MMIT Reality Check on Sickle Cell Disease (Sept 2021)

Payer Coverage:

A review of market access for sickle cell disease treatments shows that under the pharmacy benefit, about 38% of the lives under commercial formularies are covered with utilization management restrictions. About 25% of the lives under health exchange formularies are not covered for at least one of the drugs.

Only Adakveo is covered under the medical benefit. About 59% of the lives under commercial policies are covered with utilization management restrictions. Meanwhile, almost 73% of the lives under Medicare Part B policies have access to the drug without utilization management restrictions.

For about 80% of the covered lives, payer pharmacy benefit formularies do not require step therapy (ST). Of the lives that require ST, 28% require multiple steps. Around 59% of payer-controlled pharmacy benefit covered lives require prior authorization, with 44% of those lives covered by policies that are restrictive as compared with a product’s FDA-approved label.

Trends:

FDA Grants Priority Review to Oxbryta

In September 2021, the FDA accepted for filing and reviewing Global Blood Therapeutics, Inc.’s supplemental New Drug Application seeking accelerated approval for Oxbryta (voxelotor) for the treatment of sickle cell disease in children ages 4 to 11 years, as well as an NDC for a new dispersible tablet form. The FDA previously granted accelerated approval to the medication in tablet form to treat patients aged 12 years or older.

Gene Therapy May Make Big Impact

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.

FDA Approves Oxbryta

In November 2019, the FDA gave accelerated approval to Global Blood Therapeutics, Inc.’s Oxbryta (voxelotor) for the treatment of sickle cell disease in people at least 12 years old. The company says it’s the first FDA-approved treatment that targets the root cause of the disease. The agency gave the tablet fast track and orphan drug designations.
The monthly price of the drug is $10,417, with a net price of about $8,000 for many payers.

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, Global Blood Therapeutics, Inc.’s Oxbryta (voxelotor) was approved for the treatment of sickle cell disease in adults and pediatric patients 12 years of age and older. 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. Another recently approved treatment for sickle cell disease is Novartis AG’s Adakveo (crizanluzumab), which contains an antibody that blocks one of the proteins in the endothelial cells of the blood vessels. This antibody prevents the protein from binding to sickle cells, thereby reducing the pain and inflammation caused by sickle cell aggregates blocking blood vessels.

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 Implications

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 Oxbryta falls under the pharmacy benefit.

Key Players in Market:

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AIS Health Staff

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