Comment on: “Patient Registries: An Underused Resource for Medicines Evaluation: Operational Proposals for Increasing the Use of Patient Registries in Regulatory Assessments”

Abstract

I write in response to the recent article by McGettigan et al. [1]. I would like to thank the authors for their work and respectfully ask them to respond to three areas of interest. The first is in relation to the methodology employed in this study. The authors describe the utilisation of a “qualitative synthesis” methodology; while numerous recognised methodologies are well-established [2], the exact method employed in this study is not cited. Could the authors provide a citation as, without utilising a recognised scientific methodology, the work constitutes a simple summary of the four European Medicines Agency (EMA) reports and not “original research” as presented? The second is over the choice of definitions. Definitions published in an indexed journal require stringent quality control and rely on the use of recognised research terminology. The definition of registries used by the authors, “organised systems that use observational methods to collect uniform data on a population defined by a particular disease, condition, or exposure, and that is followed over time”, cites three references [3–5]; two of the three [3, 4] differ to a material degree (only one refers to “uniform data” [3]) and the third [5] refers to a link that is broken. Crucially, none of these citations are peer-reviewed articles in indexed journals. The workshops forming the basis of the article were run by the EMA, a regulator of medicines and not clinical research. National ethical and site-level governance bodies in each country are responsible for safeguarding patients recruited into research, and, therefore, it is from their ‘gate-keeper’ perspective that revisions to this definition are recommended; this group will be referred to here as ‘ethical governors’. The ethical governor could be argued as being the ‘dominant’ stakeholder, being ultimately responsible for research approval. Furthermore, key characteristics of each research activity have implications for implementation, particularly with regards to the clinical trials regulation [6]. The definition of a patient registry should therefore be approached from this perspective. In order to appraise the authors’ definition and suggest a revised one, we reviewed the websites of the clinical trials regulation [6], National Institute for Health Research [7] and clinialtrials.gov [8] but could not identify the term “organised system”. The most relevant term found was “clinical study”, defined as, “A research study involving human volunteers (also called participants) that is intended to add to medical knowledge” [8]. The patient registry is further defined as “a type of observational clinical study” [8]. Of the three main types of observational study previously described [9], the cohort is the most appropriate here, as it describes the incidence and/or natural history of a disease and is able to analyse predictors (risk factors) [9]; this group of observational studies can be further subdivided into prospective and/or retrospective designs. Therefore, a patient registry is more accurately defined as, “a prospective and/or retrospective observational cohort clinical study.” Would the authors agree that this represents a more appropriate definition to describe a patient registry in an ethics submission and, subsequently, in a peer-reviewed, indexed journal? The third area of interest is Table 4, which purports to differentiate between a “registry” and a “registry-study”, listing numerous characteristics under various headings. There are several concerns with this table from the ethical governor’s perspective. This comment refers to the article available at https ://doi. org/10.1007/s4026 4-019-00848 -9.