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Navigating Clinical Development Challenges to Advance Idiopathic Pulmonary Fibrosis Treatments

As clinical activity targeting rare respiratory disease grows, explore approaches to set your study up for success.

Patients diagnosed with idiopathic pulmonary fibrosis (IPF) — a progressive, rare lung disease marked by scarring and worsening lung function — often face a poor prognosis. While two approved treatments, pirfenidone and nintedanib, slow disease progression, there currently is no treatment that reverses the effects or offers a cure for IPF.

It’s not for lack of trying. The complexity of diagnosing IPF, understanding its root causes, and translating preclinical findings to clinical success make it a challenging disease for drug development. However, recent advancements and strategic approaches in clinical trials offer hope that additional treatments are on the way.

The road ahead for idiopathic pulmonary fibrosis treatments

The landscape of idiopathic pulmonary fibrosis treatment development is poised to evolve significantly. With the expiration of pirfenidone’s patent exclusivity in 2021, sponsors have renewed their focus on developing new therapies. The emergence of new drugs and treatment strategies has broadened treatment options that are in development and fueled research into urgently needed new therapeutic approaches for IPF. Several clinical trials have progressed to Phase III and, if successful, have the potential to alter the standard of care.

Recent advancements in the understanding of the molecular and genetic underpinnings of IPF are driving the identification of new therapeutic targets. Researchers are exploring antifibrotic agents, immune modulators and regenerative therapies as potential treatments. These approaches aim to address the underlying mechanisms of fibrosis and inflammation, offering the possibility of not just slowing disease progression but potentially reversing it.

The regulatory landscape for IPF treatments is also evolving. Agencies are increasingly recognizing the need for flexible and adaptive trial designs that accommodate the unique challenges of studying rare and progressive diseases like IPF. This includes the use of surrogate endpoints, accelerated approval pathways and real-world evidence to support drug approval. These changes are enabling the expedited development and approval of new IPF therapies, ensuring that patients have access to potentially life-saving treatments, sooner.

Prioritizing the idiopathic pulmonary fibrosis patient experience in clinical trials

Understanding the patient experience is paramount to the success of idiopathic pulmonary fibrosis clinical development. Patients with IPF suffer from symptoms like fatigue and shortness of breath, which make completing routine, low-impact tasks a challenge. Even attending their regular clinic visits can be exhausting and stressful. Common study endpoints, such as forced vital capacity (FVC) and the six-minute walk test, are difficult to capture accurately due to patients’ diminished lung capacity and reluctance or inability to undergo repeated strenuous assessments.

That’s why minimizing patient burden is critical to IPF trial success. Sponsors are able to make participation in trials easier by reducing the frequency and duration of study visits, which can be achieved through the adoption of digital solutions, optimized trial design and home health care services. Prioritizing participant comfort and convenience improves compliance as well as patient recruitment and retention rates, in turn increasing the likelihood that studies are able to be completed on time and on budget.

The emphasis on patient-centric trial designs is becoming increasingly important in IPF research. Incorporating patient input and preferences into the design and execution of clinical trials — from the earliest stages — increases participants’ trust and ultimately strengthens the research. For example, using wearable devices to monitor patients’ activity levels and respiratory function can provide valuable data while reducing the need for frequent clinic visits. Telemedicine and remote monitoring technologies also enable patients to participate in trials from the comfort of their own homes, improving their overall experience and compliance.

Patient advocacy groups and organizations play a critical role in shaping the design and implementation of IPF clinical trials. These groups offer valuable insights into the patient experience and advocate for trials that are aligned with patients’ needs and preferences. Engaging with these organizations early in the trial design process also leads to more patient-centric protocols.

The importance of site selection

Site selection is especially critical to idiopathic pulmonary fibrosis clinical trials, and identifying and partnering with sites experienced in managing IPF patients is essential. These sites have access to existing patient pools and understand the specific needs and tolerances of IPF patients. It’s also important to have knowledge of sites in different geographic regions where opportunities exist to recruit patients that may not have access to currently approved medications.

