Study results November,

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1 Study results 1 PATIENTS AND PROFESSIONALS PREFERENCES FOR TYPE 2 DIABETES MELLITUS TREATMENTS IN SPAIN AND PORTUGAL: WILLINGNESS TO PAY FOR GAINING HEALTH BENEFITS AND AVOIDING SIDE EFFECTS PhD. Antonio Ramírez de Arellano June 17 th, 2015 XXXV Jornadas AES

2 Study results November,

3 Priorities in Patient Preferences: Discrete Choice Experiment in Swedish Patients Main Features of the Analysis: Method: Discrete Choice Experiment to determine the value that patients place to change attributes & preferred options Attributes: 16 convenience (e.g. oral vs injection); 24 clinical attributes (e.g. weight reduction). No. of Questionnaires: 461 under final scrutiny Slide no 3 Main Conclusions of the Analysis: Patients placed the highest value on weight loss; in particular, the avoidance of weight gain (over HbA1c improvements) A daily injection (in relation to meal) providing at least 2.14Kg weight loss would be preferred over a tablet providing weight neutrality A daily injection (irrespective of meals) providing at least 1.3Kg weight loss would be preferred over a tablet providing weight neutrality How much do I value to get a positive outcome? Hypoglycaemia in remarkable relative position comparing to HbA1c improvements * * I accept injection provided that I can lose 2.14Kg * I accept injection provided that I can lose 1.3Kg * How much do I value to avoid a negative outcome? Ref. Jendle J. et al. 2010: Willingless to pay for health improvements associated with anti-diabetes treatments for people with type 2 diabetes. Current Medical Research & Opinion. Vol. 26:

4 Bogelund M. et al. 2011: Patients preferences for diabetes management among people with type 2 diabetes in Denmark: a discrete choice experiment. Current Medical Research & Opinion. Vol. 27: Priorities in Patient Preferences: Discrete Choice Experiment in Danish Patients Main Features of the Analysis: Method: Discrete Choice Experiment to determine the value that patients place to change attributes & preferred options Attributes: 16 convenience (e.g. oral vs injection); 24 clinical attributes (e.g. weight reduction). No. of Questionnaires: 270 under final scrutiny Comparing Danish versus Swedish Results: Patients placed the highest value on weight loss; in particular, the avoidance of weight gain (over HbA1c improvements) A daily injection (in relation to meal) providing at least 1.4Kg (2.14Kg Swedish) weight loss would be preferred over a tablet providing weight neutrality The relative value of improvements in HbA1c is higher than that of reductions in Hypoglycaemia events (the opposite in the Swedish results) Slide no 4 Weight - loose 6 kg Possession of drivers licence* Weight - loose 3 kg HbA1c - 1% decrease Improved heart function* Hypoglycaemia - 2 event less per month HbA1c - 0.5% decrease Injection - 1 injection less per day SMBG - 1 test strip less per day Hypoglycaemia - 1 event less per month Mode of administration - injection irr. of meals vs. tablet Transient nausea - 25% risk of nausea for 1 month Injections with meals instead of independent of meals HbA1c - 0.5% increase Mode of administration - injection with meals vs. tablet Hypoglycaemia - 3 more events per month Weight - gain 3 kg weight HbA1c - 1% increase Transient nausea - experience nausea for 1 month * * * How much do I value to get a positive outcome? I accept injection provided that I can lose 1.4Kg 0.5% reduction in A1c as valuable as Hypo reduction from 2 to 0 How much do I value to avoid a negative outcome? * Monthly WTP (DKK)

5 Aim: Study results November, To assess the preferences of Spanish and Portuguese patients with type 2 diabetes mellitus (T2DM) and physicians with regard to diabetes treatments, including side effects and method of administration. Objectives: To determine the willingness to pay of T2DM patients and physicians for: a) Achieving certain therapeutic benefits b) Avoiding side effects c) Alternative ways of treatment administration d) The frequency of self-monitoring blood glucose levels

6 Study results November, Methods Design Observational, descriptive, multicenter, exploratory study in the context of usual clinical practice in Spain and Portugal (not linked to any specific antidiabetic drug or treatment). Setting Patients were recruited from 11 centers (hospitals and outpatient s clinics) operating in the public healthcare system in different autonomous communities in Spain and in Portugal. Professionals were recruited from diverse hospitals and outpatient s clinics operating in the public healthcare system in different autonomous communities in Spain and in Portugal.

