by Helen Collis at the TOPRA meeting
BRUSSELS, Oct 14 (APM) - Real world data generated through observational studies as part of early access schemes across Europe may add some evidence of efficacy for future regulatory discussions, the TOPRA regulatory conference in Brussels heard on Tuesday.
As more forms of early access schemes, designed to get not-yet-authorised drugs to patients, are rolled out across the region, industry representatives have heard previous warnings that the schemes are not designed as another means of generating efficacy data for filings.
However, questioned on the use of observational studies - a requirement for some early access schemes - and how data could be used to expand the indication of a drug, Tomas Salmonson, chair of the CHMP and of Sweden’s Medicinal Products Agency (MPA), suggested that in reality, there may be room to incorporate such data into regulatory assessments.
He said: “One of the challenges of using observational data is that we can’t promote off label use. It would be through off label use to expand the label. The most likely use would be to reduce the uncertainty around the medication - we get more cost effectiveness and safety data as well as efficacy data.
“But it’s a complicated world and I don’t like to exclude that real world data will be excluded from the efficacy study. I wouldn’t exclude that we will provide some data for label expansions (from this real world data)."
While he cautioned that this was not the purpose of such studies, his comments directly oppose those of Mark Corbett, senior vice president of global access programmes at Clinigen, speaking at last week’s UK BioIndustry Association conference, in London.
Corbett at the time underlined that early access schemes were “in no way about collecting clinical data to support marketing authorisation processes,” noting that they were not controlled randomised clinical trials. (APMMA 40003)
He said that such observational data would however, potentially be useful for health technology assessments (HTA) and discussions with payers.
This mind set was echoed on Monday at TOPRA by Mimi de Ruyck, head of market access, Belgium, for Bayer's health division, who said: “This additional (real world) data can aid HTA decision makers to get a better understanding of the added value of a product when applying for regulatory reimbursement.”
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