CriticalPathInstitute2025

SPECIAL REPORT 2025

THE REGION’S BUSINESS MAGAZINE

CRITICAL PATH INSTITUTE MOVING DRUGS TO MARKET SMARTER, FASTER FOR 20 YEARS

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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.

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2005

C-Path begins with six employees, establishing itself as a key player in accelerating drug development. FDA partners with C-Path to realize FDA’s Critical Path Initiative mission.

2006

C-Path spearheads the formation of the Predictive Safety Testing Consortium, an unprecedented collaboration between C-Path, industry, and FDA.

2007

Founder and CEO Dr. Ray Woosley testifies before Congress. President George W. Bush signs Critical Path Public-Private Partnerships bill.

C-Path wins Arizona Innovation Award.

2008

C-Path global efforts lead to the first-ever FDA and EMA qualification of kidney safety biomarkers. C-Path ranked No.7 on Reader’s Digest’s “Fix Healthcare Now!” list.

2009

C-Path launches its Coalition Against Major Diseases (which evolved into C-Path’s Alzheimer’s and Parkinson’s-focused consortia), as well as its Patient-Reported Outcomes Consortium. These partnerships elevate the voices of those with living experiences and further accelerate drug development.

2010

PMDA reaches its first-ever biomarker qualification decision for kidney safety. Nature Biotechnology features C-Path’s evaluation of seven kidney biomarkers. Consortia focused on tuberculosis and polycystic kidney disease are formed.

2011

C-Path launches the Electronic Patient-Reported Outcomes Consortium, collaborates with Europe’s Innovative Medicines Initiative, develops first-ever therapeutic area data standards for Alzheimer’s in partnership with CDISC, and is named in NIMH’s “Top 10 Research Advances of 2011.”

2012

EMA issues first-ever qualification for brain imaging biomarker to aid early Alzheimer’s trials.

CDISC and C-Path launch the Coalition for Accelerating Standards and Therapies. FDA starts the Fit-for-Purpose Initiative, to review and endorse computerized drug development tools.

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concepts,” said Kirkpatrick. “The first was to do something that would speed up drug development in the precompetitive 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. www.BizTucson.com

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, CPath’s VP of neurology. “We hit it off...I was given an opcontinued on page 72 >>>

2013

C-Path and IMI hold first joint meeting “Collaborating for Cures.” FDA issues its first-ever FFP endorsement for C-Path’s Alzheimer’s disease clinical trial simulator. EMA issues first-ever qualification of a quantitative drug development tool for this same simulator. The tool is featured in the Wall Street Journal.

2014

FDA issues first-ever biomarker Letter of Support for C-Path’s kidney biomarkers and receives $1 million Flinn Foundation grant. C-Path receives $11.8 million grant from the Bill & Melinda Gates Foundation to develop computerized tools that can accelerate drug development for tuberculosis.

2015

Happy 10 years, C-Path! New consortia launched for neonatal drug development, Duchenne muscular dystrophy, and Parkinson’s disease. C-Path secures EMA and FDA support for multiple kidney biomarkers. C-Path is selected to host TB clinical trials data.

2016

C-Path co-develops biomarker qualification framework, launches further efforts in tuberculosis, releases multiple sclerosis trial database, gains EMA support for novel Parkinson’s biomarker, and receives three new FDA grants.

2017

C-Path makes a unique tuberculosis pathogen genomic sequencing database available globally, and receives a $1.1 million grant along with Arizonabased TGen for activities in Type 1 diabetes. T1D and solid organ transplantation consortia are launched, EMA gives support for liver biomarker and PRO Consortium achieves FDA endorsement of PRO instrument for major depression.

2018

Huntington’s disease consortium launches. C-Path receives first-ever qualification of composite safety kidney measure based on six biomarkers. University of Arizona and C-Path launch regulatory science graduate certificate.

2019

C-Path opens European headquarters in Dublin, launches the Friedreich’s Ataxia database with FARA and begins a biomarker project with Japan’s PMDA. A global transformative effort for data integration for rare and orphan conditions launches: The Rare Disease Cures Accelerator-Data and Analytics Platform initiative.

