OBJECTIVES

Clinical trials of new pharmaceuticals are designed to meet the requirements of regulatory authorities such as the European Medicines Agency (EMA). They also provide important evidence for use in health technology assessments (HTAs), especially for comparative effectiveness analyses. Historically, HTA evidence requirements have played second fiddle to regulatory needs, meaning the data available for HTA is often less than optimal, although developments such as joint HTA and regulatory scientific advice programmes help to reduce potential discordance.

METHODS

Although there are overlaps, HTA evidence needs differ from those of regulatory authorities. Additional foci of HTA bodies include ensuring the comparator reflects the standard of care in each country, outcomes are patient-relevant, quality of life and resource utilisation outcomes are fully considered, and long-term outcomes are evaluated in chronic and/ or progressive conditions. A major difference in evidence needs is that cost-effectiveness analysis is required by many HTA bodies but not required for regulatory purposes.

RESULTS

To further aid alignment of regulatory and HTA evidence needs, a HEOR analysis plan should be developed alongside the trial analysis plan. In designing the HEOR analysis plan, the focus is less on trial endpoints, instead covering data from the pivotal trials that are essential to inform HTA objectives. Pivotal trials often cannot meet all HTA data needs, hence the HEOR plan should further include the use of real world evidence, indirect treatment comparisons, and sourcing other economic model input data that could not be collected within the trial. As HTAs are country specific, local considerations are essential.

CONCLUSIONS

A HEOR analysis plan cannot provide the solution for all HTA needs but it can help optimise the planning and use of clinical trial data for joint marketing authorisation and HTA/ reimbursement requirements, as are increasingly happening in parallel in many countries.