Family, healthcare professional, and societal preferences for the treatment of infantile spinal muscular atrophy: a discrete choice experiment

2021 ◽  
Author(s):  
Kate A Carey ◽  
Michelle A Farrar ◽  
Nadine A Kasparian ◽  
Deborah J Street ◽  
Richard De Abreu Lourenco
Keyword(s):  
Healthcare Professional ◽  
Spinal Muscular Atrophy ◽  
Discrete Choice Experiment ◽  
Discrete Choice ◽  
Choice Experiment ◽  
Muscular Atrophy ◽  
Societal Preferences

scholarly journals Treatment Preference Among Patients With Spinal Muscular Atrophy (SMA): A Discrete Choice Experiment

2020 ◽  
Author(s):  
Alisha Monnette ◽  
Er Chen ◽  
Dongzhe Hong ◽  
Alessandra Bazzano ◽  
Stacy Dixon ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Discrete Choice Experiment ◽  
Discrete Choice ◽  
Choice Experiment ◽  
Muscular Atrophy ◽  
Route Of Administration ◽  
Oral Medication ◽  
Treatment Preference ◽  
Minimal Risk ◽  
Trade Off

Abstract OBJECTIVE: To examine patient/caregiver preference for key attributes of treatments for spinal muscular atrophy (SMA). BACKGROUND: In the rapidly evolving SMA treatment landscape, it is critically important to understand how attributes of potential treatments may impact patient/caregiver choices.DESIGN/METHODS: A discrete choice experiment survey was developed based on qualitative interviews. Patients with SMA (18 years) and caregivers of patients were recruited through a U.S. patient organization. Respondents made choices in each of 12 sets of hypothetical treatments. The relative importance of five treatment characteristics was compared (measured by regression coefficients [RC] of conditional logit models): (1) improvement or stabilization of motor function, (2) improvement or stabilization of breathing function, (3) indication for all ages or pediatric patients only, (4) route of administration (repeated intrathecal [IT] injections, one-time intravenous [IV] infusion, daily oral delivery) and (5) potential harm (mild, moderate, serious/life threatening).RESULTS: Patient ages ranged from less than 1 to 67 years (n=101, 65 self-reported and 36 caregiver-reported) and 64 were female. Total SMA subtypes included: type 1 (n=21), type 2 (n=48), type 3 (n=29), other (n=3). Prior spinal surgery was reported in 47 patients. Nusinersen and onasemnogene abeparvovec-xioi use were reported in 59 and 10 patients, respectively. Improvement in motor and breathing function was highly valued (RC: 0.65, 95% confidence interval [CI]: 0.47–0.83 and RC: 0.79, 95% CI: 0.60–0.98, respectively). Oral medication and one-time infusion were strongly preferred over repeated IT injections (RC: 0.80, 95% CI: 0.60–0.98 and RC: 0.51, 95% CI: 0.30–0.73, respectively). Patients least preferred an age-restricted label/approved use ( 2 years of age) (RC: -1.28, 95% CI: -1.47 to -1.09). Cross-attributes trade-off decision suggested a lower willingness for a high-risk therapy despite additional efficacy gain. For some patients, there may be willingness to trade off additional gains in efficacy for a change in route of administration from repeated intrathecal administration to oral medication.CONCLUSIONS: Improvements in motor/breathing function, broad indication, oral or one-time infusion, and minimal risk were preferred treatment attributes. Treatment decisions should be made in clinical context and be tailored to patient needs.

scholarly journals Treatment Preference among Patients with Spinal Muscular Atrophy (SMA): A Discrete Choice Experiment

2020 ◽  
Author(s):  
Alisha Monnette ◽  
Er Chen ◽  
Dongzhe Hong ◽  
Alessandra Bazzano ◽  
Stacy Dixon ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Discrete Choice Experiment ◽  
Discrete Choice ◽  
Choice Experiment ◽  
Muscular Atrophy ◽  
Route Of Administration ◽  
Oral Medication ◽  
Treatment Preference ◽  
Minimal Risk ◽  
Trade Off

