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

When a drug costs more than your car, you start asking: is this really worth it? For millions of Americans, that’s not a hypothetical question. Some medications - cancer therapies, gene treatments, rare disease drugs - can run $500,000 or more per year. And they often come with brutal side effects: fatigue so deep you can’t get out of bed, nausea that lasts for weeks, or immune reactions that land you in the hospital. So why do people take them? Why do doctors prescribe them? And when does the cost and suffering actually make sense?

It’s Not Just About Living Longer - It’s About Living Better

Most people assume expensive drugs are bought just to extend life. That’s part of it, but not the whole story. Take hepatitis C. Ten years ago, treatment meant weekly injections, months of flu-like symptoms, and a 50% cure rate. Today, a 12-week course of Harvoni costs around $28,000 out-of-pocket for some, but cures over 95% of patients. No more injections. No more vomiting every day. Just a pill. And for many, that’s worth every dollar.

The same goes for rheumatoid arthritis. Before biologics like Humira or Enbrel, patients faced joint destruction, constant pain, and early disability. Now, many can return to work, play with their kids, sleep through the night. The side effects? Higher risk of infections. Maybe a rash. Maybe a mild fever. Compared to losing your hands to arthritis? That’s a trade-off people choose.

This isn’t about greed. It’s about quality-adjusted life years - or QALYs. It’s a measure that doesn’t just count how long you live, but how well. A drug that adds two years of pain and bed rest isn’t the same as two years where you can walk, laugh, cook dinner. That’s what doctors and insurers are trying to measure - even if the numbers feel cold.

Why Are These Drugs So Expensive?

You hear $500,000 for a single treatment and think: that’s insane. And it is - but the reason isn’t simple greed. Many of these drugs are made for tiny groups of people. A gene therapy for a rare childhood disorder might help only 100 people in the whole country. The company spent $2 billion developing it, and now they have to recover that cost from 100 patients. That’s $20 million per person before you even get to manufacturing, storage, or doctor fees.

Then there’s the cancer drugs. The FDA approved 50 new cancer treatments between 2017 and 2018. Half of them offered barely any improvement over older, cheaper options. But they still sold for $150,000 a year. Why? Because there’s no real price control in the U.S. Unlike in Germany or France, where health agencies review whether a drug is worth its cost before approving it, American insurers often have to pay whatever the manufacturer sets.

And here’s the twist: the list price isn’t the real price. Drugmakers give huge rebates to insurance companies - so much so that the actual cost to the system might be 40% lower. But patients still pay based on the high list price, especially if they’re in Medicare’s “donut hole.” That’s why someone might pay $6,000 a year for a drug that actually costs the system $3,500. The system is broken, and patients pay the price.

When Side Effects Are the Price of Hope

CAR-T therapy for leukemia is one of the most extreme examples. It costs $475,000. You get your own immune cells pulled out, genetically altered, and pumped back in. Then you wait. And you might get cytokine release syndrome - a storm of inflammation that can spike your fever to 105°F, crash your blood pressure, and require ICU care. Some patients don’t survive the process.

But for those who do? A 78% chance of complete remission after all other options failed. One patient on an ASCO forum said, “I was dying. I chose this because I didn’t want my daughter to grow up without a dad.” That’s not a rational calculation. It’s human.

The same goes for hemophilia. A monthly shot of emicizumab costs $15,000. But before this drug, patients bled into their joints constantly. Many ended up in wheelchairs by 30. Now, they run, hike, play basketball. The side effect? A small risk of blood clots. For many, that’s a fair swap.

The side effects aren’t the deal-breaker. It’s whether the alternative is worse. If your only other option is chronic pain, frequent hospital visits, or early death - then the side effects of the expensive drug start to look manageable.

Who Gets Left Behind?

Here’s the ugly truth: not everyone can afford to make this choice. In 2022, 68% of patients taking drugs over $10,000 a month skipped doses because they couldn’t pay. 42% chose between medicine and food. That’s not a trade-off - that’s a trap.

Medicare beneficiaries without low-income subsidies paid 2.5 times more out-of-pocket than privately insured people for the same drugs. A patient on Revlimid in 2016 paid $11,538 out-of-pocket - and most of that came after they hit the “catastrophic” coverage phase, where they were still on the hook for 5% of the cost. That’s $5,000 a year just to stay alive.

Meanwhile, manufacturer assistance programs help some - covering 40% of costs on average - but they’re not automatic. You need to apply. You need paperwork. You need a case manager who knows how to navigate the system. And not everyone has that support.

The system isn’t designed for fairness. It’s designed for profit. And when you’re sick, you’re not negotiating. You’re desperate.

What Can You Do If You’re Facing a High-Cost Drug?

If you’re staring at a $100,000-a-year prescription, here’s what actually works:

• Ask for the patient assistance program - every major drugmaker has one. Call the number on the box. Don’t assume you make too much. Many programs cover people up to 500% of the federal poverty level.
• Use a specialty pharmacy - they don’t just ship your meds. They have case managers who fight with your insurer for prior authorizations and appeals. They know the tricks.
• Check nonprofit foundations - the Chronic Disease Fund, Patient Access Network, and HealthWell Foundation gave over $2 billion in help in 2022. You don’t need to be poor to qualify.
• Ask your doctor for alternatives - not every expensive drug is the only option. Sometimes a slightly older, cheaper drug works almost as well with fewer side effects.
• Know your Medicare Part D phases - the donut hole isn’t a myth. Learn when you hit it. Use tools like Medicare’s Plan Finder to compare costs across plans.

The Bigger Picture: Is This Sustainable?

The U.S. spends more on prescription drugs than any other country - and gets less value for it. In Europe, 56% of the most expensive drugs were rated as having low or no added benefit. In the U.S., they’re just covered.

The Inflation Reduction Act started drug price negotiations in 2024 - but only for 10 drugs, and only if they’re over 12 years old. Most of the ultra-expensive drugs - the ones with the biggest side effects and highest costs - are still off-limits.

Experts agree: we need a system that ties price to actual benefit. Not just innovation. Not just rarity. Real, measurable improvement in how people live. Right now, we’re paying for hope. And sometimes, hope costs too much.

Final Thought: It’s Not About the Price - It’s About the Choice

No one wakes up and says, “I want to spend half my savings on a drug that makes me sick.” But when your options are: die slowly, or risk a bad reaction to save your life - you don’t choose the cheap one. You choose the one that gives you back your days.

The real question isn’t whether these drugs are worth it. It’s whether society should let people make that choice alone - with no safety net, no price control, and no guarantee they’ll be able to afford it.

Next Steps: What to Do Right Now

If you’re considering an expensive medication:

1. Ask your doctor: “Is there a cheaper alternative with similar results?”
2. Call the drug manufacturer’s patient support line - ask for the assistance program.
3. Visit PatientAssistance.com or NeedyMeds.org to search for nonprofit aid.
4. Ask your pharmacy for a specialty pharmacy referral - they handle prior authorizations and appeals.
5. Review your Medicare Part D plan during open enrollment - switching plans can save thousands.

The system isn’t fair. But you don’t have to navigate it alone. Help exists. You just have to ask for it.