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CurrentMedStar Family Choice, Inc.Policy N/A
High-Cost Medication Prior Authorization Criteria
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This policy governs prior authorization, initial approval, renewal, documentation, dosing, quantity, and duration requirements for high-cost medications covered by MedStar Family Choice.
No material clinical or coverage changes in this revision.
3 monthsCommon approval duration
12 monthsSelected therapy approval duration
1 courseLifetime treatment limit
26 dosesVyjuvek dose limit
≥50% reductionHAE continuation response
Medication Coverage Criteria
Initial, Continuation, and Renewal Criteria
Covered when the applicable medication-specific initial, continuation, or renewal criteria below are met and required clinical documentation is submitted.
Adzynma — initial therapy
Ordered for prophylactic or on-demand enzyme replacement therapy for congenital (hereditary) thrombotic thrombocytopenic purpura (cTTP) in an adult or pediatric patient.
Patient is age 2 years or older.
cTTP is confirmed by molecular genetic testing showing an ADAMTS13 gene mutation and ADAMTS13 activity less than 10% of normal.
Chart notes are dated within the previous 1 month.
LDH is less than 2 times the laboratory upper limit of normal.
On-demand ERT is prescribed.
Ordered by, or in consultation with, a hematologist.
Adzynma — continuation therapy
Patient meets the initial Adzynma criteria.
Documentation demonstrates improved clinical response, such as fewer acute or subacute TTP events or manifestations, less supplemental therapy, increased platelet count, or decreased LDH.
Patient has not experienced treatment-restricting adverse effects.
Dosing is consistent with FDA labeling or supported by compendia.
Chart notes are updated within the previous 3 months.
Nexviazyme therapy
Ordered for treatment of late-onset Pompe disease due to lysosomal acid alpha-glucosidase (GAA) deficiency.
Patient is age 1 year or older.
Diagnostic confirmation
Laboratory testing demonstrates deficient acid alpha-glucosidase activity in a dry blood spot or fibroblasts.
Prescribed by, or in consultation with, a geneticist, neurologist, metabolic-disorder subspecialist, or physician specializing in lysosomal storage disorders.
Quantity does not exceed 100 mg powder for injection, 23 vials every 14 days; maximum 2,300 mg every 14 days.
Nexviazyme — renewal therapy
Initial criteria were met.
Chart notes document a positive response, including improvement, stabilization, or slowing of progression in motor function, walking capacity, respiratory function, or muscle strength.
Vyjuvek — initial therapy
Ordered for treatment of wounds in a patient with dystrophic epidermolysis bullosa (DEB).
Patient is age 6 months or older.
Genetic testing confirms a mutation in the collagen type VII alpha 1 chain (COL7A1) gene.
Patient has at least one recurrent or chronic open wound with adequate granulation tissue, excellent vascularization, and no active wound infection.
There is no evidence or history of squamous cell carcinoma.
Prescribed by, or in consultation with, a dermatologist experienced in DEB treatment.
Dosing is consistent with FDA-approved labeling.
Vyjuvek — continuation therapy
Patient has previously been treated with Vyjuvek.
Patient demonstrates a positive clinical response, including decreased wound size or improved wound healing.
Adequate granulation tissue and excellent vascularization are present.
There is no active wound infection or evidence or history of squamous cell carcinoma.
Dosing is consistent with FDA-approved labeling.
HAE prophylaxis — initial therapy
Ordered for routine prophylaxis to prevent hereditary angioedema (HAE) attacks in a patient age 12 years or older.
C1-inhibitor abnormality
C1-inhibitor antigenic level is below the lower limit of normal.
C1-inhibitor antigenic level is normal but functional C1-inhibitor is less than 50% or below the laboratory lower limit of normal.
Prescriber attests that baseline evaluations have been completed, prophylaxis is medically necessary, and there are no contraindications.
Patient is not receiving another approved HAE-prophylaxis product, including Cinryze, Haegarda, or Takhzyro.
Prior prophylaxis failure
Documented failure of both Haegarda and Takhzyro.
Alternatively, documented failure of one of Haegarda, Cinryze, or Takhzyro.
Quantity does not exceed 1 capsule per day.
Prescribed by a hematologist, immunologist, or allergist.
HAE prophylaxis — continuation therapy
Member meets the initial approval criteria.
Frequency of HAE attacks has decreased by at least 50% since treatment began.
Use of medications for acute attacks has decreased since treatment began.
Prescriber attests that the patient received an annual evaluation for the continued need for long-term prophylaxis.
Prescriber attests that current medications were recently reviewed and interaction-related dose adjustments were made when applicable.
Patient is not using another approved HAE-prophylaxis product concomitantly.
Adcetris — initial therapy
Prescribed for an FDA-approved indication, including one of the following: previously untreated stage III or IV classical Hodgkin lymphoma in combination with doxorubicin, vinblastine, and dacarbazine; high-risk classical Hodgkin lymphoma consolidation after autologous HSCT; classical Hodgkin lymphoma after failure of autologous HSCT or at least two prior multi-agent regimens when not an autologous-HSCT candidate; previously untreated systemic anaplastic large-cell lymphoma or other CD30-expressing peripheral T-cell lymphoma in combination with cyclophosphamide, doxorubicin, and prednisone; systemic anaplastic large-cell lymphoma after failure of at least one prior multi-agent regimen; primary cutaneous anaplastic large-cell lymphoma or CD30-expressing mycosis fungoides after prior systemic therapy; or relapsed/refractory large B-cell lymphoma in combination with lenalidomide and rituximab.
The indication is FDA-approved and the requested regimen is clinically appropriate.
Adcetris — continuation therapy
Clinical documentation is submitted for a date of service within the previous 3 months.
There is no unacceptable toxicity or disease progression.
Crysvita — initial therapy
Approved indication
Treatment of X-linked hypophosphatemia (XLH) in an adult or pediatric patient age 6 months or older.
Treatment of FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with a phosphaturic mesenchymal tumor that cannot be curatively resected or localized, in a patient age 2 years or older.
Diagnostic evidence
Genetic testing confirms a pathogenic or likely pathogenic PHEX variant.
FGF23 is elevated above the laboratory reference range.
Pretreatment tubular reabsorption of phosphate corrected for GFR (TmP/GFR) is below the normal range for age and sex.
XLH-specific criteria
FGF23 is greater than 30 pg/mL.
Serum phosphate is less than 3.0 mg/dL (0.97 mmol/L).
Serum creatinine is below the age-adjusted upper limit of normal.
Serum 25(OH)D is at least 16 mg/mL.
ANY of the following
Epiphyseal plates have not fused.
ALL of the following
Epiphyseal plates have fused.
TIO-specific criteria
Disease cannot be curatively resected or localized.
