#patient input

Patient organization-led innovation

As patient-generated data are becoming increasingly important in the R&D world, patient organizations themselves do much of the innovative work to gather this information and discern how it can be useful within their communities.

In the first panel of the workshop, representatives from the Epilepsy Foundation, COPD Foundation, and LUNGevity Foundation discussed how they have been engaging with their patient communities, successes they have seen (and hope for), and what is limiting full utilization of the data.

Andrea Ferris, president and CEO, LUNGevity Foundation, and her team are focusing on collecting robust information that is representative of their entire community, making the change from solely surveying patients to studying their preferences and understanding their needs using social-science-based approaches. “With the rapid expansion in the number of new therapies for lung cancer, we need to learn what patients want from their treatment now. What tradeoffs are they willing to make for quality of life vs. quantity of life?”

LUNGevity is launching a large national study to ensure that it receives input not only from very engaged patients but also from a broader range of people living with lung cancer. It has enlisted patient ambassadors to recruit from their local clinic sites, and it is collaborating with the National Black Church Initiative to expand outreach. Data they collect will inform industry partners, regulators, payers, and policy-makers to best benefit the community.

Sonya Dumanis, senior director of innovation, Epilepsy Foundation, reinforced the crucial nature of collaboration, noting that no perspective is useful when it exists in a silo.

Jamie Sullivan, vice president of public policy and outcomes, COPD Foundation, shared her organization’s view on the patient perspective and its role in research. “Our founder, John Walsh, always said as a patient himself that if you’re not at the table, you’re on the menu.” The COPD Foundation strives to bring the patient voice “to as many tables as possible” and has been investing in infrastructure to gather those data directly from their large communities. They have built up a registry platform and connected it to a social media platform to leverage the insights of their large community. They now attract 250,000 hits each month.  

While patient engagement in these data-gathering programs may differ across disease populations, there are similar signs of success the panelists strive for in their areas.  

For Dumanis, success is being a part of an integrated learning health-care system in which data are considered holistically and put to use. One way she and her team are already successfully utilizing the patient perspective is through the design of a seizure gauge device that reflects attributes of greatest importance to patients.

Common roadblocks identified by the panel include a lack of resources, regulatory hurdles, difficulty reaching unengaged populations, and knowing when the data are “good enough” to be useful to decision-makers.

As the science of patient input develops, it is challenging to innovate existing practices. Ferris said, “There is nothing easy about it… It is a long-term process, and you have to think of it as a long-term and iterative process that evolves.” On a hopeful note, she also shared her observation that the science of eliciting patient input has matured, and there is more willingness to rely on it.

Patient input in action at CDRH

FasterCures’ Kim McCleary, managing director and acting executive director, interviewed Jeff Shuren, director, Center for Devices and Radiological Health (CDRH), U.S. Food and Drug Administration (FDA), during the second part of the workshop.

A pioneer in the science of patient input, Shuren recalled his start at CDRH in the fall of 2009, a time during which no one was happy with the Center’s performance in getting innovative devices to market. This necessitated a deep dive into the program to define a common mission. “We always talked about protecting and promoting public health, but what does that really mean? Ultimately, for us, it means improving health and quality of life for patients.”

What followed this vision-setting was a budding commitment to better understand and consider the patient perspective when making regulatory decisions, starting with weighing benefits and risks.  

“How do you know that a benefit outweighs a risk? Do you look at the stars? Do you read the tea leaves? There’s no data that tells you the benefits outweigh the risk other than that it’s someone’s perspective,” said Shuren.

The Food and Drug Administration Safety and Innovation Act in 2012 was a turning point with regards to FDA activities to understand patient perspectives. That year, CDRH launched the Patient Preference Initiative and began a series of activities to better understand methods that could help assess how patients make tradeoffs for benefits and risks. A draft guidance on benefit-risk assessment was issued in 2014 that first recognized the importance of the patient perspective. A year later, they issued draft guidance for including patient preference information in applications. This year, they convened the first-ever FDA Patient Engagement Advisory Committee.

A priority for the Center has been ensuring that all staff, regardless of department, engage with patients. Shuren shared examples of how they have met that goal and how much they have learned in the process.

When it comes to strategic partnerships with patients in order to gather their input, Shuren makes clear his goals: “We’re in this together, and we’re going to get over the finish line together.”

Data-driven R&D

In the third discussion of the afternoon, Annie Saha, director, external expertise and partnerships, CDRH, FDA, moderated a conversation about data-driven R&D happening in public-private partnerships and the medical device industry.

