Spring Intro 2023

23/03/2023

Creating and submitting the data to HTA bodies

‘Evidence’ translation

‘Evidence’ building blocks

LOC HTA LOC HTA

Internal resource (dossier, toolkit)

Unmet need

Submission

Safety, Efficacy

Submission

HTA HTA HTA HTA

LOC LOC LOC LOC LOC

Effectiveness

Submission

Evidence synthesis

Adaptation & dissemination

Submission

Cost-effectiveness

Submission

Financial impact

Submission

Access, service impact

HTA

Submission

Input: local evidence requirements

The Organisation for Professionals in Regulatory Affairs

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Evolving Regulatory Interface

Future paradigm?

Current paradigm

MA

MA

Assessors

Regulators

Payers

Regulators

Payers

Dedicated Relative Efficacy/- Effectiveness Assessment?

Assessment Focus

Quality, Safety, Efficacy, (First 3 hurdles) Benefit-Risk Profile

Relative Efficacy / - Effectiveness, Cost vs Health Benefit, Budget Impact (4 th hurdle) Active-controlled RCT; Observational studies, Cost-effectiveness/-utility analyses, Budget impact analysis

Quality, Safety, Efficacy, Benefit-Risk Profile

Cost vs Health Benefit, Budget Impact

Relative Efficacy / - Effectiveness

Studies / Data

Emphasis on: RCT, most often placebo-controlled

Emphasis on: RCT, more often active- and placebo-controlled

Cost-effectiveness/- utility analyses, Budget impact analysis

Active-controlled RCT, Adaptive phase III-IV trials Observational studies, Meta-analysis

MA = Marketing Authorisation; RCT = randomised controlled trial

New EPAR already reflects the movement towards

How Regulatory Agencies could interact with Health Technology Bodies, Source: Lonngren et al DIA, Berlin, March 2009

The Organisation for Professionals in Regulatory Affairs

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