2021-05-07T17:24:26-05:00 2020-06-30 860683059 BEACHFLEISCHMAN PC 1985 E RIVER ROAD SUITE 201 TUCSON AZ 85718 990 2019-07-01 201991334 CRITICAL PATH INSTITUTE CRIT 5205473440 1730 EAST RIVER ROAD NO 200 TUCSON AZ 857185898 KRISTEN L SWINGLE COO 5205473440 2021-05-07 1 KELLY L MELTZER CPA P00633511 5203214600 2021-05-07 2019 2022-09-23 18:48:47Z KRISTEN L SWINGLE 1730 EAST RIVER ROAD NO 200 TUCSON AZ 857185898 18464314 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. 13 12 99 12 0 0 10698823 12978641 5883801 5292779 215046 156753 0 36141 16797670 18464314 2107859 2697926 0 0 8456998 10893350 0 0 0 4808109 3961324 15372966 17552600 1424704 911714 17507463 16657850 9360374 7630738 8147089 9027112 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 13428896 2697926 5328920 SEE SCHEDULE O 13428896 1 1 0 1 0 0 0 0 0 0 1 0 0 0 1 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 25 0 1 99 1 0 1 EI 0 0 0 0 0 0 0 0 0 0 X 13 12 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 DR MARTHA A BRUMFIELD FORMER PRESIDENT & CEO 20.00 X 236390 0 24051 DR JOSEPH SCHEEREN PRESIDENT & CHIEF EXECUTIVE OFFICER 40.00 X X 242633 0 17100 DR LYNN HUDSON CHIEF SCIENCE OFFICER 40.00 X 210012 0 17691 DR JOHN MICHEL SAUER PROGRAM OFFICER 40.00 X 195991 0 31601 DR STEPHEN COONS PROGRAM OFFICER 40.00 X 187345 0 26911 DR DIANE STEPHENSON EXECUTIVE DIRECTOR 40.00 X 175666 0 25784 DR KLAUS ROMERO CHIEF SCIENCE OFFICER 40.00 X 182952 0 17154 DR INISH O'DOHERTY EXECUTIVE DIRECTOR 40.00 X 181018 0 16206 MS SONYA EREMENCO ASSOCIATE DIRECTOR 40.00 X 160608 0 24670 DR RICHARD LIWSKI CHIEF TECHNOLOGY OFFICER 40.00 X 163994 0 17984 DR ARIANA MULLIN EXECUTIVE DIRECTOR 40.00 X 154963 0 19122 DR MARCO SHITO SCIENTIFIC DIRECTOR 40.00 X 138031 0 21660 MR STEVEN BROADBENT FORMER COO & TREASURER 40.00 X 123553 0 18079 MR LEWIS BARBIERI CORPORATE SECRETARY 40.00 X 120637 0 19858 MS KRISTEN SWINGLE CHIEF OPERATING OFFICER & TREASURER 40.00 X 126647 0 12040 DR D CRAIG BRATER BOARD MEMBER 1.00 X 0 0 0 MS KAY HOLCOMBE BOARD MEMBER 1.00 X 0 0 0 MR PETER BARTON HUTT BOARD MEMBER 1.00 X 0 0 0 MR JEFFREY JACOB BOARD MEMBER 1.00 X 0 0 0 MR SHAUN A KIRKPATRICK BOARD MEMBER 1.00 X 0 0 0 MR ALAN G LEVIN BOARD MEMBER 1.00 X 0 0 0 DR ALASTAIR JJ WOOD BOARD MEMBER 1.00 X 0 0 0 MR JAMES W NEWMAN BOARD MEMBER 1.00 X 0 0 0 DR PAULA J OLSIEWSKI BOARD MEMBER 1.00 X 0 0 0 DR TOMAS SALMONSON BOARD MEMBER 1.00 X 0 0 0 MS CINDY PARSEGHIAN BOARD MEMBER 1.00 X 0 0 0 MR RICHARD T MYERS JR BOARD MEMBER 1.00 X 0 0 0 DR MICHAEL KASSER BOARD MEMBER 1.00 X 0 0 0 DR TIMOTHY R FRANSON CHAIR 1.00 X X 0 0 0 MR M WAINWRIGHT FISHBURN JR VICE-CHAIR 1.00 X X 0 0 0 2600440 0 309911 22 1 1 0 EPHIBIAN INC 3180 N SWAN RD TUCSON AZ 85712 PLATFORM SERVICES 160376 LOTOS NILE PO BOX 90245 NASHVILLE TN 37209 PUBLIC RELATIONS 159274 EDETEK INC 101 COLLEGE RD W EAST 2ND