Evolvement of EU regulations on innovative medicines

Abstract

Innovative medicines in the EU must follow regulatory requirements appertaining to the centralised procedure for registration. The study of the evolvement of EU regulations on centrally authorised products (CAPs) could shed light on the impact of the regulations on the access to innovative medicines. The methodology consisted of two parts: 1) A review and an analysis of the use of EU regulatory early access tools including the compilation of a glossary of regulatory terms, 2) An assessment of impact on public health through the non-availability of CAPs, identification of accessibility challenges of innovative medicines, and identification of alternative existing legislative tools to increase access to innovative medicines. Part 1: The early access tools selected for analysis were conditional marketing authorisation (CMA) and marketing authorisation under exceptional circumstances. The glossary compiled defined 280 regulatory terms. The results of the review and analysis have shown that between 2001 and 2016 a total of 65 innovative medicines were centrally authorised using CMA (35) and marketing authorisation under exceptional circumstances (30); 28 given a new active substance status, 33 an orphan designation and 7 withdrawn. Part 2: Between 1995 to 2015 a total of 822 CAPs covering 522 Anatomical Therapeutic Chemical (ATC) Codes were authorised in the EU. The Maltese NHS formulary does not include 322 (61%) of the 522 ATC Codes. Identified challenges with importation of CAPs in Malta are the low volumes required and the costs of the innovative medicines. In 2015-2016, 5 CAPs were imported as unlicensed medicines on a named patient basis and 19 CAPs as exceptional cases according to Article 20 of the Malta Medicines Act to increase access to innovative medicines in Malta. Until 2015, the parallel distribution system was not used to import CAPs in Malta. A one-year pilot project with fee reductions for parallel distribution notifications for CAPs which resulted in the importation of 5 parallel distributed CAPs, was launched by the European Medicines Agency (EMA) in 2016. Early access tools specified in EU regulations such as CMA and marketing authorisation under exceptional circumstances require more awareness, and intelligent interpretation and application. The pilot parallel distribution project involving notification fee reductions specifically authorised by the EMA specifically for Malta, was beneficial to accessibility of medicines in practice. This study is being used as an example by the European Commission to show that, in the interest of European citizens, flexibility in the regulation is possible, confirming an innovative concept of changing the perception of the EU that regulations are not biased towards safety and efficacy at the detriment of accessibility.