BioMarin Generates Patient Experience Data With Early And Often Engagement

A conversation with Ebony Dashiell-Aje, Ph.D., executive director & head, patient centered outcomes science, BioMarin Pharmaceutical, Inc.

Patient engagement is just one of the things that’s never too early to start — particularly as it relates to the generation of patient experience data to inform medical product development and decision-making. In fact, many experts would argue that pharmaceutical companies, as well as sites, should be talking early and often with patients about clinical trials – what they are and what they can do for individual and community health. But getting started — and staying committed — isn’t easy.

In November 2024, the Milken Institute held the Future of Health Summit on “Drafting the Blueprint for the Next Era of Patient Engagement,” where BioMarin’s Executive Director & Head, Patient Centered Outcomes Science, Ebony Dashiell-Aje, Ph.D., participated as an industry panelist. The proceedings from this meeting were released, and Clinical Leader’s Abby Proch chatted with Dr. Dashiell-Aje about topics discussed on the panel, including thoughts on the utility of patient engagement to facilitate patient experience data generation and key learnings from the panel discussion.

Clinical Leader: What is patient engagement, and why is it so important for patient experience data (PED) generation?

Ebony Dashiell-Aje, Ph.D.: Patient engagement is dynamic — it can involve activities like providing input on study design, identifying relevant health outcomes to study, reviewing patient-facing materials, and participating in advisory committees or governance roles. However, in my experience, patient engagement is critical for facilitating the generation of high-quality data that will be used to inform medical product development and decision-making. While the depth and intensity of engagement can vary—from one-time consultations (surveys, focus groups) to continuous partnership (patients as co-investigators or decision-makers), the main goal is to incorporate patient experiences, preferences, and priorities directly into the development and evaluation of new therapies, ensuring that products better meet real-world needs.

Where does patient engagement begin if sponsors want to ensure an informative, high-quality PED is generated?

Patients and caregivers should contribute as true partners at the earliest phases of research, well before planning to go into clinic. Patient engagement work, including the science of patient input to generate patient experience data, should begin as early as the preclinical and early discovery phases of drug development. This is the time when researchers first seek to understand if we have a potential treatment candidate, understand the mode of delivery, the disease process and can begin to identify unmet needs that are critical to inform clinical trial objectives and study endpoint selection. In these earlier phases, patient insights could shape research questions and therapeutic goals.  Early patient and caregiver involvement ensures that the development process aligns with patient priorities and experiences, ultimately driving innovation, improving data reliability and validity, and patient outcomes.

How should sponsors approach patient engagement to better integrate it into the data generation process?

Before beginning any work, sponsors should make sure that internal stakeholders place the same value on patient and caregiver insights and the need for patient experience data generation to achieve program goals. Without the same value, adoption of a systematic, scientifically robust approach to engaging patients and caregivers will be difficult. Once everyone is aligned on the value of patient engagement, the level of resource commitment should also be aligned — personnel, budget, and research objectives will drive the stage appropriate scope of work throughout the product lifecycle.

In what ways are insights and data generated from patients and caregivers most useful?

Patients and caregivers should be viewed as experts in their own/their loved one’s conditions. Insights and data obtained directly from patients and caregivers can provide valuable windows into unmet needs and research gaps which can inform program strategies, end-to-end. At BioMarin, we engage with patient communities early and our Patient Advocacy team continues to engage through the entire preclinical, clinical and commercialization process. Our goal is to ensure the patient perspective is fully integrated and understood by the company’s decision-makers.

Engaging patients and caregivers early enables consideration of their perspectives n study designs, including the incorporation of fit-for-purpose measurement strategies (e.g., clinical outcome assessments, digital health technology incorporation) into study protocols, ensuring companies understand what outcomes are meaningful to patients. However, I would also invite everyone to consider what is the bi-directional benefit of patient engagement and the data we are able to generate from those activities – a feedback loop. What is the added benefit of providing patients and caregivers with information to educate them on treatment options and aid in their decision-making? How do we equip the clinical community with good data that will help inform their decision-making and ultimately help drive the potential for patient access to approved treatments?

