Importance to FDA

Personal preferences and perspectives influence how patients and consumers make decisions to use medical products, tobacco products, or participate in treatment. FDA encourages medical product sponsors to collect and submit patient and consumer perspectives (e.g., patient preference information (PPI)). The Agency collects, measures and analyzes this information to help inform how it considers benefit‒risk tradeoffs related to medical products for patients with a specific disease or condition, or identifies subpopulations with heterogeneous preferences.

Patients’ direct experience living with a disease and the available treatments, as well as the values, beliefs, attitudes, and cultural factors of consumers, patients, caregivers, and healthcare practitioners play an important role in both regulated industry’s product development and FDA’s regulatory decision-making. The Agency uses qualitative and quantitative social and behavioral data from various sources, such as regulatory submissions, research studies, and other specific outreach programs to inform pre- and postmarket decisions and regulatory policy.

FDA seeks to understand the benefit and risk tradeoffs acceptable to the patients who are using medical products and examines consumer understanding of risk statements regarding tobacco products. This includes consideration of the burden of disease and treatment on patients and their caregivers, identifying patients’ unmet needs related to disease treatment, and in the case of tobacco products, understanding the impact of tobacco product use and exposure to tobacco emissions by non-users.

Examples

FDA engages patients and consumers in the following ways.

• Conducting small, informal, non-regulatory, non-public Patient Listening Sessions, where patients directly share their experiences with a disease or condition with FDA staff. FDA, patients, caregivers, and advocates discuss a variety of topics, including: impact on daily activities, priorities to consider when developing medical products, and aspects related to clinical trial recruitment and participation.
• Conducting public Patient-Focused Drug Development Public Meetings to obtain patients’ perspectives on specific diseases and their currently available treatments from a broader representation of the patient/disease community. These meetings have a systematic format designed to engage patients and elicit their perspectives on two topic areas: (1) the most significant symptoms of their condition and the impact of the condition on daily life; and (2) their current approaches to treatment, as well as questions posed by FDA review staff to better understand patients’ perspectives on their disease, treatments, and clinical trials.
• Collecting perspectives from patients in cancer clinical trials of undesirable and harmful symptoms to help other patients and healthcare professionals evaluate potential side effects of anti-cancer therapies.
• Developing strategies for increasing and improving patient engagement in medical product development. The Patient Engagement Collaborative (PEC) is a partnership between the FDA and the Clinical Trials Transformation Initiative (CTTI). The members of the PEC share their experiences and discuss ways to enhance patient engagement to include patient perspectives in the medical product development process and regulatory discussions at the FDA.
• Collaborating with the Medical Device Innovation Consortium, a public‒private partnership, to explore the impact of patient selection methods (e.g., patients with a confirmed diagnosis from a licensed healthcare professional versus an online panel) on the evaluation of the benefit‒risk tradeoffs that people living with heart failure are willing to make for new heart failure devices.
• Measuring PPI for investigational or novel treatments, such as gene therapy for sickle cell disease and osteoarthritis of the knee, and islet cell transplantation (when insulin-producing pancreas cells from a donor are transferred to a person with diabetes) for hard-to-control type 1 diabetes. PPI data provide FDA with information about patients’ benefit‒risk tradeoff tolerance. This is particularly important for medical products that may offer benefits and risks that may not be well understood due to a small number of patients in clinical trials with limited follow-up time.
• Supporting research studying how risk claims of new tobacco products entering the market affect consumers’ judgments and intended behaviors. The research evaluates how likely nonusers (including youth and other vulnerable populations) would be to start using tobacco products, and how likely current adult users would be to transition to potentially less harmful products or stop using them altogether.
• Preparing a curriculum for teachers to educate middle and high school students regarding dietary supplements. This curriculum includes a broad range of topics impacting consumers including adverse event reporting; live microbials (commonly referred to as “probiotics”); athletic performance enhancement/bodybuilding products; and dietary supplement labeling and advertising.