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.") .. | 5,370,782 | 10,269,811 | 10,197,464 | 10,085,049 | 10,019,550 | 45,942,656 |
| 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 | 5,370,782 | 10,269,811 | 10,197,464 | 10,085,049 | 10,019,550 | 45,942,656 |
| 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).. | 21,404,373 | |||||
| 6 | Public support. Subtract line 5 from line 4. | 24,538,283 | |||||
Calendar year
(or fiscal year beginning in) ![]() |
(a) 2015 | (b) 2016 | (c) 2017 | (d) 2018 | (e) 2019 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 7 | Amounts from line 4.. | 5,370,782 | 10,269,811 | 10,197,464 | 10,085,049 | 10,019,550 | 45,942,656 |
| 8 | Gross income from interest, dividends, payments received on securities loans, rents, royalties and income from similar sources... | 70,230 | 82,059 | 80,580 | 48,471 | 83,667 | 365,007 |
| 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 | 46,307,663 | |||||
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) |
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| 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) |
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| 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 |
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| Return Reference | Explanation |
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| 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, PROGRAM SERVICE ACCOMPLISHMENTS | 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: THE COALITION AGAINST MAJOR DISEASES WAS REBRANDED AS THE CRITICAL PATH FOR ALZHEIMER'S DISEASE. IT 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) RECEIVING A LETTER OF SUPPORT FROM THE EMA ENCOURAGING INDUSTRY SPONSORS TO SHARE WITH CPAD THE PATIENT-LEVEL DATA FROM COMPLETED PHASE II AND III CLINICAL TRIALS, INCLUDING ACTIVE AND CONTROL ARMS, ALLOWING CPAD TO COMPLETE DEVELOPMENT AND VALIDATION OF A PROPOSED QUANTITATIVE MODEL FOR IMPLEMENTATION BY SPONSORS ACTIVELY DESIGNING CLINICAL TRIALS IN AMNESTIC MILD COGNITIVE IMPAIRMENT (AMCI). (2) GENERATED GRAPHICAL USER INTERFACE-BASED UPDATES TO THE AD MILD-TO-MODERATE CLINICAL TRIAL SIMULATION TOOL AND MILD COGNITIVE IMPAIRMENT (MCI) CLINICAL TRIAL SIMULATION TOOL - THE HIPPOCAMPAL NEUROIMAGING-INFORMED AMNESTIC MCI CLINICAL TRIAL SIMULATOR. (3) INCREASED USE OF THE DATABASE AND MODELS. THE CRITICAL PATH FOR PARKINSON'S 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) SUBMISSION TO EMA AND FDA OF THE FIRST MODELING TOOL FOR PARKINSON'S- "MODEL-BASED CLINICAL TRIAL ENRICHMENT TOOL FOR PARKINSON DISEASE CLINICAL TRIALS". (2) RECEIVED FDA'S FORMAL RESPONSE TO CPP'S LETTER OF INTENT OUTLINING RECOMMENDATIONS FOR WHAT SHOULD BE SUBMITTED IN THE MODELING BRIEFING DOCUMENT. (3) EMA QUALIFICATION ADVICE LETTER RECEIVED FOR THE MODEL-BASED CLINICAL TRIAL ENRICHMENT TOOL FOR PARKINSON'S DISEASE 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: (1) ELEVEN NEW TB CLINICAL DATASETS WERE ACQUIRED, CURATED, AND INTEGRATED INTO THE TB PLATFORM FOR AGGREGATION OF CLINICAL TB STUDIES (TB-PACTS) PLATFORM AND MADE ACCESSIBLE TO QUALIFIED RESEARCHERS. TO DATE, OVER 55 