Health Technology Assessment for Molecular Diagnostics: Practices, Challenges, and Recommendations from the Medical Devices and Diagnostics Special Interest Group

Sabine Vogler; N Grössmann; J C Del Paggio; S Wolf; R Sullivan; C M Booth; K Rosian; R Emprechtinger; C Wild; Nicolas Silva-Illanes; Manuel Espinoza; Laia Maynou; John Cairns; David Taylor; Laia Maynou Pujolras; John Cairns; Ajay Aggarwal; Ophira Ginsburg; Tito Fojo; Livio Garattini; Katelijne van de Vooren; Alessandro Curto; L Kandolf Sekulovic; J Guo; S Agarwala; A Hauschild; G McArthur; G Cinat; A Wainstein; C Caglevic; P Lorigan; H Gogas; M Alvarez; R Duncombe; C Lebbe; K Peris; P Rutkowski; A Stratigos; A.-M. Forsea; L De La Cruz Merino; M Kukushkina; R Dummer; C Hoeller; C Gorry; L Bastholt; D Herceg; B Neyns; R Vieira; P Arenberger; M Bylaite-Bucinskiene; N Babovic; M Banjin; K Putnik; V Todorovic; K Kirov; J Ocvirk; A Zhukavets; A Ymeri; I Stojkovski; C Garbe; M R Seeley; L E Tonner-Navarro; B D Beck; R Deskin; V J Feron; Gunnar Johanson; Hermann M Bolt; Ronald L Akehurst; Eric Abadie; Noël Renaudin; François Sarkozy; Susan Garfield; Julie Polisena; Daryl S Spinner; Anne Postulka; Christine Y Lu; Simrandeep K Tiwana; Eric Faulkner; Nick Poulios; Vladimir Zah; Michael Longacre; Peter Schneider; Sabine Vogler

doi:10.1016/j.healthpol.2018.11.003

Health Technology Assessment for Molecular Diagnostics: Practices, Challenges, and Recommendations from the Medical Devices and Diagnostics Special Interest Group

Sabine Vogler (Editor), N Grössmann, J C Del Paggio, S Wolf, R Sullivan, C M Booth, K Rosian, R Emprechtinger, C Wild, Nicolas Silva-Illanes, Manuel Espinoza, Laia Maynou, John Cairns, David Taylor, Laia Maynou Pujolras, John Cairns, Ajay Aggarwal, Ophira Ginsburg, Tito Fojo, Livio GarattiniKatelijne van de Vooren, Alessandro Curto, L Kandolf Sekulovic, J Guo, S Agarwala, A Hauschild, G McArthur, G Cinat, A Wainstein, C Caglevic, P Lorigan, H Gogas, M Alvarez, R Duncombe, C Lebbe, K Peris, P Rutkowski, A Stratigos, A.-M. Forsea, L De La Cruz Merino, M Kukushkina, R Dummer, C Hoeller, C Gorry, L Bastholt, D Herceg, B Neyns, R Vieira, P Arenberger, M Bylaite-Bucinskiene, N Babovic, M Banjin, K Putnik, V Todorovic, K Kirov, J Ocvirk, A Zhukavets, A Ymeri, I Stojkovski, C Garbe, M R Seeley, L E Tonner-Navarro, B D Beck, R Deskin, V J Feron, Gunnar Johanson, Hermann M Bolt, Ronald L Akehurst, Eric Abadie, Noël Renaudin, François Sarkozy, Susan Garfield, Julie Polisena, Daryl S Spinner, Anne Postulka, Christine Y Lu, Simrandeep K Tiwana, Eric Faulkner, Nick Poulios, Vladimir Zah, Michael Longacre, Peter Schneider, Sabine Vogler