Saha began the conversation by asking the panelists how they define patient-generated data and how they hope to improve health outcomes for patients in ways that are meaningful and purpose-driven.

Kara Haas, global lead, regulatory affairs policy and intelligence for outcomes and evidence, Johnson & Johnson, defines this information on a broad spectrum but urged that “Patient engagement is critical to the entire product lifecycle in order to address the unmet or inadequately met needs from the patient perspective.” That information should pertain to what patients value, what potential solutions should look like, how a solution is tested, and what change occurs in the patient population after the solution is marketed and utilized.

The panelists made the distinction that although most available data ultimately come from patients, this new approach to gathering information should focus on collecting data about domains that matter most to patients. Stephanie Christopher, program director, Medical Device Innovation Consortium, is asking questions such as “What are the outcomes that are important for patients? What are the things that will motivate them, what pieces of information would they want to share?”

While all speakers agreed that gathering this information is critical, the conversation turned to how complicated it can become.

Greg Daniel, deputy director and clinical professor, Fuqua School of Business, Duke-Margolis Center for Health Policy, added, “The challenge we face is – how do we use the data? How do we know that it’s good quality? When can it be used? These are really tough questions.”

Regarding this global challenge, Barry Liden, vice president, patient engagement, Edwards Lifesciences, mentioned that “The way health-care systems work, the people who determine which therapies to purchase are not always patients. As a result, we have to focus our data generation around questions asked by payers who always have patient’s interests at heart, but they also have their own perspective and look at their data needs through their own lens. So really, this is once again trying to put patients first.”

Using information from patients to inform the design of clinical trials, including identifying trial endpoints that matter to patients, is a priority for these panelists. Christopher is working on a large-scale collaborative study on Parkinson’s patient preferences that will detail their tolerance for risk and expectations of benefits. Daniel and the Duke-Margolis Center have been responding to the 21st Century Cures Act to guide how FDA could use patient-preference data as global evidence in regulatory decisions.

In thinking through ways to push this new science along, Liden spoke of the importance of strong leadership that prioritizes the patient perspective, congratulating Jeff Shuren on being a thought leader on the topic around the world. With more industry leaders like Shuren in the field, there will be greater success in fast-tracking patient-perspective initiatives in powerful ways.

The value of patient-generated data in the value discussion

Moderator Cynthia Grossman, associate director, science of patient input, FasterCures, started off the final panel of the day with the claim that “Value is almost as big a buzzword now as patient-centricity, but there are a lot of questions about what value means.”

Suz Schrandt, director, patient engagement, Arthritis Foundation, shared that value from a patient perspective is continuously redefined. “As a patient advocacy organization, you have data coming at you all the time, whether you realize it or not. Every conversation, every conference, every support group meeting – those are all loaded with data points.” She continued to say that her mantra is, “Data collection is not an activity, it’s a state of being.”

Schrandt went on to share an example of identifying crucial patient perspective at a conference over the summer. She learned that patients were struggling with an injector device that had been designed without their needs in mind. She presented that information to a group of biomedical engineers. Athough that insight was not the result of a large-scale data collection exercise, “but rather just emerged from conversation,” it was actionable data that will make a positive impact on patients.

Not only are patients a critical voice, but providers also possess viewpoints that are fundamental to a functioning biomedical ecosystem.

Erin Holve, director, Health Care Reform and Innovation Administration, Department of Health Care Finance, Government of D.C., and her team are attempting to “thread the needle” and integrate patient perspectives into their conversations with purchasers. She wants them to understand that they share the same objective, which is to reflect individual patient needs and provide patient-centric care.

In terms of “speedbumps” impacting the utilization of the patient perspective across the industry, Josh Seidman, senior vice president, Avalere, noted that “Data is everywhere, but that doesn’t mean that it’s in a format that we can do anything with it. It’s figuring out how to structure that data and collect it in a timely way.”

The panelists closed the discussion by reiterating the human value of this new science. “When we think about the science of patient input, I’m always thinking that we can’t ‘science’ the human right out of it,” said Schrandt.

Workshop wrap-up

Ending the panel discussion at the close of the afternoon, Grossman asked the panelists what specific items they find most promising about the future of the science.

One example came from Seidman, who answered, “There is a real enthusiasm around shared decision-making that is going to move things forward, from measurement to data collection to new payment models. These are going to be real leverage points over the next year or two.”