APPLICATIONS HAVE BEEN SUBMITTED AND 42 HAVE BEEN APPROVED TO SUPPORT TB RESEARCH. (2) COMPLETED THE STAGE 2 ANALYSIS OF TB RE-ANALYSIS OF FLUOROQUINOLONE EXECUTED CLINICAL TRIALS (TB-REFLECT), WHICH FOCUSED ON ISONIAZID RESISTANT SUB-COHORT AND EXAMINATION OF SAFETY FINDINGS FOR STANDARD OF CARE REGIMENS WHICH CONTAIN A FLUOROQUINOLONE. (3) COMPLETED OR SIGNIFICANTLY ADVANCED THE FOLLOWING MODELING AND SIMULATION PROJECTS: POPULATION PK/PD, LIQUID CULTURE, QUANTITATIVE SYSTEMS PHARMACOLOGY, AND CARDIAC RISK ASSESSMENT ALGORITHM. ADDITIONALLY, HFS-TB EXPERIMENTS CHARACTERIZING THE PK/PD ATTRIBUTES OF THREE DRUGS AND REGIMENS FOR TB HAVE BEEN COMPLETED, WITH FOUR EXPERIMENTS ONGOING. OTHER MODEL-BASED ANALYSES ARE ONGOING TO IDENTIFY PREFERRED METHODS FOR THE TRANSLATION OF DATA FROM IN VITRO AND IN VIVO (ANIMAL) MODELS TO PREDICT CLINICAL OUTCOMES FOR ANTI-TB DRUGS AND REGIMENS. (4) EXPANDED ANALYSES TO SUPPORT PBPK QUALIFICATION EFFORTS BASED ON FEEDBACK FROM EMA'S SCIENTIFIC ADVICE WORKING PARTY (SAWP). FURTHER ANALYSES DESIGNED TO VERIFY PBPK GRANULOMA MODEL PERFORMANCE ARE IN PROGRESS. 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: (1) LAUNCHED THE DUCHENNE'S REGULATORY SCIENCE CONSORTIUM (D-RSC) DATABASE WHICH WILL BE USED IN THE DEVELOPMENT OF A DMD DISEASE PROGRESSION MODEL AND CLINICAL TRIAL SIMULATION PLATFORM. (2) LAUNCHED THE CATALYSIS FOUNDATION ONLINE DATA REPOSITORY TO PROVIDE A PLATFORM TO MAKE DATASETS FROM A TREATMENT RESPONSE BIOMARKER STUDY OF TB POSITIVE, HIV NEGATIVE PATIENTS AVAILABLE TO QUALIFIED TB RESEARCHERS. (3) LAUNCHED THE FRIEDREICH'S ATAXIA INTEGRATED CLINICAL DATABASE FOR USE BY QUALIFIED RESEARCHERS. (4) RELEASED TB-PACTS VERSION 2.0 CONTAINING DATA FROM 14 NEW TB STUDIES FOR A TOTAL OF 17. 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: (1) ACQUIRED TWELVE DATASETS, AND DATA SHARING AGREEMENTS FOR ADDITIONAL DATASETS ARE UNDERWAY. PRESENTLY, ~1,100 INDIVIDUAL PATIENT RECORDS ARE INCLUDED IN THE D-RSC DATABASE, EXCLUDING DATA FROM A PATIENT REPORTED REGISTRY THAT WILL NOT BE USED FOR ANALYSIS. APPROXIMATELY 4000-5000 DATA-POINTS ARE IN THE DATABASE FOR EACH MEASURE OF INTEREST. PARTS OF THE DATABASE ARE OPEN FOR USE BY CONSORTIUM MEMBERS AND HAVE BEEN USED BY AT LEAST FOUR COMPANY MEMBERS AND BY PPMD IN SUPPORT OF INITIAL ANALYSIS AROUND THE DEVELOPMENT OF THE DUCHENNE PLATFORM TRIAL. (2) PUBLISHED THE CDISC THERAPEUTIC AREA USER GUIDE. (3) DEVELOPMENT OF THE LETTER OF INTENT AND MODELING PLAN 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: (1) CONTRIBUTED TO EMA'S GOOD CLINICAL PRACTICE INSPECTORS WORKING GROUP (GCP IWG) BY OFFERING THE EPRO CONSORTIUM'S STAKEHOLDER PERSPECTIVE DURING PAST MEETING OF THE GCP IWG ON TOPICS SUCH AS: RESPONSIBILITIES OF EPRO PROVIDERS REGARDING SOURCE DATA, USE OF ELECTRONIC INFORMED CONSENT, AND RESPONSIBILITIES OF EPRO PROVIDERS WHEN COLLECTING ELECTRONIC SIGNATURES. C-PATH LAUNCHED A NEW CONSORTIUM IN PARTNERSHIP WITH CHDI FOUNDATION TO WORK ON DRUG DEVELOPMENT TOOLS FOR HUNTINGTON'S DISEASE. THE ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) RECRUITED 14 INDUSTRY PARTNERS AND 16 SCIENTIFIC ADVISORS. (2) DEVELOPED AND PUBLISHED