Prices for new US drugs doubled in 4 years as focus on rare disease grows
May 22 (Reuters) - U.S. prices for newly-launched pharmaceuticals more than doubled last year compared to 2021, as companies leveraged scientific advances to develop more therapies for rare diseases, which typically command high prices, a Reuters analysis found.
The median annual list price for a new drug was over $370,000 in 2024, according to the Reuters survey of 45 medicines.
In 2021, the median price was $180,000 for the 30 drugs first marketed through mid-July, according to a study published in JAMA based on the same criteria. The median launch price was $300,000 in 2023 and $222,000 in 2022.
The increase in prices has occurred even as the U.S. government tries to rein in prescription costs. Drug pricing has become a populist issue for President Donald Trump, who has called for drugmakers to bring U.S. prices in line with other high-income nations that pay far less.
William Padula, professor of pharmaceutical and health economics at the University of Southern California, said there is no indication that the trend will slow — at least until there is progress in lowering the cost of developing new therapies.
"For years we've had pretty good technology and solutions for a lot of the common conditions that many people have, like high cholesterol, high blood pressure, and managing the more common forms of cancer," Padula said. For rare diseases, there are fewer patients "and therefore the price per course of treatment is going to go up," he said.
The percentage of drugs launched for orphan diseases, meaning they affect fewer than 200,000 Americans, rose to 72% in 2024 from 51% in 2019, according to the Iqvia Institute for Human Data Science. Over 40% of the orphan launches were for oncology.
The other 28% included drugs for larger populations, such as schizophrenia drug Cobenfy, sold by Bristol Myers (BMY.N), opens new tab at a list price of $22,500 a year.
The leading industry trade group, the Pharmaceutical Research and Manufacturers of America, said focusing on list prices for drugs that treat rare diseases "misses the broader context of how these drugs contribute to overall prescription drug spending, healthcare costs and value to patients."
Decoding of the human genome, completed in 2003, has paved the way for better understanding of the genetic and biological underpinnings of rare diseases, leading to advancements in medical science.
Drugmakers are given incentives to invest in research for rare diseases, including longer periods of market exclusivity, in part because potential sales may be limited.
Boston Consulting Group projected the 2024 crop of drug launches would reach peak annual sales of $60 billion, significantly lower than past averages due to the absence of mega-blockbusters, a term used to describe drugs with annual sales above $10 billion.
The FDA approved 57 novel drugs last year, including seven new cell and gene therapies at the agency's biologic division. In 2023, the agency approved 55 drugs and 17 new biologics.
Reuters surveyed the makers of 45 new drugs launched last year. The price analysis excludes imaging agents, vaccines, drugs used intermittently such as antibacterials, and products that have not yet launched commercially.
The highest price for a drug taken consistently was over $1 million a year for Zevra Therapeutics' (ZVRA.O), opens new tab Miplyffa for Niemann-pick disease type C, an inherited metabolic disorder diagnosed in about 900 people in the United States.
Orchard Therapeutics' Lenmeldy gene therapy for a rare inherited disorder that affects the brain and nervous system, was launched last year at a record-high price of $4.25 million for a one-time treatment.
Pfizer's (PFE.N), opens new tab hemophilia gene therapy Beqvez was priced in 2024 at $3.5 million, but the company pulled it from the market less than a year later citing soft demand.
Pharmaceutical companies say new medicines offer cost-saving value, including potentially fewer emergency room visits and hospital stays, and with some treatments using gene editing, the possibility of a cure.
Drugmakers also stress they do not determine the portion of drug costs that are born by patients under health insurance plans.
Many offer savings cards and other programs to reduce out-of-pocket costs, while insurers can receive discounts and rebates from manufacturer list prices, especially if competing treatments are available.
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