Attach to Form 990 or Form 990-EZ.
Information about Schedule A (Form 990 or 990-EZ) and its instructions is at www.irs.gov/form990.
| (i)Name of supported organization | (ii) EIN | (iii) Type of organization (described on lines 1- 9 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 | ||||||
Calendar year (or fiscal year beginning in) ![]() |
(a) 2011 | (b) 2012 | (c) 2013 | (d) 2014 | (e) 2015 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 1 | Gifts, grants, contributions, and membership fees received. (Do not include any unusual grants.) .... | 7,998,908 | 5,915,630 | 5,370,782 | 10,269,811 | 10,197,464 | 39,752,595 |
| 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 | 7,998,908 | 5,915,630 | 5,370,782 | 10,269,811 | 10,197,464 | 39,752,595 |
| 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).. | 12,673,465 | |||||
| 6 | Public support. Subtract line 5 from line 4. | 27,079,130 | |||||
Calendar year
(or fiscal year beginning in) ![]() |
(a) 2011 | (b) 2012 | (c) 2013 | (d) 2014 | (e) 2015 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 7 | Amounts from line 4.. | 7,998,908 | 5,915,630 | 5,370,782 | 10,269,811 | 10,197,464 | 39,752,595 |
| 8 | Gross income from interest, dividends, payments received on securities loans, rents, royalties and income from similar sources... | 22,660 | 21,279 | 70,230 | 82,059 | 80,580 | 276,808 |
| 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.).. | 20,745 | 20,745 | ||||
| 11 | Total support. Add lines 7 through 10. | 40,050,148 | |||||
Calendar year (or fiscal year beginning in) ![]() |
(a) 2011 | (b) 2012 | (c) 2013 | (d) 2014 | (e) 2015 | (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) 2011 | (b) 2012 | (c) 2013 | (d) 2014 | (e) 2015 | (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 2015 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-2015 |
(iii) Distributable Amount for 2015 |
|
|---|---|---|---|---|
|
1
Distributable amount for 2015 from Section C, line 6 |
||||
|
2
Underdistributions, if any, for years prior to 2015 (reasonable cause required--see instructions) |
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| 3 Excess distributions carryover, if any, to 2015: | ||||
| a | ||||
| b | ||||
| c | ||||
| d From 2013....... | ||||
| e From 2014....... | ||||
| fTotal of lines 3a through e | ||||
| g Applied to underdistributions of prior years | ||||
| h Applied to 2015 distributable amount | ||||
|
i
Carryover from 2010 not applied (see instructions) |
||||
| j Remainder. Subtract lines 3g, 3h, and 3i from 3f. | ||||
| 4Distributions for 2015 from Section D, line 7: | ||||
| $ | ||||
| a Applied to underdistributions of prior years | ||||
| b Applied to 2015 distributable amount | ||||
| c Remainder. Subtract lines 4a and 4b from 4. | ||||
|
5
Remaining underdistributions for years prior to 2015, if any. Subtract lines 3g and 4a from line 2 (if amount greater than zero, see instructions) |
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|
6
Remaining underdistributions for 2015. Subtract lines 3h and 4b from line 1 (if amount greater than zero, see instructions) |
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|
7 Excess distributions carryover to 2016. Add lines 3j and 4c. |
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| 8 Breakdown of line 7: | ||||
| a | ||||
| b | ||||
| c Excess from 2013....... | ||||
| d From 2014....... | ||||
| e From 2015....... | ||||
| Facts And Circumstances Test |
|---|
| Return Reference | Explanation |
|---|
| Software ID: | |
| Software Version: |
Attach to Form 990 or 990-EZ.
