Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers

Background Rare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context. Methods A mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents. Results Final study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk. Conclusions This is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. Researchers of novel therapeutics for rare diseases should be encouraged to invest in preference elicitation studies to generate rigorous patient evidence and specific regulatory guidance should be issued to acknowledge their importance and their use in marketing authorisations. Ausführliche Beschreibung

Gespeichert in:

Autor*in:	Morel, T. [verfasserIn] Aymé, S. Cassiman, D. Simoens, S. Morgan, M. Vandebroek, M.

Format:	E-Artikel
Sprache:	Englisch

Erschienen:	2016

Schlagwörter:	Rare diseases Patients Caregivers Benefit-risk Preferences Values Trade-offs Risk tolerance Discrete choice experiment Qualitative survey Disability Patient-centered outcomes Patient-reported outcomes Drug development

Anmerkung:	© Morel et al. 2016

Übergeordnetes Werk:	Enthalten in: Orphanet journal of rare diseases - London : BioMed Central, 2006, 11(2016), 1 vom: 26. Mai
Übergeordnetes Werk:	volume:11 ; year:2016 ; number:1 ; day:26 ; month:05

Links:	Volltext

DOI / URN:	10.1186/s13023-016-0444-9

Katalog-ID:	SPR029495806

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520			\|a Background Rare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context. Methods A mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents. Results Final study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk. Conclusions This is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. Researchers of novel therapeutics for rare diseases should be encouraged to invest in preference elicitation studies to generate rigorous patient evidence and specific regulatory guidance should be issued to acknowledge their importance and their use in marketing authorisations.
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allfields	10.1186/s13023-016-0444-9 doi (DE-627)SPR029495806 (SPR)s13023-016-0444-9-e DE-627 ger DE-627 rakwb eng Morel, T. verfasserin aut Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers 2016 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Morel et al. 2016 Background Rare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context. Methods A mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents. Results Final study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk. Conclusions This is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. Researchers of novel therapeutics for rare diseases should be encouraged to invest in preference elicitation studies to generate rigorous patient evidence and specific regulatory guidance should be issued to acknowledge their importance and their use in marketing authorisations. Rare diseases (dpeaa)DE-He213 Patients (dpeaa)DE-He213 Caregivers (dpeaa)DE-He213 Benefit-risk (dpeaa)DE-He213 Preferences (dpeaa)DE-He213 Values (dpeaa)DE-He213 Trade-offs (dpeaa)DE-He213 Risk tolerance (dpeaa)DE-He213 Discrete choice experiment (dpeaa)DE-He213 Qualitative survey (dpeaa)DE-He213 Disability (dpeaa)DE-He213 Patient-centered outcomes (dpeaa)DE-He213 Patient-reported outcomes (dpeaa)DE-He213 Drug development (dpeaa)DE-He213 Aymé, S. aut Cassiman, D. aut Simoens, S. aut Morgan, M. aut Vandebroek, M. aut Enthalten in Orphanet journal of rare diseases London : BioMed Central, 2006 11(2016), 1 vom: 26. Mai (DE-627)50900637X (DE-600)2225857-7 1750-1172 nnns volume:11 year:2016 number:1 day:26 month:05 https://dx.doi.org/10.1186/s13023-016-0444-9 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 11 2016 1 26 05
spelling	10.1186/s13023-016-0444-9 doi (DE-627)SPR029495806 (SPR)s13023-016-0444-9-e DE-627 ger DE-627 rakwb eng Morel, T. verfasserin aut Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers 2016 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Morel et al. 2016 Background Rare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context. Methods A mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents. Results Final study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk. Conclusions This is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. Researchers of novel therapeutics for rare diseases should be encouraged to invest in preference elicitation studies to generate rigorous patient evidence and specific regulatory guidance should be issued to acknowledge their importance and their use in marketing authorisations. Rare diseases (dpeaa)DE-He213 Patients (dpeaa)DE-He213 Caregivers (dpeaa)DE-He213 Benefit-risk (dpeaa)DE-He213 Preferences (dpeaa)DE-He213 Values (dpeaa)DE-He213 Trade-offs (dpeaa)DE-He213 Risk tolerance (dpeaa)DE-He213 Discrete choice experiment (dpeaa)DE-He213 Qualitative survey (dpeaa)DE-He213 Disability (dpeaa)DE-He213 Patient-centered outcomes (dpeaa)DE-He213 Patient-reported outcomes (dpeaa)DE-He213 Drug development (dpeaa)DE-He213 Aymé, S. aut Cassiman, D. aut Simoens, S. aut Morgan, M. aut Vandebroek, M. aut Enthalten in Orphanet journal of rare diseases London : BioMed Central, 2006 11(2016), 1 vom: 26. Mai (DE-627)50900637X (DE-600)2225857-7 1750-1172 nnns volume:11 year:2016 number:1 day:26 month:05 https://dx.doi.org/10.1186/s13023-016-0444-9 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 11 2016 1 26 05