Patient is age 2 years or older.
There has been failure, contraindication, or intolerance to a vitamin D analog in combination with an oral phosphate agent.
Dosing is consistent with FDA-approved labeling.
Prescribed by, or in consultation with, an endocrinologist, geneticist, nephrologist, or specialist in XLH or TIO.
Crysvita — renewal therapy
Patient has previously received burosumab.
Patient demonstrates a positive clinical response, such as enhanced height velocity, improvement in skeletal deformities, reduction in fractures, or reduced generalized bone pain.
Dosing is consistent with FDA-approved labeling.
Cinryze/Haegarda — initial therapy
Ordered for routine prophylaxis to prevent HAE attacks in a patient age 6 years or older.
Confirmed HAE diagnosis
A monoallelic mutation known to cause HAE is confirmed in SERPING1 or F12.
C4 level is below the laboratory lower limit of normal and supporting C1-inhibitor testing confirms HAE.
Prescribed by, or in consultation with, an appropriate specialist.
Cinryze/Haegarda — continuation therapy
All initial-therapy criteria remain met.
Clinical documentation is dated within the previous 3 months.
Provider attests to a positive clinical response.
Cabometyx — initial therapy
Approved indication
Advanced renal cell carcinoma.
First-line advanced renal cell carcinoma in combination with nivolumab.
Hepatocellular carcinoma previously treated with sorafenib.
Locally advanced or metastatic differentiated thyroid cancer in a patient age 12 years or older that progressed after prior VEGFR-targeted therapy and is radioactive iodine-refractory or ineligible for radioactive iodine.
Indication-specific criteria for non-small-cell lung cancer, including a positive RET rearrangement and recurrent, advanced, or metastatic disease.
Indication-specific criteria for hepatocellular carcinoma, including appropriate Child-Pugh A status and contraindication, failure, or intolerance to sorafenib, unresectable disease, metastatic disease, extensive tumor burden, or inoperable liver-confined disease.
Osteosarcoma that has progressed on prior treatment and is relapsed/refractory or metastatic.
For patients younger than 19 years, the applicable pediatric indication criteria are met.
Medication is ordered by an oncologist.
Cabometyx — continuation therapy
Clinical documentation is dated within the previous 3 months.
There is no evidence of disease progression while receiving Cabometyx.
HAE and Cabometyx — initial therapy
The requested medication is being used for an approved HAE prophylaxis indication or an approved Cabometyx oncology indication.
All medication-specific initial criteria, including diagnosis, age, specialist prescribing, required laboratory findings, prior therapy, and absence of contraindications, are met.
Required chart notes and prescriber attestations are submitted.
HAE and Cabometyx — renewal therapy
The applicable initial criteria continue to be met.
Clinical documentation is submitted within the required review period.
There is documented clinical benefit and no unacceptable toxicity or disease progression.
Cablivi — initial therapy
Ordered for treatment of adult acquired thrombotic thrombocytopenic purpura (aTTP).
Diagnosis of aTTP is confirmed.
Cablivi is administered with plasma exchange and immunosuppressive therapy.
Cablivi will not continue beyond 30 days after cessation of plasma exchange unless persistent aTTP is documented.
The patient has not experienced more than two aTTP recurrences while receiving the medication; a 28-day extension is not a recurrence.
Ordered by, or in consultation with, a hematologist.
Cablivi — continuation therapy
The request extends therapy beyond the initial Cablivi course.
Persistent underlying aTTP is documented by ADAMTS13 activity less than 10% or by microangiopathic hemolytic anemia with schistocytes, thrombocytopenia, and elevated LDH.
Cablivi continues in combination with immunosuppressive therapy.
The patient has not already received a prior 28-day extension after the initial course.
The patient has not experienced more than two aTTP recurrences while receiving Cablivi.
Brineura — initial therapy
Ordered to slow loss of ambulation in a symptomatic pediatric patient age 3 years or older with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as TPP1 deficiency.
Molecular analysis detects two pathogenic TPP1/CLN2 variants or mutations.
Clinical Scoring System for LINCL-4 shows a combined motor and language score of 3 to 6, with at least 1 point in each domain.
Ambulatory function can be preserved; the patient is not immobile.
Prescribed by, or in consultation with, a neurologist experienced in CLN2 treatment.
Brineura — renewal therapy
Motor-domain rating on the CLN2 Clinical Rating Score has remained stable or has not declined from baseline.
Patient remains ambulatory and has motor function that can be preserved.
Patient is not receiving other antisense or gene therapy.
Provider attests to continued benefit without adverse drug effects.
Dose does not exceed 30 mg/kg per week.
Benefix — initial therapy
Ordered for on-demand treatment and control of bleeding episodes or perioperative management of bleeding in adults or children with hemophilia B, or routine prophylaxis in a patient age 16 years or older.
Congenital factor IX deficiency is confirmed by blood coagulation testing.
Benefix is not prescribed for immune-tolerance induction.
Routine prophylaxis criteria
Severe hemophilia B with factor IX level less than 1%.
At least two documented spontaneous joint-bleeding episodes.
Benefix — renewal therapy
Patient continues to meet all initial criteria.
There is no unacceptable toxicity or development of neutralizing inhibitors.
Any dose increase is supported by weight gain, half-life study results, or increased breakthrough bleeding despite adherence.
Cumulative medication on hand does not exceed five doses.
Eculizumab (Soliris) — initial therapy
Approved indication
Paroxysmal nocturnal hemoglobinuria (PNH) in an adult or pediatric patient age 1 month or older, with flow-cytometric confirmation of PNH type III red cells and transfusion history, major thrombotic vascular events, or high disease activity.
Atypical hemolytic uremic syndrome (aHUS) in an adult or pediatric patient age 1 month or older, with common causes ruled out, including infection and TTP, and evidence of hemolysis, thrombocytopenia, anemia, and renal impairment or dialysis.
Generalized myasthenia gravis (gMG) in an adult who is anti-AChR antibody positive, MGFA class II, III, or IV, has MG-ADL score at least 6, and meets the required prior-treatment criteria.
Neuromyelitis optica spectrum disorder (NMOSD) in an FDA-approved age group, with neurologist-confirmed optic neuritis, acute myelitis, area postrema syndrome, acute brainstem syndrome, or symptomatic cerebral syndrome with typical lesions; positive AQP4-IgG; multiple sclerosis and other diagnoses ruled out; prior rituximab failure, intolerance, or contraindication; and the required relapse history.
Recommended vaccinations are administered at least 2 weeks before the first Soliris dose when clinically appropriate.
Soliris is not used with another medication in the same class, such as Ultomiris.
Medication is ordered by a hematologist, nephrologist, neurologist, or other appropriate specialist.
