Registering an Interest - How EMA Views the Future of Registries

Registries are inching their way up the pharmaceutical policy agenda in Europe. In October the European Medicines Agency is going to host a workshop in a bid to get registries to work more closely with other parts of the health system-notably health technology assessment bodies, regulators and industry. It is the latest straw in the wind in a wider movement-likely to build up to gale force over coming years-as the challenges become still more pressing in getting the right drugs to the right patients at the right time, and at something like the right cost.

The EMA has been pushing quietly for closer links for more than two years now. Last September, it launched a patient registry initiative to promote more interaction between registry coordinators and users, so that data could be used better throughout the lifecycle of a medicine-from early-stage development, during the evaluation of a marketing authorization application, and after the product reaches patients. The initiative's formal aims are to increase the use of existing disease registries "to create more comprehensive, flexible and sustainable resources" and map ongoing registry projects more precisely.

The plan also includes support for companies to create a new registry where none exists for a product, to provide "adequate sources of post-authorization data for regulatory decision-making." The plan puts heavy emphasis on using a standard methodological approach, with standard core data elements and standardized protocols, to give new registries wider applicability. This exercise is strongly needs-driven. The starting point for the envisaged action would come from a recognition by an EMA committee (or an applicant for marketing authorization) that additional data on a medicine is required from real-life clinical use, before or following the medicine’s authorization. A process of identifying and evaluating existing data sources (including national databases, electronic health records and existing patient registries) would ensue, to allow a determination of whether the need for data is best addressed through a registry. If that proves to be the case, then discussions would be held with registry coordinators over possible amendments or extensions to an existing registry, and in the event that more is needed, then the core components of a new registry would be defined.

Now, a year on, EMA believes the time is right to draw some conclusions from the pilot and to point the way ahead more clearly. So the workshop will reflect on the challenges and barriers that have emerged, and explore potential solutions. The agenda is ostensibly technical, in that it will focus on the technical challenges arising from disparate datasets and on how to avoid registries duplicating one another's work. But the subject is in reality deeply political, since its implications have major significance for the multiple current attempts to get a better handle on the value of ever-more sophisticated medicines. The underlying ambition is huge: EMA (and many like-minded authorities in Europe) want to see a more systematic and standardized approach to the contribution that registries can make to the benefit-risk evaluation of medicines.

Many of the challenges have become clear over recent years. Whether using existing registries or establishing new ones, the field is characterized by a lack of coordination between ongoing initiatives at national and international levels, by failures to harmonize protocols, scientific methods and data structures, by impediments to data sharing and transparency, and by crude considerations of sustainability.

The cast-list for the conference is impressive, indicating the importance being given to the subject. It includes heavyweight regulators such as Noël Wathion and Fergus Sweeney from the EMA, June Raine from the UK, Marc Guerrier from France and Tomas Salmonson from Sweden, and a broad spectrum of leading speakers from universities, HTA bodies, scientific societies and drug firms to discuss issues such as use in clinical trials, how to manage data abstraction and interoperability, and the choice between product registries and disease registries. The agency is deliberately reaching out to the medical community, patients' associations and academia too.

Some valuable conclusions may be possible from the pilot so far. Around a dozen registry owners and drug firms expressed interest in the first six months of the pilot phase, and several case studies have been identified involving setting up a new registry, adapting an existing registry, and combining both approaches. Already initial consensus has emerged that early dialogue among the parties, with the definition of a clear protocol, is helpful to determine how feasible collaboration may be, and how ethical issues such as consent and data ownership can best be addressed. Other desirable factors are clear models for collaboration between existing registries.

The dynamic is-or at least should be-irresistible. Reinforced by the recent toughening up of European pharmacovigilance rules, registries already play a central role in many EMA regulatory procedures. They are routinely required from companies making marketing authorization applications, whether in the context of risk management, or in evaluation of advanced therapies, pediatric medicines and orphan products. At the same time, the pressure is growing for more effective HTA collaboration at European level-particularly as the debate evolves around adaptive pathways for innovative targeted products, with the attendant focus on better and wider real-world data sources. And in the background (but increasingly moving to the foreground), European governments' concerns over drug pricing are conferring new significance on mechanism that can help in discussions of the effectiveness of therapies.

By the end of 2016, the EMA intends to publish recommendations arising from the pilot phase. They will be awaited with interest by everyone across the European pharmaceutical community.