Anavex Life Sciences Corp (AVXL)

[sokol]

Yes, the pressure is on for the FDA to embrace biomarkers and end points.

The 21st Century Cures Act (HR 6), if passed by Congress supposedly would quicken the pace of approval for badly needed drugs. The legislation also clears the way to use new and creative adaptive trial designs and deploy the most modern statistical and data tools. Subtitle B of Title II is slightly modified from the original draft of the legislation. It remains focused on the "qualification and use of drug development tools"—an area which has attracted significant attention from legislators in recent months. The hope is that FDA can rely on biomarkers and other surrogate endpoints to accelerate the approval of some new products for high-need populations.

"The development of biomarkers and other drug development tools can benefit the availability of new medical therapies by helping translate scientific discoveries into clinical applications," the bill explains.

The bill calls on FDA to promote the development and qualification of biomarkers by issuing new guidance documents regarding the standards for qualifying biomarkers, and to establish a process by which biomarkers can be qualified.

Interestingly, FDA already maintains a program which would appear to do exactly as Congress has asked for in its bill. The agency's Drug Development Tools Qualification Program, for example, is intended to accelerate drug development by focusing on qualifying relevant biomarkers. A 2014 guidance document, Qualification Process for Drug Development Tools, describes the process by which FDA qualifies these tools. In addition, a new guidance document released to the public in March 2015 clarifies FDA's Critical Path Innovation Meetings process, at which FDA dispenses advice on the development of biomarkers, clinical outcome assessments, natural history designs and new technologies.

Accelerated Approval: Section 2022 of the draft legislation would amend federal law to facilitate accelerated approvals—approvals based on surrogate (e.g. intermediate) endpoints intended to serve as evidence that a drug would be effective in a population. FDA has long relied on surrogate endpoints to support accelerated approvals, but only for drugs intended to treat patients with life-threatening illnesses and unmet needs.

See more at: http://www.raps.org/Regulatory Focus/News/2015/04/30/21192/Regulatory-Explainer-The-Updated-21st-Century-Cures-Act/#sthash.lioPBhF3.dpuf