National Comprehensive Cancer Network

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Data Needs: Assessing Guidance, Endpoints, and Patient-Reported Outcomes in Oncology Clinical Care

Jake B. Guinto, PhD, Manager, Oncology Drugs & Biologics

Oncology stakeholders rely on data to support clinical decision-making, and in today's environment, there is a greater demand for more data and evidence to support oncology clinical use as a result of concerns about the safety, efficacy, and quality of oncology therapies as well as the rising costs of health care. To better understand data needs among oncology stakeholders, the National Comprehensive Cancer Network® (NCCN®) conducted an NCCN Trends™ Survey from March 14-17, 2012 at the NCCN 17th Annual Conference in Hollywood, FL. The survey consisted of a variety of oncology stakeholders (n=52), including physicians, nurses, pharmacists, and others.

The first question focused on the U.S. Food and Drug Administration (FDA) and clinical endpoints. Specifically, we asked respondents whether they thought the FDA was providing enough guidance and recommendations regarding endpoints for clinical trials to support effectiveness claims in new drug applications, biologics license applications, or supplemental applications. As shown in Figure 1, results were mixed. Thirty-seven percent of survey respondents agreed or strongly agreed that the FDA was providing enough guidance and recommendations versus 31% who disagreed or strongly disagreed. Thirty-three percent did not agree or disagree.

Since clinical endpoints determine an oncology agent's effectiveness, we asked survey respondents what they considered an acceptable primary-efficacy endpoint when assessing the benefit of an oncology agent. According to the results, 73% of survey respondents found overall survival as an acceptable primary-efficacy endpoint; 62% thought progression-free survival was acceptable (Figure 2). Respondents also selected a number of other clinical endpoints as being an acceptable primary-efficacy endpoint, including objective response rate, complete response, and disease-free survival (Figure 2).

Next, we assessed the importance of patient-reported outcomes (PRO) when deciding to use a drug or biologic. As shown in Figure 3, 71% of respondents viewed PROs as moderately important to very important, while 13% thought PROs were slightly important.

The results of this survey provide a snapshot of the perceptions of oncology clinical data from oncology stakeholders. NCCN plans to dive deeper into data needs in oncology in the summer of 2012 by hosting an invitation-only Policy Summit focusing on how data are utilized in oncology for clinical, regulatory, and coverage decision-making. The decision to use a therapy to treat patients with cancer relies on a number of factors and understanding how data are generated and used in oncology will ultimately lead to better decisions regarding oncology clinical care and more effective, efficient care for patients with cancer.