FLOOR PRINCETON NJ 08540 SUBAWARD 157500 AMY C PORTER 4201 N QUAIL CANYON DR TUCSON AZ 85750 CONSULTING 156600 RTI HEALTH SOLUTIONS 200 PARK OFFICERS DRIVE RESEARCH TRIANGLE PARK NC 27709 T&M 143685 5 7179419 5799222 12978641 RESEARCH SUPPORT FEES 541700 5253708 5253708 OTHER REVENUE 900099 39071 39071 5292779 156753 156753 EARNINGS FROM SUBSIDIARY 900099 36141 36141 36141 18464314 5328920 0 156753 2499467 2499467 198459 198459 1966157 1471956 494201 7088060 5416393 1671667 487395 371690 115705 712678 521219 191459 639060 486325 152735 21109 7465 13644 44605 44605 1102875 812580 290295 243926 243926 303687 165722 137965 573272 311452 261820 573576 427632 145944 473452 406443 67009 463937 292306 171631 30922 23054 7868 90062 90062 DUES AND SUBSCRIPTIONS 31584 16672 14912 MISCELLANEOUS 8317 61 8256 17552600 13428896 4123704 0 4891967 8304830 5019135 2281093 385302 1581850 518992 993569 102576 42677 462785 378951 76758 83834 6402760 3143650 109973 226347 17507463 16657850 900763 1209284 8404571 6353841 55040 67613 9360374 7630738 X 8147089 9027112 8147089 9027112 17507463 16657850 911714 -31691 0 X 0 1 X 1 1 1 X 10197464 10085049 10019550 10698823 12978641 53979527 10197464 10085049 10019550 10698823 12978641 53979527 21605657 32373870 80580 48471 83667 215046 156753 584517 54564044 26978211 0.59330 0.51470 X RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 0 0 0 17552600 17552600 1000000 250000 0 0 909814 875586 918648 1000000 3704048 5556072 61348 60816 122164 227454 218897 229662 250000 926013 1389020 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 161531 145992 15539 259240 190945 68295 83834 DEFERRED RENT 67613 67613 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). 1 EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM 0 0 GRANTS TO RECIPIENTS LOCATED IN REGION CLINICAL RESEARCH 198459 EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM 0 0 INVESTMENT 111924 0 0 0 0 0 0 310383 0 310383 EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM SUBAWARD 69712 WIRE TRANSFER EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM SUBAWARD 10785 WIRE TRANSFER EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM UL HYPERCOMPUTING WORK 28001 WIRE TRANSFER EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM REGULATORY FEES 19801 WIRE TRANSFER EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM CLINICAL RESEARCH 15035 WIRE TRANSFER EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM T&M WORK 43752 WIRE TRANSFER EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM CLINICAL RESEARCH 11373 WIRE TRANSFER 7 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. PART III ACCOUNTING METHOD: 1 BAYLOR RESEARCH INSTITUTE 2001 BRYAN ST SUITE 2200 DALLAS TX 75201 751921898 501(C)(3) 995538 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 502984 TUBERCULOSIS