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2020

C-Path joins ERA4TB, and leads pediatric device efforts. FDA issues contracts to support outcomes work in asthma, and model-informed drug development in neuroscience. Through the Cure Drug Repurposing Collaboratory, collaborations on COVID-19 launch.

2021

C-Path’s symptom diary for irritable bowel syndrome supports pivotal FDA-labeling claim evolution for symptomatic drug. Consortia on hereditary ataxias and acute kidney injury launch. Duchenne database opens, and C-Path joins UNITE4TB.

2022

Rare Disease COA Consortium and Translational Therapeutics Accelerator launch. C-Path is selected to support the Act for ALS collaboration with FDA and NIH, launching Critical Path for Rare Neurodegenerative Diseases. C-Path relocates European headquarters to Amsterdam. EMA supports T1D biomarker initiative and TTC receives EMA endorsement for novel biomarker panel for kidney transplant trials.

2023

C-Path receives EMA endorsement for its Duchenne muscular dystrophy clinical trial simulation platform. FDA encourages and supports PSTC’s four pancreatic safety biomarkers. New consortia in lysosomal diseases and alpha-1 antitrypsin deficiency launch with FDA support.

2024

C-Path hosts inaugural Global Impact Conference, receives $1.5M Niemann-Pick grant, unveils effort in imaging method, makes Duchenne trial simulator available, and receives landmark funding to convene the Global Evidence in Medicines for Parkinson’s Disease initiative.

2025

C-Path’s 20th Anniversary Celebration: A Global Milestone! C-Path marks 20 years of innovation, collaboration, and impact with multiple unforgettable events spanning the U.S. and Europe.

portunity 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-thescenes 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 CPath 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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From left –

Dr. Klaus Romero CEO

Kristen Swingle President & COO

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Desert Duo

Critical Path Institute’s Dynamic Leadership Team

PHOTO: BRENT G. MATHIS

By Tara Kirkpatrick

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Critical Path Institute, a groundbreaking nonprofit driving better and faster drug development across the world, is led by a respected pair who are the ultimate tag team for science and structure.

Dr. Klaus Romero, CEO, and Kristen Swingle, president and COO, are truly the glue behind a global public-private partnership that, from its humble Tucson headquarters, has helped accelerate progress in new drugs and treatments for patients for 20 years.

“We have tremendous talent here and a wonderful structure,” said Romero, who served as C-Path’s Chief Scientific Officer before being named CEO in 2024. He has been with the organization for 18 of its 20 years.

“Anytime anything important happens, whether it’s day-to-day operations or something that develops for us anywhere on the planet, we stay connected as close to real-time as possible,” he said.

This C-Path leadership team achieves results– for patients, for industry, for regulatory agencies and for stakeholders worldwide.

“Klaus and Kristen are a truly exceptional team,” said C-Path founder Dr. Raymond Woosley. “They bring a rare blend of deep regulatory and scientific expertise with the interpersonal skills to lead large, complex teams across different stakeholder groups. They don’t just manage, they inspire.”

Whereas Romero sets the vision and passion for C-Path, Swingle perfects the process, driving the daily infrastructure for C-Path researchers and staff. They

are the perfect “partners in crime,” as they like to say.

“We need a CEO who’s driving ideas and has the strategic mindset,” said Swingle. “Klaus is phenomenal at that.

When he’s traveling to D.C., I’m dealing with matters here and making sure we’ve got that continuity. It’s also bouncing ideas off each other. I’m focused on the operational standpoint, making sure everyone has everything they need.”

Said Romero, “She sets a realistic expectation. I can get really excited about something around cutting-edge science or clinical development, but I need somebody to give me a reality check of what is realistically achievable.”

A prominent physician and clinical scientist from Colombia, Romero was presenting at a scientific meeting in 2007 when he had the chance to hear Woosley speak. Impressed with the nonprofit’s work in just its first two years, they immediately hit it off, so much so, that Woosley asked Romero to come work with C-Path.

