The Domains of Patient Participation in Depth
BE INSPIRED BY THE MOMENTS THAT MATTER MOST
Stakeholders in the research process need to understand and fully consider the practical, real-life challenges of living with disease. This will ensure that successful evidence generation truly reflects unmet patient needs. It will also make it possible to develop new interventions that can offer greater value to patients, healthcare systems, and society.
Without this necessary perspective, research teams tend to leverage off-the-shelf outcome measures, which can miss the things that are most meaningful to patients. Research teams might focus their attention on an aspect of disease that is not the highest priority for the patient. Healthcare companies may focus on aspects of treatment and management that are not the most pressing issues for those who are living with the condition.
“We need to re-define and re-focus our research and data collection on endpoints that are truly relevant for the patient.,” says Rosemary Jose Haaksman, PhD, a Netherlands-based pharmacologist who serves as a senior director for strategic market access at OPEN Health. “Health technology committees might define the value of innovative treatment based on clinical markers, for instance, of inflammation or mobility. For the patient, however, it is probably improvement in the ability to perform simpler everyday tasks that matters the most. These kinds of mismatches are, sadly, quite common, and it is therefore crucial to our value demonstration that we seamlessly incorporate the patient voice.”
One way we can achieve this is by involving patients in PPI activities, as described above. We can also supplement structured quantitative questionnaires with more qualitative, open-ended interviews.
Sonya Snedecor, PhD, an Executive Director of HEOR who leads the Interactive Analytics and Communications group at OPEN Health, agrees. She explains how incorporating patients’ perspectives can improve the quality and outcomes of HTAs.
“The mandate of HTA is to maximize the health of the entire population by determining how to efficiently distribute limited resources across all healthcare interventions,” she says. “The challenge that healthcare companies and governments face with HTAs is that the decisions that maximize the health of the population and optimize the use of resources are not necessarily the decisions that will make any one person happy. Everyone has different priorities, and for decision makers to take the right action, they need to have the right knowledge, the insight to interpret that knowledge, and the necessary context to understand its relevance.”
An additional way to capture the moments that matter most to patients is to involve patients not just in study designs, but in capturing outcomes, including being able to submit and track their own data and by incorporating patient-centric measures among real-world evidence (RWE). Priya Patel sees momentum in this area.
“There’s a lot more research now that involves patient input,” she says. “Patients are being encouraged to contribute their own data and are empowered to take ownership of their own health — whether this is through study questionnaires or via advanced mobile applications that enable them to monitor and track their own data. Many patients are experts in their own conditions, being the ones that are experiencing their impact on quality of life firsthand. It is still relatively unique within the industry for an RWE agency to have a team that focuses explicitly on patient-centered outcomes, as OPEN Health does, but having this brings significant benefits, including a much more in-depth understanding of patient experience. It’s important to note that it is this qualitative research that is providing these additional insights.”
“Even as treatment strategies become more tailored and personalized, the medical community may not always immediately recognize the importance of considering the patients’ perspective. I recently worked on a patient preference study where our client developed a treatment that is comparable to the standard of care in terms of efficacy and side effects. The only difference was in the mode of administration -- our client’s therapy was oral versus the standard of care which includes IV infusions and SC injections.”
“The patients we interviewed described the burden of IV/SC therapies and spoke about their strong desire to receive their treatments orally. Oral therapies would decrease the number of required visits to the doctor’s office, which can be especially meaningful to older and frailer patients. Most patients appreciated the convenience an oral drug could bring and spoke about how it would give them part of their lives back. One patient heard about the oral treatment and immediately went to the doctor’s office to advocate and request the oral drug. However, the doctor didn’t see a need for it at the time because the efficacy and adverse event profiles were largely the same.”
— Sophie Tsai, MD, Sr. Scientist, Patient-Centered Outcomes & Patient Engagement, OPEN Health
BE INSPIRED BY THE MOMENTS THAT MATTER MOST
Stakeholders in the research process need to understand and fully consider the practical, real-life challenges of living with disease. This will ensure that successful evidence generation truly reflects unmet patient needs. It will also make it possible to develop new interventions that can offer greater value to patients, healthcare systems, and society.
