2017-02-09T15:26:28-06:00 2016-06-30 860683059 BEACHFLEISCHMAN PC 1985 EAST RIVER ROAD SUITE 201 TUCSON AZ 85718 990 2015-07-01 201991334 CRITICAL PATH INSTITUTE CRIT 5205473440 1730 EAST RIVER ROAD NO 200 TUCSON AZ 857185898 STEVEN BROADBENT MBA COO 5205473440 2017-02-08 1 KAREN K MCCLOSKEY CPA P00099644 5203214600 2015 2021-01-29 14:40:06Z MARTHA BRUMFIELD PHD 1730 EAST RIVER ROAD NO 200 TUCSON AZ 857185898 15347078 0 X WWW.C-PATH.ORG X 2004 AZ CREATE AND MANAGE INTERNATIONAL COLLABORATIONS TO RESOLVE PROBLEMS AND DEVELOP NOVEL TOOLS, ENABLING THE FASTER DEVELOPMENT OF SAFER MEDICAL PRODUCTS. 14 13 64 14 0 0 10269810 10197464 3715780 5069034 13987 15089 69216 51912 14068793 15333499 3684743 2657978 0 0 4934299 6010064 0 0 0 4558896 5997897 13177938 14665939 890855 667560 15716704 18234620 10932273 12782629 4784431 5451991 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 12217622 2657978 5069034 SEE SCHEDULE O 12217622 1 1 0 1 0 0 0 0 0 0 1 0 0 0 0 1 1 0 0 0 1 1 0 0 0 0 0 1 0 1 0 0 0 0 0 0 0 0 0 0 0 0 0 1 1 0 0 0 1 37 0 1 64 1 0 0 0 0 0 0 0 0 0 0 X 14 13 1 0 0 0 0 0 0 1 1 0 0 1 1 1 1 1 1 1 1 0 AZ X STEVE BROADBENT 5206478382 1730 EAST RIVER ROAD SUITE 200 TUCSON AZ 857185898 LYLE J BOOTMAN PHD SCD BOARD MEMBER 1.00 X 0 0 0 D CRAIG BRATER MD BOARD MEMBER 1.00 X 0 0 0 THE HONORABLE JAMES C GREENWOOD 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 RICHARD T MYERS JR 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 PETER BARTON HUTT LLB LLM BOARD MEMBER 1.00 X 0 0 0 ALAN G LEVIN MS CPA BOARD MEMBER 1.00 X 0 0 0 PAULA OLSIEWSKI PHD BOARD MEMBER 1.00 X 0 0 0 JEFFREY E JACOB SM DEPUTY CHAIR 1.00 X 0 0 0 M WAINWRIGHT FISHBURN JR VICE CHAIRMAN 1.00 X X 0 0 0 TIMOTHY R FRANSON MD CHAIRMAN OF THE BOARD FROM 6/16 1.00 X X 0 0 0 PETER B CORR MD PHD CHAIRMAN OF THE BOARD TIL 6/16 1.00 X X 0 0 0 MARTHA A BRUMFIELD PHD CHIEF EXECUTIVE OFFICER 40.00 X X 296522 0 30054 STEVEN T BROADBENT MBA CHIEF OPERATING OFFICER & TREASURER 40.00 X 187269 0 26290 ENRIQUE AVILES SECRETARY 40.00 X 157130 0 22255 LYNN D HUDSON PHD CHIEF SCIENCE OFFICER 40.00 X 191637 0 18114 JOHN-MICHAEL SAUER PHD EXECUTIVE DIRECTOR PSTC 40.00 X 179842 0 26538 STEPHEN J COONS PHD EXECUTIVE DIRECTOR PROC 40.00 X 175956 0 20673 DIANE STEPHENSON PHD EXECUTIVE DIRECTOR CPP 40.00 X 162784 0 23508 DEBRA HANNA PHD EXECUTIVE DIRECTOR CPTR 40.00 X 159054 0 22591 1510194 0 190023 12 0 1 0 ADELPHI VALUES ADELPHI MILL BOLLINGTON CHESIRE UK SK105JB RESEARCH 689270 PHARSIGHT CORPORATION PO BOX 200854 PITTSBURG PA 15251 SOFTWARE AND STATISTICAL MODEL 506072 HEALTH RESEARCH ASSOCIATES 6505 216TH STREET SW SUITE 105 MOUNTLAKE TERRACE WA 98043 RESEARCH 340156 EPHIBIAN 3180 N SWAN ROAD TUCSON AZ 85712 SOFTWARE DEV, WEB AND INFO SECURITY 240494 ANN