Eculizumab (Soliris) — renewal therapy
Clinical documentation demonstrates that Soliris is providing clinical benefit.
Indication-specific response
PNH: LDH has decreased from pretreatment baseline; patient is age 18 years or older; and there is no dual therapy with Empaveli, Fabhalta, or Ultomiris.
aHUS: LDH has decreased from pretreatment baseline, STEC-HUS is absent, and the patient is not receiving Ultomiris concurrently.
gMG: at least a 2-point improvement and maintenance of improvement in MG-ADL from baseline, without treatment failure or prohibited concomitant therapy.
NMOSD: relapses or signs and symptoms have decreased and baseline immunosuppressive therapy has been maintained, reduced, or discontinued.
Ordered for treatment of adult patients with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis, as monotherapy or with glucocorticoids for glucocorticoid-resistant or glucocorticoid-dependent disease.
Patient is age 18 years or older.
MGFA Clinical Classification is class II, III, or IV at initiation of therapy.
MG-ADL total score is at least 5.
A positive serologic test for anti-AChR antibodies is documented.
Clinical documentation is dated within the previous 3 months.
Patient continues to meet all initial approval criteria.
There is no toxicity from the drug.
MG-ADL total score has improved by at least 2 points from baseline and the improvement has been sustained for at least 4 weeks.
Muscle strength testing with fatigue maneuvers shows improvement compared with baseline.
Efgartigimod alfa and hyaluronidase (Vyvgart Hytrulo) — initial therapy
Ordered for treatment of adult patients with anti-AChR antibody-positive generalized myasthenia gravis, as monotherapy or with glucocorticoids for glucocorticoid-resistant or glucocorticoid-dependent disease.
Patient is age 18 years or older.
MGFA Clinical Classification is class II, III, or IV at initiation of therapy.
MG-ADL total score is at least 5.
A positive serologic test for anti-AChR antibodies is documented.
For the applicable Hytrulo criteria, more than 50% of the baseline MG-ADL score is attributable to nonocular symptoms.
Efgartigimod alfa and hyaluronidase (Vyvgart Hytrulo) — continuation or renewal therapy
Clinical documentation is dated within the previous 3 months.
Patient continues to meet the applicable initial approval criteria.
There is no toxicity from the drug.
MG-ADL total score has improved by at least 2 points from baseline and the improvement has been sustained for at least 4 weeks.
Muscle strength testing with fatigue maneuvers shows improvement compared with baseline.
For continuous-treatment renewal, the patient requires ongoing treatment and more than 50% of baseline MG-ADL impairment is attributable to nonocular symptoms when applicable.
Coverage requires prior authorization and submission of the applicable clinical documentation, laboratory results, diagnostic testing, treatment history, prescriber specialty, and current chart notes specified above. When a criterion requires documentation within a stated period, records outside that period may be insufficient for approval.
Medications are not covered for non-FDA-approved or otherwise unproven indications unless the applicable criterion expressly allows compendia-supported use. Indications not meeting the stated approval criteria are considered experimental, investigational, or not medically necessary.
Prohibited or restricted concomitant therapy includes duplicate products in the same therapeutic class and medication-specific combinations identified above, including concurrent HAE prophylaxis products; Soliris with Ultomiris, Empaveli, Fabhalta, or specified disease-modifying, anti-IL6, or B-cell-depletion therapies; and therapies specifically excluded for Brineura, Benefix, Vyjuvek, Crysvita, or other listed medications.
Approval is subject to medication-specific quantity limits, dosing requirements, treatment-course limits, maximum lifetime courses where stated, and approval durations. Dose escalation or continued therapy requires the clinical rationale and response documentation specified in the applicable criteria.
Contraindications and denial risks include unacceptable toxicity, active infection where prohibited, disease progression when continuation requires stability, treatment-restricting adverse effects, absence of documented response, prohibited prior therapy, prohibited concomitant therapy, and failure to meet required age, diagnostic, laboratory, specialist, or vaccination requirements.
Initial Approval Requirements
Initial Approval Criteria
Initial approval is covered only when all applicable criteria are met; where an indication list is shown, one indication must be satisfied.
Adzynma: Ordered for prophylactic or on-demand enzyme replacement therapy for congenital TTP; patient is at least 2 years old; ADAMTS13 gene mutation and activity below 10% of normal are documented; chart notes are within the previous month; and applicable prophylactic laboratory thresholds are met.Age ≥2 years; ADAMTS13 activity <10% of normal; platelets >100,000/µL and LDH <2×ULN for prophylaxis
Lumizyme: Late-onset Pompe disease is supported by acid alpha-glucosidase enzyme assay or genetic testing; patient is at least 8 years old; there is no cardiac hypertrophy; and a metabolic specialist or biochemical geneticist prescribes or consults.Age ≥8 years
Xyntha: Hemophilia A requires treatment for control or prevention of bleeding, the patient does not have von Willebrand disease, and one qualifying factor-product failure, hypersensitivity, or high-inhibitor-risk pathway is documented.Three recombinant factor trials or high inhibitor risk
Vyjuvek: The patient has dystrophic epidermolysis bullosa, is at least 6 months old, has a COL7A1 mutation, and has at least one recurrent or chronic open wound with adequate granulation tissue and excellent vascularization without active infection; there is no evidence or history of squamous cell carcinoma; a DEB-experienced dermatologist prescribes or consults; and dosing follows FDA labeling.Age ≥6 months; maximum 6 months and 26 doses
