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

Michael Ermisch, Anna Bucsics, Patricia Vella Bonanno, Francis Arickx, Alexander Bybau, Tomasz Bochenek, Marc van de Casteele, Eduardo Diogene, Jurij Fürst, Kristina Garuolienė, Martin van der Graaff, Jolanta Gulbinovič, Alan Haycox, Jan Jones, Roberta Joppi, Ott Laius, Irene Langner, Antony P. Martin, Vanda Markovic-Pekovic, Laura McCullagh & 11 others Einar Magnusson, Ellen Nilsen, Gisbert Selke, Catherine Sermet, Steven Simoens, Robert Sauermann, Ad Schuurman, Ricardo Ramos, Vera Vlahovic-Palcevski, Corinne Zara, Brian Godman

Research output: Contribution to journalArticle

17 Citations (Scopus)

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.

LanguageEnglish
Article number305
Number of pages9
JournalFrontiers in Pharmacology
Volume7
DOIs
Publication statusPublished - 28 Sep 2016

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Resource Allocation
Financial Management
Licensure
Safety
Costs and Cost Analysis
Population

Keywords

  • adaptive pathways
  • EMA
  • payers
  • marketing authorization
  • Europe

Cite this

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. / Payers' views of the changes arising through the possible adoption of adaptive pathways. In: Frontiers in Pharmacology . 2016 ; Vol. 7.
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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.",
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Ermisch, M, Bucsics, A, Vella Bonanno, P, Arickx, F, Bybau, A, Bochenek, T, van de Casteele, M, Diogene, E, Fürst, J, Garuolienė, K, van der Graaff, M, Gulbinovič, J, Haycox, A, Jones, J, Joppi, R, Laius, O, Langner, I, Martin, AP, Markovic-Pekovic, V, McCullagh, L, Magnusson, E, Nilsen, E, Selke, G, Sermet, C, Simoens, S, Sauermann, R, Schuurman, A, Ramos, R, Vlahovic-Palcevski, V, Zara, C & Godman, B 2016, 'Payers' views of the changes arising through the possible adoption of adaptive pathways' Frontiers in Pharmacology , vol. 7, 305. https://doi.org/10.3389/fphar.2016.00305

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

In: Frontiers in Pharmacology , Vol. 7, 305, 28.09.2016.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Payers' views of the changes arising through the possible adoption of adaptive pathways

AU - Ermisch, Michael

AU - Bucsics, Anna

AU - Vella Bonanno, Patricia

AU - Arickx, Francis

AU - Bybau, Alexander

AU - Bochenek, Tomasz

AU - van de Casteele, Marc

AU - Diogene, Eduardo

AU - Fürst, Jurij

AU - Garuolienė, Kristina

AU - van der Graaff, Martin

AU - Gulbinovič, Jolanta

AU - Haycox, Alan

AU - Jones, Jan

AU - Joppi, Roberta

AU - Laius, Ott

AU - Langner, Irene

AU - Martin, Antony P.

AU - Markovic-Pekovic, Vanda

AU - McCullagh, Laura

AU - Magnusson, Einar

AU - Nilsen, Ellen

AU - Selke, Gisbert

AU - Sermet, Catherine

AU - Simoens, Steven

AU - Sauermann, Robert

AU - Schuurman, Ad

AU - Ramos, Ricardo

AU - Vlahovic-Palcevski, Vera

AU - Zara, Corinne

AU - Godman, Brian

PY - 2016/9/28

Y1 - 2016/9/28

N2 - 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.

AB - 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.

KW - adaptive pathways

KW - EMA

KW - payers

KW - marketing authorization

KW - Europe

U2 - 10.3389/fphar.2016.00305

DO - 10.3389/fphar.2016.00305

M3 - Article

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JO - Frontiers in Pharmacology

T2 - Frontiers in Pharmacology

JF - Frontiers in Pharmacology

SN - 1663-9812

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