2020-04-29T19:09:58-05:00 2019-06-30 860683059 BEACHFLEISCHMAN PC 1985 E RIVER ROAD SUITE 201 TUCSON AZ 85718 990 2018-07-01 201991334 CRITICAL PATH INSTITUTE CRIT 5205473440 1730 EAST RIVER ROAD NO 200 TUCSON AZ 857185898 KRISTEN SWINGLE COO 5205473440 2020-04-27 1 KELLY L MELTZER CPA P00633511 5203214600 2020-04-27 64.128.200.66 2020-04-27 11:19:01 US P 30E46978D2C59AA32200D71C98AB2CF713052E80 30E46978D2C59AA32200D71C98AB2CF713052E80 2018 2020-11-19 02:30:22Z JOSEPH SCHEEREN 1730 EAST RIVER ROAD NO 200 TUCSON AZ 857185898 16797670 0 X WWW.C-PATH.ORG X 2004 AZ CREATE AND MANAGE INTERNATIONAL COLLABORATIONS AND DEVELOP NOVEL TOOLS, ENABLING THE FASTER DEVELOPMENT OF SAFER MEDICAL PRODUCTS. 12 11 79 11 0 0 10019550 10698823 5164609 5883801 83667 215046 0 0 15267826 16797670 1359100 2107859 0 0 8046940 8456998 0 0 0 5105672 4808109 14511712 15372966 756114 1424704 14421996 17507463 7699611 9360374 6722385 8147089 X THE CRITICAL PATH INSTITUTE IS A CATALYST IN THE DEVELOPMENT OF NEW APPROACHES THAT ADVANCE MEDICAL INNOVATION AND REGULATORY SCIENCE. THIS IS ACHIEVED BY LEADING TEAMS THAT SHARE DATA, KNOWLEDGE AND EXPERTISE RESULTING IN SOUND, CONSENSUS BASED SCIENCE. 0 0 12586197 2107859 5883801 SEE SCHEDULE O 12586197 1 1 0 1 0 0 0 0 0 0 1 0 0 0 0 1 0 1 0 0 1 1 0 0 0 0 0 1 0 1 0 0 0 0 0 0 1 0 0 0 0 0 0 1 1 1 0 0 1 16 0 1 79 1 0 1 EI 0 0 0 0 0 0 0 0 0 0 X 12 11 1 0 0 0 0 0 0 1 1 0 0 1 1 1 1 1 1 1 1 0 X ANNA M MIDKIFF CPA 5206478382 1730 EAST RIVER ROAD NO 200 TUCSON AZ 857185898 D CRAIG BRATER MD BOARD MEMBER 1.00 X 0 0 0 HONORABLE JAMES C GREENWOOD BOARD MEMBER 1.00 X 0 0 0 KAY HOLCOMBE MS BOARD MEMBER 1.00 X 0 0 0 PETER BARTON HUTT LLB LLM BOARD MEMBER 1.00 X 0 0 0 MICHAEL KASSER MBA PHD BOARD MEMBER 1.00 X 0 0 0 SHAUN A KIRKPATRICK MA BOARD MEMBER 1.00 X 0 0 0 ALAN G LEVIN MS CPA BOARD MEMBER 1.00 X 0 0 0 RICHARD T MYERS JR BOARD MEMBER 1.00 X 0 0 0 PAULA OLSIEWSKI PHD BOARD MEMBER 1.00 X 0 0 0 CINDY PARSEGHIAN MBA BOARD MEMBER 1.00 X 0 0 0 ALASTAIR JJ WOOD MB CHB BOARD MEMBER 1.00 X 0 0 0 TIMOTHY R FRANSON MD CHAIRPERSON 1.00 X 0 0 0 M WAINWRIGHT FISHBURN JR VICE CHAIRPERSON 1.00 X 0 0 0 JEFFREY E JACOB SM DEPUTY CHAIRPERSON 1.00 X 0 0 0 MARTHA BRUMFIELD PHD CEO/PRESIDENT THRU 04/14/2019 40.00 X X 296638 0 29272 JOSEPH SCHEEREN PHARMD CEO/PRESIDENT AS OF 04/15/2019 40.00 X X 0 0 0 LEWIS BARBIERI JD SECRETARY 40.00 X 117553 0 19234 STEVEN T BROADBENT MBA COO/TREASURER 40.00 X 200243 0 26831 LYNN HUDSON PHD CHIEF SCIENCE OFFICER 40.00 X 205384 0 17805 JOHN-MICHAEL SAUER PHD PROGRAM OFFICER 40.00 X 195763 0 30865 KLAUS ROMERO MD EXECUTIVE DIRECTOR 40.00 X 169174 0 16075 STEPHEN J COONS PHD PROGRAM OFFICER 40.00 X 225685 0 26157 STEVE ARNERIC PHD EXECUTIVE DIRECTOR 40.00 X 172952 0 25516 RICHARD LIWSKI CHIEF TECHNOLOGY OFFICER 40.00 X 151213 0 25035 DIANE STEPHENSON EXECUTIVE DIRECTOR 40.00 X 171020 0 24949 INISH O'DOHERTY EXECUTIVE DIRECTOR 40.00 X 169749 0 15190 SONYA EREMENCO ASSOCIATE DIRECTOR 40.00 X 157741 0 24083 DANIELA CONRADO ASSOCIATE DIRECTOR 40.00 X 134743 0 12070 MARCO SCHITO SCIENTIFIC DIRECTOR 40.00 X 133863 0 18839 2501721 0 311921 24 0 1 0 RTI HEALTH SOLUTIONS 200 PARK OFFICERS DRIVE RESEARCH TRIANGLE PARK NC 27709 RESEARCH 389277 LOTOS NILE PO BOX 90245 NASHVILLE TN 37209 MARKETING 144228 AMY C PORTER 4201 N QUAIL CANYON DR TUCSON AZ 85750 SCIENTIFIC WRITING 143950 EPHIBIAN 3180 N SWAN RD TUCSON AZ 85712 