ROBBINS LLC 6430 SUN EALE LANE UNIT 301 BRADENTON FL 34210 REGULATORY EXPERT CONSULTANT 154450 10 3200284 6997180 10197464 RESEARCH SUPPORT FEES 541700 4912824 4912824 OTHER REVENUE 900099 156210 156210 5069034 28668 28668 51912 0 51912 51912 51912 13579 -13579 -13579 -13579 15333499 5069034 0 67001 X 2600182 2600182 57796 57796 707214 294552 412662 4309832 3084036 1225796 310142 224418 85724 345738 241442 104296 337138 229692 107446 62758 60040 2718 139477 102514 36963 10000 10000 3407844 3357310 50534 220154 164317 55837 224574 193398 31176 290530 247315 43215 448272 257102 191170 458967 429854 29113 457039 436880 20159 68637 47013 21624 45365 30907 14458 BAD DEBT EXPENSE 132500 132500 DUES AND SUBSCRIPTIONS 21844 18511 3333 MISCELLANEOUS 9936 7843 2093 14665939 12217622 2448317 0 2758599 4084148 7214664 8406753 403661 327557 438101 1023373 37395 42542 873242 786731 121311 86511 4727502 4248265 15471 15471 15716704 18234620 1167646 632632 9764627 12149997 10932273 12782629 X 4784431 5451991 4784431 5451991 15716704 18234620 667560 0 X 0 1 X 1 1 1 X 7998908 5915630 5370782 10269811 10197464 39752595 7998908 5915630 5370782 10269811 10197464 39752595 12673465 27079130 22660 21279 70230 82059 80580 276808 20745 20745 40050148 17330876 0.67610 0.79310 X RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 11731 11731 14654208 14665939 883297 220824 0 0 617482 551743 809246 883297 2861768 4292652 60071 60221 67262 11731 199285 154371 137936 202312 220824 715443 1073165 PART II-A, LINE 1B C-PATH EXPENDS A MINOR AMOUNT OF TIME AND RESOURCES TO EDUCATE MEMBERS OF CONGRESS IN THE AREA OF DRUG DEVELOPMENT. 164184 140425 23759 237326 236975 351 471732 409331 62401 86511 X 15333499 0 15333499 0 15333499 14665939 0 14665939 0 14665939 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. ACCORDINGLY, NO PROVISION FOR FEDERAL AND STATE INCOME TAXES IS MADE IN THE FINANCIAL STATEMENTS. 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). GAAP REQUIRES MANAGEMENT TO PERFORM AN EVALUATION OF ALL TAX POSITIONS TAKEN OR EXPECTED TO BE TAKEN IN THE COURSE OF PREPARING C-PATH'S TAX RETURNS TO DETERMINE WHETHER THE TAX POSITIONS MEET A "MORE LIKELY THAN NOT" STANDARD OF BEING SUSTAINED UNDER EXAMINATION BY THE APPLICABLE TAXING AUTHORITIES. THIS EVALUATION IS REQUIRED TO BE PERFORMED FOR ALL OPEN TAX YEARS, AS DEFINED BY THE VARIOUS STATUTES OF LIMITATIONS, FOR FEDERAL AND STATE PURPOSES. C-PATH IS ONLY SUBJECT TO INCOME TAXES ON UBTI AS APPLICABLE. AS A RESULT, C-PATH IS REQUIRED TO FILE INFORMATIONAL RETURNS FOR FEDERAL AND STATE PURPOSES AND, IF IT HAS UBTI, FEDERAL AND STATE INCOME TAX RETURNS. MANAGEMENT HAS PERFORMED ITS EVALUATION OF TAX POSITIONS TAKEN ON ALL OPEN TAX RETURNS AND HAS DETERMINED THAT THERE WERE NO POSITIONS TAKEN THAT DO NOT MEET THE "MORE LIKELY THAN NOT" STANDARD. 