Jessica Scott, Takeda’s head of R&D patient engagement, highlights how a better understanding of the value of patient engagement across the healthcare stakeholder spectrum will drive better health outcomes and why keeping an open and curious mindset is crucial in this endeavour.


By incorporating Patient Engagement (PE) into the process of drug development by using direct patient input, we elevate our ability to better meet patients’ needs in trial design, endpoints, and ultimately with new medicines

Imagine you are planning the trip of your life. Literally, this may be the last trip you will take in your life because of a severe medical condition. Maintaining your quality of life has always been important to you, not just how long you live. You want to make the most of the trip by feeling as good as you can while you are away. You sit with your doctor who wants to prescribe a new medication for your condition. You and she are optimistic about the benefits you may have over time, but like with any new medicine, there is uncertainty and potential for side effects. Now you have to make a crucial decision: Go on the trip and delay taking the medication; go on the trip knowing you are likely to have side effects on this particular medication, or just don’t go at all. But then, your doctor shares the patient reported outcome findings from a Phase 3 trial. As it turns out, the vast majority of patients did not experience side effects during the second month after starting this medicine. And, there were patients like you in the trial, similar age, race and ethnicity, and general health. Your anxiety decreases and the decision becomes much easier to make. To think, you almost decided to put off starting this medicine where a decline in function would not be recoverable had you started it later.


How do we, as developers of new medicines, know what matters most to patients to generate the right evidence for patients’ health care decision-making? By engaging patients for their input in the process of drug development, we generate the evidence patients and doctors will use to answer questions that can help guide them in conversations and decisions about treatment. Input from patients early in the drug development process can provide important information about the need for developing one compound versus another. As we move into clinical development, engaging patients for their input is vital to understanding their perspective on topics such as how to decrease the burden of clinical trials, which symptoms are most important to address, and which tradeoffs they are willing to make.


In the pharmaceutical industry, we have made assumptions about patients’ needs and wants based on traditional sources of information, such as from physicians and the scientific literature. Often the information is second-hand, superficial, or not on-point with what we need to know. By incorporating Patient Engagement (PE) into the process of drug development by using direct patient input, we elevate our ability to better meet patients’ needs in trial design, endpoints, and ultimately with new medicines.


It’s important to keep in mind; not all Patient Engagement is equal. To get the greatest value from Patient Engagement, we need to be open to discover something completely new – something that we couldn’t possibly know without hearing it directly from the patient.


The question is: How to create an environment to learn something new? To learn something new outside of our frame of reference, we need to adopt an open mindset that is deeply curious about someone else’s experience. We can accomplish this by suspending what we already know, resisting the urge to confirm existing information and ask open-ended questions that will give us powerful insights into the patient’s world and the preferences they have. For instance, with a progressive neurological condition, in a Patient Engagement discussion, patients said they would prefer a new drug that could help maintain fine motor skills and coordination (e.g., hand function) over gross motor functions (e.g., walking). This patient input shaped the choice as to primary endpoint for a trial that would, in turn, provide key information for patients and physicians if the medicine were to move successfully through development and gain regulatory approval.


One important metric to test if you have been open-minded while engaging with patients is to ask yourself, “Did I learn something new? Or, did I have an “ah-ha” moment? These revelations let you know that you have uncovered something new that could only be learned from someone living with the condition. These valuable and unique insights add a new dimension to the information gathered from traditional sources.Qualitative Patient Engagement is also valuable in the process of drug development when combined with quantitative measures. PE helps to maximize the value of Patient Reported Outcome (PROs) measures by guiding the development of survey questions to assess what really matters to patients. For example, insight from patients during a Parkinson’s Disease advisory board uncovered that patients would prefer a new medicine that enhances or prolongs cognitive functioning versus a medication that reduces the frequency of falling. With this crucial patient insight, a PRO can include survey question(s) to assess if this finding can be generalized to a broader population.


Using PRO surveys that are brief and feel relevant for patients, patients are more likely to complete them. Since the questions are informed by qualitative information obtained directly through patient input, they target symptoms and experiences important to them. Patients feel acknowledged, valued, more interested, and engaged in responding to the survey questions. With this elevated sense of partnership, we gain better quantitative results in terms of accuracy, because the questions address issues that really matter to patients, and because patients are less likely to leave questions unanswered. An innovative pilot with FDA is now underway to share PRO results for approved medications pubically so patients and physicians can consider the reported measures of symptoms in health care decision-making.[i] This pilot would enhance communication and transparency by providing a new mechanism for sharing patient symptom data via the FDA website elevating the potential for its use beyond what may be included in the product label.



Patient Engagement provides an important navigational tool in drug development that helps steers us toward developing medicines that address patients’ unmet needs, focuses us on symptoms patients care about, and guides us toward developing evidence that can ultimately support their health care decisions. Patient Engagement helps us uncover what truly matters to patients because the information comes directly from them. How we engage with patients is equally important. For it to be effective, Patient Engagement needs to be approached with an open mind, that is, to learn something new rather than confirming what we believe we already know. Combing qualitative patient insights with quantitative assessments, PROs, we ensure we are asking the right questions. Patient Engagement is a unique dimension that, when included in the process of drug development, we can ensure that we bring medicines of value to patients.


In conclusion, it may be possible for a patient to have her trip and her medicine too.





Photo Credit: Anna Shvets