Module 6

03/10/2024

Regulatory vs HTA principles

Regulators

need degree of certainty that the benefit of a new drug outweighs the risk.

leading to tightly defined trial requirements

e.g. endpoints specific to the disease state

trends:

additional benefit vs. additional risk.

“real world efficacy”

HTA bodies • assess benefit vs cost compared to treatments and interventions currently used.

• are prepared to accept much more uncertainty:

• but too much uncertainty on value will lead to rejection despite clear efficacy

• are prepared to accept alternative sources of data to address uncertainty

• may require standardised measures of net benefit such as

•

QALY (Quality Adjusted Life Year)

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Data required by agencies assessing value

Pricing, Reimbursement, Access and HTA bodies require some or all of the following: - Epidemiology data - Data to demonstrate the Public Health/Socio-economic burden *Summary of Trial Data / access to study reports - Relative Effectiveness including: * Comparison to currently used alternatives

Compliance / adherence * Longer term outcomes

* Clinical Relevance of statistically significant differences * Sub Group Analysis or tailored patient opportunities

*Quality of Life and Patient Reported Outcomes - Economic Model showing: Effect on patient direct/indirect costs

Cost effectiveness e.g. Cost/QALY within thresholds

- Overall budget impact * Safety data to support recommendation for chronic administration - Observational data to confirm results in a naturalistic setting

* Of which these may be provided by a regulatory trial package

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