Research output: Contribution to journal › Article › peer-review

Abstract

Background Decisions on the reimbursement of the same cancer drugs are different across European countries, but empirical work on the reasons behind these differences has been scarce. The main objective of this paper is to make a methodological contribution to existing research, specifically by outlining the systematic process of analysis to address such questions and determining the factors that might lead to different drug reimbursement decisions, and to explore its application in the field of oncology. Methods Reimbursement decisions on cancer drugs in six European countries (Belgium, England, Poland, Portugal, Scotland, and Sweden) between 2006 and 2014 were included in the study. A taxonomy was developed, comprising two groups of variables (system-level and product-specific) and an econometric model was specified (multilevel mixed-effects ordered probit). Results Only one in six evaluations in the sample reach the same reimbursement recommendation. Most health system variables were not determinants of a higher or lower probability of a positive reimbursement recommendation. However, the probability of reimbursement was higher when a drug was considered cost-effective by NICE/SMC and when there was a financial Managed Entry Agreement. This work also demonstrated a possible econometric approach for analysing differences in reimbursement decisions and contributes a structured approach for collecting and preparing data for such analyses. Conclusions Drug reimbursement decisions can be analysed in detail along a set of factors that are related to each decision. This information is essential, not only for understanding why a particular drug is accepted in one country and not in another but also when trying to implement a new HTA system or reform an existing one. This analysis provides policy makers and stakeholders with a model that enables a better understanding of the factors that drive HTA decisions and is adaptable to answer similar questions. Moreover, the data collection limitations encountered and described in this work shed light on the need for greater accessibility and transparency in HTA systems and regarding HTA outcomes.

Original language	English
Pages (from-to)	67-76
Number of pages	10
Journal	Value in Health
Early online date	20 Nov 2018
DOIs	https://doi.org/10.1016/j.healthpol.2018.11.003
Publication status	Published - 2018

Keywords

Access
Advocacy
Benefit
Biosimilar medicines
Biosimilars
Breast cancer medicines
Cancer drug approval
Cancer drug-indications
Cancer economics
Cancer medicines
Drug reimbursement
ESMO-MCBS
Economic evaluation
Europe
European countries
Generic medicines
HIV
Health Technology Assessment (HTA)
Health economics
Health technology assessment
Health technology assessment (HTA)
Immunooncology
Infectious disease
Innovative medicines
Markov models
Metastatic breast cancer
Metastatic melanoma
Multilevel mixed-effects Ordered Probit
Non-prescription medicines
Orphan medicinal products
Policy
Price study
Regional variations
Targeted therapy
Trastuzumab
Treatment
Vaccines
Value
Value for money
cancer
cancer drugs
colorectal cancer
decision making
diagnostics
economic evaluation
health technology assessment
pharmaceuticals
screening
structural uncertainty

Research Beacons, Institutes and Platforms

Manchester Cancer Research Centre

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

Access to Document

10.1016/j.healthpol.2018.11.003

Cite this

Vogler, S. (Ed.), Grössmann, N., Paggio, J. C. D., Wolf, S., Sullivan, R., Booth, C. M., Rosian, K., Emprechtinger, R., Wild, C., Silva-Illanes, N., Espinoza, M., Maynou, L., Cairns, J., Taylor, D., Pujolras, L. M., Cairns, J., Aggarwal, A., Ginsburg, O., Fojo, T., ... Vogler, S. (2018). Health Technology Assessment for Molecular Diagnostics: Practices, Challenges, and Recommendations from the Medical Devices and Diagnostics Special Interest Group. Value in Health, 67-76. https://doi.org/10.1016/j.healthpol.2018.11.003

@article{457a37a529714fbb99e1220ec9214538,

title = "Health Technology Assessment for Molecular Diagnostics: Practices, Challenges, and Recommendations from the Medical Devices and Diagnostics Special Interest Group",

abstract = "Background Decisions on the reimbursement of the same cancer drugs are different across European countries, but empirical work on the reasons behind these differences has been scarce. The main objective of this paper is to make a methodological contribution to existing research, specifically by outlining the systematic process of analysis to address such questions and determining the factors that might lead to different drug reimbursement decisions, and to explore its application in the field of oncology. Methods Reimbursement decisions on cancer drugs in six European countries (Belgium, England, Poland, Portugal, Scotland, and Sweden) between 2006 and 2014 were included in the study. A taxonomy was developed, comprising two groups of variables (system-level and product-specific) and an econometric model was specified (multilevel mixed-effects ordered probit). Results Only one in six evaluations in the sample reach the same reimbursement recommendation. Most health system variables were not determinants of a higher or lower probability of a positive reimbursement recommendation. However, the probability of reimbursement was higher when a drug was considered cost-effective by NICE/SMC and when there was a financial Managed Entry Agreement. This work also demonstrated a possible econometric approach for analysing differences in reimbursement decisions and contributes a structured approach for collecting and preparing data for such analyses. Conclusions Drug reimbursement decisions can be analysed in detail along a set of factors that are related to each decision. This information is essential, not only for understanding why a particular drug is accepted in one country and not in another but also when trying to implement a new HTA system or reform an existing one. This analysis provides policy makers and stakeholders with a model that enables a better understanding of the factors that drive HTA decisions and is adaptable to answer similar questions. Moreover, the data collection limitations encountered and described in this work shed light on the need for greater accessibility and transparency in HTA systems and regarding HTA outcomes.",

