Current Research Agenda

Benchmarking the Drug Development Process

  • Assessments by therapeutic class and molecule type
  • Discovery and non-clinical development metrics
  • Regulatory review metrics
  • Return on investment for oncology drugs
  • Biotechnology pipeline: development metrics and trends
  • Vaccines development metrics
  • Precision/personalized medicine, specialty pharma development
  • R&D costs and returns with single-source contract manufacturing
  • Global landscape for new active substances (NASs)
  • Personnel workload and capacity

Improving R&D Efficiency and Productivity

  • Clinical trial operations: start-up procedures, cycle times, best practices
  • Protocol design complexity
  • Investigator site selection and site management practices
  • Study conduct cycle times, patient recruitment and retention rates
  • Pharmacovigilance operations and strategies*
  • Contract service providers
  • Adaptive trial designs
  • Application of comparative oncology
  • Pediatric studies of oncology drugs in development for adults
  • Assessment of end-to-end R&D platforms
  • Adoption and impact of patient engagement initiatives*
  • Real World Evidence (RWE) use in clinical trials*
  • Innovative models to grow awareness, participation of minority populations in clinical trials

Regulatory and Policy Initiatives to Foster Bioinnovation and Patient Access

  • FDA expedited development & review programs (BTD, PRV, Fast Track, Accel Approval)
  • Orphan product designation, R&D challenges, and approval
  • Real World Evidence (RWE) use for Value-Based Pricing (VBP) in global emerging markets
  • Patient access and reimbursement for orphan drugs
  • PDUFA VI and 21st Century Cures Act impact on innovation and the regulatory environment
  • Challenges and solutions in pediatric study compliance
  • Medical countermeasures and the emerging sponsor landscape
  • Harmonization priorities for FDA, EMA, and PMDA