Peter Wehrwein

Editor

The 21st Century Cures Act that passed the Senate on December 7 is a sprawling piece of legislation that makes MACRA look minor and — at least as far as page count goes — is in the same league as the ACA.

The New York Times described it rather kindly as a throwback to legislative horse trading — a “return to a more classic approach to legislation.”

Merrill Goozner, editor of Modern Healthcare, was less charitable. Some 1,400 lobbyists got an early Christmas present, he wrote in an editorial, recounting how various interest groups managed to come out ahead. Larry Husten, author of the CardioBrief blog and a MedPage Today contributor, called it the 21st Century Giveaway Act because, he says, of all the benefits it has for drug companies and device makers.

And Shelia Kaplan at Stat provided an instructive rundown of the winners and losers. Drug companies, device makers, and the NIH (a $4.8 billion boost in funding) were atop the winner’s column. The losers — a much shorter list — included groups like Public Citizen’s Health Research Group, which fought against the bill, and Sen. Elizabeth Warren, who voted against it and had little success persuading others to join her. The Massachusetts Democrat was one of only five senators to vote nay.

If you take the dare and start roaming around in the bill’s 996 pages, you’ll discover all kinds of legislative nooks and crannies, no doubt each a victory for some special interest group.

There’s Section 2062 that calls for creation of a federal tick-borne disease working group. Bob Herman at Modern Healthcare ferreted out the story behind a provision that delays the termination of the contracts with Medicare Advantage plans that have fallen short on quality ratings.

Interested in telehealth? There’s something for you in the Cures Act — provisions about services for dual eligibles and Medicare beneficiaries with chronic conditions and a MedPAC look at billing.

Of course the Cures Act also deals with much more consequential — and controversial — matters. Here are a few of them:

Real-world evidence. The bill sets in motion a process for creating standards for real-world evidence that could be used to approve new indications for drugs already on the market. It defines real evidence as data coming from sources other than randomized trials. Don’t expect this to happen overnight; a draft guidance is due in five years.

Proponents of real-world evidence and drug companies say treatments will get to patients faster and more efficiently if researchers are allowed to tap into the oceans of data being collected in electronic health records, patient registries, and the like.

And the move to real-world evidence is already underway; the FDA issued a draft guidance for real-world evidence for devices in July.

But this is a controversial area. Some of the most pointed criticism of the Cures Act took aim at the real-world evidence provision. Real-world evidence is a way, say the critics, for drug developers to get around doing randomized clinical trials. Moreover, they say the data collected outside trials is varied and inexact so it can’t be crunched into a reliable guide for approval decisions.

Husten said the researchers he spoke with believe real-world evidence opens up the door to data dredging for favorable results.

Other changes at the FDA. The bill, of course, has many other FDA-related provisions. Putting the particulars aside for a moment, the proponents see them as modernizing the approval process and getting rid of barriers to drug and device development. Critics see a dangerous watering down of standards and giveaways to drug and device companies. There’s really no bridging that gap.

Not all of the many FDA-related provisions can be covered, so this is just a tiny sample:

  • Patient experience data. The bill will make patient experience data part of FDA approval processes and requires the development of FDA guidances about methods for collecting it. On its face, this would seem sensible and humane and not the least bit controversial, but some see patient experience data as possibly clouding FDA decision making with less rigorous evidence. The close — and increasingly financial — relationship between drug developers and patient advocacy groups that might collect this data stokes suspicion.
  • Breakthrough devices. The FDA has created several “pathways” that speed up the agency’s review of medical devices. The Cures Act would establish one for breakthrough devices, defined as devices for life threatening or debilitating conditions for which there is no clear alternative. Critics see an opportunity for device makers to escape tougher testing and higher standards of evidence.
  • Summary level review. The bill allows drug companies to submit their own data summaries when applying for approval for a new indication of a previously approved drug. They must also submit the underlying data, but the FDA decision on the new indication could be based on the review. Drug companies and experts who see the FDA as an obstacle to innovation say the summaries are sufficient for drugs already on the market and will benefit people with conditions covered by the new indications. Husten and others say an independent FDA review of the raw data is crucially important and can reveal problems with a drug that the company summaries may not include.

Mental health care. We’ll see how it plays out, but in contrast to the drug and device approval provisions, the mental health provisions seemed welcomed by all. Advocacy and professional organizations were unstinting in their praise. One major crowd pleaser: a full-court press on mental health care parity that includes guidance documents, audits of health plans with violations, a high-profile stakeholder meeting, and a parity website.

The mental health part of the bill also throws money at various aspects of the problem. There’s funding, for example, for jail diversion programs ($4.2 million per year for four years) adult suicide prevention ($30 million), and assertive community treatment ($5 million), a team-based approach to mental health care that addresses employment and housing as well as medication and therapy.

The bill also makes adjustments to problems in existing programs that have rankled providers and patients. Among the provisions singled out for praise by the American Psychological Association is one that gets rid of the “same day” exclusion, which prohibits separate Medicaid payments for mental health and primary services provided on the same day.

NIH funding. An upbeat Politico story about all the political gamesmanship that led to the broad bipartisan support for the Cures Act casts the $4.8 billion boost in NIH funding as the big quid pro quo for the regulatory changes favored by Republicans.

Vice President Joe Biden is a pivotal player in the Politico account. Included in the NIH money is $1.8 billion for Biden’s cancer moonshot, which was motivated by the death of his son, Beau Biden, from brain cancer (the moonshot has been officially renamed the Beau Biden Cancer Moonshot). To Democrats who saw the Cures Act as making too many concessions to drug companies, Biden countered that it was difficult to help patients without helping companies that provide the cures the patients need, according to Politico.

The NIH funding also had to help put a White House in need of a legacy fully on board. The Cures Act also has money for President Obama’s Precision Medicine Initiative ($1.5 billion) and the Brain Initiative ($1.5 billion).

Don’t be wowed by the NIH money, said the few dissenting Democrats and dismayed outside groups. Three reasons were given. First, over 10 years, it is just not that much money. Second, it must be appropriated every year, so down the line, the plug could be pulled on the research funding but not on the regulatory changes. Third, the Cures Act cuts $3.5 billion (about 30%, according to news accounts) from the Prevention and Public Health Fund established as part of the ACA. Some public health groups said the cuts are shortsighted because the fund supports the unglamorous work of preventing chronic disease and other health problems before they occur and become expensive to treat.