Attending the conference in person was a highlight in and of itself.  There is a certain energy that comes with meeting face to face and experiencing the presentations first hand, and DIA RWE did not disappoint.  Rather than debate the attributes of particular methods or data sources, the meeting focuses on the broader scope and challenges of reframing clinical investigation using RWE.  Here are a few of the key takeaways from a stimulating and engaging conference.

1. The FDA’s been busy this year

2022 has flown by.  Perhaps you’ve missed the 4 draft guidelines published in the last year.  The FDA is creating a working framework for drug developers looking to leverage real-world data and real-world evidence to support clinical investigations. 

The goal is to provide enough structure to take action while acknowledging that the guidance will need updates as the field gains more experience.

The FDA produces draft guidelines on a variety of RWE topics and solicits feedback from stakeholders in academia and industry before producing final versions.  Take a deep dive into the guidelines here. Or, if you are looking for a lighter read, check out this article to learn more about where we are now with RWE.

2. Start regulatory discussions early

This leads to the next takeaway, early discussions with regulatory bodies will help navigate away from avoidable pitfalls. The FDA wants the same things as everyone: better options, care and outcomes for patients, without sacrificing safety and effectiveness. Some attendees voiced concern that speaking with regulatory bodies early could open Pandora's box and derail timelines. New programs were announced that should help reduce that stigma, establish more lines of communication, and help make it the norm for the future.

3. New programs to facilitate the implementation of RWE

We heard several great talks that mentioned new programs for integrating RWE approaches into clinical development.  The Advancing Real-World Evidence Program aligns with the FDA’s commitment to opening new channels and programs to accelerate the use of RWE that meet regulatory requirements in all stages of drug development including post-market.  In Canada, the Post-Market Drug Evaluation Program (CMDE) aims to continue the collection of evidence post-approval to provide feedback on a drug's performance in the market, in the real-world.  

4. No one-size-fits-all template

There is no silver bullet when it comes to applying RWD to clinical development.  As reiterated throughout the meeting, data needs to be fit for purpose when navigating this new and exciting space.  Each use case will be wrought with nuance, shaped by biology, patient characteristics, treatment landscape, and disease specific challenges.  To find success, the researcher must know what questions to ask and then decide what data is needed to answer them. 

5. Is RWE Confounded?

A major theme to the meeting was how will we know if we are getting it right with RWE?  In other words, if randomized control trials (RCT) are the gold standard, how can we be sure that the results we get using RWD replicate what would be found in an RCT?  RCT DUPLICATE is an ongoing project aimed to evaluate the concordance between using RWE and traditional completed RCTs.  Read more about it here.  In general, the more challenges there are with emulating the RCT data, the bigger chance that RWE misses the mark. And, like most things in science, more data is better.

6. Real Examples of RWE Success

While the majority of RWE is used as supportive evidence to RCT data in regulatory filings, an entire session was devoted to examples where RWE was used to gain approvals or remove black labels from the original approvals.  It is easy to get caught up in the world of possibility using RWE, but RWE is not a magic bullet. Expecting that RWE is better, cheaper, and faster will lead to disappointment.  However, several attendees reported that internal acceptance of RWE strategies was more likely achieved with a successful project or two and these examples were nothing short of inspiring.  

7. The Future is Exciting

In the final session of the conference, speakers and panelists were asked what they are most excited about the future of RWE and here is a summary of some of the responses:

1. Longitudinal patient data or longer patient journeys
2. Collaboration from stakeholders to jointly solve the challenges and broader use of RWE
3. Leveraging tokenization and linkage
4. Integration of research and care through digital tools
5. Expanding RWE use from rare disease and oncology to more common diseases in clinical research
6. Appreciating the progress between this year and next year’s conference

Rightly spoken, and a great way to wrap! 

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