Using Individual Experiences With Experimental Medications to Predict Medication-Taking Behavior Postauthorization

From humanistic, clinical, and economic perspectives, it is important to understand patients’ health care attitudes and behavior. Of particular interest in defining the value of a pharmacologic therapy is medication adherence. A DIA workstream was convened to define medication adherence in a drug-development context, explore the relevance of medication nonadherence from various stakeholder perspectives, examine methods of collecting medication adherence data in, or alongside, drug-development programs, and propose a robust approach to predicting medication adherence in routine clinical practice from data derived in, or alongside, drug-development programs. This article summarizes the workstream findings and guidance as it pertains to these objectives. The proposed approach to predicting medication adherence involves a patient-reported outcome (PRO) questionnaire that contains a series of standardized questions for patient self-completion that asks about likelihood of medication adherence in clinical practice, and that queries about perceived benefits and barriers to adherence on exit from an efficacy trial. This methodology can be used to both gain a better understanding of the experimental medication and compare the experimental treatment to comparator therapy as warranted. This approach may assist regulators and payers in making meaningful treatment comparisons and facilitate manufacturers in developing empirically based patient-support programs. This workstream will now turn to the challenge of developing the PRO questionnaire in line with this proposal.