High-Cost Trend in New U.S. Drugs Continues, Driven by Rare Disease Therapies

May 22 (Reuters) – The median annual list price for new drugs in the U.S. surged to over $370,000 in 2024, more than double the median price from 2021 ($180,000). This escalating trend is primarily attributed to a growing focus on therapies for rare diseases, which command significantly higher prices due to smaller patient populations and specialized research.

A Reuters analysis of 45 new medicines launched last year found that 72% were for orphan diseases, affecting fewer than 200,000 Americans, a notable increase from 51% in 2019. Over 40% of these orphan drug launches were in oncology.

Experts like William Padula of USC indicate that this pricing trajectory is unlikely to slow without advancements in reducing drug development costs. While the U.S. government, including President Donald Trump, has pushed for lower prescription costs, the industry emphasizes the “value” of these new medicines, citing potential for reduced hospital visits and even cures, especially with gene therapies.

Notable high-priced launches in 2024 included Zevra Therapeutics’ Miplyffa at over $1 million annually for Niemann-pick disease type C, and Orchard Therapeutics’ Lenmeldy gene therapy at a record $4.25 million for a one-time treatment. Pfizer’s hemophilia gene therapy Beqvez, launched at $3.5 million, was later pulled due to soft demand.

Drugmakers also highlight that list prices do not reflect patient out-of-pocket costs, often mitigated by savings programs and insurer rebates. The industry attributes scientific advances, particularly from human genome decoding, as enabling these complex, high-value therapies for previously untreatable rare conditions, noting regulatory incentives for such research. The FDA approved 57 novel drugs last year, including seven new cell and gene therapies.

Packgene is dedicated to affordable gene and cell therapy by offering innovative and cost-efficient manufacturing solutions.