Albert Hauber

Treatment preferences and medication adherence of people with Type 2 diabetes using oral glucose-lowering agents

Aims  Medication non‐adherence is particularly common in patients with Type 2 diabetes. We constructed a discrete‐choice experiment to examine the relative importance of oral glucose‐lowering medication features and to estimate the likely effect of effectiveness and side effects on medication adherence in patients with Type 2 diabetes in the UK and the USA.

Patient Benefit-Risk Preferences for Targeted Agents in the Treatment of Renal Cell Carcinoma

Background: Angiogenesis inhibitor agents have been shown to be effective in increasing progression-free survival (PFS) in patients with renal cell carcinoma (RCC); however, these treatments have different toxicity profiles. Objective: Our objective was to quantify patients’ benefit-risk preferences for RCC treatments and relative importance of attributes in a common metric.Methods: US residents aged ≥18 years with RCC completed a web-enabled, choice-format conjoint survey that presented a series of 12 trade-off questions, each including a pair of hypothetical RCC treatment profiles. Each profile was defined by efficacy (PFS, when overall survival held constant), tolerability effects (fatigue/tiredness, diarrhoea, hand-foot syndrome [HFS], mouth sores) and serious adverse events (liver failure, blood clot). Trade-off questions were based on a predetermined experimental design with known statistical properties. Random-parameters logit was used to analyse the data.Results: A total of 138 patients completed the survey. PFS was the most important attribute for patients over the range of levels included in the survey, while remaining attributes were ranked in decreasing order of importance: fatigue/tiredness, diarrhoea, liver failure, HFS, blood clot and mouth sores. In order to increase PFS by 11 months, patients would be willing to accept a maximum level of absolute blood clot risk of 3.1%(95%CI 1.5, 5.3) or liver failure risk of 2.0% (95% CI 1.0, 3.3).Conclusion: A 22-month change in PFS was shown to be the most important improvement for patients. Severe fatigue/tiredness and diarrhoea were rated as the most troublesome tolerability effects of RCC treatment. Patients were likely willing to accept significant treatment-related risks of 2–3% for liver failure and blood clot to increase PFS by 11 months.

Effect of pill burden on dosing preferences, willingness to pay, and likely adherence among patients with type 2 diabetes

Purpose To quantify willingness-to-pay (WTP) for reducing pill burden and dosing frequency among patients with type 2 diabetes mellitus (T2DM), and to examine the effect of dosing frequency and pill burden on likely medication adherence. Patients and methods Participants were US adults with T2DM on oral antihyperglycemic therapy. Each patient completed an online discrete-choice experiment (DCE) with eight choice questions, each including a pair of hypothetical medication profiles. Each profile was defined by reduction in average glucose (AG), daily dosing, chance of mild-to-moderate stomach problems, frequency of hypoglycemia, weight change, incremental risk of congestive heart failure (CHF), and cost. Patients were asked to rate their likely adherence to the profiles presented in each question. Choice questions were based on a predetermined experimental design. Choice data were analyzed using random-parameters logit. Likely treatment adherence was analyzed using a Heckman two-stage model. Results Of the 1,114 patients who completed the survey, 90 had lower dosing burden (<5 pills/day taken once/day or as needed) for all medications, and 1,024 had higher dosing burden (≥5 pills/day or more than once/day). Reduction in AG was valued most highly by patients. Hypoglycemia, chance of mild-to-moderate stomach problems, weight change, incremental risk of CHF, and daily dosing were less valued. Patients with higher current dosing burden had lower WTP for more convenient dosing schedules than patients with lower current dosing burden. Changes in dosing and cost impacted likely adherence. The magnitude of the impact of dosing on likely adherence was higher for patients with lower current dosing burden than for patients with higher current dosing burden. Conclusion Patients with T2DM were willing to pay for improvements in efficacy, side effects, and dosing. Patients’ WTP for more convenient dosing depended on current dosing burden, as did the effect of these attributes on likely adherence.

