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.
Beta thalassemia and myelodysplastic syndromes (MDS)
Sickle Cell Disease (SCD)
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 - Gene Therapy |
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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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Telomerase Inhibitor |
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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, PA does not apply through the hospital outpatient and inpatient settings. Please refer to 130 CMR 433.408 for PA 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 PA status and criteria, if applicable. |
II. Therapeutic Uses
FDA-approved, for example:
- Beta thalassemia (Casgevy, Reblozyl, Zynteglo)
- Myelodysplastic syndromes associated anemia (Reblozyl, Rytelo)
- Sickle cell disease (Adakveo, Casgevy, l-glutamine, Lyfgenia, 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 is ≥ 16 years of age; and
- prescriber is a hematologist or consult notes from a hematologist are provided; and
- member has experienced ≥ two sickle cell crises in the previous 12 months; and
- member's current weight; and
- one of the following:
- 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.
Casgevy
- Documentation of all of the following is required for a diagnosis of sickle cell disease:
- appropriate diagnosis; and
- copy of genetic test confirming diagnosis of SCD; and
- prescriber is a hematologist or consult notes from a hematologist are provided; and
- member is ≥ 12 years of age; and
- history of ≥ two sickle cell crises per year in the last two years; 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
- appropriate dosing and treatment dates; and
- infusion will take place in a qualified treatment facility; and
- member will receive pre-infusion conditioning with busulfan; and
- member is clinically stable and eligible for HSCT; and
- member does not have active HIV, HBV, or HCV infection; and
- member has not received any prior SCD gene therapy.
- Documentation of all of the following is required for a diagnosis of transfusion-dependent beta thalassemia (TDT or beta thalassemia major):
- appropriate diagnosis; and
- copy of genetic test confirming diagnosis; and
- prescriber is a hematologist or consult notes from a hematologist are provided; and
- member is ≥ 12 years of age; and
- appropriate dosing and treatment dates; and
- member has required ≥ 100 mL/kg/year of pRBC or ≥ ten units per year in the previous two years; and
- infusion will take place in a qualified treatment facility; and
- member will receive pre-infusion conditioning with busulfan; and
- member is clinically stable and eligible for HSCT; and
- member does not have active HIV, HBV, or HCV infection; and
- member has not received any prior TDT gene therapy.
* Requests will be evaluated taking into account MassHealth pharmacy claims history or additional documentation addressing adherence to these agents.
l-glutamine
- Documentation of all of the following is required:
- diagnosis of sickle cell disease; and
- member is ≥ five years of age; and
- prescriber is a hematologist or consult notes from a hematologist are provided; and
- member has experienced ≥ two sickle cell crises in the previous 12 months; and
- member's current weight; and
- inadequate response, adverse reaction, or contraindication to hydroxyurea.
Lyfgenia
- Documentation of all of the following is required:
- diagnosis of sickle cell disease; and
- copy of genetic test confirming diagnosis of SCD; and
- prescriber is a hematologist or consult notes from a hematologist are provided; and
- member is ≥ 12 years of age; and
- history of ≥ two sickle cell crises per year in the last two years; 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
- medical necessity for use of requested agent instead of Casgevy; and
- member has a negative serology test for HIV; and
- member does not have α-thalassemia trait (-α3.7/-α3.7); and
- appropriate dosing and treatment dates; and
- infusion will take place in a qualified treatment facility; and
- member is clinically stable and eligible for HSCT; and
- member has not received any prior SCD gene therapy.
* Requests will be evaluated taking into account MassHealth pharmacy claims history or additional documentation addressing adherence to these agents.
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 is ≥ 18 years of age; and
- prescriber is a hematologist or consult notes from a hematologist 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 associated anemia
- Documentation of all of the following is required:
- appropriate diagnosis; and
- member is ≥ 18 years of age; and
- prescriber is a hematologist or consult notes from a hematologist are provided; and
- member's current weight.
- For recertification, documentation of positive response to therapy (e.g., decrease in transfusion requirements) is required.
Rytelo
- Documentation of all of the following is required:
- appropriate diagnosis; and
- member is ≥ 18 years of age; and
- prescriber is a hematologist or consult notes from a hematologist are provided; and
- appropriate dosing; and
- member has required ≥ four RBC transfusions in the last eight weeks; and
- inadequate response or adverse reaction to one or contraindication to all erythropoiesis stimulating agents (e.g., epoetin, darbepoetin); and
- if the member has MDS with ring sideroblasts (RS), inadequate response, adverse reaction, or contraindication to Reblozyl; and
- if the member has MDS associated with a del 5q cytogenic abnormality, inadequate response, adverse reaction, or contraindication to lenalidomide.
- 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 is ≥ 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 < 51 years of age; and
- appropriate dosing and treatment dates; and
- member has a negative serology test for HIV; 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; and
- member has not received any prior gene therapy for transfusion-dependent beta thalassemia.
Original Effective Date: 09/2003
Last Revised Date: 01/2025
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Last updated 02/10/25