By Julie Krommenhoek

Today, real-world Healthcare data can be used to evaluate potential drug recipients, drug efficacy in population subsets, and other important testing mechanisms to speed up the clinical trial process. This trend is setting the foundation for more organizations to ask the FDA to accept real-world data in place of or in conjunction with sterilized clinical reviews.

Real World Data Would Expedite Healthcare Advances

Lawmakers got it right with the Twenty First Century Cures Act by being on the front of legislation which enables the FDA to evaluate how real-world data can aid in the evaluation of drug safety and efficacy.

The shared goal of Congress, the FDA, and pharmaceutical companies to bring lifesaving treatments to broader populations more quickly is still elusive. While the Cures Act empowers the FDA to allow for drug approvals based on real-world evidence, the administrative burden is only now being embraced by policy makers as they work closely with researchers to establish when and how real-world data is appropriate.

Researchers already recognize the tremendous value that real-world data can provide.  Increased speed to market, increased recruitment and retention by leveraging virtual control arms, and the ability to cut across broader and deeper sets of the population through big data, are all use cases being pursued today. The speed and advancement of how real-world data is being used also creates opportunity and complexity as the FDA continues to get its arms around policy.

Real World Data is Not New to the FDA

When it comes to the development of new drugs, the FDA’s Center for Drug Evaluation and Research (CDER) governs all progress in oncological medicine. The FDA provides scientific and regulatory advice and certifies new pharmaceutical therapies. Each year, CDER approves new drugs and biological products, some classified as new molecular entities (“NMEs”) for purposes of FDA review. As the testing of drugs in the treatment pipeline continues to advance, those numbers likely will grow year-over-year in 2019 and beyond. Policy makers, now more than ever, need to take a bold and progressive stand on real-world data.

Early FDA approvals using real-world data have already occurred. For example, Amgen Science reports that they successfully leveraged real-world data for a Phase 2 oncology approval.

In this example and many others like it, researchers are asking:

  • Is real-world data available able to meet evidentiary rules and guidelines?
  • Is real-world data available for statistically significant sample size?
  • How can real-world data be traced and normalized to meet rules and guidelines?

There is a joint interest between the FDA and industry partners in establishing clear and compelling guidelines on RWD. To this end, the December 2018 release of a new framework for RWE, while still only providing cursory guidance has a promising future because it extends an olive branch to industry experts in three ways:

  1. Demonstration Projects – This critical opportunity allows industry to partner with the FDA on new and novel RWE/RWD based trials to allow for key insights into the final policy that is formed.
  2. Engagement with Stakeholders – Particularly when looking at new types of RWE/RWD, the FDA has indicated they would like input from stakeholders outside the organization to inform their decision making.
  3. Development of Guidance Documents – The FDA acknowledges that more documentation and feedback is needed to formalize the Agency’s view on RWE/RWD. The continued commitment of the Agency to not only shape policy, but to drive meaningful outcomes, is demonstrated through their intentionality and engagement with industry stakeholders.

Expanding Industry Engagement

Engaging industry stakeholders is a bold move in the right direction but determining the right way to do that is another challenge. One key opportunity for the FDA is to allow the researchers to be in the ‘design seat’ of any aggregated data set. Researchers should still be the voice in charge of which data is required, aggregated and digitized. However, the data they use in studies around the country could come from real patients, real doctors, seeking real results and outcomes. More test medicines could be made available to those in need. More lives could be saved, and critical life-saving drugs could reach market faster with a switch to real-world data.

To enable this, health data organizations have advanced technology to curate and refine the dataset criteria, alongside researchers, on a trial by trial basis, to develop a longitudinal view of patients and to help establish direction of causality. Health data companies can partner directly with life science organizations to execute vital clinical data-based analyses by quickly and securely sharing the right health information about the right patients, at the right time.

Such curation exercises require significant scientific, clinical, financial and technology resources for each life sciences organization. The FDA is in a unique position to work with health data companies to define standards and best practices for capturing and curating a unified research-friendly dataset.

As trends continue toward bringing better treatments to market faster, and as the FDA continues to loosen restrictions on life-saving endeavors to bring trials to patient populations, data collection and management is poised for positive change. Health data organizations stand ready to better serve large volumes of clinical data, irrespective of its operational variabilities, and to enable seamless data exchange and compatible data analytics.



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