Pharmaceutical companies and their research partners frequently express concern about incorporating the voice of patients into their clinical trials. However, capturing the thoughts and perspectives of those patients can be a challenge.
To learn more about the importance of learning and assessing patient preferences in clinical research, Outsourcing-Pharma connected with Jayne Galinsky, senior outcomes researcher with contract research organization (CRO) ICON.
OSP: When trial teams are talking about the patient voice, what might they mean?
JG: The patient voice in a clinical trial team can mean a number of different things. The patient voice may be the perspective of a patient organization in a specific disease area, which has commented on a clinical trial protocol or supported a company with a PRO strategy. The patient voice may also be the perspective of an individual patient who might sit within a research team and provide insight from a patient perspective, alongside a wider group of researchers. The patient perspective may also come from study findings, such as those from a patient preference study. It is important that patients and their families have a platform to feed into discussions around research so that future treatments can truly meet the needs of patients and their families in the real-world setting.
OSP: How do study professionals usually assess patient preferences, and when? I.e. do they check in with patients’ feelings at every stage, or at certain points in a study?
JG: Patient preferences for treatment can be measured in different ways. Researchers can conduct qualitative interviews to understand more about unmet needs and what patients want from their future treatments. Patient preferences can also be captured quantitatively, through methods that illicit patients’ preferences for hypothetical or actual treatment attributes. Such data is increasingly recognized as having a key role to play in research and development decision-making and in regulatory and health resource allocation contexts.
Patient preference studies are not usually longitudinal. They can be conducted at any point in a clinical trial. Patient preference data is critical for informing the future development and prioritization of new treatments and so the earlier the better.
OSP: Please talk about how patient preferences might be different based on the condition in question.
JG: Patient preferences for treatment differ depending on the condition. For example, cancers more typically affecting older people might mean that this patient group might have different unmet needs and preferences for treatment, to a cancer more typically affecting a working age population. Similarly, preference studies can generate data on patient subgroups, such as those living in different countries, of different ages, or stage of disease.
Patient preference data highlights the complex trade-offs between benefits and risks that different people make. This is important especially because some literature suggests that healthcare professionals can underestimate the importance of patient pain or quality of life for patients. Understanding more about the way that disease impacts particular groups of individuals means that treatments can be better tailored to what patients need and want in the future.
OSP: What can a trial team do to incorporate what they learn when soliciting patient preferences? Can they typically be used to improve an ongoing trial (without throwing the protocol for a loop)?
JG: Patient preference data is always useful for understanding what is important and relevant to patients and their families. If a preference study is conducted early enough, it can incorporate the patients' perspectives into the clinical development process to deliver treatments and outcomes that are relevant to them.
Preference data provides in-depth insight into the lived experience of patients. If clinical trial protocols cannot be amended to reflect generated preference data, then the insights gained from these studies should still help researchers understand the experiences and preferences of patients more widely and be helpful in communication, understanding of quality of life issues, and the way that illness impacts upon patients’ lives more broadly.
OSP: Could you share any real-life examples of an effort to collect patient preferences?
JG: Most of my work on patient preference studies has been in the area of myeloma. Patient preference studies, such as Discrete Choice Experiments, ask patients to select their preferred option from a set of competing alternatives (which collectively form a choice task). These choices are hypothetical and patients may not have come across questions like this before. The hypothetical nature of choice tasks, therefore, has the potential to be confusing or cognitively challenging.
It, therefore, makes sense that the studies patients have responded to the most have been surveys that are visual and engaging. In my experience, preference surveys work best when they feel realistic and reflect choices that patients may come across in the real world.
Making the survey feel simple and eye-catching is really important in generating large samples with relevant findings. Making sure that draft surveys are tested with patients is also important. To do this, interviews are conducted with individual patients to make sure the draft survey makes sense, is relevant, and uses language sensitive to patients. Making sure that native speakers check any translations is important too, especially when asking the same questions to patients working across countries and cultures.
OSP: Do you have anything to add?
JG: I think that evidence like patient preference data is becoming more and more important for decision-makers and informing the prioritization of new treatments in care pathways. I hope to see preference data assisting regulators and health technology assessment agencies in understanding the acceptability and value proposition of new treatments more in the future. I believe it is important that patient perspectives continue to be taken seriously - data we can obtain from patients on quality of life is a critical contribution to discussions and decision-making in health.
