The Future Feature, with Mike D’Ambrosio | Read now

Medable, Inc?caught up with noted Real-World Evidence (RWE) expert,? Mike D'Ambrosio from Syneos Health , to discuss what he perceives as the biggest breakthroughs in clinical research to date, & what he thinks our industry should strive towards in the future.

What's one thing you wish everybody knew about your job?

Real World Data evangelism is a real thing! I do love wine and I see my job as being analogous to building skills as a wine connoisseur –?

• Foundational is having a good palate and being able to understand basic (data) characteristics.
• It’s important to stay well informed of new varieties (of real-world data) and what pairs well for what accompaniment.
• Aging is important but not all (data) vintages are created equal.
• It is worth exploring local shops(data-marketplace/technology) and some gems can be found.
• Tasting events (read capabilities events) are very informative and can easily drive your tastes/appetite.
• Terroir (reimbursement climate, and diversity) is equally important for RWD as it is for wine!?
• Knowing the right glass (read “form”.. e.g. primary data vs. secondary data) to use is important to get the most of your data.
• Building an RWD/RWE vocabulary is important to make sure that you get the most from your interactions.
• Finally, not everyone is a fan of RWD/RWE and that’s ok! ( as is non-alcoholic beer!)

In an ideal world, how would clinical research be done?

I say this from the position of someone that sees the value and utility of RWD/RWE.? In an ideal world more adaptability to clinical research design (we have seen great advances here already) and easier accessibility and use of RWD/RWE to drive faster outcomes (positive and negative). EMEA DARWIN initiative has made good advances in their pilot phase to show how at-scale data transformation into a common data model for scalable RWD analysis could work. There is a wealth of RWD available that can be highly valuable to optimize protocols, identify gaps in treatment and empower patients and their communities. However, there is an issue of multiple data owners with overlapping data as well as a number of data privacy considerations which makes it difficult and expensive to be able to unlock the full potential of RWD/RWE in clinical research.

I would love to see patient baseline characteristics (collected usually at screening or at enrollment into clinical trials and practically validated) being mandated to be shared within broad open access disease registries where this could be extremely beneficial to both pre-identify potential patients for future clinical trials as well as to have “clinical trial ready” registry data available that could potentially be used to look at treatment gaps, protocol optimization or even for use as external comparator data. The proprietary asset data can be owned by the developers but where the broader communities can benefit from high quality RWD.

What's one barrier to research that's not being talked about enough?

100% Healthcare and Trial literacy. While healthcare literacy has probably improved over time with readily available access to the internet, the complexity of asset development, and accessibility to commercial treatments is still probably overwhelming for the vast majority of patients.? Personalized medicine has been a blessing and a curse in this respect with navigation around access also becoming more complex. More needs to be done to simplify understanding and contextualization of both clinical trials, technology and real-world data as well as navigation of healthcare systems –

• Make access to trials easier
• Empower patients with easier and broader access to their own healthcare data
• Allow patients to better understand and use information to make informed decisions about their health.
• Better use of plain language summaries

What's one consumer experience you wish you could bring to clinical research?

Google translate combined with AI (like ChatGPT…in a compliant manner) to interrogate your own personal healthcare data in order to better navigate/translate complex healthcare questions (trial access, trial data, RWD, navigating healthcare environment) in easy to understand plain language summaries.??

For the win….”rate my sponsor”/”rate my trial”/”rate my physician”

If the industry could just ______ ?things would work better.? Explain why. ?

Collaborate more

There is such a high cost to asset development that proprietary data is heavily protected (understandable from commercial stand-point). However, this can severely inhibit quick availability of data to drive patient, physician and other collaborator decision making to drive better outcomes. The rare disease communities have been probably the best templates for how communities themselves can empower the act of collaboration across industry, physicians and patient groups, where generally the communities are high in healthcare and trial literacy but also have a high level of altruism within their communities. This gives the upper hand to embed the ethos of collaboration in asset development, request for patient relevant data collection and dissemination and broader access to data. We have seen this with large scale collaborative registry approaches and also with the approach of RDCRN.

In your view, what is the smartest/best advancement in clinical trials that's happened in the past 5 years?

Broader access to genomic data and broader adoption of electronic health care records across the world. While this has been the staple for the “advanced/developed” countries for a number of years, the adoption in developing countries and particularly in the continent of Africa is still very poor.? Given that the continent of Africa is home to approximately 20% of the world’s population and approximately 25% of the global disease burden it also accounts for less than 2% of genomic data. Is it surprising that with these statistics that there is large inequality in access to clinical trials and commercial personalized medicines? We can do better.