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An EU Task Force to assess the burden of rare diseases has recently been established, comprised of
international experts who will meet regularly to map the burden of disease indicators, and identify barriers
[11]
in accessing primary, secondary, and tertiary health care services for people with RDs . This EU Task
Force will collaborate with IRDiRC to assess the impact of RDs on patients based on previously published
recommendations .
[12]
Critical Path Institute (C-Path) is an independent nonprofit, public-private partnership with the U.S. Food
and Drug Administration (FDA) created under the auspices of the FDA’s Critical Path Initiative program in
2005. IRDiRC and C-Path share common goals to improve and streamline the process of medical product
development by fostering precompetitive collaboration. A current focus is on clinical outcome assessments
for rare diseases that optimize the incorporation of the patient’s voice in the evaluation of treatment
efficacy.

In addition to the partnerships described above, IRDiRC and its members often participate as invited
speakers or panel participants in international scientific, patient group, and industry meetings such as the
Orphan Drug Congress Europe, American Society of Gene & Cell Therapy (ASGCT), and the Orphan
Drugs and Rare Diseases Global Congress. IRDiRC has also been a supporting partner for international
conferences such as the ECRD (European Rare Disease and Orphan Products) conference organized by
EURODIS (European Organization for Rare Diseases) with Orphanet, and the Orphan Drug Congress
Europe.

Global collaboration through a global pandemic
The global COVID-19 pandemic that began in December 2019 posed significant challenges not only for RD
research but also for patients seeking care for rare or undiagnosed diseases. To understand the impact of the
pandemic on the RD community, IRDiRC conducted a survey of its members in the summer of 2020 when
many countries and communities were in the midst of full lockdown measures to counter the threat from
COVID-19 . While it was evident that the pandemic did have an impact on funding, research activities,
[13]
and patient access to diagnostics, therapies and clinical trials, in general, the sentiment of the IRDiRC
membership was that the three stated goals were still achievable by 2027. The first goal of patients receiving
a diagnosis within 1 year was still achievable if research returned to pre-pandemic levels and healthcare
centers remained accessible to patients. The second goal of 1,000 new therapies remains achievable if
research funding is restored and research efforts that were previously diverted towards COVID-19 are re-
focused on RDs. The third goal of developing methodologies to measure the impact of diagnoses and
therapies on RDs was perhaps the least affected by the pandemic, as work towards this goal could be
executed by the increased use of teleconferencing linking collaborators globally.

The COVID-19 pandemic highlighted one of the major strengths of the Consortium, which is its ability to
work remotely in a highly collaborative manner. Despite the travel restrictions imposed by the pandemic,
the work of IRDiRC continued mostly unhindered, in that the inherent global nature of its membership,
which already relied heavily on teleconferencing for most of its activities. Events that were previously
conducted in person, such as the semi-annual Consortium Assembly meetings, the biennial scientific
conference, and Task Forces, pivoted to virtual meetings, enabling continued discussion amongst
consortium members. Output from TFs/WGs continued during the pandemic, albeit at a slower pace,
demonstrating the resilience of the Consortium to the global health crisis COVID-19.

As the world began to recover from the pandemic and travel restrictions were lifted, IRDiRC maintained the
use of the hybrid format meeting to ensure maximum participation from all members, irrespective of their