Download Pharmacy Medical Necessity Guidelines

Pharmacy Medical Necessity Guidelines: Orkambi™ (lumacaftor/
ivacaftor)
Effective: October 24, 2016
Prior Authorization Required
√
Type of Review – Care Management
Not Covered
Type of Review – Clinical Review
√
Pharmacy (RX) or Medical (MED) Benefit
RX Department to Review
RXUM
This Pharmacy Medical Necessity Guideline applies to the following:
Fax Numbers:
Tufts Health Plan Commercial Plans
Tufts Health Plan Commercial Plans – large group plans
Tufts Health Plan Commercial Plans – small group and individual plans
Tufts Health Public Plans
RXUM: 617.673.0988
Tufts Health Direct – Health Connector
Tufts Health Together – A MassHealth Plan
Tufts Health Freedom Plan products
Tufts Health Freedom Plan - large group plans
Tufts Health Freedom Plan - small group plans
Note: For Tufts Health Plan Medicare Preferred Members, please refer to the Tufts Health Plan Medicare
Preferred Prior Authorization Criteria. Background, applicable product and disclaimer information can be
found on the last page.
OVERVIEW
FOOD AND DRUG ADMINISTRATION-APPROVED INDICATIONS
Orkambi (lumacaftor/ivacaftor) is a combination of lumacaftor and ivacaftor, a cystic fibrosis
transmembrane conductance regulator (CFTR) potentiator, indicated for the treatment of cystic
fibrosis (CF) in patients age 6 years and older who are homozygous for the F508del mutation in the
CFTR gene. If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to
detect the presence of the F508del mutation on both alleles of the CFTR gene.
The efficacy and safety of ORKAMBI have not been established in patients with CF other than those
homozygous for the F508del mutation.
CF is caused by genetic mutations in the CFTR protein. The CFTR protein is present in the respiratory
epithelium and plays an important role in the regulation of airway surface liquid. Genetic mutations in
this protein result in abnormal airway secretions, chronic endobronchial infection, and progressive
airway obstruction. CFTR potentiators treat the underlying cause of CF by targeting the defective CFTR
protein to help facilitate increased chloride transport. Multiple CFTR potentiators are now commercially
available. Choice of agent for the treatment of CF is dependent on the specific genetic mutation
present.
COVERAGE GUIDELINES
Tufts Health Plan may authorize coverage of Orkambi (lumacaftor/ivacaftor) for Members, when all
the following criteria are met:
1. Documented diagnosis of cystic fibrosis
AND
2. Documentation from an FDA-approved CF mutation test that the member has the F508del mutation
on both alleles of the CFTR gene
AND
3. The member is at least 6 years of age
LIMITATIONS
1. Tufts Health Plan will not approve coverage for Orkambi for any non-FDA approved indications.
2. Orkambi (lumacaftor/ivacaftor) tablets will be limited to 112 tablets per 28 days.
CODES
None
REFERENCES
1. Davies JC, Alton EFW, Bush A. Cystic fibrosis. BMJ. 2007;335:1255-9.
2386562
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Medical Necessity Guidelines:
Orkambi™ (lumacaftor/ ivacaftor)
2. Elborn J, Ramsey B, Boyle M, et al. Efficacy and safety of lumacaftor/ivacaftor combination
therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function
subgroup: a pooled analysis. Lancet Respir Med. 2016 Aug;4(8):617-26.
3. Flume PA, Mogayzel PJ, Robinson KA, et al. Cystic fibrosis pulmonary guidelines. Am J Resp Crit
Care Med. 2009:180:802-8.
4. Gershman AJ, Mehta AC, Infeld M, et al. Cystic fibrosis in adults: an overview for the internist.
Cleve Clin J Med. 2006; 73(12):1065-74.
5. Mogayzel PJ, Flume PA. Update in Cystic Fibrosis 2009. Am J Respir Crit Care Med. 2010:181:53944.
6. Mogayzel PF, Flume PA. Update in Cystic Fibrosis 2010. Am J Respir Crit Care Med.
2011:183:1620-4.
