Clinical Policy Title: Botulinum toxin products

Transcription

1 Clinical Policy Title: Botulinum toxin products Clinical Policy Number: Effective Date: September 1, 2013 Initial Review Date: May 13, 2013 Most Recent Review Date: April 10, 2018 Next Review Date: April 2019 Related policies: Policy contains: Botulinum toxins A and B. Involuntary muscle activity ocular, GI, urinary, and spasticity. Hyperhidrosis. Chronic migraine. Dysphonia. CP# CP# CP# CP# CP# Hyperhidrosis treatment Spine pain trigger point injection Invasive treatment for cervicogenic headache and occipital neuralgia Temporomandibular joint disorder Deep brain stimulation ABOUT THIS POLICY: Select Health of South Carolina has developed clinical policies to assist with making coverage determinations. Select Health of South Carolina s clinical policies are based on guidelines from established industry sources, such as the Centers for Medicare & Medicaid Services (CMS), state regulatory agencies, the American Medical Association (AMA), medical specialty professional societies, and peerreviewed professional literature. These clinical policies along with other sources, such as plan benefits and state and federal laws and regulatory requirements, including any state- or plan-specific definition of medically necessary, and the specific facts of the particular situation are considered by Select Health of South Carolina when making coverage determinations. In the event of conflict between this clinical policy and plan benefits and/or state or federal laws and/or regulatory requirements, the plan benefits and/or state and federal laws and/or regulatory requirements shall control. Select Health of South Carolina s clinical policies are for informational purposes only and not intended as medical advice or to direct treatment. Physicians and other health care providers are solely responsible for the treatment decisions for their patients. Select Health of South Carolina s clinical policies are reflective of evidence-based medicine at the time of review. As medical science evolves, Select Health of South Carolina will update its clinical policies as necessary. Select Health of South Carolina s clinical policies are not guarantees of payment. Coverage policy Select Health of South Carolina considers the use of botulinum neurotoxin to be clinically proven and, therefore, medically necessary when U.S. Food and Drug Administration (FDA)-approved serotypes are used: OnabotulinumtoxinA (Botox, Allergan Inc., Irvine, California). IncobotulinumtoxinA (Xeomin (Merz Pharmaceuticals, Greensboro, North Carolina). AbobotulinumtoxinA (Dysport, Ipsen Biopharmaceuticals Inc., Basking Ridge, New Jersey)*. RimabotulinumtoxinB (Myobloc, Solstice Neurosciences Inc., Louisville, Kentucky). *PerformRx preferred agent. See Appendix for prior authorization criteria. Select Health of South Carolina has determined that separate medically necessary indications for the 1

2 botulinum toxin products will be combined into a single list. This is not meant to imply that botulinum toxin products are interchangeable. It is the responsibility of providers, however, to use each drug in accordance with FDA instructions, general medical compendia, or standard-of-care guidelines, taking into consideration the following: Dosing patterns are specific to the preparation of neurotoxin and member s condition. A member who is not responsive or who ceases to respond to one serotype may respond to another. After documented failure of conservative therapy, unless specified below. List of medically necessary indications: Cervical dystonia in members age 18 years (FDA, 2017a; FDA, 2017b; Simpson, 2016; FDA, 2009; FDA, 2015): - Includes post-radiation facial myokymia or trismus (Cohen, 2016). - Members who are botulinum toxin-naïve or previously treated with another botulinum toxin product. Blepharospasm (FDA, 2017a; Rowe, 2017; Simpson, 2016 based on Truong, 2007; FDA, 2015): - Associated with dystonia, including benign essential blepharospasm or VII nerve disorders. - Intermittent or sustained closure caused by involuntary contractions of the orbicularis oris. Focal limb spasticity in members ages 2 years (FDA, 2017a; FDA, 2017b; Simpson, 2016; FDA, 2015; Delgado, 2010). Strabismus in members age 12 years (FDA, 2017a; Rowe, 2017): - For vertical muscles. - For horizontal strabismus of up to 50 prism diopters. - For persistent VI nerve palsy of one month or longer duration. Focal primary axillary hyperhidrosis in members age 18 years (FDA, 2017a; Naumann, 2013): - Topical aluminum chloride ineffective. Prevention of episodic headaches in members age 18 years with chronic migraine (FDA, 2017a; Simpson, 2016): - At least four hours duration and 15 days per month. - Failure of four drugs proven for migraine prophylaxis, with a 60-day trial each: antiepileptics (topiramate, sodium valproate); beta-blockers (metoprolol, propranolol, timolol); and tricyclic antidepressant (amitriptyline). Overactive bladder in members age 18 years (FDA, 2017a; American Urological Association, 2014): - Symptoms of urge incontinence, frequency, and urgency. - Inadequate response to, or unable to tolerate, conservative options, anticholinergics, or vaginal estrogen. 2

