Patient preferences do matter: a discrete choice experiment conducted with breast cancer patients in six European countries, with latent class analysis.

OBJECTIVES: The evolution of breast cancer (BC) treatments has resulted in tailored therapies for the different types and stages of BC. Each treatment has a profile of benefits and adverse effects which are taken into consideration when planning a treatment pathway. This study examines whether patients' preferences are in line with what is considered important from decision makers viewpoint. METHODS: An online discrete choice experiment was conducted in six European countries (France, Germany, Ireland, Poland, Spain, UK) with BC patients. Six attributes were included: overall survival (OS), hyperglycemia, rash, pain, functional well-being (FWB), and out-of-pocket payment (OOP). Sixteen choice sets with two hypothetical treatments and a "No treatment" option were presented. Data were analyzed with the use of heteroscedastic conditional, mixed logistic, and latent class models. Marginal rate of substitution (MRS) were estimated for OOP versus the rest of attributes to establish the ranking of preferences for each attribute. RESULTS: Two hundred and forty-seven patients with advanced or metastatic BC and 314 with early-stage BC responded. Forty-nine percent of patients were < 44 years old and 65 percent had completed university education. The MRS of the analysis demonstrated that "severe pain" is the highest dis-preferred attribute level, followed by "severe impairment in FWB" and OS. Four classes of patients as "decision makers" were identified. CONCLUSIONS: This study suggests that there is heterogeneity in treatment preferences of BC patients depending on their sociodemographic and disease-related characteristics. In combination with clinical guidelines, patient preferences can support the selection and tailoring of treatment options.

Patient preferences for breast cancer treatments: a discrete choice experiment in France, Ireland, Poland and Spain.

Aim: To understand breast cancer patients' trade-offs when choosing treatments and to identify the most important treatment attributes which drive decisions. Materials & methods: A discrete choice experiment was conducted in France, Ireland, Poland and Spain. Progression-free survival, febrile neutropenia, pain, functional well-being and out-of-pocket payment were the treatment attributes. Results: 371 patients were willing to pay €6896 per year for 1 additional year of progression-free survival, €17,288 per year for perfect functional well-being and €15,138 for one pain-free year. Patients are willing to trade off progression-free survival months for better functional abilities and less pain. Conclusion: Patient preferences should be considered by regulatory agencies, reimbursement bodies, payors and clinicians for best treatment choices for the individuals.

The authors wanted to explore what breast cancer patients want the most from their cancer treatments. For this purpose, their preferences were collected in four European countries via a discrete choice experiment. The study showed that patients prefer treatments that improve their ability to function well in their daily lives and reduce their levels of pain. Patients' preferences for treatment outcomes might differ from those of the treating clinicians or regulators. Hence, these aspects can be discussed with their clinicians to make a joint decision on the choice of treatments.

Can we use existing guidance to support the development of robust real-world evidence for health technology assessment/payer decision-making?

Advances in the digitization of health systems and expedited regulatory approvals of innovative treatments have led to increased potential for the use of real-world data (RWD) to generate real-world evidence (RWE) to complement evidence from clinical trials. However, health technology assessment (HTA) bodies and payers have concerns about the ability to generate RWE of sufficient quality to be pivotal evidence of relative treatment effectiveness. Consequently, there is a growing need for HTA bodies and payers to develop guidance for the industry and other stakeholders about the use of RWD/RWE to support access, reimbursement, and pricing. We therefore sought to (i) understand barriers to the use of RWD/RWE by HTA bodies and payers; (ii) review potential solutions in the form of published guidance; and (iii) review findings with selected HTA/payer bodies. Four themes considered key to shaping the generation of robust RWE for HTA bodies and payers were identified as: (i) data (availability, governance, and quality); (ii) methodology (design and analytics); (iii) trust (transparency and reproducibility); and (iv) policy and partnerships. A range of guidance documents were found from trusted sources that could address these themes. These were discussed with HTA experts. This commentary summarizes the potential guidance solutions available to help resolve issues faced by HTA decision-makers in the adoption of RWD/RWE. It shows that there is alignment among stakeholders about the areas that need improvement in the development of RWE and that the key priority to move forward is better collaboration to make data usable for multiple purposes.