AUTHOR=Ermisch Michael , Bucsics Anna , Vella Bonanno Patricia , Arickx Francis , Bybau Alexander , Bochenek Tomasz , van de Casteele Marc , Diogene Eduardo , Fürst Jurij , Garuolienė Kristina , van der Graaff Martin , Gulbinovič Jolanta , Haycox Alan , Jones Jan , Joppi Roberta , Laius Ott , Langner Irene , Martin Antony P. , Markovic-Pekovic Vanda , McCullagh Laura , Magnusson Einar , Nilsen Ellen , Selke Gisbert , Sermet Catherine , Simoens Steven , Sauermann Robert , Schuurman Ad , Ramos Ricardo , Vlahovic-Palcevski Vera , Zara Corinne , Godman Brian TITLE=Payers' Views of the Changes Arising through the Possible Adoption of Adaptive Pathways JOURNAL=Frontiers in Pharmacology VOLUME=7 YEAR=2016 URL=https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2016.00305 DOI=10.3389/fphar.2016.00305 ISSN=1663-9812 ABSTRACT=

Payers are a major stakeholder in any considerations and initiatives concerning adaptive licensing of new medicinal products, also referred to as Medicines Adaptive Pathways to patients (MAPPs). Firstly, the scope and necessity of MAPPs need further scrutiny, especially with regard to the definition of unmet need. Conditional approval pathways already exist for new medicines for seriously debilitating or life-threatening diseases and only a limited number of new medicines are innovative. Secondly, MAPPs will result in new medicines on the market with limited evidence about their effectiveness and safety. Additional data are to be collected after approval. Consequently, adaptive pathways may increase the risk of exposing patients to ineffective or unsafe medicines. We have already seen medicines approved conventionally that subsequently proved ineffective or unsafe amongst a wider, more co-morbid population as well as medicines that could have been considered for approval under MAPPs but subsequently proved ineffective or unsafe in Phase III trials and were never licensed. Thirdly, MAPPs also put high demands on payers. Routine collection of patient level data is difficult with high transaction costs. It is not clear who will fund these. Other challenges for payers include shifts in the risk governance framework, implications for evaluation and HTA, increased complexity of setting prices, difficulty with ensuring equity in the allocation of resources, definition of responsibility and liability and implementation of stratified use. Exit strategies also need to be agreed in advance, including price reductions, rebates, or reimbursement withdrawals when price premiums are not justified. These issues and concerns will be discussed in detail including potential ways forward.