keywords = "Access, Advocacy, Benefit, Biosimilar medicines, Biosimilars, Breast cancer medicines, Cancer drug approval, Cancer drug-indications, Cancer economics, Cancer medicines, Drug reimbursement, ESMO-MCBS, Economic evaluation, Europe, European countries, Generic medicines, HIV, Health Technology Assessment (HTA), Health economics, Health technology assessment, Health technology assessment (HTA), Immunooncology, Infectious disease, Innovative medicines, Markov models, Metastatic breast cancer, Metastatic melanoma, Multilevel mixed-effects Ordered Probit, Non-prescription medicines, Orphan medicinal products, Policy, Price study, Regional variations, Targeted therapy, Trastuzumab, Treatment, Vaccines, Value, Value for money, cancer, cancer drugs, colorectal cancer, decision making, diagnostics, economic evaluation, health technology assessment, pharmaceuticals, screening, structural uncertainty",

author = "Sabine Vogler and N Gr{\"o}ssmann and Paggio, \{J C Del\} and S Wolf and R Sullivan and Booth, \{C M\} and K Rosian and R Emprechtinger and C Wild and Nicolas Silva-Illanes and Manuel Espinoza and Laia Maynou and John Cairns and David Taylor and Pujolras, \{Laia Maynou\} and John Cairns and Ajay Aggarwal and Ophira Ginsburg and Tito Fojo and Livio Garattini and \{van de Vooren\}, Katelijne and Alessandro Curto and Sekulovic, \{L Kandolf\} and J Guo and S Agarwala and A Hauschild and G McArthur and G Cinat and A Wainstein and C Caglevic and P Lorigan and H Gogas and M Alvarez and R Duncombe and C Lebbe and K Peris and P Rutkowski and A Stratigos and A.-M. Forsea and Merino, \{L De La Cruz\} and M Kukushkina and R Dummer and C Hoeller and C Gorry and L Bastholt and D Herceg and B Neyns and R Vieira and P Arenberger and M Bylaite-Bucinskiene and N Babovic and M Banjin and K Putnik and V Todorovic and K Kirov and J Ocvirk and A Zhukavets and A Ymeri and I Stojkovski and C Garbe and Seeley, \{M R\} and Tonner-Navarro, \{L E\} and Beck, \{B D\} and R Deskin and Feron, \{V J\} and Gunnar Johanson and Bolt, \{Hermann M\} and Akehurst, \{Ronald L\} and Eric Abadie and No{\"e}l Renaudin and Fran{\c c}ois Sarkozy and Susan Garfield and Julie Polisena and Spinner, \{Daryl S\} and Anne Postulka and Lu, \{Christine Y\} and Tiwana, \{Simrandeep K\} and Eric Faulkner and Nick Poulios and Vladimir Zah and Michael Longacre and Peter Schneider and Sabine Vogler",