Zynteglo: The patient is 5 through 50 years old with transfusion-dependent beta-thalassemia confirmed by HBB genetic testing or qualifying hematologic findings; at least 8 pRBC transfusions per year occurred in each of the preceding 2 years; specified cardiac, hepatic, infectious, transplant, gene-therapy, and malignancy exclusions are absent; and a hematologist or transplant specialist prescribes.Age 5–50 years; ≥8 pRBC transfusions/year in each of prior 2 years
Blincyto or Adcetris approved oncology indication: Blincyto is used for B-cell precursor ALL in first or second complete remission with MRD at least 0.1% or for relapsed/refractory disease, with FDA-approved indication and CD19 confirmation; or Adcetris is used for one of the listed approved Hodgkin lymphoma, anaplastic large cell lymphoma, peripheral T-cell lymphoma, mycosis fungoides, or relapsed/refractory large B-cell lymphoma indications.Blincyto MRD ≥0.1% for the remission pathway
Crysvita: The patient meets the XLH or TIO indication, has objective diagnostic evidence such as a PHEX variant, elevated FGF23, or low TmP/GFR, meets applicable laboratory and age requirements, has failed, cannot tolerate, or has a contraindication to vitamin D analog plus oral phosphate therapy when required, and has appropriate specialist prescribing or consultation.XLH age ≥6 months; TIO age ≥2 years; XLH FGF23 >30 pg/mL and phosphate <3.0 mg/dL
Cinryze or Haegarda: The patient requires routine HAE prophylaxis and has qualifying hereditary angioedema evidence by SERPING1 or F12 mutation, low C4, or specified C1-inhibitor abnormality. Cinryze applies to patients at least 6 years old.Cinryze age ≥6 years
Cabometyx: The patient has advanced RCC; first-line advanced RCC treated with nivolumab; HCC previously treated with sorafenib or meeting a specified Child-Pugh A exception; or locally advanced/metastatic DTC after prior VEGFR-targeted therapy with radioactive iodine-refractory or qualifying disease.DTC age ≥12 years
Cablivi: The adult patient has acquired TTP, receives Cablivi with plasma exchange and immunosuppressive therapy, has no more than two aTTP recurrences while receiving the medication, and treatment is ordered by or in consultation with a hematologist.Initial approval 30 days
Amondys 45: DMD is confirmed with a mutation amenable to exon 45 skipping; corticosteroid therapy is stable for at least 24 weeks; the patient is not ventilator dependent and is not receiving prohibited antisense or gene therapy; monitoring is attested; and a DMD-experienced pediatric neurologist prescribes or consults.30 mg/kg/dose once weekly
Brineura: The symptomatic pediatric patient has CLN2/TPP1 deficiency confirmed by enzyme activity or two pathogenic variants, is at least 3 years old, has combined motor and language score 3–6 with each domain at least 1, remains ambulatory, and has neurologist involvement.Maximum 30 mg/kg/dose weekly
Carvykti: The adult patient has relapsed or refractory multiple myeloma after at least four prior lines including a proteasome inhibitor, immunomodulatory agent, and anti-CD38 antibody; has no prohibited prior therapy or clinical condition; and receives single-agent therapy.One dose up to 100 million autologous CAR-positive viable T cells; one course per lifetime
Pombiliti: The adult patient has late-onset Pompe disease, weighs at least 40 kg, is not improving on current ERT, has diagnostic enzyme or genetic support, and will receive Pombiliti with Opfolda.Age ≥18 years; weight ≥40 kg
Benefix: Congenital factor IX deficiency is confirmed by coagulation testing. Benefix is used for bleeding control, perioperative management, or qualifying prophylaxis; immune tolerance induction is excluded. Prophylaxis requires factor IX below 1% or at least two spontaneous joint bleeds.Factor IX <1% or ≥2 spontaneous joint bleeds; long-term prophylaxis 100 IU/kg once weekly
Procysbi: The patient has nephropathic cystinosis, is at least 1 year old, has leukocyte cystine assay or genetic support, and will not receive Procysbi with Cystagon.Age ≥1 year
Deflazacort: The patient has DMD, is biologically male and at least 2 years old, used prednisone or prednisolone equivalent continuously for at least 6 months within the prior 2 years with uncontrolled weight gain, has neurologist involvement, and uses the specified dose.0.9 mg/kg once daily
Elevidys: The patient has DMD, is 4 or 5 years old, has an eligible mutation not involving deletion of exon 8 or 9, is ambulatory without an assistive device, has anti-AAVrh74 titer below 1:400, has never received Elevidys, and will not receive exon-skipping therapy concomitantly or afterward.1.33×10^14 vector genomes/kg; one lifetime course
Dinutuximab: The patient is younger than 18 years and has high-risk neuroblastoma with at least a partial response to prior first-line multiagent, multimodality therapy; treatment is combined with GM-CSF, IL-2, and 13-cis-retinoic acid.Age <18 years; one treatment per lifetime; no longer than 30 days from approval or until age 6, whichever is first
Soliris indication: The requested use is for PNH, aHUS, adult anti-AChR-positive generalized myasthenia gravis, or NMOSD, with all applicable indication-specific diagnostic, laboratory, age, prior-treatment, specialist, vaccination, and combination-therapy requirements met.
Vyvgart or Vyvgart Hytrulo: The adult patient has AChR-positive generalized myasthenia gravis treated as monotherapy or with glucocorticoids for glucocorticoid-resistant or glucocorticoid-dependent disease; is at least 18 years old; has MGFA Class II, III, or IV at initiation; has MG-ADL at least 5; and has positive anti-AChR serology where required for the product.Age ≥18 years; MGFA II–IV; MG-ADL ≥5; positive anti-AChR serology
Vyvgart Hytrulo additional pathway: For the specified Vyvgart Hytrulo pathway, more than 50% of the baseline MG-ADL score is attributable to non-ocular symptoms.>50% of baseline MG-ADL score due to non-ocular symptoms
Renewal and Continuation Requirements
Renewal and Continuation Criteria
Renewal or continuation requires all criteria applicable to the requested medication and indication.
