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Preferences for Advanced Therapy Medicinal Products: Understanding the Published Literature on the Value of Innovative Health Interventions.

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Title: Preferences for Advanced Therapy Medicinal Products: Understanding the Published Literature on the Value of Innovative Health Interventions.
Authors: Ferizović, N.1 (AUTHOR) Nermina.ferizovic.20@ucl.ac.uk, Lorgelly, P.2 (AUTHOR), Clarke, C. S.1 (AUTHOR), Hunter, R. M.1 (AUTHOR), Plackett, R.1 (AUTHOR), Abbas, W.1 (AUTHOR), Freemantle, N.1 (AUTHOR)
Source: Inquiry (00469580). 11/1/2025, Vol. 62, p1-14. 14p.
Subject Terms: *Diffusion of innovations, *Quality of life, Gene therapy, Medical information storage & retrieval systems, Risk assessment, Health insurance reimbursement, Tissue engineering, Cellular therapy, Decision making in clinical medicine, Uncertainty, Treatment effectiveness, Systematic reviews, MEDLINE
Abstract: Advanced therapy medicinal products (ATMPs) are ground-breaking genetic and cell/tissue-based therapies with the potential to treat and even cure myriad health conditions. However, they have faced multiple challenges in decision making with relatively low success in achieving reimbursement. The aim of this study was to review the published preference-based research on ATMPs to identify value attributes as a starting point in a research workstream aimed at understanding the true value of these innovative therapies. A literature review was used to identify studies across MedLine, Embase, Allied and Complementary Medicine Database, Global Health, ProQuest Central, Social Sciences Premium Collection, Applied Social Sciences Index and Abstracts, Econ Lit, Web of Science and SCOPUS from inception to 3rd March 2024. Search terms for genetic and cell/tissue-based therapies were conducted separately. Studies were included if they were a peer-reviewed primary research study or systematic literature review using stated preference measures to elicit preferences for ATMPs. Thirteen studies were included with 8 pertaining to genetic therapies and 5 to cell/tissue therapies. Attributes of value were similar across the studies and ATMP categories. Attributes were grouped into the following for genetic therapies: clinical benefits, uncertainty, risk, treatment burden, and quality of life; for cell/tissue therapies clinical benefit, uncertainty, risk and treatment burden. There is limited research on the preferences for ATMPs; but these findings suggest that the value attributes of ATMPs are consistent across gene and cell/tissue therapies with clinical benefit, uncertainty, risk, treatment burden, and quality of life being important. [ABSTRACT FROM AUTHOR]
Copyright of Inquiry (00469580) is the property of Sage Publications Inc. and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)
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  Data: Preferences for Advanced Therapy Medicinal Products: Understanding the Published Literature on the Value of Innovative Health Interventions.
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  Data: <searchLink fieldCode="AR" term="%22Ferizović%2C+N%2E%22">Ferizović, N.</searchLink><relatesTo>1</relatesTo> (AUTHOR)<i> Nermina.ferizovic.20@ucl.ac.uk</i><br /><searchLink fieldCode="AR" term="%22Lorgelly%2C+P%2E%22">Lorgelly, P.</searchLink><relatesTo>2</relatesTo> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Clarke%2C+C%2E+S%2E%22">Clarke, C. S.</searchLink><relatesTo>1</relatesTo> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Hunter%2C+R%2E+M%2E%22">Hunter, R. M.</searchLink><relatesTo>1</relatesTo> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Plackett%2C+R%2E%22">Plackett, R.</searchLink><relatesTo>1</relatesTo> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Abbas%2C+W%2E%22">Abbas, W.</searchLink><relatesTo>1</relatesTo> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Freemantle%2C+N%2E%22">Freemantle, N.</searchLink><relatesTo>1</relatesTo> (AUTHOR)
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  Data: <searchLink fieldCode="JN" term="%22Inquiry+%2800469580%29%22">Inquiry (00469580)</searchLink>. 11/1/2025, Vol. 62, p1-14. 14p.
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  Data: *<searchLink fieldCode="DE" term="%22Diffusion+of+innovations%22">Diffusion of innovations</searchLink><br />*<searchLink fieldCode="DE" term="%22Quality+of+life%22">Quality of life</searchLink><br /><searchLink fieldCode="DE" term="%22Gene+therapy%22">Gene therapy</searchLink><br /><searchLink fieldCode="DE" term="%22Medical+information+storage+%26+retrieval+systems%22">Medical information storage & retrieval systems</searchLink><br /><searchLink fieldCode="DE" term="%22Risk+assessment%22">Risk assessment</searchLink><br /><searchLink fieldCode="DE" term="%22Health+insurance+reimbursement%22">Health insurance reimbursement</searchLink><br /><searchLink fieldCode="DE" term="%22Tissue+engineering%22">Tissue engineering</searchLink><br /><searchLink fieldCode="DE" term="%22Cellular+therapy%22">Cellular therapy</searchLink><br /><searchLink fieldCode="DE" term="%22Decision+making+in+clinical+medicine%22">Decision making in clinical medicine</searchLink><br /><searchLink fieldCode="DE" term="%22Uncertainty%22">Uncertainty</searchLink><br /><searchLink fieldCode="DE" term="%22Treatment+effectiveness%22">Treatment effectiveness</searchLink><br /><searchLink fieldCode="DE" term="%22Systematic+reviews%22">Systematic reviews</searchLink><br /><searchLink fieldCode="DE" term="%22MEDLINE%22">MEDLINE</searchLink>
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  Data: Advanced therapy medicinal products (ATMPs) are ground-breaking genetic and cell/tissue-based therapies with the potential to treat and even cure myriad health conditions. However, they have faced multiple challenges in decision making with relatively low success in achieving reimbursement. The aim of this study was to review the published preference-based research on ATMPs to identify value attributes as a starting point in a research workstream aimed at understanding the true value of these innovative therapies. A literature review was used to identify studies across MedLine, Embase, Allied and Complementary Medicine Database, Global Health, ProQuest Central, Social Sciences Premium Collection, Applied Social Sciences Index and Abstracts, Econ Lit, Web of Science and SCOPUS from inception to 3rd March 2024. Search terms for genetic and cell/tissue-based therapies were conducted separately. Studies were included if they were a peer-reviewed primary research study or systematic literature review using stated preference measures to elicit preferences for ATMPs. Thirteen studies were included with 8 pertaining to genetic therapies and 5 to cell/tissue therapies. Attributes of value were similar across the studies and ATMP categories. Attributes were grouped into the following for genetic therapies: clinical benefits, uncertainty, risk, treatment burden, and quality of life; for cell/tissue therapies clinical benefit, uncertainty, risk and treatment burden. There is limited research on the preferences for ATMPs; but these findings suggest that the value attributes of ATMPs are consistent across gene and cell/tissue therapies with clinical benefit, uncertainty, risk, treatment burden, and quality of life being important. [ABSTRACT FROM AUTHOR]
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  Data: <i>Copyright of Inquiry (00469580) is the property of Sage Publications Inc. and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.)
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        Value: 10.1177/00469580251390763
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      – Code: eng
        Text: English
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        PageCount: 14
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    Subjects:
      – SubjectFull: Diffusion of innovations
        Type: general
      – SubjectFull: Quality of life
        Type: general
      – SubjectFull: Gene therapy
        Type: general
      – SubjectFull: Medical information storage & retrieval systems
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      – SubjectFull: Risk assessment
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      – SubjectFull: Tissue engineering
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      – SubjectFull: Cellular therapy
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      – SubjectFull: Decision making in clinical medicine
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      – SubjectFull: Uncertainty
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              Text: 11/1/2025
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