Critical Path Institute

“Peter hutt and jeff jacob have a business relationship. Wain fishburn and jeff jacob have a business relationship. Jeff jacob and shaun kirkpatrick have a business relationship. Tomas salmonson and jeff jacob have a business relationship.”

“Form 990 is prepared by an external cpa firm using information provided by the organization. The form is reviewed by the organization's cfo, coo, president/ceo, the board audit, finance, and risk committee, and the board of directors prior to being finalized.”

“All members of the board of directors and all officers of the organization are required to fill out a conflict of interest form annually. The results are compiled and reviewed by the board's audit, finance, and risk committee. Any actual or perceived conflicts that have the potential to bias any discussions or decisions by the board are discussed by the board. Any director with a real or potential conflict will recuse themselves from any discussion or decision where the conflict has a bearing.”

“A board of directors compensation committee meets and reviews executive compensation annually. Periodically, the committee requests a compensation analysis to determine if employee compensation has remained within market range or if adjustments are required. This information is then used as a basis for the compensation committee's recommendation to the board of directors. An overall salary program for employees must be approved by the board of directors compensation committee before implementation. All employee salary increases must be approved by the president/ceo.”

“Articles of incorporation can be found on the arizona corporation commission website. The conflict of interest policy and the financial statements (via the annual report) are posted on the institute's website.”

“The rare disease cures accelerator-data and analytics platform (rdca-dap) is an fda-funded initiative that provides a centralized and standardized infrastructure to support and accelerate rare disease characterization, with the goal of accelerating therapy development across rare diseases. This platform is made possible through a collaborative grant from the fda and in partnership with the national organization for rare disorders (nord). Rdca-dap promotes the sharing of existing patient-level data and encourages the standardization of new data collection. By integrating such data in a regulatory-grade format suitable for analytics, rdca- dap accelerates the understanding of disease progression (including sources of variability to optimize the characterization of subpopulations), clinical outcome measures and biomarkers, and facilitates the development of mathematical models of disease and innovative clinical trial designs. Rdca-dap is positioned to generate solutions to drug development bottlenecks. Accomplishments this year include: (1) signed 13 data contribution agreements and is currently negotiating agreements with 21 additional groups. As of june 30, 2021, the platform includes over 60 rare disease datasets in-house from 18 disease areas; (2) signed memoranda of understanding with five rare diseases data companies (u.s. And international) to frame collaborations that will help increase the pool of data available and the platform functionality, visibility and value to drug development; (3) completed two prototype projects to demonstrate the value of the platform by demonstrating the ability to integrate data across related diseases (polycystic kidney disease and kidney transplant) and development of a disease progression model for polycystic kidney disease. Rdca-dap also advanced pilot analytical work for disease modeling and data-driven solutions for freidreich's ataxia in collaboration with fara; and, (4) helped advance the platform development including beta-testing of the platform's first iteration (with testers from academic, data science/ quantitative modeling and platform/patient group segments of our end user base) and ongoing building of an application programming interface with nord's iamrare registry platform.”

“The goal of the rare disease clinical outcome assessment consortium (rd-coac) will be to optimize coa selection during medical product development for rare diseases. Work towards establishment of this consortium began with fda funding in 2019 as the rare disease subcommittee. The rare disease subcommittee was established within the pro consortium (proc) and includes representatives from fda, the national organization for rare disorders (nord), the national center for advancing translational science (ncats), the patient-centered outcomes research institute (pcori), biopharmaceutical firms, as well as three clinical experts. Their accomplishments this year include: (1) completed the landscape analyses for the daily function subdomains of self-care, gross motor function, fine motor function, and communication; (2) completed the gap analyses of 68 coas for the daily function subdomains of self-care, gross motor function, and fine motor function; (3) conducted a virtual workshop titled covid-19 mitigation strategies in pediatric rare disease clinical trials; 4) completed an online survey of nord's iamrare registry partners; and (5) expanded membership through agreements with new industry and non-profit members.”

“The goal of the huntington's disease regulatory science consortium (hd-rsc) is to bring together participants from across the global huntington's disease (hd) community with the goal to create and advance drug development tools for regulatory endorsement and accelerate the overall development of novel therapeutics for hd. Their accomplishments this year include: (1) submission of a letter of intent, as part of a larger briefing package, to fda under the fit-for-purpose initiative; (2) completion of two manuscripts on imaging biomarkers for submission to frontiers in neuroscience; (3) creation of the hd integrated staging system for clinical use by the regulatory science forum workgroup and a companion manuscript to be submitted to the lancet neurology; (4) expansion of the hd database, currently representing 28,300 patients.”