VIDEOS: High-priced drugs, out-of-pocket costs raise challenges for neurologists

LOS ANGELES – Neurologists can play an important role in helping patients gain access to high-cost, breakthrough drugs, while at the same time guiding patients to lower-cost options whenever possible, speakers said at the annual meeting of the American Academy of Neurology.

The use of the Orphan Drug approval pathway established in 1983 has gained a great deal of steam for rare neurologic diseases in recent years with the approval of a number of drugs, such as nusinersen (Spinraza) for spinal muscular atrophy, eteplirsen (Exondys 51) for Duchenne muscular dystrophy, and edaravone (Radicava) for amyotrophic lateral sclerosis, said Nicholas Johnson, MD, a pediatric neuromuscular disease specialist at the University of Utah, Salt Lake City.

But given that only 2% of U.S. physicians are neurologists, yet 18% of rare diseases are neurologic and 11% of drugs in development overall are for neurologic diseases, there are a great deal of challenges arising for neurologists in getting access to these new high-priced drugs for their patients, said Dr. Johnson, who leads the AAN’s Neurology Drug Pricing Task Force and is also chair of the AAN Government Relations Committee.

These challenges range from increased administrative burden on staff, getting insurance approval, finding administration sites, and the ability to perform special patient assessments, he said in an interview.

Dr. Nicholas Johnson’s interview:

Dr. Brian Callaghan’s interview:

Many high-priced drugs commonly prescribed for chronic neurologic conditions and diagnostic tests also have high out-of-pocket costs for patients, but it is remarkably hard even for well-informed experts to find the actual costs that patients will pay out of pocket for such drugs and tests, according to Brian Callaghan, MD, a neuromuscular disease specialist at the University of Michigan, Ann Arbor.