Overcoming the Limitations of Clinical Trials with Real-World Patient Data

Due to the use of newer and more sophisticated technologies, the cost associated with developing drugs is steadily rising. The trend of medication personalization is also growing, which means the investments that go into research have to be paid for by fewer individuals, further increasing the cost of drugs.

Life science organizations are dealing with unprecedented cost pressures. Further, due to an unsustainable increase in healthcare costs, payers have become skeptical about approving reimbursements. They require tangible evidence to include new drugs in the list of approved medication.

The need for real-world data (RWD)

Real-world data can empower life science organizations to respond to these challenges. RWD consists of real patient data from electronic health records (EHRs), patient registries, studies, surveys, and data from smartphones and wearable devices. It can help accurately assess the safety and efficacy of new drugs during clinical trials, thereby improving the chances of success of the drug and return on investment in the research and development.

By facilitating data analysis of the real-world use of drugs, RWD can generate real-world evidence (RWE). Access to substantial RWE can accelerate the process of getting approvals from health plans and reduce the time for the new drug to reach the marketplace. On average, it takes around 10 years for a drug to reach the marketplace from the time it was initially created.

Leveraging real-world data to support randomized controlled trials (RCT)

In RCTs, a closely defined patient population is administered treatments to assess its long and short-term effectiveness and consequences (if any) in controlled conditions. The purpose of the trial is to determine the unbiased outcomes of new treatments. So, RCTs have historically played a key role in regulatory decision making. Data from RCTs are leveraged in registering the medication, expanding a product label and more. However, a trial performed on a homogeneous patient population does not provide an accurate representation of how the medication will perform on people with different conditions.

The use of RWD and RWE will ultimately make trials more effective. Data that is inclusive of all demographics and represents real patients health status can determine how a drug will affect people with different health and social conditions. Clinical trials often, with strict inclusion and exclusion criteria, attain affected outcomes. Real-world settings are different from clinical trial settings, and the outcomes of drugs tested in simulated settings can be significantly different.

Factors to consider while accessing and leveraging real-world data

There are countless benefits to leveraging real patient data. However, certain factors need consideration before leveraging the data.

• Life science organizations need to develop pharma-provider collaboration in order to access real patient data. Providers would have to be incentivized adequately to participate in data-driven research for drug development.

• Different providers have different patient identifiers and coding standards. It can be challenging to extract and link the data, which implies that generating RWE from that data would be even more complicated.

• The provider database has to be expansive and deep. A big data set is not enough to generate accurate outcomes. It should be inclusive of patient data with different health and social status.

• Provider coding can also be inaccurate at times due to reimbursement incentives. Such anomalies can introduce unnecessary bias in the trial.

The road ahead

Life science organizations must identify new opportunities to innovate and improve operational efficiencies, clinical outcomes, and patient experience. They should leverage RWD and RWE to drive new approaches to deliver high-value insights into their research and development and commercial efforts. Overcoming persistent challenges to innovate faster, collaborating internally and externally, demonstrating the full value of their pharmaceutical portfolios, and delivering personalized solutions to meet patient demands with faster iteration cycles can help organizations attain a competitive edge.