Consulting in All Areas of Clinical Development
For Biotech/Pharma Companies
- Independent medical monitoring of clinical trials, including unblinded data review
- Design of clinical studies meeting regulatory definition of AWC ("adequate and well controlled") study to demonstrate efficacy and safety
- Complex trial designs, including basket, umbrella, adaptive, SMART/SPCD, Bayesian/BOIN, multi-part, and platform trials
- Study Protocols, IBs, ICFs
- IRB/IEC submissions
- Safety Surveillance/Management Plans
- CSRs
- PIPs/PSPs
- TPPs
- CDPs, IDPs
- Primary/secondary endpoints, biomarker/surrogate endpoints, COAs/eCOAs, imaging charters, natural history and external controls, prognostic and predictive factors
- Use of RWD and RWE, including digitals/wearables
- Patient registries (safety/efficacy)
- Pre-approval/Expanded Access Programs
- INDs (COs, GIPs)
- NDAs (eCTD Modules 1, 2, and 5, including CSEs/ISEs and CSSs/ISSs)
- Clinical input for key Regulatory Agency interactions: e.g., Clinical Hold/Type A, EoP/Type B, WRO/Type C, START/Type D (pilot by FDA), INTERACT FDA meetings
- Advanced Regulatory Designations (including Orphan Drug, Rare Pediatric Disease, Fast Track, Breakthrough, RMAT, ATMP, etc)
- Safety/Efficacy White Papers, Clinical Impact Assessments
- DSURs, PBRERs (PSURs), IND Annual Reports (PADERs)
- RMPs, REMSs
- SAE narratives, Company analyses for SUSARs
- DSMB/Data Monitoring Committee charters and meeting organization
- Advisory Boards set up, KOL ongoing engagement (e.g., establishment of standing Scientific Advisory Boards through networking with leading KOLs)
- Portfolio prioritization and clinical development program review
For Venture Capital/Hedge Funds Investing in Biotech
- Comprehensive analysis of available data to inform clinical/medical aspects of due diligence evaluations
- Preclinical/clinical data analysis for business development/investment evaluations (e.g., IND readiness)
- MoA analysis with competitive landscape assessment
- Compound viability analysis from FIH/FIP (safety) through PoC (efficacy)
- Regulatory probability of success (approval of a marketing application: NDA/BLA, MAA)
- Phase-specific CSI (Compound Success Index per each Phase of Development): data and evidence driven model providing probability of success in a given phase of clinical development
Past Experience and Areas of Expertise
- Therapeutic areas of expertise: rare diseases (e.g., mucopolysaccharidoses, glycogen storage diseases, lipid storage disorders), neuro-degenerative/-muscular diseases, infectious diseases, virology, GI/hepatology, rheumatology, dermatology, immunology, immuno-oncology
- Compounds/NMEs: small molecules, biologics/mAbs, gene therapies, cell therapies, drug/device combinations
- Development Phases: Translational: 1 & 1b; PoC: 2a & 2b; Pivotal/Registrational: 3 & 3b; Post-approval: 4 & PASS/PMRs