Patient, Family Member and Physician Perspectives and Experiences with AML Treatment Decision-Making.

INTRODUCTION: Treatment decisions in older adults with acute myeloid leukemia (AML) are challenging, particularly for those who are not candidates for intensive chemotherapy (IC), and the trade-offs patients, their families and physicians consider when choosing a treatment option are not well understood. This qualitative research explored the value of extending survival and the treatment decision-making process from a multi-stakeholder perspective. METHODS: Overall, 28 patients with AML (≥ 65 years old, unsuitable for IC), 25 of their relatives and 10 independent physicians from the US, UK and Canada took part in one-on-one, 60-minute qualitative interviews. RESULTS: Across all stakeholders, improved health-related quality of life (HRQoL), extended survival and relief of AML symptoms were recognized as most important in AML treatment decision-making. However, extending survival in 'good health' was more important than extending survival alone, particularly because of the extra time it gives patients and their relatives together, and allows patients to achieve important goals. Patients' limited understanding of available treatment options, paired with incorrect perceptions of treatment side effects, impacted their involvement in the treatment decision-making process. Patients and physicians perceived physicians to have the most influence in the decision-making process despite their priorities not always aligning. CONCLUSION: These findings illustrate the importance of having structured discussions which explicitly assess patients' goals and their understanding and expectations of treatments and also the need for patient friendly resources about the lived experience of AML and available treatment options. These measures will help to ensure that patients are fully involved in the shared decision-making process.

Incorporating Patient Advocates in Oncology Clinical Development: Lessons Learned From a Novel Pilot Program.

The advent of patient-focused drug development (PFDD) has underscored the priority of engaging the "voice of the patient" in therapy development. Industry sponsors are working to enhance engagement of patients early, particularly within decision making for design and execution of clinical trials. This trend is especially significant within oncology, as industry leaders partner with patient advocacy organizations, individual patients, and clinicians to enhance patient-centricity. These partnerships often require a willingness to change attitudes, approaches, and processes to reshape traditional models of drug development. In 2016, Bayer Oncology launched a pilot program called the Patient Advocate Advisory Council (PAAC), to design and execute a program whereby patients join clinical development teams. The PAAC, composed of experienced patient advocates from the US and Europe, worked closely with company leaders to design and execute a pilot in an ongoing clinical development program. The PAAC and Bayer teams have identified important learnings from the first phase of the program, emphasizing earlier engagement of patient advisors, launching the enhanced training platform, and recruiting additional PAAC members to expand the initiative's reach across the cancer community. A critical success factor is having champions for patient engagement within the company to ensure that activities are streamlined and standardized as patient engagement becomes more common. This is particularly important given that patient engagement should be a long-term investment with sufficient and sustained resources. PAAC members and Bayer have committed to sharing learnings, to advance opportunities for successful patient engagement in drug development throughout the oncology therapeutic landscape.

Regulatory decision-making meets the real world.

As patient input in drug development increases and new data sources are tapped, regulators need to organize and ensure the quality of data to inform decision-making.