1 EUROPE (INCLUDING ICELAND & GREENLAND) 0 0 GRANTS TO RECIPIENTS LOCATED IN REGION CLINICAL RESEARCH 57796 0 0 0 0 0 0 57796 0 57796 EUROPE (INCLUDING ICELAND & GREENLAND) CLINICAL RESEARCH 57796 WIRE TRANSFER 1 0 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 1950 N STEMMONS FREEWAY SUITE 5010 LOCKBOX 846275 DALLAS TX 75207 751921898 501(C)(3) 1287756 DEVELOP A HOLLOW FIBER MODEL FOR TUBERCULOSIS RESEARCH UNIVERSITY OF CALIFORNIA 1855 FOLSOM STREET BOX 0897 SAN FRANCISCO CA 94143 946036493 STATE OF CALIFORNIA 642125 DEVELOP A DISEASE PROGRESSION MODEL FOR TUBERCULOSIS UNIVERSITY OF FLORIDA PO BOX 113001 GAINSVILLE FL 32611 596002052 STATE OF FLORIDA 162040 TO DEVELOP A POPULATION PHARMACOKINETIC/PHARMACODYNAMIC (PK/PD) MODEL FOR TUBERCULOSIS. CLINICAL DATA INTERCHANGE STANDARDS CONSORTIUM PO BOX 2068 ROUND ROCK TX 78680 043503931 501(C)(3) 508261 DEVELOP CLINICAL DATA STANDARDS 4 PART I, LINE 2: CRITICAL PATH INSTITUTE ENTERS INTO SUBAWARD AGREEMENTS WITH SUBRECIPIENTS AND REVIEWS PROGRESS DELIVERABLES AND COSTS. X X X X X 0 0 0 0 0 0 0 0 0 MARTHA A BRUMFIELD PHD CHIEF EXECUTIVE OFFICER 296522 0 0 0 0 0 23575 0 6479 0 326576 0 0 0 STEVEN T BROADBENT MBA CHIEF OPERATING OFFICER & TREASURER 187269 0 0 0 0 0 15904 0 10386 0 213559 0 0 0 ENRIQUE AVILES SECRETARY 157130 0 0 0 0 0 11871 0 10384 0 179385 0 0 0 LYNN D HUDSON PHD CHIEF SCIENCE OFFICER 191637 0 0 0 0 0 15904 0 2210 0 209751 0 0 0 JOHN-MICHAEL SAUER PHD EXECUTIVE DIRECTOR PSTC 179842 0 0 0 0 0 14565 0 11973 0 206380 0 0 0 STEPHEN J COONS PHD EXECUTIVE DIRECTOR PROC 175956 0 0 0 0 0 14484 0 6189 0 196629 0 0 0 DIANE STEPHENSON PHD EXECUTIVE DIRECTOR CPP 162784 0 0 0 0 0 13130 0 10378 0 186292 0 0 0 DEBRA HANNA PHD EXECUTIVE DIRECTOR CPTR 159054 0 0 0 0 0 13169 0 9422 0 181645 0 0 0 FORM 990, PART III, LINE 4A, PROGRAM SERVICE ACCOMPLISHMENTS THE CRITICAL PATH INSTITUTE (C-PATH) IS AN INDEPENDENT, NON-PROFIT ORGANIZATION UNIQUELY DEDICATED TO IMPLEMENTING THE FDA'S CRITICAL PATH INITIATIVE BY CREATING COLLABORATIONS AMONG REGULATORS (SCIENTISTS FROM THE FDA AND EMA) THE REGULATED (MEDICAL PRODUCT INDUSTRY), ACADEMIA, PATIENTS ORGANIZATIONS, 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 COALITION AGAINST MAJOR DISEASES DEVELOPS TOOLS AND PROCESSES TO ACCELERATE PROGRESS IN THE PREVENTION, TREATMENT AND CURE OF MAJOR NEURODEGENERATIVE DISEASES, INITIALLY FOCUSING ON ALZHEIMER'S AND PARKINSON'S DISEASES. THIS YEAR'S ACCOMPLISHMENTS INCLUDE: DEVELOPED AN INFORMED CONSENT FORM THAT WILL ENABLE BROADER SHARING OF PATIENT DATA AND SPECIMENS FOR FUTURE RESEARCH. OBTAINED REGULATORY ENDORSEMENT OF THE STATISTICAL ANALYSIS APPROACH FOR THE HIPPOCAMPAL