Abstract OBJECTIVE: To examine patient/caregiver preference for key attributes of treatments for spinal muscular atrophy (SMA). BACKGROUND: In the rapidly evolving SMA treatment landscape, it is critically important to understand how attributes of potential treatments may impact patient/caregiver choices.DESIGN/METHODS: A discrete choice experiment survey was developed based on qualitative interviews. Patients with SMA (³18 years) and caregivers of patients were recruited through a U.S. patient organization. Respondents made choices in each of 12 sets of hypothetical treatments. The relative importance of five treatment characteristics was compared (measured by regression coefficients [RC] of conditional logit models): (1) improvement or stabilization of motor function, (2) improvement or stabilization of breathing function, (3) indication for all ages or pediatric patients only, (4) route of administration (repeated intrathecal [IT] injections, one-time intravenous [IV] infusion, daily oral delivery) and (5) potential harm (mild, moderate, serious/life threatening).RESULTS: Patient ages ranged from less than 1 to 67 years (n=101, 65 self-reported and 36 caregiver-reported) and 64 were female. Total SMA subtypes included: type 1 (n=21), type 2 (n=48), type 3 (n=29), other (n=3). Prior spinal surgery was reported in 47 patients. Nusinersen and onasemnogene abeparvovec-xioi use were reported in 59 and 10 patients, respectively. Improvement in motor and breathing function was highly valued (RC: 0.65, 95% confidence interval [CI]: 0.47–0.83 and RC: 0.79, 95% CI: 0.60–0.98, respectively). Oral medication and one-time infusion were strongly preferred over repeated IT injections (RC: 0.80, 95% CI: 0.60–0.98 and RC: 0.51, 95% CI: 0.30–0.73, respectively). Patients least preferred an age-restricted label/approved use (£ 2 years of age) (RC: -1.28, 95% CI: -1.47 to -1.09). Cross-attributes trade-off decision suggested a lower willingness for a high-risk therapy despite additional efficacy gain. For some patients, there may be willingness to trade off additional gains in efficacy for a change in route of administration from repeated intrathecal administration to oral medication.CONCLUSIONS: Improvements in motor/breathing function, broad indication, oral or one-time infusion, and minimal risk were preferred treatment attributes. Treatment decisions should be made in clinical context and be tailored to patient needs.

scholarly journals Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Alisha Monnette ◽  
Er Chen ◽  
Dongzhe Hong ◽  
Alessandra Bazzano ◽  
Stacy Dixon ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Discrete Choice Experiment ◽  
Discrete Choice ◽  
Choice Experiment ◽  
Muscular Atrophy ◽  
Route Of Administration ◽  
Oral Medication ◽  
Treatment Preference ◽  
Minimal Risk ◽  
Trade Off

Abstract Objective To examine patient/caregiver preference for key attributes of treatments for spinal muscular atrophy (SMA). Background In the rapidly evolving SMA treatment landscape, it is critically important to understand how attributes of potential treatments may impact patient/caregiver choices. Design/methods A discrete choice experiment survey was developed based on qualitative interviews. Patients with SMA (≥ 18 years) and caregivers of patients were recruited through a U.S. patient organization. Respondents made choices in each of 12 sets of hypothetical treatments. The relative importance of five treatment characteristics was compared (measured by regression coefficients [RC] of conditional logit models): (1) improvement or stabilization of motor function, (2) improvement or stabilization of breathing function, (3) indication for all ages or pediatric patients only, (4) route of administration [repeated intrathecal (IT) injections, one-time intravenous (IV) infusion, daily oral delivery] and (5) potential harm (mild, moderate, serious/life threatening). Results Patient ages ranged from less than 1 to 67 years (n = 101, 65 self-reported and 36 caregiver-reported) and 64 were female. Total SMA subtypes included: type 1 (n = 21), type 2 (n = 48), type 3 (n = 29), other (n = 3). Prior spinal surgery was reported in 47 patients. Nusinersen and onasemnogene abeparvovec-xioi use were reported in 59 and 10 patients, respectively. Improvement in motor and breathing function was highly valued [RC: 0.65, 95% confidence interval (CI): 0.47–0.83 and RC: 0.79, 95% CI: 0.60–0.98, respectively]. Oral medication and one-time infusion were strongly preferred over repeated IT injections (RC: 0.80, 95% CI: 0.60–0.98 and RC: 0.51, 95% CI: 0.30–0.73, respectively). Patients least preferred an age-restricted label/approved use (≤ 2 years of age) (RC: − 1.28, 95% CI: − 1.47 to − 1.09). Cross-attributes trade-off decision suggested a lower willingness for a high-risk therapy despite additional efficacy gain. For some patients, there may be willingness to trade off additional gains in efficacy for a change in route of administration from repeated intrathecal administration to oral medication. Conclusions Improvements in motor/breathing function, broad indication, oral or one-time infusion, and minimal risk were preferred treatment attributes. Treatment decisions should be made in clinical context and be tailored to patient needs.

