Cell and gene therapies (CGTs), which modify genes, gene expression or the biological properties of cells, have revolutionised medicine, providing personalised and targeted treatment for life-limiting diseases from genetic disorders to hard-to-treat cancers. Around twenty CGTs have been approved, and many more are in development. However, these cutting-edge therapeutics can come with a high price tag. [1]
Launched in the US in March 2024, Orchard Therapeutics’ Lenmeldy (atidarsagene autotemcel) is reported to be the most expensive drug in the world, with a price tag of $4.25 million. A one-time treatment, atidarsagene autotemcel is a gene therapy for metachromatic leukodystrophy (MLD), a fatal lysosomal disorder that leads to loss of sensation, cognition and motor skills. Infants usually die by five years of age, with patients with the juvenile and adult forms dying within 20 and 14 years of onset. Nine of the top ten most expensive drugs in the US are gene therapies, costing between $2.2 million and $4.25 million. [2, 3]
The cell and gene therapy market was worth around $18.12 billion in 2023, and is predicted to rise to $21.28 billion in 2024 and $117.46 billion in 2034, rising at an estimated compound annual growth rate (CAGR) of 18.6%. [4]
The high cost of cell and gene therapies has a number of drivers. Developing these types of therapies is high risk and complex, with years of clinical trials. The manufacturing process involves many steps, especially when the therapeutics are created for individual patients, there is only limited manufacturing capacity and only certain reagents are approved for use. The regulatory processes can be slow, especially as the technologies are new to the regulators. [1]
Looking at long-term costs
Many CGTs are ‘one and done’ treatments, and so the initial high cost needs to be balanced against a lifetime’s cost of drug and other treatments. Improving patients’ health also allows them to contribute to society through education and work, and through involvements in family and community life.
Using licensing agreements to control pricing
Licensing agreements, especially when assets arise from academic research supported by government funding, can be drafted to include clauses that keep prices lower for lower-income groups, and make sure that products are registered in lower-income countries to ensure access. The agreements could also be designed to ensure that exclusive licenses revert to non-exclusive licenses to stop companies shelving projects but retaining intellectual property.
Streamlining drug approval
Regulatory authorities can put in place programs that support CGT research, development and approval, for example the FDA’s Breakthrough Therapy Program and Regenerative Medicine Advanced Therapy designation.
The Breakthrough Therapy Program, which was implemented in 2012 and updated in 2018, was created to expedite the development and review of drugs for serious conditions that have potential to offer significant improvements over existing therapies. Companies developing a Breakthrough Therapy Program-designated drug can access: [5, 6]
· Fast Track designation – more frequent FDA meetings and written communications, and eligibility for Accelerated Approval and Priority Review and Rolling Review
· Guidance on drug development, starting from Phase I
The Regenerative Medicine Advanced Therapy (RMAT) designation, part of the 21st Century Cures Act, covers regenerative medicine therapy (cell therapy, therapeutic tissue engineering product, human cell and tissue product and certain gene therapies) developed to treat, modify, reverse or cure a serious or life-threatening condition. RMAT designated therapies gain the benefits of Fast Track and Breakthrough Therapy designation, as well as having the potential of accelerated approval. [6, 7]
Some CGTs are developed for ultra-rare disorders with existing treatments, where the patient numbers are so low that there is little chance of an economic return on the treatment. If regulators could allow continuous treatment under phase I-type cGMP (current good manufacturing...
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