Pharmacy Policy Bulletin

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Pharmacy Policy Bulletin Title: Policy #: Pulmonary Arterial Hypertensive (PAH) agents Rx.01.83 Application of pharmacy policy is determined by benefits and contracts. Benefits may vary based on product line, group, or contract. Some medications may be subject to precertification, age, gender or quantity restrictions. Individual member benefits must be verified. This pharmacy policy document describes the status of pharmaceutical information and/or technology at the time the document was developed. Since that time, new information relating to drug efficacy, interactions, contraindications, dosage, administration routes, safety, or FDA approval may have changed. This Pharmacy Policy will be regularly updated as scientific and medical literature becomes available. This information may include new FDA-approved indications, withdrawals, or other FDA alerts. This type of information is relevant not only when considering whether this policy should be updated, but also when applying it to current requests for coverage. Members are advised to use participating pharmacies in order to receive the highest level of benefits. Intent: The intent of this policy is to communicate the medical necessity criteria for sildenafil (Revatio ), tadalafil (Adcirca), riociguat (Adempas), ambrisentan (Letairis), macitentan (Opsumit), and selexipag (Uptravi) as provided under the member's pharmacy benefit. Description: Sildenafil (Revatio ), tadalafil (Adcirca), riociguat (Adempas), macitentan (Opsumit), ambrisentan (Letairis), treprostinil (Orenitram ), and selexipag (Uptravi ) are indicated for the treatment of pulmonary arterial hypertension (World Health Organization [WHO] Group I) to improve exercise ability. Riociguat (Adempas) is also indicated for the treatment of adults with persistent/recurrent chronic thromboembolic pulmonary hypertension (World Health Organization [WHO] group 4), after surgical treatment or inoperable chronic thromboembolic pulmonary hypertension, to improve exercise capacity and WHO functional class. Pulmonary arterial hypertension (PAH) is a rare disease, with an estimated prevalence of 15-50 cases per million. Idiopathic PAH (IPAH) has an annual incidence of 1-2 cases per million people in the US and Europe and is 2-4 times as common in women as in men. The mean age at diagnosis is around 45 years, although the onset of symptoms can occur at any age. Despite the true relative prevalence of IPAH, heritable PAH (HPAH), and associated PAH (APAH) being unknown, it is likely that IPAH accounts for at least 40% of cases of PAH, with APAH accounting for the majority of the remaining cases. Due to the non-specific nature of the symptoms, PAH is unfortunately most frequently diagnosed when patients have reached an advanced stage of disease (WHO Functional Class III and IV). The intent of the policy is to ensure proper usage of the medication for which it is FDA approved.

Sildenafil (Revatio ) and tadalafil (Adcirca ) are selective inhibitor of cyclic guanosine monophosphate (cgmp)-specific phosphodiesterase-5 (PDE5) in the smooth muscle of the pulmonary arteries, where cgmp is degraded by PDE5. As a result, increases cgmp within the pulmonary arteries, causing relaxation and vasodilation of the pulmonary arteries. Riociguat (Adempas) has a dual mode of action. It sensitizes soluble guanylate cyclase (sgc) to endogenous nitric oxide (NO) by stabilizing the NO-sGC binding. Riociguat (Adempas) also directly stimulates sgc independent of NO. Macitentan (Opsumit) and ambrisentan (Letairis) blocks endothelin (ET) 1 from binding to endothelin receptor subtypes ETA and ETB on vascular endothelium and smooth muscle. Stimulation of these receptors is associated with vasoconstriction, fibrosis, proliferation, hypertrophy, and inflammation. Treprostinil (Orenitram ) is a prostacyclin analogue. The major pharmacologic actions are direct vasodilation of pulmonary and systemic arterial vascular beds, inhibition of platelet aggregation, and inhibition of smooth muscle cell proliferation. Selexipag (Uptravi ) is a prostacyclin receptor agonist that works at the same pathway as the prostacyclin analogues, but activates the IP receptor. It is one of two orally administered agents that work within the prostacyclin pathway. Selexipag (Uptravi ) indicated for the treatment of PAH (WHO Group I) to delay disease progression and reduce hospitalization. Policy: Tadalafil (Adcirca ) and sildenafil (Revatio ) are approved when all of the following inclusion criteria are met: 1. Documentation of a diagnosis of pulmonary arterial hypertension (PAH) WHO Group I with New York Heart Association (NYHA) Functional Class II or III 3. Documentation of mean pulmonary artery pressure greater than 25 mm Hg at rest or greater than 30 mm Hg with exertion 4. No concurrent use of nitrates or other contraindicated medications for the drug requested, unless recommended by a cardiologist or pulmonologist Ambrisentan (Letairis ) and macitentan (Opsumit ) are approved when ALL of the following inclusion criteria are met: 3. Mean pulmonary artery pressure greater than 25 mm Hg at rest or greater than 30 mm Hg with exertion 4. Inability to tolerate or inadequate response to bosentan (Tracleer ) Treprostinil (Orenitram ), treprostinil (Tyvaso ) and iloprost (Ventavis ) are approved when ALL of the following inclusion criteria are met: 3. Mean pulmonary artery pressure greater than 25 mm Hg at rest or greater than 30 mm Hg with exertion

