Product Details
Soliris
Eculizumab10 mg/mL (300 mg/30 mL)
Solution for Infusion
Single-Use 300-mg Vial (Preservative-Free)
DIN/PIN/NPN
02322285
Manufacturer
Alexion Pharma GmbH
Formulary Listing Date
0000-00-00
Unit Price
Amount MOH Pays
Coverage Status
Exceptional Access Program Product
ODB Formulary Therapeutic Classification
Therapeutic Note
NO
ATC Code
L04AA25
Interchangeable Products
NOLU Clinical Criteria
NOEAP Criteria
Therapeutic Class | Reimbursement Criteria |
---|---|
Blood Modifiers | Eculizumab
For the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) meeting the following criteria: The diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) has been made based on the following confirmatory results:
AND at least one of the following:
The dose of eculizumab that will be considered is: Duration of Approval: 6 months Renewals will be considered for patients who:
Requests for renewal should be accompanied by confirmation of granulocyte clone size (by flow cytometry). Further, subsidized treatment may continue unless one or more of the following situations apply:
Other eligibility requirements: Note: All patients must receive meningococcal vaccination with a tetravalent vaccine at least two weeks prior to receiving the first dose of eculizumab. Exclusion criteria for both initial and renewal requests:
PREAMBLE: A confirmed diagnosis of atypical hemolytic uremic syndrome (aHUS) is required for eculizumab funding. The information below is to provide clinicians with context for how a diagnosis of aHUS will be assessed for funding consideration. Details to address these issues should be provided in the funding request. While some patients may already have a confirmed aHUS diagnosis, by clinical history and/or genetic testing, the majority of patients presenting with thrombotic microangiopathy (TMA) have no prior diagnosis of aHUS. For most patients presenting with a TMA, it is not possible to confidently separate aHUS from the vast majority of other conditions causing TMA until after appropriate testing and treatment have occurred. The majority of patients who have TMA suffer from Thrombotic Thrombocytopenic Purpura (TTP) (30-40%), or a secondary form of TMA (e.g., pregnancy, HIV, collagen vascular disease, drugs, malignancy, stem cell transplant, malignant hypertension) (> 50%), or hemolytic uremic syndrome due to a Shiga toxin (>5%). In most cases, patients who suffer from TTP will have an ADAMTS-13 of less than 10%. If TTP has been ruled out and any secondary causes have been treated and the patient still has a persisting unexplained TMA with ADAMTS-13 ≥10%, the patient would be presumed to suffer from aHUS. Patients who present with ADAMTS-13 of ≥10% and who are unresponsive to plasma therapy (>4 plasma exchanges) and do not have a known secondary explanation would also be presumed to suffer from aHUS. In the absence of a confirmed diagnosis of aHUS, there is nothing in these criteria that changes the clinical expectation for appropriate use of plasma exchange/plasma infusion in the management of patients presenting with TMA. Initiation Criteria A patient must meet all three of the following criteria to obtain funding for initial treatment with eculizumab:
Note: Patients who have extra-renal complications related to TMA (e.g., TMA-related cardiac impairment, TMA-related gastrointestinal impairment, or TMA-related pulmonary impairment) will be reviewed by an external clinical expert. Continuation Criteria (at 6 months): After six months of eculizumab therapy, a further six-month of funding will be considered if the patient demonstrates treatment response, defined as:
Continued treatment with eculizumab will not be funded beyond six months if a patient has experienced treatment failure, defined as:
Approval duration: 6 months Continuation Criteria (at 12 months):
There may be other exceptional circumstances where the patient has a high risk of recurrence and in whom consequences of a relapse are significant (e.g., complement Factor H genetic mutation, multiple clinical presentations of active TMA). These will be reviewed on a case-by-case basis by an external clinical expert. For patients in whom a pause in therapy is recommended, funding will be left in place for 3 months so that eculizumab can be quickly restarted upon evidence of recurrence per recommencement criteria. Approval duration: 12 months Recommencement Criteria: A patient previously diagnosed with aHUS and who responded to treatment with eculizumab and has not failed eculizumab is eligible to restart eculizumab if the following clinical conditions are met:
Note:
A patient who becomes eligible to restart eculizumab, in accordance with the above criteria, will be assessed every 6 months for treatment response or failure. Approval duration: 6 months Patients undergoing kidney transplantation: For patients with a confirmed aHUS diagnosis who are undergoing kidney transplantation, eculizumab funding will be provided for the time period immediately prior to (or at time of) transplant. Treatment must be started immediately prior to or at time of transplant. Approval duration: 6 months All funding requests must come from, or be submitted in consultation with, a pediatric nephrologist, a nephrologist, a pediatric hematologist or a hematologist. EAP Drug Request Form: |