CDER official reflects on a decade of patient-focused drug development

Regulatory NewsRegulatory News | 19 September 2022 |  By 

Multistakeholder meetings held as part of US Food and Drug Administration (FDA) patient-focused drug development efforts have left a huge impact on everyone involved, according to Theresa Mullin, PhD, associate director for strategic initiatives within the FDA Center for Drug Evaluation and Research (CDER).
Mullin, who leads the Patient-Focused Drug Development (PFDD) program at FDA, told attendees at a recent webinar hosted by the Alliance for a Stronger FDA that patient-focused drug development (PFDD) is “a systematic approach to help ensure that patients experiences and their perspectives and needs and priorities are captured and meaningfully incorporated into drug development and benefit-risk assessment.” Using a PFDD approach, FDA staff and industry can involve patients and their caregivers in drug development, clinical trials, and regulatory decisions, identifying what outcomes are important to them. It also includes the collection of those data points and how they are communicated to stakeholders.
“The primary goal of PFDD is to better incorporate the patient’s voice in drug development and evaluation,” Mullin said.
FDA’s PFDD program has its origins in the Prescription Drug User Fee Act (PDUFA) reauthorization in 2012 where Congress directed the agency to meet monthly with “patients and consumers and other stakeholders.” Gradually, that evolved into a format where FDA doctors and statisticians as well as other reviewers and prospective drug sponsors listened to patients and their caregivers discuss their experiences in different disease areas.
The original plan was to hold 20 meetings per year in different disease areas, but a total of 24 meetings were held due to increased demand. “The meetings were so informative and powerful, review divisions wanted to do more,” Mullin said.
The meetings have left a huge impact on everyone involved, she explained, and the information is valued and referenced in later patient reports.
“FDA doctors, including ones who continue to see patients in the clinic, come away from these meetings and say that they heard new things that they really never heard from the patients while treating them all these years in the course of these meetings,” Mullin said. “I’ve been told by reviewers that the things they heard from patients in those meetings factored into their decisions in looking at benefits and risks for treatments that they have come to review later.”
The PFDD meetings also have had broader unanticipated impacts such as inspiring colleagues at FDA to incorporate PFDD elements into a progressive multifocal leukoencephalopathy in collaboration with the National Institutes of Health and changing the career path of at least one of the collaborators, Mullin said.
How PFDD has grown
Over the last 10 years, the PFDD program has expanded to allow for more of these types of meetings to be held. In 2016, FDA began letting patient advocacy groups hold externally led PFDD meetings. Since then, patient advocacy groups have held more than 55 such meetings, Mullin said, which are a “very similar format to the FDA meeting, probing the same types of questions about severity and the impact of disease and experience with treatments.” FDA also links out to a detailed meeting report on the agency’s website from these meetings in the same way they would with an FDA-led meeting.
The PFDD program also is developing guidance documents to aid in its expansion, Mullin noted. “We also wanted to further expand the opportunity to incorporate patient perspectives in drug development and decision-making and realizing that if a patient’s perspective is going to get further incorporated in data collection during clinical development, the validity and reliability of the measures and tools would determine the extent to which it could be used in regulatory decision making,” she said.
The guidance documents, described in PDUFA VI and the 21st Century Cures Act, detail how stakeholders can move from PFDD-type meetings to collecting patient and caregiver fit-for-purpose information that could help with decision-making. The first two guidances have been finalized and outline how to appropriately collect patient and caregiver input and determine what aspects of disease burden and treatment are most important to patients. A third guidance document, currently a draft, discusses potential processes for selecting, developing, or modifying clinical outcome assessments. The fourth guidance, which reached the public workshop stage in December 2019, will outline how to take the clinical outcome assessments and incorporate them into endpoints that meet requirements for regulatory decision making. Mullin noted that the draft for the fourth guidance should be published within the next few months.
“We think the information in this PFDD series will really help patients, stakeholders and other researchers to address a number of important and challenging issues,” Mullin said.
Future of PFDD
Mullin said she’d like the next decade of PFDD at FDA to be focused on sustainability, standardizing the collection and integration of patient perspectives. While the guidance series should help, the implementation of the new guidance and processes to ensure quality and reliability of the collected data will be a team effort. Mullin said her team intends to get the word out through training opportunities such as webinars and workshops.
“Patient groups will play an important role in expecting sponsors to use high quality approaches to collect information about their patient experience so that the regulators and other health decision makers can actually rely on the data,” she said. “Patients can and they need to participate in research to develop good measures and tools.”
In particular, the development of a publicly available standard for clinical assessment measures and tools is a priority for sustainability, Mullin said.
“[T]here’s generally little coordination in the development of clinical outcome assessments, including within a given disease area,” she explained. “Reviewers are currently seeing multiple independent efforts and the duplication of effort, and the diversity of the measures and the proprietary tools really can limit the affordability and sustainability of integrating the patient’s perspective.” Since the proprietary tools are of varying quality, the tools themselves and the data they produce are of limited use in regulatory decision-making, she added.
Mullin aims to change this with a grant program to develop a core set of standardized measures. A total of five grant programs are currently being conducted in the areas of migraine, acute pain therapeutics for infants and young children, physical functioning for chronic conditions, nephrotic syndrome, and communication in patients with rare neurodevelopmental disorders.
“Our focus is on specific disease areas or impact within a disease that could apply to multiple other disease areas,” she said. “We’re looking at diseases that are chronic, symptomatic or affect functioning and activities of daily living and disease, areas that represent a broad range in terms of the size of the affected populations, so including both common prevalent conditions as well as rare disease conditions and smaller populations.”
Eventually, Mullin said she envisions a national catalog of standard core clinical outcomes and assessments that would work for every disease area, “publicly available and ideally be built to serve a range of health decision makers.”
“This national catalog could have reliable measures of clinical outcomes that are meaningful to patients, but at the same time they would be measuring disease specific impacts and they could be agnostic to the type of medical intervention,” she said. Mullin acknowledges this is a large undertaking that would involve multiple stakeholders, because it is “so important and affects so many people.”


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