 70 HMO 2250/55 + Child Dental, Sharp Premier Silver 70 HDHP HMO 2500/20% + Child Dental, Sharp Performance Bronze 60 HMO 6300/65 + Child Dental, Sharp Premier Bronze 60 HDHP HMO -
Specialty Drugs Requiring Precertification
Specialty Drugs Requiring Precertification Policy Number: Original Effective Date: MM.04.009 09/01/2008 Line(s) of Business: Current Effective Date: HMO; PPO; QUEST Integration 06/27/2014 Section: Prescription Drugs Place(s) of Service: Home; Office; Outpatient; Ambulatory Infusion Suite I. Description A specialty drug is a drug that is typically high in cost (greater than $600 per month) and has one or more of the following characteristics: Specialized patient training on the administration of the drug (including supplies and devices needed for administration) is required. Coordination of care is required prior to drug therapy initiation and/or during therapy. Unique patient compliance and safety monitoring requirements. Unique requirements for handling, shipping and storage. Restricted access or limited distribution. The intent of this policy is to require precertification of designated specialty drugs on the date of approval by the Food and Drug Administration (FDA). This policy applies to drugs covered under the medical benefit and under the prescription drug rider benefit. II. Criteria/Guidelines A. Precertification is required for specialty drugs, effective on the date of approval by the FDA. Refer to Appendices A and B for the lists of drugs requiring precertification. B. A specialty drug is covered (subject to Limitations/Exclusions and Administrative Guidelines) when the following criteria are met: 1. It is prescribed for an FDA approved indication. a. Its use is consistent with the manufacturer’s prescribing information. b. When the FDA-approved indication lacks patient selection specificity, the patient meets the eligibility criteria for the clinical study or studies upon which the FDA approval was based. -
Long-Term Follow-Up and Sudden Unexpected Death in Gaucher Disease Type 3 in Egypt
Long-term follow-up and sudden unexpected death in Gaucher disease type 3 in Egypt Magy Abdelwahab, MD, ABSTRACT PhD Objective: To describe the long-term follow-up and distinct phenotype of a large cohort of patients Derek Blankenship, PhD with Gaucher disease type 3 on enzyme replacement therapy (ERT) in Egypt. Raphael Schiffmann, Methods: A prospective cohort study of 78 patients on ERT who were followed for up to 9 years MD, MHSc with yearly evaluations that included EEG and cognitive testing. Results: Of the patients, 73% were homozygous for the L444P GBA1 mutation; all but 7 were Correspondence to neurologically symptomatic. Supranuclear gaze palsy with variable but stable cognitive function Dr. Schiffmann: was present in 91% of patients. Convergent strabismus and bulbar dysfunction were noted in Raphael.schiffmann@ baylorhealth.edu or 22% and 37%, respectively. Features of oppositional defiant disorder were present in 54% of Dr. Abdelwahab: patients. Twenty-three patients (30%) developed seizures while on ERT for 1–9 years. Of those, [email protected] 12 patients (15%) died suddenly and unexpectedly at a mean age of 6.7 6 5.0 years (range 1.5– 18). Sudden death was usually associated with a seizure disorder or a terminal seizure, but 7 of 12 patients had a preceding normal EEG. An additional 11% had background slowing or epilep- togenic activity on EEG without clinical seizures. There were 3 familial cases of sudden unex- pected death. Conclusions: Despite having the most common GBA1 genotype known to be associated with neuronopathic Gaucher disease, patients with Gaucher disease type 3 in Egypt have a phenotype and a clinical outcome on ERT that are very different from those observed in other populations. -
State and Specialty Pharmacy Drug Reimbursement Rates
State and Specialty Pharmacy Drug Reimbursement The following table lists specialty pharmacy drug reimbursement rates. ForwardHealth defines specialty drugs as drugs requiring comprehensive patient care services, clinical management, and product support services. Drugs that meet ForwardHealth’s definition of specialty drugs will be reimbursed using the following rates. However, these rates do not apply to specialty drugs purchased through the federal 340B Drug Discount Program, which will be reimbursed according to the 340B ingredient cost reimbursement methodology. If ForwardHealth uses State Maximum Allowable Cost (SMAC) reimbursement in the future, the SMAC reimbursement rates will be published in this table. Effective 11/01/2020 EAC* EAC* Label Generic Name - Label - Generic ANEMIA/NEUTROPENIA DRUGS ARANESP DARBEPOETIN ALFA IN POLYSORBAT -1.0% EPOGEN EPOETIN ALFA -1.6% FULPHILA PEGFILGRASTIM-JMDB -1.0% GRANIX TBO-FILGRASTIM -1.0% MIRCERA METHOXY PEG-EPOETIN BETA -0.4% NEULASTA PEGFILGRASTIM -1.0% NEUPOGEN FILGRASTIM -1.0% NIVESTYM FILGRASTIM-AAFI -1.0% PROCRIT EPOETIN ALFA -1.6% RETACRIT EPOETIN ALFA-EPBX -1.0% UDENYCA PEGFILGRASTIM-CBQV -1.0% ZARXIO FILGRASTIM-SNDZ -1.0% ZIEXTENZO PEGFILGRASTIM-BMEZ -1.0% ANTICOAGULANT DRUGS ARIXTRA FONDAPARINUX SODIUM -3.0% -50.0% FONDAPARINUX SODIUM FONDAPARINUX SODIUM -50.0% FRAGMIN DALTEPARIN SODIUM,PORCINE -0.4% THROMBATE III ANTITHROMBIN III (PLASMA DER) -0.4% ANTI-INFECTIVE DRUGS ABELCET AMPHOTERICIN B LIPID COMPLEX -0.4% AMBISOME AMPHOTERICIN B LIPOSOME -0.4% ANCOBON FLUCYTOSINE -0.4% -1.0% -
