What’s Next for 21st Century Cures Act?

December 8, 2016

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The 21st Century Cures Act has been long in the making. First passed by the House of Representatives as in July 2015 by an overwhelming vote of 344 – 77, the bill hit the legislative wall in the Senate where, despite rumors of advancement over many months, it stayed put until after the election. Then, with new wind in its sails, a revised version was passed again by the House last week and on December 7 the Senate also yielded a 94-5 lopsided vote to pass, sending the bill to President Obama who has been urging passage. Now that it is here, what does it mean?

As most know, the bill seeks to liberalize the approach FDA takes to drug and device approval with an eye toward speeding innovation and access. The final version of the legislation, passed in time for Christmas, has an ornament hung on it for almost everyone. It will broadly affect healthcare, with the specific aim of speeding up innovation to get new medicines to market more quickly. The means to do so are three-fold – increasing funding for some specific kinds of research; modernizing our approach to clinical trials; and removing bureaucratic obstacles.

There is increased funding for both the National Institutes of Health and the Food and Drug Administration – funding which will be subject to the appropriations process – meaning that while it will not ever be certain, it is at least, aspirational. Also included are funding for President Obama’s Precision Medicine Initiative – aimed at lofty goals in the treatment of diseases such as Alzheimer’s, as well as the Cancer Moonshot headed by Vice President Biden. In addition, the bill seeks to address issues associated with the delivery of mental health services and there is money to support state efforts in the opioid epidemic. Finally, the legislation also has multiple provisions that will strengthen mental health treatment and services. It is easy to see why a bill with something for everyone becomes law with such bipartisan support.

That said there are critics of the legislation who have had specific concerns with respect to FDA. While one of the key perceived benefits of the legislation is to speed up drug approvals – the means for acceleration is a matter of concern for those who see it as compromising what has heretofore been considered a gold standard – Phase III clinical trials. The bill would position the agency to be able to consider patient based experience when assessing drug approvals. While that is welcome news to many, if it became the norm rather than the exception – or if patient experience is used in lieu of clinical evidence instead of in addition to clinical evidence, critics see faster approvals coming with a price tag – lowered scientific scrutiny of safety and efficacy.

Ultimately, while the content is naturally important, what may be most influential in the impact of this new legislation is how it is interpreted and put to use by the person who is at the helm of FDA. The bill creates new pathways for FDA in considering the approval of new products. The question now becomes when – and how often – are those pathways to be utilized? Recently FDA made a drug approval decision that was more based on patient experience than on scientific data for a rare and serious condition. How far that type of decision-making extends in this new era to drugs under consideration is perhaps not clear. At this point, no new FDA Commissioner has been named, but when — and if — a new commissioner is appointed, it will likely shed further light on the true extent of change rendered by the passage of this landmark legislation.