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Numbers of gene and cell therapy trials are expanding rapidly. Here, we examine the market forces and regulatory pathways allowing this field to grow.
As of March 2023, 3,900 gene therapy trials were noted as ongoing, completed, or approved across 46 different countries. An analysis of the second quarter of 2024 by the American Society of Gene and Cell Therapy estimates that 2,068 gene therapies and 901 cell therapies are currently in development from pre-clinical to pre-registration stages. As of the first quarter of 2024, there were 32 gene and 68 cell therapies approved worldwide, as well as advances in genetic testing which are enabling improved treatment matching for patients with specific rare diseases.
Regulatory and market trends
As the global market continues to expand and more potential therapies like these move into development, dynamic regulatory frameworks which recognize the unique challenges, nuances, and new technologies involved in developing gene and cell therapies will play a vital role in enabling innovation in the field.
Accelerated approval pathways
Accelerated approval pathways are one key way in which governments are innovating to accelerate patient access to impactful rare disease treatments. The FDA's Accelerated Approval Program is a prominent example of this. As of January 2023, the program had approved more than 300 accelerated approval applications, including four gene therapies.
Other programs around the world include the UK’s Innovative Licensing and Access Pathway, which aims to support innovative approaches to the safe, timely and efficient development of medicines, as well as the European Medicines Agency’s (EMA) regulation of specific gene therapies as Advanced Therapy Medicinal Products.
Orphan drug designation
Orphan drug designation (ODD) is also supporting efforts to enable innovation while delivering life-changing therapies to patients at increased pace. ODD is a special status which can be granted to specific drug development efforts that focus on delivering therapies for rare diseases. Studies aiming to develop gene or cell therapies for rare disease can apply for ODD status which, in the US, comes with reductions in certain fees, exclusive marketing rights, and tax incentives.
ODD was designed to encourage companies to actively innovate in the rare disease space and is helping to drive cell and gene therapy development. Additionally, the associated tax incentives and fee reductions are an acknowledgement of the high costs involved in developing rare disease treatments, helping to make the cost of launching trials in these areas less prohibitive.
Aligning regulatory standards
There is also a growing trend towards aligning regulatory standards on a global scale, with multiple governments recognizing the need to align key benchmarks in order to scale and develop rare disease treatments in an efficient and streamlined manner.
Worldwide efforts are currently being made to unify and align standards with the aim of simplifying the cell and gene therapy process, harnessing international expertise and accelerating patient access to these treatments. For example, The International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) – a collaborative international network aiming to implement global best practices – has formed a specialist cell therapy group which focuses on discussing innovations and unique regulatory challenges within the cell and gene therapy landscape.
A careful balancing act is required to encourage and accelerate innovation in gene and cell therapies while at the same time ensuring due processes are in place to protect patient safety and understand the side effects and long term impacts of pioneering treatments.
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