RESEARCH UNIVERSITY OF FLORIDA PO BOX 113001 GAINSVILLE FL 32611 596002052 STATE OF FLORIDA 50000 DUCHENNE'S MUSCULAR DYSTROPHY MODEL BASED SIMULATION TOOL THE TRANSLATIONAL GENOMICS RESEARCH INSTITUTE 445 N FIFTH STREET SUITE 600 PHOENIX AZ 85004 753065445 501(C)(3) 35000 ANTI MICROBIAL RESISTANCE DATA PROJECT NORTHWESTERN UNIVERSITY 625 N MICHIGAN AVENUE STE 2700 CHICAGO IL 60611 362167817 501(C)(3) 162655 CLINICAL OUTCOME RESEARCH NATIONAL ORGANIZATION FOR RARE DISORDERS (NORD) DEPT 5430 PO BOX 4110 WOBURN MA 01888 133223946 501(C)(3) 712409 RARE DISEASE EFFORTS VERASCI INC NCT HOLDINGS INC 6820 CREEK WOOD DRIVE SUITE 101 CHAPEL HILL NC 27514 202104807 20680 PRO CONSORTIUM PROJECT UNIVERSITY OF COLORADO BARBARA DAVIS CENTER 1775 AURORA CT B-140 AURORA CO 80045 841231021 STATE OF COLORADO 10200 T1D CONSORTIUM RESEARCH UNIVERSITY OF ROCHESTER 265 CRITTENDEN BLVD ROCHESTER NY 14642 160743209 STATE OF NEW YORK 10000 CPP DATA CONTRIBUTION 8 PART I, LINE 2: CRITICAL PATH INSTITUTE ENTERS INTO SUBAWARD AGREEMENTS WITH SUBRECIPIENTS AND REVIEWS PROGRESS DELIVERABLES AND COSTS. X X X X 0 0 0 0 0 0 0 1 0 DR MARTHA A BRUMFIELD FORMER PRESIDENT & CEO 236390 0 0 0 0 0 17167 0 6884 0 260441 0 0 0 DR JOSEPH SCHEEREN PRESIDENT & CHIEF EXECUTIVE OFFICER 242633 0 0 0 0 0 16038 0 1062 0 259733 0 0 0 DR LYNN HUDSON CHIEF SCIENCE OFFICER 210012 0 0 0 0 0 15919 0 1772 0 227703 0 0 0 DR JOHN MICHEL SAUER PROGRAM OFFICER 195991 0 0 0 0 0 16259 0 15342 0 227592 0 0 0 DR STEPHEN COONS PROGRAM OFFICER 187345 0 0 0 0 0 15212 0 11699 0 214256 0 0 0 DR DIANE STEPHENSON EXECUTIVE DIRECTOR 175666 0 0 0 0 0 14310 0 11474 0 201450 0 0 0 DR KLAUS ROMERO CHIEF SCIENCE OFFICER 182952 0 0 0 0 0 5533 0 11621 0 200106 0 0 0 DR INISH O'DOHERTY EXECUTIVE DIRECTOR 181018 0 0 0 0 0 14438 0 1768 0 197224 0 0 0 MS SONYA EREMENCO ASSOCIATE DIRECTOR 160608 0 0 0 0 0 13411 0 11259 0 185278 0 0 0 DR RICHARD LIWSKI CHIEF TECHNOLOGY OFFICER 163994 0 0 0 0 0 11221 0 6763 0 181978 0 0 0 DR ARIANA MULLIN EXECUTIVE DIRECTOR 154963 0 0 0 0 0 12392 0 6730 0 174085 0 0 0 DR MARCO SHITO SCIENTIFIC DIRECTOR 138031 0 0 0 0 0 10566 0 11094 0 159691 0 0 0 MR STEVEN BROADBENT FORMER COO & TREASURER 123553 0 0 0 0 0 10129 0 7950 0 141632 0 0 0 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 135850 CONSULTING SERVICES - SCIENTIFIC/TECHNICAL WRITING 0 FORM 990, PART III, LINE 4A, DESCRIPTION OF PROGRAM SERVICE: 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 (CPI), BY ESTABLISHING 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: THE CRITICAL PATH FOR ALZHEIMER'S DISEASE (CPAD) CONSORTIUM DEVELOPS TOOLS AND NOVEL METHODOLOGIES TO ACCELERATE PROGRESS IN THE DEVELOPMENT OF PREVENTION AND TREATMENT STRATEGIES FOR ALZHEIMER'S DISEASE (AD). THIS YEAR'S ACCOMPLISHMENTS INCLUDE: (1) SPEAR-HEADING AN INDUSTRY DATA-SHARING INITIATIVE TO