Executive Summary
The latest meeting of the DARWIN EU advisory board heard about moves to increase the number of RWD sources that can be analyzed and to improve the efficiency of performing RWE studies.
Source: ShutterstockDARWIN SHOULD PRODUCE RELIABLE AND TIMELY REAL-WORLD EVIDENCE
EU medicine regulators are planning to produce catalogs of real-world data (RWD) sources and observational studies later this year or in early 2023, and are preparing to select partners who have access to such data.
The move is part of efforts to improve the transparency of observational study data and data sources and to make it easier to judge the “evidentiary value” of observational studies and RWD sources when used to assess the safety and effectiveness of medicines on the market, according to the European Medicines Agency.
These and other issues were discussed at the latest meeting of the advisory board of DARWIN EU, a project being set up by the EMA and the EU Heads of Medicines Agencies to help deliver timely and reliable real-world evidence (RWE) for use by regulators, health technology assessment (HTA) bodies and payers. RWE is the clinical evidence derived from the analysis of RWD.
The project seems to be making good progress. A coordination center for DARWIN EU was established in February to prepare individual data use agreements with data partners. RWE “use cases” are to be identified, RWE pilot studies are to be launched later this year and in 2023, and a technical workshop with HTA bodies and payers is planned for the third quarter of 2022. (Also see “EMA, HTA Bodies & Payers Explore Common Ground On Use Of RWE” – Pink Sheet, 28 Jun, 2022.)
Data Source Lists
The 20 April meeting of the DARWIN EU advisory board, minutes of which have just been published by the EMA, heard that the agency had produced a list of metadata for describing RWD sources and studies. The list, which was adopted by the agency at the end of May, will be used to establish two further lists.
These are a catalog of data sources, building on the ENCePP (European Network of Centres for Pharmacoepidemiology and Pharmacovigilance) resource database, and a catalog of observational studies, building on the EU PAS (post-authorization studies) register. Both catalogs are expected to be ready in the fourth quarter of 2022 or the first quarter of 2023.
Data Partners
The meeting also heard a presentation by the EMA’s Xavier Kurz on the criteria for selecting data partners to join DARWIN. Data partners have access to raw data either in-house or remotely, and they may include national authorities and the EMA itself. The coordination center is responsible for providing a structure to develop and manage the network of data partners.
Initially it is proposed that the DARWIN network should use data sources that have been converted to the Observational Medical Outcomes Partnership (OMOP) common data model (CDM). In the later operational phases, data partners who have not fully converted to a CDM will be onboarded. “This will allow a quick establishment in order to start delivering the benefits of DARWIN,” the EMA said.
Non-EU data partners could be considered for inclusion if this helps achieve DARWIN’s objectives, according to the meeting report.
RWE Study Classification
As for the kinds of RWE studies that could be performed via DARWIN EU, the EMA’s Juan Jose Abellan said that drug utilization, disease epidemiology and etiological studies were commonly used to support decision making, as were safety monitoring studies.
“It is the goal of DARWIN EU to increase both the number of data sources that can be analyzed as well as the capacity for studies that can be performed annually, while delivering efficiency gains in standardised analytics, time and cost savings,” the EMA declared.
Discussions are ongoing with the coordination center on defining each study type with the use cases in mind, as well as the outputs and typical focus of the study – for example, a particular therapeutic class, a selection of substances or a disease area. It was noted at the meeting that the data source selection for each study should be based on its utility in terms of yielding robust results.
Use Cases
An example of how Portugal is approaching RWE via use cases was given by Claudia Furtado of the national regulator, Infarmed. The use cases discussed included characterizing the utilization and prescribing of drugs, monitoring the impact of policy and regulatory actions, describing standards of care and treatment patterns, monitoring managed entry agreements and restrictions on reimbursement, and tracking effectiveness to support reassessments of regulatory decisions.
Furtado said Infarmed had access to several data sources such as hospital data prescribing and claims data, and disease registries. The registries had been developed either by Infarmed, for hepatis C or spinal muscular atrophy, or by other entities such as the National Cancer Registry.
“For example, the data from the National Cancer Registry allowed the monitoring of real-life utilization and effectiveness of pembrolizumab [MSD’s Keytruda] in patients with advanced melanoma, observing that effectiveness is slightly lower than in clinical trials and with a comparable safety profile,” according to the meeting report.
Furtado also noted some challenges that had emerged, saying it would be necessary to identify at an earlier stage those medicines for which post-marketing evidence generation would be required.
“Data quality is important for robust conclusions, as is the use of well-established methodology and availability of qualified researchers,” the meeting report says. “In discussion, it was noted that linkage between prescription and healthcare database and registries is important.”
The EMA now plans to discuss specific use cases that could be addressed through DARWIN EU, particularly in the context of international collaboration and rare diseases. Representatives of national regulators were invited to present more use cases at the next meeting of the advisory board, which took place on 6 July.
Source: https://pink.pharmaintelligence.informa.com/PS146470/EU-DARWIN-Project-To-Catalog-Real-World-Data-Sources