allfields_unstemmed	10.1186/s13023-016-0444-9 doi (DE-627)SPR029495806 (SPR)s13023-016-0444-9-e DE-627 ger DE-627 rakwb eng Morel, T. verfasserin aut Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers 2016 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Morel et al. 2016 Background Rare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context. Methods A mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents. Results Final study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk. Conclusions This is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. Researchers of novel therapeutics for rare diseases should be encouraged to invest in preference elicitation studies to generate rigorous patient evidence and specific regulatory guidance should be issued to acknowledge their importance and their use in marketing authorisations. Rare diseases (dpeaa)DE-He213 Patients (dpeaa)DE-He213 Caregivers (dpeaa)DE-He213 Benefit-risk (dpeaa)DE-He213 Preferences (dpeaa)DE-He213 Values (dpeaa)DE-He213 Trade-offs (dpeaa)DE-He213 Risk tolerance (dpeaa)DE-He213 Discrete choice experiment (dpeaa)DE-He213 Qualitative survey (dpeaa)DE-He213 Disability (dpeaa)DE-He213 Patient-centered outcomes (dpeaa)DE-He213 Patient-reported outcomes (dpeaa)DE-He213 Drug development (dpeaa)DE-He213 Aymé, S. aut Cassiman, D. aut Simoens, S. aut Morgan, M. aut Vandebroek, M. aut Enthalten in Orphanet journal of rare diseases London : BioMed Central, 2006 11(2016), 1 vom: 26. Mai (DE-627)50900637X (DE-600)2225857-7 1750-1172 nnns volume:11 year:2016 number:1 day:26 month:05 https://dx.doi.org/10.1186/s13023-016-0444-9 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 11 2016 1 26 05
allfieldsGer	10.1186/s13023-016-0444-9 doi (DE-627)SPR029495806 (SPR)s13023-016-0444-9-e DE-627 ger DE-627 rakwb eng Morel, T. verfasserin aut Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers 2016 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Morel et al. 2016 Background Rare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context. Methods A mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents. Results Final study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk. Conclusions This is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. Researchers of novel therapeutics for rare diseases should be encouraged to invest in preference elicitation studies to generate rigorous patient evidence and specific regulatory guidance should be issued to acknowledge their importance and their use in marketing authorisations. Rare diseases (dpeaa)DE-He213 Patients (dpeaa)DE-He213 Caregivers (dpeaa)DE-He213 Benefit-risk (dpeaa)DE-He213 Preferences (dpeaa)DE-He213 Values (dpeaa)DE-He213 Trade-offs (dpeaa)DE-He213 Risk tolerance (dpeaa)DE-He213 Discrete choice experiment (dpeaa)DE-He213 Qualitative survey (dpeaa)DE-He213 Disability (dpeaa)DE-He213 Patient-centered outcomes (dpeaa)DE-He213 Patient-reported outcomes (dpeaa)DE-He213 Drug development (dpeaa)DE-He213 Aymé, S. aut Cassiman, D. aut Simoens, S. aut Morgan, M. aut Vandebroek, M. aut Enthalten in Orphanet journal of rare diseases London : BioMed Central, 2006 11(2016), 1 vom: 26. Mai (DE-627)50900637X (DE-600)2225857-7 1750-1172 nnns volume:11 year:2016 number:1 day:26 month:05 https://dx.doi.org/10.1186/s13023-016-0444-9 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 11 2016 1 26 05
allfieldsSound	10.1186/s13023-016-0444-9 doi (DE-627)SPR029495806 (SPR)s13023-016-0444-9-e DE-627 ger DE-627 rakwb eng Morel, T. verfasserin aut Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers 2016 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Morel et al. 2016 Background Rare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context. Methods A mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents. Results Final study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk. Conclusions This is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. Researchers of novel therapeutics for rare diseases should be encouraged to invest in preference elicitation studies to generate rigorous patient evidence and specific regulatory guidance should be issued to acknowledge their importance and their use in marketing authorisations. Rare diseases (dpeaa)DE-He213 Patients (dpeaa)DE-He213 Caregivers (dpeaa)DE-He213 Benefit-risk (dpeaa)DE-He213 Preferences (dpeaa)DE-He213 Values (dpeaa)DE-He213 Trade-offs (dpeaa)DE-He213 Risk tolerance (dpeaa)DE-He213 Discrete choice experiment (dpeaa)DE-He213 Qualitative survey (dpeaa)DE-He213 Disability (dpeaa)DE-He213 Patient-centered outcomes (dpeaa)DE-He213 Patient-reported outcomes (dpeaa)DE-He213 Drug development (dpeaa)DE-He213 Aymé, S. aut Cassiman, D. aut Simoens, S. aut Morgan, M. aut Vandebroek, M. aut Enthalten in Orphanet journal of rare diseases London : BioMed Central, 2006 11(2016), 1 vom: 26. Mai (DE-627)50900637X (DE-600)2225857-7 1750-1172 nnns volume:11 year:2016 number:1 day:26 month:05 https://dx.doi.org/10.1186/s13023-016-0444-9 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 11 2016 1 26 05
language	English
source	Enthalten in Orphanet journal of rare diseases 11(2016), 1 vom: 26. Mai volume:11 year:2016 number:1 day:26 month:05
sourceStr	Enthalten in Orphanet journal of rare diseases 11(2016), 1 vom: 26. Mai volume:11 year:2016 number:1 day:26 month:05
format_phy_str_mv	Article
