According to a publication from SMA News Today, Scotland is now the latest country to make spinal muscular atrophy (SMA) drug Spinraza available to patients at low or no cost through their public health service.
About Spinal Muscular Atrophy
Spinal muscular atrophy is a genetic disorder characterized by the progressive weakening and wasting of muscles. This is caused by a loss of motor neurons – specialized nerve cells that control muscle movement.
There are a few different types of SMA, numbered one through four (or zero through four, depending on who you ask). They affect different groups, and vary widely in severity and prognosis. Most children diagnosed with type 1 muscular atrophy will not survive early childhood, while an adult diagnosed with type 4 has a completely normal life expectancy.
Some kind of SMA occurs roughly once in every 10,000 people. Type 1, the most common form, affects extremely young children and has low survival rates.
Spinraza to be Offered by NHS Scotland
Spinraza comes in the form of a spinally-administered injection.
Health systems around the world have been adopting it for the treatment of certain types of muscular atrophy, including those in the United States and many parts of Europe.
Scotland is the newest nation to make the drug available to SMA patients at low or no cost. It will provide the drug through its branch of the National Health Service, NHS Scotland. Starting this April, anyone using NHS Scotland can begin to receive Spinraza injections.
Some Controversy Remains
Despite the welcome news for SMA patients, a small number of countries around the world are refusing to administer Spinraza as part of their national health services. Authorities in Denmark, Norway, and England have all balked at the prospect of offering the drug to their patients.
The reason probably wouldn’t surprise you. Spinraza is one of the most expensive drugs in the world, with each injection costing an astounding $125,000. That means in the first year, just one patient taking Spinraza would rack up a bill between $625,000 to $750,000 (five to six injections). Every year of treatment after that costs an additional $375,000 (only three injections).
Some experts call the pricing “unethically high.” Norway did eventually approve funding the treatment for those under 18, reluctantly. Authorities in England have stated that Spinraza could be approved for select patients on a provisional basis called a managed access arrangement, though such an approval would still hinge on a price reduction from Spinraza’s trademark holder, Biogen Inc.
Spinraza received Orphan Drug designation in 2011. When it received its marketing approval in 2016, just two days before Christmas, Biotech was immediately granted a seven-year marketing monopoly on the drug. That means nobody can produce a competing version of nusinersen (Spinraza’s generic name) for SMA patients until the last week of 2023.
Until then, Biotech is allowed to charge what they’d like with little real fear of repercussion. Unless they prove willing to negotiate on pricing with these and future nations, access to Spinraza could remain limited in some areas.
At what point does pricing become a factor in the ultimate effectiveness of a treatment? How might a balance be struck between incentivized pharmaceutical development and affordable healthcare? Share your thoughts with Patient Worthy!