3 Urge incontinence due to detrusor overactivity associated with a neurologic condition (e.g., spinal cord injury or multiple sclerosis) (FDA, 2017a; Duthie, 2011): - Anti-muscarinic drugs were ineffective or poorly tolerated. Sialorrhea (National Institute for Health and Clinical Excellence, 2017; Naumann, 2013; Miller, 2009 [reaffirmed 2013]; Vashishta, 2013): - Refractory to pharmacotherapy. Spasmodic dysphonia (Schwartz, 2009): - As a primary or secondary treatment option. Hemifacial spasm (Awan, 2017): - Refractory to oral pharmacotherapy (e.g., carbamazepine, baclofen, and benzodiazepine). Myofascial pain syndrome (Soares, 2014; Zhou, 2014): - Head and neck. - After failed first-line therapies (e.g., local anesthetics injection, corticosteroid injections to trigger points, massage, physical therapy, and lifestyle changes to reduce stress). Oromandibular dystonia (Comella, 2018): - For jaw-closing type. - As primary or secondary treatment. Achalasia and cardiospasm (Vaezi, 2013; Stefanidis, 2012): - Failure of conventional treatments: upright after eating, achalasia wedge positioning, and proton pump inhibitors. - High risk of complications from pneumatic dilation or surgical myotomy. - Previous dilation or myotomy failure or epiphrenic diverticulum or hiatal hernia. Upper extremity tremor (Zesiewicz, 2011): - Medically refractory. - Successful first botulinum toxin trial. Hirschsprung s disease (Friedmacher, 2012; Frykman, 2012): - Internal sphincter achalasia post-endorectal pull-through. Chronic anal fissure (Ebinger, 2017; Stewart, 2017): - Failure to respond to conservative therapy: topical nitroglycerin or diltiazem. Limitations: Approved indications for botulinum toxins type A and type B differ, and off-label uses are evolving. A patient who is not responsive or who ceases to respond to one serotype may respond to another. Those indications not listed above may be considered medically necessary on a case-by-case basis if conservative options have failed and the member has a reasonable chance of responding based on condition, sufficient evidence of safety and effectiveness, or standard of care. Each preparation has distinct pharmacological and clinical profiles specified on the product insert. 3

4 Dosing patterns are also specific to the preparation of neurotoxin and are very different between different serotypes. Failure to recognize the unique characteristics of each formulation of botulinum toxin can lead to undesired patient outcomes. It is expected that physicians will be familiar with and experienced in the use of these agents, and use evidence-based medicine to select the appropriate drug and dose regimen for each patient condition. Botulinum toxin injections for cosmetic purposes are not medically necessary under any circumstances. Where FDA labeling requirements impose limitations for use, Select Health of South Carolina coverage follows FDA guidelines. When medical necessity criteria for botulinum toxin therapy are met, approval consists of a maximum of four treatments in one year (one injection every three months or 90 days). If initial criteria are met and a clinical benefit is obtained but its duration is less than 90 days, then up to six treatments per year (one every two months or 60 days) may be considered on a case-by-case basis. The off-label use of botulinum toxin in children less than age 2 years is not medically necessary, as evidence supporting its off-label use in this population is insufficient. Prior authorization requirements: See Appendix for additional information. Alternative covered services: Accepted standard of care for each indication, including, but are not limited to comparator treatments from systematic reviews detailed on pages 8-12, where comparisons other than placebo were used and reported. Background Most widely known for its role in food poisoning (botulism), botulinum toxin is a culture-derived injectable toxin from the Clostridium botulinum bacterium that blocks acetylcholine release at the neuromuscular junction. In the late 1970s, interest in botulinum toxin emerged as a less invasive treatment for movement disorders and other conditions associated with increased cholinergic activity. Botulinum toxin is injected directly into affected muscles to provide transient relief of symptoms. The paralytic effect is usually temporary, lasting approximately two to six months; recovery occurs when the affected axonal terminals sprout and re-innervate the muscle. Patients may require repeated injections to maintain treatment effects. Botulinum toxin is classified into eight distinct serologically related neurotoxins. The two main commercial types available for medical use are botulinum toxin types A and B. Table 1 lists the four FDA- 4

