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An early peek at the FDA’s RWE ‘cookbook’

The FDA is developing guidelines that will help pharma accelerate its adoption of RWE, this is what the industry should know



Pharma has welcomed long-overdue guidance on the use of real-world data and real-world evidence being shaped by the FDA, which late last year released two sets of draft guidelines as part of an industry consultation. 

The two documents, addressing the use of RWE in new indications and RWD used to support new drug submissions, are already helping pharma understand the FDA’s thinking when it comes to the standards it is looking for when it comes to the quality of data, data capture systems, standardisation, harmonisation and RWE methodologies. 

The FDA consultation is important because hitherto the industry has been left guessing what regulators expect from the industry when it comes to the use of RWE and product approval, says Ramon Hernandez Vecino, Global Head Real World Evidence in Clinical Development for Sanofi.

The documents address a range of useful issues where guidance has been lacking and pharma has been managing risk with limited guidance, says Hernandez Vecino, including risk relating to the selection of data sources, the process of developing a study design, the data provenance, data quality and creation. “Before these guidelines, all of us were dealing in one way or in another with these topics. What is different from now on is that we can address this document as a way to discuss those risks.

“These guidelines should help us to better understand the [regulator’s] worries and the standards of quality and the key elements that we should consider when we produce this evidence from real-world data.”

After two decades of discussion, practical guidance is now needed and the aspiration is almost for the industry to have a ‘cookbook’ to follow to provide clarity on how pharma establishes clarity on disease and on compounds via RWE, says Jennie Medin, Global Head Real World Science & Digital at AstraZeneca. “It's timely, it's needed.”

One of the most important aspects of the guidance will be the fact that it will define and clarify the quality bar and thus drive improvements in the way pharma and its partners gather and analyse data, says Gilles Paubert, SVP, Global Head of Cegedim Health Data.

“It's a good driver to improve the quality of data collection. We know now what key elements must be in our submission.”

Paubert stresses, however, that he hopes for more clarity about the role of software providers in gathering reliable and useful data in the final document. “I think that we can improve some recommendations about how to enrol different stakeholders to improve data collection and data quality. It could be a good idea for authorities to anticipate and to build a process with the involvement of the software providers, because we are always involved at the end of the process.”

The document includes some interesting clues as to the FDA’s directive rather than proscriptive attitude to unstructured data, says Nigel Hughes, Scientific Director, Janssen. “The section where they talk about unstructured data is very impactful, because what it says is that they will not tell us what to do. 

“It's almost like setting an exam question. We see your answer, but we'd love toyou’re your working. It's really ‘tell us what you do, be transparent and explain it’. That's a rather open approach and I think that's very encouraging.”

The final form of the guidance due to be published in 2022 will give pharma much more solid ground on which to build its future plans for RWE not just in the US but beyond, says Hughes. 

“We'll be looking forward in 2022, to further enhancements of these guidance pending the response to the consultation phases, but further deliberation on these [is] likely internationally from other stakeholders, all of whom have equal stakes in utilising this for regulatory or indeed other types of decision making.”

Listen to Real-World Evidence, Real-World Industry podcast here

Read the source FDA documents here: Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products and Data Standards for Drug and Biological Product Submissions Containing Real-World Data 


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Pharma Clinical

May 17, 2022 - May 19, 2022, Virtual Conference, Networking & Exhibition

Rebuild clinical trials in the image of patient need