title: Payers' Views of the Changes Arising through the Possible Adoption of Adaptive Pathways.
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.
authors.affiliation: Pharmaceuticals Department, National Association of Statutory Health Insurance Funds Berlin, Germany.
authors.forename: Michael
authors.initials: M
authors.lastname: Ermisch
authors.affiliation: Department of Finance, University of Vienna Vienna, Austria.
authors.forename: Anna
authors.initials: A
authors.lastname: Bucsics
authors.affiliation: Independent Pharmaceutical Consultant Mellieha, Malta.
authors.forename: Patricia
authors.initials: P
authors.lastname: Vella Bonanno
authors.affiliation: National Institute for Health and Disability Insurance Bruxelles, Belgium.
authors.forename: Francis
authors.initials: F
authors.lastname: Arickx
authors.affiliation: Zilveren Kruis Achmea Leiden, Netherlands.
authors.forename: Alexander
authors.initials: A
authors.lastname: Bybau
authors.affiliation: Department of Drug Management, Faculty of Health Sciences, Jagiellonian University Medical College Krakow, Poland.
authors.forename: Tomasz
authors.initials: T
authors.lastname: Bochenek
authors.affiliation: National Institute for Health and Disability Insurance Bruxelles, Belgium.
authors.forename: Marc
authors.initials: M
authors.lastname: van de Casteele
authors.affiliation: Clinical Pharmacology Service, Vall d'Hebron University Hospital, Fundació Institut Català de Farmacologia Barcelona, Spain.
authors.forename: Eduardo
authors.initials: E
authors.lastname: Diogene
authors.affiliation: Medicinal Products Department, Health Insurance Institute of Slovenia Ljubljana, Slovenia.
authors.forename: Jurij
authors.initials: J
authors.lastname: Fürst
authors.affiliation: Department of Pathology, Forensic Medicine and Pharmacology, Faculty of Medicine, Vilnius UniversityVilnius, Lithuania; Medicines Reimbursement Department, National Health Insurance FundVilnius, Lithuania.
authors.forename: Kristina
authors.initials: K
authors.lastname: Garuolienė
authors.affiliation: National Health Care Institute Diemen, Netherlands.
authors.forename: Martin
authors.initials: M
authors.lastname: van der Graaff
authors.affiliation: Department of Pathology, Forensic Medicine and Pharmacology, Faculty of Medicine, Vilnius UniversityVilnius, Lithuania; State Medicines Control AgencyVilnius, Lithuania.
authors.forename: Jolanta
authors.initials: J
authors.lastname: Gulbinovič
authors.affiliation: Health Economics Centre, University of Liverpool Management School Liverpool, UK.
authors.forename: Alan
authors.initials: A
authors.lastname: Haycox
authors.affiliation: Scottish Medicines Consortium Glasgow, UK.
authors.forename: Jan
authors.initials: J
authors.lastname: Jones
authors.affiliation: Pharmaceutical Drug Department, Azienda Sanitaria Locale of Verona Verona, Italy.
authors.forename: Roberta
authors.initials: R
authors.lastname: Joppi
authors.affiliation: State Agency of Medicines Tartu, Estonia.
authors.forename: Ott
authors.initials: O
authors.lastname: Laius
authors.affiliation: Wissenschaftliches Institut der AOK (WIdO) Berlin, Germany.
authors.forename: Irene
authors.initials: I
authors.lastname: Langner
authors.affiliation: Health Economics Centre, University of Liverpool Management School Liverpool, UK.
authors.forename: Antony P
authors.initials: AP
authors.lastname: Martin
authors.affiliation: Ministry of Health and Social WelfareBanja Luka, Bosnia and Herzegovina; Department of Social Pharmacy, Medical Faculty, University Banja LukaBanja Luka, Bosnia and Herzegovina.
authors.forename: Vanda
authors.initials: V
authors.lastname: Markovic-Pekovic
authors.affiliation: Pharmacoeconomics and Health Technology Assessment, Department of Pharmacology and Therapeutics, Trinity College Dublin, Ireland.
authors.forename: Laura
authors.initials: L
authors.lastname: McCullagh
authors.affiliation: Department of Health Services, Ministry of Health Reykjavík, Iceland.
authors.forename: Einar
authors.initials: E
authors.lastname: Magnusson
authors.affiliation: Norweigian Directorate for Health Oslo, Norway.
authors.forename: Ellen
authors.initials: E
authors.lastname: Nilsen
authors.affiliation: Wissenschaftliches Institut der AOK (WIdO) Berlin, Germany.
authors.forename: Gisbert
authors.initials: G
authors.lastname: Selke
authors.affiliation: Institut de Recherche et Documentation en Economie de la Santé (IRDES) Paris, France.
authors.forename: Catherine
authors.initials: C
authors.lastname: Sermet
authors.affiliation: Department of Pharmaceutical and Pharmacological Sciences, KU Leuven Leuven, Belgium.
authors.forename: Steven
authors.initials: S
authors.lastname: Simoens
authors.affiliation: Department of Pharmaceutical Affairs, Main Association of Austrian Social Insurance Institutions Vienna, Austria.
authors.forename: Robert
authors.initials: R
authors.lastname: Sauermann
authors.affiliation: National Health Care Institute Diemen, Netherlands.
authors.forename: Ad
authors.initials: A
authors.lastname: Schuurman
authors.affiliation: Health Technology Assessment, Prices and Reimbursement Department, INFARMED - National Authority of Medicines and Health Products, I. P., Parque de Saúde de Lisboa Lisbon, Portugal.
authors.forename: Ricardo
authors.initials: R
authors.lastname: Ramos
authors.affiliation: Department of Clinical Pharmacology, University Hospital Rijeka, Croatia.
authors.forename: Vera
authors.initials: V
authors.lastname: Vlahovic-Palcevski
authors.affiliation: Barcelona Health Region, Catalan Health Service Barcelona, Spain.
authors.forename: Corinne
authors.initials: C
authors.lastname: Zara
authors.affiliation: Health Economics Centre, University of Liverpool Management SchoolLiverpool, UK; Department of Pharmacoepidemiology, Strathclyde Institute of Pharmacy and Biomedical Sciences, University of StrathclydeGlasgow, UK; Division of Clinical Pharmacology, Department of Laboratory Medicine, Karolinska Institutet, Karolinska University Hospital HuddingeStockholm, Sweden.
authors.forename: Brian
authors.initials: B
authors.lastname: Godman
keywords: EMA; Europe; adaptive pathways; marketing authorization; payers