Development of Therapeutic Area-Specific Data Standards for Brain Diseases (S9.007)

OBJECTIVE: To improve the quality, efficiency and cost-effectiveness of clinical trials by developing brain disease-specific open data standards. BACKGROUND: Consensus-based clinical data standards serve two main purposes: integration of existing data, and consistency in prospective data collection. Critical Path Institute (C-Path), working with the Clinical Data Interchange Standards Consortium (CDISC), has played a leadership role in the development of data standards for neurodegenerative diseases, as well as in precompetitive data sharing for multiple disease areas. These therapeutic area-specific standards represent the preferred format for regulatory submissions of new drug applications. DESIGN/METHODS: Industry members, regulatory agencies, academics, and patient groups collectively developed data standards for Alzheimer’s disease (AD), Parkinson’s disease (PD), and multiple sclerosis (MS) in partnership with CDISC. Development is currently underway for traumatic brain injury (TBI). With input from clinical science experts (NINDS for PD, MS and TBI; ADNI for AD), developers mapped clinical concepts to the CDISC Study Data Tabulation Model (SDTM) and developed controlled terminology that supports development of standardized databases for research and regulatory submissions in these disease areas. RESULTS: CDISC therapeutic-area data standards implementation guides were developed for AD, PD and MS, as supplements to the CDISC SDTM, an FDA-recognized standard. The AD guide covers concepts including CSF biomarkers, ApoE genotype, volumetric MRI, amyloid PET imaging, and 10 clinical outcome assessment (COA) instruments, including ADAS-Cog, MMSE and CDR. The PD guide covers MRI, PET-SPECT, Deep Brain Stimulation, Neuropathology, and COAs, including both UPDRS and MDS-UPDRS. The MS guide covers disease course, relapse, multiple visual assessments, and 23 COAs. CONCLUSIONS: Use of consensus-based data standards maximizes efficiency in regulatory review and facilitates analyses across diverse studies. CDISC standards allow for integrating and pooling data in a platform-independent manner. Implementation of CDISC standards, particularly in the biomarkers arena, promises to facilitate improved efficiencies and harmonization in clinical trials. Study Supported by: Disclosure: Dr. Neville has nothing to disclose. Dr. Kopko has nothing to disclose. Dr. LeRoy has nothing to disclose. Dr. Gordon has received personal compensation for activities with Boehringer-Ingelheim as an employee. Dr. De Santi has received personal compensation for activities with Piramal Pharma as an employee. Dr. Jeromin has nothing to disclose. Dr. Mowry has received research support from Teva Neuroscience. Dr. Austi has received personal compensation for activities with Ixico PLC as an employee. Dr. Cole has received personal compensation for activities with Takeda Pharmaceutical Company as an employee. Dr. Marek has received personal compensation for activities with Molecular Neuroimaging, GE Healthcare, Piramal Group, Eli Lilly & Company, Merck & Co., Roche Diagnostics Corporation, Pronetha, Novartis, US WorldMeds, and nLife Therapeutics as a consultan Dr. Novak has received personal compensation for activities with Johnson & Johnson as an employee. Dr. Novak holds stock and/or stock options in Johnson & Johnson. Dr. Romero has nothing to disclose. Dr. Stafford has nothing to disclose. Dr. Hartley has nothing to disclose. Dr. Palmer has nothing to disclose. Dr. Facile has nothing to disclose. Dr. Chen has nothing to disclose. Dr. Fleisher has received personal compensation for activities with Eli Lilly & Company as an employee. Dr. Fleisher has received personal compensation for activities with Eli Lilly & Company, Avid Radiopharmaceuticals, Merck & Co., Inc., Grifols, Quintil Dr. Odenkirchen has nothing to disclose. Dr. Aviles has nothing to disclose. Acorda Therapeutics, Inc.; Biogen Idec; Novartis Pharmaceuticals Corp; Teva Neuroscience, Inc.; Genzyme; Sanofi; Celgene; NIH; NMSS Dr. Reiman has received personal compensation for activities with AstraZeneca, Bayer Pharmaceuticals Corporation, Esai Inc., Eli Lilly & Company, Novartis, Siemens, and Takeda Pharmaceutical Company as a scientific advisor. Dr. Manley has nothing to disclose. Dr. Hudson has nothing to disclose. Dr. Stephenson has nothing to disclose.