2018-02-23T15:04:46-06:00 2017-06-30 860683059 BEACHFLEISCHMAN PC 1985 E RIVER ROAD SUITE 201 TUCSON AZ 85718 990 2016-07-01 201991334 CRITICAL PATH INSTITUTE CRIT 5205473440 1730 EAST RIVER ROAD NO 200 TUCSON AZ 857185898 STEVEN BROADBENT MBA COO 5205473440 2018-02-17 1 KAREN K MCCLOSKEY CPA P00099644 5203214600 208.87.239.202 2018-02-17 15:58:07 US P 23AB17895BAC7DA56F918F3357BB5DF74DD723A1 EF92F78E7C0E069179DCA9050FD4B48B79C32FEB 2016 2021-01-29 14:40:06Z MARTHA BRUMFIELD PHD 1730 EAST RIVER ROAD NO 200 TUCSON AZ 857185898 15670367 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. 14 13 68 14 0 0 10197464 10085049 5069034 5531847 15089 50971 51912 2500 15333499 15670367 2657978 1853946 0 0 6010064 7188245 0 0 0 5997897 6113896 14665939 15156087 667560 514280 18234620 16508211 12782629 10541940 5451991 5966271 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 12569647 1853946 5531847 SEE SCHEDULE O 12569647 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 30 0 1 68 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 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 1.00 X X 0 0 0 MARTHA A BRUMFIELD PHD CHIEF EXECUTIVE OFFICER & PRESIDENT 40.00 X X 296577 0 31011 STEVEN T BROADBENT MBA CHIEF OPERATING OFFICER & TREASURER 40.00 X 187021 0 26155 LEWIS BARBIERI SECRETARY 40.00 X 113208 0 18707 LYNN D HUDSON PHD CHIEF SCIENCE OFFICER 40.00 X 195453 0 17522 JOHN-MICHAEL SAUER PHD EXECUTIVE DIRECTOR PSTC 40.00 X 184460 0 21850 STEPHEN J COONS PHD EXECUTIVE DIRECTOR PROC 40.00 X 179348 0 20915 STEVE ARNERIC PHD EXECUTIVE DIRECTOR 40.00 X 170534 0 21847 DIANE STEPHENSON PHD EXECUTIVE DIRECTOR CPP 40.00 X 164406 0 24104 1491007 0 182111 12 0 1 0 RESEARCH TRIANGLE INC 200 PARK OFFICES DRIVE RESEARCH TRIANGLE PARK NC 27709 RESEARCH 714099 PREMIER RESEARCH INTERNATIONAL PO BOX 347566 PITTSBURGH PA 15251 RESEARCH 264558 ADELPHI VALUES ADELPHI MILL BOLLINGTON CHESIRE UK SK105JB RESEARCH 256193 PACIFIC BIOMARKERS 45 ELLIOTT AVENUE WEST SUITE 300 SEATTLE WA 98119 RESEARCH SUPPORT SERVICES 220968 EPHIBIAN 3180 N SWAN ROAD TUCSON AZ 85712 IT SERVICES 211600 10 2824994 7260055 10085049 RESEARCH SUPPORT FEES 541700 5403668 5403668 OTHER REVENUE 900099 128179 128179 5531847 45971 45971 2500 0 2500 2500 2500 5000 0 5000 5000 5000 15670367 5531847 0 53471 X 1842400 1842400 11546 11546 681676 231086 450590 5222962 3971879 1251083 377873 284412 93461 493365 353031 140334 412369 292947 119422 103386 102629 757 125608 98153 27455 60193 59258 935 3296740 3245512 51228 196911 155096 41815 270536 231256 39280 376444 328596 47848 514007 263468 250539 475046 451235 23811 533142 522197 10945 52848 37543 15305 72767 56102 16665 DUES AND SUBSCRIPTIONS 27987 25190 2797 MISCELLANEOUS 8281 6111 2170 15156087 12569647 2586440 0 4084148 1969152 8406753 