Regulatory Rewind

A mix of the biggest hits from 2018

By Meagan Parrish, Senior Editor

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The Drug Competition Action Plan
Since its adoption in 2017, the FDA has used the Drug Competition Action Plan to push for policies that support the generic drug market. In 2018, the agency requested over $35 million from the Congressional budget to fund two major initiatives within the FDA. One will include the switch from its text-based generic drug review platform to an updated data-based assessment tool. This change will help the agency process generic drug applications in a timelier and more efficient manner.

The other initiative is aimed at helping the agency take more of an active role in keeping generic drug labeling up to date. Typically, innovator drugmakers update labeling information for the brand-name medication, which then applies to the generic. But if the innovator drugmaker takes the product off the market, the process of keeping the generic label up to date can slow down, which could result in the drug being prescribed less often. Keeping the labels updated will ultimately remove a barrier to having more generics on the market.

The CREATES Act
Although it hasn’t been passed yet, the Creating and Restoring Equal Access to Equivalent Samples Act has mostly received broad bipartisan support and is making its way through legislative channels.

According to supporters, the impetus behind the bill is to stop brand-name companies from abusing the FDA’s Risk Evaluation and Mitigation Strategy (REMS) program to keep samples out of the hands of generic drug developers. If adopted, the CREATES Act will allow generic drugmakers to bring civil lawsuits against brand-name manufacturers who fail to provide “sufficient quantities” of the drug samples needed for premarket testing.

At the time of writing, the bill was waiting for a vote in the Senate.

A New Tune for Drug Development

The need for speed hasn’t only been felt in the drug approvals stage — the FDA is also taking targeted steps to make early drug development more efficient.

Promoting Targeted Therapy Innovation
In October, the FDA released two new guidances aimed at modernizing drug development for targeted therapies.

The first draft guidance, “Hematologic Malignancies: Regulatory Considerations for Use of Minimal Residual Disease (MRD) in Development of Drug and Biological Products for Treatment,” is aimed at helping drug developers use MRD as a biomarker in clinical trials for specific blood cancer treatments. According to the agency, MRD assessments may be useful in clinical trials and have the potential to expedite product development.

Screen Shot 2018 12 21 at 3.02.38 PMThe agency also issued a final version of its guidance, “Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease.” The FDA says that the final guidance “addresses the topic of finding treatments that address underlying molecular changes that often cause or contribute to a disease,” which is becoming a more common approach to developing new therapies.

The CDER Shakeup
The FDA also unveiled plans for a major reorganization that affects one-third of the Center for Drug Evaluation and Research (CDER). In an effort to make the process of working with FDA experts on early drug development more efficient, CDER director, Janet Woodcock, announced that the changes in the agency’s various divisions will impact how drugmakers are guided through clinical trials.

Project management for the Office of New Drugs will now be centralized, and various other divisions will be reduced and reorganized into clinical divisions. Ultimately, the goal is to have drug companies work with FDA experts more aligned with the type of therapy being developed.

According to Meredith Brown-Tuttle, a principal consultant with Regulatorium, the reorganization should eventually make the clinical trials process more efficient.

“It will be better, because I’ll have experts at the table who can talk to me about GI or liver disease, for example, who focus on nothing else,” she says.

Forever in Genes

The science behind gene therapies is evolving rapidly, but the FDA has been on the industry’s heels, and working to keep regulatory standards up to date. Recognizing the promise gene therapies hold for a swath of illnesses, including previously untreatable diseases, the agency released six new guidance documents in the first half of 2018 aimed at providing more clarity at gene therapy development and manufacturing.

Some of the new guidances deal with clinical trials (see above) and creating a clarified framework for using a genetic-based approach to early drug development. In fact, the agency has been notably lenient about allowing gene therapies into phase 1 trials. But when it comes to scaling up manufacturing, the agency has begun to develop clearer regulatory controls.

That said, the agency has also indicated that even though patient safety is, of course, paramount, the FDA also accepts some level of uncertainty when it comes to the burgeoning field of manufacturing gene therapies.

“In contrast to traditional drug review, some of the more challenging questions when it comes to gene therapy relate to product manufacturing and quality, or questions about the durability of response, which often can’t be fully answered in any reasonably sized pre-market trial. For some of these products, we may need to accept some level of uncertainty around these questions at the time of approval,” the FDA said in a statement in July.

In an emailed statement, the FDA said that its series of guidances from 2018 should “lay the groundwork for continued advancement by establishing a sustainable, evidence-based regulatory framework for future developments in this innovative field.”

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