Value of Real-World Evidence in Improving Patient Outcomes in Oncology

Specialty Pharmacy TimesJanuary/February
Volume 9
Issue 1

Real-world evidence is becoming increasingly important for the development of treatment strategies in oncology.

Clinical trials play a critical role in evaluating the safety and efficacy of drugs, medical devices, and other products before they are made available to patients. However, clinical trials can take many years to complete before the results become available to apply to patient care.

Observational studies using real-world evidence (RWE) provide significant opportunities to gain insight into treatment patterns and outcomes in clinical practice outside randomized controlled trials, as patients enrolled in clinical trials may be in better health and less diverse than the general patient population.

Expanding Role of RWE

RWE is becoming increasingly important for the development of treatment strategies in oncology. In its simplest form, RWE is health care information collected outside formal clinical trials, including electronic health records (EHRs), billing databases, and product and disease registries. The integration of evidence from real-world utilization with data from clinical trials holds the promise of providing information on larger populations of patients with cancer to understand disease epidemiology, pharmacoeconomics, and patient experience.

The FDA issued guidance in August 2016 with its “Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices,” in which it describes the circumstances in which RWE may be used to support a variety of FDA regulatory decisions.1 The 21st Century Cures Act, approved by Congress in December 2016, requires the FDA to develop a framework and guidance for evaluating RWE in the context of drug regulation to support the approval of new drugs and the approval of new indications for previously approved drugs and to support post-approval study requirements.2

In what may be a preview of this pending guidance, 15 FDA officials, including FDA Commissioner Robert Califf, published an editorial about the potential uses of RWE in the New England Journal of Medicine titled “Real-World Evidence—What Is It and What Can It Tell Us?”3

Opportunities and Benefits of Real-World Evidence

To understand the treatment of oncology patients, it is critical to evaluate clinical data points (biomarkers, disease staging, location of metastases, disease progression, etc) as well as elements of the patient experience (adherence rates, reason for treatment discontinuation, toxicities, functional status, etc). RWE also provides the opportunity to better understand rare disease types in smaller populations, in which clinical trials may be difficult to perform and evidence is sparse.

The US Oncology Network is a group of more than 60 independent community oncology practices comprising over 1400 physicians treating more than 900,000 patients per year. Many practices use McKesson Specialty Health’s iKnowMed EHR, which captures outpatient medical histories from community oncology practices across 19 states. Through use of a shared EHR platform, physicians are able to perform research in collaboration with pharmaceutical companies to answer important scientific questions that are relevant for drug development and help improve the outcomes of patients. Here are 3 main ways that the medical community can use RWE to help improve health outcomes.

Efficient Drug Development for Unmet Needs

RWE complements research studies and clinical trials, delivering insight into the use of drugs and treatment patterns in daily practice. This ultimately provides a more comprehensive view of patient outcomes to medications and a better understanding of disease patterns that may not be identified in smaller samples. These patterns can reveal additional indications for medications already in widespread use.

This is a particular area of opportunity in oncology, in which treatment decisions are affected by the fact that there are many forms of disease, diseases with short survival times, and many rare conditions that have a lack of evidence, no approved treatment, or only conditional approvals. With the ability to analyze large data sets, investigators can use RWE to conduct postapproval studies to discover patterns that may not be visible in smaller sample sizes. This is critical to help support new indications for medicines and improve patient care.

One such condition is metastatic Merkel cell carcinoma (mMCC). An uncommon, aggressive skin cancer, mMCC has one of the highest mortality rates across all skin cancer types, with an overall survival rate of only 40%. Fewer than half of patients who receive the diagnosis survive more than a year. Despite incidence of mMCC dramatically increasing over the past 20 years, development of an evidence-based standard therapeutic regimen was lagging. As a result, treatment for mMCC centered on a therapy primarily used for patients with small cell lung cancer.

In a study performed through The US Oncology Network using McKesson Specialty Health’s iKnowMed oncology EHR, a substantial patient population who received a diagnosis of mMCC and were treated for it was identified. By using iKnowMed to identify an mMCC patient population, investigators were able to conduct a study of historical treatment therapy responses, to help interpret the outcomes of single-arm clinical trials, and help establish an FDA-approved first-line therapy for mMCC. Through iKnowMed, 686 patients with mMCC were identified, and one-third of these patients had metastatic disease. After applying eligibility criteria similar to that of the clinical trial being conducted in mMCC, 67 patients were deemed eligible for the RWE study.4

Real-world retrospective analyses of patients with distant mMCC who received first-line or second-line and later chemotherapy indicated that although responses were observed with chemotherapy, the duration of such responses was brief and associated with poor overall outcomes in patients with mMCC. These results underscored the need for novel therapeutic approaches. With initial evidence suggesting that immune checkpoint inhibitors had the potential to dramatically improve treatment outcomes, results were submitted. Within a year of the study’s conclusion, the FDA granted approval of a new first-line therapy for mMCC.5

“The pharmaceutical industry is increasingly realizing that observational studies using real-world data have promise to efficiently support various phases of the therapeutic innovation process,” said Jennifer Frytak, executive director of health economics and outcomes research development at McKesson Specialty Health. “This approval of a first-line therapy for Merkel cell carcinoma is a tremendous example of how real-world data provided essential information that was needed to support this successful FDA submission, which permitted this drug to become available to patients in need of treatment.”

Informed Decision Makers

Payers and other health care professionals often have to make decisions about access, coverage, reimbursement, and formulary placement for treatments for which data are limited. Offering insight into how a drug performs in real-world clinical settings, RWE can help provide a more comprehensive view of patient response to medications, a better understanding of disease patterns, and improved patient safety information and economic analyses.

