Patient-Focused Drug Development: A Systematic Approach
If you’ve been following us for a while, you may have noticed that our blog and Twitter content often points out that clinical research involves a partnership among many groups of people: sponsor companies (such as Lilly), study coordinators and principal investigators at clinical research sites, ethical review boards, and, of course, patients. The image we created on Lilly TrialGuide to illustrate the relationship among these various groups is meant to subtly show that the patient is at the center of clinical research.
While health care providers may be experts on the clinical aspects of a given medical condition, patients are experts on what it is like to live with their condition. When we are developing a potential new treatment in a specific disease, we need to understand several key points:
- What does it mean to the patient to have this disease or condition?
- What do patients experience with this disease or condition?
- What would a successful treatment look like from the patient’s perspective?
Patients have a unique viewpoint that can provide insights on the outcomes that matter most to them.
- Severity and frequency of symptoms
- Impact on daily function and daily life
This approach is aligned with upcoming guidance from the U.S. FDA on Patient-Focused Drug Development, or PFDD. PFDD is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into the development and evaluation of medical products, at all stages along the way.
For example, we often use patient questionnaires in our clinical research studies to collect information from the patient’s perspective. These patient questionnaires are one form of patient-reported outcomes, or PROs. A PRO is defined as “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else." Understanding the patient’s viewpoint through measures like PROs can be just as important as data we collect through things like laboratory samples (such as blood, urine, stool, or tissue) and imaging results (such as CT, MRI, or PET scans).
Patients are experts on what it is like to live with their condition, and we are making it a priority to listen to them. Drug development is at its best when we include the patient perspective throughout the process.