Search

Carol Mansfield,1 Lubomyra Nalysnyk,2 Dhaivat Joshi,2 Joshua Coulter,1 Ruth Pulikottil-Jacob3 1Health Preference Assessment, RTI Health Solutions, Research Triangle Park, North Carolina, USA; 2Health Economics and Value Assessment, Sanofi, Cambridge, MA, USA; 3Health Economics and Value Assessment, Sanofi, Thames Valley Park, Reading, UKCorrespondence: Ruth Pulikottil-Jacob, Sanofi, Thames Valley Park, Reading, RG6 1AD, UK, Tel +44-7525594087, Email ruth.jacob@sanofi.comPurpose: Acid sphingomyelinase deficiency (ASMD) is a rare, progressive, and potentially fatal disease affecting major organs; its symptoms present heterogeneously. Data on the most bothersome symptoms for patients with ASMD types B or A/B and their caregivers or parents are limited. We conducted a survey to quantify the relative impact of potential ASMD symptoms and risks for patients and parents/caregivers.Patients and Methods: Twenty respondents, recruited via National Niemann-Pick Disease Foundation (United States) and Niemann-Pick United Kingdom, took a preference survey: 11 patients who had a self-reported diagnosis of ASMD types B or A/B and 9 parents who had a child with ASMD types B or A/B. Using object-case best-worst scaling, we explored the most and least bothersome among a set of 15 ASMD symptoms/risks selected based on clinical input and qualitative research with patients and caregivers. In 15 experimentally designed questions containing five items each, respondents ranked the symptoms/risks, irrespective of their experiences with them. Data were analyzed using a conditional multinomial logit model.Results: Patients reported constant abdominal pain, severe pain in bones and joints, and severe fatigue to be the most bothersome potential symptoms or risks, followed by a chance of bleeding in the spleen. The next most bothersome potential symptom was constant shortness of breath. Easy bruising and noticeable abdominal enlargement were among the least bothersome symptoms. The most bothersom

Michael Serbin,1 Connie Marras,2 Carol Mansfield,3 Colton Leach,3 Charles Yonan,1 Margaret Sheehan,4 Anne Donnelly,5 Olga Klepitskaya1 1Neurocrine Biosciences, Inc., San Diego, CA, USA; 2The Edmond J. Safra Program in Parkinson’s Disease, University Health Network, University of Toronto, Toronto, ON, Canada; 3RTI Health Solutions, Research Triangle Park, NC, USA; 4Ashurst, Washington, DC, USA; 5Kellogg School of Management, Northwestern University, Evanston, IL, USACorrespondence: Carol Mansfield, RTI Health Solutions, 3040 East Cornwallis Road, PO Box 12194, Research Triangle Park, NC, 27709, USA, Tel +19195418053, Email carolm@rti.orgBackground: Several adjunctive medications are available to reduce OFF time between levodopa/carbidopa (LD/CD) doses for people with Parkinson’s disease (PD).Objective: To explore how individuals with PD balance benefits and burdens when considering adjunctive medications.Methods: US adults (30– 83 years) with self-reported PD, currently treated with LD/CD, who experienced OFF episodes were recruited through the Fox Insight study to complete a discrete-choice experiment survey. Respondents selected among experimentally designed profiles for hypothetical adjunctive PD treatments that varied in efficacy (additional ON time), potential adverse effects (troublesome dyskinesia, risk of diarrhea, risk of change in bodily fluid color), and dosing frequency or the option “No additional medicine”. Data were analyzed with random-parameters logit models.Results: Respondents (N=480) would require ≥ 60 additional minutes of daily ON time to accept either a 40% risk of change in bodily fluid color or 10 additional minutes with troublesome dyskinesia daily. Respondents would require 40 additional minutes of daily ON time to accept a 10% risk of diarrhea and 22 additional minutes of daily ON time to switch from 1 additional pill each day to 1 pill with each LD/CD dose. On average, respondents preferred adjunctive PD medication over no add

Daniel Auclair,1,* Carol Mansfield,2,* Mark A Fiala,3,* Ajai Chari,4 Craig E Cole,5 Jonathan L Kaufman,6 Gregory J Orloff,7 David S Siegel,8 Jeffrey A Zonder,9 Brennan Mange,2 Jennifer Yesil,1 Mehul Dalal,10 Joseph R Mikhael11 1Department of Research, Multiple Myeloma Research Foundation, Norwalk, CT, USA; 2RTI Health Solutions, Research Triangle Park, NC, USA; 3Division of Hematology & Oncology, Washington University School of Medicine, St. Louis, MO, USA; 4Icahn School of Medicine, Mount Sinai Hospital, New York, NY, USA; 5Department of Medicine, MSU Breslin Cancer Center, Lansing, MI, USA; 6Winship Cancer Institute, Emory University School of Medicine, Atlanta, GA, USA; 7Virginia Cancer Specialists, Fairfax, VA, USA; 8John Theurer Cancer Center, Hackensack University Medical Center, Hackensack, NJ, USA; 9Division of Clinical Hematology-Oncology, Barbara Ann Karmanos Cancer Institute, Detroit, MI, USA; 10Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical, Company Ltd, Cambridge, MA, USA; 11Translational Genomic Research Institute, City of Hope Cancer Center, Phoenix, AZ, USA*These authors contributed equally to this workCorrespondence: Daniel Auclair, AstraZeneca Oncology R&D| Hematology, 35 Gatehouse Drive, Waltham, MA, 02451, USA, Email daniel.auclair@astrazeneca.comIntroduction/Background: This study aimed to describe patient and caregiver preferences for treatments of relapsed or refractory multiple myeloma (MM).Materials and Methods: A survey including discrete-choice experiment (DCE) and best-worst scaling (BWS) exercises was conducted among US patients with relapsed or refractory MM and their caregivers. The DCE included six attributes with varying levels including progression-free survival (PFS), toxicity, and mode and frequency of administration. In addition, the impact of treatment cost was assessed using a fixed-choice question. The BWS exercise included 18 items (modes and freque