5 approved botulinum toxin products for non-cosmetic use. Each product has a different dose-to-potency ratio, and the units of one product cannot be converted into units of another product (i.e., not interchangeable) (FDA, 2018). Table 1. FDA-approved botulinum toxin products Trade name New drug name Old drug name Dosage forms Botox OnabotulinumtoxinA Boxulinum toxin type A 100 U, 200 U vials Dysport AbobotulinumtoxinA Boxulinum toxin type A 300 U, 500 U vials Xeomin IncobotulinumtoxinA Boxulinum toxin type A 50 U, 100 U vials Myobloc RimabotulinumtoxinB Boxulinum toxin type B 5,000 U/mL (in 0.5 ml, 1 ml, 2 ml single-use vials) Searches Select Health of South Carolina searched PubMed and the databases of: UK National Health Services Centre for Reviews and Dissemination. Agency for Healthcare Research and Quality s National Guideline Clearinghouse and other evidence-based practice centers. The Centers for Medicare & Medicaid Services (CMS). We conducted searches on February 17, Search terms were: botulinum toxins (MeSH) and freetext terms botox, myobloc, xeomin, dysport, onabotulinumtoxina, abobotulinumtoxina, incobotulinumtoxina, and rimabotulinumtoxinb. We included: Systematic reviews, which pool results from multiple studies to achieve larger sample sizes and greater precision of effect estimation than in smaller primary studies. Systematic reviews use predetermined transparent methods to minimize bias, effectively treating the review as a scientific endeavor, and are thus rated highest in evidence-grading hierarchies. Guidelines based on systematic reviews. Economic analyses, such as cost-effectiveness, and benefit or utility studies (but not simple cost studies), reporting both costs and outcomes sometimes referred to as efficiency studies which also rank near the top of evidence hierarchies. Findings While most, but not all, reviews identified at least some randomized controlled trials, the trials were small, underpowered, or otherwise subject to bias. Some trials found unacceptably high adverse event 5

6 rates, including those leading to early termination. Important unresolved research questions include optimal doses, treatment intervals, criteria for retreatment, and direct comparisons with other treatments. Ethical and cost issues relate to the need for lifelong repeated injections versus less-invasive or potentially harmful alternatives. Policy updates: In 2015, we identified two new systematic reviews for this policy. One found strong evidence of safety and efficacy from 12 randomized controlled trials supporting the use of abobotulinumtoxina treatment in adult patients with upper limb spasticity caused by stroke (Dashtipour, 2015). The other review found some evidence of effectiveness from eight randomized controlled trials for botulinum toxin type A injections of trigger points for myofascial pain; however, further clinical trials should account for minimizing placebo effect, repeated dosing, adequate coverage of trigger points, and using ultrasound confirmation and guidance to provide conclusive evidence (Zhou, 2014). In 2016, we identified three new systematic reviews for this policy (Chen, 2015; Cui, 2015; Wu, 2015). Two reviews found insufficient evidence to determine the therapeutic benefits of botulinum toxin for temporomandibular joint disorders or shoulder pain (Chen, 2015; Wu, 2015). Cui (2015) found onabotulinumtoxina a safe and effective treatment for idiopathic overactive bladder compared to placebo-controlled studies. These findings would not change earlier conclusions. However, the FDA has approved a new indication for incobotulinumtoxina for treatment of upper limb spasticity in adults. Treatment is limited to a total dose of up to 400 U no more frequently than every 12 weeks (Appendix B). In 2017, we added two new systematic reviews (Alviar, 2016; Morra, 2016), a Cochrane review update (Duarte, 2016), and an updated evidence-based guideline from the American Academy of Neurology (Simpson, 2016). Alviar et al. identified only one, small randomized controlled trial of botulinum toxin type A for treatment of phantom limb pain. For treating trigeminal neuralgia, results from four small randomized controlled trials found a positive effect for botulinum toxin type A compared with placebo (Morra, 2016). Results from these reviews remain inconclusive due to the paucity of evidence for both indications. The most recent Cochrane review update found that a single treatment session of either onabotulinumtoxina (100 U to 250 U) or rimabotulinumtoxinb (5,000 U to 10,000 U) is equally effective and safe in adults with certain types of cervical dystonia, which is consistent with the previous findings of this policy (Duarte, 2016). The American Academy of Neurology s updated recommendations for blepharospasm, cervical dystonia, adult spasticity, and headache are also consistent with this policy (Simpson, 2016). Therefore, no policy changes are warranted at this time. In 2018, we consolidated the indications in Appendix B into the coverage statements and added: seven new systematic reviews and meta-analyses (Comella, 2018; Awan, 2017; Drake, 2017; Ebinger, 2017; Soares, 2014; Vashishta, 2013; Friedmacher, 2012; Frykman, 2012); one systematic review update (Rose, 6

