Face-to-face discussion educates regulators on what matters most to patients, and guides future decision-making.
Regulators are increasingly asking drug developers to include the patient voice in submissions, but the best way to go about this is less clear-cut.
In the United States, learning how to navigate forums such as the FDA’s listening sessions and patient-focused drug development (PFDD) meetings could be the key to “truly moving the dial”.
Speaking during a Patients as Partners meeting, Ebony Dashiell-Aje, senior director of patient engagement and outcomes research at BioMarin Pharmaceuticals, said these small, informal, non-regulatory events helped the FDA “better understand what is most important” among specific patient groups.
The rise of patient centricity
“We know the patient voice is at the centre of medical product development,” she told delegates at the online event, Applying Patient Engagement Initiatives and Demonstrating Impact.
“Patients are experts in their own conditions and are, ultimately, the consumers of medical products.
“It’s important that the patient experience and patient voice is appropriately elevated, and that regulators, sponsors, and researchers are meaningfully engaging patient stakeholders in order to define the most high-value areas of impact.”
In recent years, this ethos has been written into the approach of most of the world’s biggest regulators, including the FDA, EMA, and MHRA, she added.
“Regulators now urge sponsors and researchers to systematically engage patients and caregivers to define the burden of disease, the burden of treatments, and unmet needs.” Yet while there has been a lot of progress in understanding which key areas patient stakeholders can and should contribute to, “there has been less focus on the how”.
“Industry and patient advocacy groups have continually requested pragmatic how-to guides on ways to generate information in traditional scientific exchange, but the patient voice is more robust than what is captured in a concept elicitation interview, clinical outcome assessment, or a preference score.
“There are times where roundtable discussions, or virtual or face-to-face meetings with regulators, can take their thinking further.”
Listening sessions and PFDD meetings enable the regulators to gain a better understanding of a particular patient and caregiver community, Dashiell-Aje explained.
“They can be via teleconference or in person, and they allow participants to connect with FDA staff first-hand. It’s about sharing of patient experiences and perspectives, and sharing information about needs that are related to patient health and or to condition in general.
“These meetings give the FDA an opportunity to connect with different patient communities, including underrepresented groups, and can inform regulatory decision making.”
PFDD meetings are organised and led by the patient community, in partnership with consultants or sponsors, who invite an FDA representative to attend.
Dashiell-Aje said these could be “very impactful”, and shared an example of a 2017 PFDD meeting on autism research.
“It was held primarily to gain feedback from the community on the most important metrics in clinical trials, and how to move forward with specific trial endpoints. The patient community was able to speak about what was most important to them, specifically around improvements in communication,” she said.
“Prior to this, sponsors would be seeking to improve stimming, which is a repetitive behaviour sometimes be seen in people with autism. However, they discovered during this meeting that this was not the most important thing to patients.”
In fact, the meeting heard, many people with autism use stimming as a coping mechanism, so reducing it could actually be detrimental.
“This particular meeting impacted division directors’ thinking on how to approach sponsors when proposing primary endpoints. It changed the entire paradigm in the field, and allowed for the better consideration of exactly what aspects of health were most important to improve in this population.”
Here to listen
Listening sessions, which can be initiated by the FDA or by a patient advocacy group (PAG), by submitting a request to the Office of Patient Affairs or the Center for Drug Evaluation and Research (CDER), can have a similar effect.
A 2019 session, on cavernous haemangioma, discussed how the typical clinical trial enrolment criteria focused only on a small portion of the patient population, challenging recruitment.
They also spoke about the experiences of a subset of patients who suffered significant functional neurological deficits, yet were often overlooked in clinical development, and the utility of symptom stability as a primary end point.
“This meeting was very successful, because the FDA showed great interest in moving the field forward, to encourage sponsors to enter this space and try to find treatment options for these patients,” Dashiell-Aje said.
A meeting request, she went on, should include specifics related to the meeting topic and goals, proposed meeting attendees and their affiliations, and discussion questions.
“It should also leave room for FDA to provide input. They are constantly seeing different applications come through, and sometimes will have topics that they want to raise with the community,” Dashiell-Aje said.
She also gave some advice on giving a meeting request the best chance of success, including inviting people with “diverse opinions” and ensuring patients, caregivers, and advocacy groups are all represented.
Ultimately, the patient voice is critical to advancing medical product development – and independently engaging with the FDA is one way to make sure regulators understand it.
“You can inform regulatory decision-making through continued collaboration with researchers, industry sponsors, and consultants. You can elevate the patient voice and educate regulators on what matters most,” Dashiell-Aje concluded.
About the author
Amanda Barrell is a freelance health and medical education journalist, editor and copywriter. She has worked on projects for pharma, charities, and agencies, and has written extensively for patients, HCPs and the public.
This post was originally published on Source Link