Christian Mumm has a genetic disease that causes severe epilepsy, experienced frequent seizures – at his worst, one every 10 minutes – that left him depleted and jumpy. Christian’s severe form of epilepsy stems from a mutation in a gene called KCNQ2. That mutation causes electrical signaling in Christian’s brain to go haywire. After a half-dozen drugs failed to help Christian, doctors suggested the family try one called Potiga. The medicine has had a powerful effect. Seizures that once struck multiple times an hour now come once every five or six days. Potiga had a unique mechanism among epilepsy medications. It worked by targeting the precise biological defect that was at the root of Christian’s disease.
GlaxoSmithKline, the British drug company that sells Potiga, pulled it off the market because of declining sales, forcing families to stockpile supplies or wean their children off a drug that dramatically improved their quality of life. Future families potentially may never have access to the drug which highlights the limitations of drug companies’ business model.
The pharmaceutical industry is investor-driven. At the end of the day, it means decisions on products that are essential to sick people are being made by those businesses' accountancy and marketing departments. But when drugs live or die depending on commercial viability, mass-market drugs that don’t fulfill their initial purpose may not be available. Potiga sales peaked in 2013, at $14 million in U.S. sales, far from analysts’ original estimates that it could reach more than $200 million per year. When GSK announced it would discontinue the drug last summer, there were fewer than 500 prescriptions per month.
“There are people who’ve already been taking the drug, and based on their perceptions, it’s helping. I actually find this one more heart-wrenching,” said Alison Bateman-House, an assistant professor in the division of medical ethics at NYU Langone Health. “Yes, we realize you were getting benefits, but because of financial realities, and just what we choose to do with our business, we’re not going to make the drug anymore.”
“Here we have 30 years of NIH-sponsored and industry-sponsored research leading to the specific understanding of a catastrophic illness. We have a drug that acts on that mechanism,” said Edward Cooper, an associate professor of neurology and neuroscience at Baylor College of Medicine. Taking it off the market “goes exactly against what our policymakers are explicitly trying to do, which is make available precision medicines to address unmet medical need.”
“If they can do this to my kid, they can pull any other drug off the shelf of your medicine cabinet and do this to anyone else, without a second glance,” Erica Mumm said. “These companies have great power, and they shouldn’t be driven solely by profit. They do have an ethical responsibility to the patient.”
GlaxoSmithKline, the British drug company that sells Potiga, pulled it off the market because of declining sales, forcing families to stockpile supplies or wean their children off a drug that dramatically improved their quality of life. Future families potentially may never have access to the drug which highlights the limitations of drug companies’ business model.
The pharmaceutical industry is investor-driven. At the end of the day, it means decisions on products that are essential to sick people are being made by those businesses' accountancy and marketing departments. But when drugs live or die depending on commercial viability, mass-market drugs that don’t fulfill their initial purpose may not be available. Potiga sales peaked in 2013, at $14 million in U.S. sales, far from analysts’ original estimates that it could reach more than $200 million per year. When GSK announced it would discontinue the drug last summer, there were fewer than 500 prescriptions per month.
“There are people who’ve already been taking the drug, and based on their perceptions, it’s helping. I actually find this one more heart-wrenching,” said Alison Bateman-House, an assistant professor in the division of medical ethics at NYU Langone Health. “Yes, we realize you were getting benefits, but because of financial realities, and just what we choose to do with our business, we’re not going to make the drug anymore.”
“Here we have 30 years of NIH-sponsored and industry-sponsored research leading to the specific understanding of a catastrophic illness. We have a drug that acts on that mechanism,” said Edward Cooper, an associate professor of neurology and neuroscience at Baylor College of Medicine. Taking it off the market “goes exactly against what our policymakers are explicitly trying to do, which is make available precision medicines to address unmet medical need.”
“If they can do this to my kid, they can pull any other drug off the shelf of your medicine cabinet and do this to anyone else, without a second glance,” Erica Mumm said. “These companies have great power, and they shouldn’t be driven solely by profit. They do have an ethical responsibility to the patient.”
Amy Buches's, Stephen, suffers from a similar problem and will receive a guarantee of a supply but she explained, “Yet, it is unsettling to know that there could be an infant born in the world this very moment that would benefit from Potiga, but will not be able to use it”.
According to a 2011 study in Pharmacoepidemiology and Drug Safety, 118 novel drugs were withdrawn from the market between 1980 and 2009. Only a fifth of them were withdrawn primarily because of safety issues.
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