Information about Schedule O (Form 990 or 990-EZ) and its instructions is at| 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 IMPLEMENTING THE FDA'S CRITICAL PATH INITIATIVE BY CREATING COLLABORATIONS AMONG REGULATORS (SCIENTISTS FROM THE FDA AND EMA) THE REGULATED (MEDICAL PRODUCT INDUSTRY), ACADEMIA, PATIENTS ORGANIZATIONS, AND OTHER ORGANIZATIONS THAT ADVANCE MEDICAL INNOVATION THROUGH THE DEVELOPMENT OF TOOLS AND METHODS BASED UPON SOUND, CONSENSUS BASED SCIENCE. THE ACCOMPLISHMENTS FOR THIS FISCAL YEAR REPORT INCLUDE: THE COALITION AGAINST MAJOR DISEASES DEVELOPS TOOLS AND PROCESSES TO ACCELERATE PROGRESS IN THE PREVENTION, TREATMENT AND CURE OF MAJOR NEURODEGENERATIVE DISEASES, INITIALLY FOCUSING ON ALZHEIMER'S AND PARKINSON'S DISEASES. THIS YEAR'S ACCOMPLISHMENTS INCLUDE: DEVELOPED AN INFORMED CONSENT FORM THAT WILL ENABLE BROADER SHARING OF PATIENT DATA AND SPECIMENS FOR FUTURE RESEARCH. OBTAINED REGULATORY ENDORSEMENT OF THE STATISTICAL ANALYSIS APPROACH FOR THE HIPPOCAMPAL VOLUME BIOMARKER QUALIFICATION EFFORT. RECEIVED THREE LETTERS OF SUPPORT FROM THE FDA ENCOURAGING THE FURTHER STUDY AND USE OF CEREBROSPINAL FLUID ANALYTES AB1-42, TOTAL-TAU, AND PHOSPHO-TAU, AND LOW BASELINE HIPPOCAMPAL VOLUME MEASURED BY MAGNETIC RESONANCE IMAGING AS EXPLORATORY PROGNOSTIC BIOMARKERS FOR ENRICHMENT IN TRIALS FOR AD; AND MOLECULAR NEUROIMAGING OF THE DOPAMINE TRANSPORTER AS AN EXPLORATORY PROGNOSTIC BIOMARKER FOR ENRICHMENT IN TRIALS FOR PARKINSON'S DISEASE. THE COALITION FOR ACCELERATING STANDARDS AND THERAPIES IS AN INITIATIVE TO ACCELERATE CLINICAL RESEARCH AND MEDICAL PRODUCT DEVELOPMENT BY CREATING AND MAINTAINING DATA STANDARDS, TOOLS, AND METHODS FOR CONDUCTING RESEARCH IN THERAPEUTIC AREAS THAT ARE IMPORTANT TO PUBLIC HEALTH. CFAST WAS INITIATED AS A PARTNERSHIP BETWEEN THE CLINICAL DATA INTERCHANGE STANDARDS CONSORTIUM (CDISC) AND THE CRITICAL PATH INSTITUTE (C-PATH). THE COALITION HAS DEVELOPED AND PUBLISHED THE FOLLOWING (22) CDISC THERAPEUTIC AREA STANDARDS: ALZHEIMER'S V1 AND 2, ASTHMA, BREAST CANCER, CARDIOVASCULAR, CHRONIC HEPATITIS C, DIABETES, DYSLIPIDEMIA, INFLUENZA, MULTIPLE SCLEROSIS, PAIN, PARKINSON'S DISEASE, POLYCYSTIC KIDNEY DISEASE, QT STUDIES, SCHIZOPHRENIA, TUBERCULOSIS V1 AND V2, VIROLOGY V1 AND V2, TRAUMATIC BRAIN INJURY, AND CHRONIC OBSTRUCTIVE PULMONARY DISEASE (COPD). A SEPARATE ANALYSIS DATA MODEL (ADAM) SUPPLEMENT WAS PUBLISHED FOR DIABETES. A NEW CONSORTIUM ENTITLED CRITICAL PATH FOR PARKINSON'S WAS LAUNCHED. ITS GOALS ARE TO DEVELOP QUANTITATIVE MODEL-BASED TOOLS TO ACCELERATE THE TREATMENT OF PARKINSON'S DISEASE, TARGETING PATIENTS AT THE FIRST CLINICAL SIGNS OF MOTOR SYMPTOM ONSET. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: RECEIVED SUPPORT FROM EMA TO PROCEED WITH QUALIFICATION PLAN FOR MOLECULAR NEUROIMAGING OF THE DOPAMINE TRANSPORTER AS A PROGNOSTIC BIOMARKER FOR ENRICHMENT IN TRIALS FOR EARLY PARKINSON'S DISEASE. RECEIVED A LETTER OF SUPPORT FROM THE EMA FOR THE USE OF DAT IMAGING AS AN ENRICHMENT BIOMARKER FOR PD CLINICAL TRIALS. THE MISSION OF THE CRITICAL PATH TO TB DRUG REGIMENS INITIATIVE IS TO ACCELERATE THE DEVELOPMENT OF NOVEL TB DRUG REGIMENS THAT ARE SAFER, SHORTER IN DURATION, AND MORE EFFICACIOUS THAN THE CURRENT STANDARD OF CARE. THEIR MISSION EXPANDED TO ALSO ACCELERATE THE DEVELOPMENT OF A CLINICALLY USEFUL IN VITRO RAPID DRUG SUSCEPTIBILITY ASSAY TO SUPPORT TB REGIMEN DEVELOPMENT AND DEPLOYMENT. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: THE EMA ISSUED A POSITIVE QUALIFICATION DECISION ON CPTR'S QUALIFIED IN VITRO HOLLOW FIBER SYSTEM MODEL FOR TUBERCULOSIS. THE WHO SELECTED C-PATH TO HOST TB CLINICAL TRIALS DATA PLATFORM. THE RESEQTB DATA PLATFORM LAUNCHED FOR USE BY RESEARCHERS, DATA CONTRIBUTORS AND CONSORTIA MEMBERS. TB-PLATFORM FOR AGGREGATION OF CLINICAL TB STUDIES LAUNCHED. DELIVERED A QUANTITATIVE MODEL FOR TIME-TO-POSITIVITY BEHAVIOR USING AN INTEGRATED DATABASE OF PHASE II STUDIES. MANUFACTURED NEW SENSITITRE PLATE CONTAINING CURRENT, NEW, AND REPURPOSED ANTI-TB DRUGS AND SHIPPED TO MULTIPLE SITES FOR ASSAY VALIDATION. COMPLETED 2 OF 7 MODELING AND SIMULATION TOOLS (PHARMACOLOGY BASED PHARMACOKINETICS AND QT INTERVAL MODELS). SUBMITTED COMMENTS TO FDA DRAFT GUIDANCE FOR INDUSTRY ON INFECTIOUS DISEASE NEXT GENERATION SEQUENCING BASED DIAGNOSTIC DEVICES: MICROBIAL IDENTIFICATION AND DETECTION OF ANTIMICROBIAL RESISTANCE AND VIRULENCE MARKERS. THE DATA COLLABORATION CENTER IS A NEWLY ESTABLISHED CENTER WITHIN C-PATH TO ENABLE MULTIPLE ORGANIZATIONS TO WORK TOGETHER IN A NEUTRAL SETTING AND SHARE CLINICAL DATA IN ORDER TO OPTIMIZE ITS VALUE IN CREATING NEW INSIGHTS AND TOOLS THAT ACCELERATE DRUG DEVELOPMENT IN AREAS WITH UNMET MEDICAL NEEDS. THE DCC SUPPORTS DATA SHARING PROJECTS ALIGNED WITH SPECIFIC C-PATH CONSORTIA AS WELL AS DATA SHARING INITIATIVES THAT ARE INDEPENDENT OF C-PATH CONSORTIA. ITS ACCOMPLISHMENTS THIS YEAR INCLUDE: PROVIDED DATA MANAGEMENT, CURATION, STANDARDIZATION, AND DATA SHARING SUPPORT FOR