Adzynma renewal: Initial criteria remain met; improved clinical response is documented; no treatment-restricting adverse effects have occurred; dosing is consistent with labeling or supported by compendia; and updated chart notes are submitted.Chart notes within previous 3 months
Nexviazyme renewal: Initial criteria remain met and chart notes document improvement, stabilization, or slowed progression in motor function, walking capacity, respiratory function, or muscle strength.Approval duration 3 months
Vyjuvek renewal: The patient was previously treated and had a positive response; wound granulation, vascularization, infection, and squamous-cell-carcinoma criteria remain satisfied; and dosing follows FDA labeling.No more than 26 doses and 6 months
HAE prophylaxis renewal: Attack frequency has decreased by at least 50%; acute-attack medication use has decreased; annual continued-need evaluation is completed; and current medications and interactions have been reviewed and managed.≥50% reduction in attack frequency; 1 capsule per day
Adcetris renewal: Clinical documentation shows benefit without unacceptable toxicity or disease progression.Approval duration 3 months
Crysvita renewal: The patient previously received burosumab, has documented positive clinical response, and receives FDA-labeled dosing.Authorization up to 12 months
Cinryze or Haegarda renewal: All initial criteria remain met; chart notes are dated within the previous 3 months; and the provider attests to positive clinical response.Chart notes within previous 3 months
Cabometyx renewal: Chart notes are dated within the previous 3 months and the patient does not show evidence of disease progression while receiving Cabometyx.Approval duration 12 months
Cablivi continuation: Persistent underlying aTTP is documented by ADAMTS13 activity below 10% or the specified MAHA, schistocyte, thrombocytopenia, and elevated-LDH findings; treatment continues with immunosuppressive therapy; no prior 28-day extension has been received; and there have been no more than two recurrences.ADAMTS13 activity <10% or specified persistent-aTTP findings
Brineura continuation: The motor domain rating remains stable or has not declined from baseline; the patient remains ambulatory with preservable motor function; and continued benefit without adverse drug events is documented.Approval duration up to 12 months
Pombiliti renewal: Documentation from the previous 3 months shows positive response, stabilization, or slowed progression in motor function, walking capacity, respiratory function, or muscle strength; initial criteria remain met; and Pombiliti continues with Opfolda.Documentation within previous 3 months
Benefix renewal: Initial criteria remain met; there is no unacceptable toxicity or development of neutralizing inhibitors; dose increases have acceptable clinical rationale; and cumulative medication on hand does not exceed five doses.Maximum five doses on hand; renewal duration 3 months
Procysbi renewal: Chart notes from the previous 3 months document positive response, stabilization, or slowed progression in renal function, leukocyte cystine concentration, or growth, and absence of hypersensitivity or other adverse reaction.Documentation within previous 3 months
Deflazacort renewal: Current weight, diagnosis, age, and prescriber specialty are verified; an Emflaza claim occurred within the previous 90 days; and the prescriber attests to clinical improvement, disease stabilization, or significant limitation of disease progression.Approval duration 6 months
Soliris PNH renewal: The patient is at least 18 years old, serum LDH has decreased from pretreatment baseline, and dual therapy with another PNH PA medication is not used.Decrease in serum LDH from pretreatment baseline
Soliris aHUS renewal: Serum LDH has decreased from pretreatment baseline, the patient does not have STEC-HUS, and dual therapy with another aHUS PA medication is not used.Decrease in serum LDH from pretreatment baseline
Soliris gMG renewal: The patient is at least 18 years old, maintains at least a 2-point reduction in MG-ADL from pretreatment baseline, and has reduced MG signs and symptoms. Escalation of immunosuppressive therapy or additional rescue therapy for worsening is treatment failure.≥2-point MG-ADL reduction
Soliris NMOSD renewal: Documentation shows reduced number or severity of NMOSD relapses or symptoms and maintenance, reduction, or discontinuation of baseline immunosuppressive therapy. Prohibited combination therapies are not used.
Vyvgart renewal: Chart notes have a date of service within the previous 3 months; initial criteria remain met; there is no drug toxicity; and clinical improvement is documented, including at least a 2-point reduction from baseline in MG-ADL sustained for at least 4 weeks where specified.Documentation within previous 3 months; MG-ADL reduction ≥2 points sustained ≥4 weeks
Vyvgart or Vyvgart Hytrulo renewal: Initial criteria remain met; there is no drug toxicity; MG-ADL has improved by at least 2 points from baseline and remained improved for at least 4 weeks; and muscle-strength testing with fatigue maneuvers has improved compared with baseline.MG-ADL reduction ≥2 points sustained ≥4 weeks
Vyvgart Hytrulo continued-treatment pathway: The patient continues to meet the documented disease, age, MGFA, MG-ADL, and anti-AChR criteria; more than 50% of the baseline MG-ADL score is attributable to non-ocular symptoms where this pathway applies; there is no drug toxicity; MG-ADL improvement of at least 2 points is sustained for at least 4 weeks; muscle-strength testing improves with fatigue maneuvers; and continuous treatment remains necessary where specified.>50% non-ocular baseline MG-ADL where required; MG-ADL reduction ≥2 points sustained ≥4 weeks
The source truncates the final factor in portions of this criterion; no additional factor is inferred.
ADAMTS13 activity< 10% of normal activity for cTTP confirmation; persistent aTTP may also be documented with activity < 10%.
Hematologic thresholdsPlatelet count > 100,000/µL and LDH < 2 times the ULN for prophylactic Adzynma therapy; aHUS requires hemoglobin < 10 g/dL, platelets 150,000/mm3, and documented hemolysis.
Naïve T-cell count< 50 naïve T cells/mm3 or < 5% of total T cells having a naïve phenotype for congenital athymia.
Treatment responseHAE prophylaxis continuation requires ≥ 50% reduction in attack frequency; gMG continuation requires at least a 2-point reduction in MG-ADL from baseline, sustained for at least 4 weeks where specified.
Patient ageMinimum ages include ≥ 18 years for specified Vyvgart and Vyvgart Hytrulo criteria; other medication-specific thresholds include ≥ 2 years for Adzynma, ≥ 8 years for Lumizyme, and ≥ 12 years for differentiated thyroid cancer treated with Cabometyx.
Disease classification and baseline scoreMGFA Clinical Classification must be Class II, III, or IV at initiation; several Vyvgart and Vyvgart Hytrulo criteria require a baseline MG-ADL total score ≥ 5.
Documentation recencyRenewal clinical documentation or chart notes generally must have a date of service within the previous 3 months; initial Adzynma chart notes must be within the previous 1 month.
Required Prior Therapy
Medication
Required trial or prerequisite
Failure, sequencing, or timing requirement
Xyntha
Trial of three specified recombinant factor VIII products: Advate, Kogenate FS, Kovaltry, NovoEight, Nuwiq, or Recombinate, as applicable.
Document failure to meet clinical goals or hypersensitivity after the three-product trial. The high inhibitor-risk pathway may apply instead, with immune tolerance therapy when specified.
Hereditary angioedema prophylaxis medication
Document failure of both Haegarda and Takhzyro; alternatively, document failure of one of Haegarda, Cinryze, or Takhzyro.
The medication must not be used in combination with another approved HAE prophylactic product, including Cinryze, Haegarda, or Takhzyro.
Crysvita
For applicable XLH or TIO pathways, use vitamin D analog therapy, such as calcitriol, paricalcitol, or doxercalciferol, in combination with an oral phosphate agent.
Document failure, contraindication, or intolerance to the prerequisite therapy. For XLH with a fused epiphyseal plate, this requirement applies along with qualifying clinical symptoms.
Cabometyx — renal cell carcinoma
Cabometyx may be used with nivolumab as first-line therapy for advanced renal cell carcinoma.
No prior Cabometyx step requirement is stated for this first-line combination pathway.
Cabometyx — hepatocellular carcinoma
Prior sorafenib (Nexavar) therapy is required unless a specified Child-Pugh A disease circumstance applies.
Document contraindication, failure, or intolerance to sorafenib, or document the applicable qualifying Child-Pugh A circumstance.
Cabometyx — differentiated thyroid cancer
Prior VEGFR-targeted therapy is required.
Disease must have progressed after prior VEGFR-targeted therapy and be radioactive iodine-refractory or otherwise ineligible for radioactive iodine.
Pombiliti
Trial of current or prior first-line enzyme replacement therapy, such as Lumizyme or Nexviazyme.
Document that the patient is not improving on current ERT. Pombiliti must be administered in combination with Opfolda (miglustat).
Deflazacort (Emflaza)
Continuous prednisone or prednisolone-equivalent treatment for at least 6 months within the previous 2 years.
