Cost vs Benefit: When Expensive Medications Make Sense Despite Side Effects

When a medication costs more than a car, or even a year’s rent, it’s natural to ask: is this worth it? Especially when the side effects are brutal - fatigue so deep you can’t get out of bed, nausea that lasts for weeks, or immune reactions that land you in the hospital. Yet, for thousands of people, these drugs are the only thing standing between them and death, or a life of constant pain. This isn’t about greed or corporate profits. It’s about a brutal, real-world math: when does the benefit outweigh the cost - and the suffering?

Why Some Drugs Cost So Much

Not all expensive drugs are created equal. The 50 most costly medications covered by U.S. Medicare in 2023 averaged over $16,000 per dose. About 62% of them are biologics - drugs made from living cells, not chemicals. These aren’t pills you pick up at your corner pharmacy. They’re complex, hard to replicate, and often require special handling and administration. Many target rare diseases - conditions that affect fewer than 200,000 Americans. Because so few people need them, the cost to develop and produce each dose gets spread across a tiny patient base. The result? A single treatment can hit $500,000 or more.

Cancer drugs, autoimmune therapies, and gene treatments dominate this space. For example, CAR-T cell therapy like tisagenlecleucel costs $475,000 per infusion. It’s not a cure-all. Patients often spend days in the hospital dealing with cytokine release syndrome - a dangerous immune overreaction. But for those with late-stage leukemia who’ve run out of options, it’s the difference between months of pain and years of remission.

The Hidden Cost of Side Effects

Side effects aren’t just annoying. They’re expensive. A drug that causes severe nausea might mean you can’t work for two weeks. One that lowers your white blood cell count could land you in the ER with an infection. These aren’t rare events - they’re built into the treatment plan.

Take hepatitis C. A decade ago, treatment meant weekly injections of interferon. Patients got fever, chills, depression, and extreme fatigue. Cure rates? Around 50%. Today, a 12-week course of Harvoni costs about $7,000 out-of-pocket for many. But it’s a pill. No injections. No hospital visits. And it cures 95% of people. The side effects? Mild headache or tiredness. The value isn’t just in survival - it’s in returning to normal life.

For someone with hemophilia, emicizumab costs $15,000 a month. But before this drug, they needed infusions three times a week to prevent bleeding. Many ended up with permanent joint damage. Now, they get one shot a week. Fewer bleeds. Less pain. Fewer surgeries. The side effects? Headaches, joint pain, occasional blood clots. For many, it’s a fair trade.

When the System Fails Patients

The problem isn’t just the price tag. It’s the system that makes you jump through hoops just to get the drug you need.

Most insurance plans require step therapy - you have to try cheaper drugs first, even if they don’t work. That can mean months of trial and error. Then there’s prior authorization. Your doctor submits paperwork. The insurance company takes 7 to 14 days to respond. If they say no, you start over.

Medicare Part D’s coverage gaps - the infamous "donut hole" - hit hard. In 2021, over 2.3 million beneficiaries hit this gap. One patient on Revlimid, a multiple myeloma drug, paid $11,538 out-of-pocket in 2016. Over 75% of that came after they’d already spent their annual limit. Many skip doses. Some choose between medicine and food. A 2022 survey found 42% of patients on drugs over $10,000 a month made that choice.

Who Gets to Decide What’s Worth It?

In Canada, the UK, and Germany, government agencies review every new drug. They ask: does it add real value? How much does it cost per extra year of healthy life? In the UK, if a drug costs more than £30,000 per quality-adjusted life year (QALY), it’s usually rejected. Daratumumab, a multiple myeloma drug, was denied by NICE in 2016 because its price was £120,000 per QALY. After negotiations, the price dropped. It was approved.

The U.S. has no such system. Drugmakers set prices. Insurers negotiate behind closed doors. Rebates are hidden. The list price you see? Often not what anyone pays. The Congressional Budget Office says rebates reduce costs significantly - but we don’t know by how much. That means patients, doctors, and even insurers are flying blind.

A 2024 study found that 56% of the 50 most expensive U.S. drugs were rated as having low or no additional benefit by independent European health agencies. Yet they’re still sold here at full price.

When It’s Worth It - Real Stories

There are moments when the math changes.

A 58-year-old woman in Edmonton with advanced rheumatoid arthritis tried every oral drug. None worked. Her joints were crumbling. She couldn’t hold a coffee cup. Her doctor offered a biologic that cost $18,000 a month. Side effects? Risk of serious infection, occasional fever. She said yes. Six months later, she was gardening again. Walking without pain. She still pays $3,000 a month after insurance. But she’s alive - and active.

A teenager with a rare genetic disorder had seizures every day. Standard meds didn’t touch them. A gene therapy was approved - $3 million one-time cost. His family fought for coverage. Took two years. He got the treatment. Now, he has one seizure every six months. He’s in school. He plays video games. He sleeps through the night.

These aren’t outliers. They’re the reason these drugs exist.

How to Navigate the System

If you’re facing a high-cost drug, here’s what actually works:

• Ask for a patient assistance program. Most drugmakers offer them. On average, they cover 40% of out-of-pocket costs for commercially insured patients.
• Use a specialty pharmacy. They don’t just fill prescriptions. They assign case managers who handle prior authorizations, appeals, and financial aid applications. They spend over 3 hours per patient.
• Know your insurance phases. Medicare Part D has four stages: deductible, initial coverage, donut hole, catastrophic. Once you hit catastrophic, you pay only 5% of the cost. That’s a lifeline.
• Check independent foundations. The Chronic Disease Fund gave out $2.1 billion in aid in 2022. Organizations like NeedyMeds and Patient Access Network Foundation help with copays.
• Ask your doctor for alternatives. Not all expensive drugs are the only option. Sometimes a different biologic, or even an older drug used off-label, gives similar results at a fraction of the cost.

The Bigger Picture

The U.S. spends more on prescription drugs than any other country. In 2022, specialty drugs - just 3% of all prescriptions - made up 54% of pharmacy spending. By 2030, that number could hit 79%.

The Inflation Reduction Act of 2022 started the first-ever Medicare drug price negotiations in 2024. But here’s the catch: 96% of the most expensive drugs are still excluded because they’re too new, too rare, or don’t meet the criteria. The real change won’t come from government alone. It will come from patients demanding transparency. From doctors refusing to prescribe drugs with no proven value. From insurers demanding real data before paying millions.

The question isn’t whether expensive drugs should exist. It’s whether we’re paying for real benefit - or just hype.

For some, the answer is clear: the cost is high, the side effects are real - but the alternative is worse. And sometimes, that’s enough.

Next steps: If you’re considering a high-cost medication, start by asking your doctor for the clinical evidence behind it. Then contact your insurer and ask for a cost estimate under your plan. Finally, reach out to a specialty pharmacy - they’ll guide you through financial aid options. You don’t have to navigate this alone.