Critical Path Institute 

Moving Drugs to Market Smarter, Faster for 20 Years

By Tara Kirkpatrick

Like many big ideas, Critical Path Institute essentially started on a napkin.

At a late summer dinner at Vivace in 2003, founder Dr. Raymond Woosley and a few trusted friends discussed how to speed up a stalled drug development pipeline. The notes they took that night would become a business plan, and ultimately in 2005, Critical Path Institute.

Today, 20 years later, Critical Path Institute is a Tucson-based, global nonprofit that has changed the trajectory of the industry. C-Path’s pioneering work as a neutral convener, trusted resource and generator of drug development solutions for companies and regulators alike has helped accelerate new therapies for diseases, both common and rare. 

“What seemed impossible – to optimize the process for drug development and make it truly efficient – is now our reality,” said Dr. Klaus Romero, CEO of C-Path. “We were able to turn the dream of someday into today.”

“C-Path’s work has fundamentally changed the way the industry approaches drug development,” said Wainwright Fishburn, chair of C-Path’s board of directors. “Through its leadership in regulatory science, data integration, and patient-centered research, the organization has made an undeniable impact on the lives of people worldwide.”

Fixing an Inefficient System

The early 2000s should have been a promising time for medicine. The first draft sequence of the human genome was announced in 2001, offering a trove of genetic information to decipher disease and human health. 

But drug development had become too complex and costly for innovation–so much so, that Dr. Janet Woodcock, then FDA’s principal deputy commissioner, led the writing of a report about the dire need for a new “critical path” for drug development, in which regulators and pharmaceutical companies worked together to better the process for everyone, focusing especially on diseases with unmet needs. 

The timing was fortuitous–Woosley, then-dean of the University of Arizona College of Medicine, was ready to start this “critical path” venture right in the desert. The City of Tucson, Pima County, Thomas R. Brown Foundations, Jim Click, and I. Michael Kasser were among the many donors who helped raise $5 million for the revolutionary idea.

Longtime C-Path board members, bioscience executives Shaun Kirkpatrick and Jeff Jacob have been involved since that pivotal 2003 dinner. 

“We had a number of things in mind when we sketched out the first business concepts,” said Kirkpatrick. “The first was to do something that would speed up drug development in the pre-competitive space, which would become available to all involved as a common good; second, was to harness the unique talent, collaboration and innovative spirit of Arizona.  When the FDA’s Critical Path Initiative White Paper was published in 2004, the timing could not have been better.”   

Jacob wrote the business plan, and when C-Path was incorporated in 2005 with six employees, he served as its first chief program officer. “I had to literally walk the halls of the FDA multiple days…looking for ideas for initiatives to make the medical product approval pathway better.”

The first effort C-Path launched was the Predictive Safety Testing Consortium, to leverage “biomarkers,” or measurable indicators of biological processes in people, to better bridge the gap from the lab bench to bedside. These efforts are also helping to minimize animal testing.

“Now, the biomarker qualification pathway is a common process pursued by many groups around the world,” said Jacob. “C-Path was not only the first, but the one who helped create the entire pathway with the FDA. C-Path did the same thing with the EMA, as well as PMDA,  Japan’s regulatory agency.”

Said Woosley, “It felt like serving on the USS Enterprise, charting unexplored territory and asking our small team to boldly go where no one had gone before. We didn’t just embrace the challenge; we thrived on it! And we attracted the very best scientists drawn to exploration and the chance to redefine what’s possible.”

Another early C-Path supporter was the Flinn Foundation, a privately endowed foundation dedicated to improving the quality of life in Arizona. Flinn had completed its first Arizona Bioscience Roadmap in 2002, a plan to boost bioscience in the state. C-Path became one of its early success stories.

“As C-Path became a trusted convener in regulatory science, Arizona gained a seat at the table in key national and international discussions on drug development, precision medicine, and rare diseases,” said Dr. Tammy McLeod, Flinn president and CEO. “This bolstered Arizona’s reputation as a serious contributor to solving complex health challenges.”

The Core of C-Path

Today, C-Path employs over 150 people across the country and globe, and has added a European headquarters in Amsterdam.

The therapeutic areas of focus in C-Path’s current portfolio include Alzheimer’s disease, Parkinson’s disease, Type 1 diabetes, as well as rare diseases such as Huntington’s disease, amyotrophic lateral sclerosis, Duchenne muscular dystrophy, Friedrich’s ataxia, polycystic kidney disease, and more.

