Which disease components are the heaviest for patients? How do researchers find this information?
To answer this question, the U.S. Food and Drug Administration (FDA) recently issued a second guidance document Patient-Centered Drug Development: Identifying What Matters for Patients. Some of the qualitative methods they recommend for gathering this information include:
one-on-one interview Provides the opportunity to explore topics in depth at a personal level using probing questions.
- method: There are three different types of interview methods: structured interview (interviewer asks a set of predetermined questions), semi-structured interview (interviewer asks a predetermined set of questions and probing questions), unstructured interview (interviewer asks a predetermined set of questions and probing questions) officer asks unplanned or spontaneous questions).
- patient population: Patients recruited for these interviews should be broadly representative of the patient population of interest, and a sufficient sample size should be collected to obtain a broad view of the disease.
- administrative: The interview method should be pilot tested before full rollout. Interviewers should consider the pros and cons of remote versus in-person interviews. Interviewers should be experienced and/or trained. Interviewers should be aware of the emotional burden these questions may place on respondents.
- Advantage: Interviews help gain a deeper personal experience; deal with sensitive topics; and explore diseases or conditions with many different symptoms that vary from patient to patient
- limit.
special group Involves a conversation with a group of participants led by a moderator. The most common type is a single focus group, where a group of participants and moderators work as a group to discuss a topic interactively in one place. Typically, the moderator uses a semi-structured discussion guide to guide group conversations.
- number of focus groups. This may depend on (i) the complexity of the subject (eg, the full or partial impact of the disease or condition on multiple dimensions of the patient’s quality of life, depending on the therapeutic area and research question, (ii) the sensitivity of the subject (eg, sexual Gender segregation on sensitive topics such as function, (iii) diversity of the participant sample, (iv) number of subgroups planned.
- focus group size. The goal is to make the group large enough to allow enough diversity of opinions, while remaining small enough so that all participants can weigh in. A sample size of 5 to 10 participants per focus group is most common.
- Moderator. As with interviews, the host (or interviewer) should be well trained and have expertise in the subject area.
- Advantage: allows participants to interact; allows a range of viewpoints to be captured in a shorter period of time (compared to one-on-one interviews).
People also have to decide whether qualitative research should be conducted in person, over the phone or via videoconferencing. The table below outlines some of the advantages and disadvantages of each method.
The FDA guidance also provides some (advanced) recommendations on quantitative surveys and mixed methods approaches, as well as a discussion on the use of social media for passive and active data collection.
