Alliance for impact – advancing CGT development in Europe

The development of cell and gene therapies (CGTs) is a dynamic and evolving area, challenging technology, legislation and financial models for the pharmaceutical industry. CGTs have positioned themselves as the new superhero drugs, swooping in to challenge unmet clinical needs, with the potential to establish new cures for previously life-limiting conditions and opportunities for one-time approaches to treating diseases.

Europe’s position in the advanced therapy market

As a hub for innovation and scientific excellence, Europe is serving as a gateway for many new drug candidates and novel drug targets. However, investment to bring these novel treatments to market in the region is a challenge. Many drugs in early development are being siphoned by large Pharma or major investors and transitioned into the North American system to complete their development and market approval.

The recent shift in global geopolitics has rippled through the financial markets, changes that have strongly impacted the pharmaceutical industry. For Europe, this has been an opportunity to demonstrate the advantages of developing such high value drugs within the region.

As a firm competitor to the US market for the entire product lifecycle, Europe has marketed itself as a diverse, strong market that enables pharmaceutical businesses to establish themselves, invest and grow within the region.

Individual EU governments are establishing initiatives to support companies and academics with new concepts to spin out into the commercial sector, heavily investing especially within the CGT sector. This includes initiatives such as ATMP 2030 led out of Sweden. Regional hubs, specialising in CGT manufacturing, process and product development and training to increase specialised workforces within the region have also been established, and thrive.

This shift in business model, to keep investigational drugs inside of Europe is impacting the ancillary service industry as well, with strong investment in contract manufacturing spaces, new quality control analytics companies and hubs for raw materials such as cell culture media.

Of course, the ultimate aim is not to shift the primary region for developing CGTs, but rather see a wealth of knowledge, experience and opportunities across the globe. Collaboration and alliance are key to the successful integration of CGTs globally into standard healthcare.

Addressing the challenges of cell and gene therapy development and market entry

Society faces unique, global challenges with the evolution and establishment of CGTs in medical practice. These include:

Manufacturing, CMC and scalability

The lack of technological solutions to manufacturing and scaling of production was once the core issue facing CGT developers.

However, scalability now enters the debate of centralised versus decentralised manufacturing. This risk-based approach, considering the product on a case-by-case basis and bespoke to product development strategy truly highlights the unique challenges the industry faces.

The speed of advancements in engineering, digitalisation, artificial intelligence (AI) and product design far outpaces the speed at which regulations move, however. The introduction of rapid microbial methods being one such example, with global regulators having different viewpoints on the introduction of these technologies, moving away from traditional compendial methods.

While regulators are in favour of advancements that benefit the patient through accessibility and speed of manufacturing and drug product release, caution remains, safeguarding the patient until technology has proven itself through risk management, comparability testing against compendial methods and, within Europe, stringent validation and QP approval.

Regulatory alignment and harmonisation

A need for regulatory alignment and harmonisation across jurisdictions, to truly move towards global availability of such specialist drug products, has received much attention recently.

For example, the US Food and Drug Administration (FDA), Japanese PMDA and the European Medicines Agency (EMA) are aligning their requirements while minimising risk to patient safety or product quality. Many CGTs have also taken advantage of expedited market entry schemes and exclusivity options.

Investment and reimbursement

Innovative medicines such as CGTs are associated with high costs. The traditional route to market through clinical trials offers unique challenges to CGT developers from onboarding investors, through to payment for the resulting drug itself.

Reimbursement remains one of the true open questions right now. Of course, the aim is to have equal access across all EU Member States, eliminating the risk that CGTs become a premium product. At present, the EU has a single regional approach to pricing. While there may be economy in scale, the challenge is to model how new drugs on the market will be valued. There needs to be balance, especially for a global marketing strategy where drug prices are historically higher in regions such as the US to that of Europe.

Where Europe exhibits strength in innovation, many countries demonstrate a creative and solution-driven environment. A collaborative approach will help deliver successful adoption of new therapeutic modalities, especially considering the current challenges of CGT development.