IT SERVICES 131445 KORN FERRY NW 5854 PO BOX 1450 MINNEAPOLIS MN 554855854 RECRUITING 127767 13 3770446 6928377 10698823 RESEARCH SUPPORT FEES 541700 5835771 5835771 OTHER REVENUE 900099 48030 48030 5883801 215046 215046 16797670 5883801 0 215046 X 1911647 1911647 196212 196212 1992133 1224986 767147 5150234 3942006 1208228 383337 294649 88688 432513 322320 110193 498781 363911 134870 23784 21462 2322 48892 36272 12620 2032457 2007948 24509 213210 155558 57652 385310 350197 35113 500061 438017 62044 551859 333730 218129 493458 465173 28285 441368 432059 9309 26250 19152 7098 45166 32953 12213 DUES AND SUBSCRIPTIONS 32061 27418 4643 MISCELLANEOUS 14233 10527 3706 15372966 12586197 2786769 0 3026764 4891967 4831456 5019135 166823 385302 596248 518992 92347 102576 663569 586811 83171 76758 5615214 6402760 9973 109973 14421996 17507463 600978 955803 7098633 8404571 7699611 9360374 X 6722385 8147089 6722385 8147089 14421996 17507463 1424704 0 X 0 1 X 1 1 1 X 10269811 10197464 10085049 10019550 10698823 51270697 10269811 10197464 10085049 10019550 10698823 51270697 24619913 26650784 82059 80580 48471 83667 215046 509823 51780520 25365071 0.51470 0.52990 X RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 0 0 0 15372966 15372966 918648 229662 0 0 883297 909814 875586 918648 3587345 5381018 11731 61348 60816 133895 220824 227454 218897 229662 896837 1345256 PART II-A, LINE 1B C-PATH DOES NOT LOBBY FOR OR AGAINST ANY SPECIFIC LEGISLATION. C-PATH DOES EXPEND A MINOR AMOUNT OF TIME AND MONEY TO EDUCATE LEGISLATORS AND THEIR STAFF IN THE AREA OF DRUG DEVELOPMENT. 42014 42014 0 188718 169212 19506 432837 375585 57252 76758 X PART X, LINE 2: FINANCIAL STATEMENT FIN 48 (ASC 740) FOOTNOTE: C-PATH IS EXEMPT FROM INCOME TAXES UNDER BOTH FEDERAL (INTERNAL REVENUE CODE SECTION 501(C)(3)) AND ARIZONA INCOME TAX LAWS, AND IS CLASSIFIED AS OTHER THAN A PRIVATE FOUNDATION UNDER INTERNAL REVENUE CODE SECTION 509(A)(1). INCOME FROM CERTAIN ACTIVITIES NOT DIRECTLY RELATED TO C-PATH'S TAX-EXEMPT PURPOSE, HOWEVER, MAY BE SUBJECT TO TAXATION AS UNRELATED BUSINESS TAXABLE INCOME (UBTI). SHOULD C-PATH EVER BE SUBJECT TO PENALTIES AND INTEREST ASSESSED BY VARIOUS TAXING AUTHORITIES, THEY WOULD BE CLASSIFIED AS MANAGEMENT AND GENERAL EXPENSES. 1 EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM 0 0 GRANTS TO RECIPIENTS LOCATED IN REGION CLINICAL RESEARCH 196212 EUROPE (INCLUDING ICELAND & GREENLAND) 0 0 INVESTMENT 100000 0 0 0 0 0 0 296212 0 296212 EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM CLINICAL RESEARCH 89424 WIRE TRANSFER EUROPE (INCLUDING ICELAND & GREENLAND) CLINICIAL RESEARCH 96785 WIRE TRANSFER EUROPE (INCLUDING ICELAND & GREENLAND) CLINICAL RESEARCH 10000 WIRE TRANSFER 3 1 0 1 0 0 0 PART I, LINE 2: CRITICAL PATH INSTITUTE ENTERS INTO SUBAWARD AGREEMENTS WITH SUBRECIPIENTS AND REVIEWS PROGRESS DELIVERABLES AND COSTS. 1 BAYLOR RESEARCH INSTITUTE 2001 BRYAN ST SUITE 2200 DALLAS TX 75201 751921898 501(C)(3) 1055538 DEVELOP A HOLLOW FIBER MODEL FOR TUBERCULOSIS RESEARCH REGENTS OF THE UNIVERSITY OF CALIFORNIA 1855 FOLSOM STREET BOX 0897 SAN FRANCISCO CA 94143 946036493 STATE OF CALIFORNIA 170000 RESEARCH UNIVERSITY OF FLORIDA PO BOX 113001 GAINSVILLE FL 32611 596002052 STATE OF FLORIDA 17000 RESEARCH CLINICAL DATA INTERCHANGE STANDARDS CONSORTIUM PO BOX 2068 ROUND ROCK TX 78680 043503931 501(C)(3) 137750 DEVELOP CLINICAL