7 2017); and eight evidence-based guidelines (National Institute for Health and Clinical Excellence, 2017; Stewart, 2017; American Urological Association, 2014; Naumann, 2013; Vaezi, 2013; Miller, 2009; Schwartz, 2009; Zesiewicz, 2011). All serotypes appear safe and effective when administered by experienced practitioners in accordance with evidence-based practice. Patients may respond differently or, conversely, equally well to various serotypes based on their underlying condition and may require trials of more than one product to achieve optimal response. New indications are emerging but currently lack strong evidence of long-term outcomes or comparative effectiveness to determine the superiority of one product over another. There is sufficient evidence of safety and effectiveness from research and clinical experience to support additional serotype-specific indications when conservative treatment fails or is not tolerated: for onabotulinumtoxina, oromandibular dystonia and spasmodic dysphonia; for abobotulinumtoxina, blepharospasm and sialorrhea; and for rimabotulinumtoxinb, spasmodic dysphonia and sialorrhea. A growing area of interest is intrapyloric injection of botulinum toxin type A for treatment of gastroparesis. One systematic review (Bai, 2010) and two evidence-based guidelines (Camilleri, 2013; Saliakellis, 2013) found insufficient evidence to recommend botulinum toxin type A for gastroparesis in adults or children. While observational studies generally found improvement in symptoms and gastric emptying, the results of the two available randomized placebo-controlled trials did not confirm these findings. At this time botulinum toxin type A cannot be recommended as medically necessary. Summary of clinical evidence: Citation Alviar (2016) Cochrane review Phantom limb pain Morra (2016) Content, Methods, Recommendations Systematic review of 14 studies (269 total participants), including one study of botulinum toxin type A. Overall quality: low or unclear risk of bias with small sample size. Inconclusive evidence for short- and long-term effectiveness of botulinum toxin type A, opioids, N-methyl D-aspartate receptor antagonists, anticonvulsants, antidepressants, calcitonins, and local anesthetics for clinically relevant outcomes. Botulinum toxin-a for trigeminal neuralgia Systematic review and meta-analysis of four randomized controlled trials (178 total patients). Compared to placebo, botulinum toxin type A was associated with a higher proportion of responders (risk ratio = 2.87, 95% confidence interval [CI] 1.76 to 4.69, p < and lower daily frequency of paroxysms (mean difference , 95% CI to , p < ); no significant heterogeneity with either measure was found. Results suggest that botulinum toxin type A may be effective and safe for patients with trigeminal neuralgia, but larger and well-designed randomized controlled trials are needed. 7

8 Citation Simpson (2016) for the American Academy of Neurology Practice guideline update summary: botulinum toxin for blepharospasm, cervical dystonia, adult spasticity, and headache Chen (2015) Botulinum toxin for temporomandibular joint disorders Wu (2015) Botulinum toxin for shoulder pain Soares (2014) Cochrane review Botulinum toxin for myofascial pain syndromes in adults, excluding head and neck Zhou (2014) Content, Methods, Recommendations Blepharospasm: onabotulinumtoxina and incobotulinumtoxina probably effective and should be considered (Level B); abobotulinumtoxina possibly effective and may be considered (Level C). Cervical dystonia: abobotulinumtoxina and rimabotulinumtoxinb established as effective and should be offered (Level A); onabotulinumtoxina and incobotulinumtoxina probably effective and should be considered (Level B). Adult spasticity: abobotulinumtoxina, incobotulinumtoxina, and onabotulinumtoxina established as effective and should be offered (Level A): - For upper limb spasticity, rimabotulinumtoxinb probably effective and should be considered (Level B). - For lower-limb spasticity, abobotulinumtoxina and onabotulinumtoxina established as effective and should be offered (Level A). Headache: onabotulinumtoxina established as effective and should be offered to increase headache-free days (Level A); probably effective and should be considered to improve health-related quality of life (Level B) in chronic migraine; established as ineffective and should not be offered for episodic migraine (Level A), and probably ineffective for chronic tension-type headaches (Level B). Systematic review of five relevant study trials (117 patients). Overall quality: low-to-moderate. Heterogeneous methods. Conflicting results. Unclear therapeutic benefits of botulinum toxin on temporomandibular joint disorders versus placebo or any other intervention. Systematic review of nine randomized controlled trials comparing botulinum toxin to steroid injection or placebo. Overall quality: unclear. Significant heterogeneity in designs limits conclusions. Compared to control, botulinum toxin injections improved pain scores and range of motion in adult patients with shoulder pain. Higher quality studies needed. Systematic review of four randomized controlled trials 233 total patients) comparing onabotulinumtoxina to placebo. Overall quality: low with significant heterogeneity, short follow-up. Inconclusive evidence of safety and effectiveness. More high-quality randomized controlled trials are needed. Botulinum toxin type A for myofascial pain syndrome Systematic review of eight double-blinded randomized controlled trials. Overall quality: high. Botulinum toxin type A is efficacious for myofascial pain syndrome. Further clinical trials need to address minimizing placebo effect, repeated dosing, 8