THE CDISC HD-SPECIFIC THERAPEUTIC AREA USER GUIDE. (3) CREATED A COMPREHENSIVE INVENTORY OF HD CLINICAL DATA SETS AND MODELING AND SIMULATION TOOLS. (4) ACQUIRED THE FIRST TWO DATA SETS, ENROLL-HD, AN ONGOING NATURAL HISTORY STUDY THAT INCLUDES 8000 SUBJECTS, AND TRACK-HD, A COMPLETED OBSERVATIONAL STUDY THAT INVESTIGATED POTENTIAL OUTCOME MEASURES IN OVER 300 SUBJECTS. ACTIVE CONVERSATIONS ARE ONGOING WITH FOUR ADDITIONAL DATA CONTRIBUTORS. (5) LAUNCHED WORKING GROUP ACTIVITIES WITH FOCUS ON CONTEXT-OF-USE, CRITERIA FOR REGULATORY READINESS OF BIOMARKERS, AND AN IMMEDIATE ACTION PLAN TO SEEK REGULATORY FEEDBACK ON CLINICAL OUTCOME ASSESSMENTS CURRENTLY IN DEVELOPMENT. |
| FORM 990, PART III, LINE 4A | 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'S CLINICAL PHARMACOLOGY WORKGROUP DEVELOPED A WHITE PAPER TO ASSIST REGULATORS IN PREPARING GUIDANCE ON THE CLINICAL PHARMACOLOGY CONSIDERATIONS FOR NEONATES, INCLUDING RECOMMENDATIONS FOR SAFER FORMULATIONS ENCOMPASSING EASE OF ADMINISTRATION. INC'S BRONCHOPULMONARY DYSPLASIA (BPD) WORKGROUP DESCRIBED POTENTIAL ENDPOINTS FOR TRIALS TO EVALUATE TREATMENTS FOR CHRONIC PULMONARY INSUFFICIENCY OF PREMATURITY (CPIP). INC'S SEIZURE WORKGROUP DEVELOPED RECOMMENDATIONS FOR USE IN A MASTER PROTOCOL TO EVALUATE MEDICATIONS TO TREAT NEONATAL SEIZURES. INC'S DATA WORKGROUP REVIEWED THE VALUE AND NEEDS FOR SHARING NEONATAL DATA AND PROPOSED A GLOBAL APPROACH TO COLLECTING, STANDARDIZING, AND SHARING DATA. INC'S CLINICAL PHARMACOLOGY ADVERSE EVENT SUBGROUP COMPLETED DEVELOPMENT OF THE NEONATAL ADVERSE EVENT SEVERITY GRADING SCALE (NAESS) AND HAVE INCORPORATED THE NEW AE TERMINOLOGY INTO THE NATIONAL CANCER INSTITUTE - ENTERPRISE VOCABULARY SERVICES (NCI-EVS) ONLINE THESAURUS. INC SUBMITTED COMMENTS ON THE NOTICE OF PROPOSED RULEMAKING (NPRM) "FEDERAL POLICY FOR THE PROTECTION OF HUMAN SUBJECTS" TO U.S. HEALTH AND HUMAN SERVICES' OFFICE FOR HUMAN RESEARCH PROTECTIONS AND ALSO SUPPLIED INPUT ON DRUGS AND THERAPEUTIC AREAS THAT REQUIRE FURTHER STUDY IN NEONATES, IN RESPONSE TO NATIONAL INSTITUTE OF CHILD HEALTH AND DEVELOPMENT'S REQUEST FOR INFORMATION ON THE BEST PHARMACEUTICALS FOR CHILDREN ACT PRIORITIES IN PEDIATRIC THERAPEUTICS 2017. THE MULTIPLE SCLEROSIS OUTCOME ASSESSMENTS CONSORTIUM 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) EXECUTED THE STATISTICAL ANALYSIS PLAN (FOR A SINGLE MEASURE) DEVELOPED FOR FDA AND SUBMITTED THE FINAL QUALIFICATION PACKAGE. (2) EXECUTED THE STATISTICAL ANALYSIS PLAN (INCLUDING FOUR MEASURES) DEVELOPED FOR EMA AND THE "VOICE OF THE PATIENT" METHODOLOGY AND SUBMITTED THE FINAL QUALIFICATION PACKAGE TO THE EMA. 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. 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. 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. TWO PRO MEASURES WERE QUALIFIED BY FDA FOR EXPLORATORY USE, THE SYMPTOMS OF MAJOR DEPRESSIVE DISORDER SCALE (SMDDS) AND THE NON-SMALL CELL LUNG CANCER SYMPTOM ASSESSMENT QUESTIONNAIRE (NSCLC-SAQ). OTHER PRO MEASURES WHICH HAVE BEEN SUBMITTED TO FDA FOR QUALIFICATION FOR EXPLORATORY USE INCLUDE: (1) THE ASTHMA DAILY SYMPTOM DIARY (ADSD) AND (2) THE FUNCTIONAL DYSPEPSIA SYMPTOM DIARY (FDSD). THE PRO CONSORTIUM HAS ISSUED A TOTAL OF 10 LICENSES FOR USE OF PRO CONSORTIUM MEASURES INCLUDING THE SMDDS, THE NSCLCSAQ, AND THE MYELOFIBROSIS SYMPTOM ASSESSMENT FORM V4.0 (MFSAF V4.0). 