By Dan Sorenson
Tucson’s Critical Path Institute, better known as C-Path, is emerging as a major player in a movement to improve and hasten the drug approval process – not just in the United States but worldwide.
At the head of that effort is C-Path’s new CEO and President Martha Brumfield, a veteran of the pharmaceutical industry.
Brumfield said C-Path is already deeply involved in providing the tools to develop new pharmaceuticals that hold promise for Alzheimer’s disease, tuberculosis, polycystic kidney disease, multiple sclerosis and Parkinson’s. And she’s optimistic that there is no limit to the diseases to which its game-changing system for research and development could be applied.
Yet success has brought inquiries about projects that are beyond the capacity of C-Path’s present 34-member staff. Brumfield said she’d like to expand the staff and its capabilities.
Don’t look for Brumfield and C-Path to be credited with creating drugs that will treat or cure some of today’s most-feared diseases.
To get society the medical products it needs, the organization’s strategy is to harness the power of “Big Data” and modern technology – by sharing the research of competing pharmaceutical companies and combining that with the eternally sought goal of streamlining bureaucracy.
Brumfield said C-Path’s scientists, technologists and administrators want to overcome the obstacles to scientific solutions – to improve and get out of the way of good science.
C-Path founder Dr. Raymond L. Woosley noted it was typically taking 15 years to develop major “important drugs” – with a 95-percent failure rate for getting to market. Woosley said he would like to see C-Path shorten that path to three years with a 95 percent success rate. He cited HIV/AIDS research as proof that such a massive change can take place. “It was done for HIV/AIDS and it can be done again,” he said.
The pace of scientific advances in the lab has skyrocketed. In the previous decade new drug approvals were declining – but this improved in recent years. Last year turned out to be a record setter for new drug approvals in the in the United States. The U.S. Food and Drug Administration blessed 39 new molecular entities in 2012 – the most in 16 years
Part of the problem is the risk of late-stage drug trials, which can cost hundreds of millions of dollars. If drug companies don’t see a good chance of a payoff, they won’t spend money on getting through those final stages of the approval process.
“The drug development process is a long process,” Brumfield said. C-Path’s efforts to break the log jam won’t happen overnight – but we are starting to have an impact.
“I wish you could measure it in months, but it’s usually measured in years,” Brumfield said of the industry’s pace. But she said the thinking behind the founding of C-Path was based on knowing that “science in a company’s lab is developing much faster than it is out in the world.”
Sharing data requires trust
It could work even faster and more effectively if the research of a number of companies were to be combined. However, the drug industry is one of the most competitive sectors in the world – and for good reason. The financial risks are huge. While the payoff for a drug that can save millions of lives worldwide is potentially huge, so are the costs racked up during the development stages and the government approval processes the drug must pass through before the profits can be rung up. No government approval, no payback and no profits.
Getting pharmaceutical companies to share information would have been unthinkable just a few years ago – and it still requires a lot of trust, Brumfield said. Credibility is everything.
Yet, she said C-Path has already made inroads with both the private pharmaceutical industry and the government agencies that regulate them and their products – thanks to a precedent-setting C-Path project.
In 2010, C-Path scientists and technologists, working with member companies, combined data from multiple Alzheimer’s clinical trials to create an integrated, standardized database. This database was used to develop a unique clinical trial simulation tool for Alzheimer’s disease now endorsed by the FDA and European Medicines Agency. This tool is now available to qualified researchers doing work in Alzheimer’s – free of charge, Brumfield said.
This data integration is not easy, considering the competitive and confidential nature of the industry. And it’s even more difficult when you add the problem of melding massive piles of proprietary data.
“Everybody talks about the Big Data concept of going out and data mining,” Brumfield said. “There are areas where it has applicability – but clearly when we’re talking about controlled clinical trials in the setting of a regulated industry such as that we work with, the data has to be formatted so that you can compare and look across multiple trials.
“That’s a huge effort, but we did that successfully and we’re doing that with some other disease areas as well. Because of all that, it’s clear to me that the companies that we work with have a lot of trust of our capability in this space.
“The benefit realized when you aggregate tens of thousands or hundreds of thousands of data points across 10 companies becomes very powerful,” Brumfield said.
