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He also talked about how RWE has evolved in recent years and the use of RWE as a tool to support Marketing Authorization Applications (MAA). Interview edited for clarity.
The Data Analysis and Real-World Interrogation Network (DARWIN EU) aims to provide the EMA, the European Commission and the competent authorities of the 27 EU Member States with access to real-world medical database analyses. increase. It was published last year. Last month, the EMA announced that the DARWIN EU had completed its first study and was looking for new data partners. (Related: EMA, HMA Outline Evolution of DARWIN EU Real World Database, Regulatory focus August 2, 2022)
Focus: How has real-world evidence gathering evolved in the EU over the past few years?
Arlett: Historically, companies set up registries specific to their products. If it’s the first cure for a particular disease, that’s fine, but when the next product comes along, that company sets another registry, and then another registry. First, we collected data for only one product, so comparisons are not possible.
The approach we are promoting is to enroll based on specific patient populations, such as multiple sclerosis patients, rather than patients treated with a specific product. This is because you can see clinical events. And the clinical progression and different drug journeys.
Patient groups are of great interest in building enrollment cohorts based on common patient groups with specific diseases and collecting useful data. Our position as regulators is that this approach is very useful.
Focus: What do you think is the biggest change in real-world evidence gathering?
Arlett: We have been using real-world evidence in industry and the regulatory arena for decades, specifically for drug safety and drug development. Most successful companies have looked to real-world evidence to understand if their products have therapeutic gaps. and to inform their clinical trial medication. For example, if we know that his 80% of all diabetics are between her 30’s and her 60’s, it helps us design clinical trial recruitment. The numbers are made up by me, but you get the point.
There are some things that are changing and changing rapidly. First, access to data is improved. The digitization of medical records has made much more data accessible. You can also access insurance records. That’s the game changer. A decade ago, electronic health records were the minority of health records, but today they are the majority of health records. By going digital, you can start analyzing. Moreover, this methodology has advanced significantly in the last few years.
Focus: What are the lessons learned from the DARWIN pilot?
Arlett: First, we’re using a common data model, OMOP. [Observational Medical Outcomes Partnership] Common Data Model – means that the data has been transformed into a common structure. It also means that the terms used in the data are mapped.So that database heart attack [the same thing as] Heart attack, a slightly different wording is mapped so you can understand medical concepts and use computer algorithms to query different data sets instead of manually pulling back data. The Common Data Model is a key enabler of DARWIN. Investigations can be done quickly and scaled up, so you can explore when onboarding not just one dataset, but 30, 40, or 50 datasets. It’s about analyzing data, and that’s one of learning.
The next learning is that we need to go step by step…the bottom line is that we need to bring in stakeholders, we need to build networks, we need to onboard trading partners and that we need to learn as we go along. We also need to bring in internal and external stakeholders. Internal Stakeholder means a Regulatory Authority. Don’t think that clinical trials and real evidence are at odds. This is very important. There are still regulatory agencies that are experienced in clinical trials and do not fully trust real evidence. So we have to go step by step.
Focus: What products are approved using RWE?
Two examples: In 2020, we had a product approved for spinal muscular atrophy, a dreaded physical disorder. [Novartis’s] Zolgensma. The actual evidence was an important part of the paperwork. There were uncontrolled phase 3 trials, single arm trials. [good clinical practice] GCP used historical data from RWE like a registry. It was considered a very important part of the document.
the other one is [Adienne Pharma & Biotech’s] Ferinun for various blood cancers. The evidence came from the full published literature and was a combination of clinical trials and observational studies. [European public assessment report] EPAR. It turns out that both of these examples relate to very rare diseases. If it is a common disease, there is no logistical reason not to conduct a randomized controlled trial, and randomized controlled trials are preferred to address the confounding bias of randomization.
Focus: We hear a lot about the Danish patient registry, can you talk about this?
Arlett: Nordic countries have a completely different story… In Denmark, the health system itself collects health records in a registry. These are national health registries, so rather than being run by patient organisations, they have a Danish population-wide registry and have a sub-registry for cancer patients. As a result, we are able to conduct excellent research.
I gave an example [at the meeting] About the effectiveness study of COVID-19 research that the EMA commissioned a Danish agency a year ago. This is one of the highest quality studies on efficacy that has been done against COVID-19, and it was possible thanks to these high quality registries in the Nordic countries.
Focus: Any Sponsor Advice for Better Use of RWD?
Arlett: I’ll give you one stock answer, especially in a rapidly evolving field like real-world evidence.
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