When my 2-year-old was diagnosed with a rare, incurable genetic disorder, I looked for every treatment available. I soon learned there was only one drug on the market. It had terrible side effects and had to be administered every six hours. My son quickly caught on to the fact that the medicine made him feel nauseated, and he would intentionally throw it up. I spent many days and nights re-dosing his medicine, hoping he’d sleep through the nausea.

When I heard about a new clinical drug trial at Stanford, I immediately enrolled him in the study. The new drug, with the same active ingredient, would replace his six-hour dosing schedule with a 12-hour version. For the first time in months, my son was doing well and was interested in eating again. We finally felt hopeful for his future.

After we finished the trial, we learned the cost of the medicine.

In 2013, the new drug received FDA approval. A heavy price tag of over $300,000 annually was placed on it. Families like ours, accustomed to paying $10,000 annually, were shocked by the pharmaceutical company’s decision. Parents began asking why it was so expensive.

Pharmaceutical companies frequently cite research and development (R&D) as their reason for establishing high costs. However, an investigation by Dr. Oliver Wouters published in The Journal of the American Medical Association in 2022 found no correlation between estimated research and development investments and treatment costs. A University of California San Diego report quoted the senior author of the study, Inmaculada Hernandez, as saying, “Our findings provide evidence that drug companies do not set prices based on how much they spent on R&D or how good the drug is. Instead, they charge what the market will bear.”

By 2018, the new drug had generated more revenue than the pharmaceutical company had spent developing it. The medication had brought in nearly $500 million. Over the years, the drug has changed corporate hands through major company acquisitions and increased in price multiple times. This year, my son’s medication will cost $1.3 million.

Patients and their families need protection from pharmaceutical price-gouging.

The price of this drug raises questions about unintended consequences of the 1983 Orphan Drug Act (ODA). Congress established the act to incentivize pharmaceutical companies to invest in diseases that affect fewer than 200,000 people in the United States. While these incentives may be helpful in motivating companies to take interest in rare illnesses, it does not protect rare disease communities from pharmaceutical price-gouging.

Market-exclusivity protections and patent incentives limit competition and allow pharmaceutical companies total control over pricing. Lack of regulation allows pharmaceutical companies to create monopolies and charge sick patients unaffordable prices without any safeguards.

Instead of government regulation, insurance companies become the barrier to acquiring the expensive drugs.

Every year, my husband and I have to get prior authorization for the medication. Every year, our insurance company denies it, and we and our physician have to submit appeals and complete peer-to-peer discussions and pray that they will ultimately approve it. It has become a grueling process that feels like a second job. The chronic anxiety, the dread of losing a job with health benefits, and the fear for my child’s health are constantly in the back of my mind.

My family lives in a country with great privilege, among amazing scientists and doctors who work tirelessly to develop new treatments. Their discoveries have allowed my son to sleep through the night without any dosing interruptions, eliminating the need to keep a throw-up bowl beneath his bed. The medication has drastically improved his quality of life and could do the same for any other child and adult affected by this disease, if insurance will pay for it. But a medication can’t help the people who need it if the cost is out of reach.

To ensure fairness, we must legislate stronger safeguards to keep medications affordable and protect vulnerable communities.

Ashton Jenkins is a mom to three kids and a master of social work student.

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