Three Papers Examining The Experience, Value Frameworks And Value-Based Purchasing Of Novel Drugs

Abstract

This dissertations explores real-world experience, value assessment frameworks, and value-based purchasing arrangements for novel medications. The first paper is a systematic literature review of outcomes of these novel therapies, where I show that the real-world experience with these medications�with never-before-seen mechanisms of action or those that address unmet medical needs for serious conditions�have high costs and do not often align with the outcomes reported in clinical trials. The need to increase access to these novel therapies is often hindered by questions about the cost and value these innovative medications. Advocates of value-based purchasing promote it as a potential solution to concerns that drug prices are increasingly misaligned with their therapeutic benefits. However, defining drug value is not easy. The second paper explores seven value-assessment frameworks developed by U.S-based organizations. These frameworks vary by the elements of a medication's value taken into account, how elements are measured and valued, how elements are combined into an overall assessment of a medicine's value and how that could then be linked to the reimbursement price. In response to increasing costs of these novel medications, state Medicaid programs have experimented with a variety of models for value-based purchasing arrangements (VBAs) to contain spending and achieve value in prescription drugs. The third paper is from in-depth, semi-structured, qualitative interviews conducted with representatives from four state Medicaid programs that are currently implementing VBAs with drug manufacturers. While interest in Medicaid VBAs grows and states experience some success, there are challenges with the negotiation process, selecting and monitoring patient outcomes, and concerns with high launch prices persist.