EXPAND THE ACQUISITION OF CONTEMPORARY PATIENT-LEVEL CLINICAL TRIAL DATA THAT WILL BE INCORPORATED INTO A COMPREHENSIVE DISEASE PROGRESSION MODEL ACROSS THE AD CONTINUUM. (2) EXPANDING THE AD CLINICAL TRIAL DATA REPOSITORY TO 41 STUDIES WITH 20,549 INDIVIDUAL ANONYMIZED PATIENT RECORDS. IT HAS BEEN UTILIZED BY 456 APPROVED APPLICANTS FROM AROUND THE GLOBE, COVERING OVER 150 DISTINCT ORGANIZATIONS FROM PHARMACEUTICAL INDUSTRY, GOVERNMENT AGENCIES, NONPROFIT ORGANIZATIONS, ACADEMIA, AND INDEPENDENT RESEARCHERS, FROM MORE THAN 40 DIFFERENT COUNTRIES. (3) CONVENING A QUANTITATIVE MODELING WORKING GROUP TO SUPPORT THE GENERATION OF ACTIONABLE AD DRUG DEVELOPMENT TOOLS. THE CRITICAL PATH FOR PARKINSON'S CONSORTIUM'S (CPP) GOALS ARE TO DEVELOP QUANTITATIVE MODEL-BASED TOOLS TO ACCELERATE THE TREATMENT OF PARKINSON'S DISEASE (PD), ESPECIALLY FOR THOSE INDIVIDUALS EXPERIENCING THE FIRST CLINICAL SIGNS OF MOTOR SYMPTOM ONSET. ACCOMPLISHMENTS THIS YEAR INCLUDE:(1) LAUNCHED STAGE 2 OF THE DIGITAL DRUG DEVELOPMENT TOOLS (3DT) PROJECT WITH A GOAL TO ACQUIRE DATA FROM DIGITAL DEVICES TO BETTER UNDERSTAND HOW TO MANAGE, DISTRIBUTE, STANDARDIZE, AND APPLY THESE DATA TO OPTIMIZE PD CLINICAL TRIALS. (2) EXPANDED THE PD INTEGRATED DATABASE TO OVER 12,000 SUBJECTS FROM FIVE OBSERVATIONAL STUDIES AND 13 CLINICAL TRIALS IN PD. 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. ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) ENRICHED THE TB PLATFORM FOR AGGREGATION OF CLINICAL TRIALS (TB-PACTS), BY INCORPORATING 17 CLINICAL TRIAL DATASETS; (2) SUBMITTED DOCUMENTATION PACKAGE FOR A PRE-SUBMISSION MEETING WITH FDA CDRH TO UPDATE THE AGENCY ON THE RELATIONAL SEQUENCING FOR TB (RESEQTB) DATABASE PROGRESS, GAIN A LETTER OF SUPPORT FROM CDRH, AND DEFINE A REGULATORY PATH FORWARD; AND (3) COMPLETED THE ADAPTIVE TRIAL DESIGN PROJECT AND FINALIZED A QUANTITATIVE PLATFORM TO DETERMINE THE UTILITY OF ADAPTIVE TRIAL DESIGNS AS NOVEL CLINICAL DEVELOPMENT STRATEGIES. THE CRITICAL PATH FOR SICKLE CELL DISEASE (CP-SCD) CONSORTIUM WAS LAUNCHED IN APRIL OF 2020. IT IS A COLLABORATION SET UP TO DEVELOP COMMUNITY CONSENSUS ON HOW TO OPTIMIZE AND ACCELERATE DRUG DEVELOPMENT FOR THIS RARE DISEASE. THE MISSION OF THE CP-SCD IS TO SUPPORT COLLABORATIVE DEVELOPMENT AND REGULATORY ENDORSEMENT OF NOVEL MEDICAL PRODUCT DEVELOPMENT TOOLS. THESE TOOLS WILL HELP TO OPTIMIZE AND DE-RISK CLINICAL TRIALS TO INCREASE THE EFFICIENCY IN DEVELOPING AND DELIVERING SAFE, EFFECTIVE TREATMENTS FOR PEOPLE LIVING WITH SICKLE CELL DISEASE. THE CURE DRUG REPURPOSING COLLABORATORY (CDRC) IS A PUBLIC-PRIVATE PARTNERSHIP INITIATED IN JUNE OF 2020 BY C-PATH AND THE U.S. FOOD AND DRUG ADMINISTRATION (FDA) IN PARTNERSHIP WITH THE NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES (NCATS), PART OF THE NATIONAL INSTITUTES OF HEALTH (NIH). CDRC, IN PARTNERSHIP WITH THE FDA-NCATS CURE ID* PLATFORM, IS A DEDICATED INITIATIVE DESIGNED TO CAPTURE REAL-WORLD CLINICAL OUTCOME DATA TO ADVANCE DRUG REPURPOSING AND INFORM FUTURE CLINICAL TRIALS FOR DISEASES OF HIGH UNMET MEDICAL NEED. THE DATA COLLABORATION CENTER (DCC) IS A PROGRAM 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. ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) LAUNCHED THE RARE DISEASE CURES ACCELERATOR - DATA AND ANALYTICS PLATFORM (RDCA-DAP) AND DEVELOPMENT OF PROTOTYPE DATA INTERROGATOR FOR THE RDCA-DAP; (2) COMPLETED THE ALZHEIMER'S DISEASE DATA INTEROPERABILITY PILOT PROJECT TO VALIDATE THE AD WORKBENCH PROOF OF CONCEPT AND JUSTIFY PROCEEDING TO THE SCALE-UP PHASE; (3) RECEIVED ADDITIONAL FUNDING FROM NIH TO PROCESS ISOLATES FOR THE RESEQTB INITIATIVE; AND (4) EXPANDED TB-PACTS 2.0 TO INCLUDE ADDITIONAL TB 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 (DMD), TO ENABLE THE EARLIEST POSSIBLE PATIENT ACCESS TO NEW TREATMENTS. ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) EXPANDED THE D-RSC DATABASE TO 16 DATASETS, REPRESENTING NEARLY 5000 PEOPLE WITH DMD; AND (2) SUBMITTED A CLINICAL TRIAL SIMULATION TOOL LETTER OF INTENT AND MODELING PLAN TO FDA AND EMA AND INITIATED WORK ON CLINICAL TRIAL SIMULATION PLATFORM. 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. ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) LAUNCHED THE ECOA: GETTING BETTER TOGETHER INITIATIVE TO ADDRESS CHALLENGES WITH THE IMPLEMENTATION OF ELECTRONIC COAS (ECOAS) IN CLINICAL TRIALS; (2) PARTICIPATED IN THE PREPARATION OF AN FDA DDT RESEARCH GRANT APPLICATION TITLED PRELIMINARY RESEARCH TO SUPPORT QUALIFICATION OF AN ACTIVITY MONITOR-BASED ENDPOINT MEASURE TO EVALUATE PHYSICAL ACTIVITY IN PERSONS WITH CHRONIC HEART FAILURE FOR DDTCOA000114; (3) LAUNCHED THE EPRO CONSORTIUM'S CHINA SUBCOMMITTEE; AND (4) IN COLLABORATION WITH THE PROC, DEVELOPED THE PRESENTATION TITLED "CORONAVIRUS DISEASE 2019 (COVID-19): RISK ASSESSMENT AND MITIGATION STRATEGIES FOR THE COLLECTION OF PATIENT-REPORTED OUTCOME (PRO) DATA THROUGH CLINICAL SITES." 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) CONTRIBUTED COMMENTS TO FDA DRAFT GUIDANCE ON THE USE OF NATURAL HISTORY STUDIES IN DRUG DEVELOPMENT FOR RARE DISEASES; (2) EXPANDED CONSORTIUM MEMBERSHIP THROUGH AGREEMENTS WITH NEW INDUSTRY AND NON-PROFIT MEMBERS; (3) SUBMITTED MANUSCRIPT ON STANDARDIZED DATA STRUCTURES IN RARE DISEASES TO CLINICAL AND TRANSLATIONAL SCIENCE; (4) FINALIZED TERMS AND CONDITIONS FOR USERS TO GAIN ACCESS TO THE HD AGGREGATED DATABASE; AND (5) EXPANDED HD DATABASE, CURRENTLY REPRESENTING 16,500 PATIENTS. 