year = "2018",

doi = "10.1016/j.healthpol.2018.11.003",

language = "English",

pages = "67--76",

journal = "Value in Health",

issn = "1524-4733",

publisher = "Elsevier BV",

}

Vogler, S (ed.), Grössmann, N, Paggio, JCD, Wolf, S, Sullivan, R, Booth, CM, Rosian, K, Emprechtinger, R, Wild, C, Silva-Illanes, N, Espinoza, M, Maynou, L, Cairns, J, Taylor, D, Pujolras, LM, Cairns, J, Aggarwal, A, Ginsburg, O, Fojo, T, Garattini, L, van de Vooren, K, Curto, A, Sekulovic, LK, Guo, J, Agarwala, S, Hauschild, A, McArthur, G, Cinat, G, Wainstein, A, Caglevic, C, Lorigan, P, Gogas, H, Alvarez, M, Duncombe, R, Lebbe, C, Peris, K, Rutkowski, P, Stratigos, A, Forsea, A-M, Merino, LDLC, Kukushkina, M, Dummer, R, Hoeller, C, Gorry, C, Bastholt, L, Herceg, D, Neyns, B, Vieira, R, Arenberger, P, Bylaite-Bucinskiene, M, Babovic, N, Banjin, M, Putnik, K, Todorovic, V, Kirov, K, Ocvirk, J, Zhukavets, A, Ymeri, A, Stojkovski, I, Garbe, C, Seeley, MR, Tonner-Navarro, LE, Beck, BD, Deskin, R, Feron, VJ, Johanson, G, Bolt, HM, Akehurst, RL, Abadie, E, Renaudin, N, Sarkozy, F, Garfield, S, Polisena, J, Spinner, DS, Postulka, A, Lu, CY, Tiwana, SK, Faulkner, E, Poulios, N, Zah, V, Longacre, M, Schneider, P & Vogler, S 2018, 'Health Technology Assessment for Molecular Diagnostics: Practices, Challenges, and Recommendations from the Medical Devices and Diagnostics Special Interest Group', Value in Health, pp. 67-76. https://doi.org/10.1016/j.healthpol.2018.11.003

TY - JOUR

T1 - Health Technology Assessment for Molecular Diagnostics: Practices, Challenges, and Recommendations from the Medical Devices and Diagnostics Special Interest Group

AU - Grössmann, N

AU - Paggio, J C Del

AU - Wolf, S

AU - Sullivan, R

AU - Booth, C M

AU - Rosian, K

AU - Emprechtinger, R

AU - Wild, C

AU - Silva-Illanes, Nicolas

AU - Espinoza, Manuel

AU - Maynou, Laia

AU - Cairns, John

AU - Taylor, David

AU - Pujolras, Laia Maynou

AU - Cairns, John

AU - Aggarwal, Ajay

AU - Ginsburg, Ophira

AU - Fojo, Tito

AU - Garattini, Livio

AU - van de Vooren, Katelijne

AU - Curto, Alessandro

AU - Sekulovic, L Kandolf

AU - Guo, J

AU - Agarwala, S

AU - Hauschild, A

AU - McArthur, G

AU - Cinat, G

AU - Wainstein, A

AU - Caglevic, C

AU - Lorigan, P

AU - Gogas, H

AU - Alvarez, M

AU - Duncombe, R

AU - Lebbe, C

AU - Peris, K

AU - Rutkowski, P

AU - Stratigos, A

AU - Forsea, A.-M.

