Critical Path Institute: Paving the Way for Global Drug Innovation

By Mary Minor Davis

The Critical Path Institute, a groundbreaking nonprofit organization that helps expedite medical product development and regulatory review processes, celebrates 15 years in Tucson as its global headquarters. 

Founded by Dr. Raymond Woosley in 2005, C-Path opened its doors in this desert city with just six employees. Today, nearly 100 scientists, pharmaceutical industry leaders and medical professionals staff the institute and its board. Today, the institute’s global presence includes a European headquarters in Dublin, Ireland.

The work of C-Path is renowned, uniting medical stakeholders and their intellectual and financial resources to bring effective drugs and treatments to fruition faster. The institute is truly a jewel in Tucson’s crown, and yet, it almost wasn’t. 

“In our early conversations with the U.S. Food and Drug Administration, there were people trying to take the program to California,” said Woosley, who was dean of the University of Arizona College of Medicine before he founded C-Path. “The people in Washington, D.C., wanted us to move it there, saying it would never survive being so far away. I was really afraid they might be right, but here we are.”

Here, C-Path has thrived.

Under Woosley’s leadership, the institute was a first-of-its-kind venture supported by the FDA, which was incubated within UArizona, to help implement the FDA’s Critical Path Initiative. The initiative’s aim was to tackle the “pipeline problem” – a gap between the number of new biomedical discoveries and the declining number of new medical treatments being submitted for FDA approval. As a neutral, trusted third party since 2005, C-Path facilitates shared knowledge and science for a more streamlined approach.

“C-Path has done that at a scale I never thought would be possible,” Woosley said. “I always believed that the drug industry would come together as long as the FDA was encouraging them. Their participation convinced me that C-Path has far exceeded what I expected. The scope of illnesses that are being addressed is just phenomenal.” 

C-Path’s model involves the formation of creative teams, or consortia, that are created when the institute is approached by regulators, patient groups or industry leaders to solve an unmet need.

There are currently 23 consortia and programs involving stakeholders in academia, industry, regulatory bodies, medical societies, healthcare and patient groups. Each team is led by an executive director and focuses on a specific clinical area such as Parkinson’s disease, pediatrics, neonatal care and drug safety. Members share research, data, ideas, patient experiences and costs to develop the tools to move medical products forward.

Dr. Klaus Romero has been with C-Path since 2007. In his role as chief science officer and former executive director of Clinical Pharmacology and Quantitative Medicine, he has seen the institute’s successful trajectory first-hand.

“It was like going to the moon,” Romero said. “We created something from scratch and built a rocket, and we reached the moon. Today, we can now conquer the entire solar system with all of the work that we are doing to expedite medical product development through the different solutions that we generate.” 

“When we started our (Critical Path to Tuberculosis Drug Regimens) consortium in 2010, the last drug that had been approved for the treatment of tuberculosis was in the 1950s,” he said. Integrating data, enabling discussions and using previous groundwork, the first new TB drug in a half century was approved in 2013. There are now over 20 TB drugs in the pipeline.  

Moving forward, Romero hopes C-Path’s proven expertise and biopharma models can be expanded to other product classes, including medical devices and biologics. 

C-Path’s proficient leadership has been instrumental in its success. C-Path President and CEO Dr. Joseph Scheeren took the helm in 2019 with a resume of more than 35 years in the pharmaceutical industry. He succeeded Martha Brumfield, who with more than 20 years of experience with Pfizer and other regulatory work had expertly guided C-Path’s stable growth since 2013, after Woosley retired. 

Under Scheeren’s leadership, the organization has grown from 68 to 100 employees in his first year, a move he said is powered by global growth and demand. After initiating activities in Europe, C-Path provided the model for the Innovative Medicines Initiative in the European Union and is actively involved in three current projects, creating an international collaboration.

“We’re developing programs for the global community,” he said. “We are trying to develop tools in such a way that medical products that become available in the U.S. can also be approved for patients globally, at an accelerated pace.”

To succeed, Scheeren said several things must be in place: streamlined review processes, a common language to interpret global clinical trials, global data, and other tools that “speak the same terminology” when talking about biomarkers and other quantitative information.

Data sharing has traditionally been a barrier as countries were unwilling to open up their research to others, but Scheeren said, “those walls have come down. We’ve made it clear that these things can be done while preserving data privacy for the patient.”

Managing the complexities of more data moving forward will be crucial to meet the FDA’s Critical Path Initiative, and similar global goals. “We need a solution that balances the need for open science with the push for data protection and data privacy, because those same things are extremely important topics that society is grappling with right now,” Scheeren said.

Other future C-Path programs include developing fellowships and internships with U.S. universities to mentor students on the institute’s role and expanding network. The COVID-19 pandemic has also made the public more aware of the need for science, as well as the necessary phases to gather information and make key conclusions. C-Path leadership hopes this awareness will continue.

“Here’s how science works,” Scheeren said. “It works by making mistakes, as well as making discoveries. Each builds on the other for successful outcomes.”