Approval criteria used by Fallon Community in MA, USA. Sound approach when dealing with HIGH PRICED rare drugs like Horizon Pharma Procysbi. Health Canada recent decision is negligent when it comes to care and cost for Canadian Cystinosis Patients.

1. Fallon Community Health Plan Department of Pharmacy Services Page 1 of 2
Prior Authorization Approval Criteria
Brand (generic)
Generic name: cysteamine bitartrate
Brand name: Procysbi
Medication class: cysteine depleting agent
Mechanism of action: converts cysteine into cysteine-cysteamine mixed disulfide thereby
increasing the depletion of cysteamine from the lysosome
FDA-approved uses: treatment of nephropathic cystinosis in patients greater than 6 years
Available dosage forms: delayed release capsules (25 and 75 mg)
Usual dose: 2Gm per day in 2 divided doses every 12 hours (1.3-1.95gm/M2/day or
90mg/mg/kg/day. If the WBC cysteine level is >1nmol half cysteine/mg
protein and the plasma cysteamine is >0.1mg/L
Start with 1/6-1/4 of maintenance dose, titrate slowly over 4-6 weeks
Goal: Decrease disease progression, kidney dysfunction, dialysis, need for
transplant, organ failure, premature death
Approximate monthly cost: $2,091.60/day, $62,748/30 days, $752,976- year
(based on AWP 2013)
Duration of therapy: indefinite
Criteria for use (bullet points below are all inclusive unless otherwise noted):
 The indicated diagnosis (including any applicable labs and /or tests) and medication
usage must be supported by documentation from the patient’s medical records.
 Must have nephrotic cystinosis
 Target WBC cysteine levels must be monitored
 End organ damage must be monitored
 Must be able to be taken on an empty stomach (30 minutes before eating or 2.5 hours
after eating)
 Must be unable or intolerant to take cysteamine immediate release tablets 4 times a
day
Criteria for continuation of therapy:
 Patient is tolerating and responding to medication and there continues to be a medical
need for the medication
 WBC cysteine levels must be kept <1nmol half cysteine/mg protein
OR
 If poor tolerability, may be able to keep WBC cysteine levels down below 2nmol half-
cystine/mg protein and the plasma cysteamine level is >0.1mg/L
Caution:
 Hypersensitivity to cysteamine or penicillamine

2. Fallon Community Health Plan Department of Pharmacy Services Page 2 of 2
 Side-effects: fever 22%, lethargy 11%, rash 7%, vomiting 35%, anorexia 31%
Monitoring: If the WBC cysteine level is >1nmol half cysteine/mg protein and the plasma cysteamine
is >0.1mg/L
Contraindication:
 If the WBC cysteine level is >1nmol half cysteine/mg protein and the plasma cysteamine
is >0.1mg/L must confirm that patient is adherent and conforming to food
administration restrictions
 If patient is adherent must look at relationship of eating and dosing
 WBC cysteine levels every month for 3 months then every 3 months for 1 year, the twice
a year after that
Not approved if:
 Have any contraindications to the use of
 Does not meet the above stated criteria.
Special considerations:
 Approval based upon study of 43 patients
 Non-inferior to immediate release cysteamine tablets
 Price of immediate release cysteamine tablets $17.08/day, $512.40/month, $6148/year
(no food restrictions with immediate release product)
FCHP Pharmacy and Therapeutics Committee approval: __________________________________________
Date: ______________________
Adopted: 12/2013