Use of the conditional marketing authorization pathway for oncology medicines in Europe

Abstract

Conditional marketing authorization (CMA) in the European Union (EU) is an early access pathway for medicines that show promising therapeutic effects, but for which comprehensive data are not available. Using a mixed quantitative-qualitative research design, we evaluated how CMA has been used in marketing authorization of oncology medicines in the period 2006 to 2013. We show that compared to full marketing authorization, CMA is granted based on less comprehensive data. However, this is accompanied by significantly longer assessment times and less consensus among regulators about marketing authorization. Moreover, development time from first-in-human testing to marketing authorization did not differ between full marketing authorization and CMA, but was significantly longer for CMA compared to accelerated approved products in the United States (US). Results indicate that CMA is not used by companies as a prospectively planned pathway to obtain early access, but as a "rescue option" when submitted data are not strong enough to justify full marketing authorization.

Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13

“…In contrast with recent studies focusing on a subset of oncology drug approvals in Europe,32 33 we included all authorised oncology indications regardless of route of approval and systematically analysed survival and quality of life benefits. We also relied exclusively on evidence generated from randomised controlled trials.…”

“…Recent FDA-EMA comparisons show differences in regulatory decisions, route of approval, and availability of cancer drugs that could have important implications for clinical practice and patient safety. [31][32][33][34][35] In particular, regulatory provisions for expediting drug development and approval differ between the US and the EU, 32 with EU regulation being more restrictive in scope. 36 This could lead to divergent outcomes between the two regions.…”

Pathways to faster access to medicines

“…32 This may indicate that conditional marketing is used by companies and regulators for medicines for which a full marketing authorisation is uncertain, rather than as a planned strategy for bringing promising medicines to market earlier.…”

Demands for access to new therapies: are there alternatives to accelerated access?

“…Numerous jurisdictions have introduced systems that allow for provisional approval of medicines on the basis of less complete data (such as surrogate markers) on the condition that post-marketing studies are done to resolve any uncertainties about safety or clinical effectiveness. [20][21][22][23] Many countries also allow individual patients to apply to regulators for use of unregistered medicines through special access schemes or early access programmes. [24][25][26] Several "managed entry" or "coverage with evidence development" schemes have also been established for therapies that have been approved by regulators but not (yet) funded.…”