Loading...

Blue Cross Blue Shield MA specialty drug Coverage Update | OpenPayer

CurrentBlue Cross Blue Shield - MassachusettsPolicy 027

Specialty pharmacy coverage for select rare-disease drugs (DMD, AHP, ALS)

Defines prior authorization, coverage criteria, and coding for specific specialty drugs (e.g., eteplirsen, golodirsen, casimersen, viltolarsen, givosiran, tofersen) for commercial BCBSMA members; applies to outpatient pharmacy benefit and certain medical benefit scenarios. Affects prescribers and pharmacy/medical authorization staff.

Policy Summary

PayerBlue Cross Blue Shield - Massachusetts

PolicySpecialty pharmacy coverage for select rare-disease drugs (DMD, AHP, ALS)

Policy CodePolicy 027

Change TypeAdded Qalsodycoding clarified

Effective Date

Next Review Date

Key ActionObtain prior authorization and provide required documentation (diagnosis, mutation confirmation, relevant test results, ambulation status, and calculated weight-based dosing) to support coverage.

SourceLink

POLICY UPDATE CHANGES

Qalsody (tofersen) was added to the policy.

Coding information was clarified in 1/2024.

Amondys 45 (casimersen) was added to the policy previously.

6named drugs covered by policy

6 monthsinitial prior authorization duration for most DMD drugs

1 yrcontinuation approval duration for Givlaari

≥300 m

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Drug-Specific Coverage Criteria

Amondys 45 (casimersen) — Initial and reauthorization

Covered when ALL of the following are met

Amondys 45 coverage: Confirmed diagnosis of Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping; concurrent use of glucocorticoids unless clinically contraindicated; documentation of ambulation without assistance or devices; prescription written by a board-certified or board-eligible neurologist; dose limited to the FDA-approved 30 mg/kg administered once weekly with weight and calculated dose provided.

Reauthorization requires the same criteria; prior authorization granted for up to six months.

Exondys 51 (eteplirsen) — Initial and reauthorization

Covered when ALL of the following are met

Exondys 51 coverage: Confirmed diagnosis of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping; documentation of ambulation without assistance or devices; concurrent use of glucocorticoids unless clinically contraindicated; prescription written by a board-certified or board-eligible neurologist; dose limited to the FDA-approved 30 mg/kg administered once weekly with weight and calculated dose provided.

Reauthorization requires the same criteria; prior authorization granted for up to six months.

Vyondys 53 (golodirsen) and Viltepso (viltolarsen) — Initial and reauthorization

Covered when ALL of the following are met

Exon 53 DMD coverage: Confirmed diagnosis of Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping; a recent (within 4 weeks) pre-treatment 6-minute walk test of at least 300 meters while walking independently (without assistive device); documentation of ambulation without assistance or devices; concurrent use of glucocorticoids unless clinically contraindicated; prescription written by a board-certified or board-eligible neurologist; member has stable pulmonary and cardiac function; member is not concurrently enrolled in a clinical trial to receive an experimental therapy for DMD; dose limited to the FDA-approved 30 mg/kg administered once weekly with weight and calculated dose provided.6MWT ≥300 m

Reauthorization requires the same criteria; prior authorization granted for up to six months.

Givlaari (givosiran) — Initial and continuation

Covered when ALL of the following are met

Givlaari coverage: Confirmed diagnosis of acute hepatic porphyria (AHP), including acute intermittent porphyria (AIP) or hereditary coproporphyria (HCP); elevated urinary or plasma porphobilinogen (PBG) or ALA values within the past year; patient meets policy age criteria; active disease with at least 2 documented porphyria attacks within the last 6 months or 1 attack involving the central nervous system (CNS); patient is not anticipating liver transplantation.

First prior authorization granted for up to six months; continuation approvals may be granted for up to one year; reauthorization requires the same criteria.

Qalsody (tofersen) — Initial and reauthorization

Covered when ALL of the following are met

Qalsody coverage: Confirmed diagnosis of amyotrophic lateral sclerosis (ALS) in adults with a mutation in the superoxide dismutase 1 (SOD1) gene; patient meets policy age criteria (prescriber must document adult status given inconsistent age ranges in policy text); prescription written by an appropriate specialist.

Reauthorization requires the same criteria; prior authorization duration noted in policy.

We do not cover the medications listed in this policy for conditions other than those explicitly listed. Requests for these drugs to treat indications not specified under the policy’s covered diagnoses may be denied.

Requests based exclusively on the use of samples will not meet coverage criteria. Additional clinical information demonstrating medical necessity must be provided by the requesting prescriber for review; sample use alone is considered insufficient for a coverage determination.

Initial Therapy Requirements

Initial therapy — diagnosis-specific initial approval rules

Initial therapy approvals require meeting diagnosis-specific criteria below

Initial DMD therapies (general): Confirmed DMD mutation amenable to the indicated exon skipping (45, 51, or 53 as specified); evidence of ambulation without assistance or devices; concurrent glucocorticoid use unless clinically contraindicated; prescription by a board-certified or board-eligible neurologist; dosing limited to FDA-approved 30 mg/kg once weekly with weight and calculated dose provided.

Initial prior authorizations for DMD exon-skipping therapies are generally granted for up to six months.

Exon-specific requirements

Exon 45: Meets the general DMD therapy criteria and confirmed mutation amenable to exon 45 skipping.
Exon 51: Meets the general DMD therapy criteria and confirmed mutation amenable to exon 51 skipping.

