Evidence inventory: A patient-centered framework for Centers for Medicare & Medicaid Services to assess the clinical benefit of drugs to inform its maximum fair price negotiation.

ABSTRACT

The Inflation Reduction Act passed in August 2022 empowers the Centers for Medicare & Medicaid Services (CMS) to negotiate maximum fair prices for certain expensive drugs. In the process of determining maximum fair prices, CMS gathers evidence on the clinical benefits of these drugs as compared with their therapeutic alternative(s) from various sources. As patients are the primary beneficiaries of the treatment, we recommend that CMS should embrace an approach that prioritizes patient experience when evaluating such diverse sources of evidence in the assessment of the clinical benefit. Thus, we propose to draw on several existing frameworks to support the concept of "evidence inventory," a patient-centered approach to systematically evaluate benefits of drugs under consideration. This 4-step process to develop an evidence inventory includes the following (1) Formulate the research question-in a PICO(T) (P = population, I = Intervention or exposure, C = comparator, O = outcome, and T = time frame) format, (2) Synthesize evidence, (3) Evaluate the evidence-using evidence inventory, and (4) Reevaluate evidence as new information become available. Patients and other relevant stakeholders play a critical role in each of these 4 steps. The proposed evidence inventory holds the potential to provide a structured and transparent patient-centered framework for evaluating the clinical benefits of drugs as compared with their therapeutic alternative(s) informing CMS maximum fair price negotiation.