New trends and challenges in the European regulation of innovative medicines.

ABSTRACT

Regulators' marketing authorizations for innovative medicines are linked into a complex process with successive crucial decisions. Objectives and decision criteria of the stakeholders in this process, e.g. health technology assessment (HTA) bodies, payers, physicians and patients, vary and may result not only in different but even mutually exclusive requirements. Reacting to changes in scientific, economic and social demands, European regulatory agencies alter content and format of their assessment procedures and their communication. New diagnostic options (e.g. genotyping and biomarkers) and pharmaceutical innovations (e.g. targeted medicines, nanomedicines) are the scientific drivers of this development. Social drivers are the price and reimbursement decisions by HTA bodies and payers, prerequisites for most patients' access to innovative medicines. The European Medicines Agency's adaptive licensing concept and priority medicines scheme foster the early authorization of innovative medicines. HTA builds on regulators' assessment, with additional requirements and economic components. An intensified exchange between all stakeholders, e.g. in multilateral scientific advice procedures has been initiated. Diminishing the differences in the requirements of regulators and HTA bodies is in the best interest of both patients and the pharmaceutical industry, avoiding duplication of work and accelerating patients' access by early decisions on price and reimbursement.