5994076 327557 1212619 1023373 400363 42542 159773 732850 659220 86511 73630 4248265 6674273 15471 24325 18234620 16508211 632632 753078 12149997 9788862 12782629 10541940 X 5451991 5966271 5451991 5966271 18234620 16508211 514280 0 X 0 1 X 1 1 1 X 5915630 5370782 10269811 10197464 10085049 41838736 5915630 5370782 10269811 10197464 10085049 41838736 16583869 25254867 21279 70230 82059 80580 48471 302619 42141355 20301825 0.59930 0.67610 X RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 61348 61348 15134932 15196280 909814 227454 0 0 551743 809246 883297 909814 3154100 4731150 60221 67262 11731 61348 200562 137936 202312 220824 227454 788526 1182789 PART II-A, LINE 1B C-PATH SPENT A MINOR AMOUNT OF FUNDS ON LOBBYING IN FYE 06/30/2017. GENERALLY, C-PATH DOES NOT LOBBY FOR OR AGAINST ANY SPECIFIC LEGISLATION. C-PATH EXPENDS SOME TIME AND RESOURCES TO EDUCATE MEMBERS OF CONGRESS IN THE AREA OF DRUG DEVELOPMENT. 42014 32361 9653 179139 179139 0 511697 447720 63977 73630 X 15670367 0 15670367 0 15670367 15156087 0 15156087 0 15156087 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 STATE 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 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. 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 11546 0 0 0 0 0 0 11546 0 11546 EUROPE (INCLUDING ICELAND & GREENLAND) CLINICAL RESEARCH 11546 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) 1327338 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 430802 DEVELOP A DISEASE PROGRESSION MODEL FOR TUBERCULOSIS UNIVERSITY OF FLORIDA PO BOX 113001 GAINSVILLE FL 32611 596002052 STATE OF FLORIDA 15000 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) 28615 DEVELOP CLINICAL DATA STANDARDS COLORADO STATE UNIVERSITY 6003 CAMPUS DELIVERY FORT COLLINS CO 80523 846000545 STATE OF COLORADO 8504 DEVELOP COMPUTER MODELS FOR TUBERCULOSIS JOHN HOPKINS UNIVERSITY 3910 KESWICK ROAD NO N4327B BALTIMORE MD 21211 520595110 501(C)(3) 32141 DEVELOP COMPUTER MODELS FOR TUBERCULOSIS 6 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 & PRESIDENT 296577 0 0 0 0 0 24000 0 7011 0 327588 0 0 0 STEVEN T BROADBENT MBA CHIEF OPERATING OFFICER & TREASURER 187021 0 0 0 0 0 15290 0 10865 0 213176 0 0 0 LYNN D HUDSON PHD CHIEF SCIENCE OFFICER 195453 0 0 0 0 0 15180 0 2342 0 212975 0 0 0 JOHN-MICHAEL SAUER PHD EXECUTIVE DIRECTOR PSTC 184460 0 0 0 0 0 14998 0 6852 0 206310 0 0 0 STEPHEN J COONS PHD EXECUTIVE DIRECTOR PROC 179348 0 0 0 0 0 14270 0 6645 0 200263 0 0 0 STEVE ARNERIC PHD EXECUTIVE DIRECTOR 170534 0 0 0 0 0 11162 0 10685 0 192381 0 0 0 DIANE STEPHENSON PHD EXECUTIVE DIRECTOR CPP 164406 0 0 0 0 0 13301 0 10803 0 188510 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, 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 COALITION AGAINST