R&D Gets Real with Real-World Evidence

Bringing RWE into the earlier stages of R&D needs a collective effort from companies, regulators and other stakeholders



Over the past few years, the collection and use of real-world evidence (RWE) has often been an afterthought, restricted to post-marketing registries and market access strategies. Now, however, RWE is pushing deeper into pharma R&D and throughout the entire healthcare decision-making progress.

One initiative that has done a lot to drive this change is the three-year GetReal project, part of the European Union’s public-private collaboration, the Innovative Medicines Initiative (IMI). By bringing together large pharmaceutical companies, SMEs, academia, HTA agencies, regulators and patient organizations, the project aimed to assess existing RWE methodologies and explore new ones.

For Chris Chinn, Head of Real World Investigations, Sanofi, a member of GetReal, the ultimate goal was to produce better-quality evidence for health and technology assessments (HTA).

“We needed evidence that was more informative and more helpful to the HTA body in identifying what the relative effectiveness of the drug was likely to be in the real world,” he says. “The problem is that you often don’t have any real-world experience of your new drug at launch, only the evidence you can generate through the clinical trial program.”

A key element of the GetReal project was to explore existing procedures, typically carried out post-launch, to see whether they could be adopted earlier. “We want to bring the real world into the drug development process, whereas lots of groups were still looking at how best to do post-launch research,” says Chinn.

We are rethinking the paradigm of when you look at real-world evidence in the traditional timeline. It is still quite slow-moving, but we’re certainly seeing more than we did 10 years ago.

Changing the entire R&D mindset could not be achieved by a single company, so collaboration was essential, as was the involvement of NICE and the EMA. “Collaboration enabled us to explore types of effectiveness evidence and more pragmatic trials with regulators and HTA bodies. We needed them to talk openly and frankly with pharma. We had to know whether, if pharma made this huge effort to change, it would be acceptable to them. We found, for example, that NICE is not so much focused on the dogma of the trial, it is much more flexible.”

Three years later, similar projects are springing up, underlining the importance of bringing real-world evidence into earlier-stage drug development, says Chinn. “We are rethinking the paradigm of when you look at real-world evidence in the traditional timeline. It is still quite slow-moving, but we’re certainly seeing more than we did 10 years ago. Pharma companies are now actively working through EFPIA and IMI to drive change, which is an extremely positive thing.”

Incorporating real-world evidence into R&D requires collaborations within companies too, he says. Get Real is now rolling out tools to offer practical help with this process, one of which is called The Navigator.

“When you start a development process in a new disease area, what steps can you take with your R&D colleagues, as well as your market access colleagues and HEOR colleagues to sit down and talk through the best strategies?” asks Chinn. “We are providing a framework to guide key conversations between different teams within pharmaceutical companies.”

Issues up for discussion include using real-world evidence to understand how a disease is treated, the pros and cons of available treatments in the real world, and understanding the potential value of a new medicine. “This means being able to design your trials so they deliver better evidence, with more information on how to extrapolate efficacy results into effectiveness results. That’s what we are after – at the time of launch, we present estimates and we want the best evidence we can in order to support accurate and credible estimates that we will then take to HTA,” explains Chinn.

“The goal is to help the market access teams and HTA bodies to make a better-informed analysis at the time of launch. If HTA bodies have more confidence in your evidence, it is likely to result in a smoother ride through market access and HTA. That’s the ultimate goal.”

 “We need R&D development teams to do a full life-cycle risk assessment, not just a regulatory risk assessment. There is a risk of not having a commercial product, even after positive regulatory approval… we know now that companies can’t just rest on their laurels after having a successful trial.”


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