Results of a unique pilot project was designed to establishing framework to evaluate real-world endpoints in medicine.

Friends of Cancer Research, an organization focusing on the collaboration among partners from every healthcare sector to power advances in science, policy and regulation that speed lifesaving treatments to patients, has released the results of a unique pilot project, The Future Use of Real-World Evidence, designed to establishing framework to evaluate real-world endpoints.

Although the definition of “real-world evidence” or RWE is still evolving, most experts believe that RWE in is generally derived from medical practice among heterogeneous sets of patients in real life practice settings. This data can, for example, be based on insurance claims data and clinical data from electronic health records. Outside clinical trials, the use of RWE is seen as a way to tailor health care decision making more closely to the characteristics of individual patients. In turn, this is seen as a step towards making health care more personalized and effective.

Proponents expected that RWE will not only tap increasing volumes of traditional data, but weave together different sources of data as well. The additional data may include clinical data, genomic data and socioeconomic data. The expectation is that this will ultimately yield a much better picture of individual patient characteristics and improve a physician’s ability to treat individual patient needs.

Long-term impact
In the presented study, data was compiled from six data partners regarding patients receiving approved immune activating drugs. The findings from the data support the use of RWE for assessing the long-term impact of a treatment.

“New sources of data may require new methods of analysis for real-world evidence to be used to its full potential,” said Friends’ President & CEO, Jeff Allen.

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“To explore how endpoints derived from healthcare data relate to more traditional measures, Friends convened a group of partners to design a pre-competitive study to compare how different data sets can be used to evaluate outcomes in lung cancer,” Allen added.

The analysis undertaken by the working group found the following key findings:

  • There is notable correlation between several real-world endpoints and overall survival (OS), indicating that real-world endpoints have the potential for evaluating treatment benefit
  • The range of OS observed in clinical trials used to approve checkpoint inhibitors is highly similar to the range observed in real-world populations, demonstrating that in this case the results from the clinical trial are generalizable to the broader population.
  • Treatment effects assessed by real-world endpoints were consistent across a variety of demographic factors, such as age and sex, although differences were observed across lines of therapy and stage of disease.

The U.S. Food and Drug Administration (FDA) also presented new data at the meeting. Their findings show that based on aggregated clinical trial data, progression free survival is highly correlated with TTD.

These results and findings are available in a whitepaper.

Clinical trials
Clinical trials provide the high quality data necessary to support claims of safety and efficacy for FDA drug approval. However, clinical trials do not necessarily reflect the breadth of data that can be collected by evaluating products in a more diverse real-world setting. While there has been a lot of interest in RWE, parameters and conditions of use have not been well defined and traditional clinical endpoints may be difficult to obtain and evaluate from real-world data making new metrics a potential necessity.

For example, the published report discusses how immunotherapies are being used to treat patients with cancers that have historically had few treatment options. Over the last couple of years, these novel therapies have generated high level of interest. However, while immunotherapies have resulted in significant improvements in some patients, other patients do not respond or only respond for a limited time.?As a result, it’s important to find understand the value of these new drugs.

Unfortunately, as the results sow, current value frameworks to immune checkpoint inhibitors has proved difficult since they tend to underestimate the benefits of long-term survival and treatment-free survival. [1]

This may, in part, be cause by the reliance on pivotal trial data. Furthermore, in the setting of expedited approvals, assessments of the full clinical endpoints have not been completed. As a result, conclusions are often based on surrogate efficacy endpoints.

[1] Ben-Aharon O, Magnezi R, Leshno M, Goldstein DA. Association of Immunotherapy with Durable Survival as Defined by Value Frameworks for Cancer Care. JAMA Oncol. 2018, 4(3):326?332.

Note: Data partners participating in this pilot project include: COTA, Flatiron, IQVIA?, Kaiser Permanente/CRN, Mayo Clinic/OptumLabs?, and PCORnet/University of Iowa.

Last Editorial Review: July 10, 2018

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