17th Annual eyeforpharma Barcelona (Commercial, Digital and Patients)

Mar 12, 2019 - Mar 14, 2019, Barcelona

Where Pharma Becomes The Solution

Patients: The New Architects Of Clinical Trials?

The industry has long flirted with the idea of getting patients to design clinical trials. But is the idea coming to fruition?



Eureka! The pharma industry is slowly realizing that the secret to successful clinical trial design is the unique insight that only a patient can provide.

However, realization is academic. This magazine is interested in the practical steps companies are taking to make it happen.

So, with that in mind, are companies making good on their promise to involve patients in clinical trial design?

“We are all in. We are certainly not perfect, there are still a lot of space for us to grow and continue to learn, but the commitment is substantial, and it's consistent across all of our therapeutic areas,” says Pfizer’s Craig Lipset.

And Pfizer certainly is “all in”. While some organisations may seek patient insights for studies being carried out in certain therapeutic areas, or in specific circumstances, Lipset explains that within Pfizer’s Global Product Development organization there is now a process and indeed, an expectation, for all studies across all therapeutic areas to come to the protocol review committee with patient feedback and insights in hand – this has been live for a year now. It hasn’t been easy, he admits.

“Innovation is really about scalability. The idea that we could do one or two innovative little experiments or have anecdotes here or there really isn’t that hard, or to find a team somewhere in the vast portfolio of studies in Pfizer that has used patient insights and can share those in a conference. In the wider industry just about every company has probably had teams that are doing this. I would be shocked if there are even larger or midsize pharma companies that aren’t engaging patients somewhere in their organisation. 

"If a company has done it once in an ethical manner and nobody has been fired or gone to jail then they can do it twice, but I think that’s where it gets hard. The hard thing for a big organisation like Pfizer is to say let’s take that and bring it to scale across the entire portfolio. But people at all levels are really excited about it.”

He adds that while there is no resistance per se, the tightly-regulated world of clinical trials meant there were some initial teething problems.

“One concern early on was whether this will only be meaningful to our organisation’s leadership if we are able to demonstrate quantitative measures of impact on study conduct, and if so how long will that take? But what I was impressed with was the willingness of the leaders in our organisation to agree that it is the right thing to do based on incremental insights from our earliest work.”

These perceived problems may be the obstacle preventing other organisations from going down the same road, notes Lipset. For example, he believes the fear – often misguided – of non-compliance is hampering meaningful patient participation, even where there is a will to do so.

“It’s not enough for an organisation to then say, ‘Ok teams, go and talk to patients’. For decades these teams have been warned that they can’t, and certainly there has been a prevailing perception among many development colleagues that you can’t talk to patients. In many cases study teams sincerely believed that there is some sort of codified rule somewhere out there that says we are not even allowed to.”

The fear of non-compliance is almost matched by the fear of the unknown. In this relatively new space, many organisations don’t even know where to start, admits Lipset.

“I think once you get people past that fear then it turns into, ‘it’s a great idea but I don't even know how to do this.  You tell me to talk to patients, do I just walk out on East 42nd street in midtown Manhattan and find people with fatty livers and rheumatoid arthritis? How do I go about actually doing this?’”

For Pfizer, it wasn’t a trivial exercise to put in place tools, processes, templates and training, as well as the manpower resources needed to achieve this across-the-board commitment to patient participation in clinical trials.

“It was a worthwhile investment,” he says, however. “Some of it is as benign as identifying a template to use for an agreement with a patient or an advocacy group and not sending agreements as if they were a consultant or a CRO. Or identifying if patients who share insights will be compensated for their time, and how much? And is that what’s considered a fair and ethical amount to compensate them? We have had to work through all that with a diverse team spanning Development, Innovation, Patient Affairs and Legal.”

Lipset is effusive about the positive impact of Pfizer’s new approach on the entire clinical trials process. “This has been a great thing for our teams in terms of study execution but also in terms of patient interaction. Because these teams are now enjoying the opportunity to actually talk to humans outside our four walls who will be impacted by these studies.”

