Parallel consultation, Scientific Advice & Early Dialogue – learnings from the past and shaping the future

Patient role/involvement

• How and when in regulatory and HT assessment process patients could be more effectively engaged?
• The role of patients in shaping outcome measurements for regulatory and reimbursement purposes

Uncertainty of evidence - feasibility of delivering an a data package acceptable for both regulators and reimbursement authorities

• Choice of comparator
• Surrogate/intermediate endpoints and extrapolating the results
• Use and acceptability of innovative clinical trial designs (adaptive trials, master protocols, use of historical controls)

Post-licensing evidence generation

Suitability of current regulatory & value frameworks for new technology based curative therapies(e.g. cell and gene) in the pipeline

Regulatory vs access views – internal alignment within industry functions?

Unmet medical need & evidentiary standards

• Professionals working in Regulatory Affairs and HTA/Market Access
• Professionals involved in drug development, e.g. clinicians, epidemiologists and biostatisticians
• Patient organisations
• Biotech companies, drug developers including SMEs
• Decision makers (Ministries of Health etc.)

• Through candid and well informed discussion help all involved stakeholders find better ways to improve communication in the regulatory-reimbursement ecosystem
• Identify the limitations in how evidence is used across the entire regulatory-reimbursement and their implications for the patients answer the question of what isn’t working perfectly
• Come up with pragmatic solutions for closing existing evidence gap and their policy implications (“what could we do” and “how we should do”).
• Trying to find solutions for patients to get access to medicines they need