9 Citation Fedorowicz (2013) Cochrane review Masseter hypertrophy Naumann (2013) for the American Academy of Neurology Evidence-based review and assessment of botulinum neurotoxin for the treatment of secretory disorders Vaezi (2013) for the American College of Gastroenterology ACG clinical guideline: diagnosis and management of achalasia Vashishta (2013) Botulinum toxin for the treatment of sialorrhea Friedmacher (2012) Content, Methods, Recommendations adequate coverage of trigger points, and using ultrasound confirmation and guidance. Systematic review of randomized controlled trials and controlled clinical trials of botulinum toxin versus placebo published through April No eligible studies identified. Systematic review and recommendations. Axillary hyperhidrosis (923 total patients): Level A recommendation for botulinum toxin type A products; Level B recommendation for abobotulinumtoxina and onabotulinumtoxina; Level U recommendation (insufficient data) for incobotulinumtoxina and rimabotulinumtoxinb. Palmar hyperhidrosis (five trials in 82 patients): Level B recommendation for botulinum toxin type A products; Level C recommendation for botulinum toxin type B products; individual formulations received a Level U rating due to insufficient data. Gustatory sweating: Level U recommendation for all botulinum toxin formulations. Sialorrhea (eight trials in 222 adults and children): Level B recommendation for abobotulinumtoxina, onabotulinumtoxina, and rimabotulinumtoxinb; Level U recommendation for incobotulinumtoxina. Allergic rhinitis (two studies in 73 patients): Level B recommendation for onabotulinumtoxina; Level U recommendation for abobotulinumtoxina, incobotulinumtoxina, or rimabotulinumtoxinb. Botulinum toxin is restricted to specific circumstances where pneumatic dilatation and surgical myotomy are not considered appropriate because of inherent patient-related risks. Botulinum toxin can be considered as an adjunct treatment in patients with residual spastic contractions above the myotomy site or lower esophageal sphincter; however, outcome-related data are lacking. Systematic review and meta-analysis of eight randomized controlled trials enrolling 181 total patients (83 placebo; 98 active) with sialorrhea of varying etiologies. Compared to placebo, botulinum toxin type A and type B significantly decreased the severity of drooling in both adult and pediatric patients. Both botulinum toxin types A and types B produced similar effects. Doses greater than 50 U produced much stronger effects. Future studies are needed to evaluate the optimal technique and dosages. Comparison of posterior internal anal sphincter myectomy and intrasphincteric Meta-analysis of 16 prospective and retrospective studies (395 total patients) with internal anal sphincter achalasia (58% myectomy, 42% botulinum toxin injection). Regular bowel movements were significantly more frequent after internal anal sphincter myectomy. 9

10 Citation botulinum toxin injection for treatment of internal anal sphincter achalasia Frykman (2012) Hirschsprung-associated enterocolitis: prevention and therapy Stefanidis (2012) for the Society of American Gastrointestinal and Endoscopic Surgeons Guidelines for the surgical treatment of esophageal achalasia Zesiewicz (2011) for the American Academy of Neurology Evidence-based guideline update: treatment of essential tremor Delgado (2010) for the American Academy of Neurology Practice parameter: pharmacologic treatment of spasticity in children and adolescents with cerebral palsy Bai (2010) Content, Methods, Recommendations No significant difference in continued use of laxatives or rectal enemas or overall complication rates between procedures. Posterior internal anal sphincter myectomy appears to be a more effective treatment option compared to intrasphincteric botulinum toxin injection, which was associated with higher rates of transient fecal incontinence, non-response, and subsequent surgical procedures. Intrasphincteric botulinum toxin injection appears to be safe, can be used for multiple injections if symptoms recur, reduces the number hospitalizations for enterocolitis, and can improve symptoms in some patients. It is difficult to predict which patients will respond. Recommendation: botulinum toxin injection can be administered safely, but its effectiveness is limited, especially in the long term. It should be reserved for patients who are poor candidates for other more effective treatment options, such as surgery or dilation. The effect of botulinum toxin type A on limb tremor in essential tremor is modest and is associated with dose-dependent hand weakness. Botulinum toxin type A may reduce head tremor and voice tremor, but data are limited. When used to treat voice tremor, botulinum toxin type A may cause breathiness, hoarseness, and swallowing difficulties. Botulinum toxin type A injections for limb, head, and voice tremor associated with essential tremor may be considered in medically refractory cases. For localized/segmental spasticity, botulinum toxin type A is established as an effective treatment to reduce spasticity in the upper and lower extremities (Level A). There is conflicting evidence regarding functional improvement. Botulinum toxin type A appears safe in children with cerebral palsy; however, the FDA investigation is ongoing. Insufficient evidence on the use of phenol, alcohol, or botulinum toxin type B injections. A systematic review on intrapyloric botulinum toxin injection for gastroparesis Systematic review included 13 non-randomized and two randomized controlled trials. Almost all the non-randomized trials reported significant improvement in subjective symptoms and objective gastric emptying study after botulinum toxin injection, but the two randomized controlled trials did not confirm the efficacy of botulinum toxin injection. None of the individual trials showed statistically significant subjective and objective improvement in the active patients when compared with patients receiving placebo. 10

