Silence Is Not Always Golden: The Value of Engagement for Collection of Patient Experience Data in Drug Development

Speaker(s)

Discussion Leader: Danny Yeh, PhD, AESARA, Burlingame , CA, USA
Discussants: Ebony Dashiell-Aje, PhD, Patient Centered Outcomes Science, BioMarin Pharmaceutical, San Rafael, CA, USA; Natalie Engmann, PhD, Denali Therapeutics, San Francisco, CA, USA; Selena Daniels, PharmD, PhD, Division of Clinical Outcome Assessment, US Food and Drug Administration, Silver Spring, MD, USA

Presentation Documents

PURPOSE:

The workshop aims to discuss practical aspects of investing in and generating patient experience data (PED) for drug development. Experts will discuss challenges, opportunities, and best practices related to engaging regulators in PED generation throughout the drug life cycle. Through interactive discussions, the goal is to inspire collaborative ideas and insights to enhance PED generation in drug development.

DESCRIPTION:

In the patient-focused drug development era, clinical trials are evolving to actively incorporate the collection of PED (information related to patients’ experience of their disease/condition). Various data sources, such as Clinical Outcome Assessments (COAs), Digital Health Technologies (DHTs), and Patient Preference Information, can collect this data. Although PED has the potential to expedite drug development, pharma and biotech firms may struggle with resource allocation to prioritize PED generation and stakeholder engagement at early stages, which can lead to insufficient data or data that fails to meet regulatory requirements. This workshop seeks to highlight the need for early investment in PED measurement strategy development. Discussions will focus on complexities, opportunities, and engagement points with regulators for input on PED collection tools, fostering a more direct path to reliable and valid PED generation.

Dr. Yeh (chair) will present an overview of PED in drug development, highlighting common challenges and opportunities. Dr. Dashiell-Aje will introduce a recently developed Biotechnology Innovation Organization (BIO) FDA-sponsor COA engagement framework, elucidating its purpose, development process, and its practical utility for industry sponsors. Dr. Engmann will share strategies and challenges for early involvement in generating PED from a small biotech perspective. Dr. Daniels will provide the regulatory perspective regarding how regulatory input can be leveraged at critical timepoints in drug development to inform a PED measurement strategy.

The workshop employs live polling for audience input on PED in drug development, leading to an interactive discussion to conclude the session.

Code

305

Topic

Patient-Centered Research