Andrew Thach,1 Jessie Sutphin,2 Joshua Coulter,2 Colton Leach,2 Eric Pappert,3 Carol Mansfield2 1Health Economics and Outcomes Research, Sunovion Pharmaceuticals Inc., Marlborough, MA, USA; 2Health Preference Assessment, RTI Health Solutions, Research Triangle Park, NC, USA; 3Medical Affairs, Sunovion Pharmaceuticals Inc., Marlborough, MA, USACorrespondence: Andrew ThachHealth Economics and Outcomes Research, Sunovion Pharmaceuticals Inc., 84 Waterford Dr, Marlborough, MA 01752, USATel +1 774-369-7123Email andrew.thach@sunovion.comIntroduction: Several on-demand treatments are available for management of “OFF” episodes in patients with Parkinson’s disease (PD). We evaluated patients’ preferences for features of theoretical on-demand treatment options.Methods: In a discrete choice experiment, US adults with self-reported PD of ≥ 5 years, or < 5 years with “OFF” episodes, taking oral carbidopa/levodopa, selected between pairs of theoretical on-demand treatments that varied by mode of administration (with and without mode-specific adverse events [AEs]), time to FULL “ON,” duration of “ON,” and out-of-pocket cost for a 30-day supply. Data were analyzed with a random parameters logit model; results were used to calculate relative importance of treatment attributes, preference shares, and willingness to pay.Results: Among 300 respondents, 98% had “OFF” episodes. Across the range of attribute levels included in the survey, avoiding $90 cost was most important to respondents, followed by a preferable mode of administration with associated AEs and decreasing time to FULL “ON.” Duration of “ON” was relatively less important. On average, respondents preferred a theoretical dissolvable sublingual film versus other theoretical treatments with alternative modes of administration. Respondents were willing to p

As new female-initiated HIV prevention products enter development, it is crucial to incorporate women's preferences to ensure products will be desired, accepted, and used. A discrete-choice experiment was designed to assess the relative importance of six attributes to stated choice of a vaginally delivered HIV prevention product. Sexually active women in South Africa and Zimbabwe aged 18-30 were recruited from two samples: product-experienced women from a randomized trial of four vaginal placebo forms and product-naïve community members. In a tablet-administered survey, 395 women chose between two hypothetical products over eight choice sets. Efficacy was the most important, but there were identifiable preferences among other attributes. Women preferred a product that also prevented pregnancy and caused some wetness (p < 0.001). They disliked a daily-use product (p = 0.002) and insertion by finger (p = 0.002). Although efficacy drove preference, wetness, pregnancy prevention, and dosing regimen were influential to stated choice of a product, and women were willing to trade some level of efficacy to have other more desired attributes.

Marjana Marinac,1 Jessie Sutphin,2 Campbell Hutton,1 Kathleen Klein,2 Sean Sullivan,3 Carol Mansfield2 1JDRF, Washington, DC, USA; 2RTI Health Solutions, Research Triangle Park, NC, USA; 3The Leona M. and Harry B. Helmsley Charitable Trust, New York, NY 10169, USACorrespondence: Carol Mansfield Email carolm@rti.orgPurpose: Hemoglobin A1c (HbA1c) is the accepted measure of effectiveness for type 1 diabetes therapies. We investigated preferences for measures of diabetes control in addition to HbA1c among adults with type 1 diabetes and caregivers of children with type 1 diabetes.Methods: Using discrete-choice experiment methodology, surveys for adults with type 1 diabetes and caregivers presented choices between hypothetical treatments described by six attributes with varying levels: HbA1c, time in optimal glucose range, weekly number and severity of hypoglycemic and hyperglycemic events, additional disease management time, and additional treatment cost. Choice data were analyzed using random-parameters logit.Results: A total of 300 adults with type 1 diabetes and 400 caregivers completed the survey. Adults and caregivers placed the most importance on reducing hypoglycemic and hyperglycemic events. For adults, avoiding 1– 5 mild-to-moderate hypoglycemic events (glucose 54– 69 mg/dL)/week was five times more important than being a half-point above target HbA1c. Avoiding 1– 5 hyperglycemic events (glucose > 180 mg/dL)/week was seven times more important than being a half-point above target HbA1c. Additional time in optimal glucose range was as important as a reduction greater than a half-point in HbA1c. Avoiding hyperglycemic and hypoglycemic events was more important than all other outcomes for caregivers of younger children. Caregivers of children > 12 years placed relatively more weight on avoiding hypoglycemic events < 54 mg/dL than those with younger children and preferred avoiding additional costs.Conclusion: Adul

The purpose of this analysis is to evaluate the potential economic impacts associated with the proposed critical habitat designation for the Coral Pink Sand Dunes (CPSD) tiger beetle. The analysis considers current and future impacts on economic efficiency as well as the distribution of economic impacts that may result from efforts to protect the CPSD tiger beetle and its habitat. In addition, the evaluation considers the benefits of the proposed action; distributional impacts on small businesses; whether the proposed listing or designation can be seen as an unfunded mandate on local government; and whether the action would significantly affect energy supply, distribution, and use. In accordance with the 10th Circuit U.S. Court of Appeals ruling in New Mexico Cattle Growers Association v. U. S. Fish and Wildlife Service (Service), this analysis considers the co-extensive cost impacts of the proposed critical habitat’s protection. Co-extensive impacts include baseline impacts, which are a result of the proposed listing, and incremental impacts, which are solely attributable to the proposed designation of critical habitat.