11 Citation Schwartz (2009) for the American Academy of Otolaryngology Head and Neck Surgery Foundation Clinical practice guideline: hoarseness (dysphonia) Content, Methods, Recommendations The clinician should prescribe, or refer the patient to a clinician who can prescribe, botulinum toxin (onabotulinumtoxina) injections for the treatment of hoarseness caused by adductor spasmodic dysphonia (based on randomized controlled trials with minor limitations and preponderance of benefit over harm). Surgical treatment for laryngeal dystonia, or adductor spasmodic dysphonia, is infrequently performed due to the widespread acceptance of botulinum toxin as the firstline treatment for this disorder. References Professional society guidelines/other: Camilleri M, Parkman HP, Shafi MA, Abell TL, Gerson L. Clinical guideline: management of gastroparesis. Am J Gastroenterol. 2013; 108(1): 18 37; quiz 38. DOI: /ajg Delgado MR, Hirtz D, Aisen M, et al. Practice parameter: pharmacologic treatment of spasticity in children and adolescents with cerebral palsy (an evidence-based review): report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology. 2010; 74(4): Reaffirmed DOI: /WNL.0b013e3181cbcd2f. Diagnosis and Treatment of Non-Neurogenic Overactive Bladder (OAB) in Adults: AUA/SUFU Guideline. Published 2012; Amended American Urological Association website. Accessed February 18, Miller RG, Jackson CE, Kasarskis EJ, et al. Practice parameter update: the care of the patient with amyotrophic lateral sclerosis: multidisciplinary care, symptom management, and cognitive/behavioral impairment (an evidence-based review): report of the Quality Standards Subcommittee of the American Academy of Neurology. Neurology. 2009; 73(15): DOI: /WNL.0b013e3181bc01a4. National Institute for Health and Clinical Excellence (NICE). Parkinson s disease in adults. NICE guideline [NG71]. Published date: July NICE website. Accessed February 18, Naumann M, Dressler D, Hallett M, et al. Evidence-based review and assessment of botulinum neurotoxin for the treatment of secretory disorders. Toxicon. 2013; 67: DOI: /j.toxicon

12 Saliakellis E, Fotoulaki M. Gastroparesis in children. Ann Gastroenterol. 2013; 26(3): Available at: Accessed April 10, Schwartz SR, Cohen SM, Dailey SH, et al. Clinical practice guideline: hoarseness (dysphonia). Otolaryngol Head Neck Surg. 2009; 141(3 Suppl 2): S1 S31. DOI: /j.otohns Simpson DM, Hallett M, Ashman EJ, et al. Practice guideline update summary: Botulinum neurotoxin for the treatment of blepharospasm, cervical dystonia, adult spasticity, and headache: Report of the Guideline Development Subcommittee of the American Academy of Neurology. Neurology. 2016; 86(19): DOI: /wnl Stefanidis D, Richardson W, Farrell TM, et al. SAGES guidelines for the surgical treatment of esophageal achalasia. Surg Endosc. 2012; 26(2): Stewart DB, Sr., Gaertner W, Glasgow S, et al. Clinical Practice Guideline for the Management of Anal Fissures. Dis Colon Rectum. 2017; 60(1): DOI: /dcr Vaezi MF, Pandolfino JE, Vela MF. ACG clinical guideline: diagnosis and management of achalasia. Am J Gastroenterol. 2013; 108(8): ; quiz DOI: /ajg Zesiewicz TA, Elble RJ, Louis ED, et al. Evidence-based guideline update: treatment of essential tremor: report of the Quality Standards subcommittee of the American Academy of Neurology. Neurology. 2011; 77(19): DOI: /WNL.0b013e318236f0fd. Peer-reviewed references: Alviar MJ, Hale T, Dungca M. Pharmacologic interventions for treating phantom limb pain. Cochrane Database Syst Rev. 2016; 10: Cd Awan KH. The therapeutic usage of botulinum toxin (Botox) in non-cosmetic head and neck conditions - An evidence based review. Saudi Pharm J. 2017; 25(1): DOI: /j.jsps Bai Y, Xu MJ, Yang X, et al. A systematic review on intrapyloric botulinum toxin injection for gastroparesis. Digestion. 2010; 81(1): DOI: / Chen YW, Chiu YW, Chen CY, Chuang SK. Botulinum toxin therapy for temporomandibular joint disorders: a systematic review of randomized controlled trials. Int J Oral Maxillofac Surg. 2015; 44(8): DOI: /j.ijom Comella CL. Systematic review of botulinum toxin treatment for oromandibular dystonia. Toxicon DOI: /j.toxicon