Benefits, Risk, and Uncertainty: Preferences of Antiretroviral-Naïve African Americans for HIV Treatments

While African Americans in the United States are disproportionately affected by HIV, they are less likely to take antiretroviral therapies. Different first-line antiretroviral therapies are associated with short-term and long-term adverse event (AE) risks. We estimated the willingness of antiretroviral-naïve, HIV-positive African Americans to accept risks of acute AEs with known outcomes and long-term AEs with uncertain outcomes in exchange for virologic suppression. We estimated the relative importance of short-term and long-term AE risks. Two hundred thirty-five subjects were recruited through eight clinics in the United States. One hundred fifty-eight subjects met study inclusion criteria. One hundred fifty-three subjects completed a series of choice-format conjoint trade-off tasks. In each task, subjects were asked to choose between two hypothetical treatments with varying levels of virologic failure, risks of hypersensitivity reaction, decreases in bone mineral density (BMD), and renal impairment, and outcome uncertainty associated with the risks of decreased BMD and renal impairment. Attributes were expressed as probabilities of occurrence. We calculated the relative importance of each AE and the level of risk subjects would accept to reduce the risk of virologic failure. Subjects indicated that short-term AEs with relatively certain outcomes are preferred to long-term AEs with uncertain outcomes. Subjects were strongly averse to the risk of decreased BMD that could not be treated successfully or when the outcome was uncertain and to the risk of renal impairment that could not be treated successfully. Subjects were willing to accept increased risks of AEs in exchange for lower risk of virologic failure.

The value to patients of reducing lesion severity in plaque psoriasis

Abstract Plaque psoriasis is associated with significant psychosocial, quality-of-life, and economic burden. The objective of this study was to quantify the value to patients of reducing the severity and size of plaque psoriasis lesions. Subjects included individuals with a self-reported diagnosis of plaque psoriasis from a nationally representative US household panel. Subjects completed a web-based conjoint analysis survey and chose between hypothetical treatments in a series of questions. Each alternative was defined by lesion severity, percentage of body surface area (BSA) covered by lesions, type of treatment, injection discomfort or pain (if treatment included injections), risk of serious lung infection, and monthly out-of-pocket cost. 28,200 panelists were invited to participate. 18,330 responded, 503 qualified, and 419 completed the survey. Mean age was 54.5 years and 52% were female. 64% (35%) of patients reported psoriasis severity as mild or mild to moderate (moderate to very severe). Patients were willing to pay

A discrete-choice experiment to quantify patient preferences for frequency of glucagon-like peptide-1 receptor agonist injections in the treatment of type 2 diabetes:

486.73 out-of-pocket per month to eliminate severe lesions covering 25% BSA on the arms and legs and

How do physicians weigh benefits and risks associated with treatments in patients with osteoarthritis in the United Kingdom

444.80 out-of-pocket per month to eliminate moderate lesions covering 4% BSA on the face. Individuals with plaque psoriasis are willing to pay substantial amounts to reduce lesion severity and percentage of BSA covered by lesions.

Patient preferences for reducing toxicities of treatments for gastrointestinal stromal tumor (GIST)

