Attach to Form 990 or Form 990-EZ.
Go to
www.irs.gov/Form990 for the latest information.
| (i) Name of supported organization | (ii) EIN | (iii) Type of organization (described on lines 1- 10 above (see instructions)) | (iv) Is the organization listed in your governing document? | (v) Amount of monetary support (see instructions) | (vi) Amount of other support (see instructions) | |
|---|---|---|---|---|---|---|
| Yes | No | |||||
|
Total |
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Calendar year (or fiscal year beginning in) ![]() |
(a) 2014 | (b) 2015 | (c) 2016 | (d) 2017 | (e) 2018 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 1 | Gifts, grants, contributions, and membership fees received. (Do not include any "unusual grant.") .. | 10,269,811 | 10,197,464 | 10,085,049 | 10,019,550 | 10,698,823 | 51,270,697 |
| 2 | Tax revenues levied for the organization's benefit and either paid to or expended on its behalf.... | ||||||
| 3 | The value of services or facilities furnished by a governmental unit to the organization without charge.. | ||||||
| 4 | Total. Add lines 1 through 3 | 10,269,811 | 10,197,464 | 10,085,049 | 10,019,550 | 10,698,823 | 51,270,697 |
| 5 | The portion of total contributions by each person (other than a governmental unit or publicly supported organization) included on line 1 that exceeds 2% of the amount shown on line 11, column (f).. | 24,619,913 | |||||
| 6 | Public support. Subtract line 5 from line 4. | 26,650,784 | |||||
Calendar year
(or fiscal year beginning in) ![]() |
(a) 2014 | (b) 2015 | (c) 2016 | (d) 2017 | (e) 2018 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 7 | Amounts from line 4.. | 10,269,811 | 10,197,464 | 10,085,049 | 10,019,550 | 10,698,823 | 51,270,697 |
| 8 | Gross income from interest, dividends, payments received on securities loans, rents, royalties and income from similar sources... | 82,059 | 80,580 | 48,471 | 83,667 | 215,046 | 509,823 |
| 9 | Net income from unrelated business activities, whether or not the business is regularly carried on.. | ||||||
| 10 | Other income. Do not include gain or loss from the sale of capital assets (Explain in Part VI.).. | ||||||
| 11 | Total support. Add lines 7 through 10 | 51,780,520 | |||||
Calendar year (or fiscal year beginning in) ![]() |
(a) 2014 | (b) 2015 | (c) 2016 | (d) 2017 | (e) 2018 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 1 | Gifts, grants, contributions, and membership fees received. (Do not include any "unusual grants.") . | ||||||
| 2 | Gross receipts from admissions, merchandise sold or services performed, or facilities furnished in any activity that is related to the organization's tax-exempt purpose | ||||||
| 3 | Gross receipts from activities that are not an unrelated trade or business under section 513 ..... | ||||||
| 4 | Tax revenues levied for the organization's benefit and either paid to or expended on its behalf... | ||||||
| 5 | The value of services or facilities furnished by a governmental unit to the organization without charge | ||||||
| 6 | Total. Add lines 1 through 5 | ||||||
| 7a | Amounts included on lines 1, 2, and 3 received from disqualified persons | ||||||
| b | Amounts included on lines 2 and 3 received from other than disqualified persons that exceed the greater of $5,000 or 1% of the amount on line 13 for the year. | ||||||
| c | Add lines 7a and 7b.. | ||||||
| 8 | Public support. (Subtract line 7c from line 6.) | ||||||
Calendar year (or fiscal year beginning in) ![]() |
(a) 2014 | (b) 2015 | (c) 2016 | (d) 2017 | (e) 2018 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 9 | Amounts from line 6... | ||||||
| 10a | Gross income from interest, dividends, payments received on securities loans, rents, royalties and income from similar sources.. | ||||||
| b | Unrelated business taxable income (less section 511 taxes) from businesses acquired after June 30, 1975. | ||||||
| c | Add lines 10a and 10b. | ||||||
| 11 | Net income from unrelated business activities not included in line 10b, whether or not the business is regularly carried on. | ||||||
| 12 | Other income. Do not include gain or loss from the sale of capital assets (Explain in Part VI.) .. | ||||||
| 13 | Total support. (Add lines 9, 10c, 11, and 12.).. | ||||||
| Section A - Adjusted Net Income | (A) Prior Year |
(B) Current Year (optional) |
||||
| 1 | Net short-term capital gain | 1 | ||||
| 2 | Recoveries of prior-year distributions | 2 | ||||
| 3 | Other gross income (see instructions) | 3 | ||||
| 4 | Add lines 1 through 3 | 4 | ||||
| 5 | Depreciation and depletion | 5 | ||||
| 6 | Portion of operating expenses paid or incurred for production or collection of gross income or for management, conservation, or maintenance of property held for production of income (see instructions) | 6 | ||||
