Overview of Duchenne Muscular Dystrophy
Muscular dystrophy is a group of genetic disorders that cause progressive muscle weakness and loss of muscle. Each type differs in the muscles it affects, age it starts, and genes that cause the disorder. Duchenne muscular dystrophy (DMD) affects 1 in 7,250 males age 5-24 and first appears between ages two and three. Females can be carriers of this disorder but rarely show signs or symptoms. In patients with DMD, muscle weakness starts in the trunk, moves to the legs, and then progresses to the arms. Children with DMD have difficulty running, jumping, and walking up steps; they are also more prone to fractures. Duchenne muscular dystrophy progresses rapidly and patients are usually wheelchair bound by their early teens. This disease ultimately leads to severe heart and lung complications. Patients with DMD have an average life expectancy of 35 years.
Treating patients with Duchenne muscular dystrophy requires the collaboration of many different health care professions and strong support from the patient’s family. Early screening for heart or lung dysfunction is an essential component to DMD management. Surgical treatment may be required for some patients to correct joint contractures or scoliosis. Physical therapy and weight management are important to help avoid the need for a wheelchair and to maintain muscle mass even after the patient becomes wheelchair bound. Pharmacologic treatment with corticosteroids—such as prednisone or deflazacort—has been proven to keep patients walking longer. Because managing this disease can be daunting, education and psychotherapy are vital resources for both the patient and their family.
Prednisone has long been the standard pharmacologic treatment for Duchenne muscular dystrophy in the United States. In February of 2017, the FDA approved deflazacort (Emflaza) for the treatment of DMD in patients five years of age and older. Emflaza is available as an oral tablet and suspension and is to be taken once daily with or without food. Similar to prednisone, Emflaza exhibits anti-inflammatory and immunosuppressive effects but the mechanism by which it treats DMD specifically is not fully understood. Clinical trials have shown that Emflaza works as well as prednisone in improving motor function and causes significantly less weight gain. However, the cost of Emflaza is considerably greater than prednisone.
MedTrakRx’s Cost Management Strategy
Due to the extremely high cost as well as its role in managing this complex condition, Emflaza has been designated as a specialty medication therapy. As a specialty drug, Emflaza is subject to MedTrak’s prior authorization (PA) approval criteria. This evidence-based PA criteria has been developed by MedTrak’s team of Clinical Pharmacists and seeks to ensure appropriate utilization of this costly therapy. When approval criteria is met, patients will have access to the designated Best in Class specialty pharmacy where MedTrak has secured competitive discounts and clinical expertise in managing DMD.
- Emflaza [package insert]. Northbrook, IL: Marathon Pharmaceuticals, LLC; 2017. https://emflaza.com/prescribing-info.pdf. Accessed March 7, 2017.
- Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurology. 2010 Jan; 9(1):77-93. http://www.sciencedirect.com.www2.lib.ku.edu/science/article/pii/S1474442209702716. Accessed March 9, 2017.
- Griggs RC, Miller JP, Greenberg CR, et al. Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology. 2016 Nov 15;87(20):2123-31. http://www.neurology.org/content/87/20/2123.full.pdf+html. Accessed March 7, 2017.
- Gloss D, Moxley RT, Ashwal S, et al. Practice guideline update summary: corticosteroid treatment of Duchenne muscular dystrophy. Neurology. 2016 Feb 2;86(5):465-72. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4773944/pdf/NEUROLOGY2015679183.pdf. Accessed March 7, 2017.
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