Patient Preferences for Benefits, Risks, and Administration Route of Hypomethylating Agents in Myelodysplastic Syndromes.

INTRODUCTION/BACKGROUND: Therapy with infused or injected hypomethylating agents (HMAs) may lead to higher treatment administration burden (ie, local reaction, visit frequency and duration) vs. oral HMAs.   OBJECTIVES: To reveal preferences of US and Canadian patients with myelodysplastic syndromes (MDS) for HMAs' benefits, risks, and administration burden through an online discrete-choice experiment (DCE). MATERIALS AND METHODS: Choice of DCE attributes and survey development were informed by literature review and interviews with clinicians, MDS patients, and caregivers serving as patient proxies, and patient advocacy groups (PAGs) representatives, including from AAMAC, AAMDS, and MDSF. DCE choice tasks were analyzed using random parameter logit models. Survey patients were recruited by the PAGs via their networks. To understand key preference drivers and how much patients were willing to trade between attributes, we calculated each attribute's relative attribute importance (RAI) and marginal rates of substitution. RESULTS: One hundred eighty-four respondents (including 158 patients; mean age, 67.2 years; male, 50.5%; White, 50.5%; US residents, 88%) completed the survey. MDS risk was low (34.8%), high (30.9%), or unknown (34.2%). RAI (in decreasing order) was as follows: risk of AML (40%), fatigue level (33%), number of visits (12%), mode of administration (6%), visit duration (5%), and administration frequency (4%). Assuming the same risk of AML transformation or level of fatigue, most respondents (76.6%) were predicted to switch to an oral pill if it were available to them. CONCLUSION: Given equivalent effectiveness across HMAs, patients' preferences for HMA administration method should be considered in treatment decision-making to minimize burden and facilitate adherence.

Physician preferences for attributes of pediatric combination vaccines in the United States.

OBJECTIVE: To understand physician preferences for various attributes of pediatric combination vaccines. METHODS: An online survey was completed by 400 US physicians (pediatricians and family physicians) who routinely administer vaccines to infants aged 1-12 months in outpatient settings. Respondents completed a discrete choice experiment (DCE) by selecting their preferred options from different hypothetical vaccine profiles with systematic variation in the levels of five attributes: vaccine presentation, number of injections administered at a single visit, completion rates, timeliness rates (within 30 days of recommended age), and years of availability for routine use, assuming similar cost, safety, and efficacy. Odds ratios and relative attribute importance scores were estimated using a random parameters logit model. RESULTS: Physicians (mean age 50.4 years, 52.5% women) preferred combination vaccines that reduced the number of injections administered at a single visit, facilitated higher completion and timeliness rates for the primary DTaP series, were available as a pre-filled syringe rather than a vial needing reconstitution and had been available for routine use for more than 1 year. All odds ratios were statistically significant. Physicians were twice as likely to prefer administering three injections in a single visit instead of four. The most important attribute was the number of injections administered at a single visit (relative importance 38%), followed by timeliness, completion rates, and vaccine presentation; years a vaccine has been available was the least important attribute. CONCLUSION: US physicians prefer pediatric combination vaccines that enable fewer injections to be administered at a single visit, facilitate higher completion and timeliness rates, are offered as a pre-filled syringe, and have been available for routine use for more than 1 year. The most important attribute of pediatric combination vaccines was a reduction in the number of injections administered at a single visit.