Patient Involvement in the Regulatory Process and Rare Disease Patient Perceptions of Treatment Benefits and Harms

Abstract

Patient involvement in healthcare decision-making is becoming an essential part of healthcare policy in today’s patient centred environment. It supports responsive and transparent healthcare programs and policies that are informed by patients for patients. While regulatory agencies, the bodies responsible for the approval of new medicines, involve patients in the regulatory process, little is known about the involvement context or the type of patients engaged. The purpose of this thesis was to explore patient involvement within the regulatory process and gain insights into rare disease patient perceptions of treatment benefits and harms. It contains three papers. The first comprises a review of proposed and current regulatory patient involvement using the International Association for Public Participation Spectrum. The second paper presents findings from three fora and surveys conducted in three different Canadian cities. The fora were used to elicit treatment harm and benefit attributes and treatment benefit priorities from rare disease patients and caregivers. Surveys were used to gather patients’ and caregivers’ levels of expectations of treatment benefit. Their input highlighted the need for survey questions to be relevant and meaningful to health contexts of the target population. The third paper, informed by the second paper, provides an understanding of harm acceptance while considering increasing levels of treatment benefit in a specific rare disease. It was found that mucopolysaccharidosis patients accept lower levels of harm than caregivers, where caregivers consistently selected the maximum level of harm for maximum treatment benefit. The findings of all three papers demonstrate that patient input around the acceptability of different benefit-harm trade-offs is needed in order to make regulatory decisions more patient-centred.