The Coalition Against Major Diseases will move to creating a database of Parkinson's disease trials after unveiling a similar product for Alzheimer's disease.

The data will come from various pharmaceutical companies, who have agreed to pool control trial data in an effort to give drug development in the space a better chance of succeeding. By combining the control data, researchers at the coalition companies can design better and more targeted clinical trials to test their new drugs.

Scientists are hoping patterns will emerge once the Parkinson's data, and later Huntington's disease data, are viewed alongside Alzheimer's data. Raymond Woosley, president and CEO of the Critical Path Institute, which manages CAMD, said he is hoping more can be understood about neurodegenerative diseases in general.

Woosley said the three diseases may turn out to be symptoms of neurodegeneration that appear in different parts of the brain.

"Once we get it together there may be overlap because some people with Alzheimer's have movement disorder and (some) people with movement disorder have dementia, or they have elements of dementia," Woosley said in an interview.

Smaller Trials Possible

Companies contributed 11 control trials to the Alzheimer's database, announced June 11, which includes information from more than 4,000 patients. More data is expected to be added as more companies join the coalition, and Woosley said members hope they can gain a better understanding of the disease by looking at a larger patient pool.

The neurodegenerative diseases have been the coalition's focus since it was formed last year (Also see "Biomarkers For Alzheimer's, Parkinson's Disease To Be Developed By Industry Coalition Through First-Of-A-Kind Clinical Data Exchange" - Pink Sheet, 18 January, 2010.).

The data is expected to help researchers simulate clinical trials. Scientists also are hoping to break the disease down into subgroups and find biomarkers that can predict disease progression. Woosley said that information can lower the required sample size for a clinical trial and increase the chances of success.

"The more variability you've got, the longer you're going to have to run it and the larger the trial's going to be," he said. "So if you can identify a subset that has a high trajectory for the disease progression and a small variability, then you have a chance to develop trials that are more likely to succeed, be shorter, and more informative."

The database represents the first milestone for Critical Path Institute, which received $1.5 million per year for five years from FDA for the project.

The agency initially funded its Critical Path initiative, which is intended to make the regulatory process more efficient and effective, in 2007. It spent $16 million on projects in fiscal year 2009 and is expected to spend $18 million this year. A $4.45 million increase has been requested in the fiscal 2011 budget (Also see "Regulatory Science: FDA Gets Help From NIH, But Is It Enough? " - Pink Sheet, 1 March, 2010.).

Database Furthers FDA Transparency Initiative

Pharmaceutical companies approached the Critical Path Institute about the Alzheimer's database idea, Woosley said. There was a general feeling the companies were running into a "stone wall" and no one had the ability to succeed on their own, he said.

"This neutral ground allows those scientists from industry and the agency to share their knowledge," Woosley said during the announcement. "The silos that have been existing in the past have to be there because of the regulatory process, but they don't have to be there for the pre-competitive or pre-commercial science."

The coalition agreements are designed so companies do not have to share their "silver bullets," Woosley said. It also allows for companies to share data from trials that failed, he said.

In a way, the move follows some ideas that FDA has proposed for itself in its transparency initiative. Some have pleaded with the agency to release more than summary data about clinical trials to further ensure drug safety.

While testifying before a house subcommittee in April, Yale University School of Medicine Professor Harlan Krumholz called for all clinical trial data to be made public following drug approval and asked for independent reviews of it (Also see "Avandia Aftershocks: Will FDA Drug Safety Review Structures Crumble?" - Pink Sheet, 3 May, 2010.).

FDA Principal Deputy Commissioner Joshua Sharfstein said during the announcement the database allows the agency to develop better approval standards and companies to build onto existing information instead of working in isolation.

"I know from having worked on the transparency initiative that it is a major goal of the scientists at FDA to encourage this kind of collaboration so that what we see in individual studies other people can see and use that to really advance the science," Sharfstein said.

Frank Casty, vice president of technical evaluations for AstraZeneca, said the company has recognized that innovation can no longer happen in one laboratory and that this coalition could be a new research model. He said the data is worth more combined than sitting on individual companies' shelves.

"For a few million dollar investment, which is not an awful lot of money for a clinical trial, we just created a database that's probably worth tens of millions of dollars," Casty said. "So the return on this investment is huge, because now's it's also accessible to all the people, all the individual researchers, whether you're part of this organization or not."

Cancer, Polycystic Kidney Diseases Databases Coming

In addition to the neurodegenerative diseases databases, Critical Path Institute is looking to create similar products for cancer and polycystic kidney diseases. In the case of polycystic kidney disease, Woosley said there have been very few trials conducted, meaning there is little data to pool, but he said the institute is working to gather data from studies sponsored by the National Institutes of Health.

"We think that has the chance of stimulating pharmaceutical interest," he said. "You can look at the database and see the disease and have greater confidence in its course..., but the other part of it is we want to use those models to qualify new testing methods."

Critical Path is taking steps to apply to FDA to qualify biomarkers based on the Alzheimer's database. Woosley said a research plan for qualification has been generated and he estimated the application could be approved in six to nine months.

Former FDA Commissioner Mark McClellan, who now directs the Engelberg Center for Health Care Reform at the Brookings Institution, said including FDA scientists, who have large amounts of experience handling hundreds of studies from multiple companies, in the coalition also is important.

"I think you can give those experts at the FDA a better platform to use to provide some of their unique insights about drug development and about development science and regulatory science that just isn't possible on a one-off basis," McClellan said.

-Derrick Gingery (d.gingery@elsevier.com)

Next Up, Parkinson's: Coalition Against Major Diseases Moves To Link Neurodegenerative Ailments In Database

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