MAJOR DISEASES DEVELOPS TOOLS AND NOVEL METHODOLOGIES TO ACCELERATE PROGRESS IN THE PREVENTION, TREATMENT AND CURE OF MAJOR NEURODEGENERATIVE DISEASES FOCUSING ON ALZHEIMER'S DISEASE. THIS YEAR'S ACCOMPLISHMENTS INCLUDE: DEVELOPED AN ADDENDUM TO THE INFORMED CONSENT FORM DEVELOPED LAST YEAR THAT WILL ENABLE BROADER SHARING OF PATIENT DATA AND SPECIMENS FOR FUTURE RESEARCH; OBTAINED REGULATORY AGREEMENT FOR THE STATISTICAL ANALYSIS APPROACH FOR THE HIPPOCAMPAL VOLUME BIOMARKER QUALIFICATION EFFORT. THE FULL QUALIFICATION PACKAGE SHOULD BE SUBMITTED IN EARLY 2018. HELD WORKSHOPS ON THE USE OF BIOMETRIC MONITORING DEVICES IN CLINICAL TRIALS AND ASSOCIATED DATA STANDARDS. PUBLISHED TWO JOURNAL ARTICLES. 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 IS A PARTNERSHIP BETWEEN THE CLINICAL DATA INTERCHANGE STANDARDS CONSORTIUM (CDISC) AND THE CRITICAL PATH INSTITUTE (C-PATH). THIS YEAR THE CONSORTIUM RECEIVED THREE FDA GRANTS AND BEGAN WORK TO DEVELOP DATA STANDARDS FOR HIV, CLOSTRIDIUM DIFFICILE-ASSOCIATED DIARRHEA, AND ONE FOR MEDICAL COUNTERMEASURE STUDIES UNDER THE ANIMAL RULE; PUBLISHED CDISC STANDARDS ON KIDNEY TRANSPLANT, RHEUMATOID ARTHRITIS, MAJOR DEPRESSIVE DISORDER, EBOLA, DIABETIC KIDNEY DISEASE AND MALARIA STANDARDS, AND CARDIOVASCULAR IMAGING; POSTED A STANDARD FOR DUCHENE MUSCULAR DYSTROPHY FOR PUBLIC REVIEW. THE CRITICAL PATH FOR PARKINSON'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: FOUR NEW PARKINSON'S CLINICAL TRIAL DATASETS HAVE BEEN ADDED TO THE DATABASE; SUBMITTED A LETTER OF INTENT AND FULL QUALIFICATION PACKAGE TO EMA FOR THE QUALIFICATION OF MOLECULAR NEUROIMAGING OF THE DOPAMINE TRANSPORTER(DAT) 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, 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: SUBMITTED INPUT TO THE FDA DRAFT GUIDANCE ON INFECTIOUS DISEASE NEXT GENERATION SEQUENCING BASED DIAGNOSTIC DEVICES; MET WITH THE EMA ABOUT CPTR'S PHYSIOLOGICALLY BASED TB-INFECTED LUNG MODEL; FORMED A NEW PARTNERSHIP WITH TGEN NORTH/NAU TO DEVELOP AN AZ-BASED SEQUENCING AND DATA PLATFORM COE FOR WHOLE GENOME SEQUENCING OF M. TUBERCULOSIS AND CONTRIBUTE TO THE RESEQTB PLATFORM. THIS WORK IS SUPPORTED BY THE BMGF SEQUENCING SUPPLEMENT GRANT AWARDED TO C-PATH. CPTR IS IN DISCUSSION WITH IMI-PREDICT-TB TO TRANSFER AND HOST THEIR CLINICAL TRIAL DATA REPOSITORY AS THEIR PROGRAM SUNSETS IN EARLY 2018. CPTR IS PARTNERING WITH COMMONWEALTH INFORMATICS TO COLLABORATE ON A COMMONWEALTH CLINICAL DATA ANALYTICS (CCDA) PILOT PROJECT. CPTR HAD A CRITICAL PATH INNOVATION MEETING ON CPTR'S LIPOARABINOMANNAN (LAM) BASED BIOMARKER. A LETTER OF INTENT WAS SUBMITTED TO THE FDA TO START THE PROCESS TO QUALIFY THE BIOMARKER. THE DATA COLLABORATION CENTER 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. ITS ACCOMPLISHMENTS