edaravone (Radicava )

Transcription

1 Applies t all prducts administered r underwritten by Blue Crss and Blue Shield f Luisiana and its subsidiary, HMO Luisiana, Inc.(cllectively referred t as the Cmpany ), unless therwise prvided in the applicable cntract. Medical technlgy is cnstantly evlving, and we reserve the right t review and update Medical Plicy peridically. When Services May Be Eligible fr Cverage Cverage fr eligible medical treatments r prcedures, drugs, devices r bilgical prducts may be prvided nly if: Benefits are available in the member s cntract/certificate, and Medical necessity criteria and guidelines are met. Based n review f available data, the Cmpany may cnsider edaravne (Radicava ) fr the treatment f amytrphic lateral sclersis (ALS) t be eligible fr cverage. Patient Selectin Criteria Based n review f available data, the Cmpany may cnsider edaravne (Radicava) when ALL f the fllwing criteria are met: Initial authrizatin (6 mnths) Patient has a definite r prbable diagnsis f Amytrphic Lateral Sclersis (ALS) as defined by the revised El Escrial (Airlie Huse) criteria; AND Requested dse is <60 mg per infusin fr <14 infusins fr the initial mnth, and <10 infusins per mnth fr each subsequent mnth f treatment; AND Patient has a baseline scre f >2 pints n each item f the ALS Functinal Rating Scale- Revised (ALSFRS-R); AND Patient has retained nrmal respiratry functin as evidenced by a Frced Vital Capacity (FVC) >80% OR decline in respiratry functin is better explained by a pulmnary disease prcess (e.g. COPD, asthma, idipathic pulmnary fibrsis). In patients with reduced baseline FVC, recrds may be requested dcumenting diagnsis f the pulmnary disease prcess leading t reduced FVC; AND Patient has a disease duratin f 2 years r less; AND Patient is n cncurrent therapy with generic riluzle unless cntraindicated r previusly nt tlerated; AND Patient has Glmerular Filtratin Rate (GFR) >50 ml/min (either measured r estimated creatinine clearance, egfr) Page 1 f 7

2 Re-authrizatin (6 mnths) Patient cntinues t have a scre f >2 n each item f the ALSFRS-R; AND The requested dse is <60mg per infusin fr <10 infusins per mnth; AND ALSFRS-R scre has nt decreased mre than 6 pints frm previus baseline 6 mnths ag When Services Are Cnsidered Nt Medically Necessary The use f edaravne (Radicava) when patient selectin criteria (except diagnsis f ALS and dsing f Radicava) are nt met is cnsidered t be nt medically necessary.** When Services Are Cnsidered Investigatinal Cverage is nt available fr investigatinal medical treatments r prcedures, drugs, devices r bilgical prducts. Based n review f available data, the Cmpany cnsiders edaravne (Radicava) fr the treatment f any indicatin ther than definite r prbable ALS r at a dse ther than the dse recmmended abve t be investigatinal.* Backgrund/Overview Radicava is indicated fr the treatment f ALS. Its mechanism f actin is unknwn, but is prpsed t be related t its antixidant prperties. Oxidative stress has been implicated as an underlying mechanism in several pathgenic pathways linked t ALS and this drug may prvide prtectin against this xidative damage. Radicava is an intravenus infusin dsed as a 60 mg infusin ver 60 minutes. Fr the first treatment mnth, it shuld be dsed daily fr 14 days fllwed by a 14 day drug-free perid. Subsequent treatment cycles invlve daily dsing fr 10 days ut f 14 day perids fllwed by 14 day drug-free perids. 90% f patients in pivtal studies f Radicava were n cncmitant therapy with riluzle. ALS is a rapidly prgressing, degenerative disease in which the patient s upper and lwer mtr neurns degenerate leading t lss f mtr functin. Patients with ALS present with painless, prgressive muscle atrphy and weakness, which eventually leads t paralysis. Death due t respiratry failure typically ccurs within 3-5 years f diagnsis. Apprximately 14,000-15,000 peple in the U.S. have ALS. The disease ccurs mst cmmnly in peple aged years. The El Escrial criteria, which were used in the clinical studies f Radicava, were develped t standardize the diagnsis f ALS (see table). Disease prgressin is mnitred using the ALSFRS-R, a 13-questin scale that assesses the patient s ability t perfrm nrmal daily activities such as speech, handwriting, cutting fd, and climbing stairs. Each questin is scred n a scale f 0-4 with higher scres representing greater functinal ability (see appendix fr full scale). The ALSFRS-R scre is cmplemented by a measurement f the FVC which is als used t predict survival and measure disease prgressin. Page 2 f 7

