Innovation and Role of Alternative Markers of Efficacy - Episode 1
The various stakeholders in oncology care, including providers, payers, the FDA, the pharmaceutical industry, and patients, share the overarching goal of improving care and outcomes, as well as advancing science. At the same time, many of these stakeholders also have market-driven goals or, in the case of the FDA, a regulatory mission.
The FDA, for example, has made great strides in moving the science forward by utilizing its Breakthrough Therapy designation, which means that the agency will expedite the development and review of a drug thus designated. Still, the FDA retains its primary role, which is to approve therapies based on trial data that can demonstrate a treatment’s efficacy and safety.
Patients with cancer are learning to be very keen assessors of value. Many must pay out of pocket for some of their care, though the drug cost is often covered for patients in clinical trials or expanded access programs. However, once a drug is approved, cost often shifts to the patient. At this point, patients may start asking, “How long do I need to take the drug?” In many cases, there is no answer. Thus, it is the individual’s choice to pay the cost and remain on the agent or stop and risk a recurrence.
Dr Ira Klein and Dr Richard Joseph discuss the issue of off-label use of approved therapies. For example, could or should pembrolizumab, which is approved as a second-line therapy for patients with melanoma who failed ipilimumab, be used in the frontline setting for this disease? There are clinical trials examining its use in treatment-naïve patients with metastatic melanoma, but the question remains, for pembrolizumab and other agents: should the paucity of data completely curtail off-label use?
Watch our related Peer Exchange, Oncology Stakeholder Summit 2014: Evidence-Based Decisions to Improve Quality and Regulate Costs