Medicine continues to evolve quickly, with new treatments and trials coming to market every month. As a doctor in practice, staying up-to-date on the newest innovations and school of thought can be overwhelming, particularly for rare diseases such as Duchenne muscular dystrophy or spinal muscular atrophy. What’s the role of a pharmacy benefit manager in cases like these?

As part of its value-based approach to pharmacy benefit management (PBM), Magellan Rx Management sees itself as an idea connector and conversation starter. Recently, a discussion forum connected doctors with two key opinion leaders to discuss the complexities and effective management strategies for Duchenne muscular dystrophy (DMD) and Spinal muscular atrophy (SMA).

  • Duchenne muscular dystrophy is a genetic disorder characterized by progressive muscle degeneration and weakness. It is one of nine types of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, usually between ages three to five. The disease primarily affects boys, but in rare cases it can affect girls.* According to the UCLA Duchenne Muscular Dystrophy Research Center, DMD affects one in every 3,500 male births.
  • Spinal muscular atrophy is a genetic disease affecting the part of the nervous system that controls voluntary muscle movement. SMA involves the loss of nerve cells called motor neuronsin the spinal cord and is classified as a motor neuron disease. The age at which SMA symptoms begin roughly correlates with the degree to which motor function is affected: The earlier the age of onset, the greater the impact on motor function.* For SMA, the disease affects one in every 6,000-10,000 births, according to the Orphanet Journal of Rare Diseases.

The physician KOLs provided their expertise and insight on a wide range of topics including review of the clinical evidence, appropriate patient selection, and a discussion of the treatment landscape.  Additionally, effective clinical polices, practical dosing considerations, and emerging therapeutics were explored in this robust webinar. Importantly, doctors who took part in this discussion were given the opportunity to ask the KOLs questions about DMD and SMA – providing an opportunity for open dialogue and discussion about these complex issues.

*Information supplied by