3 Diagnsis Definite ALS Prbable ALS Prbable ALS labratrysupprted El Escrial Revised Airlie Huse Criteria Upper mtr neurn (clinical exam) and lwer mtr neurn (clinical, electrphysilgical r neurpathlgical exam) signs: Bulbar regin and > 2 spinal regins OR Three spinal regins Upper and lwer mtr neurn signs in > 2 regins and upper signs rstral t lwer signs Upper and lwer mtr neurn signs in ne regin OR Upper signs alne in ne regin and lwer signs via electrphysilgical criteria f lwer mtr neurn lss > 2 regins Pssible ALS Upper and lwer signs in ne regin OR Upper mtr neurn signs alne in > 2 regins OR Lwer mtr neurn signs rstral t upper mtr neurn signs and unable t prve clinically prbably ALS Surce: Data n file. Radicava Prduct Dssier: Based n AMCP guidelines fr frmulary submissin, versin 2.1. MT Pharma America, Inc.; received June 14, 2017 Current treatment guidelines frm the American Academy f Neurlgy d nt yet address Radicava. The nly ther drug apprved t treat ALS is riluzle which these guidelines recmmend fr all patients with ALS unless the risk f fatigue utweighs the mdest survival benefits with this drug. The guidelines als address lithium carbnate as a ptential treatment ptin, but state that the data are insufficient t supprt r discurage its use fr ALS. Recmmendatins fr symptmatic management include btulinum txin type B fr patients with refractry sialrrhea and Nuedexta fr pseudbulbar affect. The Eurpean Federatin f Neurlgical Scieties als did nt address Radicava in their mst current guidelines (2012), but recmmend riluzle t all patients as early as pssible after diagnsis with the caveat that riluzle shuld nt be used fr patients with prgressive muscular atrphy, primary lateral sclersis, r hereditary spastic paraplegia. FDA r Other Gvernmental Regulatry Apprval U.S. Fd and Drug Administratin (FDA) Radicava is FDA apprved fr the treatment f ALS Ratinale/Surce This medical plicy was develped thrugh cnsideratin f peer-reviewed medical literature generally recgnized by the relevant medical cmmunity, U.S. FDA apprval status, natinally accepted standards f medical practice and accepted standards f medical practice in this cmmunity, Blue Crss and Blue Shield Assciatin technlgy assessment prgram (TEC) and ther nn-affiliated technlgy evaluatin centers, reference t federal regulatins, ther plan medical plicies, and accredited natinal guidelines. Page 3 f 7

4 Radicava was apprved based n a phase III, randmized, duble-blind, placeb-cntrlled study cnducted in Japan. In this study, 137 patients were randmized t receive Radicava r placeb fr 24 weeks. This was the secnd phase 3 study, and was cnducted because the first study failed t demnstrate efficacy, but shwed a greater magnitude f effect in a subppulatin identified by a pst hc analysis. T be included in this study, patients had t meet extensive inclusin criteria based n the pst hc analysis f the previus study. These criteria included: Aged years ALS f grade 1 r 2 in the Japan ALS Severity Classificatin (independent living status) Definite r prbable diagnsis f ALS based n the El Escrial criteria Scre f >2 n each item f the ALS Functinal Rating Scale (ALSFRS-R) FVC >80% Disease duratin f <2 years Decrease f 1-4 pints in the ALSFRS-R scre during a 12-week bservatin perid prir t study randmizatin N histry f spinal surgery after nset f ALS CrCl >50 ml/min The primary endpint was the change frm baseline t week 24 in the ALSFRS-R scre. Secndary endpints included the change in FVC, Mdified Nrris Scale scres, ALS Assessment Questinnaire scre (a measure f quality f life), ALS severity classificatin, and grip and pinch strength. There was fund t be significantly less decline in the ALSFRS-R scres in the Radicava grup cmpared with placeb with a change f (SE 0.64) vs -7.5 (SE 0.66). The results f the Mdified Nrris Scale favred Radicava with a least squares mean difference f 4.89, and the ALS Assessment Questinnaire scres demnstrated a significantly lwer deteriratin in quality f life in the Radicava grup. N difference was fund in FVC, grip and pinch strength, r ALS severity classificatin between the Radicava and placeb grups. References 1. Edaravne (Radicava TM ) [Package Insert]. MT Pharma America. Jersey City, NJ. Updated May Miller RG, Jacksn CE, Kasarskis EJ, et al. Practice parameter update: the care f the patient with amytrphic lateral sclersis: drug, nutritinal, and respiratry therapies (an evidence-based review). Neurlgy. 2009;73: Andersen PM, Abrahams S, Brasi GD, et al. EFNS guidelines n the clinical management f amytrphic lateral sclersis (MALS) revised reprt f an EFNS task frce. Eur J Neurl. 2012;19(3): Abe K, Aki M, Tsuji S, et al. n behalf f the edaravne (MCI-186) ALS 19 study grup. Safety and efficacy f edaravne in well defined patients with amytrphic lateral sclersis: a randmized, duble-blind, placeb-cntrlled trial. Lancet Neurl. 2017;16: Hardiman O, Van den Berg LH. Edaravne: a new treatment fr ALS n the hrizn? Lancet Neurl. 2017;16: Prudft M, Jnes A, Talbt K, Al-Chalabi A, Turner MR. The ALSFRS as an utcme measure in therapeutic trials and its relatinship t symptm nset Aug 17;17(5-6): Plicy Histry 12/07/2017 Medical Plicy Cmmittee review Page 4 f 7