| 7 | Other expenses (see instructions) | 7 | ||||
| 8 | Adjusted Net Income (subtract lines 5, 6 and 7 from line 4) | 8 | ||||
| Section B - Minimum Asset Amount | (A) Prior Year |
(B) Current Year (optional) |
||||
| 1 | Aggregate fair market value of all non-exempt-use assets (see instructions for short tax year or assets held for part of year): | 1 | ||||
| a | Average monthly value of securities | 1a | ||||
| b | Average monthly cash balances | 1b | ||||
| c | Fair market value of other non-exempt-use assets | 1c | ||||
| d | Total (add lines 1a, 1b, and 1c) | 1d | ||||
| e |
Discount claimed for blockage or other factors (explain in detail in Part VI): |
|||||
| 2 | Acquisition indebtedness applicable to non-exempt use assets | 2 | ||||
| 3 | Subtract line 2 from line 1d | 3 | ||||
| 4 | Cash deemed held for exempt use. Enter 1-1/2% of line 3 (for greater amount, see instructions). | 4 | ||||
| 5 | Net value of non-exempt-use assets (subtract line 4 from line 3) | 5 | ||||
| 6 | Multiply line 5 by .035 | 6 | ||||
| 7 | Recoveries of prior-year distributions | 7 | ||||
| 8 | Minimum Asset Amount (add line 7 to line 6) | 8 | ||||
| Section C - Distributable Amount | Current Year | |||||
| 1 | Adjusted net income for prior year (from Section A, line 8, Column A) | 1 | ||||
| 2 | Enter 85% of line 1 | 2 | ||||
| 3 | Minimum asset amount for prior year (from Section B, line 8, Column A) | 3 | ||||
| 4 | Enter greater of line 2 or line 3 | 4 | ||||
| 5 | Income tax imposed in prior year | 5 | ||||
| 6 | Distributable Amount. Subtract line 5 from line 4, unless subject to emergency temporary reduction (see instructions) | 6 | ||||
| Section D - Distributions | Current Year | |
|---|---|---|
| 1 Amounts paid to supported organizations to accomplish exempt purposes | ||
|
2
Amounts paid to perform activity that directly furthers exempt purposes of supported organizations, in excess of income from activity |
||
| 3 Administrative expenses paid to accomplish exempt purposes of supported organizations | ||
| 4 Amounts paid to acquire exempt-use assets | ||
| 5 Qualified set-aside amounts (prior IRS approval required) | ||
| 6 Other distributions (describe in Part VI). See instructions | ||
| 7Total annual distributions. Add lines 1 through 6. | ||
|
8
Distributions to attentive supported organizations to which the organization is responsive (provide details in Part VI). See instructions |
||
| 9 Distributable amount for 2018 from Section C, line 6 | ||
| 10 Line 8 amount divided by Line 9 amount | ||
| Section E - Distribution Allocations (see instructions) |
(i) Excess Distributions |
(ii) Underdistributions Pre-2018 |
(iii) Distributable Amount for 2018 |
|
|---|---|---|---|---|
|
1
Distributable amount for 2018 from Section C, line 6 |
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|
2
Underdistributions, if any, for years prior to 2018 (reasonable cause required-- explain in Part VI). See instructions. |
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| 3 Excess distributions carryover, if any, to 2018: | ||||
| a From 2013....... | ||||
| b From 2014....... | ||||
| c From 2015....... | ||||
| d From 2016....... | ||||
| e From 2017....... | ||||
| fTotal of lines 3a through e | ||||
| g Applied to underdistributions of prior years | ||||
| h Applied to 2018 distributable amount | ||||
|
i
Carryover from 2013 not applied (see instructions) |
||||
| j Remainder. Subtract lines 3g, 3h, and 3i from 3f. | ||||
| 4Distributions for 2018 from Section D, line 7: | ||||
| $ | ||||
| a Applied to underdistributions of prior years | ||||
| b Applied to 2018 distributable amount | ||||
| c Remainder. Subtract lines 4a and 4b from 4. | ||||
|
5
Remaining underdistributions for years prior to 2018, if any. Subtract lines 3g and 4a from line 2. If the amount is greater than zero, explain in Part VI. See instructions. |
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|
6
Remaining underdistributions for 2018. Subtract lines 3h and 4b from line 1. If the amount is greater than zero, explain in Part VI. See instructions. |
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|
7 Excess distributions carryover to 2019. Add lines 3j and 4c. |
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| 8 Breakdown of line 7: | ||||
| a Excess from 2014...... | ||||
| b Excess from 2015..... | ||||
| c Excess from 2016..... | ||||
| d Excess from 2017..... | ||||
| e Excess from 2018..... | ||||
| Facts And Circumstances Test |
|---|
| Return Reference | Explanation |
|---|
| Software ID: | |
| Software Version: |
Attach to Form 990 or 990-EZ.
Go to www.irs.gov/Form990 for the latest information.
| Return Reference | Explanation |
|---|---|
| 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. |
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