13 Cui Y, Zhou X, Zong H, Yan H, Zhang Y. The efficacy and safety of onabotulinumtoxina in treating idiopathic OAB: A systematic review and meta-analysis. Neurourol Urodyn. 2015; 34(5): DOI: /nau Drake MJ, Nitti VW, Ginsberg DA, et al. Comparative assessment of the efficacy of onabotulinumtoxina and oral therapies (anticholinergics and mirabegron) for overactive bladder: a systematic review and network meta-analysis. BJU Int. 2017; 120(5): /bju Drugs@FDA. FDA approved drug products. FDA website. Accessed February 21, Duarte GS, Castelao M, Rodrigues FB, et al. Botulinum toxin type A versus botulinum toxin type B for cervical dystonia. Cochrane Database Syst Rev. 2016; 10: Cd DOI: / CD pub3. Duthie JB, Vincent M, Herbison GP, Wilson DI, Wilson D. Botulinum toxin injections for adults with overactive bladder syndrome. Cochrane Database Syst Rev. 2011; (12): CD DOI: / CD pub3. Ebinger SM, Hardt J, Warschkow R, et al. Operative and medical treatment of chronic anal fissures-a review and network meta-analysis of randomized controlled trials. J Gastroenterol. 2017; 52(6): DOI: /s FDA. BOTOX (onabotulinumtoxina) for injection, for intramuscular, intradetrusor, or intradermal use. Product label. Last updated April FDA website. Accessed February 18, FDA. DYSPORT (abobotulinumtoxina) for injection, for intramuscular use. Product label. Last updated June FDA website. Accessed February 18, FDA. Myobloc (rimabotulinumtoxinb) Injection. Product label. July 31, FDA website. Accessed February 19, FDA. XEOMIN (incobotulinumtoxina) for injection, for intramuscular use. Product label. Last updated December FDA website. Accessed February 19,

14 Fedorowicz Z, van Zuuren EJ, Schoones J. Botulinum toxin for masseter hypertrophy. Cochrane Database Syst Rev. 2013; 9: CD DOI: / CD pub3. Friedmacher F, Puri P. Comparison of posterior internal anal sphincter myectomy and intrasphincteric botulinum toxin injection for treatment of internal anal sphincter achalasia: a meta-analysis. Pediatr Surg Int. 2012; 28(8): DOI: /s Frykman PK, Short SS. Hirschsprung-associated enterocolitis: prevention and therapy. Semin Pediatr Surg. 2012; 21(4): DOI: /j.sempedsurg Morra ME, Elgebaly A, Elmaraezy A, et al. Therapeutic efficacy and safety of Botulinum Toxin A Therapy in Trigeminal Neuralgia: a systematic review and meta-analysis of randomized controlled trials. J Headache Pain. 2016; 17(1): 63. DOI: /s Rowe FJ, Noonan CP. Botulinum toxin for the treatment of strabismus. Cochrane Database Syst Rev. 2017; 3: Cd DOI: / CD pub4. Soares A, Andriolo RB, Atallah AN, da Silva EM. Botulinum toxin for myofascial pain syndromes in adults. Cochrane Database Syst Rev. 2014; 7: CD DOI: / CD pub3. Vashishta R, Nguyen SA, White DR, Gillespie MB. Botulinum toxin for the treatment of sialorrhea: a meta-analysis. Otolaryngol Head Neck Surg. 2013; 148(2): DOI: / Wu T, Fu Y, Song HX, et al. Effectiveness of Botulinum Toxin for Shoulder Pain Treatment: A Systematic Review and Meta-Analysis. Arch Phys Med Rehabil. 2015; 96(12): DOI: /j.apmr Zhou JY, Wang D. An update on botulinum toxin A injections of trigger points for myofascial pain. Curr Pain Headache Rep. 2014; 18(1): 386. DOI: /s z. CMS National Coverage Determinations (NCDs): No NCDs identified as of the writing of this policy. Local Coverage Determinations (LCDs): A52381 Botulinum Toxins - Supplemental Instructions Article. CMS website. A52848 Botulinum Toxins - Supplemental Instructions Article. CMS website. 14