Without this necessary perspective, research teams tend to leverage off-the-shelf outcome measures, which can miss the things that are most meaningful to patients. Research teams might focus their attention on an aspect of disease that is not the highest priority for the patient. Healthcare companies may focus on aspects of treatment and management that are not the most pressing issues for those who are living with the condition.
“We need to re-define and re-focus our research and data collection on endpoints that are truly relevant for the patient.,” says Rosemary Jose Haaksman, PhD, a Netherlands-based pharmacologist who serves as a senior director for strategic market access at OPEN Health. “Health technology committees might define the value of innovative treatment based on clinical markers, for instance, of inflammation or mobility. For the patient, however, it is probably improvement in the ability to perform simpler everyday tasks that matters the most. These kinds of mismatches are, sadly, quite common, and it is therefore crucial to our value demonstration that we seamlessly incorporate the patient voice.”
One way we can achieve this is by involving patients in PPI activities, as described above. We can also supplement structured quantitative questionnaires with more qualitative, open-ended interviews.
Sonya Snedecor, PhD, an Executive Director of HEOR who leads the Interactive Analytics and Communications group at OPEN Health, agrees. She explains how incorporating patients’ perspectives can improve the quality and outcomes of HTAs.
“The mandate of HTA is to maximize the health of the entire population by determining how to efficiently distribute limited resources across all healthcare interventions,” she says. “The challenge that healthcare companies and governments face with HTAs is that the decisions that maximize the health of the population and optimize the use of resources are not necessarily the decisions that will make any one person happy. Everyone has different priorities, and for decision makers to take the right action,
they need to have the right knowledge, the insight to interpret that knowledge, and the necessary context to understand its relevance.”
An additional way to capture the moments that matter most to patients is to involve patients not just in study designs, but in capturing outcomes, including being able to submit and track their own data and by incorporating patient-centric measures among real-world evidence (RWE). Priya Patel sees momentum in this area.
“There’s a lot more research now that involves patient input,” she says. “Patients are being encouraged to contribute their own data and are empowered to take ownership of their own health — whether this is through study questionnaires or via advanced mobile applications that enable them to monitor and track their own data. Many patients are experts in their own conditions, being the ones that are experiencing their impact on quality of life firsthand. It is still relatively unique within the industry for an RWE agency to have a team that focuses explicitly on patient-centered outcomes, as OPEN Health does, but having this brings significant benefits, including a much more in-depth understanding of patient experience. It’s important to note that it is this qualitative research that is providing these additional insights.”
“Even as treatment strategies become more tailored and personalized, the medical community may not always immediately recognize the importance of considering the patients’ perspective. I recently worked on a patient preference study where our client developed a treatment that is comparable to the standard of care in terms of efficacy and side effects. The only difference was in the mode of administration -- our client’s therapy was oral versus the standard of care which includes IV infusions and SC injections.”
“The patients we interviewed described the burden of IV/SC therapies and spoke about their strong desire to receive their treatments orally. Oral therapies would decrease the number of required visits to the doctor’s office, which can be especially meaningful to older and frailer patients. Most patients appreciated the convenience an oral drug could bring and spoke about how it would give them part of their lives back. One patient heard about the oral treatment and immediately went to the doctor’s office to advocate and request the oral drug. However, the doctor didn’t see a need for it at the time because the efficacy and adverse event profiles were largely the same.”
— Sophie Tsai, MD, Sr. Scientist, PatientCentered Outcomes & Patient Engagement, OPEN Health
"Even as treatment strategies become more tailored and personalized, the medical community may not always immediately recognize the importance of considering the patients’ perspective."
SOPHIE TSAI, MD SR. SCIENTIST, PATIENT-CENTERED OUTCOMES & PATIENT ENGAGEMENT, OPEN HEALTH