IMI Programme Office: Why was a project like GETREAL needed in the first place?
Elaine Irving: The current drug development paradigm, which focuses heavily on marketing authorisation, means that health technology assessment (HTA) bodies and payers often have to make decisions on the value of a medicine with limited information about the likely effectiveness of the new medicine in the routine healthcare setting. GETREAL set out to address this challenge by exploring new approaches and methodologies to bring evidence generation much earlier in the drug development process. To take on such a challenge, collaboration between all the involved stakeholders was key, and IMI provided the unique framework to enable us to bring together academic and industry scientists, regulatory bodies, HTAs and patients.
IMI Programme Office: What were the project’s most important achievements?
Elaine Irving: This was a successful project so there are a number to mention and a number of publications. Firstly, we enabled open and transparent interaction with all the key stakeholders: HTAs, regulators, patients, industry scientists, academia, and really unearthed all the different perspectives, which enabled us to work together on building solutions.
Secondly, we built a TOOLBOX which enables drug developers to better understand which factors might influence how the medicine works in routine healthcare compared to clinical trials. This then tells them which areas to focus on understanding ahead of or around the time of a drug launch.
Thirdly, we created PRAGMAGIC: a great interactive tool that supports researchers embarking on designing and conducting a pragmatic trial. It helps users understand how the choices that you make when designing a study impact the operational feasibility of the study. It also demonstrates how reflective the data will be of the effectiveness of the medicine in routine healthcare.
We also did pioneering work around developing new methodologies that enable health outcome scientists to predict and model real-world effectiveness of a new medicine using existing clinical trial and real-world data (RWD). For this purpose, we further developed the ADDIS platform to enable investigators to access clinical trial data and perform analysis.
Finally, we generated the RWE Navigator, a tool that enables individuals to think through what RWE evidence they might need to demonstrate the value of the new medicine compared to existing therapies, and provides information about the different options available to deliver that evidence. The tool also provides information on the pros and cons of each of the different options, links to useful external resources and data sources.
In order to support all this, we also developed a remote e-learning platform with courses that run regularly throughout the year.
IMI Programme Office: Which accomplishment are you most proud of? What stands out?
Elaine Irving: At the moment it has to be the RWE Navigator and the associated platform around that. I really like the way it takes users on a journey from generating an evidence-generation strategy, and understanding what approaches are available, to also providing links to many useful resources that the user will need along the way. It actually helps bring transparency and openness to the limitations and benefits of the different approaches.
IMI Programme Office: Is the industry already using some of these tools in their work?
Elaine Irving: Yes, they are. For example, the Navigator is being used to help align perspectives within medicine development teams, which are made up of individuals with very different roles and perspectives. Developing a drug is really expensive: there are a number of clinical studies that have to be conducted to meet the regulatory requirements for authorisation, so sometimes when members of a medicine development team propose additional studies that they feel are needed to support medicine reimbursement, it can be quite hard to get support and funding. The Navigator provides an excellent resource to support researchers. It takes the medicine development team on a journey to identify where they might expect differences in efficacy as demonstrated in the clinical trial setting, and the effectiveness seen once used in routine healthcare. The tool also informs on the different options that can be used to address those gaps.
IMI Programme Office: What were the main ways in which the industry benefited from this project?
Elaine Irving: The main benefit was definitely the stakeholder interaction – this is one of the few forums that we would ever have to interact and conduct research alongside our HTA and regulatory partners. So that was definitely a huge benefit. For industry, understanding the perspective of regulators and HTAs, with respect to certain types of evidence and how they may or may not use that evidence in their decision-making, has been hugely informative. For us, that has been probably the biggest opportunity: to really work through real-life examples, and have those open discussions with regulators and HTAs to learn more about where they have concerns and where they see opportunities for the use of RWE.
IMI Programme Office: And what about the academic community? How did they benefit?
Elaine Irving: They also benefitted from collaboration with the different stakeholders. This is not really a group of stakeholders that would normally work together. Also, methodology development is generally not an area that is highly funded, so that’s been a big opportunity for academia. Some of the work that they have conducted in GETREAL is groundbreaking and definitely leading the field – they have published a number of peer-reviewed papers in high-impact journals. Like the other partners, they also benefitted from understanding the perspectives of the different stakeholders and from having more transparency around different RWE methodologies.
IMI Programme Office: And how did the SME in the project benefit?
Elaine Irving: Again, the benefits of collaboration come through loud and clear. They were able to demonstrate their leadership in this field, as well as work with leading academics and major pharma companies who ultimately are their key customers. Through publications and meetings, they were also able to increase their company’s visibility.
IMI Programme Office: Your project achieved a lot on different fronts – would any of it have been possible without IMI?
Elaine Irving: No, definitely not. There is a real drive to see much more RWE used in decision-making, but there is by no means any consensus around how that should be done, or around standards with respect to RWD quality or methodological approaches. Without the ability to have these open discussions, to try to get to a point where we can start to agree around some of the challenges and opportunities, some of the weaknesses and strengths of different approaches, we won’t be able to change the current drug development paradigm.
IMI Programme Office: And what are the next steps? What happens from here?
Elaine Irving: At the end of GETREAL we applied for continued funding through IMI and we hope to start up again in June this year. That really is to continue to build on the research community, drive forward some of the policy recommendations that have come from GETREAL, and take forward that global debate around some of the barriers to and the opportunities for the use of RWE in healthcare decision-making.
IMI Programme Office: Do you have anything else to add?
Elaine Irving: It’s been a great collaboration, a great group. I stepped into this role only for the last year which was daunting because there was still a lot to be done. But in this last year we really focused and we really delivered. I think the team is pretty unique, they are all very driven and aligned on the need for the project and that energy has come through from start to finish. They are all going to continue on as well, which is a good sign. It’s been a great team effort.