FDA has an ambitious plan to develop efficacy measurement standards for clinical trials in 55 diseases and therapeutic areas in the next five years – and it’s looking to the Critical Path Institute, the Clinical Data Interchange Standards Consortium and a host of other players to make its wish list a reality.

The “55n5” plan made its public debut at a two-day conference sponsored by the agency, C-Path and CDISC Nov. 30-Dec. 1. The benefits could be substantial, Raymond Woosley, founding president, CEO and chairman of C-Path, said. In October, C-Path and CDISC unveiled a new Alzheimer’s disease Therapeutic Area Standard (SDTM AD/Mild Cognitive Impairment User Guide).

“The data you saw on Alzheimer’s cost $350 million to generate, and it only generated a conclusion that the drugs didn’t work. Now that we’ve got it available, it is generating a tool that new drugs can be measured by.”

Enrique Aviles, director of data standards and management at C-Path, noted that development of this standard drew on legacy data from 15 clinical trials that enrolled 5,000 patients.

“The data you saw on Alzheimer’s cost $350 million to generate, and it only generated a conclusion that the drugs didn’t work,” Woosley said. “Now that we’ve got it available, it is generating a tool that new drugs can be measured by. We need to do that in every therapeutic area.”

Nine Months Of Gestation Per Therapeutic Area

Once the 55n5 project is fully funded, the goal is to develop a set of standards for each therapeutic area in eight to 10 months. “We have to revamp the way we develop standards because the Alzheimer’s one is taking two years and is really relying on volunteers to do the efforts and we want to take nine months,” Rebecca Kush, CEO of CDISC, noted. “So the only way to do that is to focus the efforts and get some strong governance and project management to make it happen.”

Alzheimer’s is one of nine therapeutic areas where standards development is already under way, from FDA’s list of 55.

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The others, according to Kush, are tuberculosis and acute coronary syndromes, which CDISC is doing in collaboration with Duke University, the American College of Cardiology and the TB Alliance; Parkinson’s disease, in collaboration with C-Path and NIH’s National Institute of Neurological Disorders and Stroke; pain, in collaboration with FDA and the University of Rochester Also see "Patient Selection For Analgesic Trials To Be Improved Under Public-Private ACTION Initiative" - Pink Sheet, 13 December, 2010.; virology (hepatitis B and C and HIV); polycystic kidney disease, in collaboration with Tufts University; oncology, in collaboration with the National Cancer Institute; and diabetes.

As to how the standards development will actually work, Kush cited the example of CDISC’s Clinical Data Acquisition Standards Harmonization project, which consisted of 18 “domains.” “We had 17 collaborative groups. We got funding from about five of them, and we brought in a project manager and she oversaw seven teams and had those reporting in to her and coordinate the whole thing,” she said.

“It took two years for that. That was just a touch smaller scope than this [55n5 project], and not as much science … we had to analyze what are the core data sets, what are the essential elements? And we had to do that globally. … It takes coordination, and it takes people who know what we’ve already done so we don’t duplicate efforts.”

Funding Needed, Ideas Welcomed

The 55n5 project is the first collaborative effort of the Coalition For Acceleration of Standards and Therapies (C-FAST), a partnership between CDISC and C-Path that, like the 55n5 project itself, is just getting off the ground, Kush said. Discussions began in June. If the organizations succeed in raising “a good portion of the targeted funding of $35 million or so” for the project (though the range could be anywhere from $30 million to $50 million), the five-year clock could start ticking sometime in the first half of 2012, she said.

If less is raised, the project will take more than five years to complete, Kush said. “The clock didn’t start ticking [yet] because there’s no way we can achieve 55n5 without resources. We’ve already started nine of these therapeutic standards packages because we want to do them. But if we don’t get appropriate resources, it’s going to be ‘55n15’ or something, it’s not going to be in five. We have to be able to hire more staff to oversee the volunteers, you can’t do rapid development with only volunteers. They have day jobs.”

Kush and Rick Myers, chief operating officer of the Critical Path Institute, said the groups hope to raise the needed funding from philanthropic organizations, government, foundations and other interested organizations. “We’d like to think as we start this that a lot of the disease areas will say, this is wonderful to have this, how do we get our diseases done first?” Myers said.

“That is going to be a very expensive undertaking,” Woosley noted. “I think we’ve heard a commitment from pharmaceutical companies that they want to contribute data to set the standards for those disease areas.”

C-Path and CDISC are open to suggested additions to the list of 55 therapeutic areas, Kush and Woosley said. FDA “developed this list, posted it on their website, and made us aware of it,” Kush said. “And they said, ‘Of course you may have other input or other people may want different things, but this is our wish list.’ … It’s basically a list of areas where they want standards for efficacy measurements. They went to their different centers and found out what they are looking at in terms of regulatory submissions in the various therapeutic areas.”

FDA developed the list “based on submissions likely to come in,” Woosley explained, adding that there may be one major area yet to be heard from: “There’s a lot of work being done by small bio companies. And we’re working with Jim Greenwood [CEO of the Biotechnology Industry Organization] to get the word out to them that they need to be part of this. They need to be using these biomarkers and using common data elements from Day One.”

The alliance hasn’t actually started fundraising yet, Kush said. “We got a grant from FDA to do the virology TA standards; we got a little piece of the grant that was given to Rochester to do the pain one; we’ve got a little money from the Polycystic Kidney Disease Foundation to do that one; we’ve got money from the TB Alliance/Gates Foundation to do that one. We’re getting support in dribs and drabs, so it’s taking a while.”

Efficacy Standards For 55 Diseases In 5 Years: FDA, Critical Path And CDISC’s Ambitious Plan

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