EMA authorisation procedure for drugs under observation

Placing a new medicine on the European market requires authorisation from the European Medicines Agency (EMA).

The EMA provides manufacturers with advisory support in order to ensure that the methods used to demonstrate the benefit of a product are properly applied prior to submitting an application for market authorisation. This support consists of what is known as ‘pre-submission’ activities. The aims of pre-submission activities are to assist the manufacturer to collect the necessary scientific evidence in a methodologically correct manner, to minimise the risk of exposing patients to unnecessary clinical trials, to increase the value of the data generated and to reduce administrative burden.

Participation in pre-submission activities is voluntary for the manufacturers and the recommendations of the EMA in the consultation procedure are not binding for the manufacturer.

Ombudsman identifies conflicts of interest

If a manufacturer requests advisory support from the EMA, the Scientific Advice Working Party (SAWP) appoints two coordinators with the appropriate expertise, who in turn select their own team of experts.

If the manufacturer decides to submit a marketing authorisation application after the consultation phase has been completed, two rapporteurs are appointed on the basis of the ‘best available expertise’, who in turn put together their own team of experts.

On her own initiative, the ombudsman submitted a request to the EMA in 2017, in particular to make pre-submission activities and the transition to the application procedure more transparent.

The inquiry revealed that, despite the utmost care, the current procedure has weaknesses with regard to the appointment of coordinators and rapporteurs. In 2017 and 2018, in approximately ten percent of marketing authorisation applications, at least one rapporteur was previously appointed as coordinator in the pre-submission phase for the same drug and could therefore have been prejudiced.

The EMA confirms that the selection of persons is made with the greatest care. However, in highly specialised areas of research and medicine, there is little room to manoeuvre when selecting the right experts. The EMA stresses that the members of the Committee for Medicinal Products for Human Use (CHMP) also must submit a declaration of interests to the EMA.

Instruments for greater transparency

In order to further improve the system, the Ombudsman invited interest groups to put forward their views during the public consultation. The German Statutory Health Insurance (GKV) contributed to the consultation. The Ombudsman’s decision, published in July 2019, shows that the GKV’s suggestions have been fruitful.

 

The Ombudsman recommends that there should be a separation between experts responsible for providing advice to a manufacturer and the rapporteurs responsible for assessing the market authorisation application for the same medicine.

 

If, in exceptional cases, it is not possible to separate the experts in the two procedures, the EMA should document the reasons for its decision and publish this information with the European Public Assessment Report (EPAR). However, the EMA should ensure that at least one of the two rapporteurs in the authorisation procedure did not play a prominent role in the pre-submission activities. Finally, the Ombudsman recommends that the EMA attach to the EPAR a detailed log of all relevant pre-submission activities, including the names of the experts involved.