Passed by Congress at the end of 2016, the 21st Century Cures Act is aimed at easing the process by which drugs can be developed and patients can access the treatments they need at the time they need it. It addresses everything from encouraging young medical researchers and advancing the Cancer Moonshot initiative to reducing the administrative burden on NIH grant recipients. There are several provisions in this new law that could have a significant impact on the kind of evidence that the pharmaceutical industry may use when undergoing drug approval.
Here’s what you need to know about the impacts the Cures Act will have on the pharmaceutical industry in the coming years.
A Brief introduction to the 21st Century Cures Act
While this 1000+ page piece of legislation is comprised of many ambitious initiatives intended to facilitate medical research and improve patient outcomes, the provisions of most interest to the pharmaceutical industry are those that aim to accelerate and streamline the process by which new drugs can be approved by the Food and Drug Administration (FDA).
Although this legislation is truly sweeping in scope, the sections of this lawthat should be of the greatest interest to pharmaceutical companies are those that amend the Federal Food, Drug, and Cosmetic Act and establish protocols for the FDA to use to evaluate patient experience and other forms of real-world data when assessing the risk-benefit analysis of a new drug. According to Stat News, real-world evidence (RWE) came out as a “winner” of the new legislation, because the Cures Act gives pharmaceutical companies more leeway to use this “useful and timely” kind of evidence.
What’s real-world evidence?
According to the site Health Law and Policy Matters, the Cures Act defines RWE as any drug data that is derived from a source other than a randomized clinical trial. The new legislation requires that the FDA, within two years of the passage of this act, devises and implements the guidelines by which companies may evaluate RWE. Within five years, the FDA must draft guidelines defining the appropriate ways in which the pharmaceutical industry can collect, analyze and incorporate RWE for either a) approval of a novel use of a previously approved drug, or b) to meet requirements after a drug has been approved.
To clarify, this does not mean that RWE cannot be used for other purposes and does not change the standards of evidence outlined by the Food, Drug and Cosmetic Act. Health Law and Policy Matters notes, however, that this marks a substantial change from the past, when the FDA issued RWE guidelines for medical devices but not for medications.
Why RWE is important
There are a number of reasons to be optimistic about the RWE provisions in the Cures Act, including the increased use of novel sources of information and, perhaps more importantly, the streamlining of the FDA approval process.
Utilization of novel information sources
One of the most important things about this increased use of RWE is that it will expand the sources of evidence on which a pharmaceutical company can draw in the process of drug approval.
In the past, the FDA relied largely on data gathered from clinically controlled trials to make their decisions regarding drug approval. However, authors writing in the New England Journal of Medicine (NEJM) note that with the increased use of RWE allowed by the Cures Act, other data sources can now be utilized. This can include the immense amount of data now generated from electronic health records (EHRs) and even wearable technology and mobile apps, as well as information gathered from patient billing information and entities like disease registries.
Increased expedience for drug development
The development of new drugs is, understandably, incredibly important to patient outcomes — but according to the Center for Bipartisan Policy, the current process by which a new drug comes to market is both long and expensive. It typically takes over a decade and around $2 billion to develop a new medication. However, the center believes that certain features of the Cures Act (such as the increased use of RWE) will streamline and modernize this costly and drawn-out process so that patients can receive new drugs and treatments in a more timely manner.
In short, the advent of the Cures Act will have a significant impact on the pharmaceutical industry — and it will be more important than ever for pharmaceutical companies to monitor published and presented RWE from around the world, as this evidence may be increasingly important in the FDA approval process.