“By December 2007, Olga Lucía and I were moving to Tucson and that was it,” recalled Romero. “I am indebted to Ray, for what he taught me, what he gave me personally and professionally. I will never be able to repay him.” Romero would go on to serve as executive director of both clinical pharmacology and the quantitative medicine program at C-Path before advancing to CSO and CEO. Romero is also a talented guitarist, often referring to himself as a “happy mucontinued on page 76 >>> Summer 2025 > > > BizTucson 75

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“

We have tremendous talent here and a wonderful structure.

Anytime anything important happens, whether it’s day to day operations or something that develops for us anywhere on the planet, we stay connected as close to real-time as possible.

– Dr. Klaus Romero, CEO, Critical Path Institute

continued from page 75 sician who happens to be a clinical scientist.” He plays bass, drums, keyboard, and relishes the ties between music and analytic thinking.

“There’s a number of components of the connection between music and my day-to-day and how I approach leadership,” he explained. “For any band to be successful, everyone has a role to play.”

Swingle, a Tucson native, joined CPath in 2019 with two decades of medical and molecular science experience, most recently as VP of stem cell operations for Cord Blood Registry.

“I had the benefit of joining the or-

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ganization at a time when a lot of hard work had already been put in and they had really built such a foundation for the successes we have had,” she said. Yet, Swingle has absolutely left her mark, helping to navigate C-Path through the COVID-19 pandemic as well as becoming a true bioscience ambassador in the region. She was chairwoman of the Arizona Bioindustry Association from 2018 to 2023 and is a national advisory board member of the R. Ken Coit College of Pharmacy at the University of Arizona.

Romero recalled her job interview: “I said, ‘Don’t wait, hire her!”

Both Romero and Swingle herald the opportunity to head a global, game-

”

changing organization from Southern Arizona.

“If you look at the big biopharma areas, each one of them has its own identity...the Bay Area, Boston, etc.

But what is the identity of Tucson? It’s growing as a hub for biopharma with a growing collaboration identity. Selfishly, I think we’ve contributed to that,” said Romero.

Added Swingle, “I’m a by-product of this community. There’s an element of Tucson in particular that is somewhat intangible. The people here are so invested in making Tucson better. We are literally making the world better, and we are right here doing it.”

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A Laser Focus on Alzheimer’s & Parkinson’s

By Tara Kirkpatrick Critical Path Institute continues to help drive needed therapies for neurodegenerative diseases, especially Alzheimer’s disease and Parkinson’s disease.

C-Path’s Critical Path for Alzheimer’s Disease consortium, which started in 2008, has contributed to some definite wins in accelerating drug development for this condition, over the past 20 years.

These include the first-ever computerized tool to transform clinical trial design in Alzheimer’s disease that is endorsed by the Food and Drug Administration and the European Medicines Agency. Also, the regulatory endorsement of viable drug development biomarkers in Alzheimer’s disease and Parkinson’s disease has contributed to a new era of biomarker-informed clinical trials.

“Thanks to our talented team and the fact that we had all the support from the FDA and EMA, we have worked with key opinion leaders in the field, and we were able to look at data in new ways,” said Diane Stephenson, C-Path’s VP of neurology. “So, we brought forward these modeling tools and biomarkers at a time when, again, people were uncertain as to how to link the right drug to the right patient at the right time. It’s really game-changing.”

The latest optimism centers around a protein linked to Alzheimer’s deterioration, called “tau.”. To advance the momentum of using tau imaging in clinical trials, the CPAD consortium gathered more than 50 global experts together to develop a groundbreaking standard for analyzing tau in clinical trials for earlier stages of Alzheimer’s–what C-Path calls a harmonization approach.

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The Critical Path for Parkinson’s consortium, started in 2015, has achieved numerous regulatory successes for establishing biomarkers and accepted clinical trial tools.

It’s now delving deeper into sex differences in Parkinson’s pathophysiology and disease progression that can impact drug development. Long considered “an old man disease,” new Parkinson’s cases are increasingly showing up in women.

