Background: Hydroxyurea is now firmly the standard of care for children with sickle cell disease (SCD) beginning early in life, resulting in a healthier generation of children and emerging adults. The formulation of hydroxyurea most commonly used for young children is a 100 mg/mL liquid suspension, compounded using hydroxyurea powder or by opening capsules. This formulation can be bottled in any quantity and does not need refrigeration. Liquid hydroxyurea has historically been compounded (and often delivered) by hospital pharmacies associated with a pediatric sickle cell center. The cost of hydroxyurea liquid or powder is negligible, and this medication has been easily available with no associated financial burden for decades. In mid-2025, a commercial formulation of hydroxyurea (Xromi, Nova Laboratories Ltd., Leister, UK and Rare Disease Therapeutics, Inc., Franklin, TN) became available. Xromi needs to be refrigerated and is prepackaged as 148 mL bottles with a cost of ~$900/bottle ($6 per mL). The manufacturer has no existing copay assistance program. With the availability of this commercial product, hospital or other pharmacies are now legally unable to compound hydroxyurea. Alternatives include capsules, which are often unable to be swallowed by young children and Siklos (dissolvable tabs), another commercial product requiring prior authorization and with associated financial burden. Here, we describe challenges and ongoing barriers of our real-world experience in transitioning pediatric SCD patients to an available and feasible hydroxyurea formulation amidst this abrupt change.

Methods: We identified patients in the Brown University Health (BUH) Comprehensive Sickle Center prescribed liquid hydroxyurea using our SCD Dashboard and pharmacy databases when the imminent arrival of Xromi was announced. Our multidisciplinary SCD team has an embedded pharmacist and pharmacy technician who worked on this process along with both the SCD clinical team and hospital outpatient pharmacy. For each patient, the pharmacy team investigated insurance coverage and worked to identify the best option for coverage without additional financial burden to the family. The team worked not only directly with families but also with Rhode Island Medicaid and private payors to advocate for coverage.

Results: A total of 50 patients were prescribed hydroxyurea liquid suspension (mean age was 7.2 ± 4.1 years). Insurance coverage was mostly Rhode Island or Massachusetts Medicaid (36/50=72%). Prior to Xromi, hydroxyurea was supplied by the BUH Outpatient pharmacy as a 60-day supply in whatever quantity was needed based on dose. The mean (± SD) hydroxyurea daily dose (supplied as 100 mg/mL) was 5.6 ± 3.7 mL (range 1.5-22 mL). The dispense volume, most often as a 60-day supply, was 176 ± 115 mL (range 45-660 mL). The co-pay for most patients was $0 with maximum monthly copay of $30 for one patient.

The transition to Xromi required prior authorization for all except one patient, with an expectation of renewing the prior authorization every 3 months for this lifetime medication. There are 14/50 (28%) of patients who are covered at least in part by private insurance. The out-of-pocket Xromi co-pay for these families ranges from $20-$723 per month with many requiring out of pocket payment of >$100/month. Because Xromi bottles are supplied as 148 mL, doses and timing of medication dispensation had to be adjusted (e.g. having to pickup the medication every 15-20 days). Because of changes in availability and cost of Xromi, to date, 2 patients were transitioned to capsules and 4 patients were transitioned to Siklos. There is not yet a clear viable solution for ~10% of patients.

Conclusions: The sudden integration of a commercial formulation of hydroxyurea has disrupted the ability to supply this medication to the pediatric SCD population. While patients covered by Medicaid, at least in some states, will have uninterrupted access to liquid hydroxyurea, but the ~20-30% of patients covered by private insurance will now have an overwhelming financial burden for this essential medication. This current situation creates real and significant risk of interruption or discontinuation of hydroxyurea for the vulnerable pediatric SCD population. We must act together as a hematology and SCD community to advocate for our patients to prevent disruption of hydroxyurea, which would undoubtedly increase unnecessarily morbidity and mortality for children with SCD.

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2025
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