Yet he admits it “isn’t all perfect”. “We still face the challenges of ensuring we are getting representative patient voices when we are gathering these insights. How many patients do you talk to -- is it five or is it 50? How do you ensure that the insights you are getting are representative of the geography and of the gender and ethnic diversity of the patient you want to include? How does one start to find real world patient insight gathering opportunities with tools like crowdsourcing or other types of digital platforms for patient insights?”

Unsurprisingly, Lipset is full of examples about how patient input substantially and importantly changed the fundamental design of a clinical trial. He describes one study that involved a substantial washout period.

“By that I mean the patient had to be on a certain therapy, and then they had to be off all medication for a period of time before they would be able to resume on a different arm in the study. From a clinical and scientific perspective, the study design was absolutely sound – it made great sense in terms of the pharmacokinetics of the different medicines and the importance of that washout.”

What the study design was missing was the human element, he explains.

“When you talk to actual human beings about what their life will be like during that period, without being on their medication it was simply intolerable. It was an amazing journey for that team having multiple sessions with those patients and in the end coming back with a modified protocol that was changed in terms of the design, based entirely on their input. This no doubt saved failed recruitment and retention in the study.  The impact of empathy was felt by everyone in the room – the patients shared how they approached the engagement with a study team with caution but were amazed at how the team asked questions, listened to answers, and adapted the design based on that feedback.”

“The FDA has been a champion for the need for patient input in clinical trial design over the last few years. They have really focused on this and I think that has trickled down to our industry peers, to ensure that this is a key component of the research that they are doing. “

Pfizer is not the only outfit taking up the baton.

The Michael J. Fox Foundation for Parkinson's Research (MJFF) recently launched the Fox Insight study, which is an online clinical study designed to collect information directly from patients and their loved ones about what it’s like to live with Parkinson’s disease. In the past eight months, they have enrolled over 25,0000 participants in the study, making it the largest global Parkinson’s cohort. Lauren Bataille, Senior Associate Director of Research Partnerships is set to present on this unique study in collaboration with a study particicpant, Will McIntyre, at the eyeforpharma Patient Summit USA in November.

“We see this study as an opportunity to engage the Parkinson’s community to share their unique experiences of how Parkinson's affects their daily lives – not only people living with the disease but also their caretakers and loved ones. By studying their responses to validated scales and novel patient-centric measures, scientists can gain critical insights into how the disease changes over time, differs from person to person and impacts quality of life. Our industry partners are excited about these data to better understand what’s really important to the patient and develop strategies to overcome key barriers to daily challenges,” she tells eyeforpharma.

“We aim to enroll over 100,000 people in the study and at that scale, expect to shine the spotlight on patterns among environmental exposures or experiences that may reveal potential causese of PD that can be further explored by my traditional clinical studies.”

Over the past few years, MJFF has sponsored a number of clinical trials in the digital innovation space, and Bataille says that in this new area of research the foundation has been proactive to seek patient input on study design through to implementation. “For Fox Insight, our study leadership works closely with study participants to identify what questions are most important, and to identify the clear and simple ways to pose those questions to a large, diverse study population. We have two patients who serve on the Fox Insight Steering Committee that work alongside leading researchers to develop new study intiatiives, novel recruitment and retention methodologies, and given the online nature of Fox Insight, their perspective on participating in a completely online study.”

 “We do recognize that Parkinson's disease is very heterogeneous and that one person’s experience living with the disease may be very difference else’s.”

The Foundation is now in the process of launching a beta tester program for Fox Insight. The goal of this group is to have patients give feedback on the participant experience within the study; an initial call out among the Fox Insight participants to gauge interest received an overwhelming response with about 600 people saying they were interested in participating.

Bataille acknowledges that each clinical trial is very different but notes bringing patient centricity to all types of clinical research will ultimately improve study experience and outcomes, which translates to new scientific findings and potentially new therapeutics.

The Fox Foundation may focus on Parkinson’s diseases, but collecting real-world evidence data directly from the patient community is a model that could be replicated across other therapeutic areas, says Bataille.

“We have a really powerful example of empowering and involving the Parkinson’s disease community in research. We hope our experiences can serve as a model for other chronic disease areas as well.”

 


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17th Annual eyeforpharma Barcelona (Commercial, Digital and Patients)

Mar 12, 2019 - Mar 14, 2019, Barcelona

Where Pharma Becomes The Solution

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