FDA-approved, for example:

• Duchenne muscular dystrophy (Exondys 51, Vyondys 53)
• Spinal muscular atrophy (Spinraza, Zolgensma)

Note: The above list may not include all FDA-approved indications.

Please note: In the case where the prior authorization (PA) status column indicates PA, both the brand and generic (if available) require PA. Typically, the generic is preferred when available unless the brand-name drug appears on the MassHealth Brand Name Preferred Over Generic Drug List. In general, when requesting the non-preferred version, whether the brand or generic, the prescriber must provide medical records documenting an inadequate response or adverse reaction to the preferred version, in addition to satisfying the criteria for the drug itself.

• All prior-authorization requests must include clinical diagnosis, drug name, dose, and frequency.
• A preferred drug may be designated for this therapeutic class. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing a non-preferred drug within a therapeutic class. Additional information about these agents, including PA requirements and preferred products, can be found within the MassHealth Drug List at www.mass.gov/druglist.
• For recertification requests, approval may require submission of additional documentation including, but not limited to, documentation of: some or all criteria for the original approval; response to therapy; clinical rationale for continuation of use; status of member’s condition; appropriate diagnosis; appropriate age; appropriate dose, frequency, and duration of use for requested medication; complete treatment plan; current laboratory values; and member’s current weight.
• Additional criteria may apply, depending upon the member’s condition and requested medication (see below).

Exondys 51

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • confirmed out-of-frame deletion in the DMD gene that is amenable to exon 51 skipping; and
  • one of the following:
    • prescriber is a neuromuscular neurologist; or
    • consult notes from a neuromuscular neurology office are provided; and
  • member is ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 200 meters (test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); and
  • dosing is appropriate (30 mg/kg intravenously every week); and
  • one of the following:
    • member has received a corticosteroid for at least six months prior and member will continue to use a corticosteroid in combination with the requested agent; or
    • contraindication to corticosteroids; and
  • member has at least a baseline measurement for each of the following timed function tests as shown in medical records (tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner):
    • timed ten-meter walk/run (time in seconds); and
    • timed floor (supine) to stand (time in seconds); and
    • timed four-step descend (time in seconds); and
    • timed four-step climb (time in seconds); and
    • timed sit to stand (time in seconds).

• For recertification requests, documentation of all of the following is required:
  • member remains ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 200 meters (test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); and
  • member has a stable or improving pattern of 6MWTs as shown in medical records with results of a pretreatment baseline and all interim results (all previous 6MWTs results must be included); and
  • dosing remains appropriate (30 mg/kg intravenously every week); and
  • one of the following:
    • member continues to utilize corticosteroids in combination with the requested agent; or
    • contraindication to corticosteroids; and
  • member has a stable or improving pattern of observed performance on at least two of the following five timed function tests as shown in medical records (all results for all tests must be included with the date of performance; tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner):
    • timed ten-meter walk/run (time in seconds); and
    • timed floor (supine) to stand (time in seconds); and
    • timed four-step descend (time in seconds); and
    • timed four-step climb (time in seconds); and
    • timed sit to stand (time in seconds).

Spinraza

• Documentation of all of the following is required:
  • appropriate diagnosis (type 1, 2, or 3 spinal muscular atrophy); and
  • genetic test confirming diagnosis of spinal muscular atrophy; and
  • prescriber is a neurologist or consult notes from a neurology office are provided; and
  • baseline motor function test; and
  • appropriate dose.
• For recertification requests, medical records documenting positive response to therapy (e.g., follow up information on motor function tests and/or member’s improvement or stability of function) is required.

Vyondys 53

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • confirmed out-of-frame deletion in the DMD gene that is amenable to exon 53 skipping; and
  • one of the following:
    • prescriber is a neuromuscular neurologist; or
    • consult notes from a neuromuscular neurology office are provided; and
  • member is ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 200 meters (test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); and
  • dosing is appropriate (30 mg/kg intravenously every week); and
  • one of the following:
    • member has received a corticosteroid for at least six months prior and member will continue to use a corticosteroid in combination with the requested agent; or
    • contraindication to corticosteroids; and
  • member has at least a baseline measurement for each of the following timed function tests as shown in medical records (tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner):
    • timed ten-meter walk/run (time in seconds); and
    • timed floor (supine) to stand (time in seconds); and
    • timed four-step descend (time in seconds); and
    • timed four-step climb (time in seconds); and
    • timed sit to stand (time in seconds).

• For recertification requests, documentation of all of the following is required:
  • member remains ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 200 meters (test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); and
  • member has a stable or improving pattern of 6MWTs as shown in medical records with results of a pretreatment baseline and all interim results (all previous 6MWTs results must be included); and
  • dosing remains appropriate (30 mg/kg intravenously every week); and
  • one of the following:
    • member continues to utilize corticosteroids in combination with the requested agent; or
    • contraindication to corticosteroids; and
  • member has a stable or improving pattern of observed performance on at least two of the following five timed function tests as shown in medical records (all results for all tests must be included with the date of performance; tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner):
    • timed ten-meter walk/run (time in seconds); and
    • timed floor (supine) to stand (time in seconds); and
    • timed four-step descend (time in seconds); and
    • timed four-step climb (time in seconds); and
    • timed sit to stand (time in seconds).

Zolgensma

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • prescriber is a neuromuscular specialist; and
  • member is < two years of age; and
  • genetic test confirming diagnosis of bi-allelic mutation in the SMN1 gene; and
  • genetic test confirming the member has two or three copies of the SMN2 gene; and
  • baseline AAV9 antibody test confirming titers < 1:50; and
  • member does not have evidence of complete paralysis of limbs; and
  • member does not have evidence of permanent ventilator dependence (defined as any of the following: member has an endotracheal tube, member has a tracheotomy tube, member had at least 14 days of continuous respiratory assistance for at least 16 hours per day) at the time the requested agent is to be administered.