The Value of Openness by PatientsLikeMe ®

PatientsLikeMe has a long tradition of bringing the patient voice into drug discovery and development, but it’s only been in the last year or two that industry has been tuning in to hear what that voice is saying about clinical trials. For the last 18 months, we’ve been asking many of our members about their attitudes toward and experiences with clinical trials, and working to ensure pharmaceutical companies understand what patients want most from trials, now and in the future. We talked with Jeremy Gilbert, Vice President of PLM Health, and Paul Wicks, Vice President of Innovation, about what we’ve found, and how that’s already helped to change the design and execution of trials and their future direction. Jeremy will be presenting more about this topic and details of a key project with AstraZeneca and Takeda that leverages the patient voice in new ways at the Patient-Centered Clinical Trials Conference in Philadelphia on October 11. More info about discounted tickets below.

Jeremy Gilbert, Vice President, PLM Health

You’ve surveyed more than 1,400 people about their experiences and opinions with clinical trials. What have they shared, and what has PatientsLikeMe done with that information?

Jeremy: A lot of people in our community are living with life-altering, degenerative conditions, and they’re acutely aware of the importance of new drugs coming to market to address their problems. In conditions like MS, where there are perhaps a dozen treatments on the market, people are keenly aware that the drug they’re taking may stop working, so there is great interest in helping new drugs come to market. Our members are vocal, too, about how involved they want to be. About 92 percent of our survey participants said they would be willing to help researchers design clinical trials to make them better for patients. At the same time, our industry partners are looking for ways to improve trial participation, especially since trials will grow more complex. So we’ve found these two groups want to come together to improve the trial experience for everyone. And there’s no better place to start than by involving patients in the trial design process.

Paul: The state of art in designing trials has been to use the advice of physician experts and to review literature and published papers, but patients have been left out. Researchers have told us this is because of legal and regulatory concerns about asking patients about experimental treatments; they don’t want to be seen as promoting a treatment that hasn’t yet undergone testing. They also cite their lack of experience and fears about the extra time it might take them as reasons why they’ve carried on with the status quo. We’ve long believed that online communities like PatientsLikeMe provide a great vehicle to attack those barriers. We can act as an honest broker of information between researchers and patients who want to help, we’ve developed a repeatable methodology to gain patient input, and we’re delivering results in a matter of weeks to researchers to improve their protocols. Increasingly now researchers are coming to us before they design their trial to incorporate the patient voice, which is a great step.

Does what patients want from trials depend on the condition they have?

Paul Wicks, PhD., Vice President, Innovation

Paul: Some elements, such as their desire to altruistically help other patients like them, stay remarkably consistent. But depending on their demographics and the impact of disease, we do see a lot of variation. Working age adults might want extended hours so they can participate outside of normal business hours, whereas older people might prioritize having transport organized for them. It really depends on the aim of the study as much as the condition; some trials are for drugs that slow the progression of a disease, while others are to treat specific symptoms or even to take an existing drug and make it more convenient to take, such as moving from an injection to a pill. Against that you’ve also got the overall commitment required to the trial protocol such as the number of study visits, their duration, any special testing that people have to undergo and, of course, any special measures put in place to mitigate those burdens. We think patients are interested in participating in research in general because of altruism, that they choose to enroll in a particular trial because of its objectives, and that they stay enrolled because of their relationship with trial staff and the level of burden the study incurs on their daily lives.

The design of a trial is just the start. What happens as trials begin, or end?

Jeremy: We’re starting to see another gap now, which is that companies have no way of soliciting feedback from patients as they participate in a trial, to find out what patients think of real trials. This is a surprise, because given most of us are consumers, we’re used to being able to give feedback about a product or our experience at any time. That inspires us to think about how to gather patient input not just about a trial’s design, but also during the trial itself, and even after it ends. And if we can figure out how to share the knowledge that’s gathered, that would help industry design and run even better trials in the future.

Where do you think trials are headed in the future, and how is the idea of patients as partners instrumental in guiding that direction?