DATA STANDARDS THE TRANSLATIONAL GENOMICS RESEARCH INSTITUTE 445 N FIFTH STREET SUITE 600 PHOENIX AZ 85004 753065445 501(C)(3) 394953 DEVELOP THE RESEQ TB INTEGRATED PIPELINE FOUNDATION FOR THE NATIONAL INSTITUTES OF HEALTH INC 11400 ROCKVILLE PIKE SUITE 600 NORTH BETHESDA MD 20852 521986675 501(C)(3) 11653 CLINICAL EVALUATION AND QUALIFICATION OF KIDNEY SAFETY BIOMARKERS STUDY NORTHWESTERN UNIVERSITY 625 N MICHIGAN AVENUE STE 2700 CHICAGO IL 60611 362167817 501(C)(3) 77216 RESEARCH UNIVERSITY OF ARIZONA PO BOX 3520 TUCSON AZ 85722 742652689 STATE OF ARIZONA 30616 RESEARCH UNIVERSITY OF UTAH 295 CHIPETA WAY 1S133 SALT LAKE CITY UT 84108 876000525 STATE OF UTAH 16920 RESEARCH 9 PART I, LINE 2: CRITICAL PATH INSTITUTE ENTERS INTO SUBAWARD AGREEMENTS WITH SUBRECIPIENTS AND REVIEWS PROGRESS DELIVERABLES AND COSTS. X X 1 1 X X X X 1 0 0 0 0 0 0 1 0 MARTHA BRUMFIELD PHD CEO/PRESIDENT THRU 04/14/2019 296638 0 0 0 0 0 22000 0 7272 0 325910 0 0 0 STEVEN T BROADBENT MBA COO/TREASURER 200243 0 0 0 0 0 15612 0 11219 0 227074 0 0 0 LYNN HUDSON PHD CHIEF SCIENCE OFFICER 205384 0 0 0 0 0 15612 0 2193 0 223189 0 0 0 JOHN-MICHAEL SAUER PHD PROGRAM OFFICER 195763 0 0 0 0 0 16259 0 14606 0 226628 0 0 0 KLAUS ROMERO MD EXECUTIVE DIRECTOR 169174 0 0 0 0 0 5119 0 10956 0 185249 0 0 0 STEPHEN J COONS PHD PROGRAM OFFICER 225685 0 0 0 0 0 14736 0 11421 0 251842 0 0 0 STEVE ARNERIC PHD EXECUTIVE DIRECTOR 172952 0 0 0 0 0 14423 0 11093 0 198468 0 0 0 RICHARD LIWSKI CHIEF TECHNOLOGY OFFICER 151213 0 0 0 0 0 10776 0 14259 0 176248 0 0 0 DIANE STEPHENSON EXECUTIVE DIRECTOR 171020 0 0 0 0 0 13955 0 10994 0 195969 0 0 0 INISH O'DOHERTY EXECUTIVE DIRECTOR 169749 0 0 0 0 0 13540 0 1650 0 184939 0 0 0 SONYA EREMENCO ASSOCIATE DIRECTOR 157741 0 0 0 0 0 13194 0 10889 0 181824 0 0 0 MARCO SCHITO SCIENTIFIC DIRECTOR 133863 0 0 0 0 0 8209 0 10630 0 152702 0 0 0 PART I, LINE 1A JOSEPH SCHEEREN RECEIVED A STIPEND FOR HOUSING WHEN HE RELOCATED. THIS WAS GROSSED UP FOR PAYROLL TAXES AND ADDED TO HIS FORM W-2. PART I, LINE 4A STEVE ARNERIC RECEIVED A PAYMENT IN THE AMOUNT OF $20,801. PART I, LINE 7 VARIABLE PAY MAY BE ISSUED BASED UPON PERFORMANCE AT THE DISCRETION OF THE DEPARTMENT HEAD. AMY PORTER SPOUSE OF JOHN-MICHAEL SAUER, KEY EMPLOYEE 143950 SCIENTIFIC WRITING 0 FORM 990, PART III, LINE 4A THE CRITICAL PATH INSTITUTE (C-PATH) IS AN INDEPENDENT, NON-PROFIT ORGANIZATION UNIQUELY DEDICATED TO ADVANCING MULTIPLE ASPECTS OF THE FDA'S CRITICAL PATH INITIATIVE BY CREATING COLLABORATIONS AMONG REGULATORS (SCIENTISTS FROM THE FDA AND EMA), THE REGULATED MEDICAL PRODUCT INDUSTRY, ACADEMIA, PATIENT GROUPS, AND OTHER ORGANIZATIONS THAT ADVANCE MEDICAL INNOVATION THROUGH THE DEVELOPMENT OF TOOLS AND METHODS BASED UPON SOUND, CONSENSUS-BASED SCIENCE. THE ACCOMPLISHMENTS FOR THIS FISCAL YEAR REPORT INCLUDE: CRITICAL PATH FOR ALZHEIMER'S DISEASE (CPAD) DEVELOPS TOOLS AND NOVEL METHODOLOGIES TO ACCELERATE PROGRESS IN THE DEVELOPMENT OF PREVENTION AND TREATMENT STRATEGIES FOR ALZHEIMER'S DISEASE. THIS YEAR'S ACCOMPLISHMENTS INCLUDE: (1) LAUNCHING THE C-PATH INDUSTRY DATA SHARING INITIATIVE, BASED ON CLOSE COLLABORATION WITH FDA AND INDUSTRY MEMBERSHIP. (2) EXPANSION OF THE CONSORTIUM THROUGH THE EXECUTION OF ADDITIONAL DATA SHARING AGREEMENTS AND ENROLLMENT OF NEW INDUSTRY MEMBERS. THE CRITICAL PATH FOR PARKINSON'S (CPP) CONSORTIUM'S GOALS ARE TO DEVELOP QUANTITATIVE MODEL-BASED TOOLS TO ACCELERATE THE TREATMENT OF PARKINSON'S DISEASE, ESPECIALLY FOR THOSE INDIVIDUALS EXPERIENCING THE FIRST CLINICAL SIGNS OF MOTOR SYMPTOM ONSET. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) RECEIVING FEEDBACK FROM THE FDA ENDORSING THE CPP MODELING ANALYSIS PLAN WITH ADVICE TO ACQUIRE PARKINSON'S DISEASE RANDOMIZED CLINICAL TRIALS. (2) AGGREGATING A TOTAL OF 11 CLINICAL TRIAL DATASETS TO ENRICH THE CPP DATABASE AND FURTHER THE DEVELOPMENT OF A PARKINSON'S DISEASE-DRUG-TRIAL MODEL. (3) LAUNCHING THE DIGITAL DRUG DEVELOPMENT TOOLS (3DT) PROJECT WHICH HAS HELD THREE HIGH-IMPACT REGULATORY MEETINGS IN 2019. (4) LAUNCHING TWO NEW TASK FORCES TO AID IN SUPPORTING FUTURE REGULATORY ENGAGEMENT FOR PD DIGITAL DEVICE MEASURES. THE MISSION OF THE CRITICAL PATH TO TB DRUG REGIMENS (CPTR) INITIATIVE IS TO ACCELERATE THE DEVELOPMENT OF NOVEL TB DRUG REGIMENS THAT ARE SAFER, SHORTER, AND MORE EFFICACIOUS THAN THE CURRENT STANDARD OF CARE. THEIR MISSION EXPANDED TO ACCELERATE THE DEVELOPMENT OF A CLINICALLY USEFUL IN VITRO RAPID DRUG SUSCEPTIBILITY ASSAY TO SUPPORT TB REGIMEN DEVELOPMENT AND DEPLOYMENT. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) COMPLETED THE GRAPHICAL USER INTERFACE (GUI) FOR THE LONGITUDINAL TTP DYNAMICS MODEL LINKING TIME-TO-POSITIVITY TO CLINICAL OUTCOMES. (2) DEPLOYED THE RESEQTB WEBSITE (WWW.RESEQTB.ORG), A "ONE-STOP" SOURCE FOR CURATED, AGGREGATED, AND CLINICALLY RELEVANT GENETIC DRUG RESISTANCE DATA, TO SUPPORT THE TUBERCULOSIS DRUG RESISTANCE SURVEILLANCE PROGRAM AT THE WORLD HEALTH ORGANIZATION, EXPANDING ACCESSIBILITY (BEYOND RESEARCHER-LEVEL USE) TO THE RESEQTB DATA PLATFORM AND AVAILABLE BIOINFORMATICS AND VISUALIZATION TOOLS. (3) REANALYZED DATA IN THE RESEQTB PLATFORM TO GENERATE THE ANNUAL UPDATED DRUG RESISTANCE REPORT AND PERFORMED AN ANALYSIS OF NEW DRUG RESISTANCE MUTATIONS IDENTIFIED IN COMPARISON TO LAST YEAR'S REPORT. (4) COMPLETED SEQUENCING COLLABORATION PROJECT WITH TRANSLATIONAL GENOMICS RESEARCH INSTITUTE (TGEN) TO SEQUENCE OVER 8,800 MTB. ISOLATES. (5) AUTOMATED THE RESEQTB BIOINFORMATICS PIPELINE, REDESIGNED THE RELATIONAL DATABASE INFRASTRUCTURE TO OPTIMIZE DATA ANALYTICS, AND DEPLOYED A SCALABLE INSTANCE OF THIS PLATFORM TO THE WORLD HEALTH ORGANIZATION TO SUPPORT THEIR GLOBAL TB DRUG RESISTANCE SURVEILLANCE EFFORT. (6) COMPLETED THE HOLLOW FIBER SYSTEM STAGE II WORK IN WHICH TB MONOTHERAPY AND COMBINATION DRUG THERAPIES WERE COMPARED HEAD-TO-HEAD TO IDENTIFY THE MOST PROMISING REGIMEN COMBINATIONS AND DOSING SCHEDULES. (7) DEPLOYED TB-PACTS 2.0 ENHANCED SEARCH AND QUERY TOOL FOR INTERNAL TESTING. THE DATA COLLABORATION CENTER (DCC) IS A CENTER WITHIN C-PATH TO ENABLE MULTIPLE ORGANIZATIONS TO WORK TOGETHER IN A NEUTRAL SETTING AND SHARE CLINICAL DATA IN ORDER TO OPTIMIZE ITS VALUE IN CREATING NEW INSIGHTS AND TOOLS THAT ACCELERATE DRUG DEVELOPMENT IN AREAS WITH UNMET MEDICAL NEEDS. THE DCC SUPPORTS DATA SHARING PROJECTS ALIGNED WITH SPECIFIC C-PATH CONSORTIA AS WELL AS DATA SHARING INITIATIVES THAT ARE INDEPENDENT OF C-PATH CONSORTIA. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) PARTNERED WITH THE WORLD HEALTH ORGANIZATION (WHO) AND THE FOUNDATION FOR INNOVATIVE NEW DIAGNOSTICS (FIND) TO DEPLOY THE FIRST-EVER DATA PLATFORM FOR GLOBAL SURVEILLANCE OF TUBERCULOSIS RESISTANCE BASED UPON GENOMIC SEQUENCING DATA. THIS PLATFORM LEVERAGES THE TECHNOLOGY DEVELOPED FOR THE RELATIONAL SEQUENCING TB DATA PLATFORM (RESEQTB) AND WILL EQUIP WHO'S GLOBAL TB SURVEILLANCE GROUP WITH A CONSOLIDATED DATA MANAGEMENT TOOL FOR TRACKING DATA ON DRUG-RESISTANT TB ON A GLOBAL SCALE. (2) PARTNERED WITH THE FLINN FOUNDATION TO DEVELOP AND PILOT "PREVENT-HAARM" (HEALTHCARE-ASSOCIATED ANTIMICROBIAL-RESISTANT MICROBES), A FRAMEWORK TO TRACK AND RESPOND TO ANTIMICROBIAL RESISTANCE IN ARIZONA. C-PATH HAS PARTNERED WITH THE TRANSLATIONAL GENOMICS RESEARCH INSTITUTE PATHOGEN AND MICROBIOME DIVISION (TGEN-NORTH) TO DESIGN A FRAMEWORK FOR A STATEWIDE HEALTHCARE ASSOCIATED ANTIMICROBIAL RESISTANT MICROBE SURVEILLANCE SYSTEM. (3) PUBLISHED THERAPEUTIC AREA DATA STANDARDS FOR CLOSTRIDIUM DIFFICILE ASSOCIATED DIARRHEA (CDAD) AND THE TREATMENT OF HIV. (4) DEVELOPED A CDISC STANDARD FOR THE EXCHANGE OF NONCLINICAL DATA (SEND) IMPLEMENTATION GUIDE (IG) FOR ANIMAL RULE STUDIES. THE DUCHENNE REGULATORY SCIENCE CONSORTIUM'S (D-RSC) GOAL IS TO SUPPORT COLLABORATIVE RESEARCH AND REGULATORY QUALIFICATION OF NEW DRUG DEVELOPMENT TOOLS FOR DUCHENNE MUSCULAR DYSTROPHY, TO ENABLE THE EARLIEST POSSIBLE PATIENT ACCESS TO NEW TREATMENTS. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) SELECTION OF THE ANALYSIS DATASET, INITIAL STATISTICAL ANALYSIS PLAN, COMPLETION OF DRAFT MODELING ANALYSIS PLAN AND LETTER OF INTENT FOR FDA FIT-FOR-PURPOSE PATHWAY, EMA LETTER OF SUPPORT FOR GLDH AS A LIVER SAFETY BIOMARKER IN PATIENTS WITH UNDERLYING MUSCLE DAMAGE; (2) ACCEPTANCE OF THE LETTER OF INTENT TO FDA FIT-FOR-PURPOSE PATHWAY AND TO EMA QUALIFICATION OF NOVEL METHODOLOGIES PATHWAY. (3) DEVELOPED BASE AND COVARIATE PROGRESSION MODELS FOR 6 ENDPOINTS. (4) PUBLISHED A PAPER ON THE VALUE OF REGULATORY ACCEPTANCE OF MODELS. THE GOAL OF THE ELECTRONIC PATIENT-REPORTED OUTCOME CONSORTIUM (EPROC) IS TO ADVANCE THE SCIENCE OF CLINICAL TRIAL ENDPOINT ASSESSMENT BY COLLABORATIVELY SUPPORTING AND CONDUCTING RESEARCH, DESIGNING AND DELIVERING EDUCATIONAL OPPORTUNITIES, AND DEVELOPING AND DISSEMINATING BEST PRACTICE RECOMMENDATIONS FOR ELECTRONIC COLLECTION OF CLINICAL OUTCOME DATA. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) PARTICIPATED IN EDUCATIONAL ACTIVITIES AT DIA 2019 GLOBAL ANNUAL MEETING, INCLUDING PRESENTING A SHORT COURSE TITLED "ECOA 101: THE WHAT, WHY, AND HOW OF ECOA TO REDUCE BARRIERS TO ADOPTION IN CLINICAL TRIALS. AND HOSTING A FORUM TITLED "TRAINING FOR THE ELECTRONIC CAPTURE OF PRO DATA IN CLINICAL TRIALS:? VIEWS FROM THE EPRO VENDORS, SPONSORS, SITES, AND PATIENTS." (2) LAUNCHED THE ECOA: GETTING BETTER TOGETHER INITIATIVE, WHICH IS A PRE-COMPETITIVE COLLABORATION AMONG C-PATH, CLINICAL TRIAL SPONSORS FROM THE PRO CONSORTIUM, ECOA PROVIDERS AND CROS FROM THE EPRO CONSORTIUM, AND REGULATORS (FDA), TO ADDRESS CHALLENGES WITH THE IMPLEMENTATION OF ELECTRONIC CLINICAL OUTCOME ASSESSMENTS (ECOAS) IN CLINICAL TRIALS THE HUNTINGTON'S DISEASE REGULATORY SCIENCE CONSORTIUM (HD-RSC), IN PARTNERSHIP WITH CHDI FOUNDATION, IS FOCUSED ON THE DEVELOPMENT OF DRUG DEVELOPMENT TOOLS FOR HUNTINGTON'S DISEASE. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) HOLDING AN INAUGURAL PROGRAM MEETING WITH 80+ STAKEHOLDERS IN ATTENDANCE. (2) SUBMITTING RESPONSE TO EMA REGULATORY SCIENCE TO 2025 STRATEGIC REFLECTION. (3) CONTRIBUTING COMMENTS TO THE FDA DRAFT GUIDANCE ON THE USE OF NATURAL HISTORY STUDIES IN DRUG DEVELOPMENT FOR RARE DISEASES. (4) SIGNING ON 4 NEW INDUSTRY MEMBERS AND 2 NEW NON-PROFIT MEMBERS. (5) EXECUTING 3 DATA COLLABORATION AGREEMENTS FOR THE INCLUSION OF 4 OBSERVATIONAL DATASETS. AT THE REQUEST OF THE FDA, C-PATH FORMED AN INTERNATIONAL NEONATAL CONSORTIUM (INC). THE PURPOSE OF THE CONSORTIUM IS TO ACCELERATE THE DEVELOPMENT OF SAFE AND EFFECTIVE THERAPIES FOR NEONATES. INC ENGAGES THE GLOBAL NEONATAL COMMUNITY - FAMILIES, NEONATAL NURSES, ACADEMIC SCIENTISTS, REGULATORS, PHARMACEUTICAL INVESTIGATORS, ADVOCACY ORGANIZATIONS, AND FUNDERS - TO FOCUS ON THE NEEDS OF THE NEONATE. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) INC'S SEIZURE WORKGROUP DEVELOPED RECOMMENDATIONS FOR USE IN A MASTER PROTOCOL TO EVALUATE MEDICATIONS TO TREAT NEONATAL SEIZURES.?(2) INC'S RETINOPATHY OF PREMATURITY (ROP) WORKGROUP DEVELOPED A SCALE TO DEFINE A COMMON SOURCE OF BLINDNESS IN PRETERM INFANTS.?(3) INC'S NECROTIZING ENTEROCOLITIS (NEC) WORKGROUP DEVELOPED A FRAMEWORK TO IDENTIFY AND TREAT NEONATAL GASTROINTESTINAL INJURY. FORM 990, PART III, LINE 4A THE MULTIPLE SCLEROSIS OUTCOME ASSESSMENTS CONSORTIUM (MSOAC) HAS A GOAL TO OBTAIN REGULATORY QUALIFICATION OF AN IMPROVED CLINICAL OUTCOME ASSESSMENT INSTRUMENT AS A PRIMARY OR SECONDARY ENDPOINT IN CLINICAL TRIALS OF MULTIPLE SCLEROSIS (MS) THERAPIES. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) COMPLETED ANALYSIS ON THE CURATED, REMAPPED, AND AGGREGATED CONTROL AND TREATMENT ARMS OF 16 CLINICAL TRIAL DATASETS. (2) RECEIVED A DRAFT POSITIVE QUALIFICATION OPINION FROM THE CHMP/EMA. THE POLYCYSTIC KIDNEY DISEASE OUTCOMES CONSORTIUM (PKDOC) HAS DEVELOPED AND OBTAINED REGULATORY QUALIFICATION FROM THE U.S. FOOD AND DRUG ADMINISTRATION (FDA) AND THE EUROPEAN MEDICINES AGENCY (EMA) OF TOTAL KIDNEY VOLUME (TKV) AS A PROGNOSTIC BIOMARKER FOR USE IN CLINICAL TRIALS FOR NEW THERAPIES FOR AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE (ADPKD). THE CONSORTIUM CONTINUES TO EXPLORE ALTERNATE CLINICAL ENDPOINTS, INCLUDING A COMPOSITE ENDPOINT, INNOVATIVE CLINICAL TRIAL DESIGNS, AND REGULATORY PATHWAYS TO HELP EXPEDITE NEW THERAPIES IN PKD. THE FDA HAS COMMUNICATED THAT TKV IS A "REASONABLY LIKELY SURROGATE," WHICH MAKES IT ELIGIBLE FOR AN ACCELERATED APPROVAL PROCESS AT THE FDA. SIX NEW COMPANIES ARE NOW WORKING TO DEVELOP THERAPIES FOR THIS DISEASE. THE FDA APPROVED OTSUKA'S DRUG (JYNARQUETM). THIS IS THE FIRST-EVER FDA-APPROVED TREATMENT FOR PKD. ALTHOUGH IT WAS NOT A DIRECT OUTPUT OF PKDOC, THE CONSORTIUM'S WORK POSITIVELY CONTRIBUTED TO ADVANCES IN THE FIELD CONSISTENT WITH THIS SUCCESS STORY. THERE IS RENEWED INTEREST FROM INDUSTRY MEMBERS TO FURTHER EXPAND THE EFFORTS OF THE PKDOC. THE PATIENT-REPORTED OUTCOME CONSORTIUM (PROC) HAS A GOAL TO ESTABLISH AND MAINTAIN A COLLABORATIVE FRAMEWORK WITH APPROPRIATE STAKEHOLDERS FOR THE QUALIFICATION OF PATIENT-REPORTED OUTCOME (PRO) INSTRUMENTS AND OTHER CLINICAL OUTCOME ASSESSMENT (COA) TOOLS THAT WILL BE MADE AVAILABLE FOR USE IN CLINICAL TRIALS WHERE COA-BASED ENDPOINTS ARE USED TO SUPPORT PRODUCT LABELING CLAIMS. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) TWO PRO MEASURES WERE QUALIFIED BY FDA FOR EXPLORATORY USE, FOR THE ASTHMA DAYTIME SYMPTOM DIARY (ADSD) AND THE ASTHMA NIGHTTIME SYMPTOM DIARY (ANSD). (2) FDA INDICATED THAT THE CONSENSUS-BASED HARMONIZED MYELOFIBROSIS SYMPTOM ASSESSMENT MEASURE, THE MYELOFIBROSIS SYMPTOM ASSESSMENT FORM (MFSAF) V4.0 DIARY, WOULD BE ADDED TO FDA'S COA COMPENDIUM. (3) THE PRO CONSORTIUM HAS ISSUED A TOTAL OF 22 LICENSES FOR USE OF PRO CONSORTIUM MEASURES INCLUDING THE SMDDS, THE NSCLCSAQ, AND THE MYELOFIBROSIS SYMPTOM ASSESSMENT FORM V4.0 (MFSAF V4.0). (4) THE FOLLOWING COAS ARE IN VARIOUS STAGES OF THE DEVELOPMENT AND/OR QUALIFICATION PROCESS: UCSD PERFORMANCE-BASED SKILLS ASSESSMENT FOR MILD COGNITIVE IMPAIRMENT (UPSA-MCI), DIARY FOR IRRITABLE BOWEL SYNDROME SYMPTOMS-C (DIBSS-C) FOR CONSTIPATION PREDOMINANT IBS, DIARY FOR IRRITABLE BOWEL SYNDROME SYMPTOMS-D (DIBSS-D) FOR DIARRHEA PREDOMINANT IBS, DIARY FOR IRRITABLE BOWEL SYNDROME SYMPTOMS-M (DIBSS-M) FOR MIXED IBS, AN OPTIMIZED PROMIS PHYSICAL FUNCTION SHORT FORM FOR MS PATIENTS, PEDIATRIC ASTHMA DIARY - OBSERVER (PAD-O), PEDIATRIC ASTHMA DIARY - CHILD (PAD-C), RHEUMATOID ARTHRITIS WG - PROMIS SHORT FORM V1.0 - FATIGUE 10A. THE PREDICTIVE SAFETY TESTING CONSORTIUM HAS A GOAL TO BRING TOGETHER PHARMACEUTICAL COMPANIES TO SHARE AND VALIDATE INNOVATIVE SAFETY TESTING METHODS UNDER ADVISEMENT OF THE U.S. FOOD AND DRUG ADMINISTRATION (FDA), EUROPEAN MEDICINES AGENCY (EMA), AND JAPAN'S PHARMACEUTICALS AND MEDICAL DEVICES AGENCY (PMDA). CURRENTLY PSTC IS FOCUSED ON DEVELOPING AND OBTAINING REGULATORY QUALIFICATION OF IMPROVED CLINICAL SAFETY BIOMARKERS FOR USE IN DRUG DEVELOPMENT. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) IN COOPERATION WITH MT. SINAI AND FDA, INITIATED THE PREDICTOX PRE-CONSORTIUM FOCUSED ON THE DEVELOPMENT OF QUANTITATIVE TOOLS TO BETTER PREDICT CLINICAL SAFETY FOR PRECLINICAL DATA. (2) MET WITH PMDA TO UPDATE ON STATUS OF KIDNEY CLINICAL QUALIFICATION AND REQUEST INITIATING QUALIFICATION FOR GLUTAMATE DEHYDROGENASE AS A BIOMARKER OF DRUG-INDUCED LIVER INJURY. (3) ORGANIZED AND FACILITATED WEBINAR ON ACETAMINOPHEN-INDUCED LIVER TOXICITY MECHANISMS, DIAGNOSIS AND TREATMENT. (4) PRESENTED AT THE WORLD BIOMARKER CONGRESS. THE VISION OF THE QUANTITATIVE MEDICINE PROGRAM IS TO IMPROVE INDIVIDUAL AND POPULATION HEALTH BY TRANSFORMING DRUG DEVELOPMENT THROUGH METHODOLOGICAL INNOVATION, AND TO BECOME A NATIONALLY AND INTERNATIONALLY RECOGNIZED RESOURCE FOR MODEL-INFORMED DRUG DEVELOPMENT (MIDD). THE MISSION OF C-PATH'S QUANTITATIVE MEDICINE IS TO INTEGRATE NON-CLINICAL AND CLINICAL DATA WITH ANALYTICAL EXPERTISE TO SOLVE BOTTLENECKS IN THE DRUG DEVELOPMENT PROCESS. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) ACHIEVED THE FIRST-EVER LETTER OF SUPPORT FOR A QUANTITATIVE DRUG DEVELOPMENT TOOL FOR THE MODEL-BASED TRIAL ENRICHMENT PLATFORM THAT INCORPORATES HIPPOCAMPAL VOLUME AS A PROGNOSTIC BIOMARKER IN ALZHEIMER'S DISEASE TRIALS. (2) RECEIVED CONFIRMATION FROM FDA ON FUNDING FOR THE TWO MIDD COMPONENTS OF THE CORE GRANT TO A) ESTABLISH A POST-DOCTORAL FELLOWSHIP PROGRAM IN MIDD AT C-PATH AND B) DEVELOP AN ONLINE COURSE IN MIDD FOR FDA SCIENTISTS OF VARYING BACKGROUNDS. THE TYPE I DIABETES CONSORTIUM (T1D) IS FOCUSING ON QUALIFYING ISLET AUTOIMMUNITY ANTIBODIES AS SUSCEPTIBILITY/RISK BIOMARKERS TO BE USED IN THE DEVELOPMENT OF THERAPIES FOR THE TREATMENT, AND ULTIMATELY, THE PREVENTION, OF TYPE 1 DIABETES. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) RECEIVED A POSITIVE FDA RESPONSE TO T1DC'S LETTER OF INTENT AND ENCOURAGED TIDC TO PROCEED TO DEVELOP THE QUALIFICATION PLAN. (2) SUBMITTED A FINAL LETTER OF INTENT AND BRIEFING PACKAGE TO EMA. (3) DRAFTED A PLAN FOR THE GENERATION OF THE T1D DISEASE PROGRESSION MODEL REQUIRED TO SUPPORT THE REGULATORY ENDORSEMENT OF THE ISLET AUTOANTIBODIES. THE TRANSPLANTATION THERAPEUTICS CONSORTIUM IS WORKING TO IDENTIFY MECHANISMS AND DRUG DEVELOPMENT TOOLS TO ACCELERATE DRUG DISCOVERY FOR TRANSPLANT PATIENTS, THROUGH THE COLLABORATIVE INVOLVEMENT OF KEY STAKEHOLDERS IN THE FIELD. THE CONSORTIUM IS INITIALLY FOCUSING ON KIDNEY TRANSPLANT BUT MAY EXPAND TO OTHER SOLID ORGAN TRANSPLANTS IN THE FUTURE. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) PRESENTED AT CDER OFFICE OF ANTIMICROBIAL PRODUCTS/DIVISION OF TRANSPLANT AND OPHTHALMOLOGY PRODUCTS GRAND SCIENTIFIC ROUNDS WITH APPROXIMATELY 385 FDA PARTICIPANTS. (2) RECEIVED NOTIFICATION OF AWARD OF TWO FDA BROAD AGENCY ANNOUNCEMENT PROPOSALS. (3) CONDUCTED CRITICAL PATH INNOVATION MEETING WITH FDA TO DISCUSS AND ALIGN TTC EFFORTS WITH AGENCY'S PERSPECTIVE ON UNMET NEEDS AND POTENTIAL REGULATORY SOLUTIONS TO CHALLENGES THAT HAVE LED TO STAGNATION OF DRUG DEVELOPMENT IN SOLID ORGAN TRANSPLANTATION. (4) ACQUIRED DATASETS FROM 4 PARTNERS TO DATE. FORM 990, PART VI, SECTION A, LINE 2 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. FORM 990, PART VI, SECTION B, LINE 11B FORM 990 IS PREPARED BY AN EXTERNAL CPA FIRM USING INFORMATION PROVIDED BY THE ORGANIZATION. THE FORM IS REVIEWED BY THE ORGANIZATION'S DIRECTOR OF FINANCE, COO, PRESIDENT/CEO, THE BOARD AUDIT, FINANCE, AND RISK COMMITTEE, AND THE BOARD OF DIRECTORS PRIOR TO BEING FINALIZED. FORM 990, PART VI, SECTION B, LINE 12C 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. FORM 990, PART VI, SECTION B, LINE 15 A BOARD OF DIRECTORS COMPENSATION COMMITTEE MEETS AND REVIEWS EXECUTIVE COMPENSATION ANNUALLY. EVERY FEW YEARS THE COMMITTEE SEEKS AN INDEPENDENT COMPENSATION CONSULTING ORGANIZATION. AFTER THE CONSULTING ORGANIZATION HAS BEEN RETAINED, THE COMPENSATION COMMITTEE RECEIVES THE CONSULTANT'S EXECUTIVE COMPENSATION REVIEWS AND RECOMMENDATIONS. THIS INFORMATION IS THEN USED AS A BASIS FOR THE COMPENSATION COMMITTEE'S RECOMMENDATION TO THE BOARD OF DIRECTORS. IN CONNECTION WITH THE HIRING OF OUR NEW CEO IN APRIL 2019, THE SEARCH FIRM UTILIZED FOR RECRUITMENT CONDUCTED A BENCH-MARKING ANALYSIS TO DETERMINE AN APPROPRIATE SALARY. 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. FORM 990, PART VI, SECTION C, LINE 19 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. FORM 990, PART IX, LINE 11G RESEARCH SUPPORT SERVICES: PROGRAM SERVICE EXPENSES 1,325,218. MANAGEMENT AND GENERAL EXPENSES 594. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 1,325,812. CONSULTING: PROGRAM SERVICE EXPENSES 651,056. MANAGEMENT AND GENERAL EXPENSES 20,513. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 671,569. OTHER PROFESSIONAL FEES: PROGRAM SERVICE EXPENSES 31,674. MANAGEMENT AND GENERAL EXPENSES 3,402. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 35,076. CRITICAL PATH GLOBAL LTD 20-22 BEDFORD ROW LONDON UK WC1R 4JS FACILITATE WORK WITH EUROPEAN MEDICINES AGENCY UK CRITICAL PATH INSTITUTE C 1.00000 1 CRITICAL PATH INSTITUTE LTD 33 SIR JOHN ROGERSONS QUAY DUBLIN EI 2 FACILITATE WORK WITH EUROPEAN MEDICINES AGENCY EI CRITICAL PATH INSTITUTE C 73838 1.00000 1 0 0 0 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 CRITICAL PATH INSTITUTE LTD D 100000 CASH