THE FOLLOWING C-PATH DATA PROJECTS: RELATIONAL SEQUENCING TB DATA PLATFORM, A DATA SHARING PLATFORM TO ADVANCE THE DEVELOPMENT OF TB RAPID DRUG SUSCEPTIBILITY TESTS; TB PLATFORM FOR AGGREGATION OF CLINICAL TRIAL STUDIES, A DATABASE TO AGGREGATE PHASE III TB CLINICAL TRIAL DATA SETS AND MAKE AVAILABLE TO RESEARCHERS FOR THE WORLD HEALTH ORGANIZATION; TB-ALLIANCE, AN AGGREGATED COLLECTION OF TB CLINICAL TRIAL DATA SETS THAT THE TB-ALLIANCE ORGANIZATION IS MAKING AVAILABLE TO QUALIFIED RESEARCHERS; SIX OF C-PATH'S CONSORTIA WITH DATABASE REQUIREMENTS. THE DCC ALSO COMPLETED ITS BUSINESS PLAN. A NEW CONSORTIUM ENTITLED DUCHENNE REGULATORY SCIENCE CONSORTIUM WAS LAUNCHED. ITS GOAL IS TO SUPPORT COLLABORATIVE RESEARCH AND REGULATORY QUALIFICATION OF NEW DRUG DEVELOPMENT TOOLS FOR DUCHENNE MUSCULAR DYSTROPHY, TO ENABLE THE EARLIEST POSSIBLE PATIENT ACCESS TO NEW TREATMENTS. SPECIFIC GOALS INCLUDE DEVELOPING A CLINICAL DATA SHARING PLATFORM, A CLINICAL DATA INTERCHANGE STANDARDS CONSORTIUM (CDISC) THERAPEUTIC AREA STANDARD, AND A DISEASE PROGRESSION MODEL FOR DUCHENNE. ACCOMPLISHMENTS THIS YEAR INCLUDE: HELD FIRST EXPERT MEETING IN NOVEMBER 2015 TO DISCUSS THE ELEMENTS NEEDED TO BE INCLUDED IN THE MODEL AND TO PRIORITIZE DATASETS. DEVELOPED AN INITIAL WORK PLAN FOR THE PROGRESSION MODEL, FOR FURTHER DISCUSSION WITH THE REGULATORY AUTHORITIES AND TO FOCUS DATA COLLECTION. ACQUIRED FOUR DATASETS AND DATA SHARING AGREEMENTS FOR ADDITIONAL DATASETS ARE UNDERWAY. THE GOAL OF THE ELECTRONIC PATIENT REPORTED OUTCOME CONSORTIUM IS TO ADVANCE THE SCIENCE OF CLINICAL TRIAL ENDPOINT ASSESSMENT BY COLLABORATIVELY SUPPORTING AND CONDUCTING RESEARCH, DESIGNING AND DELIVERING EDUCATIONAL OPPORTUNITIES, AND DEVELOPING AND DISSEMINATING BEST PRACTICE RECOMMENDATIONS FOR ELECTRONIC COLLECTION OF CLINICAL OUTCOME DATA. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: PARTICIPATED IN GCP INSPECTORS WORKING GROUP AND INTERESTED PARTIES JOINT MEETING AT THE EMA AND PRESENTED THE CONSORTIUM'S POSITION ON SOURCE DATA AND BYOD. COLLABORATED IN THE DEVELOPMENT OF A STUDY DESIGN TO SUPPORT A PROJECT TITLED "COMPARABILITY OF PROVISIONED DEVICE VS BRING-YOUR-OWN-DEVICE IN SUBJECTS WITH COPD." SEVEN NEW PRO INSTRUMENTS HAVE BEEN IMPLEMENTED ELECTRONICALLY. LAUNCHED A STUDY TO PROVIDE EMPIRICAL EVIDENCE TO SUPPORT THE MEASUREMENT EQUIVALENCE OF DATA COLLECTED ON VARIOUS DATA COLLECTION MODES (I.E., PAPER, HANDHELD, TABLET, INTERACTIVE VOICE RESPONSE [IVR], AND WEB). THE QUALITATIVE