“

The frequency of younger people of both sexes with Parkinson’s is growing, to even the childbearing years.

”

– Dr. Diane Stephenson

VP of Neurology Critical Path Institute

“The frequency of younger people of both sexes with Parkinson’s is growing, to even the childbearing years,” Stephenson said. “We don’t know why.

It’s probably both genetics and environment, but it really raises the urgent need for new approaches to detection and treatments.”

“And when you go and talk to women with Parkinson’s, they actually experience the disease very different,” she said. “They get diagnosed way later.

They have different responses to the currently approved medications.”

This innovative initiative – called Global Evidence in Medicine for Parkison’s Disease, or GEM-PD, was recently launched in Vienna and already, many leading experts want to be involved and offer data, Stephenson said. “What’s so incredible about this whole field...everything we do is going to make a dent.”

“We are excited about this whole project because we’re sitting on a lot of data,” Stephenson said. “We have data from 15,000 patients, and no one’s probably ever, ever looked at sex differences.”

Stephenson has a personal stake in both diseases, having a mother who died from Alzheimer’s and a brother from Parkinson’s. “To watch the two people you love...suffer from the same diseases you’re working on, it was the most hopeless feeling.”

But continuing her C-Path work in neurodegeneration gives her hope for future patients. “I feel really encouraged...the science is advancing at a pace that I thought probably wouldn’t happen in my career.”

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Decoding Rare & Orphan Diseases

By Tara Kirkpatrick Rare and orphan diseases–with the dual distinction of being both uncommon and incredibly challenging–have found a true champion in the Critical Path Institute.

Rare diseases affect fewer than 65 in 100,000 people, as labeled by the World Health Organization. The FDA and National Institutes of Health define rare diseases as those affecting fewer than 200,000 people in the country.

Yet, with more than 7,000 such diseases identified globally–often among children–they impact roughly 300 million people.

By bringing regulators, pharmaceutical companies and patients’ organizations to the same table and sharing valuable data, C-Path is creating a trusted baseline from which better studies can be designed, new patterns can be analyzed and new therapies can be developed.

“Rare disease studies can be challenging given the small numbers of patients and the limited understanding of the disease,” explained Collin Hovinga, VP of Rare/Orphan and Pediatric Disease programs at C-Path. “We bring stakeholders together, sponsors, persons with lived experience, clinicians and researchers to build solutions that generate impact.”

A LOOK AT SOME OF THE PROGRESS: Polycystic Kidney Disease One of C-Path’s major victories was helping to fuel the first-ever biomarker, followed by the first-ever drug to slow polycystic kidney disease. The genetic disorder causes clusters of cysts to grow 80 BizTucson < < < Summer 2025

in the body, mainly on the kidneys, damaging them over time.

C-Path’s Polycystic Kidney Disease Outcomes Consortium achieved a milestone when it secured the nod of the FDA and EMA for height-adjusted total kidney volume, or htTKV, as a biomarker, said Hovinga. “Building on that biomarker foundation, tolvaptan became the first approved therapy to slow...progression.”

“

Rare disease studies can be challenging given the small numbers of patients and the limited understanding of the disease

”

– Collin Hovinga VP of Rare/Orphan & Pediatric Disease programs Critical Path Institute

The consortium is focused on additional PKD biomarkers and advancing a clinical trial simulator to design better trials for future therapies.

Duchenne Muscular Dystrophy A complex disease caused by a genetic mutation, Duchenne muscular dystrophy robs patients, mostly boys, of the ability to walk and hits their hearts and lungs.

C-Path’s Duchenne Regulatory Science Consortium has developed an extensive database of information from DMD studies that helped produce the very first computational model for DMD clinical trial simulations. This sophisticated tool predicts results so a new therapy trial can be faster and less expensive.

Before this, designing a DMD trial was difficult because of the disease’s incredible complexity. The initiative has received a letter of support from the European Medicines Agency.

The DMD consortium’s work has also led to advancing glutamate dehydrogenase as a safety biomarker in trials of patients with muscle disorders.

