Rare illness cures accelerator-data and analytics platform milestones highlighted

TUCSON, Ariz. and WASHINGTON, D.C. October 21, 2020 — The Critical Path Institute (C-Path) and the National Organization for Rare Disorders (NORD®) hosted the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) 2020 Virtual Workshop on Monday, October 19. The platform, funded by a cooperative settlement by means of the U.S. Food and Drug Administration (FDA), serves as a centralized and standardized infrastructure to assist knowledge sharing and knowledge evaluation to assist within the understanding of uncommon ailments.

Theresa Mullin, Ph.D., Associate Director for Strategic Initiatives, FDA, Center for Drug Evaluation and Research (CDER) opened the workshop and defined how RDCA-DAP is a part of the FDA’s aggressive plan to advance drug growth for uncommon ailments. “The concept of RDCA-DAP has been discussed by C-Path and NORD with hundreds of stakeholders through public meetings, webinars, focus groups and one on one interviews to understand what the community would like to see in an analytics platform,” Mullin mentioned. “These learnings are being used now to make the platform as useful as possible to as many people as possible.”

The digital workshop, attended by greater than 400 people from affected person teams, academia, business and regulatory businesses knowledgeable the uncommon illness group of the initiative’s first-year progress. Topics included pilot analyses and the event of instruments facilitated by RDCA-DAP to advance characterization and speed up drug growth in uncommon ailments. Additional updates have been supplied on the platform’s knowledge sharing infrastructure and up to date enhancements, together with the power to look, kind and analyze knowledge.

C-Path RDCA-DAP Executive Director Jane Larkindale, D.Phil., detailed milestones the hassle achieved in its first 12 months and outlined plans for the long run. In 12 months one, the RDCA-DAP group labored with the group to develop an information governance construction that solutions the wants of each knowledge contributors and knowledge customers, accessed preliminary datasets and constructed actionable prototypes to reveal the utility of the platform.

“Our priority is not to compete, but to collaborate with other groups doing the same or similar work in rare disease drug development,” mentioned Larkindale. “Stakeholders are open to collaboration and data sharing and we have been meeting with different groups about what is already being done and how we can interoperate with other existing projects.”

Several case research have been introduced through the workshop to reveal the ability of the platform together with an illustration of the worth of the mixing of information and evaluation of mixed datasets. In one case, Pravin Jadhav, Ph.D., world group chief at Otsuka America Pharmaceutical, Inc. shared how patient-level knowledge aided within the acceptance of a biomarker for an accelerated approval of a brand new remedy for polycystic kidney illness.

C-Path’s Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. shared examples of prototypes constructed from RDCA-DAP patient-level knowledge that demonstrated the power to combine knowledge from a number of illness areas to generate superior fashions of illness development and to optimize the characterization of uncommon ailments, as a way to speed up drug growth for these situations.

Similarly, NORD’s Director of Research Vanessa Boulanger, M.Sc. used the instance of the NPKUA phenylketonuria affected person registry from NORD’s IAMRARE™ platform to point out how affected person registry knowledge can inform drug growth applications.

“The PKU case study is one example of the insights that patient registry and natural history study data can bring to RDCA-DAP,” mentioned Boulanger. “Patient-driven data gives us an opportunity to arrive at a more comprehensive understanding of rare diseases, to learn what matters to patients, and then to apply that knowledge to the design of studies that may result in treatments that are truly meaningful to the community.”

RDCA-DAP is positioned to generate options to drug growth bottlenecks by maximizing the utility of patient-level knowledge and making that knowledge accessible to a bigger group. C-Path President and CEO Joseph Scheeren, Pharm.D. and NORD President and CEO Peter L. Saltonstall each thanked the group for coming collectively across the idea of RDCA-DAP, acknowledging that knowledge contribution is essential to its success. FDA/CDER Office of Neuroscience Acting Director Billy Dunn, M.D. closed the workshop congratulating the groups on the work achieved since RDCA-DAP’s launch in September 2019 and discussing the three major areas of the FDA’s uncommon illness drug growth efforts, together with RDCA-DAP, normal core medical consequence assessments and medical trials community.

“The reason RDCA-DAP is so critical, is that it focuses on the characterization of the disease and if we don’t have a good characterization of the disease, we really can’t do our job,” Dunn mentioned. “Natural history, the variability of symptoms and experiences and the opportunities for clinical trial design are all addressed by a more robust characterization of disease.”


Groups occupied with contributing knowledge to the hassle, collaborating on the event of the platform or utilizing the database might go to c-path.org/rdca-dap or e-mail rdcadap@c-path.org for extra info. The platform is open to simply accept knowledge instantly, however the consumer interface and analytics shall be accessible in early 2021, with extra analytics instruments being developed concurrently.

A full recording of the workshop will be accessed here.

Funding for this program was made attainable, partially, by the Food and Drug Administration by means of grant (U18 FD 005320). Views expressed in written supplies or publications and by audio system and moderators don’t essentially replicate the official insurance policies of the Department of Health and Human Services; nor does any point out of commerce names, business practices, or group suggest endorsement by the United States Government.

About Critical Path Institute

Critical Path Institute (C-Path) is an impartial, nonprofit group established in 2005 as a private and non-private partnership. C-Path’s mission is to catalyze the event of latest approaches that advance medical innovation and regulatory science, accelerating the trail to a more healthy world. An worldwide chief in forming collaborations, C-Path has established quite a few world consortia that at the moment embrace greater than 1,600 scientists from authorities and regulatory businesses, academia, affected person organizations, illness foundations, and dozens of pharmaceutical and biotech corporations. C-Path US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with extra employees in a number of different areas. For extra info, go to c-path.org and c-path.eu.

About the National Organization for Rare Disorders (NORD)®

The National Organization for Rare Disorders (NORD®) is the main impartial advocacy group representing all sufferers and households affected by uncommon ailments. NORD started as a small group of affected person advocates that shaped a coalition to unify and mobilize assist to go the Orphan Drug Act of 1983. Since then, NORD has led the way in which in voicing the wants of the uncommon illness group, driving supportive insurance policies and schooling, advancing medical analysis and offering affected person and household companies for individuals who want them most. NORD is made sturdy along with over 325 disease-specific member organizations and their communities and collaborates with many different organizations on particular causes of significance to the uncommon illness affected person group. Visit rarediseases.org.


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