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. THIS YEAR THE CONSORTIUM'S ACCOMPLISHMENTS INCLUDE: (1) IN COOPERATION WITH THE FDA, LAUNCHED THE PILOT BIOMARKER DATA REPOSITORY (BMDR) PROJECT DESIGNED TO COLLECT AND ANALYZE KIDNEY SAFETY BIOMARKER DATA. (2) RECEIVED A LETTER OF SUPPORT FROM EMA FOR THE USE OF GLUTAMATE DEHYDROGENASE (GLDH), A SPECIFIC BIOMARKER OF DRUG-INDUCED HEPATIC INJURY IN HUMANS. (3) ACHIEVED FDA QUALIFICATION OF A PANEL OF CLINICAL DRUG-INDUCED KIDNEY INJURY BIOMARKERS FOR USE IN PHASE 1 CLINICAL TRIALS. 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 T1D SECURED THE FOLLOWING DATA SETS AND IS CURRENTLY WORKING TO BUILD AN INTEGRATED DATABASE TO SUPPORT QUALIFICATION: THE COLORADO DIABETES AUTOIMMUNITY STUDY IN THE YOUNG (DAISY), THE ENVIRONMENTAL DETERMINANTS OF DIABETES IN THE YOUNG (TEDDY), TRIALNET NATURAL HISTORY STUDY (TN01-NH), THE GERMAN BABYDIAB AND BABYDIET STUDIES, AND THE DIABETES PREVENTION TRIAL (DPT-1). THE CONSORTIUM SUBMITTED A LETTER OF INTENT TO FDA AND RECEIVED A POSITIVE RESPONSE TO THE LETTER OF INTENT FROM THE FDA AND WAS ASKED TO PROCEED TO THE NEXT STEP IN THE QUALIFICATION PROCESS: THE DEVELOPMENT OF THE QUALIFICATION PLAN. THE CONSORTIUM IS NOW WORKING ON THE DRAFTING OF THE QUALIFICATION PLAN FOCUSING, IN PART, ON THE BUILDING OF AN ASSAY PUBLICATION LIBRARY AND REVIEWING THE ASSAY METHODOLOGY USED FOR ALL DATA TO COMPLETE THE ANALYTICAL CONSIDERATIONS SECTION OF THE DOCUMENT. THE CONSORTIUM HAS INITIATED CONSTRUCTION OF A MODELING ANALYSIS PLAN TO ENABLE 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. THIS YEAR THE TTC INITIATED TWO WORKGROUPS, THE ENDPOINTS AND BIOMARKERS WORKGROUP, AND THE DRUG SAFETY PROFILE CHARACTERIZATION AND LABELING WORKGROUP. THE INITIAL FOCUS OF THE CONSORTIUM IS TO SEEK FDA ENDORSEMENT FOR A QUANTITATIVE DRUG DEVELOPMENT TOOL CAPABLE OF OPTIMIZING CLINICAL TRIAL DESIGNS IN KIDNEY TRANSPLANTATION, BY INFORMING SPONSOR DECISION MAKING REGARDING CLINICAL TRIAL DESIGN, SIZE, AND DURATION, OPTIMIZE DOSING STRATEGIES, PREDICT SAFETY SIGNALS, SUPPORT EVIDENCE OF EFFECTIVENESS, AND ENDPOINT SELECTION. THROUGH FDA'S BROAD AGENCY ANNOUNCEMENT PROCEDURE, TTC RECEIVED FUNDING TO SUPPORT THE INITIAL FOCUS AREA OF DEVELOPING A MODEL. |
| 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. 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 COMPTROLLER, 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. 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 1,404,700. MANAGEMENT AND GENERAL EXPENSES 0. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 1,404,700. CONSULTING: PROGRAM SERVICE EXPENSES 836,851. MANAGEMENT AND GENERAL EXPENSES 68,397. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 905,248. OTHER PROFESSIONAL FEES: PROGRAM SERVICE EXPENSES 118,245. MANAGEMENT AND GENERAL EXPENSES 7,976. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 126,221. |
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