A new milestone has been reached for the FDA as they embark on the ambitious journey to shift towards a more patient-centered approach to drug development and regulation. Earlier this month, the FDA released its first draft guidance in a new series titled  The Guidance for Enhancing the Incorporation of the Patient’s Voice in Drug Development and Regulatory Decision Making. The new guidance builds upon learnings the FDA acquired through research sessions conducted with patients and caregivers between 2012 to 2017 on more than 20 disease areas including autism, HIV, opioid use disorder, and Parkinson’s disease.

In a June 12, 2018 statement, the FDA commissioner, Scott Gottlieb, stated, “Our work demands that we must continue to reflect on how we can make the science of drug development and review more modern and more patient-centered, so that approved products impact the metrics that real-world patients and families value most.”

The new guidance titled, Patient-Focused Drug Development: Collecting Comprehensive and Representative Input, provides an overview of research best-practices for not only quantitative data but also qualitative data collection including insights on what real-world patients experience during their disease management journey.

Although the first draft guidance has just been released, several biopharmaceutical companies have already started to engage with the FDA and submit patient experience data in their drug approval process. Most notable is Genentech, whose submission of patient experience data to the FDA led to the first inclusion of a clinical trials sub-section titled, “Patient Experience”, on the US label for Rixtuxan Hycela. The data provided goes beyond standard safety and efficacy, detailing the results of a survey on patient preferences around drug administering.

Biopharmaceutical companies who invest in qualitative research early, before the design of a protocol, may benefit from obtaining a holistic view of the patient experience during drug development phases.

Representatives from Genentech have noted that when they submitted the patient experience data to the FDA, they had no idea that the information would make the label.  In this regard, there is still room for clarity on what patient experience data may make a US label.  However, having such information on a label is a method to reach prescribers and payers who trust FDA approved data.

As companies scramble to make sense of the new guidance, many may note how vague the draft is. What is clear, is an interest in using qualitative research methods to capture insights on the patient experience, from the patient’s perspective.

Biopharmaceutical companies who invest in qualitative research early, before the design of a protocol, may benefit from obtaining a holistic view of the patient experience during drug development phases. Utilizing qualitative research methods, such as in-depth patient interviews and workshops, will enable early insights and hypotheses to inform and focus clinical study endpoints.

Leaders looking to benefit from early insights may lean on the expertise of service designers and design researchers who utilize human-centered design practices. Service designers have developed rich toolsets to support a range of qualitative research methods, aligned to the FDA’s guidance. Service designers not only conduct the research but also translate the insights into outputs such as detailed patient experience maps.  Such maps visualize learnings about patients’ emotions, physical feelings, as well as touchpoints over the duration of a disease state and provide insights into opportunity areas. Patient experience maps are valuable tools used to align stakeholders around prioritized opportunities and increase cross-functional coordination.

Often, leaders within pharmaceutical companies choose to participate in these qualitative research workshops to develop a first-hand understanding of the patient experience – much like the FDA has done with their patient groups.  However, the primary benefit of conducting in-house patient experience qualitative research is to identify and prioritize opportunity areas which align to the organization’s pipeline.

Although the FDA still has much work to do to identify what information would be most relevant during their drug decision-making process, they have stated that they are looking for organizations to engage with them to discuss research plans. Biopharmaceutical companies who start incorporating new research methodologies to understand the patient experience may benefit from developing their own internal understanding of best practices and begin testing its use within the drug development process.