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Chan et al. Rare Dis Orphan Drugs J 2023;2:28  https://dx.doi.org/10.20517/rdodj.2023.23  Page 3 of 8

               Each member organization nominates a representative who is part of the Consortium Assembly (CA) that
               acts as the overall governing committee overseeing IRDiRC activities. From this assembly, a Chair and Vice-
               Chair are elected for time-limited terms to help manage the day-to-day operations of IRDiRC. Three
               Constituent Committees (CC) representing Funders (FCC), Patient Advocates (PACC), and Companies
               (CCC), whose membership is drawn from the CA, act as coordinating bodies for the constituents that they
               represent and help to identify gaps and priorities, execute activities aligned with their space, and report back
               to the CA on scientific and programmatic needs. In addition to the Constituent Committees, IRDiRC has
               four  Scientific  Committees  (SCs)  that  represent  key  areas  in  RD  research:  Diagnostics  (DSC),
               Interdisciplinary (ISC), Therapies (TSC), and Regulatory (RSC). Membership of these committees is
               comprised of subject matter experts drawn from academia, patient advocates, regulatory bodies, diagnostics,
               and the pharmaceutical industry, and is not restricted to Consortium members. Membership on the SCs is
               time-limited to enable key stakeholders the opportunity to serve and contribute their knowledge, with
               staggered terms of service to reduce disruption to committee activities. The main role of the Scientific
               Committees is to identify and execute actionable activities that advance the goals of IRDiRC, report on
               progress made towards those goals, and advise the CA on research priorities, meeting regularly in order to
               achieve those goals.


               An Operating Committee, comprised of the Chair and Vice-Chairs of the Consortium Assembly,
               Constituent Committees and the Scientific Committees, works in concert with the Scientific Secretariat to
               efficiently coordinate and manage the activities of IRDiRC. The Scientific Secretariat is a dedicated office
               within IRDiRC that manages the daily activities, providing administrative support, organizing meetings
               (teleconferences, in-person events), facilitating interactions both within and outside of IRDiRC, and
               disseminating information and communications. The Operating Committee plays a pivotal role in the
               exchange of information between the Constituent and Scientific Committees, evaluating new proposals and
               recommendations to present to the wider Consortium Assembly. The organizational structure of IRDiRC is
               summarized in Figure 1.


               Task forces and working groups
               To accomplish these goals, IRDiRC coordinates the formation of Task Forces (TFs) and Working Groups
               (WGs) to address key issues identified by the Constituent and Scientific Committees and prioritized
               through discussion by the CA. The TFs and WGs are designed to be time-limited activities that tackle an
               actionable topic in a small group setting, with members (who do not need to be IRDiRC members) selected
               based not only on expertise in the area of interest, but also those with diverse backgrounds to ensure that
               different perspectives are taken into account during discussions. The ultimate goal of these TFs and WGs is
               to propose solutions through policy recommendations, technical standards, guidelines, or tools that advance
               RD research, diagnosis, and therapeutic development. The activities of the TFs and WGs in the first decade
               of IRDiRC (2011-2021) have previously been summarized by Monaco et al., while a full listing of all
               completed and ongoing TFs/WGs is available on IRDiRC’s website .
                                                                       [7,8]

               IRDiRC continues to identify and address key issues in RD research with a number of recently completed
               and ongoing TFs/WGs. The Targeting shared molecular etiologies to accelerate drug development for rare
               diseases TF recently published a review highlighting the potential of using basket clinical trial design for rare
                                                                                                 [9]
               diseases to accelerate the development of new therapies and address the unmet needs of patients . Current
               TF/WG efforts are focused on many different aspects of RDs, from funding of research and development
               through access to treatment and the impact on patients. Efforts focused on enabling the development of
               new therapeutics include the Drug Repurposing Guidebook and Preparing for Genetic N-of-1 Treatments of
               Patients with Ultra-rare Mutations TFs. The Functional Analysis TF seeks to develop a framework for a
               robust and effective ecosystem to support research into RDs, while Funding Models to Support the Spectrum
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