Muscular Dystrophy Association
MDA Condemns Continuing Insurance Coverage Denials of FDA-Approved Neuromuscular Disease Treatments
August 31, 2023 by Paul Melmeyer

Statement from Muscular Dystrophy Association:
MDA Condemns Continuing Insurance Coverage Denials of FDA-Approved Neuromuscular Disease Treatments
August 31, 2023

Health insurers continue to deny coverage of FDA-approved therapies for neuromuscular diseases, and the problem only appears to be growing. In March, we noted that insurers were denying coverage of FDA-approved therapies for those with neuromuscular diseases at an unreasonable and harmful rate. Unfortunately, in the ensuing five months, it has only gotten worse. With so few FDA-approved therapies available for those living with neuromuscular diseases, every insurance denial means more time spent finding new ways to access care. Many neuromuscular diseases are progressive, and while an approved therapy may slow or stop a patient’s progression, no therapy can reverse what a patient has lost while waiting for appropriate care. 


The access barriers continue to grow. Most recently, draft plans published by Blue Cross Blue Shield plans in several states would deny coverage of the ALS drug Qalsody, stating that it is not “medically necessary”. Additionally, a boy with Duchenne muscular dystrophy was denied coverage for the newly approved gene therapy Elevidys by a Blue Cross Blue Shield insurer (Highmark). After being denied access to Elevidys at every turn, the family was left with the impossible choice of paying the high cost of the drug themselves or not receiving the life-changing therapy. Fortunately, an online petition that garnered 35,000 signatures and action by patient advocacy groups convinced Highmark to reverse its decision, but it should not take 35,000 voices and a campaign for our community to access life-changing therapies. 


These latest examples join similar barriers to access, such as those for Relyvrio for ALS, Spinraza, Zolgenzma, and Evrysdi for spinal muscular atrophy (SMA), and Elevydis, Emflaza, and exon skipping therapies for Duchenne muscular dystrophy. Anytime access to an FDA-approved treatment for our community is threatened, MDA stands staunchly opposed to such proposals and urges each insurance provider to reconsider their approach. 


Despite the fact that issues of access to approved therapies persist, MDA has been hard at work advocating for better access to care, and genuine progress has been made. As we noted in our March statement, Pharmacy Benefit Managers pose a problem for patients both in terms of pricing and coverage due to a lack of transparency. With our partners, we urged Congress to take action, and they have taken up several measures to address these issues. Similarly, we noted how Quality Adjusted Life Years are a harmful metric in the evaluation of treatments and have advocated for legislation that would ban their use in all federally funded healthcare programs. These reforms passed the House Energy and Commerce Committee this Spring and await a vote from the full House of Representatives.


Additionally, MDA and our partners have advocated for the protection of Medicaid from harmful cuts, the continuation of low-income subsidies for marketplace plans, the limitation of prior authorization (thus ensuring timely access to care), including patient voices during Medicare Part D redesign, better consideration of those with rare diseases when Medicare makes coverage decisions, and we will soon weigh in on a proposal to limit junk health plans to ensure that people can purchase health insurance that actually meet their needs.


Finally, MDA is also actively engaged in efforts to improve access to care outside of public policy. We recently launched the MDA Gene Therapy Support Network with GTx Support Specialists ready to assist the community in navigating access to gene therapies, are co-funding research at Tufts University Medical Center to gain a clearer picture of barriers to accessing FDA-approved therapies, and are funding Cure Rare Disease to develop innovative proposals for coverage of n-of-1, bespoke gene therapies.


These actions and many more represent our commitment to ensuring access to all FDA-approved therapies and we will continue to fight for access for the neuromuscular disease community.

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