Attach to Form 990 or Form 990-EZ.
Go to
www.irs.gov/Form990 for instructions and 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) 2015 | (b) 2016 | (c) 2017 | (d) 2018 | (e) 2019 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 1 | Gifts, grants, contributions, and membership fees received. (Do not include any "unusual grant.") .. | 10,197,464 | 10,085,049 | 10,019,550 | 10,698,823 | 12,978,641 | 53,979,527 |
| 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 | 10,197,464 | 10,085,049 | 10,019,550 | 10,698,823 | 12,978,641 | 53,979,527 |
| 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).. | 21,605,657 | |||||
| 6 | Public support. Subtract line 5 from line 4. | 32,373,870 | |||||
Calendar year
(or fiscal year beginning in) ![]() |
(a) 2015 | (b) 2016 | (c) 2017 | (d) 2018 | (e) 2019 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 7 | Amounts from line 4.. | 10,197,464 | 10,085,049 | 10,019,550 | 10,698,823 | 12,978,641 | 53,979,527 |
| 8 | Gross income from interest, dividends, payments received on securities loans, rents, royalties and income from similar sources... | 80,580 | 48,471 | 83,667 | 215,046 | 156,753 | 584,517 |
| 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 | 54,564,044 | |||||
Calendar year (or fiscal year beginning in) ![]() |
(a) 2015 | (b) 2016 | (c) 2017 | (d) 2018 | (e) 2019 | (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) 2015 | (b) 2016 | (c) 2017 | (d) 2018 | (e) 2019 | (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 2019 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-2019 |
(iii) Distributable Amount for 2019 |
|
|---|---|---|---|---|
| 1 Distributable amount for 2019 from Section C, line 6 | ||||
|
2
Underdistributions, if any, for years prior to 2019 (reasonable cause required-- explain in Part VI). See instructions. |
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| 3 Excess distributions carryover, if any, to 2019: | ||||
| a From 2014....... | ||||
| b From 2015....... | ||||
| c From 2016....... | ||||
| d From 2017....... | ||||
| e From 2018....... | ||||
| fTotal of lines 3a through e | ||||
| g Applied to underdistributions of prior years | ||||
| h Applied to 2019 distributable amount | ||||
|
i
Carryover from 2014 not applied (see instructions) |
||||
| j Remainder. Subtract lines 3g, 3h, and 3i from 3f. | ||||
| 4Distributions for 2019 from Section D, line 7: | ||||
| $ | ||||
| a Applied to underdistributions of prior years | ||||
| b Applied to 2019 distributable amount | ||||
| c Remainder. Subtract lines 4a and 4b from 4. | ||||
|
5
Remaining underdistributions for years prior to 2019, 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 2019. 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 2020. Add lines 3j and 4c. |
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| 8 Breakdown of line 7: | ||||
| a Excess from 2015..... | ||||
| b Excess from 2016..... | ||||
| c Excess from 2017..... | ||||
| d Excess from 2018..... | ||||
| e Excess from 2019..... | ||||
| 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, 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. |
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