COMPONENT OF THE STUDY HAS BEEN COMPLETED AND THE QUANTITATIVE PORTION IS UNDERWAY. THE MULTIPLE SCLEROSIS OUTCOME ASSESSMENT CONSORTIUM HAS A GOAL TO OBTAIN REGULATORY QUALIFICATION OF AN IMPROVED CLINICAL OUTCOME ASSESSMENT INSTRUMENT AS A PRIMARY ENDPOINT IN CLINICAL TRIALS OF MULTIPLE SCLEROSIS (MS) THERAPIES. THEIR ACCOMPLISHMENTS INCLUDE: DEVELOPED A CLINICAL DATA INTERCHANGE STANDARDS CONSORTIUM (CDISC) THERAPEUTIC AREA USER GUIDE FOR MS. ACQUIRED THE CONTROL AND TREATMENT ARMS OF 16 CLINICAL TRIAL DATASETS (14,430 PATIENT RECORDS), CURATED THE DATA, AND REMAPPED AND POOLED MOST OF THE DATA TO THE CDISC STUDY DATA TABULATION MODEL (SDTM) STANDARD. RECEIVED FEEDBACK ON THE INITIAL BRIEFING PACKAGE FROM THE U.S. FOOD AND DRUG ADMINISTRATION (FDA) AND EUROPEAN MEDICINES AGENCY (EMA). COMPLETED AN EXTENSIVE LITERATURE REVIEW. THE STATISTICS ANALYSIS PLAN WAS FINALIZED AND EXECUTED FOR THE FDA SUBMISSION. ESTABLISHED THE MSOAC PLACEBO DATABASE FOR USE IN APPROVED MS RESEARCH. WORK IS PROCEEDING ON SINGLE DOMAIN OF DISABILITY FOR THE FDA (I.E., COGNITION), WHILE MAINTAINING THE ORIGINAL FOCUS FOR THE EMA. |
| FORM 990, PART III, LINE 4A | THE GOAL OF THE INTERNATIONAL NEONATAL CONSORTIUM IS TO ACCELERATE THE DEVELOPMENT OF SAFE AND EFFECTIVE THERAPIES FOR NEONATES. IT ENGAGES THE GLOBAL NEONATAL COMMUNITY - FAMILIES, NEONATAL NURSES, ACADEMIC SCIENTISTS, REGULATORS, PHARMACEUTICAL INVESTIGATORS, ADVOCACY ORGANIZATIONS, AND FUNDERS - TO FOCUS ON THE NEEDS OF THE NEONATE. THROUGH TEAMS THAT SHARE DATA, KNOWLEDGE, AND EXPERTISE, THE CONSORTIUM WILL DEVELOP TOOLS THAT CAN BE INCORPORATED INTO CLINICAL TRIALS FOR NEONATES. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: HELD A FACE-TO-FACE WORKSHOP IN WASHINGTON, DC, ON TO DRAFT A WHITE PAPER REGARDING CONSENSUS-BASED CRITICAL ELEMENTS OF CLINICAL PHARMACOLOGY STUDIES IN NEONATES WHICH COULD BE CONSIDERED BY REGULATORS IN PREPARATION OF FUTURE GUIDANCE DOCUMENTS, INCLUDING RECOMMENDATIONS FOR SAFER FORMULATIONS ENCOMPASSING EASE OF ADMINISTRATION. SUBMITTED COMMENTS ON THE NOTICE OF PROPOSED RULEMAKING (NPRM) "FEDERAL POLICY FOR THE PROTECTION OF HUMAN SUBJECTS" TO THE OFFICE FOR HUMAN RESEARCH PROTECTIONS AND SUPPLIED INPUT ON DRUGS AND THERAPEUTIC AREAS THAT REQUIRE FURTHER STUDY IN NEONATES IN RESPONSE TO NICHD'S REQUEST FOR INFORMATION ON THE BEST PHARMACEUTICALS FOR CHILDREN ACT PRIORITIES IN PEDIATRIC THERAPEUTICS 2017. THE GOAL OF THE POLYCYSTIC KIDNEY DISEASE OUTCOMES CONSORTIUM IS TO DEVELOP