Cell and gene therapy (CGT) provides advanced medical treatments that revolutionise the approach and treatments where traditional pharma and small-molecule drugs are not viable. The development of CGT is among the most exciting innovations occurring in pharma research today. CGT could lead to more personalised treatments and hold the promise to cure/ treat rare diseases and selected cancers. From the first USFDA approval in the last decade, by the end of 2023, 34 CGT received approval in the US, with hundreds more proposed therapies in the pipeline. However, despite promising outcomes, these emerging therapies are among the world's most expensive treatments, between $0.4m and above $4 million per dose.

Rare diseases getting boost amid CGT

There are significant synergies and opportunities between the Orphan Drug designation and CGTs, including economic incentives and rare disease targeting. According to the FDA, about 80% of rare diseases are caused by a single-gene defect, and gene therapies are used to treat them. With funding becoming increasingly competitive and labour shortages driving the cost of research and development up, the incentives offered by orphan drug status may become a cornerstone for the viability and success of numerous CGTs.

Promising treatment but expensive affaire

High production costs are limiting patient access to cell and gene therapies. According to the Institute for Clinical and Economic Review (ICER), the average cost of a gene therapy is between $1 million and $2 million per dose. The ICER also says that the average cost per treatment for cell therapy is $1 million, but prices vary depending on the product type and indication. The most expensive drug in the U.S. is now Lenmeldy, a $4.25 million gene therapy the FDA approved on March 18 for children with a rare genetic disease.

Drug manufacturers believe that their high prices are justifiable because their treatments have the potential to cure patients, which would ultimately lead to savings for the healthcare system and other societal benefits. In addition, drug companies want to recuperate the expenses they incurred in developing these treatments, which often target small populations, thereby limiting their revenue potential.

Top 5 most expensive approved CGT treatment