Cell and gene therapies: how to encourage and promote the move to market

The evolution of advanced therapy medicinal products, or ATMPs, is bringing real world results.  

The number of cell and gene therapy products now on the market around the world is near the 100 mark, according to recent research by McKinsey & Company, with many more in development. These ground-breaking therapeutics can have a profound impact on previously intractable conditions. Products like Novartis’s gene therapy Zolgensma, for example, are costly but have the potential to act as a single-dose cure for young patients with spinal muscular atrophy. London-based Orchard Therapeutics has had some important wins with its ex vivo autologous gene therapy approach, as it seeks to address multiple therapeutic areas with profound unmet need.

The UK’s powerful research base and established life sciences sector means that it is well placed to be part of this story. The Cell and Gene Therapy Catapult reports that, in 2020, the UK was host to around 12% of ongoing ATMP global clinical trials – some 154 separate studies. A striking statistic. 

As more products are rolled out to patients, the journey for others both in overcoming technical obstacles and gaining public acceptance improves. But in the global race to bring these sophisticated products through from concept to clinic, some specific challenges stand in the way.

One of these is the need for a different style of regulatory engagement. The traditional methods used by regulators in dealing with small molecule pharmaceuticals show major limitations when applied to ATMPs. Regulators have many years of in-depth experience with small molecule medicines. This can mean that regulatory guidance focuses on this section of the market and tends to be prescriptive in nature. In contrast, regulation of large molecule, cell and gene therapies should, say those close to the industry, be based more on a philosophy rather than prescriptive rules, and should allow a greater degree of flexibility. 

To remain at the forefront of this exciting sector, rapid technological progress needs to be matched by a surefooted regulatory response. With many ATMP projects created in the hands of smaller spin-outs or growing mid-sized companies there is no spare cash or capacity for mistakes. Developers and regulators need to work together to make sure that safe and effective therapies can reach the patients that so badly need them.

A collaborative approach

Developers of innovative therapies can gain real benefits from early and regular engagement with regulatory agencies. The aim here is to smooth the product development path and, at the same time, avoid wasted effort and expense. 

With development costs high, collaboration is a must to drive down complexity and uncertainty. The pressures of COVID-19 pandemic may have helped in strengthening the collaborative approach between regulators and developers, and many hope to see these changes embedded in day-to-day practice.

Think holistically

Regulatory strategy needs to form part of the overall development picture. For example, the shelf-life of ATMPs, and also their starting materials, can be very short. Building an understanding of the practical issues into the supply chain is essential. Issues around cross-border transfers with possible delays for customs checks can seriously undermine the business model for this generation of products.  Likewise, point of care manufacturing may be a necessary element of treatment – something regulators may allow for if they appreciate its importance. 

New ways to shape clinical trials

Designing clinical trials of ATMPs to make sure that they are not excessively cumbersome or open-ended requires early planning and engagement. Regulators are working on tools that can help innovators in this area, such as novel trial structures like basket or umbrella trials. These can be deployed to evaluate multiple hypotheses with the overall goal of improving the efficiency of trial evaluation. As clinical development progresses it may become necessary to adapt the trial, and regulators need to recognise this. Long term follow-up may also be necessary.

Regulators learning from each other

With advanced therapies setting new challenges, developers can take heart from seeing regulators learning from each other and sharing lessons internationally. Many products in this area address rare diseases, so a coordinated approach makes sense in serving a group of patients spread around the world. Likewise, regulators can benefit from looking to novel examples elsewhere. 

The Japanese authorities offer a special conditional approval that looks at safety and predicted efficacy, before allowing products a provisional authorisation for the market. This enables treatment availability to patients for a number of years during which evidence to establish efficacy is gathered.

An unexpected benefit

The COVID-19 pandemic, while stretching healthcare provision to its utmost, has had some positive impacts. Widespread recognition of the importance of life science innovation is one. But accelerating new approaches and ways of working with and between regulators is another less obvious benefit. We have looked at how this can strengthen and support cell and gene therapy development, and are optimistic that these changes are here to stay. And the UK government’s recent Life Sciences Vision shows a real commitment to pushing through in many of these areas.

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