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Future-proofing health innovation regulation

IHI’s Regulatory Science Summit provided a forum to discuss how IHI projects can help overcome current regulatory challenges and contribute to regulatory science

21 March 2024
IHI and IMI projects can address research gaps in regulatory science. Image credit: EtiAmmos via Shutterstock.
IHI and IMI projects can address research gaps in regulatory science. Image credit: EtiAmmos via Shutterstock.

IHI funds health research and innovation at the precompetitive stage, and the results of our projects often go on to be used by the health industry to develop medical products. All pharmaceuticals and medical devices must undergo strict regulatory tests before being released onto the market, but there are several challenges associated with evaluating cutting-edge innovations.

IHI organised a Regulatory Science Summit on 27-28 February in Brussels, Belgium, to discuss the current issues that regulators see and identify how future IHI research projects could address these concerns.

Because IHI is a public-private partnership, it provides a neutral setting for regulators from the EU and the US to speak openly with the European Commission and industry partners about the research gaps that they encounter, and to brainstorm on whether IHI projects would be a good fit for addressing those gaps. During the Regulatory Science Summit, examples were provided on how to maximise the regulatory impact of IHI projects and optimise regulatory engagement.

This is a format that has worked under IMI – for instance, recommendations from a previous IMI Regulatory Science Summit were integrated into the topic text for IMI2’s eighteenth call related to advanced therapies. The two resulting IMI projects, T2Evolve and ARDAT, have engagement with regulators embedded into their work on cell and gene therapies.  

“This is a unique forum for us to meet across different sectors, discuss regulatory science and reflect on how IHI can contribute,” said IHI’s Executive Director Niklas Blomberg, speaking at the summit. “We are looking at the whole area of healthcare – medtech, diagnostics and pharmaceuticals in this collaborative and cross-sectorial space.”

The summit focused on five main themes: rare diseases, paediatric healthcare, real-world data, artificial intelligence (AI) and regulatory sandboxes.

New approaches for children and people with rare diseases

More than half of all rare diseases occur in childhood, and most are poorly understood with limited treatment options. Very often there are not enough rare disease patients for researchers to conduct traditional clinical trials.

The fourth IHI call specifically searched for new ways of doing clinical trials for rare and ultra-rare diseases. The summit heard about what else could be done to optimise approaches and tools for the development of medicines, devices and diagnostics for rare diseases as well as about the possibility of clustering diseases and the implications that this could have.

Alongside recruitment, there are other challenges making it tricky to run clinical trials in children.  There are two key differences between adults and children: they are smaller than adults and they grow. In the domain of medical devices, that means various sizes are needed, and the device needs to be able to grow with the child. Although frequent updates are made to adult versions of devices, children’s devices rarely go through the same improvement cycles, and there are far fewer medical devices designed specifically for children to begin with. The IMI c4c and ITCC-P4 projects have been transformational for paediatrics development but more is needed.

At the summit, participants debated what could be done to optimise paediatric and neonatal research more broadly, and pondered how it might be possible to build an integrated ecosystem to speed up paediatric drug and technologies development and improve access to health solutions adapted for children.

Harnessing real-world data for sound science

Using real-world data – harnessing data that is already in existence in hospitals for instance – could amplify what we know about patients and healthcare. But is that data robust enough for regulators to accept? Standards for real-world data need to be clearly defined to ensure that data of sound scientific quality is collected, and IHI research could help to achieve that.

In addition to success stories from IMI projects on health data such as EHDEN, IHI is already taking steps in this space. The IHI IDERHA project is developing a platform integrating real-world data from diverse sources (e.g. collected using wearable devices or routine clinical care) which could yield insights into how robust real-world data can be collected, and IHI’s sixth call is currently searching for proposals to develop practical guidance on how to use real-world evidence.

Building on the IMI/IHI portfolio and other EU initiatives, the discussion at the summit focused on how to generate evidence that meets diverse needs and requirements as well as considering how best to achieve alignment on common methodologies and foster greater understanding on the use of real-world evidence to support decision-making.

Preparing safe deployment for AI in healthcare

The use of AI within healthcare settings is still in its infancy but there is no doubt that the importance of this technology will rapidly grow. However, only the most trustworthy AI tools should be utilised for healthcare purposes; at present, some AI systems like ChatGPT fabricate data when they do not have sufficient information, and this would be unacceptable in a healthcare setting.

IMI projects have already pioneered AI solutions to further health innovation – the IMI2 MELLODDY project developed a machine learning (ML) platform using blockchain that joins up multiple small molecule datasets without compromising confidentiality. The summit examined what IHI could do to contribute to safe deployment of AI.

De-risking innovation with regulatory sandboxes

A regulatory sandbox allows a health innovation that might challenge conventional regulatory frameworks to be tested under the supervision of a regulator. They are “safe spaces” for innovation, transforming the way that regulatory agencies typically work and opening the door towards the development of innovative medicinal products and technologies and their combinations.

Considering the introduction of regulatory sandboxes in the Commission proposal for revising the EU’s pharmaceutical legislation, the summit explored how IHI projects could be useful to enable the successful implementation of regulatory sandboxes that could enable cutting-edge developments and disruptive innovations.

Wrapping up the summit, Executive Director Niklas Blomberg spoke about the “seeds of ideas” that were planted for future IHI projects and how open discussions with regulators in a trusted environment could help to accelerate regulatory science going forwards. A more detailed report on the IHI Regulatory Science Summit will be published in the coming months – watch this space! Don’t forget to sign up to our newsletter to stay up-to-date with all the latest news from IHI.