Friedreich’s Ataxia With the largest integrated data and analytics platform for rare diseases, CPath helped foster the first-ever treatment for Friedreich’s ataxia, an inherited disorder that damages the spinal cord, part of the brain and the heart.

The new drug, Skyclarys from Reata Pharmaceuticals, was approved in 2023 to slow the disease progression and improve neurological function.

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Leveraging AI, Tech for Future Drugs Hailey Davenport

Preventing, Delaying Type 1 Diabetes

By Tara Kirkpatrick Type 1 diabetes, despite several advances, is still a momentous challenge for patients and their families. What’s more, the number of people diagnosed with T1D in the U.S. alone is projected to triple by 2050.

Critical Path Institute, which formed the T1D Consortium in 2017, is working to accelerate the process to new therapies to both prevent and delay this disease, with a keen eye toward finding it before the life-altering symptoms begin.

As a disease, T1D starts well before the symptoms emerge, so the ability to screen for risk beforehand is an incredible opportunity to impact the disease. Where that work starts is by identifying biomarkers to better select patients in the disease’s early stages for future clinical trials, as well as, gaining regulatory endorsement for clinical simulation tools to help design better trials.

“If we can measure these actionable markers of the body’s immune system attacking the pancreas, then certain therapies could delay that, or even prevent the autoimmune process from starting in the first place,” said Hailey Davenport, senior project manager for the T1D consortium, who lives with T1D herself.

One such tool the C-Path consortium has advanced is based on a biomarker called C-peptide. Measuring C-peptide levels in the blood over time can become an important biomarker to better track disease progression. This revolutionary tool was developed from analyzing data from 20 randomized clinical trials that included 781 people.

“Moving forward, we are looking at... incorporating more tools that would be applicable to both new onset and prevention,” added Davenport. “There are so many unmet needs, even with all the incredible advancements that we’ve had since I’ve been diagnosed.”

Because of this work, “future generations will have it so much better than we have it now,” added Davenport. “That’s exciting to be on the forefront of something really new and exciting that could be tangible in the next 10 years or so.”

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Looking ahead, Critical Path Institute CEO Dr. Klaus Romero sees the drug development industry at an inflection point.

“We’re needing to develop therapies that are leveraging technologies...that are at the true cutting edge of the intersection of science and people’s needs,” he said. “Gene-based therapies, cell-based therapies, anti-sense oligonucleotides–those types of products have the potential to be really transformative, but there are also innovative small molecules that can have major impact too.”

Yet, those possibilities reinforce the need to make sure potential risks and harms are equally as understood as the potential benefits, Romero said. C-Path’s core competencies are well-positioned to provide that analysis.

Take artificial intelligence, or AI, which C-Path has actually been utilizing since 2016.

“We recognized then that this was something that could have a transformative power and we started working on building the first foundation for us to start integrating artificial intelligence into the development of our drug development solutions,” he said. “Now, we have an advanced approach that keeps evolving.”

Moving forward, Romero noted AI’s vast potential in drug development will require proper checks, which C-Path, as a trusted neutral party, can offer. “More specifically, how can we be sure that a given AI solution is actually doing what it’s supposed to do to inform a drug development decision.”

The increased usage of digital health technologies, such as smart watches and phones, and other wearable sensors, will help elevate patient data, as long as they consistently capture meaningful information. The algorithms that process such data also must be reliable. The FDA and EMA have already issued guidance on their usage in clinical trials.

For example, C-Path VP of Neurology Dr. Diane Stephenson noted these tools’ utility for Parkinson’s patients to track their experience. “Their experience with the disease changes, not only each day, but within the day. It’s highly variable. So, if you expect that a doctor visit every six months or a year is going to be able to accurately predict a patient experience? How would you know if a drug worked?”

“This is just the beginning of those devices,” Stephenson said. “I can only imagine they’re going to get more and more sophisticated.”

Added Romero, “As we advance in those areas that will have a bearing in unlocking the potential of all drugs to be used for new kinds of indications, that’s where I see the future in advancing the science.”

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PHOTO: BRENT G. MATHIS

By Tara Kirkpatrick

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