Overview of Final Patient-Focused Drug Development (PFDD) Guidance 1

By: Maddie Mason, Senior Associate, Policy

On June 16, 2020, the Food and Drug Administration (FDA) released the Final Patient-Focused Drug Development (PFDD) Guidance 1 entitled “Patient-Focused Drug Development: Collecting Comprehensive and Representative Input.” Guidance 1 is the first in a series of four guidance documents that the FDA is developing to direct stakeholders in collecting and submitting information on the patient experience in regulatory decision making and medical product development. The guidance’s finalization represents a historic moment for the FDA, as it formally demonstrates the agency’s commitment to elevating the voice of the patient in medical product research and review.

Guidance 1 reviews the different sampling methods that can be utilized when developing a study that uses patient input. It also gives a broad overview of the relationship between potential research questions and methods for deciding from whom to collect research.

The National Health Council (NHC) worked with our members to develop and provide substantive recommendations to the FDA for their consideration prior to the release of  Draft Guidance 1 and commented on the draft guidance in a letter to the FDA, applauding them for their efforts to encourage meaningful patient engagement. The NHC recommendations covered how draft Guidance 1 could be strengthened and encouraged the FDA to provide more details on how engagement efforts will be operationalized and reviewed by the FDA.

The NHC provided the following general overarching comments for Guidance 1:

1. Articulate that when patient engagement is conducted appropriately, such interaction is not regarded by the agency to be promotion of unapproved products.

• • This comment was not addressed in the final Guidance 1, though other communications by FDA have indicated they will not consider patient engagement activities to be promotional.

2. Incorporate hypothetical case examples into the draft guidance that demonstrate a range of regulatory decisions that may be informed by patient experience data (PED).

• • Some case examples are incorporated, but it still links to the website.

3. Further articulate that the guidance is relevant for all stages of drug development and approval, not just in clinical trials.

• • This concept is more directly stated in the final guidance.

The NHC also provided section specific comments:

In the General Considerations for Collecting Patient Experience Data (PED), the NHC encouraged the FDA to continue to expand the discussion of the methodology and best practices to achieving representativeness in studies. Compared to the first draft, this section is cleaner and more concise, expands upon the explanations of target populations, who will be providing PED, and subgroups. The overall increase in explanation helps create a better picture of representativeness and encourages considering different types of representativeness.

The Methods for Collecting and Analyzing Patient Experience Data section was removed and condensed into the General Considerations section. Previously, this section of Guidance 1 focused heavily on well-established methods that most researchers are familiar with, and the NHC commented that there are other emerging methods to gather or analyze individual patient experiences that can be useful for informing study protocols, informed consent documents, and conceptual models. The NHC believes the removal of this section does not have a significant negative impact and may help streamline the document.

Under the Operationalizing and Standardizing Data Collection and Data Management section, the NHC made a few recommendations. First, we recommended that regarding locating patients and sites, the FDA should suggest that patient groups should assist with enrolling patients to ensure representativeness. This was not included in the final guidance. We also noted that under Collecting Data “Social Media and Identifiable Patient Communities” needed to be added to the Collecting Data list, as this represents a distinct method. This recommendation was incorporated into the final but was modified to: “social media and verified patient communities.” The NHC also suggested that the FDA should include language that patient input can help ensure questionnaires and other materials intended to be used by patients are easier to understand, provide the right level of information, and reflect patient vernacular. However, the entire section on questionnaires was removed from the final guidance.

In the Glossary the NHC recommended that the FDA distinguish the role of the caregiver from the patient, including examining when it appropriate for caregivers to serve as proxy or observer. We suggested that the FDA consider how the relationship between caregiver and patient, and caregiving burden, may impact reporting of data and care delivery. We also encouraged the FDA to acknowledge that Clinical Outcome Assessments (COAs), such as patient-reported outcomes (PROs), in drug research and development should be patient focused. This glossary was not modified in any significant way, but the FDA has indicated that it intends to make modifications during the remainder of the guidance-development process.

Overall, the NHC is very pleased with the Final Guidance 1. It remains very consistent with the NHC’s consensus recommendations and sets a strong foundation for the future guidances. We will continue to work with our members and the FDA as we await the final three guidances.