Introduction

Being informed your life expectancy could be cut in half is a harsh reality for those diagnosed with Pulmonary Arterial Hypertension. While significant advances have been made, the REVEAL registry shows just a 60% survival rate over five years for class III patients. These patients are not only confronted with a grave prognosis but also a decline in their quality of life. While there are numerous treatments available, they primarily focus on symptom management without curing the condition. However, there's a glimmer of hope as researchers are actively exploring new therapies that might one day extend life beyond current expectations.

The unique challenges in PAH trials

PAH clinical trials face unique hurdles due to the rarity of the condition, and congested trial landscape with over 50 drugs currently in development, which makes patient recruitment highly competitive. This means sponsors need to form strong partnerships with specialist investigators to stand out and provide trials that appeal to both patients and healthcare professionals.

For patients, these trials present their own set of challenges. Many individuals, already dealing with severe health issues, find the prospect of traveling long distances to participate daunting. It becomes important for sponsors to design trials that ease these burdens as much as possible.

Additionally, trial assessments like the six-minute walk test, though straightforward, can be demanding for those who experience breathlessness. When combined with complex procedures such as Right Heart Catheterization, the entire process can become overwhelming, potentially deterring participation.

Sponsors should effectively communicate the scientific benefits of their research and focus on enlisting patients who stand to gain the most. By fostering open dialogue and offering robust support, both patients and investigators can work together to advance PAH treatment and improve patients’ lives.

Improving the trial experience

To improve trials, aligning procedures with standard of care practices is key to reducing patient burden. For example, if typical care involves a six-minute walk test every six months, trials should adopt this frequency instead of increasing it unnecessarily. Similarly, accepting recently performed RHC as screening / baseline data significantly reduces burden for both patient and site.

IQVIA Biotech’s PAH specialist Clinical Trial Educator team are instrumental in this process, providing on-site support and collaborating with investigators to facilitate trials smoothly. At IQVIA Biotech, our team excels at managing site relationships and aiding investigators, ensuring trials are both efficient and considerate of patient needs.

A common practice in PAH trials is the open-label extension, allowing patients continued access to medication after study completion. This approach helps maintain patient commitment, ensuring they benefit directly from their involvement.

Building strong relationships

The sites involved in PAH trials are highly specialized, with principal investigators who are truly passionate about their work. They have nurtured strong connections with their patients, and sponsors must establish similar bonds with these sites, becoming part of the care framework instead of an external burden.

Sponsors must engage deeply with these centers, demonstrating shared enthusiasm for patient care, which is fundamental to maintaining credibility. Recognizing the relationships between medical staff and patients is often overlooked yet vital for successful collaboration.

IQVIA Biotech prioritizes building close partnerships with these experts to ensure seamless integration into patient care. Consistent protocols, such as the six-minute walk test, are important for reliable results. As new therapies emerge, we remain adaptable, continually refining study designs and criteria to keep at the forefront of PAH research.

How IQVIA Biotech can help

At IQVIA Biotech, our Cardiovascular, Renal and Metabolic (CVRM) team is all about making clinical trials successful. We've got a wealth of PAH specific expertise and work closely with Clinical Trial Educators to build strong relationships with sites and investigators. This collaboration helps us deliver smooth and effective trial experiences, making us leaders in PAH clinical development.

Our team is made up of dedicated project managers and clinical operations professionals well-versed in PAH trial delivery. We understand the fast-paced nature of biotech, and we're here to support our clients every step of the way—from planning the initial study design to navigating regulatory inspections and celebrating new drug approvals. We partner with biotech sponsors to help them successfully journey through the complexities of drug development.