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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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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II. Therapeutic Uses
FDA-approved, for example:
- Beta thalassemia (Reblozyl)
- Myelodysplastic syndromes with ring sideroblasts or myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (Reblozyl)
- Sickle cell disease (Adakveo, Endari, Oxbryta)
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.
- 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).
Adakveo
- Documentation of all of the following is required:
- appropriate diagnosis; 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
- an inadequate response to hydroxyurea at the maximally tolerated dose for at least three months; or
- an adverse reaction or contraindication to hydroxyurea.
- For recertification, documentation of postive 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:
- appropriate diagnosis; 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
- an inadequate response, adverse reaction, or contraindication to hydroxyurea.
Reblozyl for beta thalessemia
- 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
- appropriate dose.
- For recertification, documentation of postive 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
- one of the following:
- inadequate response or adverse reaction to one erythropoiesis stimulating agent; or
- contraindication to all erythropoiesis stimulating agents; and
- member has required red blood cell transfusions in the past eight weeks; and
- appropriate dose.
- For recertification, documentation of postive response to therapy (e.g., decrease in transfusion requirements) is required.
Oxbryta
- Documentation of all of the following is required:
- appropriate diagnosis; and
- member ≥ 12 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
- appropriate dosing; and
- member has a baseline hemoglobin level ≤ 10.5 g/dL (Lab work should be drawn within the last 60 days).
- For recertification, documentation of postive 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.
Original Effective Date: 09/2003
Last Revised Date: 12/2020
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Last updated 01/26/21