IPF Trial Practices Often Exclude Certain Patients, Perspectives

Clinical trials for idiopathic pulmonary fibrosis could benefit from improved patient engagement to include diverse perspectives and inclusive practices.

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Current methods of conducting trials related to idiopathic pulmonary fibrosis (IPF) therapies do not do enough to engage the perspectives of a significant portion of the patient population, according to a new report.

The study, which was based on patient insight reports and interviews with patients and clinicians, suggests drug developers and regulators need to do more to ensure a wide spectrum of patients is included in future research. The study was published in Health Expectations.¹

IPF is considered a rare disease. The global prevalence of IPF ranges from just 7 to 1,650 cases per 100,000 persons, according to a 2023 study.² Those patients currently have access to 2 antifibrotic medications, though the therapies can only reduce—not reverse—disease progression, and some patients do not benefit from the therapies.

If patients want or need different therapeutic approaches, they can access new treatments and pulmonary rehabilitation strategies by participating in clinical trials. Yet, access to clinical trials depends on a number of factors, including awareness of trials and the ability to meet necessary requirements for participation. 

Julia Frost, PhD, of the University of Exeter Medical School, in the United Kingdom, and colleagues, wanted to better understand the perspective of patients who participate in clinical trials of IPF, and learn more about why certain patients do not engage with trials.

Frost and colleagues were given access to international patient insight reports that were used to inform marketing and clinical trial design for IPF therapies. These reports were based on interviews and surveys with patients, caregivers, and physicians. Frost and colleagues also conducted interviews with 32 people with IPF and 19 staff members who support patients with IPF.

The investigators found significant limitations in how participants in the insight reports were chosen, as well as problems with how broad conclusions were drawn. Some reports, for instance, did not indicate why particular patients and staff were chosen, making it difficult to understand how representative the respondents were of the general population.

Other reports seem to make broad generalizations based on small sample sizes. One patient insight report concluded that patients in the UK “reach a cold acceptance with an untimely death,” which the report said translates into less motivation to seek new treatments. However, Frost and colleagues noted that the conclusion was based on interviews with just 2 patients in the UK.

The investigators also found many patients were confused about the trial information they were provided, and many raised concerns about the time and travel requirements associated with trials.

Another issue identified by the report was that many trials rely on contract research organizations and patient organizations to design trials and recruit and manage participants. However, this practice excludes people who do not engage with either type of organization, the authors noted.

Frost and colleagues said their interviews “showed the complexity of information given to patients, the burden of trial participation and the absence of opportunities to engage in trial design further negate some patient voices.”

The voices of clinicians likewise may not be representative of all healthcare providers, the authors said. They noted that clinical staff who were engaged in patient insight reports tended to be those who were more enthusiastic about new therapies.

Frost and colleagues also found that the standardization of the tools used to glean patient insights appear to be designed for efficiency rather than comprehensiveness.

“[A]lthough such tools might optimize the return on the time invested in an interview, they limit the extent to which a patient can raise and discuss the issues that are more important to them, but which fall outside of the remit of the research brief,” they explained.

Frost and colleagues said new requirements are needed to ensure that future trials do a better job of engaging a broader swath of the patient population.

“We propose that methodological rigor (e.g., sampling for equality, diversity and inclusivity) and deliberative practices (e.g., developing drug trial research with rather than on patients) are required to enable a wider group of patients to have a stake in the design of drug trials for IPF and access to new treatments,” they wrote.

However, they said such practices will require a willingness on the part of the pharmaceutical industry and perhaps mandates on the part of regulators.

References

1. Frost J, Mandizha J, Jones S, et al; Widening Engagement Patient Advisory Group. Widening patient engagement for rare disease drug trials: the perspectives of patients with idiopathic pulmonary fibrosis on participating in clinical drug trials and drug trial design. Health Expect. 2025;28(2):e70260. doi:10.1111/hex.70260
2. Shah Gupta R, Koteci A, Morgan A, George PM, Quint JK. Incidence and prevalence of interstitial lung diseases worldwide: a systematic literature review. BMJ Open Respir Res. 2023;10(1):e001291. doi:10.1136/bmjresp-2022-001291