*

Median annual list price for new drugs over $370,000 in

2024

*

72% of new drugs in 2024 for orphan diseases

*

Drugmakers emphasize value, offer savings programs amid

rising

list prices

By Deena Beasley

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 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."

FOLLOWING THE SCIENCE

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 ) 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 ) 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.