In short
- Interstitial lung disease (ILD) trials are challenging due to disease rarity, older patient populations with comorbidities and polypharmacy, variability in diagnosis, strict trial eligibility criteria, and a highly competitive therapeutic landscape
- With multiple ILD trials enrolling globally, sponsors require effective strategies to ensure timely delivery of new therapies
- Thoughtful site selection – emphasizing strong patient databases, respiratory expertise, and established imaging and lung function capabilities – accelerates enrollment
- Effective recruitment and retention strategies include:
- Structured site engagement through webinars, investigator portals, and enrollment support resources
- Reducing patient burden through travel assistance, and providing clear patient-facing educational materials through collaboration with Patient Advocacy Group liaisons, among others
- Patient-centric trial design, including flexible visit structures and streamlined screening pathways
- Reliable longitudinal data underpins successful ILD trials. Proven experience in lung function assessment (including spirometry) and partnerships with specialized vendors, in addition to rigorous quality controls and training, minimizes variability and maximizes confidence in study outcomes
- These strategies together enable consistent enrollment performance, and improve the likelihood of timely entry to market in a competitive landscape.
The challenge
A large pharmaceutical sponsor selected PSI for a pivotal ILD trial, based on respiratory expertise and experience in complex global studies.
In a highly competitive therapeutic landscape, with multiple concurrent programs, the key question was not to ensure consistent enrollment performance while maintaining data quality and protocol adherence across the trial’s geography.
The factors that make ILD clinical trials uniquely complex
Patient factors
Rare disease population
ILDs are uncommon, resulting in a limited pool of eligible patients across geographiesOlder, comorbid population
Most patients are older adults, often with multiple comorbidities that complicate participation and increase dropout riskHeterogeneous disease severity
Patients range from early, minimally symptomatic disease where patients may feel too well to participate, to advanced stages where participation may be clinically challengingTreatment concerns
Existing therapies may have tolerability limitations, and concerns about treatment changes, placebo exposure, or washout periods can reduce willingness to enroll
Clinical trial factors
Inconsistent diagnostic pathways
Evolving, complex, regional diagnostic definitions and interpretation of imaging can delay the identification of trial-ready patientsVariability in lung function measurement
Lung function testing is sensitive to technique, patient effort, and site experience, creating variability in key endpointsHigh participation burden
Frequent visits, imaging, and assessments can be challenging for older patients with comorbidities, limited mobility, or oxygen dependenceStrict inclusion/exclusion criteria
Requirements around lung function stability, disease classification, and prior therapy use significantly reduce the eligible trial populations and reduce the interest of patients and PIsCompetitive treatment landscape
Across ILDs, approved therapies exist, but with limited efficacy and tolerability in some patients. Evolving treatment standards continue to shape physician and patient preferences, influencing trial participation dynamics
Compared to many therapeutic areas, ILD trials are more difficult to execute on time. The above-mentioned challenges often require expanded site networks, longer recruitment timelines, and increased operational support.
In ILD trials, success is often determined less by the protocol and more by the execution. Sponsors need a CRO with proven respiratory experience, strong relationships with high-performing sites, and the operational flexibility to navigate a fragile patient population. Even well-designed trials can suffer if not partnered with the right CRO.
The solutions
Site selection
Getting site selection right is key to accelerating enrollment. Priority was given to sites with:
- Strong respiratory and ILD patient databases
- Experience in high-quality lung function testing and imaging workflows
- Prior participation in ILD studies
- Minimal competing studies
- Lower SoC use
Recruitment and retention strategy
Two patient pathways were considered for this trial: Newly diagnosed patients who are seeking treatment options, and patients with insufficient response to existing standard therapies. The tailored recruitment and retention strategy included:
Site engagement
- One-to-one calls with investigators, specialist nurses, and PSI in targeted countries, accompanied by a webinar and Q&A campaign
- An investigator portal – accessible only for the duration of the study, this portal contains digital toolkits, sample letters, and other materials to facilitate discussion
- Patient enrollment specialists to support sites by handling administrative tasks (database searches, visit scheduling for ICF completion, pre-screening visits, and travel/logistic needs) and answering patient questions
- Local or regional meetings between the sponsor and site teams, including PIs and study coordinators (and including one-to-one support)
- Web-based townhall meetings with whole site teams
- A patient enrollment boosting toolkit – MMs used this as guidance for their one-to-one calls with PIs and site staff to optimize the conversations around increasing recruitment
Patient engagement
- Travel assistance (e.g., a vendor for travel, and reimbursement for accommodation) to reduce patient/caregiver burden and make the trial more appealing
- Patient portals and study-specific website; patient-facing information materials and interactive educational resources can both improve patient awareness of the trial, but also help them understand the need to adhere to the trial protocol
- Collaboration with PAGs – both nationally and internationally – to increase awareness
- Fully anonymized, semi-structured interviews for patients to provide their feedback on their experiences in the trial – with the opportunity to review their concerns and amend operational elements of the trial accordingly
Lung function data quality
Lung function measures, including spirometry, were central to the study’s endpoints, meaning consistency and reproducibility were essential. Key operational strategies to reduce variability and produce high-confidence endpoint data included:
- Selecting sites with proven experience in lung function testing (for example, those familiar with the relevant ATS/ERS guidelines)
- Standardizing equipment across participating centers
- Ensuring training and retraining for site personnel
- Close collaboration with specialized vendors to support site qualification and performance monitoring – thus reducing deviations or screen failures
The outcome
This late-stage ILD trial continues to make steady progress.
This is a huge achievement that would not have been possible without all of your hard work, energy, and dedication. From planning every detail during the startup phase to making sure everything ran smoothly at the site, each of you played a crucial role in getting us here. […] This is just the start, and we can’t wait to see us hit even more milestones together.
Key takeaways
Successful ILD trial execution depends on getting the fundamentals right:
- Selecting experienced, high-performing sites
- Ensuring reliable and standardized data collection
- Reducing patient burden wherever possible
- Aligning operational strategy with the realities of the disease population
While conceptually straightforward, trials in ILD require discipline, therapeutic experience, and careful operational design and execution to stay on track in a competitive environment.
Get in touch with PSI to run your ILD trial the right way
Abbreviations
ATS, American Thoracic Society;
ERS, European Respiratory Society;
ICF, Informed Consent Form;
ILD, Interstitial Lung Disease;
PAG, Patient Advocacy Group;
PI, Principal Investigator;
Q&A, Question and Answer;
SoC, Standard of Care.