5 12/20/2017 Medical Plicy Implementatin Cmmittee apprval. New Plicy Next Scheduled Review Date: 12/2018 Cding The five character cdes included in the Blue Crss Blue Shield f Luisiana Medical Plicy Cverage Guidelines are btained frm Current Prcedural Terminlgy (CPT ), cpyright 2016 by the American Medical Assciatin (AMA). CPT is develped by the AMA as a listing f descriptive terms and five character identifying cdes and mdifiers fr reprting medical services and prcedures perfrmed by physician. The respnsibility fr the cntent f Blue Crss Blue Shield f Luisiana Medical Plicy Cverage Guidelines is with Blue Crss and Blue Shield f Luisiana and n endrsement by the AMA is intended r shuld be implied. The AMA disclaims respnsibility fr any cnsequences r liability attributable r related t any use, nnuse r interpretatin f infrmatin cntained in Blue Crss Blue Shield f Luisiana Medical Plicy Cverage Guidelines. Fee schedules, relative value units, cnversin factrs and/r related cmpnents are nt assigned by the AMA, are nt part f CPT, and the AMA is nt recmmending their use. The AMA des nt directly r indirectly practice medicine r dispense medical services. The AMA assumes n liability fr data cntained r nt cntained herein. Any use f CPT utside f Blue Crss Blue Shield f Luisiana Medical Plicy Cverage Guidelines shuld refer t the mst current Current Prcedural Terminlgy which cntains the cmplete and mst current listing f CPT cdes and descriptive terms. Applicable FARS/DFARS apply. CPT is a registered trademark f the American Medical Assciatin. Cdes used t identify services assciated with this plicy may include (but may nt be limited t) the fllwing: Cde Type Cde CPT HCPCS N cdes C9493 ICD-10 Diagnsis G12.21 *Investigatinal A medical treatment, prcedure, drug, device, r bilgical prduct is Investigatinal if the effectiveness has nt been clearly tested and it has nt been incrprated int standard medical practice. Any determinatin we make that a medical treatment, prcedure, drug, device, r bilgical prduct is Investigatinal will be based n a cnsideratin f the fllwing: A. Whether the medical treatment, prcedure, drug, device, r bilgical prduct can be lawfully marketed withut apprval f the U.S. FDA and whether such apprval has been granted at the time the medical treatment, prcedure, drug, device, r bilgical prduct is sught t be furnished; r B. Whether the medical treatment, prcedure, drug, device, r bilgical prduct requires further studies r clinical trials t determine its maximum tlerated dse, txicity, safety, effectiveness, r effectiveness as cmpared with the standard means f treatment r diagnsis, must imprve health utcmes, accrding t the cnsensus f pinin amng experts as shwn by reliable evidence, including: 1. Cnsultatin with the Blue Crss and Blue Shield Assciatin technlgy assessment prgram (TEC) r ther nnaffiliated technlgy evaluatin center(s); 2. Credible scientific evidence published in peer-reviewed medical literature generally recgnized by the relevant medical cmmunity; r 3. Reference t federal regulatins. **Medically Necessary (r Medical Necessity ) - Health care services, treatment, prcedures, equipment, drugs, devices, items r supplies that a Prvider, exercising prudent clinical judgment, wuld prvide t a patient fr the purpse f preventing, evaluating, diagnsing r treating an illness, injury, disease r its symptms, and that are: Page 5 f 7

