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

Jan, 4 2026

When a drug costs more than a car, or even a year’s rent, it’s hard not to wonder: is this worth it? For many people living with serious illnesses, the answer isn’t about money-it’s about survival. But deciding whether to take an expensive medication isn’t just a personal choice. It’s a balancing act between cost, side effects, and real-world outcomes. And for some, the trade-off makes perfect sense.

Why Some Drugs Cost So Much

The price tag on modern medicines isn’t random. Many of the most expensive drugs are biologics, gene therapies, or treatments for rare diseases. These aren’t your typical pills. They’re complex, often made using living cells, and require years of research to develop. The Orphan Drug Act of 1983 gave companies financial incentives to treat diseases affecting fewer than 200,000 people in the U.S. That’s why drugs for conditions like hemophilia, spinal muscular atrophy, or certain rare cancers now carry prices between $500,000 and $2 million per treatment.

In 2023, the median cost per dose of the 50 most expensive drugs covered by Medicare hit $16,178. Thirty-one of those were biologics. Three were gene therapies. And 72% of them had orphan drug status. These aren’t just expensive-they’re engineered for precision. But that precision comes at a steep price.

Side Effects Aren’t the Only Risk

When people think of side effects, they imagine nausea, fatigue, or rashes. But with high-cost drugs, the risks go deeper. CAR-T cell therapy, for example, can trigger cytokine release syndrome-a dangerous immune overreaction that requires hospitalization. Some patients get sick enough to need intensive care. But for those with terminal leukemia who’ve run out of options, that risk is acceptable. In a 2023 ASCO patient forum, 78% of people who received CAR-T therapy said it was worth the side effects, even with the hospital stays and recovery time.

Similarly, hepatitis C drugs like Harvoni cost $7,153 out-of-pocket in 2016. But they cured 95% of patients in just 12 weeks. Before Harvoni, the standard treatment involved weekly interferon injections that caused flu-like symptoms, depression, and often failed. The side effects of the old treatment were brutal. The side effects of Harvoni? Mild headaches or tiredness. The benefit? A clean bill of health. For many, that trade-off was obvious.

When the Alternative Is Worse

Sometimes, the real question isn’t whether the expensive drug is worth it-it’s whether the alternatives are worse. Take rheumatoid arthritis. Methotrexate is cheap. But for many patients, it doesn’t work well enough. The pain, joint damage, and loss of mobility become unbearable. Biologics like Humira or Enbrel can cost over $70,000 a year. But they stop the disease in its tracks. Patients report being able to play with their kids again, go back to work, or sleep through the night.

A 2022 survey by the National Health Council found that 68% of patients on drugs costing over $10,000 a month skipped doses because they couldn’t afford them. But those same patients also said they’d rather risk financial ruin than go back to uncontrolled pain or worsening symptoms. For them, the cost of doing nothing was higher than the cost of the drug.

Insurance and the Hidden Cost of Access

Even if your insurance covers the drug, you’re not out of the woods. Medicare Part D has a coverage gap-the infamous “donut hole”-where patients pay nearly everything out of pocket until they hit catastrophic coverage. In 2021, over 2.3 million Medicare beneficiaries hit that gap. For a drug like Revlimid, which costs $11,538 out-of-pocket in a single year, that means paying thousands after insurance kicks in.

And here’s the kicker: the list price you see isn’t what insurers pay. Manufacturers offer secret rebates to pharmacy benefit managers. The real cost is hidden. A 2023 study found that the net price after rebates was often 30-60% lower than the sticker price. But patients don’t benefit from those rebates unless they’re on Medicaid or have special coverage. Most people still pay based on the inflated list price.

Who Gets Left Behind?

The system isn’t fair. People with lower incomes are far more likely to skip doses. In 2023, 68% of Medicare Part D enrollees earning below 150% of the federal poverty level delayed or skipped their medication due to cost. For higher-income patients, that number was 22%. That’s a massive gap.

Patients with hemophilia, for example, might need emicizumab, which costs $15,000 a month. Without financial help, that’s impossible. But through manufacturer assistance programs, many get their co-pays reduced by 40% on average. Foundations like the Chronic Disease Fund gave out $2.1 billion in aid in 2022 alone. Still, navigating these programs takes time, paperwork, and persistence. Many patients give up before they even start.

When Does It Make Sense?

So when is an expensive drug worth it? Here’s a simple framework:

• It saves your life or prevents irreversible damage. Think cancer remission, preventing liver failure from hepatitis C, or stopping joint destruction in autoimmune disease.
• It’s significantly better than existing options. If the new drug cuts treatment time from a year to a month, or reduces side effects from severe to mild, the cost may be justified.
• There are no cheaper alternatives that work. If generics or older drugs have failed, the expensive option becomes the only real choice.
• You can access financial help. Manufacturer programs, nonprofit aid, and state assistance can cut costs by half or more. Don’t assume you can’t afford it until you’ve explored every option.

What You Can Do

If you’re facing a high-cost medication:

1. Ask your doctor for the net price after rebates. You might be surprised how much lower it is.
2. Request a prior authorization letter. Sometimes, insurers will approve the drug if you prove other treatments failed.
3. Contact the drug manufacturer. Nearly all have patient assistance programs. You can often get the drug for free or at a steep discount if you qualify.
4. Work with a specialty pharmacy. They assign case managers who handle insurance, appeals, and paperwork-often spending over 3 hours per patient to get coverage.
5. Apply for aid from foundations like the Patient Access Network Foundation or the Chronic Disease Fund. They don’t ask for collateral or credit checks.

The Bigger Picture

The U.S. spends more on drugs than any other country-$621 billion in 2022. Yet, only 15% of new drugs are rated by independent groups like Prescrire as offering major therapeutic progress. Many are slight improvements over existing drugs, priced 10x higher. That’s the system’s flaw: profit often drives pricing, not value.

But for the people who need these drugs, the system works-not because it’s fair, but because it gives them a chance. A child with spinal muscular atrophy who can now walk. A woman with multiple sclerosis who no longer needs a cane. A man with hepatitis C who’s cured after 20 years of liver damage.

These aren’t just statistics. They’re lives. And sometimes, paying $500,000 for a one-time cure is the only rational choice.