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RegBits: All the Regulatory News You Might Have Missed This Week

Posted 15 May 2015 | By Alexander Gaffney, RAC

RegBits: All the Regulatory News You Might Have Missed This Week

It's been a busy week in the world of regulatory affairs, and there's a lot of news we hoped to cover but never got around to. Read on to check our brief synopses of the stories we missed.

CDRH's New Adaptive Design Guidance

Adaptive design trials are trials in which a company is able to modify a trial based on pre-planned criteria, thereby allowing the company to adjust the size or scope of a trial. For example, a company might conduct an interim analysis to either increase or decrease the size of a trial.

The US Food and Drug Administration's (FDA) Center for Devices and Radiological Health (CDRH) has now published a new draft guidance document on adaptive design trials, laying out its expectations for the trials in the hopes of encouraging companies "to consider the use of adaptive design in their clinical trials."

Guidance: Adaptive Designs for Medical Device Clinical Studies (FR)

Guidance on Enzyme Replacement Therapy

CDRH wasn't the only regulatory center releasing guidance this week. FDA's Center for Drug Evaluation and Research (CDER) released a new draft guidance document, Investigational Enzyme Replacement Therapy Products: Nonclinical Assessment.

The document "is intended to advise the sponsors and individuals involved in the design and implementation of nonclinical studies on the substance and scope of nonclinical information needed to support first-in-human clinical trials, ongoing clinical development, and eventual approval of enzyme replacement therapy (ERT) products for the treatment of rare, life-threatening conditions," FDA explained.

Investigational Enzyme Replacement Therapy Products: Nonclinical Assessment (FR)

FDA's New Emergency use Authorization

In February, Regulatory Focus reported the US Department of Health and Human Services (DHHS) had authorized FDA to authorize the use of new products under its emergency authority. The risk, DHHS said, was an emerging outbreak of enterovirus.

This week FDA quietly authorized the first device to detect enterovirus using its emergency use authorization (EUA) authority. The device, the Centers for Disease Control and Prevention's (CDC) Enterovirus D68 2014 Real-time RT-PCR Assay, is an in vitro diagnostic intended to detect the Enterovirus D68 strains detected in the US last year.

FDA Notice

Petitioning FDA on Right to Try

As Regulatory Focus has been reporting for several months, more than a dozen states have passed so-called "Right to Try" bills intended to facilitate access to experimental drugs for critically ill patients. The laws, in general, shield product manufacturers from state-level liability and allow products which have passed Phase 1 safety testing to be distributed to consumers under limited circumstances.

Now the advocacy group behind many of those bills, the Goldwater Institute, is launching a petition to ask FDA to support similar measures on a national scale. The petition already boasts 35,000 signatures and counting.

Press Release

ICH bin ein Harmonizationer

The US Food and Drug Administration (FDA) this week announced the availability of grant funds intended to support the work of the International Conference on Harmonization (ICH), a prominent pharmaceutical harmonization group primarily focused on innovative pharmaceutical products.

ICH members include the US, EU and Japan, although many other regulators are involved in various capacities. The group, FDA notes, "ensures that safe, effective and high quality medicines are developed and registered in the most resource-efficient manner."

ICH will be awarded $500,000 to further its harmonization efforts, FDA said.

FDA Notice

More FDA Funding

ICH isn’t the only recipient of FDA largesse this week. In two separate funding notices, FDA said it would be granting up to $1 million to strengthen medical product regulatory systems in the Americas and, separately, $500,000 to evaluate quality metrics for risk-based surveillance of drug manufacturing operations.

FDA Funding Notice / FDA Funding Notice

Warning on SGLT2 Inhibitors

Diabetes drugs have long been the subject of FDA concerns for various reasons, including the large population in which they are used. On 15 May 2015 FDA issued a warning regarding a class of diabetes drugs known as SGLT2 inhibitors—a class which includes drugs like dapaglifozin and empaglifozin.

The reason for the safety warning, FDA said, was the risk the drugs could cause ketoacidosis, "a serious condition where the body products high levels of blood acids called ketones that may require hospitalization."

The agency is "continuing to investigate this safety issue," it said, and is still considering what regulatory responses, including changes in the prescribing information, are necessary.

FDA Warning

Brookings to Congress: Why no Postmarketing Measures in 21st Century Cures?

The Brookings Institute, a venerable think tank highly involved in FDA regulatory matters, authored a post this month arguing that legislative proposals to rethink FDA need to "look beyond FDA approvals." Instead, the think tank argues, legislators need to consider what happens to products after they're approved.

That's not necessarily a new concept—it was a massive part of the FDA Amendments Act of 2007 which established mandatory postmarket monitoring programs for some drugs. But as Brookings argues, its full promise and potential hasn't yet been met under current legislation.

By better collecting real-world data in post-marketing trials, the group argues, companies can better target their medical products to the ideal patients, collect valuable data to drive future drug development, and ensure the safety and effectiveness of new products (especially ones approved based on limited data).

It's a strong argument, and one to keep an eye on as the Senate and House move forward with their legislative plans in the coming months.

Brookings Post

Security Pilot Program

FDA said this week it is seeking sources capable of developing a pilot program for the new Drug Supply Chain Security Act. The pilot program is intended to "explore and evaluate methods to enhance the safety and security of the pharmaceutical distribution supply chain," FDA said.

Pilot Program Solicitation

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