7 Study results November, Conjoint analysis Technique used in market research to assess how consumers value the characteristics that comprise an specific product. In recent years its use has extended to analyzing patients preferences in relation to different treatment options. Oral None 100 Mode of administration Blood glucose monitoring Payment per month Injection 3/week 50

8 Study results November, Conjoint analysis concepts Attributes are the characteristics that can vary from product to product. Levels are the options for each attribute. Levels Levels Attribute Scenario Oral None 100 Mode of administration Blood glucose monitoring Payment per month Injection 3/week 50 Scenario

9 Study results November, Utility values Utility correspond to the value that individuals assign to a product through the combination of attributes, so that its value is the maximum for the choice made in the set of options. The utility value obtained comes from an appropriate combination of attributes, weighted by the relative importance of each in contributing to the overall usefulness of the product in question. Utility = β 1 mode of administration + β 2 blood glucose monitoring + β 3 Payment per month Partial utility values Attribute importance

10 Professional s preferences for T2DM treatments and willingness to pay for gaining health benefits and avoiding side effects in Spain and Portugal Study results November,

11 Study results November, Distribution of professional s sample by country Spain 81% Portugal 19%

12 Study results November, Distribution of professional s sample by age and gender 59.0% 41.0% Age Mean SD n Spain Portugal % Total %

13 Study results November, Distribution of professional s sample by age and gender Family Medicine doctors 58% Endocrinologists 42% Family Medicine doctors 100% Endocrinologists 0%

14 Study results November, Professional s preferences for the convenience attributes Based on data from the discrete choice experiment, utility values (U) for each treatment alternative have been estimated by adding the partial utility values (β) of each attribute that comprise it. U = AAAAA 1 β 1 + AAAAA 2 β 2 + AAAAA 3 β 3 The logit conditional model calculates the partial utility values for the studied attributes.

15 Professional s preferences for the convenience attributes Hypothetic Treatment Mode of administration Blood glucose monitoring Additional payment per month 1 Injection once a day irrespective of meals 3 times per week Oral antidiabetics up to three times a day with meals No need for testing Injection once a day in relation to meal No need for testing Injection once a day irrespective of meals No need for testing Injection once a day irrespective of meals Once a day Oral antidiabetics up to three times a day with meals Once a day Inyección 1/día con la comida 3 times per week Utility value 8 Oral antidiabetics up to three times a day with meals 3 times per week Oral antidiabetics up to three times a day with meals 3 times a day Injection twice a day in relation to meal 3 times per week Injection twice a day in relation to meal Once a day Injection once a day in relation to meal 3 times a day Injection once a day in relation to meal Once a day Injection twice a day in relation to meal No need for testing Injection once a day irrespective of meals 3 times a day Injection twice a day in relation to meal 3 times a day Study results November, Hypothetical treatments with higher utility values are the most preferred ones Utility values must be interpreted in a linear distribution

16 Professional s preferences for the clinical attributes Hypothetic Treatment Number of hypoglycemic events HbA1c Weight change in 6 months Nauseas Study results November, Cardiovascular risk 1 Once a year 6-7% Lose 6 kg None Decreases Once a year <6% Remains the same weight None Remains the same Once a month <6% Lose 6 kg Mild nausea for up to 3 months Decreases Once a week <6% Lose 3 kg None Decreases None 7-8% Lose 6 kg None Remains the same None 6-7% Lose 3 kg Mild nausea for up to 3 months Decreases None >8% Remains the same weight None Decreases None <6% Gain 3 kg Mild nausea for up to 3 months Remains the same Once a year 7-8% Lose 3 kg Mild nausea for up to 3 months Remains the same Once a month 6-7% Invariable Mild nausea for up to 3 months Remains the same Once a year >8% Gain 3 kg Mild nausea for up to 3 months Decreases Once a month 7-8% Gain 3 kg None Decreases Once a week 7-8% Remains the same weight Mild nausea for up to 3 months Decreases Once a month >8% Lose 3 kg None Remains the same Once a week 6-7% Gain 3 kg None Remains the same Once a week >8% Lose 6 kg Mild nausea for up to 3 months Remains the same Cost Utility value Hypothetical treatments with higher utility values are the most preferred ones Utility values must be interpreted in a linear distribution

17 Study results November, Willingness to pay Convenience attributes Unit change Estimated coefficient (β) Standard Error P value Adm: Injection twice a day in relation to meals [ref] Adm: Injection once a day in relation to meal < Adm: Injection once a day irrespective of meals < Adm: Oral antidiabetics (OAD) up to three times a day with meals < Blood glucose monitoring (per month) < Additional payment per month < For estimating the willingness to pay for each attribute, the marginal rate of substitution was calculated by dividing the estimated coefficient for each attribute by the estimated coefficient of additional payment per month. One injection less per day = Adm: Injection once a day in relation to meal Additional payment per month = ~ Manual calculation might vary from the one performed by the model due to the decimals included.