Training and partnering with sites in emerging regions is one strategy to further expand the geographic reach of studies, ensuring high-quality data production and extending trial capabilities. Establishing and fostering strong relationships with these sites is crucial for accessing the existing patient pool and advancing trials in a competitive environment.

With that said, effective site selection involves more than just identifying experienced sites. It also requires a thorough assessment of each site’s infrastructure, capabilities and patient population. Sites with specialized expertise in respiratory diseases, access to advanced diagnostic tools enabling early disease diagnosis, and a history of successful IPF trials are more likely to produce reliable and high-quality data. Additionally, sites located in regions with diverse patient populations can help to ensure that trial results are generalizable to a broader patient population.

Once sites are selected, implementing regular communication and feedback mechanisms enables any challenges or issues that arise during the trial to be addressed quickly to keep the study on track. Building strong relationships with site investigators and staff — rooted in proactive and timely communication — promotes a shared sense of ownership and commitment to the trial, leading to better patient recruitment and retention.

Tapping a CRO for IPF trials

As the idiopathic pulmonary fibrosis landscape evolves, sponsors can advance their studies by partnering with an experienced contract research organization (CRO) that offers extensive indication expertise, innovative trial designs and a history of successful regulatory submissions.

When selecting a CRO partner for your IPF trial, you want to ensure that they possess the capabilities you need to succeed. Look for a CRO that offers:

  1. Expert site selection and management: The right CRO will help identify experienced investigators and sites that have the capacity to manage IPF patients effectively. They ensure that sites are well-trained and capable of producing the high-quality data that is crucial for trial success.
  2. Operational expertise: Managing complex endpoints like spirometry and the six-minute walk test requires operational expertise to ensure accuracy and consistency. Your CRO partner should provide detailed training and close monitoring to address inter-site and inter-patient variability, ensuring consistent, high-quality data collection.
  3. Patient-centric approaches: A CRO can support your efforts to emphasize patient-centricity by minimizing the trial burden through the integrations of digital solutions and home health care services that improve the overall patient experience.
  4. Regulatory support: Experienced CROs have a history of successful regulatory interactions, including novel trial designs and developing agency briefing books. A trusted CRO guides sponsors through the regulatory landscape, ensuring that therapies in development meet all necessary requirements.
  5. Clinical science liaison (CSL): At the PPD™ clinical research business of Thermo Fisher Scientific, the CSL represents sponsors, interacting with sites from feasibility through recruitment. They support direct discussions around trial conduct to expedite trial delivery and ensure sites remain focused on the study at hand. Sponsor visibility and interactivity with sites can be key to success, a CSL can take on that role for your organization.
  6. Data management and analysis: Access to sophisticated data management and analysis capabilities through a CRO ensures that trial data is collected, stored and analyzed accurately and efficiently. This is critical for identifying trends, making informed decisions and, ultimately, demonstrating the efficacy and safety of new IPF treatments.
  7. Global reach and local expertise: For IPF trials, it’s important to choose a CRO with both a global presence and local expertise, which enables more seamless navigation of diverse regulatory environments and cultural considerations. Global reach is essential for conducting multi-center trials and ensuring that new treatments are effectively evaluated across diverse patient populations.

The right partner for your IPF trial

For drug developers at biotech and biopharma organizations, considering all these elements and leveraging the strengths of an experienced provider of CRO solutions can make a substantial difference in the success of clinical trials and the development of effective idiopathic pulmonary fibrosis therapies.

Thermo Fisher Scientific’s PPD clinical research business has extensive experience spanning many respiratory diseases, including IPF. Over the past five years, we’ve partnered on:

  • 27 IPF studies
  • Across 883 sites
  • Enrolling more than 3,000 participants

By leveraging our expertise, you are better positioned to advance your IPF clinical trials. Our focus on patient-centric approaches, strategic site selection and expert partnerships enables us to collaborate with drug developers to make the path toward better treatments for IPF clearer and more achievable.

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