For a drug to go to market it must first undergo a clinical trial to test its safety and efficacy to obtain FDA approval. However, this current structure poses a significant limitation: clinical trials offer a limited view of the patient population and how the drug fares post-trial.
To address this, in December 2018 the FDA released a framework for evaluating the potential use of real-world evidence (RWE) to streamline the current model for drug approvals. RWE relies on the analysis of real-world data (RWD), which are patient data collected from non-clinical trial settings, such as electronic health records, claims data, patient registries, and patient-generated data. This framework looks to leverage RWE to support the discovery of new indications for drugs that are already approved and available to patients. If new indications for approved drugs exist or can offer new insight into varying approaches to drug administration, dosage, etc., a company won’t be subject to an additional clinical trial, saving time, money, and resources.
While the FDA is largely focused on RWE for new indications and regulatory decision making, the life sciences industry has a unique and necessary opportunity to harness and advance its potential to demonstrate the long-term value of therapies.
RWE and the Value Equation
Science that has been tested for decades is finally becoming reality for patients in need. New, breakthrough treatments and one-time cures are coming down the pipeline and striving to gain market access, but many come with a high price tag. Industry is faced with the perpetual challenge of demonstrating the value – and therefore justifying the price – of these drugs, which ultimately determines the level of access for patients. That is where RWE comes in.
By using RWE to collect patient reported outcomes and help determine costs avoided, industry can better demonstrate the long-term value a drug has on patient lives. For example, a breakthrough drug could cost $100,000, but if it saves a patient from a $500,000 organ transplant, and the associated costs of additional medical procedures and hospital stays, that drug can actually be a major source of cost savings and of immense value to the patient, the overall healthcare system, and society.
The success of RWE in demonstrating value rests in the hands of the industry, which should invest in its capabilities beyond using it for new indications. As we move towards this new era of innovation, there are several companies that are currently doing this to different degrees by either building their own tools to collect RWE and RWD or taking advantage of existing platforms. NY-based Flatiron is a good example of a company that’s currently building the tools and the network for industry to leverage RWE on a greater scale.
Real World Evidence in the Real World
Flatiron Health was founded with a focus on oncology and a goal to revolutionize the way patients are treated by harnessing RWE. In 2016, only 4 percent of the adult US cancer population was enrolled in clinical trials, forcing providers to make treatment decisions based on a very limited view of the disease population. Flatiron seeks to address this gap by aggregating all of the data on patients not included in clinical trials, organizing it, and making it available to physicians so they can truly gauge the impact of therapies in the real world and improve patient care.
Furthering this mission, Flatiron partnered with the FDA to research how different treatment options, based on patient data captured outside of clinical trials, can provide new insights into the safety and efficacy of cancer therapies. Through this partnership, Flatiron wanted to use RWE to deliver more accurate insights into expected treatment outcomes, thus enabling patients and providers to understand the long-term value of different treatment options.
Flatiron and Beyond
Today, Flatiron has over 2.2 million active patient records collected through clinics, medical centers, and hospitals and has since expanded its partnership with the FDA. Through this expansion, the company will continue its work validating clinical data, improving regulatory decision making, and developing real-world control arms (so patients in a clinical trial don’t have to fear receiving the placebo instead of treatment). The company will also work with the FDA to evaluate cancer populations underrepresented in clinical trials to best understand how all patients respond to certain treatments.
Through its vast network of electronic health records and patient reported outcomes, Flatiron has developed the tools for other industry players to use and advance RWE. In partnering with the FDA, many companies have made significant steps towards leveraging RWE to understand the real-world impact of therapies on the broader population – beyond clinical trials. However, there is still much to be done to specifically use this data to measure costs avoided and improved outcomes – the crucial determinants of value.
As the debate around drug pricing intensifies, RWE will help provide the data we need to demonstrate the full value of a drug – value that is based on improved quality of life and costs avoided, not an arbitrary number set by stakeholders outside of the industry. It is our responsibility to ensure access for patients, so they can get the medications they need and the treatment they deserve, without unnecessary restrictions. RWE can help take us there.
To learn more about real-world evidence and how it can help the industry address the drug pricing debate, register to attend Part I of our three part series exploring the value of health: How to Define the Value of Prescription Drugs to Ensure Patient Access.