Whatever life throws at Phil Green, he tackles. When he wanted to be a kicker with the University of Washington football team, he hit the gym daily until he scored a spot and ultimately won the 1991 National Championship. When he wanted to enter the business sector, he went from a start-up company to mingling with Sony's top executives in just a few years. And when he was diagnosed with amyotrophic lateral sclerosis (ALS) in 2018, he took on researchers and public policy to effectively transform ALS clinical trials, expanding access and making them better aligned with the needs of patients.

For better or worse, I don't know how to back down from a challenge," said Phil. "It was really never an option for me to feel sorry for myself or retreat. If anything, my diagnosis made me more motivated to drive change. I thought, 'Okay, so my time is more limited; how much of a difference can I make with what I've got left?'"

As it turns out, a lot. Phil jumped into ALS research with both feet, currently sitting on the Clinical Trials Transformation Initiative's (CTTI) Steering Committee as a patient rep, along with 12 other advisory boards for trial design. Phil is also a team leader for CTTI's new Disease Progression Modeling project, and a former team lead and team member for CTTI's Decentralized Clinical Trials Update project and Novel Endpoints Acceptance project, respectively.

Don't think one person can advance meaningful change for an entire community? Phil's story will convince you otherwise. Here is the story of how he is bringing CTTI's Transforming Trials 2030 vision for patient-centered, easily accessible trials to life.

The rocky road to navigate ALS patients' biggest hope: investigational research

ALS is a relentless neurodegenerative disease that attacks nerve cells in the brain and spinal cord. There is currently no cure, so investigative therapies represent the best hope for slowing or halting the condition's progress. But, as Phil learned, ALS trials are few, and, in 2018, the barriers to participants were many.

"The ALS study that was really getting attention for its potential was a stem-cell trial," said Phil. "I knew I wanted in, but so did everyone else-patient demand was extremely high. When I found out I was accepted, it was the first time since my diagnosis that I felt real hope."

Phil had good reason for that hope. He credits that trial with slowing his ALS progression and extending his ability to speak for longer than anyone expected. But the trial asked a lot of participants. To begin, it had a three-month "observation window" during which participants had to wait for treatment. While these observation windows are common in clinical research, ALS's rapid progression made the wait frustrating.

"Decline that could take years in other conditions can happen in a single month for an ALS patient," said Phil. "From the very start, there was this sense of a disconnect between clinical trial design and the urgency that comes with the lived ALS experience."

There were more challenges, too. Although Phil was lucky enough to live only an hour and a half away from the site, other participants had to travel much further-many even had to fly with no reimbursement. Once treatment began, it was a 50/50 chance of getting the investigational treatment or placebo. However, regardless of receiving placebo or treatment, every participant was subjected to seven lumbar punctures and a bone marrow harvest-incredibly invasive procedures. The study also offered no open-label extension, so even if you received the treatment, you couldn't continue with it once the study ended. For the unlucky placebo participants, no open label meant they'd spent valuable months jumping through hoops for a trial that would ultimately offer them nothing at all.

"The lack of patient centricity in this trial didn't sit right with me," said Phil. "I teamed up with other participants, and after two years of advocating, we were able to convince the research team to offer expanded access. This was the turning point when we realized that as patients, we have the power to demand change. So we went to work."

Changing the landscape of ALS trials

A "go big or go home" guy at heart, Phil didn't stop at changing one trial-he wanted to change them all. And he wasn't alone. He'd already built a connected community of people living with ALS from his clinical trial, which he expanded further by partnering with I AM ALS, a patient-led community that gives support and resources to people living with ALS.

"They are my partners in ALS crime," Phil said with a smile. "We shook things up. We made good trouble."

That "good trouble" took the form of a bold decision: ALS advocates would no longer tolerate ALS trials that don't have an open label extension. Further, they created a list of nine criteria for patient-centricity they wanted to see in ALS trials and publicly rated active studies to encourage participation in those that considered patient needs in their design. They called this tool the Patient-Centric Trial Design Rating Tool, or PACTD.

"The beauty of PACTD is that it got sponsors' attention because it reminded them of the power of patients," said Phil. "If they can't fill their trials, they lose money. But that's not what we want. We want to advance ALS research, and for that to happen, patients and researchers need to work together. PACTD lays the groundwork for us to all succeed."

Phil and his colleagues counsel trial sponsors on how open label extensions and expanded access are often well worth the cost, given the spike such programs drive in patient participation and, consequently, faster results. Their efforts paid off. Today, every single ALS trial-even Phase II trials-has an open-label extension.

Not today, ALS

The success in transforming ALS research that Phil sees has only invigorated his drive for change. He now sits on 13 advisory boards to support better trial design and recently met with the U.S. Food and Drug Administration (FDA) to update the guidance document for ALS clinical trials. Today, sponsors come to him asking how they can improve their research to bring in more ALS participants. Phil's daily schedule is filled with phone calls that would rival any executive agenda-he regularly advises lawmakers, research teams, other people with ALS, and progressive initiatives with a passion to improve research. The latter includes CTTI.

"CTTI is on top of all things patient-centered," said Phil. "All the tactics I want to start seeing in ALS research, like decentralized trials and novel endpoints, CTTI is dedicated to furthering and developing best practice. So many issues we are tackling in ALS research, CTTI is already addressing."

CTTI's Transforming Trials 2030 initiative takes a macro-view of what it will take to bring the same kind of accelerated, patient-centered research Phil has championed in ALS to the entire clinical research spectrum. The input of patient partners like Phil is essential to bringing that vision to life, but Phil sees his role as a patient representative of CTTI's Steering Committee as a two-way street.

"I bring ALS to CTTI, but I also bring a huge amount of knowledge from CTTI back to the ALS community," he said. "CTTI really gives me the opportunity to have the biggest impact on my disease."