What’s stopping sponsors from “baking in” patient engagement into their scientific data generation processes?

Several factors create barriers for sponsors to fully “bake” patient engagement into the patient-centered data generation strategy across the product lifecycle. Barriers could include difficulties with internal buy-in and financial constraints – they can also be logistical, or be created by doubts about utility for regulators, payers, or the medical community.

Without strong champions advocating for its importance, sponsors can struggle to integrate patient engagement into their workflows because it is seen as secondary to quantitative clinical outcomes and real-world evidence generation. Additionally, time and budget pressures often lead to shortcuts, with engagement efforts reduced or omitted entirely to save on resources –metrics and data to support rationale for the work are critical here for driving adoption.

Is industry doing enough?

Many industry leaders are doing a lot, but we can do more.  You have to have a champion to say why this is important, so that meaningful, systematic patient engagement doesn’t get deprioritized. Fostering a patient-inclusive company culture, with senior leadership sponsorship and support, is critical to ensure this work is embedded at the enterprise level.

How can sponsors ensure patient engagement is a long-term commitment to ensure the right data is generated?

First, patient engagement should be viewed as a scientific exercise – a component in driving the science of patient input to generate high-quality patient experience data for each development program. Second, there needs to be a strong strategic plan, mapping key patient engagement activities to study objectives and program goals; without a direct tie to the business, robustness of this work will be ancillary. Lastly, there needs to be a strong infrastructure built upon systematic processes and data generation hubs to allow for the practices to be embedded into the scientific culture – this requires effective and intentional cross-functional partnership, a patient-inclusive company culture, and building professional capabilities (e.g., in a dedicated patient advocacy function) for success. This infrastructure has to be viewed as immovable and status quo and integrated into the core R&D and commercial business strategies.

What did you learn or find in common with other folks on the Milken Institute Panel?

One key takeaway from our conversation that is relevant for today is that as a pharmaceutical or biotech partner, we often forget that patients are our ultimate customers. Why don't we approach them the same way we would approach any type of other product we’d like them to use? You need to know if they want it. You need to figure out what would be best for them and get opinions on whether it is suitable to meet a need. And patients should be viewed as experts in their own condition, not negating their expertise or devaluing it compared to clinical experts or biomarker data. Well-planned patient engagement activities can generate high-quality, usable data that can be leveraged as valuable evidence to inform decision-making.

Another thing I took away from this panel was hope. Despite having a way to go, the industry has champions that are evolving our thinking to a point where I can see the path to better patient engagement integration and prioritization – but perhaps in a different way than we first thought. Buy-in can be hard, but it’ll take speaking the language of the business. It’s time to reframe our thinking about sustainability science and begin to approach this a different way to drive long-lasting adoption.

About The Expert:

Ebony Dashiell-Aje, Ph.D., is a leading expert in patient-centered outcomes research, including clinical outcome assessment (COA) and digital health technology development and optimization, measurement science strategy, and patient engagement across the medical product lifecycle. Currently, she serves as the executive director & head of patient-centered outcomes science at BioMarin. Dr. Dashiell-Aje has also served within the Division of Clinical Outcome Assessment (DCOA) in the Office of New Drugs at the FDA as a scientific lead and consultant to CDER, CBER, and CDRH; she was a key player in shaping patient-focused drug development (PFDD) policy efforts. In addition, she has served as an expert consultant to both pharmaceutical industry and federal government clients. As a health outcomes researcher and methodologist, Dr. Dashiell-Aje is driven by her passion for public health and promoting patient-centered medical product development, commercialization, and clinical care. Her research expertise has significantly contributed to evidence-based solutions and has helped shape health policy in multi-stakeholder environments.