DTF Prior Authorization Drug List
DTF Prior Authorization Drug List The following is a list of the prescribed drugs included in your prior authorization program and is comprehensive as of the date of publication. Express Scripts Canada® makes every effort to ensure this list is updated regularly. Please note that prior authorization applies to the brand drug and its generic alternatives (if available). As of July 23, 2020 Drug Chemical Ingredient ABSTRAL fentanyl ACTEMRA tocilizumab ADCIRCA tadalafil ADEMPAS riociguat AFINITOR everolimus AIMOVIG erenumab AJOVY fremanezumab ALECENSARO alectinib ALUNBRIG brigatinib ARZERRA ofatumumab AUBAGIO teriflunomide AVASTIN bevacizumab AVSOLA infliximab BALVERSA erdafitinib BANZEL rufinamide BAVENCIO avelumab BENLYSTA belimumab BESPONSA inotuzumab ozogamicin BEOVU brolucizumab BOSULIF bosutinib BOTOX onabotulinumtoxinA BRENZYS etanercept BUDESONIDE budesonide CABLIVI caplacizumab CABOMETYX cabozantinib CALQUENCE acalabrutinib CAPRELSA vandetanib CARBAGLU carglumic acid CERDELGA eliglustat CIMZIA certolizumab CINQAIR reslizumab COSENTYX secukinumab COTELLIC cobimetinib CRESEMBA isavuconazole 1 © Express Scripts Canada. All rights reserved. CRYSVITA burosumab CUVPOSA glycopyrrolate CYRAMZA ramucirumab CYSTADROPS cysteamine DAKLINZA daclatasvir DAURISMO glasdegib DEMYLOCAN decitabine DIACOMIT stiripentol DUODOPA levodopa/carbidopa DUPIXENT dupilumab DYSPORT THERAPEUTIC abobotulinumtoxinA EGRIFTA tesamorelin EMGALITY galcanezumab ENBREL etanercept ENTYVIO vedolizumab EPCLUSA sofosbuvir/velpatasvir ERELZI etanercept ERIVEDGE vismodegib -
Intravenous Enzyme Replacement Therapy (ERT) for Gaucher Disease
UnitedHealthcare® Oxford Clinical Policy Intravenous Enzyme Replacement Therapy (ERT) for Gaucher Disease Policy Number: PHARMACY 270.16 T2 Effective Date: April 1, 2021 Instructions for Use Table of Contents Page Related Policies Coverage Rationale ....................................................................... 1 • Acquired Rare Disease Drug Therapy Exception Prior Authorization Requirements ................................................ 3 Process Applicable Codes .......................................................................... 3 • Drug Coverage Guidelines Background ................................................................................... 3 • Experimental/Investigational Treatment Benefit Considerations .................................................................. 4 • Experimental/Investigational Treatment for NJ Plans Clinical Evidence ........................................................................... 4 U.S. Food and Drug Administration ............................................. 6 • Provider Administered Drugs – Site of Care References ..................................................................................... 7 Policy History/Revision Information ............................................. 8 Instructions for Use ....................................................................... 8 Coverage Rationale See Benefit Considerations This policy refers to the following drug products, all of which are intravenous enzyme replacement therapies used in the treatment of Gaucher disease: -
Cerezyme, INN-Imiglucerase
ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS 1 1. NAME OF THE MEDICINAL PRODUCT Cerezyme 400 Units Powder for concentrate for solution for infusion 2. QUALITATIVE AND QUANTITATIVE COMPOSITION Each vial contains 400 units* of imiglucerase**. After reconstitution, the solution contains 40 units (approximately 1.0 mg) of imiglucerase per ml (400 U/10 ml). * An enzyme unit (U) is defined as the amount of enzyme that catalyses the hydrolysis of one micromole of the synthetic substrate para-nitrophenyl β-D-glucopyranoside (pNP-Glc) per minute at 37°C. ** Imiglucerase is a modified form of human acid β-glucosidase and is produced by recombinant DNA technology using a mammalian Chinese Hamster Ovary (CHO) cell culture, with mannose modification for targeting macrophages. Excipients: For the full list of excipients, see section 6.1. This medicinal product contains sodium and is administered in 0.9% sodium chloride intravenous solution (see section 6.6). After reconstitution, the solution contains 1.24 mmol sodium (400 U/10 mL). To be taken into consideration by patients on a controlled sodium diet. 3. PHARMACEUTICAL FORM Powder for concentrate for solution for infusion. Cerezyme is a white to off-white powder. 4. CLINICAL PARTICULARS 4.1 Therapeutic indications Cerezyme (imiglucerase) is indicated for use as long-term enzyme replacement therapy in patients with a confirmed diagnosis of non-neuronopathic (Type 1) or chronic neuronopathic (Type 3) Gaucher disease who exhibit clinically significant non-neurological manifestations of the disease. The non-neurological manifestations of Gaucher disease include one or more of the following conditions: • anaemia after exclusion of other causes, such as iron deficiency • thrombocytopenia • bone disease after exclusion of other causes such as Vitamin D deficiency • hepatomegaly or splenomegaly 4.2 Posology and method of administration Disease management should be directed by physicians knowledgeable in the treatment of Gaucher disease.