Understanding drug preferences, different perspectives

Abstract

AIMSTo compare the values regulators attach to different drug effects of oral antidiabetic drugs with those of doctors and patients. METHODSWe administered a 'discrete choice' survey to regulators, doctors and patients with type 2 diabetes in The Netherlands. Eighteen choice sets comparing two hypothetical oral antidiabetic drugs were constructed with varying drug effects on glycated haemoglobin, cardiovascular risk, bodyweight, duration of gastrointestinal complaints, frequency of hypoglycaemia and risk of bladder cancer. Responders were asked each time which drug they preferred. RESULTSFifty-two regulators, 175 doctors and 226 patients returned the survey. Multinomial conditional logit analyses showed that cardiovascular risk reduction was valued by regulators positively (odds ratio 1.98, 95% confidence interval 1.11-3.53), whereas drug choices were negatively affected by persistent gastrointestinal problems (odds ratio 0.24, 95% confidence interval 0.14-0.41) and cardiovascular risk increase (odds ratio 0.49, 95% confidence interval 0.27-0.87). Doctors and patients valued these effects in a similar manner to regulators. The values that doctors attached to large changes in glycated haemoglobin and that both doctors and patients attached to hypoglycaemia and weight gain also reached statistical significance. No group's drug choice was affected by a small absolute change in risk of bladder cancer when presented in the context of other drug effects. When comparing the groups, the value attached by regulators to less frequent hypoglycaemic episodes was significantly smaller than by patients (P = 0.044).

Quantifying Preferences in Drug Benefit‐Risk Decisions

“…Tommi Tervonen 1, *, Aris Angelis 2 , Kimberley Hockley 3 , Francesco Pignatti 4 and Lawrence D. Phillips 5 Benefit-risk assessment is used in various phases along the drug lifecycle, such as marketing authorization and surveillance, health technology assessment (HTA), and clinical decisions, to understand whether, and for which patients, a drug has a favorable or more valuable profile with reference to one or more comparators. Such assessments are inherently preference-based as several clinical and nonclinical outcomes of varying importance might act as evaluation criteria, and decision makers must establish acceptable trade-offs between these outcomes.…”

“…Different stakeholder groups sometimes exhibit different preferences, and within one group preferences may vary. [3][4][5][6] Although not every decision made on drug benefit-risk balance needs detailed analysis to support it, this paper argues that decisions about drugs should more often include formal quantification of preferences. This is especially true for "preference-sensitive" decisions where multiple treatment options exist and there is no option that is clearly superior for all patients, or the evidence supporting one option over others is considerably uncertain or variable, or patients' views about the most important benefits and acceptable risks of a technology vary considerably within a population or differ from those of healthcare professionals.…”

“…Different stakeholder groups sometimes exhibit different preferences, and within one group preferences may vary . Although not every decision made on drug benefit‐risk balance needs detailed analysis to support it, this paper argues that decisions about drugs should more often include formal quantification of preferences.…”

Precision medicine in diabetes and diabetic kidney disease: Regulatory considerations

“…Moreover, increasingly caregivers and clinicians involve willing patients in shared decision‐making, choosing a therapy based on (expected) outcomes that are most relevant to these patients. Patients and regulators may have mostly similar values with regards to major drug effects of antidiabetic drugs, but do differ in the value they attach to minor short‐term (adverse) drug effects . Whereas patients are already involved in a range of activities at the EMA and FDA, a European public private partnership project, IMI‐PREFER (https://www.imi-prefer.eu/) investigates how and when to include patient‐preference in decision‐making during the drug life cycle.…”

“…Patients and regulators may have mostly similar values with regards to major drug effects of antidiabetic drugs, but do differ in the value they attach to minor short-term (adverse) drug effects. 9 Whereas patients are already involved in a range of activities at the EMA and FDA, a European public private partnership project, IMI-PREFER (https:// www.imi-prefer.eu/) investigates how and when to include patientpreference in decision-making during the drug life cycle. Moving forward, regulatory systems should help ensure that these best patienttherapy pairs can be identified and brought together.…”