Patient Preference in Medical Product Development - July Newsletter Feature

What is patient preference? We have FDA's definition, which describes preference as patients' desire for outcomes or attributes given a choice.  But, what does patient preference really mean?  And, how can patient preference help medical product sponsors bring their product to market?  Preference is much more than making a simple choice between treatments. Patient preference is complex assessment of patients' priorities and desired treatment outcomes.  Sponsors can harness these data to support product development, regulatory decisions, commercialization, and more. But, it is necessary for sponsors to understand 1) when a preference study will help, 2) how to design a preference study that will provide meaningful data, and 3) how to leverage preference data across the medical product life-cycle.  Let’s talk about each of these in some detail.

When should a medical product company embark on a patient preference study?  Given the cost and time involved in conducting these studies, a sponsor should ensure that the results of the effort will be worth it.  The first place to start is to determine whether you have a preference sensitive question.  A preference sensitive question is one that legitimately presents the patient with a trade-off choice.  For example, let’s say you have a medical product that will treat a chronic, debilitating disease, but not cure that disease.  And, let’s say that your treatment provides immediate improvement in function and quality of life, but the long-term health effects are uncertain.  This scenario presents us with a preference sensitive issue – are patients willing to tolerate not having a cure for their disease and uncertain long-term health impact to obtain immediate and dramatic improvements in function and quality of life?  The choice may seem obvious to patients, but the FDA may look at your uncertain long-term health impact and lack of a cure and decide your treatment shouldn’t be approved.  In this situation, patient preference data that quantitatively captures patients’ willingness to forgo a cure and tolerate some uncertainty to obtain the benefits of your treatment can influence FDA’s decision to approve your treatment.  It is scenarios like these that can make obtaining patient preference data worthwhile.

Let’s look at another example.  Let’s pretend you are developing a treatment for a condition that is typically associated with a myriad of symptoms and is experienced by patients in variable ways. In this scenario, your preference sensitive question is:  in which symptoms and quality of life outcomes do patients want to see the most improvement from my treatment?  To find out, you could conduct a preference study using methods that quantitatively rank outcomes, or symptoms, or other criteria in such a way that you can determine which outcomes are most important to your patients.  This can drive product development, clinical trial design, and regulatory decision-making.  

Once you’ve decided you have a preference sensitive question, the time has come to determine how you are going to conduct your study. The design of a patient preference study will depend on several factors:  study objectives, treatment attributes, timeline, budget, patient availability, disease area, treatment types, and regulatory agency.  The study objective and patient availability are probably the two most critical aspects on which you will base your study design.  Numerous different designs – both quantitative and qualitative – are available.  A handful of qualitative interviews with patients at the beginning of your preference study will be critical for ensuring your study addresses what patients want, not what you think they want.  Interviews with treating clinicians can also be helpful during development of your preference study.  However, it is important to keep in mind the ultimate reason you are conducting this study – the patients and their priorities.

Once you’ve designed and implemented your patient preference study, how can a sponsor make the most of the data obtained?  Preference data, if collected properly, can support many aspects of the medical product life-cycle.  These include design and development, clinical study implementation, regulatory decision-making, value proposition and reimbursement, and post-market assessment.  While the popular focus right now is on how to use patient preference in regulatory decision-making, sponsors should be aware of the different ways their patient data can be of value throughout the total product life-cycle.

Want to learn more about patient preference?  The Patient x Design July newsletter featured this post along with additional commentary and resources focused on the theme, Patient Preference.  To get your own copy of this and future monthly newsletters covering topics in patient-focused policy, subscribe here!