Standards for organizing clinical data from research in some major diseases are taking shape as FDA moves to clear a regulatory path that eventually could allow all submissions to be done electronically.

A private consortium, with support from the agency, is close to announcing data standards have been agreed to for formatting Alzheimer’s disease research, meaning drug sponsors would use the same methods to compile and ultimately submit data to the agency as part of an application.

It is another step in efforts by FDA not only to increase the number of electronic data submissions, but also to enable all the data in its library to be searchable for comparative effectiveness and regulatory research.

Raymond Woosley, president and CEO of the Critical Path Institute, which is working on the Alzheimer’s data standard with the Clinical Data Interchange Standards Consortium, said work on a standard for Parkinson’s disease trials will begin next.

“People are beginning to understand,” Woosley said. “Companies are beginning to recognize that they have to have these standards for efficiency and once they’ve got them, they recognize that our consortia are being very effective at getting the FDA to talk about how you can use the data.”

The Critical Path Institute already has developed a database of Alzheimer’s control data that sponsors can use to design more targeted clinical trials (Also see "Next Up, Parkinson's: Coalition Against Major Diseases Moves To Link Neurodegenerative Ailments In Database" - Pink Sheet, 11 June, 2010.).

Woosley said standards for other diseases, like Parkinson’s and soon polycystic kidney disease, will be developed faster now that relationships are established among the various stakeholders.

For a condition like polycystic kidney disease, which few companies had been willing to go after, a collection of National Institutes of Health data created using a common standard could attract more sponsors to the disease, Woosley said.

“You start with standards, you get the data aggregation that can come from those standards, and then you can really incentivize new drug development,” he said.

CDISC also is helping develop FDA’s Clinical Trial Repository, the organization announced in May, which is expected to be ready in 2013.

In conjunction with the National Cancer Institute Center for Biomedical Informatics and Information Technology, CDSIC is creating the database to automatically validate, load and manage clinical trial data to support research across similar products.

Agency Eventually Wants Only Electronic Submission Of Trial Data

FDA plans to issue a rule requiring electronic submission of all study data.

FDA listed increasing the use of electronic information among its priorities for regulatory reform in the HHS regulatory reform plan released Aug. 22. That included “regulatory revisions to permit electronic submission of clinical study data for drug trials,” according to the plan.

The agency also wants to move to electronic post-market event reporting for drugs and biological products and registration and listing of drugs and medical devices, according to the plan.

FDA said CDER and CBER plan to issue a rule that requires electronic submission of all study data submitted as well as issue guidance outlining expectations for standardized data submissions.

Draft guidance on electronic submission of study data is expected in 2012.

FDA’s goal is to eventually receive all clinical trial data and post-marketing reports electronically. But the agency said it will continue for the foreseeable future to provide alternative means for individuals to submit information, such as the MedWatch phone line for adverse event reports.

Receiving only electronic submissions is a laudable goal, but Robert Billings, vice president of policy for the Generic Pharmaceutical Association, said more sponsors will accept it when sponsors can realize more benefits – which could already be happening.

“I believe the best way to make sure that we go to a paperless system as quickly as possible is to show that it saves time on approvals,” Billings said.

FDA received an average of 10 NDAs, BLAs and efficacy supplements per month with electronic datasets from April 2010 through March 2011. The agency began tracking that statistic in April 2010 as a measure of efforts to encourage sponsors to send submissions in the preferred electronic format. No monthly targets were listed.

The average dropped from 10.5 to 9.5 between the last six months of fiscal year 2010 and the first half of FY 2011.

FDA also is tracking the number of applications submitted with electronic datasets that reached mid-cycle review during a specific month. Overall, the agency averaged more than nine applications per month reaching the mid-cycle milestone from April 2010 through March 2011. Once again, the average was higher in the final six months of FY 2010 than the first half of FY 2011 (11.5 v. 7.3, respectively).

FDA said CDER has expanded resources to facilitate electronic submissions that are compliant with CDISC standards.

The agency has been working on electronic data standards for some time, so application data can be more easily analyzed and reviews can be more predictable. Those submitted on paper or in alternative formats prevent staff from asking in-depth questions and cause delays while data is converted to the desired format.

CDER Director Janet Woodcock has said significant improvements in regulatory efficiency are possible with common data standards, among other changes (Also see "NDA Data Standardization Efforts Emerging Along Public, Private Fronts" - Pink Sheet, 7 February, 2011.).

User fees paid by prescription drug sponsors also would be used to standardize electronic submissions and enhance the agency’s capacity to develop data standards, according to the pending agreement between FDA and industry (Also see "PDUFA Proposal Sheds Tier 3 Funding" - Pink Sheet, 2 May, 2011.).

E-Package Inserts On The Table, Too

Among the proposed regulatory reforms in the plan, FDA also included a requirement for electronic package inserts for drugs and biologics.

The rule would require prescribing information for practitioners be made available electronically, so it is the most up-to-date, rather than solely on paper. A notice of proposed rulemaking on the subject is expected in October, according to an inventory of intended rulemaking recently published in the Federal Register (Also see "FDA's Rulemaking To-Do List Includes Dampening Pedigrees, Boosting Electronic Labeling" - Pink Sheet, 25 July, 2011.).

Several groups seemed to favor a similar electronic system regarding patient medication information (PMI) leaflets. During a two-day hearing on the design and content of the PMI – what has become known as the “one-document solution” for giving prescription information to patients – they said a repository allowing pharmacists, physicians and others to print PMI on-demand would be ideal (Also see "Assurance Vs. Flexibility: Unit-Of-Use Packaging Offers Trade-Offs For Patient Leaflets" - Pink Sheet, 4 October, 2010.).

By Derrick Gingery

E-Submission Part Of FDA Reg Reform Plan; Alzheimer’s Data Standard About To Debut

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