Document uncontrolled weight gain as the adverse effect prompting the change to deflazacort.
Elevidys
Do not use Amondys 45, Exondys 51, Viltepso, or Vyondys 53 with Elevidys.
Exon-skipping therapies may not be administered concomitantly with or after Elevidys treatment.
Soliris (eculizumab) — gMG or NMOSD
For gMG, document prior failure of Soliris or Ultomiris and, as applicable, failure of immunosuppressive therapy or inadequate response despite plasmapheresis/plasma exchange and/or IVIG. For NMOSD, document failure, contraindication, or intolerance to rituximab.
For gMG, failure may include failure of at least two immunosuppressive agents or failure of one immunosuppressive therapy with at least four plasmapheresis/plasma exchange and/or IVIG courses without symptom control. For NMOSD, the required relapse history must also be documented.
Dosing, Duration, and Lifetime Limits
Quantity limits, dose limits, and approval durations
Nexviazyme100 mg powder for injection; 23 vials every 14 days; maximum 2,300 mg every 14 days.
HAE prophylaxis medication110 mg or 150 mg; quantity limit of 1 capsule per day.
VyjuvekNo more than 26 doses; initial and renewal authorization is limited to no more than 6 months.
Amondys 45 and BrineuraAmondys 45: 30 mg/kg/dose once weekly. Brineura: 30 mg/kg/dose weekly.
CarvyktiOne dose of up to 100 million autologous CAR-positive viable T-cells; limited to one treatment course per lifetime.
Benefix and deflazacortBenefix long-term prophylaxis: 100 IU/kg once weekly, with cumulative medication on hand not exceeding five doses. Deflazacort/Emflaza: 0.9 mg/kg once daily.
Approval durations and product strengthSoliris: 6 months (5 therapy cycles). Vyvgart: 3 months. Vyvgart Hytrulo: 400 mg/20 mL injection.
Prior Authorization and Documentation Requirements
Prior Authorization
Prior Authorization, Specialist, REMS, and Submission Requirements
Prior authorization is required for high-cost and specialty medications listed in this policy, including medication-specific, initial, renewal, Vyvgart, and Vyvgart Hytrulo requests. Submit the applicable clinical criteria, specialist-ordering information, REMS confirmations, step-therapy history, and current records through the required internal workflow.
High-cost medication PA required; medication-specific PA required; medication-specific prior authorization required; prior authorization required before dispensing or administration.
Submit separate initial prior authorization and renewal prior authorization requests with the applicable approval and continuation criteria.
Vyvgart prior authorization and initial Vyvgart authorization require documentation of adult AChR-positive generalized myasthenia gravis, age ≥18 years, MGFA class II, III, or IV at treatment initiation, MG-ADL score ≥5, positive anti-AChR serology, and the applicable specialist and treatment information.
Vyvgart renewal authorization requires current chart notes dated within the previous 3 months, continued initial eligibility, absence of toxicity, at least a 2-point reduction from baseline in MG-ADL sustained for at least 4 weeks, and improvement in muscle-strength testing. Document continued benefit and safety and provide renewal response documentation.
Vyvgart Hytrulo requires prior authorization. Initial submissions must include the applicable AChR-positive gMG criteria, age, MGFA classification, MG-ADL score, anti-AChR antibodies, and—when applicable—documentation that more than 50% of baseline MG-ADL impairment is attributable to nonocular symptoms. Renewals require continued eligibility, continuous-treatment need when applicable, absence of toxicity, sustained MG-ADL response, and improved strength testing.
Yescarta REMS facility requirement: the dispensing and treatment facility must be enrolled in and comply with the Yescarta Risk Evaluation and Mitigation Strategy (REMS).
Adcetris indication review: verify that the requested use is an approved indication, including the applicable Hodgkin lymphoma, systemic anaplastic large cell lymphoma, CD30-expressing PTCL, pcALCL/MF, or relapsed/refractory large B-cell lymphoma indication, and document specialist prescribing.
Cabometyx indication review: verify the approved RCC, HCC, differentiated thyroid cancer, or other applicable FDA-labeled indication, age and disease requirements, oncology prescribing, and required prior treatment. For HCC, document prior sorafenib (Nexavar) contraindication, failure, or intolerance when applicable; Cabometyx prior sorafenib requirement applies to the applicable HCC indication.
Specialist ordering rules: document prescribing by or consultation with the required specialist, such as a hematologist, oncologist, neurologist, pediatric neuromuscular specialist, endocrinologist, geneticist, nephrologist, allergist, immunologist, dermatologist, or metabolic-disorder specialist, as specified for the medication.
REMS and gene-therapy submissions: document facility enrollment, treatment-center compliance, required genetic and antibody testing, specialist oversight, vaccination or infectious-disease screening, dosing, and lifetime-use restrictions.
Step-therapy documentation: provide trial dates, response or failure, intolerance, contraindication, adverse effects, and reason for discontinuation. This includes the recombinant factor VIII trial requirement for Xyntha; HAE prophylaxis step therapy with Haegarda, Takhzyro, or Cinryze as applicable; Crysvita prerequisite therapy with vitamin D analog plus oral phosphate when applicable; Cabometyx prior-treatment requirements; Pombiliti documentation of inadequate response to current ERT; deflazacort prior corticosteroid trial; and Soliris prior-treatment requirements.
Medication-specific submission: include the applicable diagnosis, laboratory or genetic confirmation, age, disease severity, prior treatment, current regimen, dose and quantity, prescriber specialty, baseline and follow-up testing, current chart notes, response measures, adverse-effect monitoring, and requested duration.
Denial Risk
Denial Risks and Failure Conditions
Requests may be denied when the diagnosis, indication, clinical criteria, treatment history, safety requirements, response, documentation, or renewal conditions are not established. Approval is also limited by medication-specific exclusions, prohibited concomitant therapy, quantity or duration limits, and lifetime-treatment restrictions.
Insufficient confirmation of cTTP: Adzynma requires molecular confirmation of an ADAMTS13 mutation and ADAMTS13 activity <10% of normal, with current chart notes; incomplete diagnostic confirmation or missing official laboratory results may result in denial.
Rethymic exclusion criteria: deny when SCID is not conclusively ruled out, HIV is present, the patient is a poor surgical candidate, heart surgery is anticipated within the specified period, or the patient previously received thymus-tissue transplantation. Rethymic is limited to one treatment course per lifetime.
Failure to meet indication or diagnostic requirements: deny an unapproved indication, unsupported diagnosis, incorrect age, missing FDA/compendia support, or failure to satisfy disease-specific criteria.
