(021) How to improve Patient centricity in Benefit-Risk Assessments (BRA)?

Background: Patients have valuable insights from living with a condition and its treatment. They can provide a unique perspective on symptoms, natural history, quality of life, unmet needs, prioritization of clinical outcomes and preferences for future treatments, that when collected correspond to patient experience data (PED).

Objectives: To better understand how the patient’s perspective can inform medicinal product BRA, further exploration into PED and its use to inform decision-making should be encouraged, as expressed by Health Authorities (HA).

Methods: A literature review was conducted to produce a comprehensive overview of past or ongoing initiatives from HA and public/private partnerships aiming at improving patient centricity in BRAs. To better understand how PED have supported HA decision, regulatory documents relating to recently approved products were also reviewed to identify products for which PED have supported the BRA and approval decision.

Results: Many initiatives worldwide such as ICH M4E(R2) Guideline dated July 2016, EMA’s multistakeholder workshop on “Patient-experience data in medicines development and regulatory decision-making” held in September 2022, the CIOMS XII on benefit-risk balance of medicinal products (public consultation completed in August 2023), and the US FDA – Benefit-Risk Assessment for New Drug and Biologic Products guidance for industry published in October 2023, convey the need to propose a transformation of the perspective in BRA, by providing an opportunity to better understand and describe the benefits and harms of interventions based on patients’ perspective. Another key initiative was the IMI PREFER public private partnership, that developed recommendations on how to assess and use patient preference studies (PPS) in medical product decision-making, including in the regulatory evaluation of medicinal products.

In examples of BRA of recently approved products, PED were used to:
• Describe the therapeutic context and unmet needs from a patient’s perspective,
• Identify benefits and risks relevant to patients,
• Assess the importance of the observed benefits and risks, based on the results of the PPS; and their view towards uncertainties and acceptability of risks,
• Perform quantitative BRA with explicit relative weighing of trade-offs from PPS.

Conclusions: PED is intended to inform decision-making as supplementary information to clinical efficacy and safety evidence. Patient-centered BRA should be conducted all along the medicinal products life’s cycle, to inform decision making. More interactions with Regulators on this topic are required during the drug development program, as well as early planning.