3 Use Cases for Real World Evidence in Pharma Drug Development

September 10, 2021 - Real world evidence (RWE) in pharmaceutical drug development analyzes real world data, such as electronic health records and data from wearable devices, to complement clinical trials data.

RWE provides insights that trials can’t, including safety and effectiveness details from a patient’s daily life. FDA defines RWE as “the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real-world data.” 

RWE helps researchers understand how patient characteristics and behaviors affect health outcomes, which helps to predict the progression of a disease and a patient’s response to a vaccine or therapy.

As the use of RWE continues to expand, pharmaceutical industry professionals can see key use cases in the vaccine development process, the testing of medications, and the design of digital therapeutics. 

Vaccines

The evolving analytics techniques to generate RWE have allowed the research and medical communities to boost clinical research.

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RWE addresses questions about a vaccine that may remain unanswered after being studied in a clinical trial. 

Top healthcare companies had an increased desire to expand the use of RWE beyond traditional safety-related applications because of the urgency to develop vaccines and medications during the COVID-19 pandemic. 

In March, a CDC study used RWE to find that mRNA COVID-19 vaccines effectively prevent SARS-CoV-2 infections among the healthcare personnel, first responders, and other essential workers who got the shots in the vaccine rollout’s first wave.

The study looked at the two FDA-approved COVID-19 vaccines, the first from Pfizer and BioNTech and the second from Moderna, in 3,950 study participants over 13 weeks between December 2020 and March 2021. 

Researchers found that the vaccines reduced the risk infection by nearly 80 percent two or more weeks after the first dose and by 90 percent two or more weeks after the second dose.  

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Meanwhile, a recent RWE trial found that AstraZeneca’s COVID-19 vaccine was 92 percent effective against severe disease or hospitalization due to the Delta variant and 86 percent effective against the Alpha variant. 

The vaccine was highly effective against the virus across all disease severities, demographics, ethnicities, and adult age groups.

RWE also showed the effectiveness of the COVID-19 vaccine against death, hospitalization, and transmission in tens of millions of vaccinated people globally.

“Vaccine effectiveness studies provide growing evidence that mRNA COVID-19 vaccines protect as well in real-world conditions as they have in clinical trial settings. These studies show that the vaccines reduce the risk of COVID-19, especially severe illness, among people who are fully vaccinated,” a CDC spokesperson said in a press release.

Medications

But RWE was in use before the pandemic, as pharmaceutical researchers sought to understand how medications and other pharmaceutical therapies affected patients in real life.

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Novartis has focused on reimagining how integrated RWE can transform healthcare decision-making and address development gaps throughout the years. The company has launched RWE clinical trials studying various treatments, including CAR-T cell therapy, Kymriah, and its psoriasis treatment, Cosentyx.

In September 2018, multiple real-world sources confirmed that Cosentyx improved quality of life in a real-world setting for 59 percent of patients at 24 weeks. Eighty-sevent percent of psoriasis patients remained on Cosentyx for at least 12 months. 

Additionally, 85.8 percent of individuals treated with the drug remained on treatment. 

And in December 2019, Novartis released results from two analyses of RWE with Kymriah, the only CAR-T cell therapy approved in two distinct indications.

In the study, patients who received Kymriah saw an overall response rate of 58 percent, while 40 percent achieved a complete response. 

Along with Novartis, Eli Lilly & Company and Pfizer have continuously leveraged RWE to understand the impact of certain drugs in a target population. 

A June 2020 RWE study found that Eli Lilly & Company’s Trulicity had significantly higher patient adherence and longer persistence than weekly injections of semaglutide or exenatide in individuals with type 2 diabetes. 

About 59.7 percent of those taking Trulicity adhered to the medication versus 42.7 percent of those taking semaglutide. And individuals saw 143.6 days of persistent use of Trulicity versus 129.9 days for semaglutide.

And in March 2021, Pfizer announced the peer-reviewed publication of RWE, demonstrating that first-line therapy with Ibrance, combined with letrozole, improved real-world progression-free survival and overall survival in women with metastatic breast cancer. 

These findings represent the first comprehensive comparative effectiveness analysis of survival outcomes for a CDK 4/6 inhibitor in routine clinical practice.

Other top pharmaceutical companies that have initiated RWE studies for medications include Johnson & Johnson for its pulmonary arterial hypertension drug and Gilead for its large B-cell lymphoma drug.

Merck also launched a RWE evidence trial for two drugs from its diabetes portfolio, Januvia and Steglatro.

Digital Therapeutics 

Digital therapeutics present potential solutions for chronic diseases, ranging from Alzheimer’s disease, type 2 diabetes, congestive heart failure, and cancer. In the new era of healthcare digitalization, there is a notable connection between digital health and RWE. 

In November 2018, FDA unveiled an open-source mHealth app, MyStudies, to foster the collection of RWE via patients’ mobile devices. 

The agency stated that connected health platforms will improve the development of new mobile health technologies by giving developers and researchers a direct link to the patients using the technology.

“There are a lot of new ways that we can use real world evidence to help inform regulatory decisions around medical products as the collection of this data gets more widespread and reliable,” former FDA Commissioner Scott Gottlieb, MD, said in a press release. 

“Better capture of real world data, collected from a variety of sources, has the potential to make our new drug development process more efficient, improve safety and help lower the cost of product development,” Gottlieb continued. 

A recent real-world study evaluated a prescription digital therapeutic (PDT) and associated outcomes of opioid use in patients with opioid use disorder (OUD) treated with buprenorphine medication. PDTs are software-based disease treatments evaluated for safety and effectiveness in randomized clinical trials and authorized by FDA.

Researchers observed patients who received a 12-week prescription for Pear Therapeutics and Northwell Health’s reSET-O PDT. 

In the study, 91 percent of patients met the responder definition of urine drug screening (UDS) negative.

Overall, reSET-O was readily and broadly used by patients with OUD. And high real-world engagement with the therapeutic was positively associated with abstinence and retention in treatment.

The future of RWE in digital therapeutics is promising. RWE allows real-time analysis and inclusion of specific patient information, which can ultimately provide higher quality care for patients.