肺动脉高压临床试验教育者的演变与影响

The Evolution and Impactof Pulmonary Arterial Redefining success in pulmonary hypertension clinical trials. SARAH THINNESEN, Global Oversight PAH/PH/IPF, Director CTE Project Management, Research& Development Solutions Table of contents IntroductionWhat is PAH?The evolution of CTEs in Pulmonary Arterial Hypertension?PAH clinical trial caseStrategic CTE implementation: how to’sImproving long-term achievements Introduction Within the clinical trial setting, the niche world ofpulmonary arterial hypertension (PAH) requirestailored care. The importance of a multidisciplinary Despite substantial progress made in recent yearsregarding PAH disease awareness, together with definitive guidelines for the diagnosis and treatment of PAH,7there is more work to be done. Giventhe disease’s rare nature, not all cardiologists or pulmonologists have expertise in treating PAH.8Accessto treatment can also be variable across differentcountries given factors such as cost, location andinsurance coverage.9As such, some patients present Clinical trials can face many unique challengesin striving to advance innovative therapies while IQVIA has an established and bespoke PAHfootprint within the clinical trial setting and a deepunderstanding of the white glove approach neededto support this balance. As such, the role of PAH The number of specialist physicians and PAH referralcenters, combined with the low incidence/prevalence ofPAH patients, presents additional real-world obstacles inthe trial setting. The PAH global clinical trial landscape ishighly competitive, with most studies undertaken within What is Pulmonary Arterial A chronic and progressive disease, PAH can bedescribed as high blood pressure in the lungs that detrimentally affects the way in which the heart and lungs work together.1This rare disease affects 5 to 15 Of the PAH patients in clinical trials, most havereceived one or more therapies developed to workon the three established but complex PAH treatmentpathways, namely the nitric oxide (NO), endothelin (ET)and prostacyclin (PGI2) pathways.11However, more people per million,2,3whilst it affects women more thantwo to one.4According to recent estimates, PAH affects PAH is a complex pathophysiological disordercharacterized by the presence of antiproliferative vasculopathy in the pulmonary arterioles, leadingto a combination of endothelial and smooth muscle The evolution of CTEs in PAH The role of CTE has undergone a significanttransformation over the last decade.13, 14Originally introduced to energize trials by acceleratingpatient recruitment, a number of these field-basedprofessionals have become PAH global ambassadors atthe site level, essential in managing complex PAH trialenvironments. PAH CTEs are instrumental in fostering screen and reduced screen failures (SF)), site-specificrecruitment strategies (driving to surpass enrollmenttargets) and educating on retention activities and PAH CTEs can hold scientific discussions regarding thestudy of the investigational product (IP) they support.They often provide supplementary education tostudy site staff on inclusion and exclusion criteria to PAH CTE responsibilities have expanded from basiceducation and recruitment support to dynamicfunctions. These encompass supplementary study PAH clinical trial case: Supporting with education on dosing and potential side Background and challenges: •Barrier identification and resolution• High-touch support with assistance in developing The key challenge was recruiting participants foran IP with a mechanism of action that could yieldcommonly known side effects in this classificationof drug. The complex protocol required education Outcomes: • Screenings:CTEs supported 61% of all subjects • Randomization:CTEs supported 64% of all subjectsrandomized, measuring 105% of the recruitment Role of PAH CTEs: In addition to prescreening, screening andrecruitment, the fully dedicated PAH CTE team • Competitive advantage:Utilized specific diseaseacumen to discuss relevant data and position the • Support for awareness of potential clinical •Education on proficient IP dosing, together with Improving long-term Strategic CTE implementation: In the landscape of PAH trials, companies like IQVIAhave distinguished themselves through an innovativeapproach to integrating PAH CTEs into the process. Deployment of dedicated PAH CTEs represents asignificant evolution in clinical trial management.While their study impact is multifaceted, their Clinical trials are an enormous undertaking involvingsignificant funding and studies that can often takeyears to complete. Despite the noted success of PAHCTEs, some sponsors hesitate to add another resource PAH CTEs are particularly vital in trials where patientpopulations are small and dispersed, and treatment • Solid backgrounds:IQVIA CTEs selected for PAHtrials typically are medical professionals with strong • Immersion in PAH training:Comprehensiveonboarding ensures all CTEs ar