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, president/coo, 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/coo.”

“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 24 data contribution agreements (dcas), twelve mous and thirteen ndas negotiated and signed to date to date; (2) the rdca-dap platform currently includes over 75 rare disease data sets representing 25 disease states from 53,001 patients; (3) manuscript, "innovations in therapy development for rare diseases through the rare disease cures accelerator data and analytics platform," submitted to therapeutic innovation & regulatory science completed and published white paper on rdca-dap website titled "rare diseases cures accelerator data and analytics platform (rdca-dap) best practices and recommendations for fair data, toward alignment with international regulatory agencies," sharing lessons learned for storing and sharing data and strategies for collecting registry data that will maximize impact on the drug and diagnostic tools ecosystem; (4) technical advisory group meeting held with fda and nord to demonstrate the platform to a wider fda audience.”

“The goal of the cure drug repurposing collaboratory (cdrc) is to be the central global source of validated real-world data of repurposed drugs for diseases with the highest levels of unmet medical need. To accomplish this goal, cdrc is working with multiple stakeholders to develop open-sourced tools to automate the extraction, aggregation, analysis and sharing of key clinical data elements from electronic health records in a deidentified manner regardless of the vendor or data model for identifying fda approved drugs prescribed for new indications. In addition, cdrc is working to develop a web-based patient portal for patients to provide their treatment journey experience to alleviate symptoms of disease that is not on the current drug label or ameliorate side effects of existing fda approved and prescribed treatments. The analysis of real-world data from both clinicians and patients will be used to generate hypothesis that can supplement existing clinical trial data or inform clinical trial design in how existing fda approved drugs can be evaluated in randomized trials to assess treatment efficacy. The utility of systematically collecting existing real-world data to generate real-world evidence and linkage to clinical trials will help inform prescribers how to maximize the use of existing fda approved drugs and leverage existing data to change clinical practice and help address the high unmet clinical need of patients with diseases that have little or no treatment options. Accomplishments this year include: (1) designed and constructed a bioinformatic data processing pipeline in amazon web services (aws) to digest data from cure id and generate informative insights for drug repurposing; (2) defined rare disease pilots for oncology (sarcoma subtypes: angiosarcoma, perivascular epithelioid cell tumors and epithelioid hemangioendothelioma) and non-oncology (rasopathies with the exception of neurofibromatosis type 1), engaged clinical experts to review and define data elements, established drug repurposing task force with patient groups; (3) secured funding for create from three pharma companies who expect to come to market soon with new products for ibd but the project requires additional investment to build the registry infrastructure; (4) finalized protocol synopsis and identified pilot sites (baylor, hopkins and uab) for securing congenital cmv data from ehrs; (5) successfully uploaded covid-19 data automatically extracted from ehr from first pilot site to c-path using the edge tool; (6) established who collaboration on sexually transmitted infection treatment failures for gonorrhea and mycoplasma genetalium.”

“The objectives of c-path's real-world data analytics platform (rw-dap) project are to assimilate clinical trial datasets, electronic health records (ehr), and registry data from global partners and generate actionable solutions to accelerate drug development in neonatology. This project entails generating a real-world data analytics platform (rw-dap) that will aggregate, standardize, and integrate patient-level real world data (rwd) to (1) establish neonatal laboratory value reference ranges by gestational and postnatal age, and other patient-level characteristics, and (2) develop a quantitative disease progression model for bronchopulmonary dysplasia (one of the most challenging respiratory complications of prematurity). Any gaps that interfere with optimal use of existing rwd will be identified, and solutions that will improve the usability of rwd in the future will be proposed. During the past one year, we have continued to meet and make significant progress with pharmaceutical companies, academic institutes, and neonatal registry networks for the project. Discussions have been ongoing with 22 academic institutes who have indicated a willingness to collaborate with us. In aggregate, c-path has received datasets representing >200,000 patients and expects 100,000 additional patients in future datasets with partners and collaborators. In order to focus and leverage the experience and expertise of the large inc stakeholder network, we have created bpd and laboratory value workgroups. The bpd workgroup has 30 members, comprised of academic, industry, and regulatory researchers with expertise in bpd research as well as parent members. The laboratory value workgroup has 20 members, comprised of academic and industry researchers who have a longstanding interest in creating generalizable reference ranges for laboratory values in neonates.”

“Research support services: program service expenses 1,481,156. Management and general expenses 0. Fundraising expenses 0. Total expenses 1,481,156. Consulting: program service expenses 1,166,698. Management and general expenses 415,577. Fundraising expenses 0. Total expenses 1,582,275. Other professional fees: program service expenses 400,050. Management and general expenses 287,427. Fundraising expenses 0. Total expenses 687,477.”