Posted 06 January 2014
By Alexander Gaffney, RAC
Mere months after releasing a development tools guidance intended for medical device manufacturers, the US Food and Drug Administration (FDA) is following its own lead by releasing an extensive final guidance for the pharmaceutical industry outlining the qualification process for developing and validating drug development tools.
FDA has placed a strong focus on drug development tools (DDTs) in recent years. At its most basic, a DDT is intended to help guide the development of a product for a population in which the product can create the most benefit.
For example, a drug intended to treat a rare genetic condition would benefit from a test that would validate the presence of that genetic marker in a patient-a so-called biomarker.
At present, FDA runs a DDT Qualification Program within its Center for Drug Evaluation and Research (CDER). The program has been described by agency officials as "a mechanism for formal review by CDER to qualify drug development tools, and provide a framework for interactions with the Center to better identify data needed to support qualification of these tools."
In addition to biomarkers, DDTs can include clinical outcomes assessments (COAs) and pathways under the Animal Efficacy Rule ('Animal Rule'), used to develop products for rare and dangerous pathogens.
While DDTs may vary in their individual purposes, their overarching purposes are the same: Allow products to reach patients more efficiently, more quickly, more cheaply and with better assurances of safety. DDTs, the thinking goes, can allow a relatively small clinical trial to have the same impact as a much larger trial unsupported by equivalent DDTs, saving a company both time and money.
Already, FDA has unveiled a similar development tools process for medical devices. In November 2013, the agency released a draft guidance, Medical Device Development Tools (MDDTs), to accelerate development.
FDA said that in the case of devices, it was interested in achieving four overarching goals:
- enabling faster and more efficient development of products
- promoting the development of new development tools
- leveraging advances in regulatory science into new development tools
- better communicating to stakeholders which development tools are appropriate for use in development programs
FDA also stressed one additional point: Under its new policy, any MDDTs cleared for use will be qualified per the tool's "context of use"-not for a specific company. "Previously, if there was interest in using a particular MDDT for multiple products or different clinical settings, each FDA device review team would typically evaluate the data justifying the MDDT use for each product or setting separately," FDA wrote. Under the new policy, the qualification process will allow the tools to be used within that same context in the future, reducing the need for redundant reviews by CDRH.
FDA's DDT guidance, Qualification Process for Drug Development Tools, is in many ways similar to its MDDT guidance.
Rather than focus on narrow, one-off DDTs, FDA has instead constructed a framework by which it can "expedite development of publicly available DDTs that can be widely employed." Once qualified, a DDT may be used by any other company to accelerate drug development so long as the tool is used within the same context of use (COU).
"Once a DDT has been qualified, CDER reviewers can feel confident of the application of the DDT within the qualified COU and not have to re-confirm the DDT's utility," regulators write in the guidance. In addition, "subject to review and discussion with CDER staff, a DDT may be used in IND programs for a purpose outside of the qualified COU," FDA added.
Qualification, however, will require "substantial effort," FDA said. Given the public nature of the tools, FDA said it will be best for companies to come together and work collaboratively to tackle common drug development problems. However, FDA will consider an application regardless of the number of applications, it said.
Companies can still validate the use of a DDT within an individual drug development program (i.e. on a drug-specific basis), potentially preventing future competitors from using the same tool as easily.
The guidance goes on to explain the specifics of the qualification process, including how to initiate a request, receiving advice from FDA regarding the application, and FDA's review of the DDT qualification application.
All recommendations made through the DDT qualification process will be made public through FDA's website and the Federal Register, FDA said.
Qualification Process for Drug Development ToolsFederal Register