On Thursday, May 11, the Senate Committee on Health, Education, Labor and Pensions marked up the proposed Food and Drug Administration (FDA) user fee reauthorization bill and voted 21-2 to advance it to the full Senate. The bill would reauthorize the fee system for the FDA’s approval of drugs, medical devices, generic drugs, and biosimilars. Recognizing both the must-pass nature of the bill and the partisan climate in Congress right now, the committee and its staff smartly managed to keep the bill fairly clean, adding few non-funding related provisions relative to some past user fee agreements (UFAs). Still, the full text clocks in at just under 200 pages, including amendments.
I review some of the key provisions below.
Eschewing The President’s Proposed User Fee Hikes
Most importantly, the bill reauthorizes the different user fees drug and device sponsors pay the agency in exchange for review of their products. What is most notable about this set of provisions, which make up Titles I through IV of the bill, is perhaps what is not there. President Trump’s budget blueprint aimed to decrease general funding for the FDA and make up for the shortfall by doubling the size of user fees from industry. This bill does not do that and instead moves forward with the fee levels that were negotiated between the FDA, industry, and consumer groups prior to the release of that blueprint.
Broadening The Definition Of Patient Experience Data
Other provisions of the bill are noteworthy for the ways in which they interface with the 21st Century Cures Act, signed into law in December. Sections 3001 and 3002 of that Act first instruct the FDA to make publicly available statements about patient experience data submitted as part of an approved new drug application (NDA) or biologics license application (BLA). They then instruct the FDA to develop a plan to issue draft and final guidance documents on the collection of patient experience data. Section 3001 defines patient experience data as including data that “are intended to provide information about patients’ experiences with a disease or condition, including (A) the impact of such disease or condition, or a related therapy, on patients’ lives.” [emphasis added]
Section 605 of the user fee bill makes a small change to the italicized text, instead including “physical and psycho-social impacts of such disease or condition, related therapy, or clinical investigation.” Substantively, this section makes clear that “impact” on patients’ lives ought to be considered broadly, going beyond mere physical impacts. Procedurally, the 21st Century Cures Act’s first deadline on this subject is coming up in less than a month—new Commissioner Gottlieb must develop a plan to issue draft guidance on the subject—making this a particularly timely provision.
Limited Restriction On Drug Importation Likely Motivated By Specific Safety Issues
A more substantive provision of note is section 607, “protecting and strengthening the drug supply chain.” This provision blocks (with two exceptions) the importation of drugs that are subject to 21 U.S.C. § 353(b)(1), which includes drugs that are “not safe for use except under the supervision of a practitioner licensed by law to administer such a drug,” or are, more generally, subject to Risk Evaluation and Mitigation Strategy (REMS) proposals.
It is difficult to be sure about the motivations behind this particular section, but I would caution against viewing it as an attempt to keep drug prices high by preventing importation more generally. Portions of the regulated industry may see it that way, but its restriction to drugs which are of potential public health concern, in terms of their potential for abuse or otherwise, suggests that it has more positive motivations. It may be a response to a safety concern the FDA and patient groups have observed, or it may be an attempt to ensure that future importation provisions do not include such drugs.
Instructing The FDA To Examine Barriers To Clinical Trial Participation
In Thursday’s hearing, two additional amendments were added to the bill. The first, from Senator Hatch, aims to expand patient access to experimental treatments. Senator Hatch has co-sponsored “right to try” legislation in the past, and this bill takes a different approach to addressing the underlying concerns behind those pieces of legislation. Specifically, the amendment (more formally known as the Enhanced Clinical Trial Design Act) instructs the FDA to examine clinical trial inclusion and exclusion criteria and consider the barriers to patient access created by those criteria. Again, this provision dovetails with some of the 21st Century Cures Act’s requirements—its instruction to consider the enrollment of pregnant and lactating women in clinical trials is closely related to section 2041 of that Act. However, Hatch’s amendment is aimed more at patient access, rather than at ensuring that innovation meets the needs of broader populations in a safe way.
Formalizing FDA’s Goal Of Prompt Review Of Generics Where Little Competition Exists
The second amendment, from Senators Collins and Franken, would require the FDA to prioritize review of generic drug applications in situations where there are three or fewer approved generics on the market, suggesting that more competition may be valuable. This provision would require the FDA to act on such applications within eight months. In general, the FDA has aimed to hit a ten-month benchmark under the Generic Drug User Fee Act (GDUFA).
However, the FDA has already stated that one of their goals for this round of UFAs is to approve “priority original ANDAs [abbreviated new drug applications]” within eight months of submission. The FDA has also already begun to prioritize review of “sole-source” products. As such, the primary contribution of this amendment may be to formalize and enshrine into law those agency goals. This amendment does not go as far as existing proposals, such as the legislation introduced by Representatives Bilirakis (R-FL) and Schrader (D-OR) in the House, which would create a six-month timeline for priority ANDA filings.
Avoiding The Minefield Of Drug Importation From Canada
One amendment of note was rejected by the Committee on Thursday: a proposal by Senator Bernie Sanders to more broadly allow the importation of prescription drugs from Canada. The amendment was voted down, likely due to both its substance and potential political divisiveness. Especially because other similar provisions have been previously rejected in the Senate, it is easy to see how this amendment could have created problems for the bill going forward.
For those who are interested in this provision, it appears to track identically an importation provision in a recent drug pricing bill introduced in both houses of Congress, about which I’ve previously written.
The user fee bill includes a number of other uncontroversial provisions on topics like over-the-counter availability of hearing aids, facilitating international harmonization of clinical trial standards, and the like. But I do not expect these provisions to pose obstacles to the bill’s passage in the near future.