AND OBTAIN REGULATORY QUALIFICATION FROM THE U.S. FOOD AND DRUG ADMINISTRATION (FDA) AND THE EUROPEAN MEDICINES AGENCY (EMA) OF TOTAL KIDNEY VOLUME (TKV) AS A PROGNOSTIC BIOMARKER FOR USE IN CLINICAL TRIALS FOR NEW THERAPIES FOR AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE (ADPKD). AFTER SUCCESSFULLY ACCOMPLISHING THE PRIMARY GOAL, THE CONSORTIUM IS NOW WORKING ON OTHER REGULATORY CHALLENGES, CLINICAL TRIAL DESIGNS, EXPLORATORY ENDPOINTS, AND THE POSSIBILITY OF QUALIFYING TKV AS A SURROGATE ENDPOINT. THEIR ACCOMPLISHMENTS FOR THIS YEAR INCLUDE: RECEIVED A LETTER OF SUPPORT FROM THE FDA FOR THE EXPLORATORY USE OF TKV IN CLINICAL TRIALS FOR ADPKD. RECEIVED A FORMAL QUALIFICATION OF TKV AS A PROGNOSTIC BIOMARKER FROM THE FDA IN THE FORM OF A DRAFT GUIDANCE (AUGUST, 2015) AND FROM THE EMA AS A QUALIFICATION OPINION (NOVEMBER, 2015). HELD A VERY SUCCESSFUL ADPKD SUMMIT MEETING WITH INDUSTRY, ACADEMIA, AND INTERNATIONAL REGULATORS TO DISCUSS POTENTIAL SOLUTIONS TO ADDRESS THE NEED FOR BETTER CLINICAL ENDPOINTS FOR CLINICAL TRIALS OF NEW THERAPIES FOR ADPKD. FORMAL QUALIFICATION OF TKV AS A PROGNOSTIC BIOMARKER WAS RECEIVED FROM THE FDA IN THE FORM OF FINAL GUIDANCE ON SEPTEMBER 15TH, 2016. TKV WAS ACCEPTED AS A "REASONABLY LIKELY SURROGATE" BY THE FDA ALLOWING IT TO BE USED IN ACCELERATED APPROVAL APPLICATIONS. THE GOAL OF THE PATIENT-REPORTED OUTCOME CONSORTIUM IS TO ESTABLISH AND MAINTAIN A COLLABORATIVE FRAMEWORK WITH APPROPRIATE STAKEHOLDERS FOR THE QUALIFICATION OF PATIENT-REPORTED OUTCOME (PRO) INSTRUMENTS AND OTHER CLINICAL OUTCOME ASSESSMENT (COA) TOOLS THAT WILL BE PUBLICLY AVAILABLE FOR USE IN CLINICAL TRIALS WHERE COA-BASED ENDPOINTS ARE USED TO SUPPORT PRODUCT LABELING CLAIMS. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: THE FOLLOWING PRO INSTRUMENTS ARE IN VARIOUS STAGES OF THE QUALIFICATION PROCESS: FOR THE ASTHMA DAILY SYMPTOM DIARY, A QUANTITATIVE PILOT STUDY WAS COMPLETED AND QUANTITATIVE PILOT STUDY REPORT AND QUANTITATIVE DATA SUBMITTED TO FDA QRT FOR REVIEW. FOR THE SYMPTOMS OF MAJOR DEPRESSIVE DISORDER SCALE, A QUANTITATIVE PILOT STUDY WAS COMPLETED AND QUANTITATIVE PILOT STUDY REPORT AND QUANTITATIVE DATA WAS SUBMITTED TO FDA FOR REVIEW. FOR THE FUNCTIONAL DYSPEPSIA SYMPTOM DIARY, THE QUALITATIVE RESEARCH WAS COMPLETED AND QUALITATIVE RESEARCH SUMMARY REPORT IS IN DEVELOPMENT. FOR THE THREE INSTRUMENTS FOR IRRITABLE BOWEL SYNDROME, THE QUANTITATIVE PILOT STUDY PROTOCOL AND ANALYSIS PLAN WAS REVIEWED BY FDA, THEIR