A new guidance document just finalized by the US Food and Drug Administration (FDA) aims to clarify how pharmaceutical companies and the public can help accelerate drug development through the agency's Critical Path Initiative (CPI).

Background

FDA's Critical Path Initiative was created in March 2004 as an attempt to identify and dismantle unnecessary roadblocks to new product innovation and regulatory approval. These roadblocks, which would exist on a product's "critical path" to approval, initially included dozens of "opportunities" identified by regulators and the public.

For example, better biomarkers might allow for regulators to approve products more quickly based on the use of surrogate endpoints. New imaging techniques might improve clinical trials and clinical data reliability. Innovative clinical trial designs might allow for shorter clinical trials with the same degree of statistical certainty.

These are tricky problems, FDA explains on its website.

"The monumental effort involved in creating the scientific tools and processes necessary to support 21st-Century medical product regulation cannot be undertaken by any one entity alone. It requires the collaboration of all stakeholders—federal agencies, patient groups, academic researchers, industry, healthcare practitioners, and others."

FDA says it is "uniquely positioned" to help bring together stakeholders to "identify the scientific hurdles to developing new treatments." Those efforts have already borne some results, including the launch of FDA's drug development tools (DDT) program and other related projects.

Critical Path Meetings

So when those stakeholders want to help FDA, what does that process look like?

FDA's newly finalized guidance document, Critical Path Innovation Meetings, explains how stakeholders can set up meetings with FDA to discuss a methodology or technology which might enhance drug development.

Potential topics of discussion can include biomarkers, clinical outcome assessments (COAs), natural history study designs, emerging technologies, new uses for existing technologies and new conceptual approaches to clinical trial design and analysis.

The meetings are not intended to give entities advice on a specific product, but instead to allow FDA to provide its perspective "on the potential for use of proposed new tools and methods in drug development," the agency explains in its guidance document. Regulators say they also expect that the meeting "will provide FDA with exposure to methods and techniques that may have value in drug development."

Requests for meetings should include details about the requester, as well as a six-page (or less) explanation of the background and purpose of the meeting, efforts to advance the project and, if necessary, specific questions for FDA.

The guidance finalizes a draft guidance released in October 2014.

Critical Path Innovation Meetings (FR)