THIS YEAR INCLUDE: REMAPPED PARKINSON'S AND DUCHENNE CLINICAL TRIAL DATASETS AND INCLUDED THEM IN THEIR RESPECTIVE DATABASES ACCESSIBLE BY CONSORTIUM MEMBERS; MADE THE TB-ALLIANCE CLINICAL TRIAL DATASETS AVAILABLE TO EXTERNAL QUALIFIED RESEARCHER; DEVELOPED AN INTERACTIVE INTERFACE FOR THE TB PLATFORM; BEGAN DATA CURATION ACTIVITIES FOR THE NEW TYPE 1 DIABETES CONSORTIUM. THE DUCHENNE REGULATORY SCIENCE CONSORTIUM'S 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: ACQUIRED SEVERAL ADDITIONAL CLINICAL TRIAL DATASETS FOR INCLUSION IN ITS DATABASE; COMPLETED A NEW DRAFT CDISC DATA STANDARD THAT IS IN PUBLIC REVIEW; DEVELOPED PROPOSED FORMAT FOR DISEASE PROGRESSION MODEL TO BE PRESENTED TO REGULATORS. 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: INITIATED A PROJECT ENTITLED: COMPARABILITY OF PROVISIONED DEVICE VS BRING-YOUR-OWN-DEVICE IN SUBJECTS WITH COPD; WORKED WITH THE PRO CONSORTIUM TO DEVELOP A QUESTIONNAIRE ENTITLED, "BARRIERS TO ADOPTION OF ELECTRONIC DATA COLLECTION OF CLINICAL OUTCOME ASSESSMENTS IN CLINICAL TRIALS" THAT WAS SENT TO INDUSTRY MEMBERS. C-PATH INITIATED A PROJECT IN PARTNERSHIP WITH CHDI FOUNDATION TO FORM A NEW CONSORTIUM AND WORK ON DRUG DEVELOPMENT TOOLS FOR HUNTINGTON'S DISEASE. THIS YEAR C-PATH LED THE EFFORT TO DEVELOP A NEW CDISC DATA STANDARD FOR HUNTINGTON'S DISEASE. INVENTORIES OF EXISTING CLINICAL TRIAL DATASETS AND DISEASE MODELS WERE COMPILED TO INFORM A DATA AGGREGATION PLAN. A KICKOFF MEETING IS PLANNED FOR NOVEMBER OF 2017. 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. THIS PAST YEAR INC PROVIDED INPUT TO NICHD FOR INFORMATION OF PRIORITIES IN PEDIATRIC THERAPEUTICS; PUBLISHED SEVERAL MANUSCRIPTS; WORKED TO DEVELOP A GRADING CRITERIA FOR ADVERSE EVENT SEVERITY; EXPANDED THEIR SCOPE TO INCLUDE RETINOPATHY OF PREMATURITY, NECROTIZING ENTEROCOLITIS, AND HEMODYNAMIC ADAPTATION. THE MULTIPLE SCLEROSIS OUTCOME ASSESSMENTS 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: EFFORTS CONTINUE TO QUALIFY NEW ENDPOINTS FOR CLINICAL TRIALS IN MS; FINAL QUALIFICATION PACKAGES SHOULD BE SUBMITTED TO BOTH THE FDA AND EMA LATER IN 2017; FIVE NEW ARTICLES WERE PUBLISHED; THE PLACEBO ARM DATA WITHIN THE MS DATABASE HAS BEEN MADE AVAILABLE TO QUALIFIED EXTERNAL RESEARCHERS. THE POLYCYSTIC KIDNEY DISEASE OUTCOMES CONSORTIUM 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. FORM 990, PART III, LINE 4A THE PATIENT-REPORTED OUTCOME CONSORTIUM 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. THE NON-SMALL CELL LUNG CANCER WORKING GROUP COMPLETED ITS QUANTITATIVE PILOT STUDY AND SUBMITTED THE REPORT TO THE FDA. WITH POSITIVE FEEDBACK FROM THE FDA, PROC IS WORKING ON A PERFORMANCE OUTCOME INSTRUMENT TO ASSESS COGNITIVE IMPAIRMENT IN CLINICAL TRIALS FOR MILD COGNITIVE IMPAIRMENT DUE TO