SUGGESTIONS WERE INCORPORATED INTO THE STUDY DOCUMENTS, AND A QUANTITATIVE PILOT STUDY IS BEING LAUNCHED. FOR THE NON-SMALL CELL LUNG CANCER SYMPTOM ASSESSMENT QUESTIONNAIRE, THE QUANTITATIVE PILOT STUDY WAS COMPLETED AND THE QUANTITATIVE PILOT STUDY REPORT IS IN DEVELOPMENT. THE PREDICTIVE SAFETY TESTING CONSORTIUM BRINGS TOGETHER PHARMACEUTICAL COMPANIES TO SHARE AND VALIDATE INNOVATIVE SAFETY TESTING METHODS UNDER ADVISEMENT OF THE U.S. FOOD AND DRUG ADMINISTRATION, EUROPEAN MEDICINES AGENCY, AND JAPAN'S PHARMACEUTICALS AND MEDICAL DEVICES AGENCY. CURRENTLY THE PSTC IS FOCUSED ON DEVELOPING AND OBTAINING REGULATORY QUALIFICATION OF IMPROVED CLINICAL SAFETY BIOMARKERS FOR USE IN DRUG DEVELOPMENT. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: RECEIVED EMA AND FDA LETTERS OF SUPPORT FOR FOUR SKELETAL MUSCLE INJURY BIOMARKERS IN RODENTS. SUBMITTED LIMITED CONTEXT OF USE QUALIFICATION PACKAGE TO FDA AND EMA OF A COMPOSITE MEASURE OF KIDNEY SAFETY BIOMARKERS FOR USE IN CLINICAL DRUG DEVELOPMENT STUDIES. SUBMITTED LETTER OF INTENT FOR THE QUALIFICATION OF GLUTAMATE DEHYDROGENASE AS A SPECIFIC BIOMARKER OF DRUG-INDUCED HEPATIC INJURY IN HUMANS. SUBMITTED PACKAGES FOR DILI, DIKI, AND DIVI BIOMARKERS TO EMA AND FDA FOR REVIEW WITH THE INTENT OF RECEIVING LETTERS OF SUPPORT. RECEIVED FDA LETTERS OF SUPPORT FOR LIVER SAFETY BIOMARKERS FOR USE IN CLINICAL DRUG DEVELOPMENT. A NEW CONSORTIUM ENTITLED PEDIATRICS TRIAL CONSORTIUM WAS LAUNCHED THIS YEAR. THE GOAL IS TO PROVIDE THE SUPPORT AND GUIDANCE NECESSARY FOR THE CRITICAL PATH INSTITUTE (C-PATH) TO CREATE A NEW NON-PROFIT ORGANIZATION THAT WILL WORK INDEPENDENTLY (BUT WITH INPUT FROM RELEVANT SECTORS) TO ADDRESS THE GAPS IN PEDIATRIC CLINICAL DEVELOPMENT. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: LAUNCHED A NEW INDEPENDENT ORGANIZATION CALLED INSTITUTE FOR ADVANCED CLINICAL TRIALS FOR CHILDREN. APPLIED FOR A 501(C)(3) NON-PROFIT STATUS THAT IS PENDING. INITIAL DIRECTORS AND OFFICERS HAVE BEEN APPOINTED AND BYLAWS HAVE BEEN ADOPTED. DEVELOPED AN OPERATIONAL PLAN ADDRESSING: GLOBAL INTEROPERABILITY WITH OTHER INITIATIVES; RECOMMENDATIONS ON LEADERSHIP ROLES; FUNDING STRATEGY. WROTE AND HAD EXTENSIVE LEGAL REVIEW ON AN ADVISORY REPORT. |
| FORM 990, PART VI, SECTION A, LINE 2 | LYLE BOOTMAN AND SHAUN KIRKPATRICK HAVE A BUSINESS RELATIONSHIP. MIKE KASSER AND JEFF JACOB HAVE A BUSINESS RELATIONSHIP. ALAN LEVIN AND PETER CORR HAVE A BUSINESS RELATIONSHIP. |