scholarly journals Societal preferences for rheumatoid arthritis treatments: evidence from a discrete choice experiment

Rheumatology ◽  
2015 ◽  
Vol 54 (10) ◽  
pp. 1816-1825 ◽  
Author(s):  
Mark Harrison ◽  
Carlo Marra ◽  
Kam Shojania ◽  
Nick Bansback
Keyword(s):  
Rheumatoid Arthritis ◽  
Discrete Choice Experiment ◽  
Discrete Choice ◽  
Choice Experiment ◽  
Societal Preferences

scholarly journals Society’s View on Autonomous Agriculture: Does Digitalization Lead to Alienation?

2021 ◽  
Vol 9 (1) ◽  
pp. 12
Author(s):  
Olivia Spykman ◽  
Agnes Emberger-Klein ◽  
Andreas Gabriel ◽  
Markus Gandorfer
Keyword(s):  
Weed Control ◽  
Discrete Choice Experiment ◽  
Discrete Choice ◽  
Choice Experiment ◽  
Agricultural Market ◽  
The Public ◽  
Control Technologies ◽  
Societal Preferences

Digital and autonomous technologies enter the agricultural market at an increasing rate, yet little is known about society’s view on this development, although the public is an important stakeholder. By means of a discrete choice experiment (n = 645), societal preferences for different weed control technologies and tractor types of different degrees of autonomy are investigated. The model applied focuses on emotion-related covariates. The results indicate preferences for conventional or autonomous tractors and for methods of weed control that reduce the need for herbicides. Additionally, positive associations with images of robots correlate with the rejection of conventional tractors in the discrete choice experiment.

scholarly journals Societal preferences for the treatment of impulsive-violent offenders: a discrete choice experiment

BMJ Open ◽  
2021 ◽  
Vol 11 (6) ◽  
pp. e033935
Author(s):  
Stella Settumba ◽  
Tony Butler ◽  
Peter Schofield ◽  
Georgina M Chambers ◽  
Marian Shanahan
Keyword(s):  
Discrete Choice Experiment ◽  
Discrete Choice ◽  
Choice Experiment ◽  
Latent Class ◽  
Violent Offenders ◽  
South Wales ◽  
Trade Offs ◽  
Study Participants ◽  
Tax Payers ◽  
Societal Preferences

ObjectivesThe aim of this study is to quantify societal preferences for, and assess trade-offs between characteristics of treatment programmes for impulsive-violent offenders.SettingThe study was conducted in New South Wales, Australia’s largest state.ParticipantsThe study participants were income tax payers, aged over 18 and who were able to provide informed consent.MethodsA discrete choice experiment was used to assess the preferences for treatment programmes for impulsive violent offenders. The survey presented participants with six choice sets in which they chose between two unlabelled treatment scenarios and a ‘no treatment’ choice. A random parameters logistic (RPL) model and a latent class (LC) model were used to analyse the societal preferences for treatment and estimate willingness to pay values based on marginal rates of substitution. Respondents were asked to self-identify if they ever had experiences with violence and subgroup analysis was done.ResultsThe survey was completed by 1021 highly engaged participants. The RPL model showed that society had a preference for more effective programmes, programmes that provided full as opposed to partial treatment of all co-occurring health conditions, compulsory over voluntary programmes, those with flexibility in appointments and programmes that are provided with continuity of care postprison. Respondents were willing to pay an additional annual tax contribution for all significant attributes, particularly compulsory programmes, continuity of treatment and effectiveness.The LC model identified two classes of respondents with some differences in preferences which could be largely identified by whether they had experiences with violence or not.ConclusionThe results are important for future programme design and implementation. Programmes for impulsive violent offenders that are designed to encompass societal preferences are likely to be supported by public and tax payers.

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