How Many Lives Need to Be Saved to Make It “Worth It”?
Rare diseases were never something that I was interested in…in fact I never saw myself working with “sick” individuals at all. It was the furthest thing from my mind in grade school and through high school and even undergrad – I was never going to be a doctor…at least not that kind, working in a hospital. While the rest changed a few years ago, I recently attended a lecture hosted by PCF here at the college to thank for making me see rare diseases differently too. Dr. Frame was the guest speaker and spoke about his personal experiences with his daughter, who has a debilitating rare disease, and as a pharmacist who has experience treating rare diseases that no one can figure out.
This lecture was not only inspiring, but extremely insightful into the plight of families dealing with these conditions, as well as how treatment comes around and what all goes into getting the ball rolling once these rare diseases are discovered. One thing I was shocked to learn was that the FDA defines a “rare disease” as < 200,000 individuals in the United States being diagnosed with a condition. That seems like a huge number to call a “rare disease” to me – but it is only 0.5% of the US population, after all. I found it really disheartening to hear about how difficult it was for Dr. Frame to find anyone interested in researching his daughter’s disease, especially once they started learning about other children who were also diagnosed. His training made him uniquely positioned to be able to get it off the ground and fundraise for research, etc., but what about patients whose parents are not? I remember reading a story during my junior year of high school in class about a husband whose wife was diagnosed with Creutzfeldt-Jakob disease, and he became a scientist researching prions to solve the case and find the cure with no scientific experience or money, and I just thought, wow…how lonely, depressing, and anxiety-inducing racing against the clock like that?
Thankfully, the FDA has passed the Orphan Drug Act in 1983 to incentivize large drug companies that have the capabilities to look into creating orphan drugs. We all know that creating new drugs is extremely expensive, so being able to get tax credits, market exclusivity, and grants is hugely beneficial to many of these companies. However, the problem remains: they want a money-maker, and they don’t want to sink themselves by synthesizing an expensive drug that very few people can buy. This leads to more problems with orphan drugs: frequently, there isn’t a huge benefit despite costing hundreds of thousands of dollars (or more, if you’re unlucky).
I wanted to talk about an orphan drug I found while researching this topic – Yuviwel for achondroplasia. This is more commonly known as dwarfism. According to the FDA’s approval announcement, “Patients receiving Yuviwel grew an average of 1.5 centimeters taller over the 52 weeks compared to those who received a placebo. In the open-label extension period, patients who received two years of Yuviwel treatment maintained the rate of height growth during the second year.” The problem? That equates to 3cm of growth that they can show this drug will likely help – with a price tag of a cool $1mil for two years of treatment. While this treatment isn’t necessarily life-saving, it demonstrates the small changes or improvements that orphan drugs can make…all while dropping millions on research and development (paid for by grants and tax breaks) just to sell likely only a few vials since this is so unlikely to be covered by insurance or in high-demand out-of-pocket. Many patients can skirt these costs by enrolling in clinical trials, however, many patients don’t want to subject themselves to the risks of trial drugs either.
Right now, it’s an unbalanced scale of condition improvement vs. cost, but my hope is that in the future, more legislation and advocacy can happen to improve the outcomes of patients living with rare diseases and balance the scale.
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