On Monday, the House of Representatives released the latest version of the proposed Food and Drug Administration (FDA) user fee reauthorization bill. The House is expected to vote on the must-pass bill as early as today, but the Senate has not yet set forth its own timeline for voting on the bill. I have previously written here about the version of the bill marked up in the Senate, and about the committee staff’s valiant efforts to keep the bill free of politically controversial provisions.
In this post, I review some of the key provisions in the newly released bill and consider its implications for the FDA.
Widespread Agreement Between the Senate And House Bills
With one exception (as noted below), the bill includes few surprises for those who have followed the markups in the Senate and House. The two amendments added in the Senate hearing—Senator Hatch’s (R-UT) expanded access amendment and Senators Collins’s (R-ME) and Franken’s (D-MN) generic competition amendment—are included as sections 610 and 801 of the bill.
Importantly, small changes have been made to both of those amendments. Most are not substantive. For instance, as voted on in the Senate, the generic competition amendment gave the FDA “240 calendar days” to act on the submission of a priority generic, while section 801 of the House bill gives the FDA “eight months” to act. Several changes in the expanded access amendment impact scheduling, although perhaps not significantly. As one example, the amendment as voted on in the Senate provided for a public meeting on the topic “not later than 180 days” after the Act’s enactment. The House bill changes the deadline to “270 days.”
Other changes in language may have more substantive implications. In promulgating guidance on the subject of expanded access trials—which would include people who might benefit from the drug but would be ineligible for standard clinical trials—the FDA is now specifically instructed to address methodological approaches that are appropriate “for drugs intended for the treatment of rare diseases or conditions” (Section 610(a)(3)(B)(iii)). The FDA has already exercised flexibility in evaluating clinical trials for rare disease drugs, given challenges presented by those diseases (such as small sample sizes). Congress may be looking for the FDA to extend that flexibility to the expanded access context.
Finally, as voted on in the Senate, the generic competition amendment would not have expedited review for generic applications “submitted more than 30 months in advance” of the expiration of relevant patents or exclusivity periods. That language has been simplified to say that expedited review will attach for drugs “for which there are no blocking patents and exclusivities.” If generic manufacturers must wait until all patents and exclusivity periods expire before applying for expedited review, it may well delay the approval of at least some drugs under this provision relative to the original wording of the amendment, particularly if an abbreviated new drug application (ANDA) sponsor intended to file a Paragraph III certification (indicating that the generic manufacturer will not enter the market until relevant brand-name patents expire).
The many amendments added by the House Energy & Commerce Committee during its markup are also included. Of particular interest may be Representative Schakowsky’s (D-IL) amendment, noting the “sense of Congress on lowering the cost of prescription drugs.” Now included as section 609, this amendment has no legal force but serves as a reminder that many members of Congress, on both sides of the aisle, are very interested in the question of drug pricing. Of course, it also serves as a reminder that Congress has as yet been unable to agree on what to do about it.
More generally, provisions from the Senate bill have been moved around or retitled in the House bill, but they are still present. For instance, section 607 of the House bill, “orphan drugs,” requires sponsors for orphan drugs that are the “same drug” as already-approved orphan drugs to demonstrate clinical superiority. This language was included in the Senate bill under section 608, “technical corrections,” and has simply been relocated. For those who are interested in this topic, FDA Law Blog has a clear summary of this provision and its relationship to ongoing FDA litigation and interpretations.
Incorporating The RACE Act
One significant change from the mark-ups does stand out: the inclusion of substantive provisions strongly resembling the Research to Accelerate Cures and Equity (RACE) for Children Act. The Act, which had been introduced in both houses of Congress by sponsors from both parties, is aimed at requiring pharmaceutical companies to do more to evaluate cancer therapies in children.
The primary substantive provisions of the Act had not been included in the bills considered by committees in either chamber. However, there were still indications that the committees were interested in the RACE Act. The Act includes a procedural section providing for early meetings with the FDA on a sponsor’s pediatric study plan, and even the Senate bill contained a section tracking that language closely (Section 502).
Section 504 of the House bill is much more detailed than the original text of the Act; it requires sponsors of drugs intended to treat adult cancers to study those drugs in pediatric populations if the drugs are “directed at a molecular target that the Secretary determines to be substantially relevant to the growth or progression of a pediatric cancer” (Section 504(a)(1)(A)(iv)). As the FDA moves toward approving more cancer drugs on the basis of the biomarkers they target, as opposed to the location of the cancer within the body, this provision may be increasingly relevant.
There are a number of interesting items in this long, detailed section of the bill, but I want to highlight just one. The bill instructs the FDA Commissioner to “establish and update regularly” a list of molecular targets that meet this “substantially relevant” requirement. But it goes on to specify the process by which the FDA Commissioner is to develop this list, stating that the FDA must “consult the National Cancer Institute” and others in doing so (Section 504(a)(3)(B)).
I have often written (including for this Blog) about the importance of the FDA’s relationship with other administrative agencies which are involved in the health technology space. A number of agencies within the Department of Health and Human Services have valuable contributions to make to the discovery, approval, and purchase of innovative new health technologies. However, it is relatively rare (although it does happen) to see Congress order such collaboration. The NCI has valuable information to add here, and I look forward to the partnership between the agencies.
What’s At Stake
The last round of user fee agreements, passed in 2012 and currently in place, expires on September 30. Congress must past this bill to renew the FDA’s authority to collect user fees, which make up on average 60 percent of the FDA’s costs. If the bill is not reauthorized by the end of July, on August 1 the FDA would need to send prospective layoff notices to over 5,000 employees.
In theory, the House is voting on the bill with plenty of time for it to pass the Senate and be signed by the President before the end of the month. But the Senate has not yet put forth its own schedule for voting on the bill. As Zach Brennan of RAPS reported, a spokesman for Senate Majority Leader Mitch McConnell stated, “I would gently remind you that we are working on health care right now.”
It would be a shame if the collaborative, bipartisan reauthorization of the FDA’s funding were jeopardized and career public servants fired because Senator McConnell has chosen to prioritize partisan bickering on health care reform. It is the Senate’s choice to continue working on Affordable Care Act repeal to the exclusion of all else.
Under ordinary circumstances, I would expect this bill to pass both the House and Senate easily, as the House bill’s inclusion of the Senate’s amendments reflects the priorities of lawmakers in that chamber. I will stay tuned for news of the timing on which the Senate plans to consider the bill.
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