Research Platforms and Current Research Agenda:

Benchmarking the Drug Development Process

  • Time, cost, and risk of bringing new drugs and biologics to market
  • Assessments by therapeutic class and molecule type
  • Discovery and non-clinical development metrics
  • Regulatory review metrics
  • Return on investment
  • Biotechnology pipeline: development metrics and trends
  • Vaccines development
  • Biosimilar and Biobetter development
  • Precision/personalized medicine, specialty pharma development
  • Operating function performance
  • Personnel workload and capacity

Improving Biopharmaceutical R&D Efficiency

  • Clinical trial operations - Industry best practices
  • Protocol design complexity
  • Investigator site selection and site management practices
  • Study conduct cycle times, patient recruitment and retention rates
  • Contract service providers
  • Adaptive trial designs
  • Strategic partnerships and integrated alliances
  • Application of Comparative Oncology
  • Pediatric studies of oncology drugs in development for adults
  • Patient-centricity and use of Big Data
  • Real World Evidence use in clinical trials
  • Public and private sector contributions to biopharmaceutical R&D
  • Innovative models to address awareness, participation in clinical trials among minority populations

Regulatory Initiatives to Foster Bio-innovation

  • FDA expedited development & review programs (BTD, PRV, Fast Track, Accel Approval)
  • Orphan product designation, R&D challenges, and approval
  • Impact of PDUFA VI, 21st Century Cures Act, and Trump agenda on regulatory environment
  • Addressing problems and solutions in pediatric study compliance
  • Medical countermeasures and the emerging sponsor landscape
  • Harmonization priorities for FDA, EMA, and PMDA
  • Pharmacovigilance and safety assessment

Pharmaceutical Value and Patient Access

  • Utilization and economic impact of comparative effectiveness research, cost-effectiveness, health technology assessment, and budget impact studies
  • International comparisons of pricing and reimbursement strategies and practices
  • Formulary management of cancer and orphan drugs
  • Impact of biotechnology products on health care quality and value
  • Patient access to abuse-deterrent formulations of opioid analgesics
  • Patient access to neglected disease medicines
  • Measuring innovation and value, and impact of reimbursement on innovation
  • Use of labeling and other prescribing information in the health care system