The Newest 21st Century Cures Draft Moderates, But Doesn’t Eliminate, Controversy

By Rachel Sachs

Earlier this evening, the House of Representatives released the most recent draft of the 21st Century Cures Act. This is the fifth time I’ve blogged about the Act (prior posts here, here, here, and here), which has ballooned from a 200-page discussion draft in April 2015 to a 996-page draft version today. (The House has a 44-page summary here for those with more limited time.) To be fair, the Act now contains a whole set of provisions around mental health, substance abuse, and child and family services which were not originally part of the Act. The 21st Century Cures Act is the biggest Christmas tree bill I’ve ever had occasion to read.

There will be an enormous amount of commentary on different parts of the bill, so here are some quick thoughts on the new draft, focusing not only on the provisions which are likely to attract the most attention, but also on a few quieter provisions that are nonetheless worthy of scrutiny.

Some controversial provisions have been eliminated entirely or softened greatly. One of the most controversial provisions in the last draft of the bill would’ve “farm[ed] out the certification of safety of modified devices to third parties, circumventing the FDA altogether.” That provision seems to be absent from the new draft. The last draft, in creating a program for breakthrough review of medical devices, controversially called for the use of “shorter or smaller clinical trials” for those devices. The new draft asks the Secretary only to ensure that the design of such clinical trials is “as efficient and flexible as practicable, when scientifically appropriate” (section 3051).

Other controversial provisions remain, sometimes under new names. One of the most troubling provisions in the previous draft of the bill would’ve created a program for the use of “clinical experience” evidence in drug approvals. Rather than relying on the gold standard of randomized clinical trials, this provision “would[‘ve] require the Secretary to establish a draft framework for implementing” such evidence. The new draft keeps this provision but changes the term “clinical experience” to “real world evidence” (section 3022). To be sure, this provision gives enormous discretion to the Secretary to limit (and maybe even reject) the use of such evidence. But in light of recent high-profile clinical trial failures, most notably just two days ago, we ought to be concerned about claims that the FDA is too slow and imposes too stringent requirements on drug approvals.

You get a PRV! You get a PRV! And you get a PRV! The new draft of the 21st Century Cures Act not only reauthorizes the existing Priority Review Voucher (PRV) program for rare pediatric diseases. (If you’ve read this far, you probably already know what the PRV program is. If you somehow don’t, here’s my favorite explainer.) It also creates a new PRV program for drugs for “medical countermeasures.” Seemingly no changes are made to the neglected tropical disease PRV program. Helpfully, the new draft retains a provision from the old draft (now section 3014) requiring the GAO to study the PRV program, including for its effectiveness. Earlier this year, the GAO released its first report on the subject (tl;dr they say it’s too early to tell for the pediatric voucher) but it’s important to remember here that the FDA hates the PRV program. Expanding it even further is possibly not the best way to get the FDA on board with this particular agenda.

The Eureka Prize program is fascinating. Section 2002 of the draft bill instructs the Director of the NIH to establish prize competitions in service of one or both of the following goals: 1) “Identifying and funding areas of biomedical science that could realize significant advancements through a prize competition” and 2) “Improving health outcomes, particularly with respect to human diseases and conditions for which public and private investment in research is disproportionately small relative to Federal Government expenditures on prevention and treatment activities… such that Federal expenditures on health programs would be reduced.” This new part of the bill comes out of an amendment proposed by Representatives Todd Young (R-IN) and Andy Harris (R-MD) last time the bill was passed in the House. I’ve blogged previously in more detail about the provision. I’m sad to see some of the detail about the members of the Prize Board omitted from this version, and I’m nervous that the House does not want to consider the intersection of intellectual property protection with the prize program, but this is one to watch.

I’m sure there will be much more coverage of the new draft bill in the coming days. But this may be the bipartisan bill that finally makes it through Congress.

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This entry was posted in FDA, Health Law Policy, Mental Health, Pharmaceuticals, Rachel Sachs, Research Funding by rachelsachs. Bookmark the permalink.

About rachelsachs

Rachel Sachs is an Associate Professor at the Washington University in St. Louis School of Law. Previously, she was an Academic Fellow at the Petrie-Flom Center. Rachel earned her J.D. in 2013 magna cum laude from Harvard Law School, where she was the Articles Chair of the Harvard Law Review and a student fellow with both the Petrie-Flom Center and the John M. Olin Center for Law, Economics, and Business. Rachel has also earned a Master of Public Health from the Harvard School of Public Health, during which she interned at the United States Department of Health and Human Services. Rachel's primary research interests lie at the intersection of patent law and health law, with a particular focus on problems of innovation and access and the ways in which law helps or hinders these problems.