institution	findex.gbv.de
topic_facet	Rare diseases Patients Caregivers Benefit-risk Preferences Values Trade-offs Risk tolerance Discrete choice experiment Qualitative survey Disability Patient-centered outcomes Patient-reported outcomes Drug development
isfreeaccess_bool	true
container_title	Orphanet journal of rare diseases
authorswithroles_txt_mv	Morel, T. @@aut@@ Aymé, S. @@aut@@ Cassiman, D. @@aut@@ Simoens, S. @@aut@@ Morgan, M. @@aut@@ Vandebroek, M. @@aut@@
publishDateDaySort_date	2016-05-26T00:00:00Z
hierarchy_top_id	50900637X
id	SPR029495806
language_de	englisch
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This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context. Methods A mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents. Results Final study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk. Conclusions This is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. 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author	Morel, T.
spellingShingle	Morel, T. misc Rare diseases misc Patients misc Caregivers misc Benefit-risk misc Preferences misc Values misc Trade-offs misc Risk tolerance misc Discrete choice experiment misc Qualitative survey misc Disability misc Patient-centered outcomes misc Patient-reported outcomes misc Drug development Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers
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topic_title	Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers Rare diseases (dpeaa)DE-He213 Patients (dpeaa)DE-He213 Caregivers (dpeaa)DE-He213 Benefit-risk (dpeaa)DE-He213 Preferences (dpeaa)DE-He213 Values (dpeaa)DE-He213 Trade-offs (dpeaa)DE-He213 Risk tolerance (dpeaa)DE-He213 Discrete choice experiment (dpeaa)DE-He213 Qualitative survey (dpeaa)DE-He213 Disability (dpeaa)DE-He213 Patient-centered outcomes (dpeaa)DE-He213 Patient-reported outcomes (dpeaa)DE-He213 Drug development (dpeaa)DE-He213
topic	misc Rare diseases misc Patients misc Caregivers misc Benefit-risk misc Preferences misc Values misc Trade-offs misc Risk tolerance misc Discrete choice experiment misc Qualitative survey misc Disability misc Patient-centered outcomes misc Patient-reported outcomes misc Drug development
topic_unstemmed	misc Rare diseases misc Patients misc Caregivers misc Benefit-risk misc Preferences misc Values misc Trade-offs misc Risk tolerance misc Discrete choice experiment misc Qualitative survey misc Disability misc Patient-centered outcomes misc Patient-reported outcomes misc Drug development
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title	Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers
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title_full	Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers
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author_browse	Morel, T. Aymé, S. Cassiman, D. Simoens, S. Morgan, M. Vandebroek, M.
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doi_str_mv	10.1186/s13023-016-0444-9
title_sort	quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers
title_auth	Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers
abstract	Background Rare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context. Methods A mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents. Results Final study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk. Conclusions This is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. Researchers of novel therapeutics for rare diseases should be encouraged to invest in preference elicitation studies to generate rigorous patient evidence and specific regulatory guidance should be issued to acknowledge their importance and their use in marketing authorisations. © Morel et al. 2016
abstractGer	Background Rare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context. Methods A mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents. Results Final study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk. Conclusions This is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. Researchers of novel therapeutics for rare diseases should be encouraged to invest in preference elicitation studies to generate rigorous patient evidence and specific regulatory guidance should be issued to acknowledge their importance and their use in marketing authorisations. © Morel et al. 2016
abstract_unstemmed	Background Rare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context. Methods A mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents. Results Final study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk. Conclusions This is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. Researchers of novel therapeutics for rare diseases should be encouraged to invest in preference elicitation studies to generate rigorous patient evidence and specific regulatory guidance should be issued to acknowledge their importance and their use in marketing authorisations. © Morel et al. 2016
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title_short	Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers
url	https://dx.doi.org/10.1186/s13023-016-0444-9
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author2	Aymé, S. Cassiman, D. Simoens, S. Morgan, M. Vandebroek, M.
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Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers

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