How the 21st Century Cures Act and Real World Evidence Impacts the Drug Approval Process

The 21st Century Cures Act

In late December 2016 and with only three days remaining in session for the year, the U.S. Congress passed the 21st Century Cures Act, now commonly referred to as the Cures Act. This act laid out goals to “accelerate the discovery, development, and delivery of 21st century cures.” The law authorized $6.3 billion in funding, mostly for the National Institutes of Health. The Cures Act was first introduced nearly two years prior by seven bipartisan cosponsors, many of whom resided on the Science, Space, and Technology Committee. The legislation was supported by pharmaceutical manufacturers, as it had several important measures that would allow the healthcare and pharma industry to make more informed decisions to ultimately improve the patient journey. President Obama said the Cures Act will “modernize research and accelerate discovery” using data so that “treatment and healthcare can be tailored to individual patients.” It is a highly significant piece of legislation that will impact every pharmaceutical company and the way in which they manage, analyze, and leverage their real world data.

How does the Cures Act fit into today’s technological landscape? Sophisticated analytical capabilities — including artificial intelligence (AI) and machine learning — have immense potential, but they require greater access to large amounts of data. The Cures Act addresses medical research and focuses on leveraging technology to improve interoperability and electronic health information exchange.

A provision within the Cures Act establishes a program to evaluate the potential use of real world evidence (RWE). This is expected to drastically change the FDA drug approval process.

real world.jpegWhat is Real World Evidence?

For the purposes of the Cures Act, real world evidence is defined as “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomized clinical trials.” Real world evidence sources can include:

  • Ongoing safety surveillance
  • Observational studies
  • Registries
  • Health claims
  • Patient-centered outcomes
  • Prescription records
  • Lab results
  • Drug safety
  • Drug interaction data

The Cures Act requires the FDA to expand the role of real world evidence in its drug approval process.

The Previous FDA Drug Approval Process

The previous drug approval process requires randomized clinical trials (RCTs). It also necessitates treating patients with a certain level of severity. These trials yield placebo and drug results that must then be compared against each other to determine if the drug can be approved by the FDA.

If a drug is on the market for three years and begins to show that it also has a benefit for a related indication, it must first be sent back to clinical trials. In order to legally treat another condition and update the label to include these benefits, the pharmaceutical company would be required to conduct RCTs for the new goal.

Take, for example, a new chemical entity that has been approved, deemed safe for consumption, and qualified as effective. It’s undergone the rigorous drug approval process by the FDA and passed. After the drug has been on the market for a time, the pharmaceutical company wants a different formulation (for example, a gel-filled pill or a higher strength) or has noticed the drug addresses symptoms of a different type of condition that wasn’t tested in the initial RCTs. In the past, the company must put the drug back through the entire process for the new indication. This takes years and millions of dollars.

How Real World Evidence Modifies the FDA Drug Approval Process

Now, organizations are able to submit real world data as evidence in order to get drug approvals. The pharma company trying to approve a new indication for a previously approved drug can turn to RWE. The Cures Act intends to expedite the process by which new drugs and devices are approved. It allows companies (under certain conditions) to provide data summaries and real world evidence, such as observational studies, insurance claims data, patient input, and anecdotal data. It’s important to note that real world evidence does not eliminate the need for randomized clinical trials. Initial drug approval still requires RCT. Post-launch RWE may be enough to get approval for a different delivery mechanism, treatment for a new condition, or to claim a new benefit. However, depending upon the potential safety impact, RCTs may also be required here. Regardless, the number and length of clinical trials can be reduced (and costs lowered) when supported by significant RWE.

Recruitment tactics informed by real world patient data

RWE and random clinical trials aren’t necessarily competing with one another. There are some ways to use the new method to improve upon the old method. Real world evidence can be used to improve clinical trials. Using RWE, drug companies can target clinical trial locations in an effort to streamline and reduce errors within the trials themselves. Real world data can “show” actual available patient populations that meet protocol criteria. The data will allow for more efficient recruitment tactics that result in better understanding of treatment patterns. This reduces drug development time and costs by selecting active sites in high-potential regions.

What are the Implications of RWE’s Impact on Drug Approval?

There are positive and negative implications of real world evidence’s impact on the drug approval process.

While randomized clinical trials (RCT) have been the gold standard for new drug approvals, real world data and real world evidence have been topics of interest for many years. The Cures Act legislation is the impetus to bring RWE to the forefront of pharma R&D. This saves the medical community many years and millions of dollars. Pharmaceutical companies don’t have to pay doctors to recruit patients — real world data can inform better recruitment tactics.

The FDA, however, has said that real world evidence could potentially generate “incorrect or unreliable conclusions.” Real world data isn’t collected with the specific intent of supporting research, so real world data (RWD) hasn’t been optimized for this purpose.

RWD is simply an opportunity to bring new data to the equation. In addition to the legislation itself, there are a couple of factors that should be noted.

  1. Explosion of data sources

More and more data is made available through electronic health records (EHR),

disease registries, biomarker data, genomics data, and sensor and wearables data. All of this information can be used to improve individual patient insight.

  1. Cutting-edge technology

Pharma organizations can team up with technology companies that specialize in

the healthcare industry in order to analyze these massive amounts of data in real time. RxDataScience is one such partner — this company can ingest pharma data, link it all together, and complete analyses. They’re currently working with pharmaceutical companies to both modernize RWD infrastructures and implement RWD strategies. The Cures Act is a catalyst for investment in new programs for those companies figuring out what their RWD platforms need to be.

What Pharma Companies Need to be Doing

Pharmaceutical companies must look at the current way in which they manage their real world data. In order to take advantage of the possibilities RWE creates, pharma must:

  1. Integrate RCT data with RWE data
  2. Validate RWD
  3. Look at new technology platforms that will enable them to properly (and quickly) integrate/validate — current platforms aren’t suited for this challenge

The acceptance of RWE in the FDA drug approval process has created new opportunities and proven that the status quo has changed. Traditional pharma procedures are suddenly inadequate, from both a technological and strategic standpoint, so companies need a new approach. RWE can be used to modernize the drug development process. The goal is to improve regulatory clarity regarding the use of RWE and policies for information sharing. This data can also help explore new, innovative ways to modernize drug development.

RxDataScience is already partnering with several pharma companies to modernize their RWD platforms. RxDataScience is uniquely capable of addressing this challenge, modernizing RWD structure, and helping pharma take full advantage of the opportunities created by the Cures Act. They have created several purpose-specific micro-apps, including CMS OpenPayments Analytics, Data Prep ETL accelerator, Cohort Builder, Rare Disease Patient Finder, and more.

Larry Pickett, Jr., CEO of RxDataScience, says that his company has a fresh approach with differentiating technology for RWD which “simplifies and accelerates the RWE process at a fraction of the time and cost of current technologies.” RxDataScience helps pharma companies address the challenges — and opportunities — posed by RWE. How? As Larry explains, this is done by modernizing their RWD infrastructure with a “faster, more agile capability to link, integrate, manage, and analyze very large data sets.”

Anonymous patient level data (APLD), the backbone of RWD and RWE, presents unique challenges to current tools and technologies due to the sheer volume of data and variety of sources. “By looking at this opportunity in a new way, using new tools and technologies,” says Larry,  “RxDataScience is able to link the disparate RWD data sets and make them available for real-time analysis.”

Learn more about RxDataScience’s product and service offerings