Cluster

Core considerations

Stakeholders and target mapping

Landscape assessment

Ÿ Who are the regulatory bodies in target markets, and for which remits and geographical jurisdictions (national, regional, etc.) are they responsible?

Stakeholder decision drivers

Priorities/awareness

Ÿ Which diseases and unmet needs do regulators prioritise?

Ÿ Do they prefer specific types of interventions for an indication?

Need/preferences (value-fit)

Ÿ Which are the most meaningful safety and efficacy data in general (across regulators) and specifically (per regulator, e.g. data on domestic cohorts)?

Ÿ How can data collection be streamlined for requirements across regulators? E.g. by international trialling of intervention(s) in various domestic cohorts.

Ÿ Does own product conflict with other treatments?

Ÿ Are new methods of demonstrating safety/efficacy accepted (e.g. in silico modelling)?

Ÿ Are regulatory data requirements pre-determined by previous competitor applications (and approvals)?

Application/indications/label (decision points)

Ÿ Which are the likely indications at initial approval; which is the “cleanest” label possible?

Ÿ Which resource consumption (type and quantity of efficacy/safety data) will likely lead to a less or more restricted label (forecasting)?

Ÿ Which likely impact will different TPPs/labels have on uptake?

Ÿ Are providers using product out of indication and does data support the application for indication extension?

Ÿ Might post-launch data collection commitments change basis for cost assessment? Feeds into HTA for payers in next section.

Ÿ Are future changes to regulations (e.g. new hurdles) likely due to externalities and competitor products?

Stakeholder engagement

Engagement/relationship

Ÿ Has the value fit of TPP been tested and validated with stakeholders in market (e.g. does intervention really address unmet need or improve outcomes)? A TPP “endorsed” by physicians can considerably improve negotiations with regulators.