Abstract
In order to monitor the net public health benefit of new drugs, especially in the light of recent stepwise approval approaches, there is a need to optimize real-time post-marketing evaluation of new drugs using data collected in routine care. Sweden, with its unique possibilities for observational research, can provide these data. We herein propose a framework for continuous monitoring of the effectiveness, safety, and cost-effectiveness of new drugs, using prospectively determined protocols designed in collaboration between all relevant stakeholders. We believe that this framework can be a useful tool for healthcare authorities and reimbursement agencies in the introduction of new drugs.
Highlights
Before the marketing of a new drug, its efficacy and safety are evaluated using randomized controlled trials (RCTs)
The population eventually treated with a new drug often differs in many respects from the sample studied in the RCTs
We propose a framework for such studies, using data collected in routine care
Summary
Before the marketing of a new drug, its efficacy and safety are evaluated using randomized controlled trials (RCTs). Effectiveness and Safety of Newly Introduced Medicines’ (16), we developed and validated a ‘sequentially evaluated non-randomized comparative effectiveness (SENCE)’ framework for continuous follow-up of new treatments using data from routine care in Sweden. This model is based on EHR data but can utilize data from other sources. This is a very strong assumption, and analyses should be accompanied by sensitivity analyses investigating how the study findings may be affected by the presence of unmeasured confounding We have in this framework included one commonly used approach proposed by Lin et al (26) that involves evaluating how powerful an unmeasured confounder would have to be to change the observed results (Figure 4)
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