FDA releases draft of patient-focused drug development guidance document

Regulatory Update: Patient Engagement in Drug Development

BY Rosamund Round, Director, Patient Innovation Center, Parexel - 10.9.19 -

This month, the FDA released a draft of its second patient-focused drug development (PFDD) guidance document, detailing best practices for understanding patient experiences and the related burdens of disease and treatment.   The document focuses primarily on selecting and deploying the most effective methodology and offers a clear overview of qualitative, quantitative and mixed method approaches, along with the pros and cons of each.

Firstly, qualitative methods are explored, detailing a number of approaches but focusing primarily on patient and caregiver interviews, which should be conducted sequentially until concept saturation is achieved, and focus groups, ideally 5-10 participants in size.  In both instances, the Agency stresses the importance of framing questions without bias and avoiding leading questions in order to achieve clear and accurate information from participants.

The guidance document lays out numerous quantitative methods as well, including on either self- or interviewer-administered surveys. No preference is given to the medium through which this occurs, but mention of paper, phone, video conference and online/via social media are all mentioned.

Interestingly, the Agency specifically highlights trial screening and exit interviews, stating that they can enhance understanding not only of patient perspectives on treatment benefit and harm but also on the burden of disease and treatment.  Such information could prove incredibly useful during protocol design to ensure that the most appropriate endpoints are selected to accurately reflect what is of greatest benefit to patients.  In addition, potential study challenges can be proactively identified and mitigated for, enhancing the patient experience as well as recruitment and retention in the study.

Finally, the benefits of a mixed method design are highlighted, including elements such as enabling triangulation of results from different sources, expanding results of a research question, and discovering new perspectives.

In my own research exploring patient experiences, I have favoured a mixed method as I find it beneficial when sharing results more broadly to be able to quantify the result (e.g. 75% of patients would decline for their child to participate in the trial due to high visit burden) and then explain the rationale from qualitative feedback (e.g. the main barriers to participation caused by the high visit burden were lack of childcare for their other children and an unwillingness or inability to disrupt their child’s education for the study duration).  This fuller picture tends to facilitate change as more complete understanding of the issues at hand are possible.

Patient Engagement via Social Media

The guidance document also devoted a significant section on leveraging social media to deploy quantitative, qualitative, or mixed methods.  Further, the document reflects the FDA’s positive inclination towards acceptance of this approach to eliciting patient feedback. Guidance is clear and pragmatic:

  • Consider which social media platform or community is most appropriate to answer the research question, due to differences in participant groups e.g. strong advocacy presence, capturing academic perspectives.
  • The degree of anonymity afforded to community participants may impact user willingness to share views and experiences.  However, this needs to be balanced with the ability to verify patient diagnosis and other characteristics.  Where patients prefer to remain anonymous, data analysis should reflect this limitation.
  • Feedback from a range of social media networks and communities will enable collection of data that is more likely to be applicable to the broader patient population.

A More Inclusive Approach to Surveys

It was also incredibly encouraging to see the FDA’s focus on an inclusive approach to survey usage, with guidance stating that materials should be created at a level appropriate for the literacy and numeracy levels of the patient group, provided in their local language, and avoiding clinical terminology that might be challenging to understand.

User-testing of survey tools by patients before being administered more broadly is also recommended, as well as providing the tool in a format that is clear and simple to use.  Continuing the theme of inclusivity was guidance for research with specific populations (rare diseases, cognitively impaired, children) and culturally diverse patients.

Some highly empathetic and practical considerations are detailed, such as considering the health status of participants, for example whether fatigue could make long surveys challenging, how to create surveys that accommodate the limited attention span of certain populations, and the benefit of remote assessment if patients are geographically dispersed.  The importance of considering the emotional burden of participation, changing perspectives as disease progresses, best practices for inclusion of caregivers, and the value of accurate translations are also all mentioned.

Future guidance will cover methods to measure impact in clinical trials and the collection and analysis of clinical outcome assessment (COA) data for regulatory decision-making, so the current document is not intended to be all-encompassing.  However, there were a couple of omissions.  For example, guidance seemed to focus on accessing expert patients on social media when many lay patients may use such online communities as a source of information and support, particularly soon after diagnosis.

There was also no mention of the LGBTQ+ community under “specific populations” and the inadvertent exclusion or marginalization of these patients can not only be distressing or frustrating for them but may mean that an important source of information for improving clinical research is not accessed and, therefore, not wholly representative of the burden of disease and treatment.

However, the release of the guidance itself should positively impact more widespread adoption of engaging patients in clinical research planning and conduct, though it could be useful to highlight to stakeholders under which circumstances in particular the FDA advocates for this approach.

Aside from screening and exit surveys there was mention of using data to understand what is important to patients to aid selection of COAs and to generate patient preference data.  By highlighting other key areas of the drug development process that the FDA view as important to incorporate the patient perspective, such as during protocol design, could act as a best practice checklist for industry that has potential to facilitate real change.  



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We are always available for a conversation.


Communication Preference

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