During testimony before the US Senate Committee on Appropriations regarding President Trump’s fiscal year (FY) 2019 budget request, US Food and Drug Administration (FDA) Commissioner Scott Gottlieb highlighted two of the agency’s key priorities, addressing drug pricing by increasing generic-drug competition and real-time product performance data in post-market surveillance.

During his April 24, 2018 testimony, Gottlieb proposed a solution to increasing generic-drug competition with two policy components. “Our proposal to modernize our generic-drug platform comprises two policy components,” Gottlieb said in his testimony. “The first element of our proposal will help make the approval of new generic drugs more efficient by establishing a structured application. The new process will also make submission of new generic drug applications, and their review, more seamless and efficient,” he said. “We believe a structured application, which allows key sections of the generic-drug file to be captured in a structured template, will speed the review of the application and cut down on the number of review cycles that applications must undergo, thereby reducing generic-drug development timelines.”

The second component to increase generic-drug competition, he said is “aimed at promoting the more widespread use of existing generic drugs, by looking for ways to keep generic drug labels up-to-date with the latest information about each medicine’s safety and benefits.”

With respect to labeling, Gottlieb pointed out that current laws generally require generic drugs to have the same labeling as the brand drug that they reference. However, when brand reference drugs voluntarily withdraw their marketing applications and therefore stop updating their labeling, the FDA loses a mechanism to update generic labels. The number of generic drugs that reference discontinued or withdrawn brand drugs essentially comprise about one-fifth of the total number of reference-listed drugs and consists of approximately 460 unique active ingredients, according to information from the FDA.

“If the brand drug sponsor has voluntarily withdrawn [its] marketing application, there’s no sponsor who is responsible for making any necessary label updates that other generic applicants would follow,” said Gottlieb in his testimony. “The result is that these drug labels get frozen in time.”

To put into context, he provided one estimate that there are about 5,600 reference-listed drugs that correspond to generic medicines. And of these reference drugs, 1,170 are tagged as discontinued or withdrawn by their original, branded sponsors for reasons other than safety or effectiveness. “Assuming each discontinued or withdrawn reference listed drug corresponds to five generic drug products that, on average, refer to it, then it could mean that about 20 percent of all generic drug products on the market have been discontinued or withdrawn reference listed drugs,” explained Gottlieb. “For the generic drugs with a withdrawn reference listed drug, that means there’s no single sponsor that’s responsible for updating the generic drug label. This is about 5,000 drugs,” he said. 

“FDA can assume more responsibility to help bring these drug labels up-to-date,” said Gottlieb. “I believe that if we take on this role; it can promote more widespread use of generic medicines. Our Budget requests money to do just this.” The FDA says it expects to play a more active role in updating more of these generic labels, which builds upon a pilot to update the labels on older generic cancer drugs.

Regarding Gottlieb’s second priority for real-time data, Gottlieb says this effort will help ensure that patients and providers have the most up-to-date information on which to make decisions–in addition to improving the use of real-world evidence for many of the products the FDA oversees.

He said that the FDA is seeking to expand new and existing active surveillance systems to enhance its real-time understanding of medial-product performance by broadening its use of healthcare data to use data available from electronic health records.

To accomplish this, Gottlieb listed several objectives. “First, the data must be high quality and ‘inter-operable,’ meaning that the data can be collected in one system, but used in many systems to inform patient care,” Gottlieb said in his testimony. “Second, we must work toward better integration of clinical care and clinical research….Finally, we must support development of tools that will allow these data to be properly used to inform our understanding of not only medical-product safety, but also its efficacy.”

Currently, the FDA’s active post-market data monitoring systems—principally its National Evaluation System for Health Technology database for medical devices and its Sentinel system for drugs and biologics—depend largely on the secondary use of claims data, according to Gottlieb.

Gottlieb says having a real-time, post-market, real-world experience system will also give the FDA additional ways to use real-world evidence to expand what it knows about the effectiveness of new products. For example, he said that the agencycan use electronic healthcare data to broaden the indications for use of approved medical products, eventually perhaps conducting much of late stage development in the “real world.”

The testimony provided by Gottlieb was to answer questions on the President’s budget request for the agency. President’s FY 2019 Budget request for the FDA requests $5.8 billion in total resources for the FDA, which includes an increase of $473 million in budget authority and $190 million in user fees.

Source: FDA and Senate Committee of Appropriations (hearing)