18 Study results November, Professional s monthly willingness to pay for diabetes treatment attributes Avoid one hypoglycemic event per week Avoid one hypoglycemic event per month Avoid one hypoglycemic event per year Avoid gaining 3 Kg in 6 months 114,18 141,42 166,87 287,18 Hypoglycaemic events Reduce 6Kg in 6 months Reduce 3Kg in 6 months 18,59 65,80 Weight change in 6 months Decrease cardiovascular risk Avoid nauseas Avoid increasing 1% of HbA1c 143,30 125,92 154,30 Cardiovascular risk Nauseas HbA1c One injection less per day 69,07 Administration irrespective of meals instead of in relation to meals Oral antidiabetics instead of injections in relation to meals Oral antidiabetics instead of injections irrespective of meals 1,84 26,66 24,82 Mode of administration One blood glucose monitoring test less per day One blood glucose monitoring test less per month 1,32 39,55 Blood glucose monitoring

19 Patient s preferences for T2DM treatments and willingness to pay for gaining health benefits and avoiding side effects in Spain and Portugal Study results November,

20 Study results November, Distribution of patients sample by country Spain 84% Portugal 16%

21 Study results November, Distribution of patients sample by age and gender 51.4% 47.8% 40.4% 59.6%

22 Study results November, Distribution of patients sample by age and gender 51.4% 47.8% Age Mean SD n Spain Portugal % Total %

23 Patients preferences for the convenience attributes Hypothetic Treatment Mode of administration Blood glucose monitoring Additional payment per month 1 Injection once a day irrespective of meals 3 times per week Oral antidiabetics up to three times a day with meals No need for testing Injection once a day in relation to meal No need for testing Oral antidiabetics up to three times a day with meals 3 times a day Injection once a day irrespective of meals Once a day Utility value 6 Oral antidiabetics up to three times a day with meals Once a day Injection once a day irrespective of meals No need for testing Injection twice a day in relation to meal Once a day Injection twice a day in relation to meal 3 times per week Injection once a day in relation to meal 3 times a day Injection once a day in relation to meal 3 times per week Oral antidiabetics up to three times a day with meals 3 times per week Injection twice a day in relation to meal 3 times a day Injection once a day in relation to meal Once a day Injection once a day irrespective of meals 3 times a day Injection twice a day in relation to meal No need for testing Study results November, Hypothetical treatments with higher utility values are the most preferred ones Utility values must be interpreted in a linear distribution

24 Study results November, Patients preferences for the clinical attributes Hypoglycemic event: 1 per week [ref] Estimated coefficient (β) Standard Error P value Hypoglycemic events: 1 per year <0.001 Hypoglycemic events: 1 per month Hypoglycemic events: None <0.001 HbA1c (%) <0.001 Weight: Lose 6 Kg [ref] Weight: Remains the same Weight: Lose 3 kg Weight: Gain 3 kg <0.001 Nausea: Mild nausea for up to 3 months [ref] Nausea: None <0.001 Cardiovascular risk: Remains the same [ref] Cardiovascular risk: Decreases <0.001 Additional payment per month <0.001 Coefficient magnitude Relative weight of the attribute level that contributes to the utility of each hypothetical treatment. When comparing the coefficients, attention to the different units of measurements must be paid. Coefficient sign +: preference increases for the attribute level with respect of the reference level - : preference decreases as the attribute level increases Significant p value (<0.05) The attribute level has a influence in the decision making process