HAE prophylaxis criteria not met: deny when HAE is not confirmed by the required genetic, C4, or C1-INH testing; prophylaxis is not medically necessary; required attack-frequency or clinical criteria are absent; or the prescriber has not documented the evaluation and medication review.
Disease-specific criteria not satisfied: deny when required disease severity, laboratory thresholds, functional status, specialist evaluation, prior therapy, or clinical eligibility is absent.
Cablivi duration and recurrence limits: deny continuation beyond the authorized initial course or permitted 28-day extension without documented persistent aTTP; more than two recurrences, missing plasma-exchange association, absent immunosuppressive therapy, or failure to document persistent disease also precludes approval.
Administration and Site-of-Care Requirements
Billing Rule
Confirm site of care and administration requirements
Use the required administration setting and treatment process for cellular, plasma-exchange, and facility-restricted therapies. For products where the policy identifies only an intravenous or subcutaneous route and does not specify a setting, follow the documented route and applicable clinical requirements.
Yescarta must be dispensed and administered by an enrolled, REMS-compliant treatment facility.
Cablivi initial treatment must be given with plasma exchange for acquired thrombotic thrombocytopenic purpura and in combination with immunosuppressive therapy.
Carvykti requires leukapheresis, required infectious-disease screening before cell collection, and cellular-therapy administration.
Dinutuximab billing code C9399 is identified for hospital outpatient use only.
Vyvgart is administered by intravenous injection; the policy does not specify a separate administration setting in the cited sections.
Vyvgart Hytrulo is administered by subcutaneous injection; the policy identifies the subcutaneous formulation but does not impose a separate site-of-care restriction in the cited sections.
Policy Terms and Clinical Definitions
Definitions
cTTP and congenital athymiacTTP is congenital thrombotic thrombocytopenic purpura confirmed by an ADAMTS13 gene mutation and ADAMTS13 activity < 10% of normal. Congenital athymia is defined by < 50 naïve T cells/mm3 or < 5% of total T cells having a naïve phenotype, with the specified genetic or syndromic context.
Transfusion-dependent beta-thalassemia and TIOTransfusion-dependent beta-thalassemia involves regular RBC transfusions or at least 8 pRBC transfusions per year in each of the 2 years preceding therapy. TIO is FGF23-related hypophosphatemia associated with a phosphaturic mesenchymal tumor that cannot be curatively resected or localized.
B-cell precursor ALLMRD-positive or relapsed/refractory B-cell precursor ALL means first- or second-complete remission with MRD ≥ 0.1%, or relapsed or refractory disease.
Cablivi continuation and aTTP recurrenceCablivi continuation is an extension after the initial course given with plasma exchange for acquired TTP. Recurrence means the patient must reinitiate plasma exchange; a 28-day extension does not count as a recurrence. Persistent aTTP may be shown by ADAMTS13 activity < 10% or MAHA with schistocytes, thrombocytopenia, and elevated LDH.
CLN2, PNH, and aHUSCLN2 is late-infantile neuronal ceroid lipofuscinosis type 2, also called TPP1 deficiency. PNH is paroxysmal nocturnal hemoglobinuria, a complement-mediated disorder with documented PNH cells and qualifying disease activity or transfusion/thrombotic history. aHUS is atypical hemolytic uremic syndrome after common causes are ruled out, with hemolysis, thrombocytopenia, renal dysfunction, or dialysis as specified.
gMG and NMOSDgMG is generalized myasthenia gravis; applicable criteria include anti-AChR positivity, MGFA Class II-IV disease, MG-ADL requirements, and treatment-history requirements. NMOSD is neuromyelitis optica spectrum disorder supported by qualifying neurologic manifestations and positive AQP4-IgG/NMO-IgG antibodies after alternative diagnoses are excluded.
AChR-positive gMG, MGFA, and MG-ADLAChR-positive generalized myasthenia gravis is adult generalized disease with positive anti-acetylcholine receptor antibodies, treated as monotherapy or with glucocorticoids for glucocorticoid-resistant or glucocorticoid-dependent disease. MGFA Clinical Classification means Class II, III, or IV at initiation. MG-ADL is the Myasthenia Gravis-Specific Activities of Daily Living total score used for baseline impairment and response; renewal generally requires a reduction of at least 2 points from baseline sustained for at least 4 weeks. Where specified, more than 50% of baseline MG-ADL is attributable to non-ocular symptoms.
Policy Scope and Clinical Context
This policy establishes prior authorization and coverage requirements for high-cost specialty therapies used in rare inherited disorders, hematologic malignancies, cellular and gene therapies, enzyme deficiencies, hereditary angioedema, and advanced cancers. Covered use generally requires an FDA-approved or otherwise accepted indication, objective diagnostic confirmation, specialist involvement when specified, and documentation of clinical response for continuation.
Requirements may include medication-specific clinical criteria, prior treatment history, laboratory or genetic testing, dosing and quantity limits, approval duration, and renewal documentation. Providers should submit the records needed to demonstrate the requested indication and continued benefit for the specific therapy.
Clinical signs or symptoms such as limited mobility, musculoskeletal pain, or bone fractures are present.
There has been failure, contraindication, or intolerance to a vitamin D analog in combination with an oral phosphate agent.
Dosing is consistent with FDA-approved labeling.
Neuromuscular and CLN2 eligibility: deny Amondys 45 or Brineura when required genetic or enzyme confirmation, age, ambulatory or preservable motor function, clinical scores, specialist oversight, or monitoring is absent.
Carvykti eligibility and lifetime limit: deny when the patient lacks relapsed/refractory multiple myeloma after the required prior lines, is under age 18, has prior CAR-T or B-cell-maturation-antigen therapy, contraindicating disease or infection, or has already received Carvykti. Limit to one dose/treatment course as specified.
Elevidys eligibility and lifetime limit: deny when the patient is not age 4 or 5, is not ambulatory without an assistive device, lacks the required DMD mutation documentation, has an excluded exon 8/9 deletion, elevated anti-AAVrh74 antibody titer, prior Elevidys, or prohibited concomitant/follow-on exon-skipping or gene therapy. Limit to one treatment per lifetime.
Cystinosis and DMD exclusions: deny Procysbi with concomitant Cystagon; deny DMD antisense therapy when ventilator dependence, prohibited RNA antisense or gene therapy, or failure to meet the required corticosteroid trial and safety conditions is present.
Soliris indication, documentation, and combination-therapy failures: deny without the required PNH, aHUS, gMG, or NMOSD diagnosis and supporting laboratory, serologic, neurologic, or clinical documentation; deny for Shiga-toxin E. coli HUS where excluded, absent required prior-treatment history, or failure to meet relapse or response requirements.
