OBJECTIVES

: In 2010, the European Medicines Agency (EMA) with Health Technology Assessment bodies (HTAbs), established parallel scientific advice (SA) that allows manufacturers to receive formal, consolidated feedback from the European Union (EU) regulators and HTAbs on their development plans for new medicines. This study aimed to assess the impact of integrated SA (ISA) on clinical development and health technology assessment (HTA) of new medicines in Europe.

METHODS

: A systematic search was performed for publications that analysed the impact of ISA. Manufacturers’ compliance with the advice provided by EMA and HTAbs, the outcome of marketing authorisation (MA) and reimbursement were analysed. ‘Impact’ was defined as the influence on the manufacturers’ clinical development programme and the outcome of MA and HTA appraisal.

RESULTS

: The systematic search identified twelve studies; six studies explored the impact of SA on evidence generation programmes, authored by EMA or HTAb representatives. The publications analysed previous SA advice meetings and explored questions asked by the manufacturers and level of compliance between stakeholders in a qualitative and quantitative manner.

There has been a marked increase in integrated scientific engagement in recent years. Manufacturers most frequently complied with EMA advice, followed by compliance with at least one HTAb. SA was frequently followed for study design and least followed was comparator advice (57%) where frequently the HTAb and EMA were not aligned.

For parallel SA performed between 2010-2015, the products which gained MA, also gained positive reimbursement decisions from most of the HTAbs. For parallel and HTA-only SA, the majority of remaining products continued in clinical development and other products failed development.

CONCLUSIONS

: ISA offers a valuable dialogue opportunity for stakeholders from different perspectives. This analysis demonstrates that SA may increase manufacturers’ chance to obtain regulatory and reimbursement recommendation. Research from other stakeholders (e.g. patient representatives) will provide further understanding of the impact of SA.