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Background Showing how engagement adds value for all stakeholders can be an effective motivator for broader implementation of patient engagement. However, it is unclear what methods can best be used to evaluate patient engagement. This paper is focused on ways to evaluate patient engagement at three decision‐making points in the medicines research and development process: research priority setting, clinical trial design and early dialogues with regulators and health technology assessment bodies. Objective Our aim was to review the literature on monitoring and evaluation of patient engagement, with a focus on indicators and methods. Search strategy and inclusion criteria We undertook a scoping literature review using a systematic search, including academic and grey literature with a focus on evaluation approaches or outcomes associated with patient engagement. No date limits were applied other than a cut‐off of publications after July 2018. Data extraction and synthesis Data were extracted from 91 publications, coded and thematically analysed. Main results A total of 18 benefits and 5 costs of patient engagement were identified, mapped with 28 possible indicators for their evaluation. Several quantitative and qualitative methods were found for the evaluation of benefits and costs of patient engagement. Discussion and conclusions Currently available indicators and methods are of some use in measuring impact but are not sufficient to understand the pathway to impact, nor whether interaction between researchers and patients leads to change. We suggest that the impacts of patient engagement can best be determined not by applying single indicators, but a coherent set of measures.

The International Rare Diseases Research Consortium (IRDiRC) has created a Guidebook to facilitate drug development for rare diseases by organizing available tools into a standardized framework. The International Rare Diseases Research Consortium (IRDiRC) has created a Guidebook to facilitate drug development for rare diseases by organizing available tools into a standardized framework.

The European orphan regulation incentivizes the development of medicines for conditions for which the pharmaceutical industry would be unwilling to develop medicines under normal market conditions. Defining an orphan condition is the starting point of every orphan designation, and it is also the entity used to determine prevalence, which must not be more than 5 in 10, 000 for orphan designation to be permitted. In practice, most orphan conditions can be easily identified ; recognized distinct medical entities would generally be considered as valid conditions, defined in terms of their specific characteristics . However, in certain areas, there may be challenges in defining orphan conditions such that a condition is in keeping with the regulatory framework and consistent across all applications that are presented to the European Medicines Agency (EMA)’s Committee for Orphan Medicinal Products (COMP).

Background Evidence-based clinical practice is challenging in all fields, but poses special barriers in the field of rare diseases. The present paper summarises the main barriers faced by clinical research in rare diseases, and highlights opportunities for improvement. Methods Systematic literature searches without meta-analyses and internal European Clinical Research Infrastructure Network (ECRIN) communications during face-to-face meetings and telephone conferences from 2013 to 2017 within the context of the ECRIN Integrating Activity (ECRIN-IA) project. Results Barriers specific to rare diseases comprise the difficulty to recruit participants because of rarity, scattering of patients, limited knowledge on natural history of diseases, difficulties to achieve accurate diagnosis and identify patients in health information systems, and difficulties choosing clinically relevant outcomes. Conclusions Evidence-based clinical practice for rare diseases should start by collecting clinical data in databases and registries; defining measurable patient-centred outcomes; and selecting appropriate study designs adapted to small study populations. Rare diseases constitute one of the most paradigmatic fields in which multi-stakeholder engagement, especially from patients, is needed for success. Clinical research infrastructures and expertise networks offer opportunities for establishing evidence-based clinical practice within rare diseases. Electronic supplementary material The online version of this article (doi:10.1186/s13063-017-2287-7) contains supplementary material, which is available to authorized users.