VOLUME BIOMARKER QUALIFICATION EFFORT. RECEIVED THREE LETTERS OF SUPPORT FROM THE FDA ENCOURAGING THE FURTHER STUDY AND USE OF CEREBROSPINAL FLUID ANALYTES AB1-42, TOTAL-TAU, AND PHOSPHO-TAU, AND LOW BASELINE HIPPOCAMPAL VOLUME MEASURED BY MAGNETIC RESONANCE IMAGING AS EXPLORATORY PROGNOSTIC BIOMARKERS FOR ENRICHMENT IN TRIALS FOR AD; AND MOLECULAR NEUROIMAGING OF THE DOPAMINE TRANSPORTER AS AN EXPLORATORY PROGNOSTIC BIOMARKER FOR ENRICHMENT IN TRIALS FOR PARKINSON'S DISEASE. THE COALITION FOR ACCELERATING STANDARDS AND THERAPIES IS AN INITIATIVE TO ACCELERATE CLINICAL RESEARCH AND MEDICAL PRODUCT DEVELOPMENT BY CREATING AND MAINTAINING DATA STANDARDS, TOOLS, AND METHODS FOR CONDUCTING RESEARCH IN THERAPEUTIC AREAS THAT ARE IMPORTANT TO PUBLIC HEALTH. CFAST WAS INITIATED AS A PARTNERSHIP BETWEEN THE CLINICAL DATA INTERCHANGE STANDARDS CONSORTIUM (CDISC) AND THE CRITICAL PATH INSTITUTE (C-PATH). THE COALITION HAS DEVELOPED AND PUBLISHED THE FOLLOWING (22) CDISC THERAPEUTIC AREA STANDARDS: ALZHEIMER'S V1 AND 2, ASTHMA, BREAST CANCER, CARDIOVASCULAR, CHRONIC HEPATITIS C, DIABETES, DYSLIPIDEMIA, INFLUENZA, MULTIPLE SCLEROSIS, PAIN, PARKINSON'S DISEASE, POLYCYSTIC KIDNEY DISEASE, QT STUDIES, SCHIZOPHRENIA, TUBERCULOSIS V1 AND V2, VIROLOGY V1 AND V2, TRAUMATIC BRAIN INJURY, AND CHRONIC OBSTRUCTIVE PULMONARY DISEASE (COPD). A SEPARATE ANALYSIS DATA MODEL (ADAM) SUPPLEMENT WAS PUBLISHED FOR DIABETES. A NEW CONSORTIUM ENTITLED CRITICAL PATH FOR PARKINSON'S WAS LAUNCHED. ITS GOALS ARE TO DEVELOP QUANTITATIVE MODEL-BASED TOOLS TO ACCELERATE THE TREATMENT OF PARKINSON'S DISEASE, TARGETING PATIENTS AT THE FIRST CLINICAL SIGNS OF MOTOR SYMPTOM ONSET. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: RECEIVED SUPPORT FROM EMA TO PROCEED WITH QUALIFICATION PLAN FOR MOLECULAR NEUROIMAGING OF THE DOPAMINE TRANSPORTER AS A PROGNOSTIC BIOMARKER FOR ENRICHMENT IN TRIALS FOR EARLY PARKINSON'S DISEASE. RECEIVED A LETTER OF SUPPORT FROM THE EMA FOR THE USE OF DAT IMAGING AS AN ENRICHMENT BIOMARKER FOR PD CLINICAL TRIALS. THE MISSION OF THE CRITICAL PATH TO TB DRUG REGIMENS INITIATIVE IS TO ACCELERATE THE DEVELOPMENT OF NOVEL TB DRUG REGIMENS THAT ARE SAFER, SHORTER IN DURATION, AND MORE EFFICACIOUS THAN THE CURRENT STANDARD OF CARE. THEIR MISSION EXPANDED TO ALSO 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: THE EMA ISSUED A POSITIVE QUALIFICATION DECISION ON CPTR'S QUALIFIED IN VITRO HOLLOW FIBER SYSTEM MODEL FOR TUBERCULOSIS. THE WHO SELECTED C-PATH TO HOST TB CLINICAL TRIALS DATA PLATFORM. THE RESEQTB DATA PLATFORM LAUNCHED FOR USE BY RESEARCHERS, DATA CONTRIBUTORS AND CONSORTIA MEMBERS. TB-PLATFORM FOR AGGREGATION OF CLINICAL TB STUDIES LAUNCHED. DELIVERED A QUANTITATIVE MODEL FOR TIME-TO-POSITIVITY