On March 19, the U.S. Food and Drug Administration (FDA) held its second public meeting to obtain input on the Patient-Focused Drug Development Guidance currently under development by the Agency. Evidera had representation from our Patient-Centered Research team in attendance to hear and contribute to the discussion. The topic of this meeting was specific to how external stakeholders can develop and submit proposed draft guidance relating to patient experience data to the Agency, and the meeting was attended by a wide variety of patient stakeholder groups. Key highlights and discussion points of interest from the meeting are noted below.
1. Potential Types of Patient Experience Data of Interest to the FDA
Theresa M. Mullin, PhD, Associate Director for Strategic Initiatives, Center for Drug Evaluation and Research at the FDA opened the meeting with a panel discussion on the types of patient experience data the FDA might find useful in medical product development and regulatory decision-making, which are outlined below.
Patient experience with their disease, including what disease impacts matter most to patients, challenges with adhering to prescribed treatment regimens, attitudes toward or tolerance of risk or side effects, how the patient experience varies by subgroups, and the use of natural history studies as comparator data in platform trials.
Patient-defined endpoints, including patient-reported outcomes that measure patient-defined key symptoms and impacts, as well as other types of endpoints that are considered meaningful and important to the overall value of a medical product from the patients’ perspective.
Acceptable trade-offs to benefits, noting that acceptable trade-offs may vary in different settings (e.g., point-of-care vs. clinical trial) and by different subgroups (e.g., older vs. younger patients, patients with vs. without comorbidities).
Trial design and dissemination, such as options to make trials more accessible to patients (e.g. in-home study visits and data collection, patient concierge services), and to ensure that trial results are summarized and shared with study participants and the respective patient communities.
2. Pilot Repository for Patient Experience Materials
The FDA has launched a pilot ‘repository’ for patient experience data and references. The goal is to encourage sharing of resources related to patient experience data, many of which would not be available through peer-reviewed, published content. Over time this has the potential to become an important resource of publicly available surveys, interviews, focus groups, and workshops that provide insight into the patient experience with various diseases.
External stakeholders can submit content by emailing [email protected]. This includes study reports, hyperlinks, survey results, etc.
Content will need to be reviewed by the FDA before it is posted, ensuring its relevance to the intended scope of the repository. It is important to note that approval to post in the repository is not an endorsement of that content by the FDA.
3. Patient Medication Information
A rule has been proposed by the FDA that would require companies to submit a one-page document for patients that highlights the most important information about a prescription drug. This proposal is currently open for comment, and it is expected that the FDA will make a ruling on this soon. More details on this proposal can be found here.
This document is intended to be in addition to the package insert, enabling consumers to easily access the most important information about the prescription drug they are taking.
Each one page document must be consumer tested.
4. Patient Stakeholder Perspective
The meeting included a panel discussion of invited patient stakeholder representatives, and encouraged open dialogue and feedback from patient stakeholder representatives in attendance.
The resounding message from stakeholder groups was the need for clearly defined channels for communicating and sharing information with the FDA.
Developing a draft guidance is one important channel, but may not always be the best way forward for all patient stakeholders. For this specific guidance on “how to develop a patient experience guidance”, the Agency was encouraged to consider different channels for communicating and sharing information with the FDA in addition to the development of draft guidance.
We were excited to see Theresa Mullins delineate potential approaches to the use of patient experience data in the future. The concept of a repository for content not necessarily available through other channels is also encouraging, and a positive step towards expanding access to patient experience data. Although a clear road map for how the FDA intends to use patient experience data in regulatory decision-making is yet to be defined, each public meeting provides additional clarity and direction for stakeholders.
How Can Evidera Help You Prepare for These Changes?
Capture patients’ experience with existing treatments, unmet needs, and key impacts of their disease through patient surveys, interviews, focus groups, and workshops in a format suitable for submission to the FDA.
Quantify patients’ experience with their disease in a real-world setting with epidemiological and burden of illness studies.
Understand patients’ experience with treatments by using an evidence-based approach to the design and implementation of clinical trial endpoints, such as patient-reported outcomes.
Understand the patient experience in the clinical trial setting by conducting exit studies that qualify the patient experience with the treatment, inform patient-defined meaningful change, and provide insights to improve future trial design.
Design patient-centric trials, including innovative designs to bring the trial to the patient, keep patients engaged throughout the study; and obtain patient input on protocol and endpoint design.
Identify patients’ acceptable trade-offs to benefits through patient preference studies, including minimum acceptable benefit to offset treatment risks; the maximum acceptable risk to justify treatment benefits; willingness to pay; probability that a patient selects or adheres to a particular treatment.
Prepare and test patient information documents with skilled medical writing and healthcare communication specialists and patient-centered researchers who can work together seamlessly to write and consumer test patient-focused treatment information.
Understand payers’ perspectives on patient experiences and patient-centered data to appropriately plan for pricing and reimbursement challenges in various global markets.
The next meeting on this proposed guidance will be in the fall of 2018, with subsequent meetings occurring over the next two years and a draft guidance expected in 2020. There is still a lot of work that needs to happen to truly accomplish the goal of including the patients’ voice and experience into the drug development and access process, but we are encouraged by the movement we have seen towards that goal.