Convergence: Using PREFER as a roadmap for patient preference studies

Regulatory NewsRegulatory News | 12 September 2022 | By

PHOENIX, AZ – Applying patient preference data to the drug development process and regulatory decision-making is an emerging area, but experts stress that it’s important to ask “why” before launching a study.
 
“You have to understand that not every decision can be meaningfully informed by a preference study,” Kristin Bullok, PhD, an advisor on benefit-risk management at Eli Lilly and Company, said during a session on patient preferences in regulatory at Convergence 2022. “You don’t need a preference study for everything.”
 
However, if there is uncertainty about whether patients would accept a particular risk-benefit tradeoff, a preference study might be able to help answer that question, she said. Additionally, patient preference studies are valuable in better understanding or developing patient-relevant endpoints.
 
IMI-PREFER
 
In April 2022, PREFER, a public-private collaborative research project under the umbrella of the Innovative Medicines Initiative, released recommendations on why, when and how to assess and use patient preference information. They also provided information to help guide method selection when conducting patient preference studies.
 
Additionally, the PREFER framework explains how to present the preference data alongside clinical trial data to further inform decision-making, Bullok said.
 
In May 2022, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive qualification opinion “generally” endorsing the PREFER framework and its points to consider on methods selection as a “comprehensive reference document for planning and conducting patient preference studies.”
 
CHMP endorsed the framework as “a good place to start discussions between decision-makers, such as HTA bodies and regulators, as well as patients, for how to design and conduct these preference studies,” Bullok said.
 
But there are still open topics, Bullok said, including how do you know when it is most informative to collect patient preference data and what methods to use in collecting the data.
 
Choosing methods
 
The PREFER framework provides information on methods to consider but does not recommend any one method as being preferred. Instead, the PREFER framework spells out five common, promising methods and provides points to consider when assessing any method. For instance, can the method account for and/or test for sources of preference heterogeneity or does the method directly elicit relative importance or trade-offs?
 
“There is no cookbook approach to which method is going to be most appropriate in which situation,” said Brett Hauber, PhD, a patient preference subject matter expert at Pfizer. “What we want to do is figure out a way to assess the ability of each type of method to answer a question. And then once we have that in place, we can think about which is going to be most fit for purpose.”
 
When choosing a method for patient preference data, it’s essential to do it in consultation with the clinical team as well as the regulators, Hauber said. He advised talking to the regulators and getting early scientific advice about a study design that they will find acceptable.
 
Francesco Pignatti, head of oncology, hematology and diagnostic products at the European Medicines Agency echoed that idea, noting that there is no single, one-size-fits-all method.
 
Patient preference studies can come in a variety of formats, but it is critical that the data is high quality and that it is applicable and complementary to the other clinical data, said Sara Eggers, PhD, director of the decision support and analysis staff at the US Food and Drug Administration’s Center for Drug Evaluation and Research.
 
Regulators need to have confidence in the representativeness of the data, as well as its interpretability and relevance, she said.
 
Challenges
 
While the PREFER recommendations and the CHMP qualitative opinion are a boost to patient preference research, there are still challenges.
 
A need for culture change is part of it, EMA’s Pignatti said, since some people perceive these studies as adding an extra level of bureaucracy to the drug development process. However, he said he thinks the opposite is true, with patient preference data offering the potential for greater flexibility and possible cost savings.
 
Hauber agreed that advancing patient preference data in the drug development process will involve a culture shift. While the interest is there, there is some pushback because of the cost and administrative burden of obtaining patient input, he said.
 
“There is more and more interest in patient preferences as a source of patient input and a source of information, but there is still a lot of concern because it seems so new,” Hauber said.
 
The lack of regulatory experience is another challenge, Pignatti said, but he said regulatory bodies are ready for it. “Bring it on,” he told session attendees.

 

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