| FORM 990, PART VI, SECTION B, LINE 11 | FORM 990 IS PREPARED BY AN EXTERNAL CPA FIRM USING INFORMATION PROVIDED BY THE ORGANIZATION. THE FORM IS REVIEWED BY THE ORGANIZATION'S COMPTROLLER, COO, PRESIDENT/CEO, AN OUTSIDE CPA, THE BOARD AUDIT, FINANCE, AND RISK COMMITTEE, AND THE BOARD OF DIRECTORS PRIOR TO BEING FINALIZED. |
| FORM 990, PART VI, SECTION B, LINE 12C | ALL MEMBERS OF THE BOARD OF DIRECTORS AND ALL OFFICERS OF THE ORGANIZATION ARE REQUIRED TO FILL OUT A CONFLICT OF INTEREST FORM ANNUALLY. THE RESULTS ARE COMPILED AND REVIEWED BY THE BOARD'S AUDIT, FINANCE, AND RISK COMMITTEE. ANY ACTUAL OR PERCEIVED CONFLICTS THAT HAVE THE POTENTIAL TO BIAS ANY DISCUSSIONS OR DECISIONS BY THE BOARD ARE DISCUSSED BY THE BOARD. ANY DIRECTOR WITH A REAL OR POTENTIAL CONFLICT WILL RECUSE THEMSELVES FROM ANY DISCUSSION OR DECISION WHERE THE CONFLICT HAS A BEARING. |
| FORM 990, PART VI, SECTION B, LINE 15 | A BOARD OF DIRECTORS COMPENSATION COMMITTEE MEETS AND REVIEWS EXECUTIVE COMPENSATION ANNUALLY. EVERY FEW YEARS THE COMMITTEE SEEKS AN INDEPENDENT COMPENSATION CONSULTING ORGANIZATION. AFTER THE CONSULTING ORGANIZATION HAS BEEN RETAINED, THE COMPENSATION COMMITTEE RECEIVES THE CONSULTANT'S EXECUTIVE COMPENSATION REVIEWS AND RECOMMENDATIONS. THIS INFORMATION IS THEN USED AS A BASIS FOR THE COMPENSATION COMMITTEE'S RECOMMENDATION TO THE BOARD OF DIRECTORS. THE LAST INDEPENDENT COMPENSATION SURVEY WAS CONDUCTED FOR THE CEO IN AUGUST 2011. THE CEO'S SALARY HAS REMAINED CONSTANT SINCE THE TIME OF HER HIRE. AN OVERALL SALARY PROGRAM FOR EMPLOYEES MUST BE APPROVED BY THE BOARD OF DIRECTORS COMPENSATION COMMITTEE BEFORE IMPLEMENTATION. ALL EMPLOYEE SALARY INCREASES MUST BE APPROVED BY THE PRESIDENT/CEO. |
| FORM 990, PART VI, SECTION C, LINE 19 | ARTICLES OF INCORPORATION CAN BE FOUND ON THE ARIZONA CORPORATION COMMISSION WEBSITE. THE CONFLICT OF INTEREST POLICY AND THE FINANCIAL STATEMENTS (VIA THE ANNUAL REPORT) ARE POSTED ON THE INSTITUTE'S WEBSITE. |
| FORM 990, PART IX, LINE 11G | RESEARCH SUPPORT SERVICES: PROGRAM SERVICE EXPENSES 1,405,309. MANAGEMENT AND GENERAL EXPENSES 0. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 1,405,309. CONSULTING: PROGRAM SERVICE EXPENSES 1,027,701. MANAGEMENT AND GENERAL EXPENSES 37,144. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 1,064,845. OTHER PROFESSIONAL FEES: PROGRAM SERVICE EXPENSES 924,300. MANAGEMENT AND GENERAL EXPENSES 13,390. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 937,690. |
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