Prohibited concomitant therapy: deny or stop approval when the medication is combined with prohibited alternatives or same-class therapies, including Soliris with Ultomiris, Empaveli, Fabhalta, certain MS disease-modifying or anti-IL-6/B-cell-depletion therapies; HAE prophylaxis products used together; Procysbi with Cystagon; Elevidys with exon-skipping or gene therapy; or other medication-specific prohibited combinations.
Vyvgart renewal failure: deny when renewal documentation is late or incomplete, initial criteria are no longer met, toxicity is present, the required MG-ADL response or muscle-strength improvement is not demonstrated, or ongoing treatment is not clinically justified.
Dinutuximab lifetime and age limit: deny outside the approved high-risk neuroblastoma indication, when the patient is age 18 or older, or after the maximum permitted treatment course.
Unapproved indication or age: deny use outside the FDA-approved indication or applicable age range, including unsupported oncology, enzyme-replacement, rare-disease, neuromuscular, or gene-therapy requests.
Failure to meet renewal criteria: deny when the patient does not continue to meet initial criteria, lacks positive response, has disease progression, unacceptable toxicity, neutralizing antibodies, or other medication-specific safety failure.
Insufficient documentation: deny incomplete submissions, missing diagnostic or functional testing, absent recent chart notes, missing prescriber attestations, or records that do not establish medical necessity.
Initial criteria not met / failure to meet initial disease criteria: deny when required diagnosis, age, severity, laboratory, genetic, prior-treatment, specialist, dosing, or exclusion criteria are absent.
Renewal response or safety criteria not met / failure to meet renewal response criteria: deny without measurable improvement, stabilization, or slowing of progression, or when toxicity, adverse reactions, progression, or lack of benefit is documented.
Failure to meet clinical criteria / failure to meet initial clinical criteria: deny when the requested medication is not medically necessary under the applicable disease-specific criteria or when the clinical record cannot support safe and effective treatment.
Failure to demonstrate safe and effective continuation: deny continuation without current monitoring, sustained benefit, acceptable safety, appropriate dosing, and required follow-up testing.
Failure to meet initial or renewal criteria / clinical eligibility not met: deny whenever any mandatory criterion is missing, contradicted, or unsupported.
Failure to meet initial approval criteria: deny when the requested therapy does not satisfy the complete initial approval pathway, including required diagnosis, prior treatment, specialist, safety, and documentation elements.
Incomplete renewal documentation: require current chart notes—generally within the previous 3 months—plus response, safety, dosing, and continued-benefit documentation before renewal.
Pompe disease diagnostic and response documentation: Nexviazyme requires confirmed late-onset Pompe disease and renewal documentation of improvement, stabilization, or slowed progression in motor, walking, respiratory, or muscle function. Pombiliti requires acid alpha-glucosidase testing, inadequate response to current ERT, combination use with Opfolda, and recent positive-response documentation.
Xyntha trial history: deny without documented failure to meet goals, hypersensitivity, high inhibitor risk, or required immune-tolerance history involving the specified recombinant factor VIII products; renewals require positive response and no von Willebrand disease use.
Vyjuvek wound and genetic documentation: require COL7A1 confirmation, recurrent or chronic open wound with adequate granulation and vascularization and no active infection, no squamous-cell-carcinoma history, dermatologist oversight, and dose and quantity compliance.
HAE prophylaxis documentation: require current C1-INH/HAE diagnostic testing, attack history or clinical necessity, prior-treatment trials and outcomes, medication-interaction review, prescriber attestation, and renewal evidence of at least 50% attack reduction and reduced acute-attack medication use.
Hematology and gene-therapy documentation: require disease confirmation, transfusion or treatment history, genetic testing, organ-function and infection screening, antibody testing, specialist notes, facility/REMS compliance, and lifetime-treatment history for applicable products.
Specialty medication documentation and recency: submit recent clinical chart notes, laboratory and genetic results, functional testing, medication history, dosing, quantity, specialist notes, and attestations; stale or incomplete records are insufficient.
Diagnostic and functional testing: deny without required enzyme, genetic, flow-cytometry, serologic, antibody, imaging, laboratory, neurologic, MG-ADL, muscle-strength, ambulatory, wound, or disease-activity testing.
Soliris diagnostic and clinical documentation: require the applicable PNH, aHUS, gMG, or NMOSD diagnosis, laboratory and serologic evidence, prior-treatment history, vaccination documentation, specialist notes, and response measures. NMOSD documentation must support the diagnosis, AQP4-IgG positivity, exclusion of multiple sclerosis, relapse history, and required treatment history.
NMOSD documentation: include neurologist confirmation of optic neuritis, acute myelitis, area-postrema syndrome, acute brainstem or symptomatic cerebral syndrome with typical lesions, positive AQP4-IgG, exclusion of alternative diagnoses, required rituximab history, and relapse frequency.
Recent clinical documentation / recent clinical chart notes: records must be dated within the medication-specific required period, commonly the previous 1 or 3 months; missing recency may cause denial.
Renewal response and safety documentation: document continued benefit, objective response, no unacceptable toxicity, no disease progression, appropriate dose, and absence of treatment-restricting adverse effects.
MGFA disease classification and anti-AChR serology: Vyvgart and Vyvgart Hytrulo requests require MGFA class II–IV at initiation and positive anti-AChR serology; failure to document either may result in denial.
Internal workflow compliance: follow MedStar Family Choice reporting, tracking, submission, and documentation procedures. Failure to complete the required workflow may prevent approval even when clinical information is otherwise favorable.
Clinical documentation for approval and renewal documentation: include the diagnosis, indication, baseline status, treatment plan, specialist assessment, response measures, safety monitoring, and continuation rationale for every request.
Recombinant factor VIII trial requirement: Xyntha requires the applicable three-product trial, hypersensitivity history, high-inhibitor-risk exception, or immune-tolerance documentation; absent evidence is a denial risk.
HAE prophylaxis step therapy: document failure, contraindication, intolerance, or inadequate response to the required HAE prophylaxis products and confirm that prohibited combinations are not being used.
Crysvita prerequisite therapy: document failure, contraindication, or intolerance to vitamin D analog therapy used with an oral phosphate agent when required for XLH or TIO.
Cabometyx prior treatment requirements: document the required prior VEGFR-targeted therapy for DTC and sorafenib history or applicable contraindication, failure, or intolerance for HCC.
Pombiliti inadequate ERT response: document that the adult patient weighing ≥40 kg is not improving on current ERT and that Pombiliti will be administered with Opfolda.
Deflazacort prior corticosteroid trial: require at least 6 months of continuous prednisone/prednisolone-equivalent use within the prior 2 years and the specified adverse effect before approval.
Soliris prior-treatment requirements: document required prior immunosuppressive, rescue, rituximab, or complement-therapy history for the applicable indication; failure to meet those requirements or prohibited combination therapy is a denial condition.