Patients as Partners in Medical R&D


Medical devices and therapies must go through a series of rigorous clinical trials in order to prove they are safe and effective for patient use. But more and more companies are looking at patient-reported outcomes (PRO) on a larger scale to ensure the solutions they are developing are sustainable, affordable, and accessible for people living with chronic disease, such as type 1 diabetes.

At T1D Exchange’s recent annual meeting, Dr. Alicia McAuliffe-Fogarty, vice present of the T1D Exchange’s Patient Centered Research, moderated a panel of experts who shared how and why industry should partner with patients to more deeply understand their needs and preferences before bringing new products to market. The general consensus of the panel was that a patient’s lifestyle impacts their ability and desire to fit therapies and devices into their everyday life with diabetes, and that without their input, products may never provide the solution its developers intended.

“One of the big challenges with the current therapies for treating diabetes, insulin-requiring diabetes, is that they’re not designed for sustainable use from the actual real world user,” said Jeffrey Brewer, president and CEO of Bigfoot Biomedical and type 1 diabetes caregiver, noting that diabetes and other chronic illnesses means doing something every day for the rest of your life. “How do we take that perspective and make it fit into a person’s life–actually make it more compatible with all of their other priorities, such as their family, their friends, or their job? That perspective, I think, is really lacking.”

Echoing that sentiment was Soren Skovlund, a 20-year patient-reported outcome specialist and behavioral scientist currently working in academia and in an entrepreneurial role at DrugsStars.

Skovlund said the healthcare industry and technology development environment is opening up more and more to partnering with the patient to take in the science of qualitative research and preference assessments to make the patient experience the starting point for product development through all phases.

“You always maintain that patient perspective and understand that there are different populations that may be going through different types of challenges every day,” he added.

Magaly Perez-Nieves, a research scientist at Eli Lilly and Company, and a 25+ year patient with type 1 diabetes, shared that patient-centered research is valuable to multiple stakeholders, including health care providers, regulators, and payers, as well as patients. Clinicians will have better communication with their patients if they understand what is important to them, regulators will understand which products are preferred and easy to use, and payers will have the information they need to understand the value these products provide in order to make coverage decisions.

“[Patient-reported outcomes] are important in different areas, not only from a research perspective, but also a real world perspective,” she said. “We need to start incorporating it into these different aspects.”

Brewer added that certain products will appeal to different people, such as the current choices in blood sugar monitoring. He said that PRO data is a great tool for understanding the differences amongst different people, so that we develop solutions that work for people with different preferences.

“There are some people that are willing to put [chronic disease] at the forefront of their lives and give a lot to it in order to optimize their health. And then there are people who just refuse to do that,” said Brewer. “We should have solutions that meet the needs of both of these kinds of patients.”

The fourth panelist, Anne Busk, is a global health economics and outcomes researcher at Novo Nordisk. Busk’s work is focused on working with clinical trials, determining how and what data to measure. She is hopeful that all of the stakeholders will begin to see the value of patient-reported outcomes research so that in the end, everyone wins.

“When you use PRO intelligently in the development process, you can predict what will happen in the real world. Regulators are starting to acknowledge that all of these patient perspectives actually mean a lot. We just need to make the payers understand the same thing.”

Skovlund and the other panelists agreed that people with diabetes should be looked at as partners in the research and development process, not just input-providers.

“Many people have thought that patient reported outcomes, by FDA guidance, is based on focus groups, qualitative research that defines the domains you measure and so move on. But there’s more to patient involvement than that,” said Skovlund. “We have the opportunity now, we have the experience from the industry on successful patient partnering, to begin to revisit how we optimize the application of PRO for the benefit of all of the stakeholders in the healthcare systems.”

Brewer and Perez-Nieves pointed out that the current system of clinical trials sources patients from a skewed population, and will never truly mirror the real-world experience of the majority of patients.

“We’re measuring these PROs from the best, most engaged doctors, the most engaged patients usually with the best insurance and the best education levels and support at home,” Brewer said. “The real world is that two-thirds of people in this country are treated by primary care providers and that primary care provider is literally along for the ride, mostly writing prescriptions. It is all the patient, and they don’t have much support. The trials, they don’t ever approximate that environment.”

Perez-Nieves added, “We concentrate a lot on the clinical aspect and on the data. We also need to educate ourselves and our physicians and other stakeholders on how to interpret the data.”

T1D Exchange is dedicated to understanding patient needs and preferences as new therapies come to market. By building a large cohort of people with diabetes as well as their caregivers and other supporters, they hope to understand the impacts of both the medical and psychosocial outcomes of these therapies. For more information, visit

 –Amy Bevan/GluMom

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