Payers' views of the changes arising through the possible adoption of adaptive pathways

Ermisch, Michael and Bucsics, Anna and Vella Bonanno, Patricia and Arickx, Francis and Bybau, Alexander and Bochenek, Tomasz and van de Casteele, Marc and Diogene, Eduardo and Fürst, Jurij and Garuolienė, Kristina and van der Graaff, Martin and Gulbinovič, Jolanta and Haycox, Alan and Jones, Jan and Joppi, Roberta and Laius, Ott and Langner, Irene and Martin, Antony P. and Markovic-Pekovic, Vanda and McCullagh, Laura and Magnusson, Einar and Nilsen, Ellen and Selke, Gisbert and Sermet, Catherine and Simoens, Steven and Sauermann, Robert and Schuurman, Ad and Ramos, Ricardo and Vlahovic-Palcevski, Vera and Zara, Corinne and Godman, Brian (2016) Payers' views of the changes arising through the possible adoption of adaptive pathways. Frontiers in Pharmacology, 7. ISSN 1663-9812

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    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.