Reactions to the Report from the EMA Workshop on Patients Registries


In 2014, the European Medicines Agency (EMA) commenced a Registry Initiative aiming to optimise the use of registries in supporting medicines authorisations. Engagement between marketing authorisation applicants and registry holders requires establishing a strategy and a supporting task force. An undertaken pilot phase aimed to identify the barriers and enablers and to develop recommendations to optimise the use of registries in marketing authorisation. The perspectives of multiple stakeholders including registry holders, patients, the pharmaceutical industry, health technology assessment representatives and regulators were then explored in the EMA Registries Workshop held on the 28th October 2016. Participants described the challenges and barriers to collaboration, and made recommendations to address them and to develop the next steps of the EMA Registry initiative.

The report from the workshop sets out participants’ observations and recommendations in five theme areas: benefits of patient registries and obstacles to be overcome, benefits and challenges of collaborations, technical considerations, governance, and sustainability.

Members of the RD-Connect Patient Advisory Council have followed the EMA patient registry pilot project during the last two years.

Patient organisations are very interested in the final outcome in the use of registries specifically for regulatory approval, and whether new applicants will implement disease based registries instead of product registries.

As patient representatives, we have often stressed the importance of integrating patient perspectives in the regulatory approval process – not only the patient preferences but also what is clinically important for us, through the various Clinical Trial mechanisms like primary endpoints etc. With the new concept of these registries we could take a step further towards the collection of Real World Evidence about what works, by how much it works and for whom it works best.

So it becomes essential for all of us to develop the proper robust governance to safeguard transparency, accessibility of data and independence of registries.

Disease registries and post-authorization pharmacovigilance

EMA requires pharmaceutical laboratories to monitor, using product registries, the safety of their medicinal products after market authorization to track large-scale adverse events that could have gone undetected through clinical trials. However, more and more pathologies are endowed with an epidemiological patient registry whose aims include a better understanding of the natural history of the disease and the clinical characteristics of the population.

Within its Registry Initiative, EMA wants to encourage Pharma companies to switch from using Product registries to Patient registries in their pharmacovigilance studies. With this in mind, EMA invited stakeholders to feedback on the report summarising the observations and recommendations arising from the Registries Workshop This is a tremendous opportunity to improve quality of disease registries while helping to evaluate new drugs, so important in rare diseases.

Registries represent an invaluable source of data especially in the field of rare diseases as they can for example help assess the feasibility of a study by evaluating the number of patients eligible for a clinical trial.

Registries-Pharma partnerships could provide funding to registry managers for a given period of time. These should be considered as a bonus, an extra ad-hoc support to help improve the quality of registries and facilitate compliance with good clinical practices, rather than being the main funding provider. Setting up a drug registry requires the pharmaceutical laboratory to respect these good clinical practices, it is therefore likely that the EMA will also impose it on patient registries.

New ways for Patient Organisations to Contribute to Drug Evaluation

In 2011, the importance of the role of associations in the health system was reaffirmed by the French national Agency for drug safety. The law reinforces transparency and puts the patient at the heart of the process that ensures his/her safety. Approved patient organisations can report suspected adverse reactions. They can put a request to the Agency recommending the temporary use of a medicinal product before its marketing authorization when clinical trials showed a significant efficacy and no or limited adverse events. This is particularly important in the field of rare diseases where few drugs are available. Some patient organisations are members of the board of directors and of the three advisory committees. A specific Agency committee provides an ongoing dialogue on cross-cutting issues with patient and user organizations.

The Agency also encourages through annual calls for projects, competitive and independent associative initiatives aimed at promoting the good use of medicines.

In 2016, the French Health Authority implemented a tool allowing patient organisations to give their opinion on a medicinal product before its market authorization.

See also, this short song/animation encouraging patients to report adverse side effects due to medication after marketing authorisation.

~The article was written by the members of the RD-Connect Patient Advisory Council.