Paul: Today, taking part in a clinical trial is like having a part-time job. In the future, we want it to be much more convenient and to harness the digital technologies that have become a normal part of daily life. We’re also looking at novel technologies to see if we can do away with some of the aspects of trials that patients dislike, such as placebo arms. Using online digital platforms, it might be possible to produce enough carefully-matched historical controls to more rapidly identify when a treatment is having an effect. That could reduce costs, speed up trials and ultimately increase the number of “shots on goal” that researchers can take to fight disease.

 
Note: Jeremy will be presenting more about this topic at the Patient-Centered Clinical Trials Conference in Philadelphia on Tuesday, October 11 at 2:00 PM. He’ll be joined by Arnold Degboe, Director of Patient Reported Outcomes, Health Economics and Outcomes Research at AstraZeneca. They will talk for the first time about their work to change clinical trials with patient feedback. Discounted tickets for patients and industry participants are available from the conference organizer. Please contact Katie Gadsby for further information at kgadsby@eyeforpharma.com.

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Picture this: you’re a medical professional and are about to open the doors to a conference you’ve spent years pulling together. You’ve booked your venue, have your sponsors lined up, got top headliners to give keynotes, picked your Twitter hashtag and have invited every industry pro to be a part of the event.

In walk the physicians; check. Allied health professionals; check. Pharmacists; check. Industry leaders, life science CEO’s and top researchers; check, check, check.

Notice anyone that’s missing? If medical conferences exist to create a network, to stimulate new collaborations, overturn redundant thinking, and to help attendees return to work reinvigorated, the conference that excludes patients misses hearing the most important voices in medicine.

But luckily for you, patients living with a variety of conditions aren’t just sitting on the sidelines anymore. They’re proactively participating in these conferences any way they can, even if that means going through the virtual window. They watch the hashtag, pose questions, say they wish the conference were live-streamed, and ask attendees to smuggle out knowledge they could use to improve their care or offer them hope. If they’re ignored or marginalized they take to the Twittersphere and let their displeasure be known.

“The slow boring of hard boards”
Over a decade ago the Outcome Measures in Rheumatology (OMERACT) began inviting patients to its meetings, initially as observers, later as participants and then finally as true co-producers. It was a long journey, and as patient research partner Maarten de Wit has described, not always easy for its patient members. However the team has documented the benefits of patient inclusion such as: “widening the research agenda; including patient relevant outcomes in core sets; enhancing patient reported instruments; changing the culture of OMERACT… identifying previously neglected outcome domains such as fatigue, sleep disturbances and flares which prompted collaborative working on new programmes of research.”

A new mechanism for change
On May 1^st 2015, a group of volunteers hope to take patient inclusivity to the next level by releasing version 1.0 of a crowdsourced charter called #PatientsIncluded. It contains five clauses based on Lucien Englen’s astute observation that there are too many conferences lacking patients on the planning committee, on stage, and in the audience. Medical conferences that are certain they meet the criteria have the right to use the “Patients Included” logo, and we hope will rapidly come to realize the benefits that can accrue.

The charter is by no means a silver bullet – it is a tool to encourage conference organizers to think past tokenism, be ambitious in their striving to integrate, and to reward those who take the uncomfortable step to try something different. My hope is that a wellspring of advice, best practices, and strategies will emerge from conference organizers as they deal with issues of cost, inclusivity, managing tensions, and reflecting on the changes made. Patients will develop their own resources to help one another plan, influence, participate, and engage in what might well be an uncomfortable and unfamiliar environment themselves.

As a member of the 5-strong patient panel at this month’s International Forum on Safety and Quality, hosted by the BMJ, there was much to admire from the way patients were celebrated and invited to speak and chair sessions. It was also clear that the scientific advisory board is taking seriously the notion that patients are  experts in their own right with much to offer. However it’s clear too that advocating and just being physically present at a medical conference is hard and exhausting work.

I hope actors on both sides of the qualification fence will work to support one another and acknowledge that this is a journey. Mistakes will be made. Iterations will have to occur. The name itself, version 1.0, demands that we constantly realign  and have the confidence to continue forward. The reward for the future will be that of professionals and patient-experts joining forces to advance our collective understanding of illness, formulate new plans of attack, and forge new weapons in the fight against disease.

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