CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Deflazacort (Emflaza) — Coverage Criteria for Duchenne Muscular Dystrophy

Policy governing coverage criteria, quantity limits, and continuation requirements for deflazacort (Emflaza) for treatment of Duchenne muscular dystrophy (DMD) for members in the payer's Medicaid scope.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyDeflazacort (Emflaza) — Coverage Criteria for DMD

Policy CodePolicy N/A

Change TypeAnnual review — no material changes

Effective Date02/01/2021

Next Review DateN/A

Key ActionObtain prior authorization and provide documentation of DMD diagnosis and baseline functional measures to request a 6-month approval.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

2 yrsMinimum member age

6 moAuthorization duration

2 tabs/6 mg dayTablet quantity limit

MedicaidPayer scope

Coverage Criteria for Deflazacort (Emflaza)

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Initial Approval for Deflazacort: Member is at least 2 years of age; Emflaza is prescribed by or in consultation with a neurologist who specializes in the treatment of Duchenne muscular dystrophy; diagnosis of DMD confirmed by either genetic testing demonstrating a mutation in the DMD gene or muscle biopsy demonstrating absent dystrophin; documentation that the member meets one of the following: unmanageable or clinically significant psychiatric/behavioral issues (e.g., abnormal behavior, aggression, irritability) while on prednisone or prednisolone; unmanageable and/or clinically significant weight gain/obesity while receiving prednisone or prednisolone for ≥6 months as evidenced by BMI in the overweight or obese category (refer to appendix for pediatric and adult BMI/weight-status categories); or clinically significant growth stunting while receiving prednisone or prednisolone as evidenced by decreased growth trajectory or growth velocity, decline in mean height percentile for age from baseline, or reduction in serum biomarkers of bone formation or bone turnover (e.g., osteocalcin, PINP, CTX1); AND baseline documentation of one or more of the following measures (e.g., dystrophin level, timed function tests such as TTSTAND/6MWT/TTRW/TTCLIMB/4SC, upper limb function tests, FVC% predicted, NSAA).

Authorization duration: 6 months

Continuation Therapy

Covered when ALL of the following are met

Continuation of Therapy: Member meets all initial authorization criteria; documentation that the member is receiving clinical benefit from deflazacort compared to pretreatment baseline in one or more of the following: increase in dystrophin level; stability, improvement, or slowed rate of decline in timed function tests (e.g., TTSTAND, 6MWT, TTRW, TTCLIMB/4SC); stability, improvement, or slowed rate of decline in upper limb function tests; stability, improvement, or slowed rate of decline in North Star Ambulatory Assessment (NSAA) score; stability, improvement, or slowed rate of decline in FVC% predicted; or improvement in quality of life.

Authorization duration: 6 months

Age, BMI Thresholds and Coding Notes

Age and BMI thresholds

Minimum age for therapyMember must be at least 2 years of age.

Pediatric BMI categories (ages 2–19)<5th percentile = Underweight; 5th–<85th percentile = Healthy weight; 85th–<95th percentile = Overweight; ≥95th percentile = Obese.

Adult BMI categories (≥20 years)<18.5 = Underweight; 18.5–24.9 = Healthy weight; 25.0–29.9 = Overweight; ≥30.0 = Obese.

Use of BMI for eligibility determinationUnmanageable weight gain/obesity on prednisone or prednisolone is defined by BMI in the overweight or obese categories (see appendix) after ≥6 months of therapy.

Prescribing, Authorization, and Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. An authorization of 6 months may be granted for initial therapy when all initial criteria are met. Continuation (renewal) authorizations may also be granted for 6 months when all continuation criteria are satisfied and the member demonstrates clinical benefit from deflazacort compared to pretreatment baseline.

Authorization duration: 6 months for initial approval
Continuation authorization: 6 months when continuation criteria met

Denial Risk

Indication Requirement — DMD only

Emflaza (deflazacort) must be used only for the FDA‑approved indication: treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. Use for indications other than the FDA‑approved indication or other compendial uses is considered experimental/investigational and is not medically necessary.

Documentation Required

Documentation Expectations

For initial approval, documentation must confirm the member is at least 2 years old and that DMD diagnosis is confirmed by genetic testing showing a DMD gene mutation or by muscle biopsy demonstrating absent dystrophin. Additional supporting documentation for baseline status and prior intolerance or adverse effects to prednisone/prednisolone should be provided as applicable.

Member age >= 2 years
Confirmation of DMD by genetic testing or muscle biopsy showing absent dystrophin

Background

Deflazacort (Emflaza) is FDA‑approved for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 2 years and older. The policy requires that treatment be limited to the approved DMD indication (and recognized compendial uses); uses outside this indication are treated as experimental/investigational and are not covered. Baseline and follow‑up clinical measures (for example, timed function tests, 6‑minute walk test, NSAA, upper limb function tests, and FVC% predicted) and documentation of diagnosis are expected to support authorization and continuation decisions.

Definitions and Clinical Measures

Timed function tests

Timed function tests (examples)Includes Time to Stand (TTSTAND), 6‑Minute Walk Test (6MWT), Time to Run/Walk 10 meters (TTRW), Time to Climb 4 Stairs (TTCLIMB or 4SC).

PurposeUsed as baseline and continuation measures to document stability, improvement, or slowed rate of decline while on therapy.

Other functional measures referencedUpper limb function tests, North Star Ambulatory Assessment (NSAA) score, and FVC% predicted are complementary outcome measures.

BMI categories

Children (2–19 years) weight status by BMI percentile<5th percentile = Underweight; 5th–<85th percentile = Healthy weight; 85th–<95th percentile = Overweight; ≥95th percentile = Obese.

Adults (≥20 years) weight status by BMI<18.5 = Underweight; 18.5–24.9 = Healthy weight; 25.0–29.9 = Overweight; ≥30.0 = Obese.

Reference locationDefinitions and thresholds are provided in the policy appendix (BMI percentile table for children and BMI cutoffs for adults).