The patient’s voice WILL be heard in drug development: draft FDA guidance on collecting patient experience data

Written by Angela Rylands on Thursday 21st June 2018

The US Food and Drug Administration (FDA) officially acknowledge a deeper appreciation for the expertise that patients and caregivers can bring to the drug development process. According to new draft guidance, capturing the patient voice, in drug development, can be achieved by gathering ‘patient experience‘ data, using innovative methodologies, including quantitative and qualitative data collection.

Of course, in some ways this news is old news, as we have been referring to the 2009 guidance for incorporating Patient Reported Outcome (PRO) measures for labelling claims for some time already. However, these new (65 page) guidelines focus on patient experience data and are the first of a four-part series of guidance documents for patient-focused drug development, aiming to provide up to date and innovative methodological recommendations for HOW to collect and submit information gathered from patients and caregivers as part of regulatory submissions. This guidance is not just about using PRO data for labelling claims, but the focus is on methodological considerations, for example, WHO (e.g. patient, caregiver) to target to gather this critical information.

Of particular interest to us at pH Associates Patient Centred Outcomes, is the information incorporated within the guidelines on qualitative and quantitative research methods for analysing patient experience data, as well as the information around how to operationalise and standardise data collection and management. To our knowledge, this is the first time the FDA guidance documents have provided such a detailed description, particularly with a focus on qualitative data analysis methods. The level of detail provided demonstrates that the authorities do indeed value innovative and varied methods of data acquisition as part of regulatory submissions.

At pH Associates Patient Centred Outcomes, we work with pharmaceutical industry clients and healthcare providers to design qualitative and quantitative research studies and ensure optimal methods are used to capture the patient experience across the product life-cycle. With our in-house team of experts, we carry out research studies, often in real world settings, with patients and caregivers experiencing a wide range of diseases. We also provide strategic input to our clients throughout this process and advise on the technical implementation of such strategies.

For these draft guidelines, we intend to harness the details within, but more importantly, we acknowledge that this not only reaffirms the work that we are already doing with our clients, but they further advocate the use of innovative methods for capturing the patient voice in the drug development process, and this is certainly something that the wider Patient Centred Outcomes research community is fully behind.

Next, will be a series of the guidelines that will focus on technical aspects on the HOW to collect this information from patients and caregivers e.g. how best to measure patient centred outcomes in clinical trials. Watch this space.

If you would like to get in touch to discuss your patient centred data needs, please don’t hesitate to make contact Angela.

+44 (0)1628 481112