Abstract Objective: Understanding patients’ preferences for attributes of injectable antihyperglycemic regimens may improve patient satisfaction and medication adherence. Our objective was to quantify the preferences of patients with type 2 diabetes mellitus (T2DM) for reducing the frequency of glucagon-like peptide-1 receptor agonist injections from once daily to once weekly. Methods: A total of 643 respondents with a self-reported physician diagnosis of type 2 diabetes completed a web-based discrete-choice experiment survey. The sample included four prespecified subgroups: currently using exenatide once weekly (n = 150), liraglutide once daily (n = 153), insulin (but not exenatide once weekly or liraglutide) (n = 156), and no injectable treatment (n = 184). Device attributes included type of injection device, needle size and pain, injection frequency, refrigeration, and injection-site reactions. Random-parameters logit was used to estimate the relative impact of device attributes on treatment choice for each subgroup. Results: In all subgroups, changing injection frequency from daily to weekly (independent of the effect of injection frequency on preferences for other attributes) was the most important predictor of treatment choice. Switching from a longer and thicker needle to a shorter and thinner needle and eliminating injection-site reactions were also statistically significant predictors of device choice (P < 0.05). Exenatide once weekly users and those not currently using injections were more likely to choose a treatment with characteristics similar to exenatide once weekly. Conclusions: The treatment attribute most important to patients choosing among hypothetical injectable treatments for T2DM was injection frequency: patients preferred weekly over daily injections. Limitations: The primary limitations of this study are that it included only a limited number of attributes that may not reflect the full complexity of patient choices, diagnosis was self-reported, and patients were recruited from an Internet panel and may not be representative of the T2DM patient population.

Physicians’ stated trade-off preferences for chronic hepatitis B treatment outcomes in Germany, France, Spain, Turkey, and Italy:

Objective. To quantify the relative importance that UK physicians attach to the benefits and risks of current drugs when making treatment decisions for patients with osteoarthritis (OA). Methods. Physicians treating at least 10 patients with OA per month completed an online discrete-choice experiment survey and answered 12 treatment-choice questions comparing medication profiles. Medication profiles were defined by 4 benefits (reduction in ambulatory pain, resting pain, stiffness, and difficulty doing daily activities) and 3 treatment-related risks [bleeding ulcer, stroke, and myocardial infarction (MI)]. Each physician made medication choices for 3 of 9 hypothetical patients (varied by age, history of MI, hypertension, and history of gastrointestinal bleeding). Importance weights were estimated using a random-parameters logit model. Treatment-related risks physicians were willing to accept in exchange for various reductions in ambulatory and resting pain also were calculated. Results. The final sample was 475. A reduction in ambulatory pain from 75 mm to 25 mm (1.6 units) was 1.1 times as important as an increase in MI risk from 0% to 1.5% (1.5 units). The greatest importance was for eliminating a 3% treatment-related risk of MI or stroke. On average, physicians were willing to accept an increase in bleeding ulcer risk of 0.7% (95% CI 0.4%–1.7%) for a reduction in ambulatory pain of 75 mm to 50 mm. Conclusion. When presented with well-known benefits and risks of OA treatments, physicians placed greater importance on the risks than on the analgesic properties of the drug. This has implications for the reporting of the results of clinical research to physicians.

Physician preferences for bone metastasis drug therapy in Canada

Purpose: To quantify gastrointestinal stromal tumor (GIST) patients’ preferences for reducing treatment toxicities and the likely effect of toxicities on patients’ stated adherence. Methods: English-speaking members of the Life Raft Group, a GIST patient advocacy and research organization, aged 18 years and older, completed a web-enabled survey including a series of treatment-choice questions, each presenting a pair of hypothetical GIST medication toxicity profiles. Each profile was defined by common or concerning toxicities verified via pretest interviews including: severity of edema, diarrhea, nausea, fatigue, rash, hand-foot syndrome, and heart failure; and risk of serious infection. Each subject answered 13 choice-format questions based on a predetermined experimental design with known statistical properties. Subjects were asked to rate the likelihood that they would miss or skip doses of medications with different toxicity profiles. Random-parameters logit was used to estimate a relative preference weight for each level of toxicity. Results: 173 subjects completed the survey. Over the ranges of toxicity levels included in the study, heart failure was the most important toxicity. Edema was the least important. For all toxicities, reducing severity from severe to moderate was more important to subjects than reducing severity from moderate to mild. Reducing heart failure from moderate to mild and diarrhea from severe to moderate had the largest effects on subjects’ evaluation of adherence. Conclusions: All toxicities included in the study are important to patients. Treating or reducing severe toxicities is much more important to patients than treating or reducing moderate toxicities. Focused reductions of certain toxicities may improve treatment adherence.