Pharmacy management at many hospitals was recently asked to prepare for the arrival of nusinersen for treatment of spinal muscular atrophy for patients. I had no idea what this drug was (or how to pronounce it) and then I started to receive emails on a listerv from other pediatric institutions discussing how they were going to prepare for the new drug. At that point I got interested in learning a little more. Here's some background for you...
"The U.S. Food and Drug Administration (FDA) approved nusinersen under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. Nusinersen is the first and only treatment approved in the U.S. for SMA, a leading genetic cause of death in infants and toddlers that is marked by progressive, debilitating muscle weakness." Biogen.com
"SMA involves the loss of nerve cells called motor neurons in the spinal cord and is classified as a motor neuron disease. In the most common form of SMA (chromosome 5 SMA, or SMN-related SMA), there is wide variability in age of onset, symptoms and rate of progression. In order to account for these differences, the chromosome 5 SMA often is classified into types 1 through 4. 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. Children who display symptoms at birth or in infancy typically have the lowest level of functioning (type 1). SMA onset in children (types 2 and 3), teens or adults (type 4) generally correlates with increasingly higher levels of motor function." MDA.org
So it's a "wonder drug". It's honestly fantastic news for everyone diagnosed and suffering with this horrible condition. The results of the drug have been groundbreaking with some patients even seeing a cure of their condition! Sign them all up, right? Well, here's the issue: AWP for this agent is $125,000 per vial. Patients receive 6 vials in year one ($750,000/year) and three vials yearly after that ($375,000/year). There are some small savings through 340B programs for eligible hospitals, but the cost of therapy is still over $500,000/year in year one. Insurance companies haven't reached decisions yet on whether they will pay for the agent (as far as I know), although I did hear a rumor that at least one state Medicaid said they would not.
When asked how the company priced the agent they said: the company balanced “a number of important factors, including its clinical value, its impact to patients and the health care system as a whole, and the need for [the drug company] to fund further research and development to make the next innovation possible.”
We've had some instances over the past 5-10 years where high-priced "wonder drugs" were introduced for cancer and provided some marginal benefits with questionable quality of life and they obviously sparked some debate. As far as I can remember though, we've never had this kind of agent introduced where it will significantly improve quality of life-- allowing toddlers to crawl and roll over who were previously unable to move-- and potentially reduce longer term healthcare costs. So, if you're a pharmacist working in a hospital, what do you do? Pursue prior authorization? Increase your drug budget? What if you don't get paid? What if your hospital has 20 patients who want to receive the drug and you can only afford to provide care to 5 of them? Who gets prioritized? Do you create an ethics committee to decide?
At the end of the day I'm having an internal struggle about what to do in this case. How do you ethically help patients to have a better life (like we signed up for when we became pharmacists) but keep your place of employment in business? I need to keep a paycheck coming in just like everyone else. I just keep saying to myself "this is so unsustainable". We're not going to be able to print more money to keep paying for high drug costs. It's not going to be magically fixed by any one political candidate. We want America to be a great place for everyone and to extend longevity and improve health and quality of life, but at what cost?
I encourage you to visit this website to learn more about The Campaign for Sustainable Rx Pricing (CSRxP).