7. Mogayzel PJ, Naureckas ET, Robinson KA, et al. Pulmonary Clinical Practice Guidelines Committee.
Cystic fibrosis pulmonary guidelines. Chronic medications for maintenance of lung health. Am J
Respir Crit Care Med. 2013 Apr 1;187(7):680-9.
8. Orkambi (lumacaftor/ivacaftor) [prescribing information]. Boston, MA: Vertex Pharmaceuticals
Inc.; September 2016.
9. O’Sullivan BP, Freedman SD. Cystic fibrosis. Lancet. 2009;373(9678):1891-1904.
10. Pittman J, Ferkol T. The evolution of cystic fibrosis care. Chest. 2015 Aug;148(2):533-42.
11. Rehman A, Baloch N, Janahi I. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for
Phe508del CFTR. N Engl J Med. 2015 Oct;373(18):1783.
12. Staab D. Cystic fibrosis-therapeutic challenge in cystic fibrosis children. Eur J Endicrinol. 2004;
151:S77-S80.
13. Stoltz D, Meyerholz D, Welsh M. Origins of cystic fibrosis lung disease. N Engl J Med. 2015;
372(4):351-362.
14. Wainwright CE, Elborn JS, Ramsey BW, et al. Lumacaftor-ivacaftor in patients with cystic fibrosis
homozygous for Phe508del CFTR. N Engl J Med. 2015 Jul;373(3):220-31.
15. Wainwright CE, Elborn JS, Ramsey BW, et al. Lumacaftor-ivacaftor in patients with cystic fibrosis
homozygous for Phe508del CFTR. N Engl J Med. 2015 Oct;373(18):1783-4.
16. Zhang W, Zhang X, Zhang YH, et al. Lumacaftor/ivacaftor combination for cystic fibrosis patients
homozygous for Phe508del-CFTR. Drugs Today (Barc). 2016 Apr;52(4):229-37.
APPROVAL HISTORY
October 6, 2015: Reviewed by the Pharmacy and Therapeutics
Subsequent endorsement date(s) and changes made:


September 13, 2016: No changes. Effective September 13, 2016, Medical Necessity Guideline
applies to Tufts Health Together.
October 18, 2016: Updated approval criteria to members at least 6 years of age based on
updated package labeling.
BACKGROUND, PRODUCT AND DISCLAIMER INFORMATION
Pharmacy Medical Necessity Guidelines have been developed for determining coverage for plan
benefits and are published to provide a better understanding of the basis upon which coverage
decisions are made. They are used in conjunction with a Member’s benefit document and in
coordination with the Member’s physician(s). The plan makes coverage decisions on a case-by-case
basis considering the individual Member's health care needs. Pharmacy Medical Necessity Guidelines
are developed for selected therapeutic classes or drugs found to be safe, but proven to be effective in
a limited, defined population of patients or clinical circumstances. They include concise clinical
coverage criteria based on current literature review, consultation with practicing physicians in the
service area who are medical experts in the particular field, FDA and other government agency
policies, and standards adopted by national accreditation organizations. Tufts Health Plan revises and
updates Pharmacy Medical Necessity Guidelines annually, or more frequently if new evidence becomes
available that suggests needed revisions.
This Pharmacy Medical Necessity Guideline does not apply to Uniformed Services Family Health Plan
Members or to certain delegated service arrangements. Unless otherwise noted in the Member’s
benefit document or applicable Pharmacy Medical Necessity Guideline, Pharmacy Medical Necessity
Guidelines do not apply to CareLinkSM Members. For self-insured plans, drug coverage may vary
depending on the terms of the benefit document. If a discrepancy exists between a coverage guideline
and a self-insured Member’s benefit document, the provisions of the benefit document will govern.
Applicable state or federal mandates will take precedence.
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Pharmacy Medical Necessity Guidelines:
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For Tufts Health Plan Medicare Preferred, please refer to Tufts Health Plan Medicare Preferred Prior
Authorization Criteria.
Treating providers are solely responsible for the medical advice and treatment of Members. The use of
this policy is not a guarantee of payment or a final prediction of how specific claim(s) will be
adjudicated. Claims payment is subject to Member eligibility and benefits on the date of service,
coordination of benefits, referral/authorization and utilization management guidelines when applicable,
and adherence to plan policies and procedures and claims editing logic.
Provider Services
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