Continuation / Reauthorization Criteria

Continuation therapy — reauthorization rules that mirror initial criteria

Continuation/reauthorization decisions mirror the initial coverage criteria and require documentation of ongoing benefit

Continuation for DMD exon-skipping drugs: Reauthorization requires the same clinical criteria as initial approval (confirmed mutation amenable to the indicated exon skip, ambulation without assistance, glucocorticoid use unless contraindicated, prescriber specialty, and dose within FDA-approved 30 mg/kg weekly).

Prior authorizations for DMD exon-skipping therapies are generally granted for up to six months on reauthorization.

Continuation for Givlaari: Reauthorization requires the same clinical criteria as initial approval (AHP diagnosis with biochemical evidence and attack history); continuation approvals may be granted for up to one year.

Continuation for Qalsody: Reauthorization requires the same clinical criteria as initial approval (adult ALS with SOD1 mutation); prior authorization duration noted in policy.

Provider Requirements and Documentation

Prior Authorization

Prior Authorization Required

Prior authorization is required for all medications listed in this policy. Typical initial approvals are issued for up to six months (Givlaari initial approval up to six months; continuation approvals up to one year when specified). Providers should contact Blue Cross Blue Shield of Massachusetts Pharmacy Operations to submit requests: Tel: 1-800-366-7778; Fax: 1-800-583-6289. Individual consideration is available for requests that do not meet clinical criteria — see the Individual Consideration section.

Blue Cross Blue Shield of Massachusetts Pharmacy Operations Department, 25 Technology Place, Hingham, MA 02043
Prior authorization phone: 1-800-366-7778; fax: 1-800-583-6289

Step Therapy

Step Therapy and Formulary

Step therapy/formulary status: All drugs addressed by this policy are on the standard formulary and are PA Required. Providers must document prior trials only when explicitly required by the clinical criteria for the specific drug. To request coverage, submit the Formulary Exception/Prior Authorization form by phone, fax, or mail as noted above.

Billing and Coding

HCPCS CodesHCPCS

C9071	Injection, viltolarsen (Viltepso) 10 mg.
C9399	Unclassified drugs or biologicals (Outpatient Use ONLY).
J0223
J1304	Injection, tofersen, 1 mg (Qalsody).
J1428	Injection, eteplirsen, 10 mg (Exondys 51).
J1429	Injection, golodirsen, 10 mg (Vyondys 53).
J3490	Unclassified drugs.
J3590	Unclassified biologics.

6-Minute Walk Test requirement for exon 53 DMD drugs

ThresholdPre-treatment 6-Minute Walk Test (6MWT) ≥ 300 meters

Timing requirement6MWT must be performed within 4 weeks prior to the authorization request (recent pre-treatment test)

Ambulation requirement6MWT must be performed while walking independently (without assistive devices such as cane, walker, or wheelchair)

Applies toVyondys 53 (golodirsen) and Viltepso (viltolarsen) — DMD exon 53 skipping therapies

Authorization durationIf approved, prior authorization is typically granted for up to six months (reauthorization requires same criteria)

Step Therapy Requirements

Step	Requirement	How to Document
1
Providers may be required to demonstrate trials of preferred formulary medications or provide a clinical rationale for exceptions before approval.
List the formulary alternatives tried (name, strength), dates of therapy, reason for failure or intolerance, and any relevant clinical notes.

Site-of-Care / Dispensing

Billing Rule

Use in‑network retail specialty pharmacy (exceptions noted)

Members (except certain Medicare plans) must fill prescriptions for these medications at a retail specialty pharmacy in BCBSMA’s in‑network retail specialty pharmacy list; see BCBSMA specialty pharmacy list for participating providers.

Applies to BCBSMA members other than Medex, Blue MedicareRx, and Medicare Advantage plans that include prescription coverage.

Key Definitions

Exon-skipping — definition and examples

DefinitionExon-skipping therapy: an antisense oligonucleotide designed to skip a specific exon (e.g., exon 45, 51, 53) in dystrophin pre-mRNA to produce a truncated but functional dystrophin protein.

Therapy class examplesAntisense oligonucleotide exon-skipping drugs: Amondys 45 (casimersen) for exon 45, Exondys 51 (eteplirsen) for exon 51, Vyondys 53 (golodirsen) and Viltepso (viltolarsen) for exon 53.

Clinical intentRestores the mRNA reading frame for specific DMD mutations to enable production of a shortened, functional dystrophin protein.

Coverage contextCoverage requires confirmed DMD mutation amenable to the indicated exon skipping and additional clinical documentation per drug-specific criteria.

Background and Clinical Context

Duchenne muscular dystrophy (DMD) is a genetic, progressive muscle-wasting disorder caused by mutations in the DMD gene that can be amenable to targeted exon-skipping antisense oligonucleotide therapies. Exon-skipping agents (for example those targeting exon 45, 51, or 53) are designed to restore the dystrophin reading frame for a specific mutation, producing a truncated but functional dystrophin protein. Policy-specific requirements for exon-skipping drugs include confirmation that the patient’s DMD mutation is amenable to the indicated exon skip, documentation of ambulation without assistance, concurrent glucocorticoid use unless contraindicated, prescriber specialty (board-certified or board-eligible neurologist), and dosing limits consistent with FDA-approved dosing (for the exon 53 agents the policy limits dose to 30 mg/kg once weekly). For exon 53 therapies the policy also requires a recent (within 4 weeks) pre-treatment 6-Minute Walk Test ≥ 300 meters while walking independently and evidence of stable pulmonary and cardiac function.

Policy Revision History

2024-01-01coding clarificationLatest

Clarified coding information in the policy history.

2023-09-01addition

Qalsody (tofersen) was added to the policy and individual consideration language was aligned with state statute 118E MGL § 51A.

2021-07-01addition