The United States’ Orphan Drug Act (ODA) was enacted in 1983. Years later, it has failed to fuel the creation of orphan drugs to treat rare diseases, including spinal muscular atrophy (SMA).
First, what are orphan diseases? Those like SMA that affect less than 200,000 Americans. According to recent research, the ODA allows pharmaceutical corporations to maximize revenue without addressing the needs of patients. In other words, these companies could, theoretically, raise their prices astronomically and then expect patients to pay for the medicine. If patients cannot pay for it, then they are without the medicine or treatment they need to live a better quality of life.
However, the ODA has served its purpose of significantly increasing the number of orphan drugs on the market.
Now, researchers believe that the government needs to intervene. Authorities should provide an economic incentive or balance. This should give patients access to needed medicines.
One study in the Orphanet Journal of Rare Diseases touched on the ethical predicament of providing people who suffer from rare diseases with reasonably priced treatment.
I agree that it is unethical to develop treatments that work and then force patients to pay a high price just to be treated. I think this is especially horrible for those who could benefit from a lifesaving treatment.
Late 2016, the FDA approved a treatment for SMA, Spinraza, and the list price was a whopping $125,000 for each injection. For six injections within a year, the price goes up to $750,000.
The drug price is ridiculous, but can you put a numerical price on life? Is this how much a life is worth to drug companies?
