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8th Blueprint for Breakthrough: Validating Real-World Endpoints for an Evolving Regulatory Landscape

8th Blueprint for Breakthrough: Validating Real-World Endpoints for an Evolving Regulatory Landscape

“Real-World Evidence is already here.” – Ned Sharpless, Acting FDA Commissioner

On September 18, 2019, Friends of Cancer Research (Friends) and Alexandria Real Estate Equities, Inc. hosted a meeting to present data from the Real-World Evidence (RWE) Pilot 2.0, building on the Friends RWE Pilot 1.0. The meeting focused on the opportunities and challenges associated with using RWE as advances in data analytics and data capture through electronic health records (EHRs) and medical/pharmacy claims become potential tools for measuring medical product effectiveness. The meeting debuted a whitepaper that was created by a multi-stakeholder working group convened by Friends and Alexandria and can be found here. For a full list of panelists from the meeting, please click here.

The meeting began with a conversation between Acting FDA Commissioner, Ned Sharpless MD, and Friends’ Founder and Chairperson, Ellen Sigal PhD, during which Dr. Sharpless discussed the need for FDA to update its infrastructure to improve workflows within the agency and interactions with industry. Dr. Sharpless’ remarks emphasized the importance of effective information storage and handling systems and a focus on collaboration within the agency to be “better ready to use the tsunami of data that is coming.” He further emphasized that FDA is excited about the future use of real-world data and is determining the most meaningful way to operationalize the information, referencing its current use to support label expansions. To see the full morning keynote, click here.

“Real-world endpoints can serve as important post-marketing research tools. They may also help describe long-term value for oncology drugs.” – Jeff Allen

Following the morning keynote, Jeff Allen PhD, President and CEO of Friends, presented the initial findings of RWE Pilot 2.0. The use of a common methodology and analysis framework enabled 10 healthcare research organizations to extract similar patient populations with advanced non-small cell lung cancer (aNSCLC) across 10 data sets. However, unique characteristics of patients were identified from the different datasets, which may lead to variations in outcomes that exists amongst real-world patients as observed in the preliminary findings. Data from this pilot also demonstrated interesting trends in clinical care and the possibility of providing insights into therapeutic effectiveness in broader patient populations not enrolled in clinical trials. Further refinement of project protocols and more exhaustive analysis of the data collected from this pilot project will be conducted over the coming months. To access the presentation slides, click here.

“It’s remarkable how 10 “frenemy” organizations, that are typically competing, came together to create common definitions to help advance the field.” – Wendy Rubinstein, ASCO-CancerLinQ

Next, Panels 1 and 2 discussed the primary RWE 2.0 project and future analyses to assess the internal consistency of real-world endpoints within the project, respectively. Each of the collaborating organizations were represented across the first two Panels. Donna Rivera from SEER-Medicare, moderated panel 1 and led the panelists through an informative dialogue about the preliminary results of the pilot. The panel discussed processes to handle challenges associated with the availability and consistency of data across provider types and settings, and these differences may require tailored methods and definitions depending on the underlying patient population. Panelists also discussed variability due to missing data or data not captured during a clinical care visit that could impact the interpretation of RWE. It was noted that collaborative efforts, such as the RWE 2.0 project, can ultimately help improve the format, quality, and validity of RWE. To access the Panel 1 slides, click here.

Panel 2, moderated by Nicholas Robert MD of McKesson, explored the concept of internal consistency of real-world data as a control for real-world outcomes measures. Several of the panelists noted that it is likely RWE will differ from clinical trials, but RWE can help understand the impact of clinical care once a drug is on the market. Because RWE will have biases, statistical tools need to be utilized to improve the usefulness of the data. The panelists discussed the challenges with aligning real-world data elements with clinical trial inclusion and exclusion criteria to identify “clinical trial eligible” real-world patients for comparative analyses to clinical trials. This may help further assess the sensitivity of real-world endpoints and further elucidate sources of variability.

The lunch keynote was a conversation between Deputy Commissioner of the FDA Amy Abernethy MD, PhD, and Lynne Zydowsky of Alexandria Real Estate Equities, Inc. Dr. Abernethy announced the FDA’s Technology Modernization Action Plan (TMAP) and talked about her vision for and intent of the TMAP. She emphasized that this has been a priority for her since joining the agency and addressed what she sees as the most important steps to standardizing and maximizing the utility of RWE. To see the full lunch keynote, click HERE.

“We need to raise the question of how we can work together to help physicians gather real-world data.” – Kimberly Blackwell, Eli Lilly and Company

Last, the meeting closed with a provocative conversation from members of Panel 3 on the future of RWE informing regulatory and payer decisions, moderated by Kate Rawson of Prevision Policy. The panelists thought about the direction of healthcare and the role that RWE will play in advancing drug development and patient care. It was noted that RWE has a role throughout the life cycle of a drug. The panel agreed that the most obvious use of RWE is in informing value-based payment arrangements and that equally important to the ability to learn about drug efficacy is to learn about how it is used in daily clinical practice. These data can help guide therapy selection and provide critical information for patients trying to learn more about long-term efficacy and impact on daily living.

Several next steps are currently underway to assess potential difference in the population characteristics, data source, and methodological variations that could impact outcomes. Subsequent analyses will assess the treatment effect across frontline therapy regimens for aNSCLC using real-world endpoints and further evaluate sources of variability.


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