CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Evrysdi

Defines prior authorization, documentation, prescriber specialty, initial and continuation coverage criteria, dosing limits, and exclusions for Evrysdi for treatment of spinal muscular atrophy (SMA). Applies to pediatric and adult patients when all criteria are met.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyEvrysdi

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionSubmit genetic testing confirming SMN1 deletion/mutation and baseline motor assessment records for initiation and provide most recent (within 1 month) assessment showing improvement or stabilization for continuation prior authorization review.

SourceLink

POLICY UPDATE CHANGES

No material changes

3Covered SMA Types (1/2/3)

5Accepted baseline/assessment tools

4Age/weight dosing tiers

Coverage Summary

Provider Actions & Initiation Requirements

Prior Authorization

Prior authorization required with documentation

Submit genetic testing confirming SMN1 deletion/mutation and medical records of the baseline motor assessment using at least one specified motor function test to initiate prior authorization. For continuation, submit the most recent assessment (less than 1 month prior to the continuation request) documenting a positive clinical response. Prior authorization review will use these documents.

Documentation Required

Baseline and follow-up motor assessments required

Provide a baseline motor assessment for initiation using at least one accepted test (HINE-2, HFMSE, CHOP-INTEND, MFM32, or BSID-III). For continuation requests submit the most recent assessment performed less than 1 month prior to the request demonstrating improvement or stabilization (per the policy response criteria). Members previously on gene therapy should provide documentation of impact or stabilization when applicable.

Accepted tests: HINE-2, HFMSE, CHOP-INTEND, MFM32, BSID-III
Continuation assessment must be <1 month prior to request and show positive clinical response per specified thresholds

Denial Risk

Coverage exclusion for non-FDA indications and concomitant therapy

All indications outside FDA‑approved treatment of SMA are considered experimental/investigational and not medically necessary. Concomitant use of Evrysdi with Spinraza is not allowed and may be denied.

Billing Rule

Dose limit enforcement

Prescribed daily dose must not exceed the policy’s age/weight maximums. Doses above these limits are outside the policy and may not be covered.

Members <2 months: 0.15 mg/kg daily
Members 2 months to <2 years: 0.2 mg/kg daily
Members ≥2 years and <20 kg: 0.25 mg/kg daily
Members ≥2 years and ≥20 kg: 5 mg daily

Continuation Therapy Criteria

Continuation of Therapy - Initial Continuation Criteria (general note)

Authorization of 12 months may be granted for continued treatment of SMA when all of the following criteria are met:

ALL of the following

Member has type 1, type 2, or type 3 SMA
Respiratory support exclusions
- Member is not dependent on invasive ventilation or tracheostomy
- Member is not dependent on use of non-invasive ventilation beyond naps and nighttime sleep
Acceptable evidence of positive clinical response
- HINE-2 response options
  - Member exhibited improvement or maintenance of previous improvement of at least a 2-point (or maximal score) increase in ability to kick
  - Member exhibited improvement or maintenance of previous improvement of at least a 1-point (or maximal score) increase in any other HINE-2 milestone (excluding voluntary grasp)
  - Member exhibited improvement or maintenance of previous improvement in more HINE-2 motor milestones than worsening (net positive improvement)
  - Member achieved and maintained any new motor milestones when they would otherwise be unexpected to do so (e.g., sit or stand unassisted, walk)
- HFMSE response options
  - Member exhibited improvement or maintenance of previous improvement of at least a 3-point increase in score
  - Member has achieved and maintained any new motor milestone from pretreatment baseline when they would otherwise be unexpected to do so
- CHOP-INTEND response options
  - Member exhibited improvement or maintenance of previous improvement of at least a 4-point increase in score
  - Member has achieved and maintained any new motor milestone from pretreatment baseline when they would otherwise be unexpected to do so
- MFM32
  - Member has experienced an increase in their MFM32 score from baseline
  - That increase correlates with a clinically significant functional improvement
- BSID-III: Member exhibited the ability to sit without support for at least 5 seconds after 12 months of treatment
- Member was prescribed Evrysdi due to clinical worsening after receiving gene replacement therapy (e.g., Zolgensma) and there is documentation of stabilization or improvement in clinical status with Evrysdi therapy (e.g., impact on motor milestones)
Member will not use Evrysdi and Spinraza concomitantly
Continuation daily dose limits by age/weight (must not exceed):
- Members less than 2 months of age: 0.15 mg/kg
- Members 2 months to less than 2 years of age: 0.2 mg/kg
- Members 2 years of age and older weighing less than 20 kg: 0.25 mg/kg
- Members 2 years of age and older weighing 20 kg or more: 5 mg

ref”:

Note regarding Gene Replacement Therapy

ALL of the following

Members previously established on Evrysdi who subsequently received gene replacement therapy must meet all initial criteria prior to re-starting therapy on Evrysdi

Unproven Indications / Exclusions

Exclusions: All indications outside the FDA-approved treatment of SMA are considered experimental/investigational and not medically necessary. Concomitant use of Evrysdi with nusinersen (Spinraza) is excluded and may result in claim denial.

Clinical Evidence & Background

Background: Evrysdi (risdiplam) is FDA-approved for treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. This policy restricts coverage to FDA-approved SMA indications and requires genetic confirmation (SMN1 deletion or mutation) and standardized motor function assessments (HINE-2, HFMSE, CHOP-INTEND, MFM32, or BSID-III) to establish baseline motor ability and to document response to therapy.

Key thresholds and dose limits

HINE-2 worsening (post-gene therapy)>= 2 point decline on kicking; >= 1 point decline on any other milestone (excluding voluntary grasp)

HFMSE worsening (post-gene therapy)>= 3 points decline

CHOP-INTEND worsening (post-gene therapy)>= 4 points decline

MFM32 worsening (post-gene therapy)Decline from baseline

BSID-III worsening (post-gene therapy)Inability to sit without support for more than 5 seconds (per item 22)

HINE-2 continuation improvement - kicking>= 2-point increase (or maximal score)

HINE-2 continuation improvement - other milestones>= 1-point increase (or maximal score), excluding voluntary grasp

HFMSE continuation improvement>= 3-point increase

CHOP-INTEND continuation improvement>= 4-point increase

BSID-III continuation improvementAbility to sit without support for at least 5 seconds after 12 months of treatment

Daily dose limit - <2 months0.15 mg/kg

Daily dose limit - 2 months to <2 years0.2 mg/kg

Daily dose limit - >=2 years & <20 kg0.25 mg/kg

Daily dose limit - >=2 years & >=20 kg5 mg