MMIT Reality Check on Sickle Cell Disease (September 2022)

Payer Coverage:

A review of market access for sickle cell disease treatments shows that under the pharmacy benefit, about 44% of the lives under commercial formularies are covered with utilization management restrictions. Around 37% of the lives under Medicare formularies are not covered for at least one of the drugs.


Only Adakveo is covered under the medical benefit. About 67% of the lives under commercial policies are covered with utilization management restrictions. Almost 69% of the lives under Medicare policies have access to the drug without utilization management restrictions.


For about 79% of the covered lives, payer pharmacy benefit formularies do not require step therapy (ST). Of the lives that require ST, about 22% of the lives require multiple steps. Around 56% of payer-controlled pharmacy benefit covered lives require prior authorization, with 56% of those lives covered by policies that are restrictive as compared with a product’s FDA-approved label.



FDA Gives Oxbryta Accelerated Approval

In December 2021, the FDA gave accelerated approval to Global Blood Therapeutics, Inc.’s Oxbryta (voxelotor) for the treatment of sickle cell disease in children ages 4 to less than 12. The agency initially approved the drug on Nov. 25, 2019. Dosing for people at least 12 years old is 1,000 mg once daily. For younger patients, dosing is weight-based. lists the price of 90 500 mg tablets as more than $10,884.00.

Gene, Orphan, CAR-T Therapies Are in Pipeline Spotlight

Mesfin Tegenu, R.Ph., CEO of RxParadigm, Inc., anticipates gene therapy could have a big impact in 2021. “Drugs for rare diseases should always be on payers’ radar due to their high costs. Additionally, over the next few years there’s a good chance we will see several new gene therapy drugs hit the market that should very much concern payers due to associated very high costs. Gene therapy treatments that are in the pipeline include ones for hemophilia, beta thalassemia and sickle cell disease,” he says.

Looming FDA Decisions Could Swell Number of Gene Therapies on Market

Bluebird bio, Inc.’s sickle cell disease treatment, lovotibeglogene autotemcel — as well as Vertex/CRISPR Therapeutics’ exagamglogene autotemcel — could both be filed with the FDA by the end of 2022 or the first quarter of 2023, according to Arash Sadeghi, clinical pharmacist, Pipeline & Drug Surveillance, Optum Rx. Sickle cell disease is a key area of interest for gene-therapy-focused firms “because of the large target population relative to other orphan conditions,” he tells AIS Health, a division of MMIT.

Key Findings:

Market Events Drive Changes

Several approved medications can help improve the symptoms of sickle cell disease. These include hydroxyurea and Endari (L-glutamine). Most recently, the FDA gave accelerated approval to Global Blood Therapeutics, Inc.’s Oxbryta (voxelotor) for the treatment of sickle cell disease in children ages 4 to less than 12. The medication was approved for the treatment of sickle cell disease in adults and pediatric patients 12 years of age and older in 2019. The therapy targets the underlying cause of sickle cell disease, the abnormal hemoglobin. The drug works by preventing hemoglobin from sticking together, which allows the red blood cells to maintain a more normal and flexible shape.

Competitive Market Landscape

Medunik USA Inc.’s Siklos (hydroxyurea), Bristol Myers Squibb Co.’s Droxia (hydroxyurea) and Emmaus Medical, Inc.’s Endari are other competitors in this indication. Several drugs are in Phase III clinical trials.

Pharmacy Benefit Implication

Endari and Siklos have very little unrestricted access versus Droxia in this indication. Adakveo requires prior authorization and is covered under both the pharmacy and medical benefit, while the other agents fall under the pharmacy benefit.

Key Players in Market:


© 2024 MMIT
AIS Health Staff

AIS Health Staff

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