BEHAVIOR USING AN INTEGRATED DATABASE OF PHASE II STUDIES. MANUFACTURED NEW SENSITITRE PLATE CONTAINING CURRENT, NEW, AND REPURPOSED ANTI-TB DRUGS AND SHIPPED TO MULTIPLE SITES FOR ASSAY VALIDATION. COMPLETED 2 OF 7 MODELING AND SIMULATION TOOLS (PHARMACOLOGY BASED PHARMACOKINETICS AND QT INTERVAL MODELS). SUBMITTED COMMENTS TO FDA DRAFT GUIDANCE FOR INDUSTRY ON INFECTIOUS DISEASE NEXT GENERATION SEQUENCING BASED DIAGNOSTIC DEVICES: MICROBIAL IDENTIFICATION AND DETECTION OF ANTIMICROBIAL RESISTANCE AND VIRULENCE MARKERS. THE DATA COLLABORATION CENTER IS A NEWLY ESTABLISHED 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. ITS ACCOMPLISHMENTS THIS YEAR INCLUDE: PROVIDED DATA MANAGEMENT, CURATION, STANDARDIZATION, AND DATA SHARING SUPPORT FOR THE FOLLOWING C-PATH DATA PROJECTS: RELATIONAL SEQUENCING TB DATA PLATFORM, A DATA SHARING PLATFORM TO ADVANCE THE DEVELOPMENT OF TB RAPID DRUG SUSCEPTIBILITY TESTS; TB PLATFORM FOR AGGREGATION OF CLINICAL TRIAL STUDIES, A DATABASE TO AGGREGATE PHASE III TB CLINICAL TRIAL DATA SETS AND MAKE AVAILABLE TO RESEARCHERS FOR THE WORLD HEALTH ORGANIZATION; TB-ALLIANCE, AN AGGREGATED COLLECTION OF TB CLINICAL TRIAL DATA SETS THAT THE TB-ALLIANCE ORGANIZATION IS MAKING AVAILABLE TO QUALIFIED RESEARCHERS; SIX OF C-PATH'S CONSORTIA WITH DATABASE REQUIREMENTS. THE DCC ALSO COMPLETED ITS BUSINESS PLAN. A NEW CONSORTIUM ENTITLED DUCHENNE REGULATORY SCIENCE CONSORTIUM WAS LAUNCHED. ITS 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. SPECIFIC GOALS INCLUDE DEVELOPING A CLINICAL DATA SHARING PLATFORM, A CLINICAL DATA INTERCHANGE STANDARDS CONSORTIUM (CDISC) THERAPEUTIC AREA STANDARD, AND A DISEASE PROGRESSION MODEL FOR DUCHENNE. ACCOMPLISHMENTS THIS YEAR INCLUDE: HELD FIRST EXPERT MEETING IN NOVEMBER 2015 TO DISCUSS THE ELEMENTS NEEDED TO BE INCLUDED IN THE MODEL AND TO PRIORITIZE DATASETS. DEVELOPED AN INITIAL WORK PLAN FOR THE PROGRESSION MODEL, FOR FURTHER DISCUSSION WITH THE REGULATORY AUTHORITIES AND TO FOCUS DATA COLLECTION. ACQUIRED FOUR DATASETS AND DATA SHARING AGREEMENTS FOR ADDITIONAL DATASETS ARE UNDERWAY. THE GOAL OF THE ELECTRONIC PATIENT REPORTED OUTCOME CONSORTIUM 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: PARTICIPATED IN GCP INSPECTORS WORKING GROUP AND INTERESTED PARTIES JOINT MEETING AT THE EMA AND PRESENTED THE CONSORTIUM'S POSITION ON SOURCE DATA AND BYOD. COLLABORATED IN THE DEVELOPMENT OF A STUDY DESIGN TO SUPPORT A PROJECT TITLED "COMPARABILITY OF PROVISIONED DEVICE VS BRING-YOUR-OWN-DEVICE IN SUBJECTS WITH COPD." SEVEN NEW PRO INSTRUMENTS HAVE BEEN IMPLEMENTED ELECTRONICALLY. LAUNCHED A STUDY TO PROVIDE EMPIRICAL EVIDENCE TO SUPPORT THE MEASUREMENT EQUIVALENCE OF DATA COLLECTED ON VARIOUS DATA COLLECTION MODES (I.E., PAPER, HANDHELD, TABLET, INTERACTIVE VOICE RESPONSE [IVR], AND WEB). THE QUALITATIVE COMPONENT OF THE STUDY HAS BEEN COMPLETED AND THE QUANTITATIVE PORTION IS UNDERWAY. THE MULTIPLE SCLEROSIS OUTCOME ASSESSMENT CONSORTIUM HAS A GOAL TO OBTAIN REGULATORY QUALIFICATION OF AN IMPROVED CLINICAL OUTCOME ASSESSMENT INSTRUMENT AS A PRIMARY ENDPOINT IN CLINICAL TRIALS OF MULTIPLE SCLEROSIS (MS) THERAPIES. THEIR ACCOMPLISHMENTS INCLUDE: DEVELOPED A CLINICAL DATA INTERCHANGE STANDARDS CONSORTIUM (CDISC) THERAPEUTIC AREA USER GUIDE FOR MS. ACQUIRED THE CONTROL AND TREATMENT ARMS OF 16 CLINICAL TRIAL DATASETS (14,430 PATIENT RECORDS), CURATED THE DATA, AND REMAPPED AND POOLED MOST OF THE DATA TO THE CDISC STUDY DATA TABULATION MODEL (SDTM) STANDARD. RECEIVED FEEDBACK ON THE INITIAL BRIEFING PACKAGE FROM THE U.S. FOOD AND DRUG ADMINISTRATION (FDA) AND EUROPEAN MEDICINES AGENCY (EMA). COMPLETED AN EXTENSIVE LITERATURE REVIEW. THE STATISTICS ANALYSIS PLAN WAS FINALIZED AND EXECUTED FOR THE FDA SUBMISSION. ESTABLISHED THE MSOAC PLACEBO DATABASE FOR USE IN APPROVED MS RESEARCH. WORK IS PROCEEDING ON SINGLE DOMAIN OF DISABILITY FOR THE FDA (I.E., COGNITION), WHILE MAINTAINING THE ORIGINAL FOCUS FOR THE EMA. FORM 990, PART III, LINE 4A THE GOAL OF THE INTERNATIONAL NEONATAL CONSORTIUM IS TO ACCELERATE THE DEVELOPMENT OF SAFE AND EFFECTIVE THERAPIES FOR NEONATES. IT 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. THROUGH TEAMS THAT SHARE DATA, KNOWLEDGE, AND EXPERTISE, THE CONSORTIUM WILL DEVELOP TOOLS THAT CAN BE INCORPORATED INTO CLINICAL TRIALS FOR NEONATES. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: HELD A FACE-TO-FACE WORKSHOP IN WASHINGTON, DC, ON TO DRAFT A WHITE PAPER REGARDING CONSENSUS-BASED CRITICAL ELEMENTS OF CLINICAL PHARMACOLOGY STUDIES IN NEONATES WHICH COULD BE CONSIDERED BY REGULATORS IN PREPARATION OF FUTURE GUIDANCE DOCUMENTS, INCLUDING RECOMMENDATIONS FOR SAFER FORMULATIONS ENCOMPASSING EASE OF ADMINISTRATION. SUBMITTED COMMENTS ON THE NOTICE OF PROPOSED RULEMAKING (NPRM) "FEDERAL POLICY FOR THE PROTECTION OF HUMAN SUBJECTS" TO THE OFFICE FOR HUMAN RESEARCH PROTECTIONS AND SUPPLIED INPUT ON DRUGS AND THERAPEUTIC AREAS THAT REQUIRE FURTHER STUDY IN NEONATES IN RESPONSE TO NICHD'S REQUEST FOR INFORMATION ON THE BEST PHARMACEUTICALS FOR CHILDREN ACT PRIORITIES IN PEDIATRIC THERAPEUTICS 2017. THE GOAL OF THE POLYCYSTIC KIDNEY DISEASE OUTCOMES CONSORTIUM IS TO DEVELOP AND OBTAIN 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). AFTER SUCCESSFULLY ACCOMPLISHING THE PRIMARY GOAL, THE CONSORTIUM IS NOW WORKING ON OTHER REGULATORY CHALLENGES, CLINICAL TRIAL DESIGNS, EXPLORATORY ENDPOINTS, AND THE POSSIBILITY OF QUALIFYING TKV AS A SURROGATE ENDPOINT. THEIR ACCOMPLISHMENTS FOR THIS YEAR INCLUDE: RECEIVED A LETTER OF SUPPORT FROM THE FDA FOR THE EXPLORATORY USE OF TKV IN CLINICAL TRIALS FOR ADPKD. RECEIVED A FORMAL QUALIFICATION OF TKV AS A PROGNOSTIC BIOMARKER FROM THE FDA IN THE FORM OF A DRAFT GUIDANCE (AUGUST, 2015) AND FROM THE EMA AS A QUALIFICATION OPINION (NOVEMBER, 2015). HELD A VERY SUCCESSFUL ADPKD SUMMIT MEETING WITH INDUSTRY, ACADEMIA, AND INTERNATIONAL REGULATORS TO DISCUSS POTENTIAL SOLUTIONS TO ADDRESS THE NEED FOR BETTER CLINICAL ENDPOINTS FOR CLINICAL TRIALS OF NEW THERAPIES FOR ADPKD. FORMAL QUALIFICATION OF TKV AS A PROGNOSTIC BIOMARKER WAS RECEIVED FROM THE FDA IN THE FORM OF FINAL GUIDANCE ON SEPTEMBER 15TH, 2016. TKV WAS ACCEPTED AS A "REASONABLY LIKELY SURROGATE" BY THE FDA ALLOWING IT TO BE USED IN ACCELERATED APPROVAL APPLICATIONS. THE GOAL OF THE PATIENT-REPORTED OUTCOME CONSORTIUM IS 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 PUBLICLY AVAILABLE FOR USE IN CLINICAL TRIALS WHERE COA-BASED ENDPOINTS ARE USED TO SUPPORT PRODUCT LABELING CLAIMS. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: THE FOLLOWING PRO INSTRUMENTS ARE IN VARIOUS STAGES OF THE QUALIFICATION PROCESS: FOR THE ASTHMA DAILY SYMPTOM DIARY, A QUANTITATIVE PILOT STUDY WAS COMPLETED AND QUANTITATIVE PILOT STUDY REPORT AND QUANTITATIVE DATA SUBMITTED TO FDA QRT FOR REVIEW. FOR THE SYMPTOMS OF MAJOR DEPRESSIVE DISORDER SCALE, A QUANTITATIVE PILOT STUDY WAS COMPLETED AND QUANTITATIVE PILOT STUDY REPORT AND QUANTITATIVE DATA WAS SUBMITTED TO FDA FOR REVIEW. FOR THE FUNCTIONAL DYSPEPSIA SYMPTOM DIARY, THE QUALITATIVE RESEARCH WAS COMPLETED AND QUALITATIVE RESEARCH SUMMARY REPORT IS IN DEVELOPMENT. FOR THE THREE INSTRUMENTS FOR IRRITABLE BOWEL SYNDROME, THE QUANTITATIVE PILOT STUDY PROTOCOL AND ANALYSIS PLAN WAS REVIEWED BY FDA, THEIR SUGGESTIONS WERE INCORPORATED INTO THE STUDY DOCUMENTS, AND A QUANTITATIVE PILOT STUDY IS BEING LAUNCHED. FOR THE NON-SMALL CELL LUNG CANCER SYMPTOM ASSESSMENT QUESTIONNAIRE, THE QUANTITATIVE PILOT STUDY WAS COMPLETED AND THE QUANTITATIVE PILOT STUDY REPORT IS IN DEVELOPMENT. THE PREDICTIVE SAFETY TESTING CONSORTIUM BRINGS TOGETHER PHARMACEUTICAL COMPANIES TO SHARE AND VALIDATE INNOVATIVE SAFETY TESTING METHODS UNDER ADVISEMENT OF THE U.S. FOOD AND DRUG ADMINISTRATION, EUROPEAN MEDICINES AGENCY, AND JAPAN'S PHARMACEUTICALS AND MEDICAL DEVICES AGENCY. CURRENTLY THE PSTC IS FOCUSED ON DEVELOPING AND OBTAINING REGULATORY QUALIFICATION OF IMPROVED CLINICAL SAFETY BIOMARKERS FOR USE IN DRUG DEVELOPMENT. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: RECEIVED EMA AND FDA LETTERS OF SUPPORT FOR FOUR SKELETAL MUSCLE INJURY BIOMARKERS IN RODENTS. SUBMITTED LIMITED CONTEXT OF USE QUALIFICATION PACKAGE TO FDA AND EMA OF A COMPOSITE MEASURE OF KIDNEY SAFETY BIOMARKERS FOR USE IN CLINICAL DRUG DEVELOPMENT STUDIES. SUBMITTED LETTER OF INTENT FOR THE QUALIFICATION OF GLUTAMATE DEHYDROGENASE AS A SPECIFIC BIOMARKER OF DRUG-INDUCED HEPATIC INJURY IN HUMANS. SUBMITTED PACKAGES FOR DILI, DIKI, AND DIVI BIOMARKERS TO EMA AND FDA FOR REVIEW WITH THE INTENT OF RECEIVING LETTERS OF SUPPORT. RECEIVED FDA LETTERS OF SUPPORT FOR LIVER SAFETY BIOMARKERS FOR USE IN CLINICAL DRUG DEVELOPMENT. A NEW CONSORTIUM ENTITLED PEDIATRICS TRIAL CONSORTIUM WAS LAUNCHED THIS YEAR. THE GOAL IS TO PROVIDE THE SUPPORT AND GUIDANCE NECESSARY FOR THE CRITICAL PATH INSTITUTE (C-PATH) TO CREATE A NEW NON-PROFIT ORGANIZATION THAT WILL WORK INDEPENDENTLY (BUT WITH INPUT FROM RELEVANT SECTORS) TO ADDRESS THE GAPS IN PEDIATRIC CLINICAL DEVELOPMENT. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: LAUNCHED A NEW INDEPENDENT ORGANIZATION CALLED INSTITUTE FOR ADVANCED CLINICAL TRIALS FOR CHILDREN. APPLIED FOR A 501(C)(3) NON-PROFIT STATUS THAT IS PENDING. INITIAL DIRECTORS AND OFFICERS HAVE BEEN APPOINTED AND BYLAWS HAVE BEEN ADOPTED. DEVELOPED AN OPERATIONAL PLAN ADDRESSING: GLOBAL INTEROPERABILITY WITH OTHER INITIATIVES; RECOMMENDATIONS ON LEADERSHIP ROLES; FUNDING STRATEGY. WROTE AND HAD EXTENSIVE LEGAL REVIEW ON AN ADVISORY REPORT. FORM 990, PART VI, SECTION A, LINE 2 LYLE BOOTMAN AND SHAUN KIRKPATRICK HAVE A BUSINESS RELATIONSHIP. MIKE KASSER AND JEFF JACOB HAVE A BUSINESS RELATIONSHIP. ALAN LEVIN AND PETER CORR HAVE A BUSINESS RELATIONSHIP. FORM 990, PART VI, SECTION B, LINE 11 FORM 990 IS PREPARED BY AN EXTERNAL CPA FIRM USING INFORMATION PROVIDED BY THE ORGANIZATION. THE FORM IS REVIEWED BY THE ORGANIZATION'S COMPTROLLER, COO, PRESIDENT/CEO, AN OUTSIDE CPA, 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. THE LAST INDEPENDENT COMPENSATION SURVEY WAS CONDUCTED FOR THE CEO IN AUGUST 2011. THE CEO'S SALARY HAS REMAINED CONSTANT SINCE THE TIME OF HER HIRE. 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 1,405,309. MANAGEMENT AND GENERAL EXPENSES 0. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 1,405,309. CONSULTING: PROGRAM SERVICE EXPENSES 1,027,701. MANAGEMENT AND GENERAL EXPENSES 37,144. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 1,064,845. OTHER PROFESSIONAL FEES: PROGRAM SERVICE EXPENSES 924,300. MANAGEMENT AND GENERAL EXPENSES 13,390. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 937,690. THE CRITICAL PATH FOUNDATION 1730 EAST RIVER ROAD SUITE 200 TUCSON AZ 85718 204780896 RAISING AND CONTRIBUTING FUNDS TO CRITICAL PATH INSTITUTE AZ 501(C)(3) 11 CRITICAL PATH INSTITUTE 1 CRITICAL PATH GLOBAL LTD 20-22 BEDFORD ROW LONDON UK WC1R 4JS FACILITATE WORK WITH EUROPEAN MEDICINES AGENCY UK CRITICAL PATH INSTITUTE 1 0 0 0 0 0 0 0 0 0 0 0 0 0 1 1 0 0 0 0