15 L34635 Botulinum Toxin Type A & Type B. CMS website. L35172 Botulinum Toxin Types A and B. CMS website. L35170 Botulinum Toxin Types A and B Policy. CMS website. L33949 Botulinum Toxins. CMS website. L33274 Botulinum Toxins. CMS website. L33646 Botulinum Toxins. CMS website. L33458 Chemodenervation. CMS website. Commonly submitted codes Below are the most commonly submitted codes for the service(s)/item(s) subject to this policy. This is not an exhaustive list of codes. Providers are expected to consult the appropriate coding manuals and bill accordingly. CPT Code Description Comment Laryngoscopy; indirect; with vocal cord injection Laryngoscopy; direct with injection in to the vocal cord(s) Laryngoscopy; direct with injection into vocal cord(s) with operating microscope Esophagoscopy, rigid, transoral; with directed submucosal injection(s), any substance Esophagoscopy, flexible, transoral, with directed submucosal injection(s), any substance EGD, flexible, transoral; with directed submucosal injection(s), any substance EGD, flexible, transoral, with ultrasound-guided transmural injection(s) of diagnostic or therapeutic substance Chemodenervation of internal anal sphincter Cystourethroscopy with injection(s) for chemodenervation of the bladder Chemodenervation of parotid and submandibular glands, bilateral Chemodenervation of muscles innervated by facial nerve, e.g., 15

16 CPT Code Description Comment blepharospasm Chemodenervation of muscles innervated by facial, trigeminal, cervical spinal and accessory nerves, e.g., chronic migraine Chemodenervation of neck muscles, excluding larynx muscles, e.g., cervical dystonia, spasmodic torticollis Chemodenervation of larynx, unilateral, percutaneous for spasmodic dystonia Chemodenervation of eccrine glands, bilateral ICD-10 Code Description Comment G24.3 Spasmodic torticollis G24.5 Blepharospasm G Chronic migraine without aura, not intractable, with status migrainosus G Chronic migraine without aura, not intractable, without status migrainosus G Chronic migraine without aura, intractable, with status migrainosus G Chronic migraine without aura, intractable, without status migrainosus G51.2 Melkersson's syndrome G51.3 Clonic hemifacial spasm G51.4 Facial myokymia G51.8 Other disorders of facial nerve G51.9 Disorder of facial nerve, unspecified K11.7 Disturbances of salivary secretion K22.0 Achalasia of cardia K60.0 Acute anal fissure K60.1 Chronic anal fissure K60.2 Anal fissure, unspecified L Primary focal hyperhidrosis, axilla L Primary focal hyperhidrosis, face L Primary focal hyperhidrosis, palms L Primary focal hyperhidrosis, soles L Primary focal hyperhidrosis, unspecified M M62.49 Contracture of muscle M Muscle spasm of calf M Other muscle spasm N32.81 Overactive bladder N Other specified urinary incontinence R68.2 Dry mouth, unspecified HCPCS Level II Code N/A Description Comment Appendix A 16

17 Select Health of South Carolina pharmacy prior authorization criteria for botulinum toxin products Field Name Prior Authorization Group Description Drugs Covered Uses Exclusion Criteria Required Medical Information Age Restrictions Prescriber Restrictions Coverage Duration Other Criteria Botulinum Toxins A&B Field Description OnabotulinumtoxinA (Botox ), IncobotulinumtoxinA (Xeomin ), AbobotulinumtoxinA (Dysport ), RimabotulinumtoxinB (Myobloc ), or any newly marketed agent Medically accepted indications are defined using the following sources: the Food and Drug Administration (FDA), Micromedex, American Hospital Formulary Service (AHFS), United States Pharmacopeia Drug Information for the Healthcare Professional (USP DI), the Drug Package Insert (PPI), or disease state specific standard of care guidelines. N/A N/A N/A None If all of the conditions are met, the request will be approved for 12 month duration. If the conditions are not met, the request will be sent to a Medical Director/clinical reviewer for medical necessity review. **The use of these medications for cosmetic purposes is NOT a covered benefit under the Medical Assistance program.** For Approval: Dysport is the preferred agent for all indications that it is FDA approved for. If a request is for any other botulinum toxin product, a medical reason (e.g., intolerance, hypersensitivity, contraindication) was provided why Dysport is not able to be used to treat the condition. The request is for a FDA-approved indication and dose or is supported by compendia or standard of care guidelines. Documentation was submitted, that the patient had an adequate trial (consistent with pharmacy claims) of standard first line therapy for their disease state and/or has a documented medical reason (intolerance, hypersensitivity, contraindication, etc.) for not taking first line therapy to treat their medical condition. If the diagnosis is Chronic Migraines ( 15 days per month with headache lasting 4 hours a day or longer), the patient has a documented adequate trial (consistent with pharmacy claims data) of beta blockers (e.g., propranolol), tricyclic antidepressants (e.g., amitriptyline), Depakote, and topiramate, or a medical reason was submitted (intolerance, hypersensitivity, contraindication, etc.) why patient is not able to utilize these therapies. If the diagnosis is Overactive Bladder, the patient has a documented adequate trial (consistent with pharmacy claims data) of at least 2 formulary medications (e.g., oxybutynin) Revision/Review Date 7/2017 PARP approved 8/2017 Physician/clinical reviewer must override criteria when, in his/her professional judgement, the requested item is medically necessary. 17