FDA Offers Methods, Research Practices for Patient Input in Drug Development

October 7, 2019

The FDA issued a 49-page draft guidance on how industry should go about asking patients what’s most important to them about how they are dealing with their disease and its treatment, to help companies better develop new drugs.

The second in a series of four planned guidances on facilitating a systematic approach towards collecting and using patient and caregiver input, the draft goes into detail on various research methods and notes how those methods can help in the development of clinical outcome assessments and the generation of patient preference information.

For example, it offers considerations for the most common qualitative methods used for gathering patient input, including one-on-one interviews and focus groups, as well as how to go about asking effective questions. It also details survey research methods, which are frequently used for collecting quantitative data, and the accompanying instruments used to survey patients.

Additionally, it includes a section on mixed methods — combinations of qualitative and quantitative research approaches — and aspects to consider when using social media to question patients. For example, the guidance points out that “different social media communities appeal to different segments of the population” and the level of anonymity afforded by a community can affect how much a patient is willing to share.

“When possible, social media research should examine a variety of social media networks and communities to obtain data that can be most generalized to the population of interest,” the guidance says.

The guidance also lists special considerations to keep in mind when studying specific populations — children, cognitively impaired patients and patients with rare diseases. For example, a patient’s health status is important to consider, as fatigue due to illness or travel could negatively impact data quality. Similarly, the emotional burden of both the patient (anxieties or discomfort, potential for heightened emotions) and the interviewer (potential for emotional distress during the interview) could affect responses as well.

The agency issued a first draft guidance on this topic in June 2018, which offers sampling methods for researchers to use in collecting patient experience information that can assist in drug development and evaluation (CenterWatch Weekly, June 18, 2018).

Comments on the draft guidance are due by Dec. 30.

Read the full guidance here:

By James Miessler