Cost vs Benefit: When Expensive Medications Are Worth the Price Despite Side Effects

When a medication costs more than a car-or even a year’s rent-how do you decide it’s worth it? For many people living with rare diseases, cancer, or severe autoimmune conditions, this isn’t a theoretical question. It’s daily life. These drugs can save lives, but they often come with brutal side effects and financial burdens that stretch families to the breaking point. So when does the price and the risk actually make sense?

Why Some Drugs Cost More Than Your Salary

The most expensive medications today aren’t just expensive because they’re new. They’re priced that way because they’re engineered for tiny patient groups. Think of drugs like emibizumab for hemophilia or tisagenlecleucel for certain leukemias. These aren’t pills you pick up at the corner pharmacy. They’re gene therapies, monoclonal antibodies, or personalized cell treatments made one patient at a time. The manufacturing process alone can take months and cost hundreds of thousands of dollars just to produce a single dose.

In 2024, the median cost per dose for the 50 most expensive drugs covered by U.S. Medicare hit over $16,000. Thirty-one of those were biologics-complex proteins made from living cells. Sixteen were small-molecule drugs. Three were gene therapies. And 72% of them were for rare diseases, protected under the Orphan Drug Act. That law was meant to encourage innovation for conditions affecting fewer than 200,000 Americans. It worked. But it also created a market where companies can charge whatever they want because there’s no competition.

Side Effects Aren’t Just a Nuisance-They’re a Trade-Off

No drug is perfect. Even the most effective ones come with trade-offs. For example, CAR-T therapy can wipe out cancer cells in patients who’ve run out of options. But it can also trigger cytokine release syndrome-a dangerous immune overreaction that requires ICU-level care. Patients might spend weeks in the hospital, feverish, shaky, and hooked to IVs. The risk is real. So why do people still choose it?

Because the alternative is worse. A 2023 patient forum from the American Society of Clinical Oncology found that 78% of people who received CAR-T therapy said it was worth the side effects. Why? They were dying. Their bone marrow was failing. Their tumors were spreading. They’d tried chemo, radiation, targeted drugs-all failed. CAR-T gave them a shot at remission. Side effects? Temporary. Death? Permanent.

Same goes for hepatitis C drugs like Harvoni. In 2016, a full course cost over $7,000 out-of-pocket. But before Harvoni, the standard treatment was interferon injections-given weekly for up to a year. Side effects included severe fatigue, depression, nausea, and flu-like symptoms so bad that many patients quit. Harvoni cured 95% of patients in 12 weeks. The side effects? Mild headaches or tiredness. The cost was high, but the benefit was life-changing.

How Doctors Decide: It’s Not Just About the Disease

Doctors don’t just look at whether a drug works. They weigh five things:

1. How much longer will it extend life? Some drugs add a few months. Others add years.
2. How much better is daily life? Can you walk without pain? Sleep through the night? Work again?
3. Are there safer, cheaper alternatives? If yes, why isn’t that being tried first?
4. What’s the long-term cost of not treating? Untreated hemophilia leads to joint destruction. Untreated MS leads to paralysis. The cost of ignoring the disease can be higher than the drug.
5. Can you manage the side effects? Some side effects can be controlled. Others can’t.

Take Revlimid, used for multiple myeloma. In 2016, patients paid over $11,500 out-of-pocket in a single year. Many skipped doses because they couldn’t afford it. But for those who stuck with it, survival rates jumped from under 3 years to over 7. The side effects? Low blood counts, fatigue, nerve pain. But with monitoring and supportive care, most patients could manage them.

Insurance Isn’t a Safety Net-It’s a Maze

You might think insurance covers these drugs. It doesn’t-not really. Medicare Part D has a coverage gap called the “donut hole.” Even after you pay your deductible, you might still owe 25% of the drug’s list price. And for drugs over $10,000 a month, that’s still thousands of dollars.

A 2022 survey found that 68% of patients taking drugs over $10,000 a month skipped doses because of cost. 42% chose between medicine and food. That’s not a statistic. That’s a parent skipping their insulin so their kid can eat.

Prior authorization can take two weeks. Specialty pharmacies require you to call daily. Insurance companies demand step therapy-try three cheaper drugs first, even if they’ve already failed. And even when you get approved, the drug might not be in stock. One patient with a rare blood disorder waited six weeks for her drug to arrive. By then, her platelet count had dropped dangerously low.

Help Is Out There-If You Know Where to Look

You don’t have to fight this alone. Manufacturer patient assistance programs cover an average of 40% of out-of-pocket costs for commercially insured patients. Foundations like the Chronic Disease Fund gave over $2 billion in aid in 2022. Specialty pharmacy case managers spend over three hours per patient just navigating paperwork.

Here’s what actually works:

• Ask your doctor for the drug’s patient assistance program-most have one.
• Call your pharmacy and ask for a case manager. They handle insurance, appeals, and delivery.
• Check NeedyMeds.org or GoodRx for coupons and discounts-even for ultra-expensive drugs.
• Apply for Medicaid expansion if you’re under 138% of the poverty line. Some states cover drugs that Medicare won’t.
• If you’re on Medicare, ask about Extra Help (Low-Income Subsidy). It can cut your out-of-pocket costs by 90%.

When the Price Is Just Too High-Even If It Works

Not every expensive drug is worth it. A 2024 study found that 56% of the top 50 most expensive drugs were rated as having low or no additional benefit by European health agencies. Some cancer drugs extend life by just 2.5 months at a cost of $150,000. That’s $60,000 per extra month. Is that value? Or exploitation?

Dr. Peter Bach from Memorial Sloan Kettering built a tool called DrugAbacus to measure drug value. He found that four out of ten new cancer drugs approved in 2018 offered little benefit for their price. These drugs weren’t curing anyone. They were just delaying death a little longer-at a cost most people couldn’t afford.

That’s why some doctors push back. They’ll say: “We have a cheaper drug that works 80% as well. Let’s try that first.” Or: “This new drug adds three months, but you’ll be in the hospital half that time. Is that what you want?”

What’s Changing-and What’s Not

The Inflation Reduction Act of 2022 lets Medicare negotiate prices for 10 drugs starting in 2026. But here’s the catch: 96% of the most expensive drugs are currently excluded. Why? Because they’re too new, too rare, or not on the list. The law doesn’t fix the core problem: we still have no national standard for what’s a fair price.

In the UK, the National Institute for Health and Care Excellence (NICE) rejects about 25% of new drugs because they’re too expensive for the benefit they offer. In the U.S., there’s no such filter. A drug can cost $500,000 a year and still be covered-because there’s no one saying no.

Final Thought: It’s Not About the Drug. It’s About the Person.

There’s no universal answer. For one person, a $475,000 CAR-T treatment is a miracle. For another, the same drug is a financial ruin. The difference isn’t the drug. It’s the person’s values, their support system, their access to help, and their tolerance for risk.

If you’re facing an expensive medication, ask yourself:

• What happens if I don’t take this?
• What’s the worst side effect I can live with?
• What help is available to reduce the cost?
• Is this giving me more life-or just more suffering?

There’s no right answer. But there’s an informed one. And that’s the only thing that matters when your life is on the line.

When the price tag reads like a mortgage and the side effects feel like a battle, the choice isn’t easy. But it’s yours. And with the right information and support, you can make it without losing your dignity-or your wallet.