Overcoming bottlenecks in the advanced therapy supply chain

In recent years, the pharmaceutical industry has had to grapple with the scalability of advanced therapies. Yet as the traditional issue of production capacity eases, what has been given far too little attention, so far, is whether the required digital and supply chain management infrastructure is in place.

The personalised nature of each treatment means every application comes with an unusually high administrative burden, making the wider adoption simply unworkable in many clinical settings. In fact, while there have been workable systems introduced for approved therapies, these remain unique, and are forcing hospitals and doctors to deal with an unmanageable amount of admin, since every new product that comes to market comes with a new system.

In this Q&A, Prof Martin Bornhäuser – Principal Investigator at University Hospital Carl Gustav Carus Technische Universität Dresden and the founding member of the Center of Regenerative Therapy Dresden discusses real-world challenges facing clinicians working on advanced therapies.

He believes that while there is not yet a single panacea to this problem, what we need is the ability to link more systems together, for example, an interoperable advanced therapy medicinal products (ATMP) platform that aims to connect existing systems and consolidate them into a single solution. The objective is to build a new platform that not only addresses these admin-related problems but also has the potential to transform how we collect, store and analyse data. Collaborating with technology providers and designing new links between systems that help clinicians overcome these administrative burdens will play a crucial role in simplifying the complexities associated with advanced therapy delivery.

What is the current situation in hospitals with cell and gene therapy tracking?

The first thing is to distinguish between clinical trials and approved therapies. In the EU there are already some five advanced therapies approved. The issue is that each have very different approaches to tracking. In data collected, in data input and different systems, it is a large administrative challenge. For clinical trials, this is obviously even more complicated with the need for extra safety data. But for this, we are more prepared and supported by the trial sponsors. However, for approved products there is very often little support and this limits our work due to an increased administrative burden.

What problems are you facing in Germany?

Germany has 16 federal states and each one has different regulatory issues, and this introduces a further level of complexity in ordering and supply. The systems we have in place were not developed with advanced therapies in mind that go from the patients, are adapted and then returned to the patient.

What is the regulatory perspective on these issues?

The administrative burden is not really something that the regulatory bodies make any effort in addressing, as they are solely interested in safety. The ideal solution would be a centralised approach to this from the regulators down.

What is the ideal solution for moving past these obstacles?

The biggest challenge is with different stakeholders being willing to work together to create entry points into their systems. For example, could the patient management system we use talk to another system from a cell and gene therapy company? At present, most of the approved therapies use completely separate systems outside of the patient management system (PMS).

Therefore, the ideal is a new system that can talk directly with the existing PMS. Having one cell and gene therapy system for a number of approved therapies rather than just one, may be needed before a new system can be put in place.

One technology solution we are conducting a pilot programme with is Hataali. This is an interoperable ATMP platform for innovators and clinicians and that tracks therapies and potentially provides biobanking and smart lab procurement. The current trial is to investigate the feasibility of using the platform layer to talk directly with the existing PMS and already we are seeing some promising results.

How feasible is bringing onboard several advanced therapies in early-stage trials onto one system?

Onboarding several ATMPs in pre-clinical trials onto one system might be a step change approach. This is because it is more likely than having the approved therapies open their existing systems up. Unfortunately this is unlikely as they have a working solution for their therapy; the by-product is that this is also helping block future market entrants. So there is not much incentive for the big pharma innovators in this case to open up. However, for biotechs in clinical trials, this might be the way to go because it would bring many benefits to all parties involved and would hopefully help expedite these products to patients. Anything that takes away some of the burden of multiple data and patient records entry would be a tremendous improvement. It would also lower the cost of the trials for the sponsors who need to provide a lot of staff to help clinicians overcome this administrate challenge.

What are the advantages of a shared or open platform for advanced therapies?

What we are talking about here is a step towards this. Firstly, I am sure we are heading, albeit slowly, towards open platforms for advanced therapies. One advantage is that with a more open cross trial platform, there would be tremendous improvements in our ability to do research. For example, one could do joint analyses and off desk activities. It would vastly increase the flexibility and help in the design of new trials through learning from similar studies and help usher in a new age of collaborative data. The long-term benefits of this increased data access and shareability could transform the insights we garner both post trail and future therapies.

What is your call to action for the industry?

We must have the current information system providers and/or innovators open up access to their systems and the regulators need to push innovators to use common standards or approaches too. We are not talking about access to everything, but perhaps a cloud-based system – or a blockchain one that sits above existing ones and takes data security seriously.

However, as cell therapies are so expensive, I believe within the next five years insurance companies could say they are too expensive, and that we need a new approach to bring these costs down. So while manufacturing costs are high, the administrative burden of these therapies is also huge. Ultimately, this might force the change upon us, but it would be far better if as an industry we could implement it proactively, seeing the tremendous improvements this could bring and the longer-term ecosystem that all stakeholders could benefit from.