Clinical trials for neurological conditions can be some of the hardest to fill and conduct. But there are dedicated groups working with patient and caregiver communities, and with neurological disease researchers to make progress. Clinical Informatics News spoke with three such initiatives to explore how participant recruitment and other aspects of clinical trials are changing. –The Editor  

By Maxine Bookbinder

February 12, 2018 | The Michael J. Fox Foundation (MJFF) for Parkinson’s Research is using patient reported outcomes to find a cure for Parkinson’s Disease (PD) with Fox Insight, a “patient-powered”, observational online clinical study. “It flips the traditional [clinical trial] model on its head,” bridging the gap between researchers and patients’ unmet needs, says Katie Kopil, Director, Research Partnerships at the Michael J. Fox Foundation.

MJFF meets volunteers at their needs level, offering an online space apart from a traditional clinical trial to allow patients and caregivers a place to connect and vent. The Foundation believes that patients living with the disease are the experts and, therefore, should be active participants in finding a cure.  Will McIntyre, a Parkinson’s patient and MJFF contributor, wants to see more patients participate with MJFF or other PD foundations. “This disease is different for everyone. The more we know how each person experiences it, the better. The more we learn.  As Michael [J. Fox] said, ‘The cure won’t fall from the sky and hit us over the head.  We have to go up and get it.’”

MJFF launched Fox Insight in beta in March 2015, and announced the study’s formal launch in April 2017. Fox Insight is, “a digital platform and dynamic online clinical study, to build a large, diverse cohort of people with Parkinson’s and age-matched control volunteers.” Volunteers answer quarterly questionnaires, considered study visits, for five years. Their responses will be de-identified and made available to the scientific community in real time.

Before joining Fox Insight, McIntyre, a 51-year-old technology account manager, completed a traditional clinical trial for an experimental drug but wanted to put his technology expertise and passion for finding a cure to use. By chance, he attended a MJFF clinical trial fair in Philadelphia. “The total market strategy was appealing,” says McIntyre. “I know the Michael J. Fox name, that it is dedicated to research. The cure comes through research, they have people pull together for a cure and aggregate data. It’s an organization that moves the needle to find a cure.”

McIntyre started in a Fox Insight pilot study using a smart watch. Now he is on a patient steering committee for the platform and regularly participates in MJFF projects, including multi-media efforts, to increase awareness of the importance of clinical trial participation to find a cure.  

McIntyre prefers Fox Insight’s online format to “intimidating” in-person visits. He says the questionnaires take about 30 minutes to complete, are pertinent and intuitive, well-designed, and easy to navigate. As PD progresses and mobility decreases, some patients become increasingly isolated. “It’s easier to answer questions from the comfort of home,” says McIntyre. “I am as honest as I can be. I’m not a guy to sit in support circles and weep and be sad. I’d rather dive into it online, say how I’m feeling, and contribute.”

According to a Harris Insights & Analytics survey, 80% of PD patients are interested in participating in a clinical study, but only about 25% actually do. The usual reasons of work, family, and daily obstacles prevent some from entering trials.

For patients like McIntyre, the Fox Insight online platform is intended to remove these obstacles and give its 15,900 volunteers a platform through which to share their experiences including symptom assessment and medication response, directly with researchers. While most of the Fox Insight participants self-report that they have Parkinson’s Disease, approximately 20% of are control subjects, most of whom have family or friends with PD. The only requirements are that participants be 18 or older, speak English, and have an Internet connection. Participants register online, and a virtual study coordinator sends reminder emails for quarterly questionnaires.

Real World Insight

Fox Insight is not meant to replace traditional clinical trials, but instead to provide engagement for patients, lending an authentic voice to the data on issues such as daily struggles of PD, examining how heterogeneous PD is among patients, and optimizing care based on patient-centered approaches.

“As an online platform, Fox Insight can make research participation much easier for patients. For researchers, the platform is a low-cost way to study individuals over longer periods of time, including people who might have already participated in in-person research studies,” says Kopil. “In general, collecting self-reported information from a very large number of people complements these traditional in-person assessments which might capture more diverse and robust data from a small number of people.”

Impediments to Parkinson’s disease and other trials can include trial eligibility criteria and insufficient health insurance. Trials that pay for diagnostics, such as MRIs and PET scans, to ensure they recruit appropriate patients are inclusive of all socio-economic groups. However, trials that rely on these tests to ensure eligibility automatically disqualify patients who can’t afford the tests or doctor visits.

Parkinson’s disease itself presents increasing mobility challenges.  Most PD trials are at limited academic medical centers, and referrals are usually made by a movement disorder specialist; however, many PD patients see a general practitioner. Eligibility has severe restrictions and sometimes the intensity of a protocol is prohibitive. For these reasons, Fox Insight can be the first entry into a clinical trial for many patients.

Collecting online data gives real-world insight. Researchers can examine volunteer data submitted through Fox Insight for inconsistencies, common points, and other areas to ascertain what new questions to ask, problems to address, and unmet needs to tackle to eventually find a cure or at least more effective therapies.

“Curated, de-identified Fox Insight data will be made available to Parkinson’s researchers worldwide in real time in 2018. Researchers can use this robust dataset from a large, diverse cohort of patients and control volunteers to test and generate scientific hypotheses and form a more complete picture of Parkinson’s disease,” says Kopil.

In addition to quarterly study questionnaires, Fox Insight volunteers have other options, such as one-time questionnaires sponsored by independent researchers and most recently, a genetic study for those diagnosed with PD and living in the United States.

Despite the possibility of self-reported inaccuracies, the large number of responses ensures statistical significance, says Kopil. And participants have a vested interest in honesty.

“All data leads to a cure. I want to give every bit of data I have,” says McIntyre, a Fox Insight participant for more than a year. “What if the next researcher sees something no one else has thought of before? There is no hidden agenda. We all know what the goal is.”

He is optimistic, although he knows today’s research may not benefit him. “I’m investing for my daughter and potential grandchildren. I have a good feeling about contributing something for the common good.”