Clinical trials play a critical role in evaluating the safety and efficacy of drugs; however, there is rarely comparative effectiveness data available at the time of approval. That is, what is the gap between efficacy and effectiveness? With the ability to track more patients over a longer period of time, RWE plays an important role in capturing evidence to demonstrate how a drug may affect patients and health care costs in real-world settings by helping to answer the following questions:

  • How does one drug perform versus another in terms of treating a particular condition or subset of patients?
  • How does one drug perform against previously approved medications?
  • How does the drug perform when patients have multiple conditions with multiple medications?
  • Which environmental factors affect the effectiveness of the treatment?
  • Are there drug interactions that affect performance or safety?
  • What are the observed benefits of the medication as it is prescribed and used by doctors and patients in real clinical settings?

The Centers for Medicare & Medicaid Innovation’s Oncology Care Model pilot, which began in July 2016, is an alternative payment model designed to test whether specific changes in cancer care delivery can result in “better care, smarter spending and healthier people.”6 As new reimbursement models evolve with a focus on outcomes and affordability, the use of EHR data enables the retrospective assessment of known findings in the context of real clinical practice settings, adding new insights at a large scale. For example, payers and manufacturers are currently evaluating novel pricing and reimbursement models based on real-world evidence. RWE may also help improve formulary placement decisions based on longer-term medical cost and outcomes data.

“Real-world evidence represents a meaningful step toward personalized health care, as it helps pinpoint patient characteristics that can be used to improve medicine’s ability to meet the needs of individual patients. With more comprehensive data about the real clinical performance of a medication, we can make more informed decisions about access and formulary inclusion, which will help improve patient outcomes, especially when it comes to chronic or rare disease states,” said Nicholas Robert, MD, medical oncologist with The US Oncology Network and medical director for heath informatics and health economics and outcomes research at McKesson Specialty Health.

Quality Data: Safety and Outcomes

Collecting posttrial information about a drug in real time, RWE provides insights into utilization and treatment patterns, patient adherence, and disease management opportunities that can improve patient care and support quality-improvement programs.

Following FDA approval of a new drug, the postapproval monitoring process occurs. The growing use of expedited reviews for FDA approvals increases the need for longer-term postmarketing safety studies to identify adverse events, thereby limiting potential harm. Traditional adverse event reporting from clinicians, patients, and manufacturers remains the primary source of safety information. RWE provides considerable opportunity to improve post-market surveillance activities through other methods of data collection. RWE also provides the opportunity to examine longer-term outcomes among populations in which conditional approvals may exist.

The use of oral oncolytics is rapidly expanding, giving patients promising new treatment options. In 2015, 9 oral cancer drugs were approved by the FDA,7 and of the 836 cancer medications and vaccines in clinical development, approximately one-quarter are oral agents.8

While oral oncolytics offer significant convenience for patients, the transition of treatment from the physician office reduces the ability of clinicians to track how patients are taking the medications. Research shows that nonadherence rates with oral oncolytics are more than 20%.9 Analyses of RWE— including pharmacy and administrative claims data—may help identify potential problem areas and enable pharmacists, manufacturers, and providers to support adherence and positive outcomes by offering education, resources, and behavioral coaching.

RWE can also help identify whether eligible patient populations are getting access to the treatment with specific targeted therapies. Postlaunch studies can improve understanding of prescriber preferences for treatment options available in treatment guidelines or FDA-approved labels, as well as provide real-world insights into trends regarding physician adoption, sequencing, or education issues that may affect adoption.

Conclusion: The Importance of Real-World Evidence

To understand the treatment of oncology patients, it is critical to evaluate clinical data points and elements of the patient experience. In addition to more efficient drug development and commercialization for the pharmaceutical industry, the generation of RWE from comprehensive EHR databases provides value in improving patient care through better understanding of real-world treatment patterns, utilization, and patient safety. Ultimately, the ability to quickly gather evidence can help improve health outcomes for patients through better understanding of the effectiveness of medications in the real world.


  • US Food and Drug Administration. Use of real-world evidence to support regulatory decision-making for medical devices: guidance for industry and Food and Drug Administration staff. Published August 31, 2017.

  • Hills B, Zegarelli B. 21st Century Cures Act requires FDA to expand the role of real world evidence. Health Law & Policy Matters. Published December 19, 2016.
  • Sherman, RE, Anderson SA, Dal Pan GJ, et al. Real-world evidence —what is it and what can it tell us? New Eng J of Med. 2016;375(23):2293-2297. doi: 10.1056/NEJMsb1609216.
  • Kaufman HL, Russell J, Hamid O, et al. Avelumab in patients with chemotherapy-refractory metastatic Merkel cell carcinoma: a multicenter, single-group, open-label, phase 2 trial. Lancet Oncol. 2016;17(10):1374-1385. doi: 10.1016/S1470-2045(16)30364-3.
  • Avelumab (Bavencio). US Food and Drug Administration website. Updated May 30, 2017.
  • Oncology Care Model. US Centers for Medicare & Medicaid Services Updated November 14, 2017.
  • Egerton, N. In-office dispensing of oral oncolytics: a continuity of care and cost mitigation model for cancer patients. Am J Manag Care®. Published March 18, 2016.
  • Ibid
  • Brown, MT, Bussell, JK. Medication adherence: WHO cares? Mayo Clin Proc. 2011;86(4):304-314. doi: 10.4065/mcp.2010.0575.

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