She said that C-Path’s work with the Alzheimer’s disease database generated further inquiries about collaborative efforts.
Getting governments to streamline and coordinate their drug approval processes is another major part of the C-Path formula for breaking the new product log jam – and one for which C-Path founder Woosley said Brumfield is well qualified.
“Martha Brumfield is ideally qualified to lead C-Path,” Woosley said. “She has over 20 years of experience at Pfizer, one of our nation’s most successful pharmaceutical companies – and she fully understands the challenges facing drug developers and regulators. She brings an additional sphere of expertise that is essential for C-Path’s future success – and that is her experience with international regulatory agencies in Europe and Asia. Since drug development is a global enterprise, her experience and expertise in global regulatory affairs will be invaluable.”
Among the streamlining efforts are coordinating the submission and approval processes in the U.S. and European Union. Brumfield said the submission documents have already been synchronized.
Beyond the already strong ties between the U.S. FDA and EU’s regulatory body, Brumfield said China is an undeniably massive – and massively different – market for the industry. In addition to her experience in the Far East while with Pfizer, Brumfield recently visited China on behalf of C-Path.
That relationship may have implications for Tucson. Brumfield took two visiting Chinese regulators to meet with the University of Arizona College of Pharmacy and C-Path to discuss potential collaboration on projects such as biomarker and standards development.
“China is definitely a horse of a different color,” Brumfield said. “But there’s interest in what we’re doing. I believe there is the potential for some work and some funding in China. This (latest visit) is a first step in the water.
“They are going to be a player in the future. If we could bring some projects where the Tucson area could collaborate with China, I think that could be great for our community. There’s a lot of money to be spent in China. If we could tap into it in a way that adds value to what we’re doing with the biotech community here in Southern Arizona, I think that would be phenomenal.”
What lies ahead
As an administrator Brumfield’s work will also involve growing the organization and its funding. The original federal grant that established C-Path expires next year. And Brumfield said getting funding from the industry is a delicate situation – because maintaining C-Path’s independence and credibility is paramount.
“Originally we were funded by the FDA, which was able to give us a five-year grant of $8.5 million dollars,” Brumfield said. “That grant expires August of 2014. Science Foundation Arizona was able and willing to match that grant. Were it not for Science Foundation Arizona we would not be here today. They have contributed more than $13 million over the life cycle of C-Path.
“In addition we’ve raised about $9.8 million through philanthropy in the Tucson area. That was the genesis. Were it not for the FDA, Science Foundation Arizona and the local community we wouldn’t have gotten started. Since then we now have brought in some grants from the Bill & Melinda Gates Foundation, National Multiple Sclerosis Society and Polycystic Kidney Disease Foundation.”
In response to the end of the FDA and Science Foundation Arizona grants, C-Path has already entered a new phase – where its funding is coming in part from industry.
“Our model now is that we have a fee structure. So when a group of companies and academia come together and propose they want to accomplish X – whether it’s a new biomarker or data standard or modeling tool – we put a price tag around that and each of the members pays a certain fee. That money goes into a protected bank account where it’s spent to support the staffing needs, the technology needs, the data-basing needs etc. to accomplish that goal,” Brumfield said.
She said this fee structure was put in place “to allow us to continue to do the work that we’re doing. The companies have been fairly accepting of that. They see value in the money that they’re putting into these consortia – because they know what they’ll get out of it at the other end.”
Defining Paths to Accelerate Drug Development Process
C-Path works with academic scientists, industry scientists and regulatory scientists in the pharmaceutical industry to collaborate, innovate and accelerate the process of getting newer, safer drugs from the lab to the marketplace.
According to C-Path CEO and President Martha Brumfield, C-Path improves efficiency of the development of drugs and other medical products by identifying pathways to convey new scientific advances through existing regulatory processes.
As a trusted and neutral third party, C-Path works closely with the U.S. Food and Drug Administration, European Medicines Agency and other regulatory agencies to define the pathway to reach an appropriate level of regulatory input specific to the new science.
When formal regulatory decisions are necessary (such as qualification or fitness-for-purpose determinations), C-Path gathers the necessary evidence – which requires developing data, measurement and methods standards, aggregating data, as well as preparing regulatory documentation of the evidence.