AU - Merino, L De La Cruz

AU - Kukushkina, M

AU - Dummer, R

AU - Hoeller, C

AU - Gorry, C

AU - Bastholt, L

AU - Herceg, D

AU - Neyns, B

AU - Vieira, R

AU - Arenberger, P

AU - Bylaite-Bucinskiene, M

AU - Babovic, N

AU - Banjin, M

AU - Putnik, K

AU - Todorovic, V

AU - Kirov, K

AU - Ocvirk, J

AU - Zhukavets, A

AU - Ymeri, A

AU - Stojkovski, I

AU - Garbe, C

AU - Seeley, M R

AU - Tonner-Navarro, L E

AU - Beck, B D

AU - Deskin, R

AU - Feron, V J

AU - Johanson, Gunnar

AU - Bolt, Hermann M

AU - Akehurst, Ronald L

AU - Abadie, Eric

AU - Renaudin, Noël

AU - Sarkozy, François

AU - Garfield, Susan

AU - Polisena, Julie

AU - Spinner, Daryl S

AU - Postulka, Anne

AU - Lu, Christine Y

AU - Tiwana, Simrandeep K

AU - Faulkner, Eric

AU - Poulios, Nick

AU - Zah, Vladimir

AU - Longacre, Michael

AU - Schneider, Peter

AU - Vogler, Sabine

A2 - Vogler, Sabine

PY - 2018

Y1 - 2018

N2 - Background Decisions on the reimbursement of the same cancer drugs are different across European countries, but empirical work on the reasons behind these differences has been scarce. The main objective of this paper is to make a methodological contribution to existing research, specifically by outlining the systematic process of analysis to address such questions and determining the factors that might lead to different drug reimbursement decisions, and to explore its application in the field of oncology. Methods Reimbursement decisions on cancer drugs in six European countries (Belgium, England, Poland, Portugal, Scotland, and Sweden) between 2006 and 2014 were included in the study. A taxonomy was developed, comprising two groups of variables (system-level and product-specific) and an econometric model was specified (multilevel mixed-effects ordered probit). Results Only one in six evaluations in the sample reach the same reimbursement recommendation. Most health system variables were not determinants of a higher or lower probability of a positive reimbursement recommendation. However, the probability of reimbursement was higher when a drug was considered cost-effective by NICE/SMC and when there was a financial Managed Entry Agreement. This work also demonstrated a possible econometric approach for analysing differences in reimbursement decisions and contributes a structured approach for collecting and preparing data for such analyses. Conclusions Drug reimbursement decisions can be analysed in detail along a set of factors that are related to each decision. This information is essential, not only for understanding why a particular drug is accepted in one country and not in another but also when trying to implement a new HTA system or reform an existing one. This analysis provides policy makers and stakeholders with a model that enables a better understanding of the factors that drive HTA decisions and is adaptable to answer similar questions. Moreover, the data collection limitations encountered and described in this work shed light on the need for greater accessibility and transparency in HTA systems and regarding HTA outcomes.

AB - Background Decisions on the reimbursement of the same cancer drugs are different across European countries, but empirical work on the reasons behind these differences has been scarce. The main objective of this paper is to make a methodological contribution to existing research, specifically by outlining the systematic process of analysis to address such questions and determining the factors that might lead to different drug reimbursement decisions, and to explore its application in the field of oncology. Methods Reimbursement decisions on cancer drugs in six European countries (Belgium, England, Poland, Portugal, Scotland, and Sweden) between 2006 and 2014 were included in the study. A taxonomy was developed, comprising two groups of variables (system-level and product-specific) and an econometric model was specified (multilevel mixed-effects ordered probit). Results Only one in six evaluations in the sample reach the same reimbursement recommendation. Most health system variables were not determinants of a higher or lower probability of a positive reimbursement recommendation. However, the probability of reimbursement was higher when a drug was considered cost-effective by NICE/SMC and when there was a financial Managed Entry Agreement. This work also demonstrated a possible econometric approach for analysing differences in reimbursement decisions and contributes a structured approach for collecting and preparing data for such analyses. Conclusions Drug reimbursement decisions can be analysed in detail along a set of factors that are related to each decision. This information is essential, not only for understanding why a particular drug is accepted in one country and not in another but also when trying to implement a new HTA system or reform an existing one. This analysis provides policy makers and stakeholders with a model that enables a better understanding of the factors that drive HTA decisions and is adaptable to answer similar questions. Moreover, the data collection limitations encountered and described in this work shed light on the need for greater accessibility and transparency in HTA systems and regarding HTA outcomes.

KW - Access

KW - Advocacy

KW - Benefit

KW - Biosimilar medicines

KW - Biosimilars

KW - Breast cancer medicines

KW - Cancer drug approval

KW - Cancer drug-indications

KW - Cancer economics

KW - Cancer medicines

KW - Drug reimbursement

KW - ESMO-MCBS

KW - Economic evaluation

KW - Europe

KW - European countries

KW - Generic medicines

KW - HIV

KW - Health Technology Assessment (HTA)

KW - Health economics

KW - Health technology assessment

KW - Health technology assessment (HTA)

KW - Immunooncology

KW - Infectious disease

KW - Innovative medicines

KW - Markov models

KW - Metastatic breast cancer

KW - Metastatic melanoma

KW - Multilevel mixed-effects Ordered Probit

KW - Non-prescription medicines

KW - Orphan medicinal products

KW - Policy

KW - Price study

KW - Regional variations

KW - Targeted therapy

KW - Trastuzumab

KW - Treatment

KW - Vaccines

KW - Value

KW - Value for money

KW - cancer

KW - cancer drugs

KW - colorectal cancer

KW - decision making

KW - diagnostics

KW - economic evaluation

KW - health technology assessment

KW - pharmaceuticals

KW - screening

KW - structural uncertainty

UR - https://www.scopus.com/pages/publications/85057054742

U2 - 10.1016/j.healthpol.2018.11.003

DO - 10.1016/j.healthpol.2018.11.003

M3 - Article

SN - 1524-4733

SP - 67

EP - 76

JO - Value in Health

JF - Value in Health

ER -

Health Technology Assessment for Molecular Diagnostics: Practices, Challenges, and Recommendations from the Medical Devices and Diagnostics Special Interest Group

Abstract

Keywords

Research Beacons, Institutes and Platforms

UN SDGs

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