6 A. In accrdance with natinally accepted standards f medical practice; B. Clinically apprpriate, in terms f type, frequency, extent, level f care, site and duratin, and cnsidered effective fr the patient's illness, injury r disease; and C. Nt primarily fr the persnal cmfrt r cnvenience f the patient, physician r ther health care prvider, and nt mre cstly than an alternative service r sequence f services at least as likely t prduce equivalent therapeutic r diagnstic results as t the diagnsis r treatment f that patient's illness, injury r disease. Fr these purpses, natinally accepted standards f medical practice means standards that are based n credible scientific evidence published in peer-reviewed medical literature generally recgnized by the relevant medical cmmunity, Physician Specialty Sciety recmmendatins and the views f Physicians practicing in relevant clinical areas and any ther relevant factrs. Indicated trademarks are the registered trademarks f their respective wners. NOTICE: Medical Plicies are scientific based pinins, prvided slely fr cverage and infrmatinal purpses. Medical Plicies shuld nt be cnstrued t suggest that the Cmpany recmmends, advcates, requires, encurages, r discurages any particular treatment, prcedure, r service, r any particular curse f treatment, prcedure, r service. ALSFRS-R Scale Scre Descriptin 1. Speech 4 Nrmal speech prcesses 3 Detectable speech disturbance 2 Intelligible with repeating 1 Speech cmbined with nn-vcal cmmunicatin 2. Salivatin 4 Nrmal 3 Slight, but definite excess f saliva in muth; may have nighttime drling 2 Mderately excessive saliva; may have minimal drling (during the day) 1 Marked excess f saliva with sme drling 0 Marked drling; requires cnstant tissue r handkerchief 3. Swallwing 4 Nrmal eating habits 3 Early eating prblems ccasinal chking 2 Dietary cnsistency changes 1 Needs supplement tube feeding 0 NPO (exclusively parenteral r enteral feeding) 4. Handwriting 4 Nrmal 3 Slw r slppy: all wrds are legible 2 Nt all wrds are legible 1 Able t grip pen, but unable t write 0 Unable t grip pen 5a. Cutting Fd and Handling Utensils (skip if patient has gastrstmy) 4 Nrmal 3 Smewhat slw and clumsy, but n help needed 2 Can cut mst fds (>50%), althugh slw and clumsy; sme help needed 1 Fd must be cut by smene, but can still feed slwly 0 Needs t be fed 5b. Cutting Fd and Handling Utensils (WITH gastrstmy nly) 4 Nrmal 3 Clumsy, but able t perfrm all manipulatins independently Page 6 f 7

7 2 Sme help needed with clsures and fasteners 1 Prvides minimal assistance t caregiver 0 Unable t perfrm any aspect f task 6. Dressing and Hygiene 4 Nrmal functin 3 Independent and cmplete self-care with effrt r decreased efficiency 2 Intermittent assistance r substitute methds 1 Needs attendant fr self-care 0 Ttal dependence 7. Turning in bed and adjusting bed clthes 4 Nrmal functin 3 Smewhat slw and clumsy, but n help needed 2 Can turn alne, r adjust sheets, but with great difficulty 1 Can initiate, but nt turn r adjust sheets alne 0 Helpless 8. Walking 4 Nrmal 3 Early ambulatin difficulties 2 Walks with assistance 1 Nn-ambulatry functinal mvement 0 N purpseful leg mvement 9. Climbing stairs 4 Nrmal 3 Slw 2 Mild unsteadiness r fatigue 1 Needs assistance 0 Cannt d 10. Dyspnea 4 Nne 3 Occurs when walking 2 Occurs with ne r mre f the fllwing: eating, bathing, dressing (ADL) 1 Occurs at rest: difficulty breathing when either sitting r lying 0 Significant difficulty: cnsidering using mechanical respiratry supprt 11. Orthpnea 4 Nne 3 Sme difficulty sleeping at night due t shrtness f breath, des nt rutinely use mre than tw pillws 2 Needs extra pillws in rder t sleep (mre than tw) 1 Can nly sleep sitting up 0 Unable t sleep withut mechanical assistance 12. Respiratry Insufficiency 4 Nne 3 Intermittent use f BiPAP 2 Cntinuus use f BiPAP during the night 1 Cntinuus use f BiPAP during day and night 0 Invasive mechanical ventilatin by intubatin r trachestmy Page 7 f 7