25 Study results November, Patients preferences for the clinical attributes Hypothetic Treatment Number of hypoglycemic events HbA1c Weight change in 6 months Nauseas Cardiovascular risk 1 Once a year 6-7% Lose 6 kg None Decreases Once a week <6% Lose 3 kg None Decreases None 7-8% Lose 6 kg None Remains the same Once a month <6% Lose 6 kg Mild nausea for up to 3 months Decreases None >8% Remains the same weight None Decreases None 6-7% Lose 3 kg Mild nausea for up to 3 months Decreases Once a month >8% Lose 3 kg None Remains the same Once a week 7-8% Remains the same weight Mild nausea for up to 3 months Decreases Once a year <6% Remains the same weight None Remains the same None <6% Gain 3 kg Mild nausea for up to 3 months Remains the same Once a month 6-7% Remains the same weight Mild nausea for up to 3 months Remains the same Once a year 7-8% Lose 3 kg Mild nausea for up to 3 months Remains the same Once a year >8% Gain 3 kg Mild nausea for up to 3 months Decreases Once a week 6-7% Gain 3 kg None Remains the same Once a month 7-8% Gain 3 kg None Decreases Once a week >8% Lose 6 kg Mild nausea for up to 3 months Remains the same Cost Utility value Hypothetical treatments with higher utility values are the most preferred ones Utility values must be interpreted in a linear distribution

26 Study results November, Patient s monthly willingness to pay for diabetes treatment attributes Avoid gaining 3 Kg in 6 months 68,14 Reduce 3 Kg in 6 months 44,07 Reduce 6 Kg in 6 months Avoid one hypoglycemic event per month Avoid one hypoglycemic event per week 25,75 Weight change in 6 months 54,80 51,59 Avoid one hypoglycemic event per year 3,87 Hypoglycaemic events Decrease cardiovascular risk Avoid nauseas 43,01 42,74 Cardiovascular risk Nauseas Oral antidiabetics instead of injections in relation to meals One injection less per day 25,65 30,16 HbA1c Administration irrespective of meals instead of in relation to meals Oral antidiabetics instead of injections irrespective of meals Avoid increasing 1% of HbA1c 16,23 13,93 24,28 Mode of administration One blood glucose monitoring test less per day One blood glucose monitoring test less per month 0,34 10,19 Blood glucose monitoring

27 Comparison between patient s and professional s preferences for T2DM treatments and willingness to pay for gaining health benefits and avoiding side effects in Spain and Portugal Study results November,

28 Comparison between professional s and patient s monthly willingness to pay for diabetes treatment Patients attributes Professionals Avoid one hypoglycemic event per week Avoid one hypoglycemic event per month Avoid one hypoglycemic event per year Avoid gaining 3 Kg in 6 months Reduce 6Kg in 6 months Reduce 3Kg in 6 months Decrease cardiovascular risk Avoid nauseas Avoid increasing 1% of HbA1c One injection less per day Administration irrespective of meals instead of in relation to meals Oral antidiabetics instead of injections in relation to meals Oral antidiabetics instead of injections irrespective of meals One blood glucose monitoring test less per day One blood glucose monitoring test less per month 3,87 25,75 65,80 44,07 18,59 43,01 24,28 51,59 54,80 42,74 68,14 25,65 69,07 16,23 26,66 30,16 24,82 13,93 1,84 10,19 39,55 0,34 1,32 114,18 141,42 125,92 154,30 143,30 166,87 Study results November, ,18 Hypoglycaemic events Weight change in 6 months Cardiovascular risk Nauseas HbA1c Mode of administration Blood glucose monitoring

29 Study results November, Conclusions Decreasing hypoglycemias and reducing or maintaining weight were the T2DM treatment preferred attributes by patients and professionals in Spain and Portugal. Professionals in Spain and Portugal were willing to pay for health benefits associated with improvements in diabetes treatment, the most important of these being avoiding hypoglycemias, followed by avoid gaining weight and decreasing cardiovascular risk. Patients with T2DM were willing to pay for health benefits associated with improvements in diabetes treatment, the most important of these being reducing weight, followed by avoiding hypoglycemias. Patients were willing to pay more than professionals for reducing weight and for receiving oral antidiabetics instead of injections either in relation or irrespective of meals.

30 Study results November,

31 Study results November, Mohamed AF, et al. Avoidance of weight gain is important for oral type 2 diabetes treatments in Sweden and Germany: Patient preferences Germany Sweden There was a slight disordering of preference weights for frequency of monthly mild to moderate hypoglycemia, with the preference weight for four events being higher than the preference weight for two events, however, this anomaly was not statistically significant (p>0.05).

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