Patient Involvement in the Development and Use of Safe Medicines Introductory Section – Concepts to Consider CIOMS XI WG Second Meeting October 23-24, 2018 Theresa Mullin CIOMS XI Work Group 1

CIOMS XI WG Report Introduction –For Discussion The following concepts are offered as a “strawman” proposal for what might be included in the Introduction section of the report Proposed goals for the Introduction section Address “why” this topic is so important—why engaging patients and gaining their perspective is so important to medicines development and safe use Examples of opportunities to significantly add value to drug development and safe use through patient engagement Overview of topics covered in the report to enable and achieve this value-added

1. Why this topic is so important—concepts for discussion Patients’ perspective and value-added of inclusion is both unique and critical Patients have direct extensive experience with living with the disease; they know first hand what the chief burdens are and impact on their life, how they feel and function. Patients will be the only ones to directly experience any benefits that may result from treatment and be the only ones to experience the harms Patient likewise best understand the burdens and limitations associated with currently available medicines They will be expected to manage in large part their own adherence to therapy and how feasible that will be based on practical considerations and tolerability. They will be responsible to a large degree for safely using medicines based on the labeling and other information they are provided. They are often a primary source for recognizing and reporting serious and unexpected effects.

2. Examples of opportunities –concepts for discussion Looking across the life cycle of medicines, from Discovery through Post-market surveillance and risk management, opportunities for gaining the benefit of patients’ perspectives and expertise include but are not limited to: Understanding therapeutic context for medicines development and evaluation (e.g., severity of condition and degree of unmet medical need) Development of endpoints that reflect benefits and risks that matter most to patients Aspects of clinical trial design that impact patient access, enrollment, tolerability, retention, and satisfaction Informing regulatory decision making including content of labeling and effective risk management Value measurement for Health Technology Assessment and payment policy Pharmacovigilance strategy and operations

2. Examples of opportunities –concepts for discussion (cont) “Patient engagement” may suggest a passive or reactive role on the part of patients and this is one component But patient advocacy groups are also becoming better organized and informed to position themselves in more active roles and are taking proactive steps to engage in work including but not limited to: Disease registries Natural history studies Studies to describe therapeutic context (e.g., patient-focused meetings) Surveys of patients’ perspectives on specific benefit –risk tradeoffs that might be posed by a new medicine, and other topics relevant to sponsor or regulatory or health authority decision making

3. Topics covered in the report to enable and achieve this value-added Purpose of the document is to describe pragmatic approaches for involvement of patients in the development and safe use of medicines. Topics to be addressed include Review of the landscaped of current patient initiatives Patient involvement during drug development Patient involvement in pharmacovigilance Patient involvement in benefit risk assessment Rules of engagement Key stakeholders Future directions