Riociguat (Adempas ) is approved when ALL of the following criteria are met: 1. Member has a diagnosis of ONE of the following: a. Pulmonary arterial hypertension (PAH) WHO Group I with New York Heart b. Persistent/recurrent Chronic Thromboembolic Pulmonary Hypertension (CTEPH) (WHO Group 4) after surgical treatment or inoperable CTEPH 3. Documentation of mean pulmonary artery pressure greater than 25 mm Hg at rest or greater than 30 mm Hg with exertion 4. No concurrent use of specific (i.e. sildenafil) or non-specific (i.e. dipyridamole, theophylline) phosphodiesterase inhibitors. 5. No concurrent use of nitrates or nitric oxide donors (i.e. amyl nitrite) Selexipag (Uptravi) is approved when ALL of the following documentation is provided: Association (NYHA) Functional Class II-IV; and ; and 3. Documentation of mean pulmonary artery pressure greater than 25mm Hg at rest or greater than 30mm Hg with exertion; and 4. Inadequate response or inability to tolerate TWO of the following: a. Endothelin Receptor Antagonist (Tracleer if naïve to the class) b. Phosphodiesterase Type 5 Inhibitor (sildenafil if naïve to the class) c. Riocuguat (Adempas) 5. Not taken in combination with a prostanoid/prostacyclin analogue (e.g. epoprostenol, iloprost, treprostinil) Initial authorization: 6 months Reauthorization criteria: Documentation of stabilization or improvement as evaluated by a cardiologist or pulmonologist Reauthorization duration: 12 months Black Box Warning: Adempas, Opsumit and Letairis: Embryo-fetal toxicity: Do not administer riociguat to a pregnant patient because it may cause fetal harm. Female patients of reproductive potential: Prevent pregnancy during treatment and for 1 month after stopping treatment by using acceptable methods of contraception. Guidelines: Refer to the specific manufacturer's prescribing information for administration and dosage details and any applicable Black Box warnings. BENEFIT APPLICATION Subject to the terms and conditions of the applicable benefit contract, the applicable drug(s) identified in this policy is (are) covered under the pharmacy benefits of the Company s products when the

medical necessity criteria listed in this pharmacy policy are met. Any services that are experimental/investigational or cosmetic are benefit contract exclusions for all products of the Company. References: ADCIRCA prescribing information. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2015/022332s007lbl.pdf. Accessed on October 6, 2015. ADEMPAS prescribing information. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2014/204819s002lbl.pdf. Accessed on LETAIRIS prescribing information. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2015/022081s033lbl.pdf. Accessed on OPSUMIT prescribing information. Actelion Pharmaceuticals. South San Francisco, CA. Accessed on ORENITRAM prescribing information. United Therapeutics. Research Triangle Park, NC. Accessed on REMODULIN prescribing information. United Therapeutics. Research Triangle Park, NC. Accessed on Revatio [package insert]. New York, NY: Pfizer, Inc.; 2009. Also available online at: http://www.pfizer.com/files/products/uspi_revatio.pdf. Revised 03/2014. Accessed on October 6, 2015. Uptravi prescribing information. Actelion Pharmaceuticals US, Inc. San Francisco, CA. December 2015. Available at: https://www.uptravi.com/pdf/uptravi-full-prescribing-information.pdf. Accessed on May 6, 2016. Applicable Drugs: Inclusion of a drug in this table does not imply coverage. Eligibility, benefits, limitations, exclusions, precertification/referral requirements, provider contracts, and Company policies apply. Cross References: Brand Name Generic Name Adcirca Adempas Letaris Opsumit Orenitram Revatio Tyvaso Uptravi Ventavis tadalafil riociguat ambrisentan macitentan treprostinil sildenafil trepostinil selexipag iloprost

Policy Version Number: 9.00 P&T Approval Date: April 14, 2016 Policy Effective Date: June 01, 2016 Next Required Review Date: October 08, 2016 The Policy Bulletins on this web site were developed to assist Independence Blue Cross ("Independence") in administering the provisions of the respective benefit programs, and do not constitute a contract. If you have coverage through the Independence organization, please refer to your specific benefit program for the terms, conditions, limitations and exclusions of your coverage. Independence does not provide health care services, medical advice or treatment, or guarantee the outcome or results of any medical services/treatments. The facility and professional providers are responsible for providing medical advice and treatment. Facility and professional providers are independent contractors and are not employees or agents of Independence. If you have a specific medical condition, please consult with your doctor. Independence reserves the right at any time to change or update its Policy Bulletins. 2016 Independence Blue Cross. All Rights Reserved. Independence Blue Cross is an independent licensee of the Blue Cross and Blue Shield Association.