C-Path’s involvement in each area starts with an “ask”, either by regulators, a patient group, a pharmaceutical company or a health association wanting to partner. Those asks happen a lot, Romero explained, so C-Path weighs its participation based on a due diligence process to define the unmet need.

C-Path’s core competencies include biomarkers, data science and management, clinical outcome assessments, modeling and analytics and regulatory science. “We build each partnership around these,” Romero said. 

“Each one of our efforts has a highly experienced and trained scientific lead and a core team,” said C-Path President and COO Kristen Swingle. “It’s a unified and cohesive approach to how the work gets conducted.”  

The consortia are inclusive. “We convene all key stakeholders but most importantly, we insure the patient voice is central to informing the solutions we devise,” Swingle said. “You need to make sure it’s being incorporated into the process for developing treatments that directly impact them.”

A foundational distinction of C-Path is its custodial role over the vast amount of data contributed to the institute

C-Path has earned the trust of both government and industry, so they share information once kept strictly proprietary. For example, if a certain drug trial fails, the details about sample size, participants, study design and more were often lost, when they could potentially hold the key to a future trial success. C-Path secures such crucial data, and protects its sources.

“To overcome concerns about the legality of sharing pre-competitive scientific data, we relied on a rock-solid legal foundation and an exceptional legal agreement for our consortia…,” said Woosley. 

Over the years, C-Path has compiled 700,000+ anonymized, de-identified records of individuals who participated in research, among many trials, studies and additional data sources. “This is unique to C-Path, there is not another entity that has data this comprehensive,” said Romero. 

In analyzing this rich data and engaging many perspectives, drug development in each therapeutic area becomes smarter and more streamlined, to encourage innovation and inform decisions, all with the regulators’ insights along the way.

“Data are like Lego bricks,” said Romero. “We have the ability to work with data and create any possible drug development solution with those Lego bricks, and what we create is always based on addressing unmet needs, where patients are waiting to have treatments.”

Engaging the Stakeholders

Through its work, C-Path also engages and inspires patients and advocates. 

Sarah Zenner-Dolan is a vibrant 58-year-old woman living with young onset Parkinson’s disease. Forced to quit her job as her symptoms worsened, she found solace participating in clinical trials and joining the bicycle riding team for the Davis Phinney Foundation for Parkinson’s. 

During one ride, she met Dr. Diane Stephenson, C-Path’s VP of neurology. “We hit it off…I was given an opportunity to share my experience living with Parkinson’s and as a clinical trial participant.” 

Zenner-Dolan has since joined the advisory committee for the FDA’s Peripheral and Central Nervous System leadership team. “My personal mission of living my best today is buoyed by the trust I have in the behind-the-scenes work of the brilliant teams at Critical Path Institute,” she said.

Hailey Davenport, a Tucson native and UA graduate diagnosed with Type 1 diabetes at age 14, was drawn to C-Path because of its important T1D research. She’s now the senior project manager for its T1D Consortium. 

“Being on calls with experts in the field from both academia and industry, people that are actually working on developing therapies, just kind of being in the room is so special, and really gets me excited about the future,” Davenport said.

Rich Brennan, VP of federal affairs for the ALS Association, is working with C-Path to give those diagnosed with ALS a chance to live longer. “The public-private partnership and C-Path’s work, especially in the rare disease community, and their work in the neurology space, has been really important for us…”

“What we’re trying to accomplish is to make research data more shareable or more readily available, reusable, scalable and also, person-centric,” Brennan said. 

20 Years and Onward

Woosley believes that, over these 20 years, what C-Path has truly created is collaborative regulatory science–an entirely new discipline. 

‘Today, scientists from traditionally competitive pharmaceutical companies are working side by side with regulatory and academic researchers to share data and rigorously validate tools for new drug development,” he said. “I never could have imagined the widespread, global recognition and trust C-Path has achieved from both the scientific community and patient advocacy groups alike.”

C-Path’s work “is changing lives,” U.S. Rep Juan Ciscomani, from Arizona’s 6th congressional district, told the team at its 20th celebration this spring. “It’s driving innovation, and it’s strengthening Arizona’s role as a leader in modern drug development.”

U.S. Sen Mark Kelly of Arizona, whose wife former U.S. Rep. Gabrielle Giffords, was the lead congressional sponsor on the bill to enable C-Path’s public-private partnership, also gave his praise. “It’s meant more people have access to life-changing medicines and it’s given hope to those with rare diseases,” he said.

This milestone achieved, Romero looks toward future promise: “It’s been a very successful couple of decades, but we still have so much more work to do because there are still so many more unmet needs in the drug development process. We hope to continue to beat the odds and transform the process.”

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