12:55 PM
Oct 12, 2017
 |  BC Innovations  |  Finance

IRDiRC dreams bigger

Public funding roundup 3Q17

After presiding over rapid growth in the global effort to diagnose and treat rare diseases over the past six years, the International Rare Diseases Research Consortium (IRDiRC) announced three new goals, one of which is finding new therapies for 1,000 indications.

In 2011, NIH and the European Commission created the consortium as a global coalition that has now grown to 52 government and non-profit funding bodies, biotech and pharmaceutical companies, patient advocacy groups and individual researchers. IRDiRC's founders wanted a goal-oriented collaboration like the Human Genome Project, but sought to also include companies such as Pfizer Inc. and Roche that could aid in drug development.

Individual funders, who have collectively spent over $2 billion on rare disease research since the consortium's launch, adopted changes recommended through IRDiRC's guidelines and policies on a series of topics impacting research and development.

Data on rare disease therapeutics and diagnostics are tracked by IRDiRC member Orphanet, an initiative coordinated by Institut National de la Santé et de la Recherche Médicale (INSERM) with European Commission support.

IRDiRC's original goal was for the rare disease community to get 200 new therapies approved in its first decade, a milestone it hit earlier this year.

Consequently, the consortium has decided to set new, more ambitious goals, IRDiRC Chair Christopher Austin told BioCentury. According to Austin, who is also director of NIH's National Center for Advancing Translational Sciences (NCATS), IRDiRC estimates that at current rates 600 new orphan medicinal products will be available in the next decade.

IRDiRC wants to push that number up even further. "We landed on 1,000 as an achievable goal," Austin said, adding that number would include any newly approved treatment, whether a new molecular entity or repurposed compound.

One of the reasons he thinks industry can meet the goal is that gains made in understanding disease biology have revealed connections between rare diseases once thought to be unrelated. That has led to a "changing paradigm" wherein multiple diseases are now being targeted via a shared pathway, he said.

He cited advances in technologies underlying gene therapy and gene editing, as well as better coordination of the field through IRDiRC, as additional contributors.

Austin said IRDiRC is developing a mechanism to help government funders coordinate their disease-specific research.

The consortium's second goal is diagnostic. Back in 2011, IRDiRC had also set a goal of developing means to diagnose most of the nearly 7,000 rare diseases within a decade. That goal has yet to be met, but Austin noted genomic analyses and clinical tools such as facial recognition software will soon bring it within reach.

In the meantime, IRDiRC's second new goal is for every patient worldwide who might have a previously identified rare disease to receive a definitive diagnosis within a year of reporting symptoms. "That should be a solvable problem, because it's a systems problem," Austin said. "The current diagnostic odyssey is somewhere between three and eight years, even a decade sometimes."

Patients without a diagnosis...

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