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. ACCOMPLISHMENTS THIS YEAR INCLUDE DEVELOPING RECOMMENDED NEONATAL BLOOD PRESSURE MEASUREMENT METHODS BASED ON A SYSTEMATIC LITERATURE REVIEW AND ANALYSIS, INCLUDING ORIGINAL ARTWORK WITH A DIAGRAM ILLUSTRATING PROPER BLOOD PRESSURE CUFF PLACEMENT. FORM 990, PART III, LINE 4A, DESCRIPTION OF PROGRAM SERVICE: 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) RECEIVED FINAL POSITIVE QUALIFICATION OPINION ADOPTED BY THE CHMP/EMA; AND (2) RECEIVED POSITIVE DETERMINATION LETTER FROM FDA COA QUALIFICATION PROGRAM ACCEPTING THE QUALIFICATION PLAN FOR USE OF SYMBOL DIGIT MODALITIES TEST TO ASSESS COGNITION IN TRIALS OF MS THERAPIES. THE POLYCYSTIC KIDNEY DISEASE OUTCOMES CONSORTIUM (PKD-OC) 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 SOLUTIONS TO ACCELERATE THE DEVELOPMENT OF NOVEL MEDICAL PRODUCTS FOR INDIVIDUALS LIVING WITH PKD, WHICH INCLUDE QUANTITATIVE DISEASE PROGRESSION MODELS, 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. A NEW TKV MODELING PROPOSAL HAS BEEN INITIATED THIS YEAR; THIS EFFORT WILL RESULT IN THE DEVELOPMENT OF TOOLS TO OPTIMIZE CLINICAL TRIAL DESIGN FOR PKD THERAPIES. 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. ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) RECEIVED ADVICE LETTER FROM FDA IN RESPONSE TO THE INITIAL BRIEFING PACKAGE SUBMITTED BY THE MULTIPLE SCLEROSIS WORKING GROUP FOR QUALIFICATION OF THE PROMIS SHORT FORM V1.0-FATIGUE-MULTIPLE SCLEROSIS 8A (PROMIS FATIGUEMS-8A); (2) THE SMALL CELL LUNG CANCER WORKING GROUP SUBMITTED A LETTER OF INTENT (LOI) TO FDA FOR A NEW MEASURE DERIVED FROM THE NSCLC-SAQ V1.0 AND RECEIVED A REVIEWABILITY MEMORANDUM FROM FDA; (3) RECEIVED THE OK FROM FDA TO MOVE FORWARD WITH DEVELOPMENT OF A QUALIFICATION PLAN FOR THE SYMPTOMS OF MAJOR DEPRESSIVE DISORDER DIARY (SMDDD); AND (4) IN COLLABORATION WITH THE EPROC, DEVELOPED THE PRESENTATION TITLED "CORONAVIRUS DISEASE 2019 (COVID-19): RISK ASSESSMENT AND MITIGATION STRATEGIES FOR THE COLLECTION OF PATIENT-REPORTED OUTCOME (PRO) DATA THROUGH CLINICAL SITES." THE PREDICTIVE SAFETY TESTING CONSORTIUM (PSTC) 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. THIS YEAR THE CONSORTIUM'S ACCOMPLISHMENTS INCLUDE: (1) INITIATED THE PREDICTOX PROJECT, FOCUSED ON THE DEVELOPMENT OF QUANTITATIVE TOOLS TO BETTER PREDICT CLINICAL SAFETY FOR PRECLINICAL DATA, IN COOPERATION WITH THE ICAHN SCHOOL OF MEDICINE AT MT. SINAI AND FDA; (2) A LETTER OF INTENT FOR QUALIFICATION OF DRUG-INDUCED MUSCLE INJURY BIOMARKERS ADVANCED TO THE STAGE TWO REVIEW AT FDA; AND (3) THE QUALIFICATION PLAN FOR GLUTAMATE DEHYDROGENASE (GLDH) AS A BIOMARKER TO DETECT DRUG-INDUCED LIVER INJURY ADVANCED TO THE STAGE TWO REVIEW AT FDA. THE WORK OF THE QUANTITATIVE MEDICINE PROGRAM (QUANTMED) FOCUSES ON THE DEVELOPMENT AND INNOVATION OF QUANTITATIVE SOLUTIONS FOR MEDICAL PRODUCT DEVELOPMENT AND CLINICAL NEEDS, WITH THE AIM OF IMPROVING POPULATION AND INDIVIDUAL HEALTH BY TRANSFORMING DRUG DEVELOPMENT THROUGH METHODOLOGICAL INNOVATION. THE PROGRAM GOAL IS TO LEVERAGE KNOWLEDGE FROM A NETWORK OF EXPERTS IN INDUSTRY, ACADEMIA, NON-PROFIT AND REGULATORY SCIENCES COMBINED WITH DATA ACQUIRED FROM MULTIPLE SOURCES TO DEVELOP QUANTITATIVE METHODOLOGIES IN PHARMACOMETRICS, STATISTICS, SYSTEMS PHARMACOLOGY, ARTIFICIAL INTELLIGENCE AND DIGITAL DATA ANALYTICS. ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) RECEIVED FUNDING FROM FDA TO DEVELOP TRAINING MODULES FOR MODEL-INFORMED DRUG DEVELOPMENT (MIDD) FOR REGULATORS; (2) PROVIDED CRITICAL SUPPORT TO D-RSC THE SAWP MEETING WITH EMA FOR THE DUCHENNE CLINICAL TRIAL SIMULATION TOOL; AND (3) REPRESENTED RARE DISEASE CURES ACCELERATOR-DATA AND ANALYTICS PLATFORM (RDCA-DAP) AT FDA RARE DISEASE DAY, LEADING A PANEL ON RARE DISEASE PROGRESSION MODELING; (4) ADVANCED TO CRITICAL DEMONSTRATION PROJECTS WITHIN RDCA-DAP, FOR THE GENERATION OF QUANTITATIVE SOLUTIONS TO ACCELERATE DRUG DEVELOPMENT IN ORPHAN INDICATIONS; (5) SUPPORTED CPP'S SUCCESSFUL 3DT PROJECT; (6) SUPPORTED THE T1D CONSORTIUM IN THE SUCCESSFUL LAUNCH OF THE TRIAL OUTCOME MARKERS INITIATIVE (TOMI); AND (7) SUPPORTED THE TTC IN THE SUBMISSION OF THE LETTER OF INTENT TO FDA BIOMARKER QUALIFICATION PROGRAM TO SEEK FORMAL QUALIFICATION OF A REASONABLY LIKELY SURROGATE ENDPOINT (RLSE) FOR USE IN TRANSPLANTATION CLINICAL TRIALS. 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) INITIATIVE LAUNCHED; (2) INTEGRATED DATA FROM ~28,000 PATIENTS INTO THE RDCA-DAP; AND (3) EXPLORED PARTNERSHIPS TO FACILITATE SHARING OF DATA AND TECHNOLOGY WITH THE COMMUNITY, OUTREACH AND COMMUNICATIONS TO 100+ GROUPS INCLUDING INDUSTRY, NIH AND PATIENT GROUPS. 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. THIS YEAR'S ACCOMPLISHMENTS INCLUDE: (1) INITIATED TRIAL OUTCOME MARKERS INITIATIVE (TOMI) AND CONVENED STAKEHOLDERS IN 2020 IN RESPONSE TO T1D COMMUNITY DRUG DEVELOPMENT NEEDS; (2) RECEIVED FDA FUNDING TO BEGIN A COMPREHENSIVE ASSESSMENT TO UNDERSTAND THE RELATIONSHIPS BETWEEN CONTINUOUS GLUCOSE MONITORING MEASURES AND ESTABLISHED MEASURES OF GLYCEMIC CONTROL; AND (3) INITIATED AN ISLET AA ASSAY MODERNIZATION PROJECT. 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. THIS YEAR'S ACCOMPLISHMENTS INCLUDE: (1) SUBMITTED LETTER OF INTENT TO FDA BIOMARKER QUALIFICATION PROGRAM TO SEEK FORMAL QUALIFICATION OF A REASONABLY LIKELY SURROGATE ENDPOINT (RLSE) FOR USE IN TRANSPLANTATION CLINICAL TRIALS; AND (2) INITIATED THE CREATION OF AN AGGREGATED PATIENT-LEVEL DATABASE USING REAL-WORLD EVIDENCE AND HISTORICAL CLINICAL TRIAL DATA. 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. 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. 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. 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 36141 680634 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 111924 CASH