Regulatory officials with the US Food and Drug Administration (FDA) have announced the launch of a project they're calling the Therapeutic Area Standards Initiative Project Plan (TASIPP), which aims to develop and implement new standards to aid in the regulatory review of drugs and biologics.

Background

FDA has long been interested in addressing common bottlenecks in the regulatory review process, believing that such work can generate industry-wide returns in productivity and bring new therapies to patients even faster.

In 2012, the agency announced it would begin funding and working with two groups, the Clinical Data Interchange Standards Consortium (CDISC) and the Critical Path Institute (C-Path), on an initiative known as the Coalition for Accelerating Standards and Therapies (CFAST). The intent of the group is to create new and harmonized data standards in areas prioritized by regulators.

"We need a means to scale the process and manage the development of a very large number of therapeutic area standards," explained C-Path's president, Carolyn Compton. "The establishment of CFAST embodies our resolve to take what we have learned from the initial projects to formulate a new process that will be significantly more efficient while retaining the rigor that is essential to this work."

"As part of the changing tide of drug regulation, we are seeing ever-increasing streams of data coming into the agency," added Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER). "However, much like standardized firefighting equipment, we need to develop standardized definitions for individual diseases and the therapeutic approaches to treat them to be able to tap into this data stream."

"Establishing common standards for data reporting will provide new opportunities to transform the massive amount of data from drug studies on specific diseases into useful information to potentially speed the delivery of new therapies to patients," she added.

One Year Later

One year later, and the group has already announced some of the results of its early-stage work, such as a tool intended to help combine and report data from multiple studies for tuberculosis drugs. Apparently pleased with the work done so far, FDA has announced an additional $2 million in funding for CFAST to help it maintain its current operations and expand into new therapeutic standards areas for hepatitis C, diabetes, lipid-lowering drugs and QT studies, among others.

At present, FDA said, "The lack of standardized data affects CDER's review processes by curtailing a reviewer's ability to perform integral tasks such as rapid acquisition, analysis, storage, and reporting of regulatory data."

"Improved data quality, accessibility, and predictability will give reviewers more time to carry out complex analyses, ask in-depth questions, and address late-emerging issues," FDA continued. "Standardized data will allow reviewers to increase review consistency and perform evaluations across the drug lifecycle. This will enhance the Center's performance across key drug regulatory functions and ongoing business operations, including premarket review, post-market safety, oversight of drug quality, and oversight of drug promotion."

In other words, by helping CFAST, FDA can help both itself and industry by expediting review times and providing a more stable and reliable review platform.

TASI Report

Now FDA is out with a formal project plan for its Therapeutic Area Standards Initiative, meant to guide the agency's efforts at it embarks on a multi-year effort to develop and implement the standards.

The 21-page report explains that FDA has five over-arching goals for the program right now:

• Make significant progress in developing and implementing TA standards.
• Implement binding guidance with a consistent and predictable approach.
• Establish a consistent process that supports continued TA development.
• Define a forward-looking model and timeline for study data standards to ensure sustainability and flexibility over time.
• Make inroads in establishing interoperability with healthcare data standards.

As the second point indicates, FDA is interested in issuing guidance in the future on the requirements for data submissions. The plan states that the data will need to be in a standardized format to facilitate the processing, review and archival of submitted data.

"The final guidance (after public comment) will specify the electronic study data standards, formats, and terminologies that the FDA can process, review, and archive," FDA writes. "In addition, the guidance will provide the Agency's current recommendations on the best means for implementing standardized study data."

In addition, FDA said it is "presently engaged" in a project to model CDER and the Center for Biologic Research and Evaluation's (CBER) specific review requirements such that the agency can better inform the standards development process and make it more consistent and understandable for agency reviewers.

"An initial assessment indicates that this approach is producing useful results with minimal impact in terms of reviewers' time," FDA said. The end goal of this project is to model core requirements for a group of identified therapeutic areas over the next several years.

Once those standards are developed-typically by an outside organization like CFAST-FDA's report indicates that it will still need to undergo a lengthy period of testing, followed by adoption, implementation and guidance phases. FDA said it is still in the process of developing an acceptance testing process to take into account different approaches and measurement criteria to assess readiness.

"The testing approach is envisioned as collaborative FDA-industry effort to assess readiness to generate, transmit, receive, and use data in the standardized form," it wrote.

Comments on the report are due to FDA by 23 December 2013.

FDA's Therapeutic Area Standards (TAS) Initiative Project Plan

Federal Register

Priority Therapeutic Areas for Development (FDA)