IMI Impact: reinventing regulatory science, together (Guest blog)

The pace of medical innovation is accelerating, driven by big data, digital tools, and personalised medicine. This promises advances in how medical technologies are developed and deployed. More than that, it has the potential to deliver solutions that are more relevant to patients’ needs in the real world.
Ensuring that safe and effective innovations reach patients is the shared task of industry and regulators. The big question is: How can Europe’s regulatory system keep up with the fast-moving world of science and technology? The answer is collaboration.
The Innovation Medicines Initiative (IMI) is playing a key role in modernising clinical trials using new technologies and sources of data. By bringing together companies and regulatory authorities, along with HTA bodies and other stakeholders, IMI projects are creating a common understanding of the challenges we face. More than that, collaborative initiatives are delivering new knowledge that will shape the evolution of regulatory practice.
This is in line with the Regulatory Road to Innovation – our vision of an efficient, patient-centred system. Working within the existing legislative framework, it identifies four areas for action: the use of new types of clinical trials; greater use of real-world data; closer dialogue on emerging innovations; streamlined regulation of medicines and other healthcare products.  
Bigger, better data
As a public-private partnership, the IMI is perfectly placed to test the best in cutting-edge regulatory practice. Take ADAPT-SMART, for example. It has enabled regulators, industry, and others to test the feasibility of adaptive pathways to patients and has helped shape EFPIA’s concept of ‘dynamic regulatory assessment’. It has also helped to develop further our collective understanding of what decision-makers need to ensure patients have access to new therapies.
Several IMI projects have served as a catalyst for embracing new sources of evidence alongside data from clinical trials. BD4BO focussed on harmonising and standardising the use of big data, FAIRplus and EHDEN are building capacity by developing training in the use of data, while GetReal and ROADMAP explored how real-world data can answer scientific and regulatory questions more efficiently.
As regulatory science evolves, the patient should be centre stage at all times. Indeed, new tools can help ensure that clinical trials are better designed to meet the needs of patients. The Trials@Home project is exploring how digital technologies and wearable devices can be used to dramatically reduce the number of clinic visits patients are expected to make during a trial.
At the same time, IDEA-FAST is helping to identify digital endpoints that matter to patients while MOBILISE-D is exploring how digital mobility assessments could be integrated into regulatory decisions.
Patients first   
Together, we can make trial protocols more patient friendly and ensure that the fruit of this work reaches those who need it as efficiently as possible. For example, conducing individual trials with a single compound to test one hypothesis may generate high quality data. However, it is slow and expensive when scientists wish to test multiple compounds against similar hypotheses, or to test a single compound against multiple hypotheses.
IMI projects, including EU-PEARL, are demonstrating that we can achieve efficiency without sacrificing quality. For example, if multiple companies can test their candidate drugs simultaneously against a shared placebo group, studies become more efficient and patient friendly. Patients will be more likely to enrol if their chance of receiving a promising new therapy (rather than a placebo) is significantly higher.
By bringing stakeholders together, the IMI is helping to ensure that trials are more inclusive. Some patients have, historically, been excluded from the regulatory process. Less than half of all medicines commonly used in children have been properly tested in this group, while only 5% of medicines have adequate safety information on their use in pregnant or breastfeeding women.
The c4C project is focused on finding ways to ensure that children are involved in trials for paediatric medicines, while ConcePTION is making better use of existing data on medication use during pregnancy and breastfeeding, and generating new evidence from pregnant women.
The voice of the patient has becoming increasingly important in medicines development. Initiatives such as PARADIGM and PREFER are ensuring the patients can have meaningful input in decision-making on medicines which, after all, should be designed to address their needs.
Digital futures
Our shared future will be digital. The lines will blur between drugs and devices, and between diagnostics and treatment. Wearables and smartphones could take on new roles in medicines development, patient monitoring and even in treatment.
As Europe looks towards a new PPP for health, we should be encouraged by the success of IMI in clearing bottlenecks in the regulatory system. The collaborative spirit of these projects is playing a vital role in embracing new tools to make our regulatory system better for all. In particular, they show the potential for upgrading regulatory science to better suit patients.
For those of use working to optimise regulatory science, the goal must be to ensure patients have access to safe, high quality tools that can improve their lives. It is not an easy task – none of us has all the answers. But we should all agree that our best chance of success is in working together to provide holistic solutions for all patients via medicines, devices and diagnostics.

Judith Macdonald

Judith Macdonald is Senior Director, Global Regulatory Policy and Intelligence at Pfizer. Judith holds a B.Sc,...
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Nick Sykes

Nick Sykes has worked in Regulatory Affairs since 1992 at various companies and joined Pfizer’s regulatory affairs...
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