Table 45: Beta Thalassemia, Myelodysplastic Syndrome, and Sickle Cell Disease Agents
A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
Drug Category: Blood Disorder Agents
Medication Class/Individual Agents: Hematopoietic Agents
I. Prior-Authorization Requirements
Beta Thalassemia Agents - Erythroid Maturation Agents |
Clinical Notes |
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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.
1.Yawn BP, Buchanan GR, Afenyi-Annan AN, Ballas SK, Hassell KL, James AH, et al. Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members. JAMA. 2014 Sep 10;312(10):1033-48. |
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Beta Thalassemia Agents - Gene Therapy |
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Sickle Cell Disease Agents - Antimetabolites |
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Sickle Cell Disease Agents - Hemoglobin S (HbS) Polymerization Inhibitor |
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Sickle Cell Disease Agents - Not Otherwise Classified |
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Sickle Cell Disease Agents - P-Selectin Inhibitors |
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| CO | Carve-Out. This agent is listed on the Acute Hospital Carve-Out Drugs List and is subject to additional monitoring and billing requirements. |
| PD | Preferred Drug. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing a non-preferred drug within a therapeutic class. |
| MB | This drug is available through the health care professional who administers the drug or in an outpatient or inpatient hospital setting. MassHealth does not pay for this drug to be dispensed through the retail pharmacy. If listed, prior authorization does not apply through the hospital outpatient and inpatient settings. Please refer to 130 CMR 433.408 for prior authorization requirements for other health care professionals. Notwithstanding the above, this drug may be an exception to the unified pharmacy policy; please refer to respective MassHealth Accountable Care Partnership Plans (ACPPs) and Managed Care Organizations (MCOs) for prior authorization status and criteria, if applicable. |
II. Therapeutic Uses
FDA-approved, for example:
- Beta thalassemia (Reblozyl, Zynteglo)
- Myelodysplastic syndromes with ring sideroblasts or myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (Reblozyl)
- Sickle cell disease (Adakveo, Endari, Oxbryta, Siklos)
Note: The above list may not include all FDA-approved indications.
III. Evaluation Criteria for Approval
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 PA requests must include clinical diagnosis, drug name, dose, and frequency.
- Dispensing in a 90-day supply of medication may be mandated or allowable for agents in this therapeutic class (designated by M90 or A90, respectively, in the Drug Notes section above). Applicable quantity limits are described below as units per day, per month, per 28 days, or as clinically appropriate, and may be extrapolated for fills of longer day supply.
- 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 diagnosis and/or requested medication (see below). Other factors, including rebate and FDA-approval status may change independently of scheduled MassHealth updates, which may result in additional restrictions.
Adakveo
- Documentation of all of the following is required:
- diagnosis of sickle cell disease; and
- member ≥ 16 years of age; and
- prescriber is a hematologist or consult notes from a hematologist are provided; and
- member has experienced two or more sickle cell crises in the previous 12 months; and
- member's current weight; and
- inadequate response to hydroxyurea at the maximally tolerated dose for at least three months; or
- adverse reaction or contraindication to hydroxyurea.
- For recertification, documentation of positive response to therapy (e.g., decrease in VOCs, reduction in need for pain management, decrease in hospitalizations) is required.
Endari
- Documentation of all of the following is required:
- diagnosis of sickle cell disease; and
- member ≥ five years of age; and
- prescriber is a hematologist or consult notes from a hematologist are provided; and
- member has experienced two or more sickle cell crises in the previous 12 months; and
- member's current weight; and
- inadequate response, adverse reaction, or contraindication to hydroxyurea.
Oxbryta
- Documentation of all of the following is required:
- diagnosis of sickle cell disease; and
- member ≥ four years of age; and
- prescriber is a hematologist or consult notes from a hematologist are provided; and
- member has experienced at least one sickle cell crises in the previous 12 months; and
- one of the following:
- inadequate response to hydroxyurea therapy at the maximally tolerated dose for at least three months; or
- adverse reaction or contraindication to hydroxyurea; and
- member has a baseline hemoglobin level ≤ 10.5 g/dL (lab work should be drawn within the last 60 days); and
- for Oxbryta 300 mg tablet for oral suspension, medical necessity for the requested formulation as noted by one of the following:
- member is < 12 years of age; or
- member utilizes tube feeding (G-tube/J-tube); or
- member has a swallowing disorder or condition affecting ability to swallow; and
- appropriate dosing.
- For recertification, documentation of positive response to therapy (e.g., decrease in VOCs, Hb increase of at least one g/dL from baseline, reduction in laboratory markers associated with hemolysis) is required.
Reblozyl for beta thalassemia
- Documentation of all of the following is required:
- medical records and genetic testing supporting diagnosis of transfusion-dependent beta thalassemia; and
- member ≥ 18 years of age; and
- prescriber is a hematologist or consult notes from a hematologist supporting the use of the requested agent are provided; and
- member's current weight.
- For recertification, documentation of positive response to therapy (e.g., decrease in transfusion requirements) is required.
Reblozyl for myelodysplastic syndromes with ring sideroblasts or myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis
- Documentation of all of the following is required:
- appropriate diagnosis; and
- member ≥ 18 years of age; and
- prescriber is a hematologist or consult notes from a hematologist supporting the use of the requested agent are provided; and
- inadequate response or adverse reaction to one or contraindication to all erythropoiesis stimulating agents; and
- member has required red blood cell transfusions in the last eight weeks; and
- member's current weight.
- For recertification, documentation of positive response to therapy (e.g., decrease in transfusion requirements) is required.
Siklos
- Documentation of all of the following is required:
- diagnosis of sickle cell disease; and
- member ≥ two years of age; and
- prescriber is a hematologist or consult notes from a hematologist are provided; and
- member's current weight; and
- medical necessity for the requested formulation as noted by one of the following:
- member is < 13 years of age; or
- member utilizes tube feeding (G-tube/J-tube); or
- member has a swallowing disorder or condition affecting ability to swallow.
Zynteglo
- Documentation of all of the following is required:
- diagnosis of transfusion dependent beta thalassemia; and
- copy of genetic test confirming diagnosis; and
- prescriber is a hematologist or consult notes from a hematologist are provided; and
- member is ≤ 50 years of age; and
- appropriate dosing and treatment dates; and
- member does not have pre-existing HIV, HBV, or HCV infection; and
- member has required ≥ 100 mL/kg/year of pRBC or ≥ eight transfusions within the last 12 months; and
- infusion will take place in a qualified treatment center; and
- member is clinically stable and eligible for HSCT.
Original Effective Date: 09/2003
Last Revised Date: 06/2023
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Last updated 09/25/23