ALZHEIMER'S DISEASE; SUBMITTED THE DEPRESSION QUANTITATIVE PILOT STUDY REPORT AND DATA TO FDA FOR REVIEW; INITIATED AN EFFORT TO DEVELOP A PATIENT-REPORTED MEASURE TO ASSESS FATIGUE IN PATIENTS WITH RHEUMATOID ARTHRITIS; SUBMITTED THE ASTHMA QUANTITATIVE PILOT STUDY REPORT AND STUDY DATA TO FDA; PUBLISHED EIGHT ARTICLES; QUALIFICATION BRIEFING PACKAGES WERE SUBMITTED FOR DEPRESSION AND NON-SMALL CELL LUNG CANCER FOR FDA REVIEW. 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. THIS YEAR THE CONSORTIUM: CO-HOSTED THE DRUG INDUCED LIVER INJURY WORKSHOP; RECEIVED LETTERS OF SUPPORT FOR LIVER, KIDNEY, AND VASCULAR INJURY SAFETY BIOMARKERS FROM THE FDA AND EMA; HELD A SERIES OF WEBINARS ON BILE SALT EXPORT PUMP INHIBITION; SUBMITTED A QUALIFICATION BRIEFING PACKAGE OF GLUTAMATE DEHYDROGENASE AS A SPECIFIC BIOMARKER OF DRUG-INDUCED LIVER INJURY, AND HELD AN ADVICE AND CONSULTATION MEETING WITH THE FDA; HELD A CRITICAL PATH INNOVATION MEETING WITH THE FDA TO DISCUSS A RESEARCH PLAN FOR THE QUALIFICATION OF TRANSLATIONAL DRUG-INDUCED PANCREATIC INJURY SAFETY BIOMARKERS. THE PEDIATRIC TRIALS CONSORTIUM ACCOMPLISHED ITS GOAL OF ESTABLISHING A NEW, INDEPENDENT NON-PROFIT ORGANIZATION TO SUPPORT AND ADVANCE RESEARCH AND EDUCATION ON INNOVATIVE PEDIATRIC TREATMENTS. THE NEW INSTITUTE FOR ADVANCED CLINICAL TRIALS FOR CHILDREN SERVES AS A NEUTRAL FORUM FOR PUBLIC-PRIVATE COLLABORATION IN ADDRESSING COMMON CHALLENGES IN PEDIATRIC TRIALS TO HELP DEMYSTIFY THE STUDIES. IT WILL ALSO ASSIST IN OPTIMIZING STUDY DESIGNS, PROTOCOLS, BEST PRACTICES, TRAINING, AND ENGAGEMENT OF PATIENTS AND PARENTS. TWO NEW CONSORTIA WERE INITIATED THIS YEAR. THE FIRST IS FOCUSING ON QUALIFYING ISLET AUTOIMMUNITY ANTIBODIES AS PROGNOSTIC BIOMARKERS TO BE USED IN THE DEVELOPMENT OF THERAPIES FOR THE TREATMENT, AND ULTIMATELY, THE PREVENTION, OF TYPE 1 DIABETES. THE SECOND 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. FORM 990, PART VI, SECTION A, LINE 2 MIKE KASSER AND JEFF JACOB HAVE A BUSINESS RELATIONSHIP. PETER HUTT AND JEFF JACOB HAVE A BUSINESS RELATIONSHIP. WAIN FISHBURN AND JEFF JACOB 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 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, WHICH IS DEVELOPED BASED ON SALARY SURVEY INFORMATION, 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 1,924,899. MANAGEMENT AND GENERAL EXPENSES 0. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 1,924,899. CONSULTING: PROGRAM SERVICE EXPENSES 665,528. MANAGEMENT AND GENERAL EXPENSES 23,996. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 689,524. OTHER PROFESSIONAL FEES: PROGRAM SERVICE EXPENSES 655,085. MANAGEMENT AND GENERAL EXPENSES 27,232. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 682,317. 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