For many biopharmaceutical companies, the future of drug development lives in the real world

Health Care Current | July 10, 2018

This weekly series explores breaking news and developments in the US health care industry, examines key issues facing life sciences and health care companies, and provides updates and insights on policy, regulatory, and legislative changes.

My Take

For many biopharmaceutical companies, the future of drug development lives in the real world

By Greg Reh, Vice Chairman, US and Global Life Sciences Leader, Deloitte LLP

Many biopharmaceutical companies are putting greater emphasis on real-world evidence (RWE) to address regulatory requirements, drive the development of new therapies, and support value-based contracts, according to the results of our second annual RWE benchmarking survey.

These results, combined with examples of how the industry is starting to use RWE, help to reinforce the value of RWE and real-world data (RWD) in the delivery of more personalized, value-driven medicine.

One area in which we are seeing a lot of momentum is in clinical trials. Of the 1,800 clinical trials worldwide that involve RWE or RWD, half of them began within the last three years, and 300 were initiated in 2017, according to a recent Reuters analysis of the National Institutes of Health’s website. Historically, biopharma firms have relied primarily on randomized controlled trials (RCTs) to develop and test new therapies. While RCTs remain a critical component in this process, RWE sometimes offers deeper insight into how a drug or a class of drugs actually performs in the real world.

Most surveyed biopharma firms are investing in RWE

We surveyed 20 leading biopharmaceutical companies and found that nearly all of them (90 percent) have either established or are investing in RWE capabilities for use across the entire lifecycle of their products. However, we also found that only half of these companies have capabilities that are mature enough to take full advantage of RWE.

The use of RWE across key strategic business priorities, including the drug-approval process, is only going to grow in importance. The Food and Drug Administration (FDA) has been helping to move RWE forward: under both the 21st Century Cures Act and the Prescription Drug User Fee Act VI, FDA has set timetables to release guidance for the use of RWE. The 21st Century Cures Act directs FDA to establish a program to evaluate the expanded use of RWE in making regulatory decisions. During a National Academy of Sciences workshop last fall, FDA Commissioner Scott Gottlieb, M.D., said that advancing the adoption of RWE is a top priority for the agency. “As the breadth and reliability of RWE increases, so do the opportunities for FDA to also make use of this information,” he told attendees.

Three emerging RWE trends among biopharmaceutical firms

Here are a few of the interesting RWE trends that turned up in our survey results:

  1. Biopharma firms are moving data analysis in-house: The majority of respondents (70 percent) said they are either building or increasing capabilities to conduct more of their RWE studies internally. Over the next year, 10 respondents said they would increase spending on talent by 30 percent, and eight respondents said they would increase spending on technology by 25 percent. Hiring experts to build and implement RWE-enabling systems—such as machine learning systems—could be the prime and immediate focus.
  2. Partnerships to source non-traditional data could become a priority: Health information generated by apps, wearables, and other non-traditional sources are being eyed by more biopharma companies. RWD generated from these sources could give biopharma companies a new perspective into the progression of diseases, treatment pathways, and patient outcomes. Although less than 60 percent of our survey respondents say they now use these sources, some intend to boost their reliance over the next 12 to 18 months. These data will most likely be accessed through strategic partnerships. The majority of respondents expect that strategic data partnerships could have a significant impact on the future data landscape.
  3. Machine learning and artificial intelligence (AI) could become invaluable: The combination of machine learning, AI, and RWE could help biopharma companies produce powerful data that help them understand outcomes and improve care. Although just 60 percent of our respondents said they now use machine learning to analyze health data, nearly every respondent expects to use such technology in the future. As the volume and variety of structured and unstructured RWD grow, AI and machine learning will likely be needed to take full advantage of the information.

Are health plans and health systems keeping up?

While biopharma companies recognize the potential of RWD and RWE, many of our respondents suggested that some external stakeholders aren’t yet on board. Three-quarters of biopharma companies said some health plans and health systems haven’t yet figured out a way to use RWE, which could slow down their efforts to use the data to its potential. Biopharma firms will likely want to work with these stakeholders to break down some of these barriers. One way is to create more transparent communication by giving stakeholders better access to data.

In just the past year, the volume and variety of RWD being generated have continued to expand, according to our survey results. As the technology platforms, talent, and maturity of the tools needed to manage and derive insights from the data continue to evolve, along with the regulatory environment, I expect to see many of the barriers start to diminish. It is clear that RWE and RWD are critical to the transformation of health care, and I am excited to see their application and usage evolve.


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Breaking Boundaries

Innovations in prenatal and newborn care can lead to healthier mothers and babies

Preterm birth is a leading cause of newborn death, as well as later health problems for infants born too early. Researchers at Stanford University have developed a blood test to detect fetal RNA (genetic material) circulating in the mother’s bloodstream. This test can identify mothers who might be at risk for preterm delivery. A similar test can predict a baby’s due date more accurately than the standard method, which is based on a woman recalling her last menstrual period, combined with an ultrasound.

Though preterm births (defined as being born before 37 weeks gestation) in the US declined from 2007 to 2014—due in part to declining teen pregnancy rates—preterm births rose in 2015 and 2016. According to public health officials, about 10 percent of babies were born before 37 weeks in 2016. The rate of preterm births is higher in the US than in other developed countries.

A separate study, published in the May issue of the Journal of Perinatology, profiles a test that can identify a potential complication during the second trimester of a pregnancy. Researchers identified a biomarker that, when combined with the mother’s age and poverty status, can determine increased risk for preeclampsia. Preeclampsia is a complication associated with high blood pressure that might also damage the liver or kidneys. Left untreated, preeclampsia can cause serious or fatal complications to the woman and the fetus. The researchers noted that this test could help improve outcomes for high-risk women and babies in the most at-risk populations, including low-income women, women of color, and women living in low-income countries.

RELATED: Developers have been working on innovations for pregnant women who are not at high risk for complications. A startup called Babyscripts developed a software app to reduce the number of doctor visits for healthy women with low-risk pregnancies. Reducing the number of visits needed for healthy women could give physicians more time to focus on higher-risk patients. The app allows pregnant women to have virtual visits from their home or office. The company sells the app to physician offices and health systems.

The ob-gyn can enroll patients in the Babyscripts program during the first weeks of pregnancy. A kit shipped to the home allows the patient to take her blood pressure and send the data to the physician. Patients can also test their blood sugar if necessary. Patients still come in for prenatal visits at different points in the pregnancy, but some might be able to cut their visits in half. Since most ob-gyns receive a one-time, bundled payment for about 14 visits for each pregnant patient, this program can lead to savings for physicians who can manage low-risk women remotely for some of the visits.

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